1
00:00:05,301 --> 00:00:07,303
HOW TO ADVANCE INNOVATIVE LUNG 

2
00:00:07,303 --> 00:00:09,238
AND SLEEP THERAPIES FOR PATIENTS

3
00:00:09,238 --> 00:00:11,040
WITH HIGH UNMET NEEDS.

4
00:00:11,040 --> 00:00:18,848
MY NAME IS MARRAH 

5
00:00:18,848 --> 00:00:19,449
LACHOWICZ-SCROGGINS FROM THE 

6
00:00:19,449 --> 00:00:22,352
PEDIATRIC DISEASES BRANCH AND 

7
00:00:22,352 --> 00:00:23,619
CO-ORGANIZER OF THE WORKSHOP AND

8
00:00:23,619 --> 00:00:25,555
LOOKING FORWARD TO AN EXCITING 

9
00:00:25,555 --> 00:00:25,988
TWO DAYS TOGETHER.

10
00:00:25,988 --> 00:00:27,023
BEFORE WE KICK OFF THE MEETING 

11
00:00:27,023 --> 00:00:30,593
WE'LL HAVE A BRIEF INTRODUCTION 

12
00:00:30,593 --> 00:00:33,596
FROM DR. MARISHKA BROWN THE 

13
00:00:33,596 --> 00:00:34,464
DIRECTOR OF THE NATIONAL CENTER 

14
00:00:34,464 --> 00:00:39,502
ON SLEEP DISORDERS RESEARCH.

15
00:00:39,502 --> 00:00:41,104
>> BEFORE THAT I'M GOING
TO GO 

16
00:00:41,104 --> 00:00:43,606
OVER THE HOUSEKEEPING.

17
00:00:43,606 --> 00:00:43,940
GOOD MORNING.

18
00:00:43,940 --> 00:00:46,275
THIS PRESENTATION IS BEING 

19
00:00:46,275 --> 00:00:46,542
RECORDED.

20
00:00:46,542 --> 00:00:48,644
PLEASE MAINTAIN RESEARCH 

21
00:00:48,644 --> 00:00:50,413
INTEGRITY AND CONFIDENTIALITY.

22
00:00:50,413 --> 00:00:51,748
DISCUSSION WITH OTHER WORKSHOP 

23
00:00:51,748 --> 00:00:53,383
PATIENTS AND ATTENDEES.

24
00:00:53,383 --> 00:00:57,453
THE TOOL WILL BE USED FOR OPEN 

25
00:00:57,453 --> 00:00:58,588
DISCUSSION BETWEEN VIRTUAL 

26
00:00:58,588 --> 00:00:59,822
PARTICIPANTS AND COMMENTS 

27
00:00:59,822 --> 00:01:01,758
CAPTURED BY THE WORKSHOP 

28
00:01:01,758 --> 00:01:02,892
SPONSORS AND USE Q&A AND THE

29
00:01:02,892 --> 00:01:04,994
NAME OF THE PERSON YOU'RE 

30
00:01:04,994 --> 00:01:06,863
DIRECTING YOUR QUESTION TO.

31
00:01:06,863 --> 00:01:08,965
THIS COULD BE USED TO ASK 

32
00:01:08,965 --> 00:01:09,966
SPEAKERS A QUESTION AND THEY'LL 

33
00:01:09,966 --> 00:01:13,236
BE CAPTURED FOR REVIEW.

34
00:01:13,236 --> 00:01:15,037
KINDLY STAY ON MUTE AND OFF 

35
00:01:15,037 --> 00:01:15,271
CAMERA.

36
00:01:15,271 --> 00:01:25,448
THANK YOU.

37
00:01:29,519 --> 00:01:30,953
>> THANK YOU FOR THE 

38
00:01:30,953 --> 00:01:31,287
INTRODUCTION.

39
00:01:31,287 --> 00:01:33,856
AS MENTIONED MY NAME IS MARISHKA

40
00:01:33,856 --> 00:01:34,690
BROWN THE DIRECTOR OF THE 

41
00:01:34,690 --> 00:01:37,760
NATIONAL CENTER ON SLEEP STOREDS

42
00:01:37,760 --> 00:01:42,465
RESEARCH HOSTED WITHIN THE 

43
00:01:42,465 --> 00:01:43,099
NATIONAL HEART, LUNG AND BLOOD 

44
00:01:43,099 --> 00:01:47,036
INSTITUTE WELCOME TO HOW TO 

45
00:01:47,036 --> 00:01:48,471
ADVANCE INNOVATIVE LUNG AND 

46
00:01:48,471 --> 00:01:49,772
SLEEP THERAPIES TO PATIENTS WITH

47
00:01:49,772 --> 00:01:50,773
HIGH UNMET NEEDS.

48
00:01:50,773 --> 00:01:53,276
WE HOPE TO FACILITATE AN OPEN 

49
00:01:53,276 --> 00:01:56,412
DIALOGUE WITH THE PULMONARY 

50
00:01:56,412 --> 00:01:57,947
SLEEP COMMUNITIES AND CIRCADIAN 

51
00:01:57,947 --> 00:01:59,515
COMMUNITIES WITH THE GOAL OF 

52
00:01:59,515 --> 00:02:01,517
ADVANCING IDEAS AND STRATEGIES 

53
00:02:01,517 --> 00:02:03,586
TO MOVE THERAPIES FROM THE BENCH

54
00:02:03,586 --> 00:02:05,188
TO CLINIC WHERE INVESTIGATIONAL 

55
00:02:05,188 --> 00:02:07,824
NEW DRUG OR DEVICES THAT REQUIRE

56
00:02:07,824 --> 00:02:08,624
REGULATORY APPROVALS.

57
00:02:08,624 --> 00:02:10,760
THE SPEAKERS ON TODAY'S AGENDA 

58
00:02:10,760 --> 00:02:13,162
WILL PROVIDE UPDATE ON 

59
00:02:13,162 --> 00:02:15,031
REGULATORY PATHWAYS INCLUDING 

60
00:02:15,031 --> 00:02:17,266
DESIGNATIONS SUCH AS FAST TRACK 

61
00:02:17,266 --> 00:02:18,201
AND BREAKTHROUGH THERAPY THAT 

62
00:02:18,201 --> 00:02:21,304
COULD EXPEDITE DEVELOPMENT FOR 

63
00:02:21,304 --> 00:02:24,006
INTERVENTIONS IN AREAS OF UNMET 

64
00:02:24,006 --> 00:02:24,207
NEED.

65
00:02:24,207 --> 00:02:25,808
THIS INCLUDES HOW TO LEVERAGE 

66
00:02:25,808 --> 00:02:26,876
DISCUSSIONS WITH REGULATORY 

67
00:02:26,876 --> 00:02:29,912
BODIES AND FEEDBACK THAT GUIDES 

68
00:02:29,912 --> 00:02:31,814
THE DEVELOPMENT PROCESS.

69
00:02:31,814 --> 00:02:34,250
SPEAKERS WILL EMPHASIZE THE NEED

70
00:02:34,250 --> 00:02:35,751
TO FIND YOUR MARKET AND 

71
00:02:35,751 --> 00:02:37,153
HIGHLIGHT UNIQUE PRODUCTS IN 

72
00:02:37,153 --> 00:02:38,821
COMPARISON TO UNIQUE THERAPIES 

73
00:02:38,821 --> 00:02:41,691
IN PULMONARY AND SLEEP MEDICINE 

74
00:02:41,691 --> 00:02:43,659
AND NOVEL TECHNOLOGIES, IMPROVED

75
00:02:43,659 --> 00:02:46,162
SAFETY PROFILES OR ENHANCED 

76
00:02:46,162 --> 00:02:46,662
EFFICACY.

77
00:02:46,662 --> 00:02:49,465
WE'LL ALSO HEAR ABOUT WAYS TO 

78
00:02:49,465 --> 00:02:51,033
MOVE IDEAS FROM THE LABORATORY 

79
00:02:51,033 --> 00:02:52,034
THROUGH THE COMMERCIALIZATION 

80
00:02:52,034 --> 00:02:53,536
PROCESS AS DEVELOPING NEW 

81
00:02:53,536 --> 00:02:56,239
TREATMENTS REQUIRES SIGNIFICANT 

82
00:02:56,239 --> 00:02:58,074
RESOURCES INCLUDING TIME, 

83
00:02:58,074 --> 00:03:00,076
FINANCIAL INVESTMENTS AND TEAMS 

84
00:03:00,076 --> 00:03:01,110
OF RESEARCHERS AND EXPERTS TO 

85
00:03:01,110 --> 00:03:04,614
TRANSPORT A PRODUCT TO MARKET.

86
00:03:04,614 --> 00:03:06,916
WE THANK YOU ALL FOR ATTENDING 

87
00:03:06,916 --> 00:03:07,850
TODAY.

88
00:03:07,850 --> 00:03:09,485
AND WE LOOK FORWARD TO THE 

89
00:03:09,485 --> 00:03:12,421
DISCUSSION OVER THE NEXT TWO 

90
00:03:12,421 --> 00:03:14,524
DAYS AS WE DIG THROUGH AND HOPE 

91
00:03:14,524 --> 00:03:17,894
NOT ONLY THAT YOU PARTICIPATE IN

92
00:03:17,894 --> 00:03:19,929
THE WORKSHOP BUT THAT YOU 

93
00:03:19,929 --> 00:03:22,098
ACTIVELY ENGAGE IN TOMORROW'S 

94
00:03:22,098 --> 00:03:23,032
BREAKOUT SESSIONS TO DISCUSS 

95
00:03:23,032 --> 00:03:25,167
BARRIERS AND FACILITATORS TO 

96
00:03:25,167 --> 00:03:28,371
TRANSLATING SCIENTIFIC FINDINGS 

97
00:03:28,371 --> 00:03:31,274
AND PULMONARY SLEEP MEDICINE AND

98
00:03:31,274 --> 00:03:32,942
CIRCADIAN BIOLOGY INTO MEDICAL 

99
00:03:32,942 --> 00:03:34,677
INTERVENTIONS TO HELP PATIENTS 

100
00:03:34,677 --> 00:03:38,014
WHO NEED IT THE MOST.

101
00:03:38,014 --> 00:03:38,481
BE

102
00:03:38,481 --> 00:03:39,715
AGAIN, WE WOULD LIKE TO THANK 

103
00:03:39,715 --> 00:03:42,151
EACH OF YOU FOR YOUR ATTENDANCE 

104
00:03:42,151 --> 00:03:43,019
AND ACTIVE PARTICIPATION AND 

105
00:03:43,019 --> 00:03:51,294
I'LL TURN IT BACK TO THE 

106
00:03:51,294 --> 00:03:57,366
ORGANIZERS AND MARRAH AND OTHERS

107
00:03:57,366 --> 00:03:59,068
JOHN SHER I DAN.

108
00:03:59,068 --> 00:04:01,604
>> I WANT TO CONTINUE TO 

109
00:04:01,604 --> 00:04:03,039
INTRODUCE THE OTHER CO-ORGANIZER

110
00:04:03,039 --> 00:04:05,041
OF THE WORKSHOP.

111
00:04:05,041 --> 00:04:07,577
AS DR. BROWN NOTED, MY 

112
00:04:07,577 --> 00:04:10,846
COLLEAGUE, DR. JOHN SHERIDAN OF 

113
00:04:10,846 --> 00:04:13,749
THE RESTRICTIVE AND VASCULAR 

114
00:04:13,749 --> 00:04:14,350
DISEASES BRANCH WILL BE 

115
00:04:14,350 --> 00:04:17,353
CO-LEADING THIS MEETING.

116
00:04:17,353 --> 00:04:19,021
WE ALSO HAVE TWO WONDERFUL 

117
00:04:19,021 --> 00:04:20,289
WORKSHOP CO-CHAIRS YOU'LL HEAR 

118
00:04:20,289 --> 00:04:25,761
FROM THROUGHOUT THE WORKSHOP.

119
00:04:25,761 --> 00:04:28,230
FIRST IS DR. KATELYN KRIVCHENIA 

120
00:04:28,230 --> 00:04:30,199
OF THE INTERSTITIAL AND RARE 

121
00:04:30,199 --> 00:04:32,768
LUNG DEED PROGRAM AND DIRECTOR 

122
00:04:32,768 --> 00:04:36,806
OF THE INFANT SYSTEM FIBROSIS 

123
00:04:36,806 --> 00:04:38,307
PROGRAM AT NATIONWIDE CHILDREN'S

124
00:04:38,307 --> 00:04:38,574
HOSPITAL.

125
00:04:38,574 --> 00:04:40,876
SHE CARES FOR INFANTS AND YOUNG 

126
00:04:40,876 --> 00:04:41,978
ADULTS WITH A WIDE SPECTRUM OF 

127
00:04:41,978 --> 00:04:43,879
LUNG DISEASES.

128
00:04:43,879 --> 00:04:47,650
OUR OTHER CO-CHAIR IS DR. JEFF 

129
00:04:47,650 --> 00:04:49,185
MOFFIT FROM CARBON BIO SCIENCES 

130
00:04:49,185 --> 00:04:54,790
AND ALSO HAS OVER 15 YEARS OF 

131
00:04:54,790 --> 00:04:57,059
PHARMACEUTICAL EXPERIENCE AND IS

132
00:04:57,059 --> 00:05:01,597
A CERTIFIED TOX KOL FWIFT.

133
00:05:01,597 --> 00:05:03,065
AND ALSO HAS EXPERIENCE ON 

134
00:05:03,065 --> 00:05:07,236
SEVERAL AREAS MAINLY FOCUSES ON 

135
00:05:07,236 --> 00:05:13,376
RARE DISEASES.

136
00:05:13,376 --> 00:05:16,045
THE PURPOSE IS TO EDUCATE THE 

137
00:05:16,045 --> 00:05:17,179
RESEARCH COMMUNITY HOW TO BRING 

138
00:05:17,179 --> 00:05:18,180
THEIR IDEAS FROM THE BENCH TO 

139
00:05:18,180 --> 00:05:19,015
THE CLINIC.

140
00:05:19,015 --> 00:05:21,550
WE ALSO WANT TO DISCOVERY 

141
00:05:21,550 --> 00:05:23,953
BARRIERS AND FACILITATORS FOR 

142
00:05:23,953 --> 00:05:25,688
INNOVATION IN DEVELOPING NOVEL 

143
00:05:25,688 --> 00:05:26,889
THERAPIES FOR LUNG AND SLEEP 

144
00:05:26,889 --> 00:05:28,958
CONDITIONS WITH HIGH DISEASE 

145
00:05:28,958 --> 00:05:29,191
BURDEN.

146
00:05:29,191 --> 00:05:31,560
WE KNOW THERE'S A MISALIGNMENT 

147
00:05:31,560 --> 00:05:34,830
BETWEEN DISEASE BURDEN AND NOVEL

148
00:05:34,830 --> 00:05:37,533
THERAPY INNOVATION AND SLOW 

149
00:05:37,533 --> 00:05:40,069
SPEED IN WHICH THEY'RE MAKING IT

150
00:05:40,069 --> 00:05:43,005
TO MARKET AND MAY CONTRIBUTE TO 

151
00:05:43,005 --> 00:05:45,975
INCREASED MORBIDITY AND 

152
00:05:45,975 --> 00:05:49,111
MORTALITY AND EXACERBATE HEALTH 

153
00:05:49,111 --> 00:05:49,912
DISPARITIES AND CLOSING THESE 

154
00:05:49,912 --> 00:05:51,380
GAPS IN THE LUNG AND SLEEP 

155
00:05:51,380 --> 00:05:52,848
MEDICINE COMMUNITY NEEDS TO 

156
00:05:52,848 --> 00:05:54,216
HAPPEN THROUGH RESEARCH 

157
00:05:54,216 --> 00:05:55,685
COLLABORATIONS IN ORDER TO HELP 

158
00:05:55,685 --> 00:05:58,921
REDUCE THESE HEALTH DISPARITIES 

159
00:05:58,921 --> 00:06:00,289
AS WELL AS IMPROVE HEALTH 

160
00:06:00,289 --> 00:06:02,391
OUTCOMES AND LOWER SOCIETAL 

161
00:06:02,391 --> 00:06:03,059
COSTS.

162
00:06:03,059 --> 00:06:05,061
WE ALSO ALREADY RECOGNIZE THAT 

163
00:06:05,061 --> 00:06:06,829
THERE ARE SEVERAL CHALLENGES WE 

164
00:06:06,829 --> 00:06:09,331
HOPE PARTICIPANTS CAN WEIGH IN 

165
00:06:09,331 --> 00:06:12,468
ON DURING THE BREAKOUTS TOMORROW

166
00:06:12,468 --> 00:06:13,703
INCLUDING RECOMMENDS STRATEGIES 

167
00:06:13,703 --> 00:06:15,905
TO THE LUNG AND SLEEP RESEARCH 

168
00:06:15,905 --> 00:06:17,606
COMMUNITIES TO SPUR AND 

169
00:06:17,606 --> 00:06:18,674
FACILITATE INNOVATION THAT 

170
00:06:18,674 --> 00:06:19,942
ADDRESSES THE UNMET NEEDS AND 

171
00:06:19,942 --> 00:06:28,284
REDUCES HEALTH DISPAURTS.

172
00:06:28,284 --> 00:06:29,752
SOME CHALLENGES INCLUDE WHAT IS 

173
00:06:29,752 --> 00:06:31,787
THE GREATEST UNMET NEEDS AND 

174
00:06:31,787 --> 00:06:33,889
UNMET NEEDS DUE TO A LACK OF 

175
00:06:33,889 --> 00:06:36,892
ACCESS TO EXISTING THERAPIES.

176
00:06:36,892 --> 00:06:37,927
ALSO, DO WE UNDERSTAND ENOUGH 

177
00:06:37,927 --> 00:06:40,563
ABOUT THE DECISION MAKING 

178
00:06:40,563 --> 00:06:45,668
PROFESSION FOR INVESTMENT BY FOR

179
00:06:45,668 --> 00:06:47,403
PROFIT AND PRIVATE SECTOR 

180
00:06:47,403 --> 00:06:48,504
DEVELOPERS AND THEIR POTENTIAL 

181
00:06:48,504 --> 00:06:51,774
RISK AND RETURNED?

182
00:06:51,774 --> 00:06:53,142
WHERE DOES THE DRUG INVESTMENT 

183
00:06:53,142 --> 00:06:54,610
PIPELINE COME IN TO HELP SUPPORT

184
00:06:54,610 --> 00:06:58,214
UNMET NEEDS WHERE STANDARD OF 

185
00:06:58,214 --> 00:06:59,014
CARE IS INADEQUATE FOR WHERE 

186
00:06:59,014 --> 00:07:00,316
OUTCOMES CAN BE POTENTIALLY 

187
00:07:00,316 --> 00:07:01,617
IMPROVED BY INNOVATIVE 

188
00:07:01,617 --> 00:07:02,151
THERAPIES.

189
00:07:02,151 --> 00:07:03,052
FINALLY, THERE ARE STRATEGIES IN

190
00:07:03,052 --> 00:07:05,020
THE COMMUNITY AND WE NEED TO 

191
00:07:05,020 --> 00:07:06,756
THINK ABOUT THESE TOGETHER IN 

192
00:07:06,756 --> 00:07:08,190
ORDER TO FACILITATE GREATER 

193
00:07:08,190 --> 00:07:09,959
INVESTMENT IN INNOVATION TO 

194
00:07:09,959 --> 00:07:13,162
CONTINUE TO ADDRESS THESE UNMET 

195
00:07:13,162 --> 00:07:14,764
NEEDS AS WELL AS TO DISCOURAGE 

196
00:07:14,764 --> 00:07:15,231
PRACTICES THAT IMPEDE 

197
00:07:15,231 --> 00:07:25,441
INNOVATION.

198
00:07:25,908 --> 00:07:28,277
WE'RE HOPING TO DEMYSTIFY THE 

199
00:07:28,277 --> 00:07:29,211
PROCESS FROM WHICH YOU'RE 

200
00:07:29,211 --> 00:07:31,046
GETTING YOUR IDEAS THROUGH THE 

201
00:07:31,046 --> 00:07:32,414
REGULATORY AND COMMERCIALIZATION

202
00:07:32,414 --> 00:07:35,484
PIPELINES AND THE FOCUS OF THIS 

203
00:07:35,484 --> 00:07:36,519
WORKSHOP WILL BE THE IND 

204
00:07:36,519 --> 00:07:38,154
ENABLING PATHWAY WHICH IS THE 

205
00:07:38,154 --> 00:07:40,356
PATHWAY WHICH YOU'LL TAKE YOUR 

206
00:07:40,356 --> 00:07:42,091
IDEAS THROUGH RELEVANT MODEL 

207
00:07:42,091 --> 00:07:45,995
SYSTEMS INCLUDING PKPD AND 

208
00:07:45,995 --> 00:07:48,130
TOXICOLOGY STUDIES, THINKING 

209
00:07:48,130 --> 00:07:49,665
ABOUT HOW YOU'LL ACCESS CAPITAL 

210
00:07:49,665 --> 00:07:57,072
TO MOVE TO MANUFACTURING AND 

211
00:07:57,072 --> 00:07:58,073
PRODUCTION TO ENTER CLINICAL 

212
00:07:58,073 --> 00:07:58,507
TRIALS.

213
00:07:58,507 --> 00:08:00,976
YOUR IND PACKAGE WILL REQUIRE 

214
00:08:00,976 --> 00:08:03,679
CLINICAL PROTOCOLS FOR THE 

215
00:08:03,679 --> 00:08:04,346
TRIALS.

216
00:08:04,346 --> 00:08:05,981
AT THAT POINT YOU'LL NEED TO 

217
00:08:05,981 --> 00:08:07,116
PARTNER WITH PATIENTS AND PEERS 

218
00:08:07,116 --> 00:08:08,517
IN THE COMMUNITY WHO MAY HAVE 

219
00:08:08,517 --> 00:08:10,019
BEEN SUCCESSFUL IN THIS PROCESS.

220
00:08:10,019 --> 00:08:11,086
YOU'LL CONTINUE TO SEE THESE 

221
00:08:11,086 --> 00:08:20,462
THEMES THROUGHOUT THE WORKSHOP .

222
00:08:20,462 --> 00:08:21,697
AS DR. BROWN MENTIONED IN THE 

223
00:08:21,697 --> 00:08:24,266
AGENDA WE'LL PROVIDE AN UPDATE 

224
00:08:24,266 --> 00:08:28,737
TO PATHWAYS AND DESIGNATION MAY 

225
00:08:28,737 --> 00:08:30,339
HELP EXPEDITE INTERVENTIONS IN 

226
00:08:30,339 --> 00:08:32,608
AREAS OF UNMET NEEDS.

227
00:08:32,608 --> 00:08:36,579
THIS WILL CONTINUE TO HELP YOU 

228
00:08:36,579 --> 00:08:37,980
LEVERAGE YOUR DISCUSSIONS WITH 

229
00:08:37,980 --> 00:08:39,615
REGULATORY BODIES AND USE THE 

230
00:08:39,615 --> 00:08:41,050
FEEDBACK THEY GIVE YOU FOR 

231
00:08:41,050 --> 00:08:42,551
DEVELOPING YOUR INNOVATIONS.

232
00:08:42,551 --> 00:08:47,189
WE WILL HEAR FROM SPEAKERS WHO 

233
00:08:47,189 --> 00:08:48,524
WILL EMPHASIZE MARKET AS 

234
00:08:48,524 --> 00:08:50,459
DR. BROWN MENTIONED.

235
00:08:50,459 --> 00:08:51,327
THIS MEANS AN INVESTIGATIONAL 

236
00:08:51,327 --> 00:08:54,463
PRODUCT YOU MAY BE WORKING ON 

237
00:08:54,463 --> 00:08:56,699
NEEDS TO BE UNIQUE TO THERAPIES 

238
00:08:56,699 --> 00:08:59,468
IN PULMONARY AND SLEEP MEDICINE 

239
00:08:59,468 --> 00:09:01,537
AND NOVEL TECHNOLOGIES THAT HAVE

240
00:09:01,537 --> 00:09:03,105
IMPROVED SAFETY PROFILES OR 

241
00:09:03,105 --> 00:09:03,973
ENHANCED EFFICACY.

242
00:09:03,973 --> 00:09:05,741
YOU ALSO NEED TO THINK ABOUT HOW

243
00:09:05,741 --> 00:09:08,744
TO MOVE YOUR IDEAS FROM THE 

244
00:09:08,744 --> 00:09:10,880
BENCH THROUGH THE 

245
00:09:10,880 --> 00:09:13,549
COMMERCIALIZATION PROCESS.

246
00:09:13,549 --> 00:09:16,652
DEVELOPING YOUR TREATMENT WILL 

247
00:09:16,652 --> 00:09:17,987
REQUIRE SIGNIFICANT INVESTMENT 

248
00:09:17,987 --> 00:09:19,688
AND IF THE MARKET SEEMS LIMITED 

249
00:09:19,688 --> 00:09:21,657
MAY DETER INVESTMENT FROM 

250
00:09:21,657 --> 00:09:23,058
POTENTIAL PARTNERS.

251
00:09:23,058 --> 00:09:23,859
TOMORROW WE'LL CONTINUE TO 

252
00:09:23,859 --> 00:09:26,495
ENGAGE STAKEHOLDERS AND RELEVANT

253
00:09:26,495 --> 00:09:33,469
FEDERAL AGENCIES AND ACADEMICS 

254
00:09:33,469 --> 00:09:34,904
AND CLINICIANS AND PATIENT 

255
00:09:34,904 --> 00:09:37,606
GROUPS AND INDUSTRY TO BUILD 

256
00:09:37,606 --> 00:09:38,540
CAPACITY AND INNOVATION IN 

257
00:09:38,540 --> 00:09:39,975
THERAPEUTIC DEVELOPMENT AND 

258
00:09:39,975 --> 00:09:43,012
ENSURING BROAD EQUITABLE ACCESS 

259
00:09:43,012 --> 00:09:44,280
FOR SAFE AND EFFECTIVE NOVEL 

260
00:09:44,280 --> 00:09:44,680
THERAPIES.

261
00:09:44,680 --> 00:09:45,981
WE WANT PARTICIPANTS TOE NOT 

262
00:09:45,981 --> 00:09:47,650
ONLY LEARN FROM THIS WORKSHOP 

263
00:09:47,650 --> 00:09:49,451
BUT TO NETWORK WITH EACH OTHER.

264
00:09:49,451 --> 00:09:51,587
AS WE MENTIONED EARLIER, WE DO 

265
00:09:51,587 --> 00:09:53,022
HAVE THE CHAT OPEN FOR YOU TO 

266
00:09:53,022 --> 00:09:55,057
CONNECT TO EACH OTHER DURING THE

267
00:09:55,057 --> 00:09:55,758
WORKSHOP.

268
00:09:55,758 --> 00:09:58,060
ADDITIONALLY, WE WILL HAVE THE 

269
00:09:58,060 --> 00:09:59,795
ZOOM ROOM OPEN FOR TWO 

270
00:09:59,795 --> 00:10:02,197
NETWORKING BREAKS DURING THE 

271
00:10:02,197 --> 00:10:02,431
MEETING.

272
00:10:02,431 --> 00:10:04,099
AND FINALLY AS I SAID EARLIER, 

273
00:10:04,099 --> 00:10:05,134
WE HOPE YOU'LL PARTICIPATE IN 

274
00:10:05,134 --> 00:10:10,339
THE BREAKOUT SESSIONS ON DAY 2 

275
00:10:10,339 --> 00:10:11,640
TO HELP US DETERMINE THE 

276
00:10:11,640 --> 00:10:16,078
BARRIERS AND FACILITATORS TO AID

277
00:10:16,078 --> 00:10:17,146
IN FINDINGS AND CIRCADIAN 

278
00:10:17,146 --> 00:10:18,981
BIOLOGY INTO MEDICAL 

279
00:10:18,981 --> 00:10:20,015
INTERVENTIONS FOR PATIENTS WHO 

280
00:10:20,015 --> 00:10:29,525
NEED THEM MOST.

281
00:10:29,525 --> 00:10:34,630
I'D LIKE TO TURN IT OVER TO OUR 

282
00:10:34,630 --> 00:10:41,303
MEETING CO-CHAIR, DR. MOFFITT.

283
00:10:41,303 --> 00:10:43,072
>> TODAY I WANT TO DISCUSS
THE 

284
00:10:43,072 --> 00:10:45,040
EARLY CONCEPTS YOU CAN LEVERAGE 

285
00:10:45,040 --> 00:10:46,909
TO IMPROVE THE QUALITY OF 

286
00:10:46,909 --> 00:10:47,576
CLINICAL CANDIDATES EARLY IN THE

287
00:10:47,576 --> 00:10:50,012
RESEARCH AND DEVELOPMENT 

288
00:10:50,012 --> 00:10:58,887
PROCESS.

289
00:10:58,887 --> 00:11:00,122
MANY APPROACHES CAN GO TO 

290
00:11:00,122 --> 00:11:02,024
TRANSLATIONAL ASPECTS OF EARLY 

291
00:11:02,024 --> 00:11:04,760
DRUG DEVELOPMENT.

292
00:11:04,760 --> 00:11:08,030
OBVIOUSLY PATIENT SAFETY 

293
00:11:08,030 --> 00:11:09,865
CONSIDERATIONS ALWAYS COME FIRST

294
00:11:09,865 --> 00:11:12,968
AND IT'S NEVER TOO EARLY TO 

295
00:11:12,968 --> 00:11:16,205
START TO THINK ABOUT SAFETY 

296
00:11:16,205 --> 00:11:19,108
PROFILES FOR EARLY POTENTIAL 

297
00:11:19,108 --> 00:11:20,142
DRUGS IN THE PROCESS.

298
00:11:20,142 --> 00:11:26,048
IF YOU WERE TO OPEN A BOOK YOU'D

299
00:11:26,048 --> 00:11:32,054
SEE TARGET ORGANS AND TEXAS  

300
00:11:32,054 --> 00:11:36,158
TOXICITY AND THE RANGE OF DOSES 

301
00:11:36,158 --> 00:11:38,327
AND WE'RE TRYING TO FEED THIS TO

302
00:11:38,327 --> 00:11:39,762
CLINICAL COLLEAGUES ON WHAT TO 

303
00:11:39,762 --> 00:11:45,034
LOOK OUT FOR IN PATIENTS.

304
00:11:45,034 --> 00:11:46,735
SPECIFICALLY A PRESUMED 

305
00:11:46,735 --> 00:11:47,603
EFFICACIOUS DOSE AND AT THE 

306
00:11:47,603 --> 00:11:49,605
SAFETY MARGIN LOOKS LIKE TO 

307
00:11:49,605 --> 00:11:51,540
WHERE ADVERSE EFFECTS MAY START 

308
00:11:51,540 --> 00:11:52,708
CREEPING IN.

309
00:11:52,708 --> 00:11:56,045
OF COURSE WE INCLUDE SOME SAFETY

310
00:11:56,045 --> 00:11:58,714
MARGINS JUST TO ADDRESS 

311
00:11:58,714 --> 00:12:00,649
UNCERTAINTY AS YOU'LL HEAR ABOUT

312
00:12:00,649 --> 00:12:01,116
TODAY.

313
00:12:01,116 --> 00:12:02,518
AND WHAT I HOPE TO SHOW YOU OVER

314
00:12:02,518 --> 00:12:04,853
THE SLIDES ARE THE CONCEPTS YOU 

315
00:12:04,853 --> 00:12:07,056
CAN LEVERAGE EARLY ON TO BE 

316
00:12:07,056 --> 00:12:09,792
TRANSPARENT WITH YOUR PATIENTS, 

317
00:12:09,792 --> 00:12:11,460
YOUR CLINICAL COLLEAGUES AND 

318
00:12:11,460 --> 00:12:13,262
REGULATORS TO SHOW THEM HOW YOU 

319
00:12:13,262 --> 00:12:15,397
ARRIVED AT THE RECOMMENDATIONS.

320
00:12:15,397 --> 00:12:18,934
BUT I HAVE TO SAY THE LONGER I'M

321
00:12:18,934 --> 00:12:20,702
IN THE BUSINESS THE LESS I 

322
00:12:20,702 --> 00:12:24,006
BELIEVE DRUG SAFETY IS JUST THIS

323
00:12:24,006 --> 00:12:24,573
TWO-DIMENSIONAL RISK VERSUS 

324
00:12:24,573 --> 00:12:25,908
BENEFIT CON SEFT.

325
00:12:25,908 --> 00:12:27,709
-- CONCEPT.

326
00:12:27,709 --> 00:12:30,212
SO THIS IS REALLY HOW I THINK OF

327
00:12:30,212 --> 00:12:32,781
DRUG SAFETY AND IT'S HIGHLY 

328
00:12:32,781 --> 00:12:34,850
INFLUENCED BY YOUR PERSONAL 

329
00:12:34,850 --> 00:12:36,351
EXPERIENCE, THE NOVELTY AND 

330
00:12:36,351 --> 00:12:37,886
SEVERITY OF THE DRUGS THAT 

331
00:12:37,886 --> 00:12:39,188
YOU'RE GOING AFTER AND CERTAINLY

332
00:12:39,188 --> 00:12:40,322
THE SIZE AND PHILOSOPHY OF YOUR 

333
00:12:40,322 --> 00:12:50,566
ORGANIZATION.

334
00:12:50,566 --> 00:12:52,167
WHAT SOME CONSIDER TO BE RISK 

335
00:12:52,167 --> 00:12:55,104
ACCEPTABLE AND IN A FATAL RARE 

336
00:12:55,104 --> 00:12:57,239
DISEASE YOU'LL HAVE A DIFFERENT 

337
00:12:57,239 --> 00:12:58,474
PERSPECTIVE THAN IN SOMETHING 

338
00:12:58,474 --> 00:13:00,709
LIKE JUVENILE ASTHMA WHERE 

339
00:13:00,709 --> 00:13:02,077
THERE'S GOOD SAFE DRUGS OUT 

340
00:13:02,077 --> 00:13:04,847
THERE ALREADY.

341
00:13:04,847 --> 00:13:06,315
SO, THESE ARE SOME OF THE 

342
00:13:06,315 --> 00:13:07,049
CONCEPTS YOU HAVE TO CONSIDER 

343
00:13:07,049 --> 00:13:08,650
MOVING FORWARD.

344
00:13:08,650 --> 00:13:11,286
SO AS MOST OF US AT THE WORKSHOP

345
00:13:11,286 --> 00:13:13,455
ARE FROM ACADEMIC, GOVERNMENT OR

346
00:13:13,455 --> 00:13:16,291
SMALL BIO TECH, WE CAN'T 

347
00:13:16,291 --> 00:13:23,065
POSSIBLY HOPE TO LEVERAGE ALL 

348
00:13:23,065 --> 00:13:28,270
THE RESOURCES AT THE DISPOSAL OF

349
00:13:28,270 --> 00:13:32,074
BIG PHARMA COMPANIES.

350
00:13:32,074 --> 00:13:37,346
THEY'RE INCENTIVED AND THEY HAVE

351
00:13:37,346 --> 00:13:47,990
A WEALTH OF COMPOUNDS PRESUMABLY

352
00:13:48,390 --> 00:13:49,992
OF MOVING FORWARD AND WE DON'T 

353
00:13:49,992 --> 00:13:52,761
HAVE THE LUXURY OF THOSE OPTIONS

354
00:13:52,761 --> 00:13:56,532
AND OFTEN ARE MORE PERSISTENT 

355
00:13:56,532 --> 00:13:59,067
WITH OUR LIMITED ASSETS TO BE 

356
00:13:59,067 --> 00:14:01,270
MORE RISK TOLERANT OFTEN OUT OF 

357
00:14:01,270 --> 00:14:03,438
NECESSITY TAKING TIME TO 

358
00:14:03,438 --> 00:14:04,406
UNDERSTAND THE BARRIERS BETWEEN 

359
00:14:04,406 --> 00:14:07,042
US AND THE CLINIC AND UNDERSTAND

360
00:14:07,042 --> 00:14:10,112
THE STRENGTHS AND WEAKNESSES 

361
00:14:10,112 --> 00:14:10,479
ASSETS.

362
00:14:10,479 --> 00:14:11,613
SPENDING MORE TIME TO DIVE IN 

363
00:14:11,613 --> 00:14:13,515
THE MECHANISM, FINDING NOVEL 

364
00:14:13,515 --> 00:14:16,418
WAYS AND ASSAYS TO REALLY ASSESS

365
00:14:16,418 --> 00:14:19,388
THIS TO DRIVE FORWARD THROUGH 

366
00:14:19,388 --> 00:14:21,223
THE ISSUES.

367
00:14:21,223 --> 00:14:22,424
IN EITHER SITUATION I'M 

368
00:14:22,424 --> 00:14:23,992
CERTAINLY NOT ADVOCATING AROUND 

369
00:14:23,992 --> 00:14:25,194
TAKING AN UNSAFE MEDICINE 

370
00:14:25,194 --> 00:14:27,329
FORWARD BUT I'M JUST REALLY 

371
00:14:27,329 --> 00:14:28,397
MAKING IT CLEAR THAT RISK 

372
00:14:28,397 --> 00:14:30,365
TOLERANCE IS DIFFERENT DEPENDING

373
00:14:30,365 --> 00:14:32,334
ON WHERE YOU STAND IN THE 

374
00:14:32,334 --> 00:14:33,569
PROFESSION AND I WOULD ARGUE 

375
00:14:33,569 --> 00:14:38,073
THAT'S A VERY GOOD AND IMPORTANT

376
00:14:38,073 --> 00:14:39,341
THING WHERE WE BOTH BENEFIT FROM

377
00:14:39,341 --> 00:14:49,151
EACH OTHER.

378
00:14:49,151 --> 00:14:50,018
HERE'S AN OVERVIEW OF THE 

379
00:14:50,018 --> 00:14:52,187
PROCESS FOR THE TYPICAL SMALL 

380
00:14:52,187 --> 00:14:52,854
MOLECULE.

381
00:14:52,854 --> 00:14:54,189
WE'LL FOCUS ON THE DRIVE TO THE 

382
00:14:54,189 --> 00:14:56,258
IND AS MARRAH MENTIONED AND 

383
00:14:56,258 --> 00:14:58,327
THAT'S YOUR INVESTIGATIONAL NEW 

384
00:14:58,327 --> 00:15:01,730
DRUG APPLICATION YOU SUBMIT WITH

385
00:15:01,730 --> 00:15:02,931
THE FDA OR IND.

386
00:15:02,931 --> 00:15:03,999
WE'LL HAVE REGULATORY FOLKS TALK

387
00:15:03,999 --> 00:15:06,001
TO US ABOUT WHAT THE MILESTONES 

388
00:15:06,001 --> 00:15:11,473
MEAN AND WHAT YOU NEED TO GET 

389
00:15:11,473 --> 00:15:11,773
THERE.

390
00:15:11,773 --> 00:15:13,108
ONE OF THE THINGS WE WANT TO 

391
00:15:13,108 --> 00:15:15,043
FOCUS IN ON TOMORROW IS AROUND 

392
00:15:15,043 --> 00:15:17,112
THE CLINICAL DEVELOPMENT AND 

393
00:15:17,112 --> 00:15:18,247
ASSESSMENT STANDPOINT AS YOU 

394
00:15:18,247 --> 00:15:19,648
DRIVE TOWARD AN APPROVAL.

395
00:15:19,648 --> 00:15:22,351
SO MAYBE IF YOU COULD MOVE TO 

396
00:15:22,351 --> 00:15:23,652
THE NEXT SLIDE WE'LL TALK MORE 

397
00:15:23,652 --> 00:15:24,653
SPECIFICALLY AROUND THINGS YOU 

398
00:15:24,653 --> 00:15:27,055
CAN START TO THINK ABOUT FROM A 

399
00:15:27,055 --> 00:15:31,159
DRUG SAFETY STANDPOINT VERY 

400
00:15:31,159 --> 00:15:32,160
EARLY IN THE PROFESSOR.

401
00:15:32,160 --> 00:15:34,329
-- PROCESS.

402
00:15:34,329 --> 00:15:36,365
I WOULD ARGUE THE EARLIER YOU 

403
00:15:36,365 --> 00:15:37,933
CAN THINK OF DRUG SAFETY THE 

404
00:15:37,933 --> 00:15:39,801
BETTER OFF YOU'LL BE.

405
00:15:39,801 --> 00:15:46,475
OFTEN IT WORKS COLLABORATELY -- 

406
00:15:46,475 --> 00:15:48,076
COLLABORATIVELY TO GUIDE THE 

407
00:15:48,076 --> 00:15:49,978
PHARMACOLOGY AND CAN IMPROVE THE

408
00:15:49,978 --> 00:15:51,947
QUALITY OF THE CANDIDATES THAT 

409
00:15:51,947 --> 00:15:55,050
ARE VERY COST EFFECTIVE.

410
00:15:55,050 --> 00:16:01,023
WHILE TOXICOLOGISTS CAN CLEARLY 

411
00:16:01,023 --> 00:16:04,660
DEFINE WINNERS AND LOSERS IN THE

412
00:16:04,660 --> 00:16:07,296
PROCESS WE CAN INDICATE 

413
00:16:07,296 --> 00:16:10,465
COMPOUNDS THAT OFTEN HAVE MORE 

414
00:16:10,465 --> 00:16:11,233
DRUG-LIKE CHARACTERISTICS TO USE

415
00:16:11,233 --> 00:16:13,502
TO THEN PRIORITIZE AND HELP 

416
00:16:13,502 --> 00:16:15,070
STEER THAT RESEARCH PROCESS AS 

417
00:16:15,070 --> 00:16:17,406
THE EFFICACY AND TRANSLATIONAL 

418
00:16:17,406 --> 00:16:18,707
ASPECTS OF YOUR PROGRAM ARE 

419
00:16:18,707 --> 00:16:21,143
EVOLVING IN PARALLEL.

420
00:16:21,143 --> 00:16:23,345
I THINK SOME OF THE IMPORTANT 

421
00:16:23,345 --> 00:16:25,380
THINGS TO THINK ABOUT ARE HOW 

422
00:16:25,380 --> 00:16:29,418
CAN YOU DO THESE AT LOW DOLLAR 

423
00:16:29,418 --> 00:16:31,320
COST OPPORTUNITIES TO REALLY 

424
00:16:31,320 --> 00:16:33,555
DRIVE THOSE FORWARD AND REALLY 

425
00:16:33,555 --> 00:16:35,023
MAKING A WEIGHT OF EVIDENCE 

426
00:16:35,023 --> 00:16:37,359
APPROACH TO IMPROVE THE QUALITY 

427
00:16:37,359 --> 00:16:45,267
OF THE CLINICAL CANDIDATES.

428
00:16:45,267 --> 00:16:49,104
I DO WANT TO TAKE A MOMENT TO 

429
00:16:49,104 --> 00:16:49,838
FRAME WHAT SUCCESS LOOKS LIKE 

430
00:16:49,838 --> 00:16:51,540
EARLY IN DRUG SAFETY.

431
00:16:51,540 --> 00:16:53,408
THERE WAS A GREAT PAPER FROM 

432
00:16:53,408 --> 00:16:56,044
OLSON IN 2000 THAT FOUND 

433
00:16:56,044 --> 00:16:59,081
CONDUCTING THE LONGEST TERM THE 

434
00:16:59,081 --> 00:17:00,782
PIVOTAL TOXICOLOGY STUDY, 26 

435
00:17:00,782 --> 00:17:06,455
WEEK IN A RODENT AND 39 WEEK IN 

436
00:17:06,455 --> 00:17:09,691
A LARGE ANIMAL SPECIES WAS ONLY 

437
00:17:09,691 --> 00:17:13,362
ABLE TO IDENTIFY 71% OF HUMAN 

438
00:17:13,362 --> 00:17:14,029
RELEVANT TOXICITIES WHEN YOU 

439
00:17:14,029 --> 00:17:17,132
CONSIDER THE FINDINGS OF BOTH OF

440
00:17:17,132 --> 00:17:19,434
THOSE PIVOTAL STUDIES.

441
00:17:19,434 --> 00:17:22,371
THIS IS NOT INCLUDING ALL THE 

442
00:17:22,371 --> 00:17:24,506
ADDITIONAL TOXICOLOGY STUDIES 

443
00:17:24,506 --> 00:17:27,175
FOR GENE TOX, SAFETY 

444
00:17:27,175 --> 00:17:29,911
PHARMACOLOGY AND OTHERS GET YOU 

445
00:17:29,911 --> 00:17:33,081
TO 80% OF BEING ABLE TO PICK OUT

446
00:17:33,081 --> 00:17:35,050
HUMAN RELEVANT TOXICITIES.

447
00:17:35,050 --> 00:17:37,419
SO AS YOU CONSIDER THIS IT'S 

448
00:17:37,419 --> 00:17:38,820
ACTUALLY QUITE SOBERING TO 

449
00:17:38,820 --> 00:17:41,923
REMEMBER THAT MEANS 20% OF THE 

450
00:17:41,923 --> 00:17:43,425
TIME HUMANS ARE THE SENSITIVE 

451
00:17:43,425 --> 00:17:43,658
SPECIES.

452
00:17:43,658 --> 00:17:45,427
THIS MEANS NO MATTER WHAT YOU 

453
00:17:45,427 --> 00:17:49,798
DO, WHAT ASSAYS YOU PROFILE, YOU

454
00:17:49,798 --> 00:17:53,902
COULD NOT PREDICT THESE 

455
00:17:53,902 --> 00:17:55,003
TOXICITIES WITH ANY AVAILABLE 

456
00:17:55,003 --> 00:17:55,370
ASSAY.

457
00:17:55,370 --> 00:17:57,038
AS SOBERING AS THAT IS YOU CAN 

458
00:17:57,038 --> 00:17:59,174
THINK OF TURNING THIS AROUND THE

459
00:17:59,174 --> 00:18:00,809
OPPOSITE WAY AROUND WHAT TRYING 

460
00:18:00,809 --> 00:18:02,611
TO GUIDE YOU AND WHAT ASSAYS TO 

461
00:18:02,611 --> 00:18:03,612
RUN AS PART OF THIS.

462
00:18:03,612 --> 00:18:06,214
AT SOME POINT YOU'RE NO LONGER 

463
00:18:06,214 --> 00:18:07,649
GENERATING VALUABLE DATA.

464
00:18:07,649 --> 00:18:10,519
YOU'RE JUST GENERATING MORE DATA

465
00:18:10,519 --> 00:18:12,921
TO ASSESS AND NOT NECESSARILY 

466
00:18:12,921 --> 00:18:14,222
ADDING DECISION MAKING CRITERIA.

467
00:18:14,222 --> 00:18:17,159
IF YOU GO TO ANY OF THESE 

468
00:18:17,159 --> 00:18:19,094
SCIENTIFIC CONFERENCES OR I'M 

469
00:18:19,094 --> 00:18:21,997
SURE YOUR IN BOX BOMBARDED BY 

470
00:18:21,997 --> 00:18:23,165
ASSAY COMPANIES SAYING RUN THESE

471
00:18:23,165 --> 00:18:25,534
ASSAYS TO HELP YOU UNDERSTAND 

472
00:18:25,534 --> 00:18:28,770
THIS, HOWEVER, YOU HAVE TO 

473
00:18:28,770 --> 00:18:30,338
UNDERSTAND AT SOME POINT YOU'RE 

474
00:18:30,338 --> 00:18:31,540
HAVING THIS EFFECT OF 

475
00:18:31,540 --> 00:18:32,307
DIMINISHING RETURNS.

476
00:18:32,307 --> 00:18:35,010
THIS IS ONE OF THE STRENGTHS OF 

477
00:18:35,010 --> 00:18:38,079
OUR NEXT SESSION YOU'LL HEAR 

478
00:18:38,079 --> 00:18:39,614
ABOUT HOW CERTAIN FOLKS HAVE 

479
00:18:39,614 --> 00:18:41,716
DEVELOPED ASSAYS ENRICHING THE 

480
00:18:41,716 --> 00:18:43,084
DECISION MAKING CRITERIA THAT 

481
00:18:43,084 --> 00:18:45,353
DON'T FIT INTO THE TRADITIONAL 

482
00:18:45,353 --> 00:18:46,455
WAYS NECESSARY OF ARRIVING AT A 

483
00:18:46,455 --> 00:18:48,223
DRUG CANDIDATE.

484
00:18:48,223 --> 00:18:50,826
AND I THINK THIS IS SO IMPORTANT

485
00:18:50,826 --> 00:18:51,493
TO THINK ABOUT BECAUSE THE 

486
00:18:51,493 --> 00:18:54,329
EARLIER THAT YOU CAN PARTNER AS 

487
00:18:54,329 --> 00:18:56,064
YOU'RE GOING THROUGH THE PROCESS

488
00:18:56,064 --> 00:18:59,734
OF LEARNING AND WORKING WITH A 

489
00:18:59,734 --> 00:19:04,306
REGULATORY EXPERT OR A 

490
00:19:04,306 --> 00:19:05,774
TOXICOLOGY CONSULTANT YOU CAN 

491
00:19:05,774 --> 00:19:07,042
ALIGN WHAT THE MINIMUM PACKAGE 

492
00:19:07,042 --> 00:19:10,378
IS YOU WANT TO GET IN THE CLINIC

493
00:19:10,378 --> 00:19:13,048
AND START WORKING BACKWARDS TO 

494
00:19:13,048 --> 00:19:14,416
LOOK AT THE ADDITIONAL ASSAY 

495
00:19:14,416 --> 00:19:19,054
GIVE THE MOST DECISION MAKING 

496
00:19:19,054 --> 00:19:19,621
CRITERIA FOR YOUR SPECIFIC 

497
00:19:19,621 --> 00:19:26,995
PROGRAM.

498
00:19:26,995 --> 00:19:28,096
WHAT WHERE SOME THINGS YOU CAN 

499
00:19:28,096 --> 00:19:28,864
DO EARLY ON?

500
00:19:28,864 --> 00:19:32,667
I THINK OF RESEARCH AS THE 

501
00:19:32,667 --> 00:19:33,635
SCREENING AND I REALIZE SMALL 

502
00:19:33,635 --> 00:19:35,070
ORGANIZATIONS OR ACADEMIA YOU 

503
00:19:35,070 --> 00:19:38,573
MAY ONLY HAVE A HANDFUL OF 

504
00:19:38,573 --> 00:19:41,476
COMPOUNDS TO DRAW FROM BUT 

505
00:19:41,476 --> 00:19:42,444
DOESN'T MEAN YOU CAN'T GO 

506
00:19:42,444 --> 00:19:43,712
THROUGH THE PROCESS OF THINKING 

507
00:19:43,712 --> 00:19:44,446
ABOUT WHICH ONES LOOK MORE LIKE 

508
00:19:44,446 --> 00:19:47,048
A DRUG.

509
00:19:47,048 --> 00:19:48,917
I THINK YOU CAN DO COST 

510
00:19:48,917 --> 00:19:51,052
CONSCIOUS THINGS LIKE A TARGET 

511
00:19:51,052 --> 00:19:52,587
RISK ASSESSMENT.

512
00:19:52,587 --> 00:19:54,523
THAT'S COMBING THROUGH THE 

513
00:19:54,523 --> 00:19:56,057
LITERATURE TO LOOK TO INCLUDE 

514
00:19:56,057 --> 00:20:00,128
FROM KNOCKOUT ANIMALS AND WHAT 

515
00:20:00,128 --> 00:20:00,996
DOES EXAGGERATED PHARMACOLOGY 

516
00:20:00,996 --> 00:20:03,698
LOOK LIKE IF I KNOCK DOWN THE 

517
00:20:03,698 --> 00:20:03,999
TARGET.

518
00:20:03,999 --> 00:20:08,103
YOU CAN GET CLUES AROUND WHAT TO

519
00:20:08,103 --> 00:20:08,703
ADDRESS.

520
00:20:08,703 --> 00:20:11,973
IT'S IMPOSSIBLE TO PICK UP OFF 

521
00:20:11,973 --> 00:20:17,612
TARGET TOXICITY WHICH ARE MAJOR 

522
00:20:17,612 --> 00:20:20,348
DRIVERS FOR SMALL MOLECULE BUT 

523
00:20:20,348 --> 00:20:22,517
OTHERS WILL ADDRESS THOSE 

524
00:20:22,517 --> 00:20:22,817
PROACTIVELY.

525
00:20:22,817 --> 00:20:24,853
THIS IS UNDERSTANDING YOUR 

526
00:20:24,853 --> 00:20:27,422
TARGET AND SOME LOW TARGET 

527
00:20:27,422 --> 00:20:29,824
EVALUATIONS ARE IMMUNOGENICITY 

528
00:20:29,824 --> 00:20:32,093
ASSESSMENTS LOOKING AT 

529
00:20:32,093 --> 00:20:33,528
RELATIONSHIPS WHETHER YOU USE A 

530
00:20:33,528 --> 00:20:35,997
COMPUTERIZED SYSTEM OR COMBING 

531
00:20:35,997 --> 00:20:38,800
THE LITERATURE ARE EFFECTIVE 

532
00:20:38,800 --> 00:20:41,636
TOOLS TO HELP IDENTIFY 

533
00:20:41,636 --> 00:20:44,105
PROBLEMATIC COMPOUNDS VERY EARLY

534
00:20:44,105 --> 00:20:46,107
IN THE PROCESS AND EASY TO 

535
00:20:46,107 --> 00:20:46,942
ENGINEER AROUND WHEN YOU 

536
00:20:46,942 --> 00:20:49,611
IDENTIFY THEM WORKING WITH A 

537
00:20:49,611 --> 00:20:55,250
MEDICINAL CHEMIST YOU CAN PIVOT 

538
00:20:55,250 --> 00:20:56,151
WITHOUT LOSING A LOT OF 

539
00:20:56,151 --> 00:20:56,751
PHARMACOLOGY.

540
00:20:56,751 --> 00:20:58,520
THERE'S OTHER STUDIES HERE ON 

541
00:20:58,520 --> 00:21:01,089
THE LEFT YOU CAN ALSO DO THAT 

542
00:21:01,089 --> 00:21:03,592
WE'LL HAVE TO BRIEFLY PASS OVER 

543
00:21:03,592 --> 00:21:05,794
TODAY BUT IT'S NOT -- IT'S ALSO 

544
00:21:05,794 --> 00:21:08,463
DONE IN A RISK APPROPRIATE 

545
00:21:08,463 --> 00:21:09,731
MANNER AS YOU TWO THROUGH THE 

546
00:21:09,731 --> 00:21:11,099
SCREENING FUNNEL, THE 

547
00:21:11,099 --> 00:21:12,300
REQUIREMENTS FOR THE AMOUNT OF 

548
00:21:12,300 --> 00:21:14,102
DRUG PRODUCT YOU NEED AND THE 

549
00:21:14,102 --> 00:21:15,971
COST ASSOCIATED WITH THEM RISE 

550
00:21:15,971 --> 00:21:18,273
SO YOU WANT TO GET RID OF THOSE 

551
00:21:18,273 --> 00:21:24,479
LOW HANGING FRUIT EARLY.

552
00:21:24,479 --> 00:21:26,615
AS YOU PIVOT TO THE IN VIVO 

553
00:21:26,615 --> 00:21:28,016
STAGE OF DEVELOPMENT, WHAT ARE 

554
00:21:28,016 --> 00:21:29,684
SOME OF THE THINGS THAT ARE 

555
00:21:29,684 --> 00:21:33,822
REALLY GOOD TO DO VERY EARLY ON?

556
00:21:33,822 --> 00:21:36,424
AS YOU MATURE IN TO RUNNING A 

557
00:21:36,424 --> 00:21:39,060
RODENT STUDY OR A LARGE ANIMAL 

558
00:21:39,060 --> 00:21:40,261
STUDY HOPEFULLY AFTER YOU HAVE 

559
00:21:40,261 --> 00:21:44,099
DONE A RODENT STUDY, SOME OF THE

560
00:21:44,099 --> 00:21:48,336
EARLY NON-GLP RANGE FINDING 

561
00:21:48,336 --> 00:21:49,838
STUDIES ARE A TYPICAL 14-DAY 

562
00:21:49,838 --> 00:21:51,640
RANGE FINDING STUDY.

563
00:21:51,640 --> 00:21:54,042
YOU CAN STAGGER THE INITIATION 

564
00:21:54,042 --> 00:21:56,444
OF DOSING STARTING WITH A LOW 

565
00:21:56,444 --> 00:21:56,911
DOSE.

566
00:21:56,911 --> 00:21:58,513
SAY YOU KNOW LITTLE ABOUT YOUR 

567
00:21:58,513 --> 00:21:58,780
COMPOUND.

568
00:21:58,780 --> 00:22:06,821
YOU MAY START AT DOSE LEVEL OF 

569
00:22:06,821 --> 00:22:09,924
10 MIGS PER DAY AND IF THINGS 

570
00:22:09,924 --> 00:22:11,393
LOOK GOOD IN THE STUDIES YOU MAY

571
00:22:11,393 --> 00:22:13,995
STAGGER THE START OF A HIGH DOSE

572
00:22:13,995 --> 00:22:14,195
GROUP.

573
00:22:14,195 --> 00:22:18,199
DOSE FOR A COUPLE DAYS IF YOU 

574
00:22:18,199 --> 00:22:19,734
HAVEN'T IDENTIFIED INTOLERANCE 

575
00:22:19,734 --> 00:22:21,803
OR TOXICITY YOU MAY WANT TO PUSH

576
00:22:21,803 --> 00:22:23,872
THE DOSES FURTHER.

577
00:22:23,872 --> 00:22:26,808
AGAIN, A TOXICOLOGY STUDY THAT 

578
00:22:26,808 --> 00:22:28,443
DOES NOT IDENTIFY SAFETY 

579
00:22:28,443 --> 00:22:29,911
LIABILITIES IS NOT REALLY 

580
00:22:29,911 --> 00:22:31,980
DRIVING AND EDUCATING YOU AROUND

581
00:22:31,980 --> 00:22:33,882
WHAT YOU NEED TO LOOK FOR.

582
00:22:33,882 --> 00:22:37,385
AND HOPEFULLY YOU CAN IDENTIFY 

583
00:22:37,385 --> 00:22:39,054
WHAT YOUR KEY LIABILITIES MIGHT 

584
00:22:39,054 --> 00:22:40,655
BE WITH A COMPOUND AND USE THOSE

585
00:22:40,655 --> 00:22:45,927
TO ROLLOVER ULTIMATELY TO YOUR 

586
00:22:45,927 --> 00:22:48,196
GLP TOXICOLOGY AND HERE I LIKE 

587
00:22:48,196 --> 00:22:51,166
TO DO A FOUR-WEEK STUDY BECAUSE 

588
00:22:51,166 --> 00:22:53,868
THIS WILL ENABLE UP TO A MONTH 

589
00:22:53,868 --> 00:22:54,703
CLINICAL DOSING FROM YOUR 

590
00:22:54,703 --> 00:22:57,038
COLLEAGUES WHICH ALLOWS THEM TO 

591
00:22:57,038 --> 00:23:00,241
RUN SAD AND MAD TYPE CLINICAL 

592
00:23:00,241 --> 00:23:02,510
STUDY DESIGNS IN BOTH THE RODENT

593
00:23:02,510 --> 00:23:03,745
AND LARGE ANIMAL SPECIES 

594
00:23:03,745 --> 00:23:04,612
TYPICALLY.

595
00:23:04,612 --> 00:23:06,214
AND THE LARGE ANIMAL SPECIES 

596
00:23:06,214 --> 00:23:09,017
DOES NOT NEED TO BE NON-HUMAN 

597
00:23:09,017 --> 00:23:09,250
PRIMATE.

598
00:23:09,250 --> 00:23:14,122
IT COULD BE MINI PIG, DOG, NON 

599
00:23:14,122 --> 00:23:19,494
HUMAN PRIMATE OR ANY JUSTIFIED 

600
00:23:19,494 --> 00:23:20,128
SPECIES.

601
00:23:20,128 --> 00:23:22,263
IDEALLY YOUR STUDY WOULD BE SET 

602
00:23:22,263 --> 00:23:25,266
UP TO IDENTIFY MOST IMPORTANTLY 

603
00:23:25,266 --> 00:23:28,737
A NO-OBSERVABLE ADVERSE EFFECT 

604
00:23:28,737 --> 00:23:29,104
LEVEL.

605
00:23:29,104 --> 00:23:30,805
IF YOU CAN IDENTIFY LOW LEVELS 

606
00:23:30,805 --> 00:23:33,007
OF TOXICITY AT A MISS DOUGHED 

607
00:23:33,007 --> 00:23:35,477
AND MAXIMUM TOLERATED DOSE ARE 

608
00:23:35,477 --> 00:23:37,645
THE SPECTRUM WHERE YOU LEARN THE

609
00:23:37,645 --> 00:23:40,915
MOST FROM THE STUDY DESIGNS.

610
00:23:40,915 --> 00:23:44,119
NOW, OBVIOUSLY I'VE GLOSSED OVER

611
00:23:44,119 --> 00:23:45,253
A LOT OF KEY DATA HERE AND 

612
00:23:45,253 --> 00:23:46,855
DETAILS AROUND THE STUDY DESIGNS

613
00:23:46,855 --> 00:23:47,489
AND CONSIDERATIONS.

614
00:23:47,489 --> 00:23:50,158
SO I REALLY ENCOURAGE YOU TO GO 

615
00:23:50,158 --> 00:23:53,361
TO ICH DOT ORG AND LOOK AT THE 

616
00:23:53,361 --> 00:23:56,131
GUIDANCE ALIGNED BY A GLOBAL 

617
00:23:56,131 --> 00:23:58,299
REGULATORY AUTHORITIES AROUND 

618
00:23:58,299 --> 00:24:00,435
THE PLAYBOOKS AND EXPECTATIONS 

619
00:24:00,435 --> 00:24:03,071
AROUND DEVELOPING THE 

620
00:24:03,071 --> 00:24:03,671
THERAPEUTICS.

621
00:24:03,671 --> 00:24:06,908
FOR SMALL MOLECULES THAT'S 

622
00:24:06,908 --> 00:24:09,144
PROTEIN THERAPEUTICS HAS THEIR 

623
00:24:09,144 --> 00:24:12,180
OWN GUIDE AS WELL AS GENE 

624
00:24:12,180 --> 00:24:14,015
THERAPY.

625
00:24:14,015 --> 00:24:15,049
THE GUIDANCE AROUND GENE THERAPY

626
00:24:15,049 --> 00:24:19,053
IS GIST  JUST STARTING TO EMERGE

627
00:24:19,053 --> 00:24:20,688
SO LOOK AT THE FDA LONG-TERM 

628
00:24:20,688 --> 00:24:31,099
SAFETY GUIDANCE AS WELL.

629
00:24:38,173 --> 00:24:41,009
I WANT TO HIGHLIGHT BRIEFLY 

630
00:24:41,009 --> 00:24:44,579
AROUND MONOCLONAL ANTIBODIES AND

631
00:24:44,579 --> 00:24:45,079
PROTEINS.

632
00:24:45,079 --> 00:24:48,316
SOME MAJOR DIFFERENCES IS THE 

633
00:24:48,316 --> 00:24:50,118
EMPHASIS ON EXAGGERATED 

634
00:24:50,118 --> 00:24:50,652
PHARMACOLOGY.

635
00:24:50,652 --> 00:24:54,422
THE SPECIFICITY FOR THE 

636
00:24:54,422 --> 00:24:57,959
THERAPEUTICS ARE SO SPECIFIC 

637
00:24:57,959 --> 00:24:59,694
OVER TARGET TOXICITY IS 

638
00:24:59,694 --> 00:25:00,762
REFLECTED AROUND THE WAY WE 

639
00:25:00,762 --> 00:25:05,466
ASSESS THE PROGRAMS AND ASSETS.

640
00:25:05,466 --> 00:25:06,634
KEEP IN MIND SOME OF THE BIG 

641
00:25:06,634 --> 00:25:08,336
DIFFERENCES WE HAVE WITH SMALL 

642
00:25:08,336 --> 00:25:10,205
MOLECULES IS THE LONG HALF LIFE 

643
00:25:10,205 --> 00:25:10,805
OF THE THERAPEUTICS.

644
00:25:10,805 --> 00:25:13,641
IT'S NOT UNUSUAL TO RUN A 

645
00:25:13,641 --> 00:25:15,643
FOUR-WEEK GLP TOX STUDY TO 

646
00:25:15,643 --> 00:25:20,582
ENABLE CLINICAL DOSING FOLLOWED 

647
00:25:20,582 --> 00:25:25,720
BY A 13-WEEK RECOVERY NON-DOSING

648
00:25:25,720 --> 00:25:27,121
PHASE BECAUSE THE LONG-ACTING 

649
00:25:27,121 --> 00:25:28,122
NATURE OF THE THERAPEUTICS ARE 

650
00:25:28,122 --> 00:25:29,324
SO LONG ACTING.

651
00:25:29,324 --> 00:25:31,226
WE WANT TO MAKE SURE WE'RE 

652
00:25:31,226 --> 00:25:32,861
ASSESSING ALL THE POTENTIAL 

653
00:25:32,861 --> 00:25:34,429
SAFETY LIABILITIES OVER THE 

654
00:25:34,429 --> 00:25:38,633
DURATION OF EXPOSURE HERE.

655
00:25:38,633 --> 00:25:41,603
AND THERE ARE ALSO VERY SPECIFIC

656
00:25:41,603 --> 00:25:43,404
TISSUE CROSS REACTIVITY STUDY WE

657
00:25:43,404 --> 00:25:44,305
CONDUCT TO MAKE SURE WE 

658
00:25:44,305 --> 00:25:45,807
UNDERSTAND WHAT THE FINDING 

659
00:25:45,807 --> 00:25:47,041
POTENTIAL IS BUT A LOT OF THESE 

660
00:25:47,041 --> 00:25:50,912
EFFECTS WE TYPICALLY LOOK FOR 

661
00:25:50,912 --> 00:25:53,047
WITH PROTEIN THERAPEUTICS OR 

662
00:25:53,047 --> 00:25:55,483
MONOCLONAL ANTIBODIES ARE REALLY

663
00:25:55,483 --> 00:25:58,586
THE EFFECT ON THE IMMUNOGENICITY

664
00:25:58,586 --> 00:26:00,688
AND HOW THE IMMUNE SYSTEM REACT 

665
00:26:00,688 --> 00:26:02,123
TO CLEAR THESE THERAPEUTICS 

666
00:26:02,123 --> 00:26:05,393
EARLY ON, WHICH MAY ACTUALLY 

667
00:26:05,393 --> 00:26:08,096
MINIMIZE THE EFFECTIVENESS OF 

668
00:26:08,096 --> 00:26:11,065
OUR TOXICOLOGY STUDY TO INFORM 

669
00:26:11,065 --> 00:26:14,135
CLINICAL IMPLICATIONS OR 

670
00:26:14,135 --> 00:26:15,503
ALTERNATIVELY THINKING HOW THE 

671
00:26:15,503 --> 00:26:16,938
IMMUNE SYSTEM MAY BE EITHER 

672
00:26:16,938 --> 00:26:19,540
EXPRESSED OR ACTIVATED DEPENDING

673
00:26:19,540 --> 00:26:21,075
ON THE INTENDED PHARMACOLOGY OF 

674
00:26:21,075 --> 00:26:28,549
THE THERAPEUTIC.

675
00:26:28,549 --> 00:26:29,984
TO BRIEFLY TALK ABOUT GENE 

676
00:26:29,984 --> 00:26:32,487
THERAPIES, AGAIN THESE ARE VERY 

677
00:26:32,487 --> 00:26:33,488
DIFFERENTIATED FROM SMALL 

678
00:26:33,488 --> 00:26:35,023
MOLECULES OR PROTEIN 

679
00:26:35,023 --> 00:26:35,356
THERAPEUTICS.

680
00:26:35,356 --> 00:26:36,257
A LOT OF TIMES NOW IF WE'RE 

681
00:26:36,257 --> 00:26:38,626
LOOKING AT GENE THERAPIES WE 

682
00:26:38,626 --> 00:26:41,696
REALLY WANT TO UNDERSTAND THE 

683
00:26:41,696 --> 00:26:43,398
TROPISM AND BIO DISTRIBUTION 

684
00:26:43,398 --> 00:26:45,667
SPECIFICALLY WHAT TYPES OF CELLS

685
00:26:45,667 --> 00:26:48,603
OR TISSUES DOES THE VIRUS 

686
00:26:48,603 --> 00:26:50,371
TRANSDUCE OR A LIPID NANO 

687
00:26:50,371 --> 00:26:52,006
PARTICLE, LET'S SAY.

688
00:26:52,006 --> 00:26:53,675
WITHIN GENE THERAPIES THERE'S 

689
00:26:53,675 --> 00:26:58,379
DIFFERENT SUB HEADINGS AROUND 

690
00:26:58,379 --> 00:27:00,448
OLIGO NUCLEOTIDES AND THIS AREA 

691
00:27:00,448 --> 00:27:00,949
IS EXPLODING.

692
00:27:00,949 --> 00:27:03,051
WE CAN'T POSSIBLY COVER ALL THE 

693
00:27:03,051 --> 00:27:05,787
BEST PRACTICES IN THIS SPACE 

694
00:27:05,787 --> 00:27:06,754
WITHOUT INTO SPECIFICS.

695
00:27:06,754 --> 00:27:09,457
BUT AT A HIGH LEVEL WE REALLY 

696
00:27:09,457 --> 00:27:10,858
WANT TO UNDERSTAND THE BIO 

697
00:27:10,858 --> 00:27:13,294
DISTRIBUTION I MENTIONED.

698
00:27:13,294 --> 00:27:18,533
THE PHARMACO KINETIC, PHARMACO 

699
00:27:18,533 --> 00:27:20,134
DYNAMIC PROFILE AROUND THE 

700
00:27:20,134 --> 00:27:23,037
VECTOR GENOMES OR DNA 

701
00:27:23,037 --> 00:27:23,738
DISTRIBUTION YOU'RE DELIVERING 

702
00:27:23,738 --> 00:27:27,709
WITH YOUR VECTOR LIPID NANO 

703
00:27:27,709 --> 00:27:28,743
PARTICLE AND THE PROTEIN 

704
00:27:28,743 --> 00:27:30,378
EXPRESSION YOU HAVE SECONDARY TO

705
00:27:30,378 --> 00:27:31,045
THIS.

706
00:27:31,045 --> 00:27:33,514
THEN AS WE TALKED ABOUT WITH 

707
00:27:33,514 --> 00:27:36,451
MONOCLONAL ANTIBODIES THE 

708
00:27:36,451 --> 00:27:37,085
IMMUNOGENICITY COMPONENT.

709
00:27:37,085 --> 00:27:39,721
ARE THERE PREEXISTING ANTIBODIES

710
00:27:39,721 --> 00:27:41,956
THAT MAY SUPPRESS THE EFFECT TO 

711
00:27:41,956 --> 00:27:45,293
MODEL THIS THERAPY IN YOUR 

712
00:27:45,293 --> 00:27:45,526
SPECIES.

713
00:27:45,526 --> 00:27:47,962
AND THE IMMUNE RESPONSES.

714
00:27:47,962 --> 00:27:52,300
REMEMBER IF YOU'RE DELIVERING A 

715
00:27:52,300 --> 00:27:53,234
VECTOR-BASED VIRAL-BASED GENE 

716
00:27:53,234 --> 00:27:56,104
DELIVER HADDY APPROACH YOUR 

717
00:27:56,104 --> 00:27:57,605
IMMUNE SYSTEM RECOGNIZES THIS 

718
00:27:57,605 --> 00:27:58,806
AND ACTIVELY THINKING IT'S UNDER

719
00:27:58,806 --> 00:28:01,476
ATTACK AND WILL FIGHT BACK.

720
00:28:01,476 --> 00:28:05,613
ARE THERE DOWN STREAM EFFECTS ON

721
00:28:05,613 --> 00:28:06,214
CYTOKINE OR COMPLEMENT 

722
00:28:06,214 --> 00:28:09,083
ACTIVATION AND HOW DOES THAT FIT

723
00:28:09,083 --> 00:28:14,155
IN WITH YOUR INTENDED 

724
00:28:14,155 --> 00:28:19,260
THERAPEUTIC DOSE.

725
00:28:19,260 --> 00:28:21,729
THIS IS DIFFERENT THAN OTHER 

726
00:28:21,729 --> 00:28:22,730
MODALITIES WHERE YOU'RE DOSING 

727
00:28:22,730 --> 00:28:27,068
DAILY FOR A SMALL MOLECULE, SAY 

728
00:28:27,068 --> 00:28:29,070
WEEKLY OR BIWEEKLY FOR 

729
00:28:29,070 --> 00:28:30,671
THERAPEUTIC POTENTIAL OR LONGER 

730
00:28:30,671 --> 00:28:31,039
CADENCE.

731
00:28:31,039 --> 00:28:34,208
AND THEN MAYBE A SINGLE DOSE 

732
00:28:34,208 --> 00:28:35,943
ONLY FOR THESE TYPES OF IND 

733
00:28:35,943 --> 00:28:38,179
ENABLING STUDIES.

734
00:28:38,179 --> 00:28:39,981
IT'S REALLY IMPORTANT TO 

735
00:28:39,981 --> 00:28:40,948
UNDERSTAND WHAT YOUR LIABILITIES

736
00:28:40,948 --> 00:28:42,417
ARE AND DEVELOP THE RIGHT 

737
00:28:42,417 --> 00:28:44,085
PACKAGE AND THIS IS WHERE IT'S 

738
00:28:44,085 --> 00:28:47,555
SO IMPORTANT TO WORK WITH A GOOD

739
00:28:47,555 --> 00:28:48,589
REGULATORY OR TOXICOLOGY 

740
00:28:48,589 --> 00:28:50,158
CONSULTANT AS YOU'RE STARTING TO

741
00:28:50,158 --> 00:28:52,093
THINK ABOUT THIS IF YOU'RE NEW 

742
00:28:52,093 --> 00:28:53,961
TO THE FIELD TO STEER IN AND 

743
00:28:53,961 --> 00:28:56,264
IT'S NOT A ONE-SIZE FITS ALL 

744
00:28:56,264 --> 00:29:02,170
TYPE OF SITUATION.

745
00:29:02,170 --> 00:29:04,305
FINALLY, I WANTED TO OFFER SOME 

746
00:29:04,305 --> 00:29:04,906
PARTING THOUGHTS AROUND DRUG 

747
00:29:04,906 --> 00:29:07,708
SAFETY.

748
00:29:07,708 --> 00:29:09,577
YOU HAVE TO UNDERSTAND UP FRONT 

749
00:29:09,577 --> 00:29:11,045
YOU CAN'T PREDICT EVERYTHING.

750
00:29:11,045 --> 00:29:13,314
AS YOU'RE THINKING ABOUT YOUR 

751
00:29:13,314 --> 00:29:16,517
ASSETS EARLY ON, IT'S OKAY TO 

752
00:29:16,517 --> 00:29:19,087
BELIEVE AND ADVOCATE FOR YOUR 

753
00:29:19,087 --> 00:29:19,520
DRUGS.

754
00:29:19,520 --> 00:29:20,154
THERE ARE THINGS YOU KNOW AND 

755
00:29:20,154 --> 00:29:23,057
THINGS YOU DON'T KNOW.

756
00:29:23,057 --> 00:29:26,561
PART OF THE DRUG DEVELOPMENT 

757
00:29:26,561 --> 00:29:28,162
PROCESS AS MARRAH MENTIONED IS 

758
00:29:28,162 --> 00:29:32,200
TALKING TO INVESTORS TALKING TO 

759
00:29:32,200 --> 00:29:33,568
CLINICIANS AND KEY OPINION 

760
00:29:33,568 --> 00:29:36,104
LEADERS AND GET THEM EXCITED 

761
00:29:36,104 --> 00:29:36,938
ABOUT WHAT NOVEL THERAPEUTIC 

762
00:29:36,938 --> 00:29:39,207
OPTIONS YOU BRING TO DISEASES 

763
00:29:39,207 --> 00:29:41,542
POTENTIALLY WITH HIGH UNMET 

764
00:29:41,542 --> 00:29:41,742
NEED.

765
00:29:41,742 --> 00:29:43,444
IT'S OKAY TO BE ENTHUSIASTIC 

766
00:29:43,444 --> 00:29:44,412
ABOUT YOUR DATA.

767
00:29:44,412 --> 00:29:47,548
IT'S ALSO SOMETHING YOU NEED TO 

768
00:29:47,548 --> 00:29:48,249
APPRECIATE THERE ARE LIMITS TO 

769
00:29:48,249 --> 00:29:49,684
WHAT YOU KNOW AND DON'T KNOW 

770
00:29:49,684 --> 00:29:52,353
AROUND THE ROBUSTNESS OF THAT 

771
00:29:52,353 --> 00:29:52,620
DATA.

772
00:29:52,620 --> 00:29:55,089
SO IT'S SO IMPORTANT TO BE 

773
00:29:55,089 --> 00:29:57,825
TRANSPARENT WITH PATIENT, YOUR 

774
00:29:57,825 --> 00:29:58,493
CLINICAL COLLEAGUES AND 

775
00:29:58,493 --> 00:30:01,729
CERTAINLY REGULATORS AROUND WHY 

776
00:30:01,729 --> 00:30:03,931
YOU BELIEVE A DRUG IS SAFE AND 

777
00:30:03,931 --> 00:30:05,900
EFFICACIOUS AND MAYBE AREAS YOU 

778
00:30:05,900 --> 00:30:08,202
DON'T KNOW OF UNCERTAINTY AND 

779
00:30:08,202 --> 00:30:13,674
HOW YOU PLAN ON ADDRESSING THAT 

780
00:30:13,674 --> 00:30:15,676
AROUND SAFETY MARGINS OR SAFETY 

781
00:30:15,676 --> 00:30:17,011
MARKERS YOU CAN FOLLOW IN 

782
00:30:17,011 --> 00:30:18,312
PATIENTS TO UNDERSTAND THIS.

783
00:30:18,312 --> 00:30:19,914
I THINK IT'S OKAY TO BE PARANOID

784
00:30:19,914 --> 00:30:21,048
AS YOU TWO THROUGH DEVELOPMENT.

785
00:30:21,048 --> 00:30:26,220
THIS IS ACTUALLY A GOOD THING 

786
00:30:26,220 --> 00:30:27,755
AROUND UNDERSTANDING TYPICAL 

787
00:30:27,755 --> 00:30:29,090
ISSUES THAT MAY COME UP AND 

788
00:30:29,090 --> 00:30:30,825
YOU'LL HAVE SPURIOUS FINDINGS IN

789
00:30:30,825 --> 00:30:35,730
SOME OF YOUR STUDIES OR DATA NOT

790
00:30:35,730 --> 00:30:37,698
CONGRUENT WITH A -- THERE MAY BE

791
00:30:37,698 --> 00:30:40,067
OPPOSING DATA SETS THAT SAY IN 

792
00:30:40,067 --> 00:30:42,436
ONE SPECIES IT MAY BE UNSAFE AND

793
00:30:42,436 --> 00:30:43,504
ANOTHER SPECIES SAFE AND THEN 

794
00:30:43,504 --> 00:30:44,605
YOU HAVE TO GO THROUGH AND 

795
00:30:44,605 --> 00:30:46,340
FIGURE OUT WHICH IS THE RIGHT 

796
00:30:46,340 --> 00:30:49,043
ONE TO BELIEVE.

797
00:30:49,043 --> 00:30:50,178
ALL THOSE ARE ACCESSIBLE AND 

798
00:30:50,178 --> 00:30:50,678
OKAY.

799
00:30:50,678 --> 00:30:55,049
IT COMES BACK TO THE 

800
00:30:55,049 --> 00:30:56,984
TRANSPARENCY EFFECT.

801
00:30:56,984 --> 00:30:59,086
YOU WANT TO AVOID BOX CHECKING.

802
00:30:59,086 --> 00:31:00,354
IF YOU GO AND ASK PEOPLE WHAT 

803
00:31:00,354 --> 00:31:03,057
THEY DID FOR THEIR PROGRAMS, YOU

804
00:31:03,057 --> 00:31:04,992
MAY GET STUDIES YOU ARE 

805
00:31:04,992 --> 00:31:06,727
ESSENTIALLY UNNECESSARY.

806
00:31:06,727 --> 00:31:08,162
THIS IS A BIG CONSIDERATION WHEN

807
00:31:08,162 --> 00:31:11,098
IN VIVO STUDIES COME INTO PLAY.

808
00:31:11,098 --> 00:31:13,734
WHERE WE DON'T WANT TO BE 

809
00:31:13,734 --> 00:31:15,336
RUNNING UNNECESSARY IN VIVO 

810
00:31:15,336 --> 00:31:17,004
STUDIES THAT DON'T NECESSARILY 

811
00:31:17,004 --> 00:31:19,006
ADD VALUE.

812
00:31:19,006 --> 00:31:20,408
THE EARLIEST YOU CAN PARTNER 

813
00:31:20,408 --> 00:31:22,843
WITH REGULATORY EXPERTS OR FOLKS

814
00:31:22,843 --> 00:31:24,312
IN THE NON-CLINICAL SAFETY 

815
00:31:24,312 --> 00:31:26,747
SPACES, CONSULTANTS OR OTHERWISE

816
00:31:26,747 --> 00:31:28,516
AND UNDERSTAND THE MINIMUM 

817
00:31:28,516 --> 00:31:29,750
PACKAGE THAT'S NECESSARY TO GET 

818
00:31:29,750 --> 00:31:32,720
INTO THE CLINIC, IT'S MOST COST 

819
00:31:32,720 --> 00:31:34,322
EFFECTIVE AND STREAMLINED AND 

820
00:31:34,322 --> 00:31:36,457
THEN START WORKING BACKWARDS TO 

821
00:31:36,457 --> 00:31:39,060
SAY, DOES THIS ADEQUATELY 

822
00:31:39,060 --> 00:31:41,629
ADDRESS MY SAFETY LIABILITIES 

823
00:31:41,629 --> 00:31:44,398
AND EFFICACY OBJECTIVES IN ORDER

824
00:31:44,398 --> 00:31:46,267
TO TREAT THIS DISEASE?

825
00:31:46,267 --> 00:31:48,502
THEN YOU START BUILDING OUT FROM

826
00:31:48,502 --> 00:31:48,703
THERE.

827
00:31:48,703 --> 00:31:50,171
I THINK ABOUT IT THIS WAY 

828
00:31:50,171 --> 00:31:52,773
BECAUSE IT MINIMIZES THE 

829
00:31:52,773 --> 00:31:54,475
UNNECESSARY STUDIES THAT DON'T 

830
00:31:54,475 --> 00:31:56,177
ULTIMATELY BRING VALUE TO YOUR 

831
00:31:56,177 --> 00:31:56,477
THERAPEUTIC.

832
00:31:56,477 --> 00:31:58,713
AND AS YOU'LL HEAR FROM OTHERS 

833
00:31:58,713 --> 00:32:02,183
IN THE REGULATORY SPACE, PART OF

834
00:32:02,183 --> 00:32:04,318
THIS DYNAMIC PROCESS WE HAVE IS 

835
00:32:04,318 --> 00:32:06,120
DISCUSSING WITH REGULATORS 

836
00:32:06,120 --> 00:32:09,657
AROUND HERE'S WHAT I BELIEVE IS 

837
00:32:09,657 --> 00:32:12,226
THE RIGHT PATH FORWARD AND THEY 

838
00:32:12,226 --> 00:32:12,760
KNOW THINGS FROM THEIR 

839
00:32:12,760 --> 00:32:14,428
EXPERIENCE AND SAY WE UNDERSTAND

840
00:32:14,428 --> 00:32:17,331
THAT PERSPECTIVE BUT HAVE YOU 

841
00:32:17,331 --> 00:32:17,965
CONSIDERED THIS?

842
00:32:17,965 --> 00:32:19,867
WE WOULD RECOMMEND YOU DO THIS 

843
00:32:19,867 --> 00:32:21,402
GOING FORWARD.

844
00:32:21,402 --> 00:32:23,437
IT'S THE DYNAMIC PROCESS.

845
00:32:23,437 --> 00:32:26,040
ALL THE CREDIT IN THE WORLD TO 

846
00:32:26,040 --> 00:32:26,674
THE FDA.

847
00:32:26,674 --> 00:32:27,708
THEY'VE BEEN GREAT AROUND TRYING

848
00:32:27,708 --> 00:32:29,110
TO IMPROVE THE COLLABORATIVE 

849
00:32:29,110 --> 00:32:31,012
DISCUSSION AROUND TRYING TO FIND

850
00:32:31,012 --> 00:32:34,815
THE RIGHT PATH FORWARD FROM A 

851
00:32:34,815 --> 00:32:35,483
NON-CLINICAL SAFETY PERSPECTIVE 

852
00:32:35,483 --> 00:32:39,787
TO MINIMIZE RISK FOR PATIENTS.

853
00:32:39,787 --> 00:32:42,556
AND THEN FINALLY'S OKAY TO APPLY

854
00:32:42,556 --> 00:32:44,191
RISK IN A THOUGHTFUL, CALCULATED

855
00:32:44,191 --> 00:32:44,525
MANNER.

856
00:32:44,525 --> 00:32:46,761
I CAN TELL YOU HAVING ON EIGHT 

857
00:32:46,761 --> 00:32:48,095
MARKETED THERAPEUTICS RIGHT NOW,

858
00:32:48,095 --> 00:32:50,231
THERE WAS A COMPELLING REASON TO

859
00:32:50,231 --> 00:32:52,366
TERMINATE ALL OF THEM.

860
00:32:52,366 --> 00:32:55,036
IT IS A VERY LONG, DRAWN OUT 

861
00:32:55,036 --> 00:32:56,170
PROCESS THAT INVOLVES A LOT OF 

862
00:32:56,170 --> 00:32:57,538
FOLKS ALONG THE WAY.

863
00:32:57,538 --> 00:32:59,907
IT'S OKAY TO FIGHT AND CONVINCE 

864
00:32:59,907 --> 00:33:02,109
OTHERS TO GET THEM TO BELIEVE 

865
00:33:02,109 --> 00:33:03,044
WHAT YOU BELIEVE AND ADVOCATE 

866
00:33:03,044 --> 00:33:04,345
FOR PATIENTS AND WHAT YOU 

867
00:33:04,345 --> 00:33:07,381
BELIEVE IS AN EFFECTIVE THERAPY.

868
00:33:07,381 --> 00:33:10,618
IT'S OKAY TO DO THIS.

869
00:33:10,618 --> 00:33:13,321
JUST REMEMBER THAT SUCCESSFUL 

870
00:33:13,321 --> 00:33:13,788
PHARMACEUTICALS ARE 

871
00:33:13,788 --> 00:33:15,323
EXCEPTIONALLY RARE EVENTS AND 

872
00:33:15,323 --> 00:33:16,424
YOU NEED TO PUSH FORWARD AND 

873
00:33:16,424 --> 00:33:17,925
BELIEVE IN THEM.

874
00:33:17,925 --> 00:33:20,094
AT THE SAME TIME ALWAYS 

875
00:33:20,094 --> 00:33:20,695
BALANCING THAT WITH PATIENT 

876
00:33:20,695 --> 00:33:23,631
SAFETY GOING FORWARD.

877
00:33:23,631 --> 00:33:25,166
WITH THAT I'LL CONCLUDE MY 

878
00:33:25,166 --> 00:33:26,233
PRESENTATION AND TURN IT BACK 

879
00:33:26,233 --> 00:33:31,639
OVER TO MARRAH.

880
00:33:31,639 --> 00:33:35,042
THANK YOU VERY MUCH.

881
00:33:35,042 --> 00:33:37,745
>> THANK YOU, JEFF.

882
00:33:37,745 --> 00:33:42,983
SO WE'LL MOVE INTO SESSION 1 AND

883
00:33:42,983 --> 00:33:48,189
I WOULD LIKE TO OVER TO MY 

884
00:33:48,189 --> 00:33:54,929
COLLEAGUE, JOHN SHERIDAN.

885
00:33:54,929 --> 00:33:56,430
>> THIS IS DRUG DEVICE 

886
00:33:56,430 --> 00:33:57,798
DEVELOPMENT PROCESS MODELS AND 

887
00:33:57,798 --> 00:34:01,469
METHODS CHIEF OF THE BRANCH OF 

888
00:34:01,469 --> 00:34:07,708
THE DIVISION OF LUNG DISEASES AT

889
00:34:07,708 --> 00:34:11,045
NHLBI AND MY CO-MODERATOR IS 

890
00:34:11,045 --> 00:34:15,316
CHARLIE REN.

891
00:34:15,316 --> 00:34:21,589
>> I'M FROM CARNEGIE MELON

892
00:34:21,589 --> 00:34:22,356
UNIVERSITY.

893
00:34:22,356 --> 00:34:23,891
>> THESE ARE WHAT MAY BE 

894
00:34:23,891 --> 00:34:25,126
EMPLOYED THROUGH THE 

895
00:34:25,126 --> 00:34:26,060
THERAPEUTICS FOR LUNG AND SLEEP 

896
00:34:26,060 --> 00:34:29,163
DISEASES AND DISORDERS.

897
00:34:29,163 --> 00:34:31,632
SO WE HAVE FOUR EXCITING 

898
00:34:31,632 --> 00:34:33,300
PRESENTATIONS THAT WILL COVER 

899
00:34:33,300 --> 00:34:36,170
BOTH IN VITRO AND IN VIVO 

900
00:34:36,170 --> 00:34:36,404
MODELS.

901
00:34:36,404 --> 00:34:39,974
THE FIRST SPEAKER -- NEXT SLIDE.

902
00:34:39,974 --> 00:34:42,209
THE FIRST SPEAKER IS ALFRED 

903
00:34:42,209 --> 00:34:44,245
TAMAYO, A PRINCIPAL SCIENTIST 

904
00:34:44,245 --> 00:34:45,946
FROM CARBON BIO SCIENCES.

905
00:34:45,946 --> 00:34:48,282
HE'LL BE FOLLOWED BY ERIN 

906
00:34:48,282 --> 00:34:49,750
HENNESSEY A Ph.D. CANDIDATE FROM

907
00:34:49,750 --> 00:34:52,253
THE BOSTON UNIVERSITY SCHOOL OF 

908
00:34:52,253 --> 00:34:54,388
MEDICINE AND ERIN IS UNABLE TO 

909
00:34:54,388 --> 00:34:56,257
ATTEND LIVE SO HER PRESENTATION 

910
00:34:56,257 --> 00:34:59,059
WAS RE-RECORDED.

911
00:34:59,059 --> 00:35:03,597
WE'LL NEXT HAVE A PRESENTATION 

912
00:35:03,597 --> 00:35:08,903
FROM KRISTIN BIRCSAK A PRINCIPAL

913
00:35:08,903 --> 00:35:11,272
SCIENTIST AND THE FINAL 

914
00:35:11,272 --> 00:35:12,440
PRESENTATION IS FROM JOHN 

915
00:35:12,440 --> 00:35:15,042
ENGLEHARDT FROM THE UNIVERSITY 

916
00:35:15,042 --> 00:35:16,210
OF IOWA.

917
00:35:16,210 --> 00:35:19,046
AND AS A REMINDER, WE'LL BE 

918
00:35:19,046 --> 00:35:20,214
HOLDING Q&A UNTIL THE PANEL 

919
00:35:20,214 --> 00:35:21,649
DISCUSSION AND TURG THE TALKS 

920
00:35:21,649 --> 00:35:26,120
YOU CAN ENTER ANY QUESTIONS YOU 

921
00:35:26,120 --> 00:35:27,488
HAVE USING THE Q&A FEATURE.

922
00:35:27,488 --> 00:35:29,256
WITH THAT I'LL TURN IT OVER TO 

923
00:35:29,256 --> 00:35:39,700
OUR FIRST SPEAKER, ALFRED.

924
00:35:40,668 --> 00:35:45,005
>> THANK YOU FOR THAT, DR

925
00:35:45,005 --> 00:35:46,574
DR. SHERIDAN.

926
00:35:46,574 --> 00:35:50,244
THANK YOU FOR THE MODERATORS FOR

927
00:35:50,244 --> 00:35:53,514
CREATING THIS GREAT WORKSHOP AND

928
00:35:53,514 --> 00:35:55,416
INVITING ME AND THE AMAZING 

929
00:35:55,416 --> 00:35:57,618
SPEAKERS IN THE GROUP.

930
00:35:57,618 --> 00:36:01,455
I'LL SHARE A CASE STUDY FROM 

931
00:36:01,455 --> 00:36:02,456
ACADEMIC ROOTS TO PATIENTS WHO 

932
00:36:02,456 --> 00:36:03,057
NEED THEM.

933
00:36:03,057 --> 00:36:07,661
IT'S A FEAT OF MONUMENTAL 

934
00:36:07,661 --> 00:36:11,065
PROPORTIONS AND WORKED WITH AN 

935
00:36:11,065 --> 00:36:11,732
INCREDIBLE GROUP.

936
00:36:11,732 --> 00:36:13,467
I THINK WE HAVE A GOOD SHOT.

937
00:36:13,467 --> 00:36:15,703
THAT'S BY FAR BEEN THE MOST 

938
00:36:15,703 --> 00:36:19,073
IMPORTANT LESSON TO DATE WITH 

939
00:36:19,073 --> 00:36:20,441
THE RIGHT PEOPLE AND MIND SET, 

940
00:36:20,441 --> 00:36:23,043
ANYTHING IS POSSIBLE.

941
00:36:23,043 --> 00:36:27,014
WITH THE VIGNETTE I CAN OFFER 

942
00:36:27,014 --> 00:36:30,084
SNIPPETS OF THE MANY CHALLENGES 

943
00:36:30,084 --> 00:36:31,519
WE'VE FACED AND THOSE THAT ARE 

944
00:36:31,519 --> 00:36:32,019
SHORT COMES.

945
00:36:32,019 --> 00:36:34,788
AS I SAID FROM THE START IT'S AN

946
00:36:34,788 --> 00:36:36,056
UNFINISHED CASE STUDY SO I CAN'T

947
00:36:36,056 --> 00:36:38,259
TELL YOU HOW IT ENDS BUT CAN 

948
00:36:38,259 --> 00:36:39,860
SHARE WHAT I'VE LEARNED ALONG 

949
00:36:39,860 --> 00:36:42,263
THE WAY FROM THE PERSPECTIVE OF 

950
00:36:42,263 --> 00:36:43,397
A CURRENT INDUSTRY SCIENTIST AND

951
00:36:43,397 --> 00:36:53,507
FORMER ACADEMIC RESEARCHER.

952
00:36:53,507 --> 00:36:55,576
A YEAR AND A HALF AT CARBON 

953
00:36:55,576 --> 00:36:57,878
MARKS MY FIRST LUNG FOCUSSED 

954
00:36:57,878 --> 00:36:59,179
INDICATION AND WORKED WITH A 

955
00:36:59,179 --> 00:37:01,849
COMPANY ATTEMPTING TO TACKLE 

956
00:37:01,849 --> 00:37:08,789
DRUG RESIST TACT -- RESISTANCE 

957
00:37:08,789 --> 00:37:12,259
OF THE LUNG AND AT THE MICROBES 

958
00:37:12,259 --> 00:37:14,328
AND NOT SO MUCH THE LUNG.

959
00:37:14,328 --> 00:37:16,397
WE FACED CHALLENGES HOW TO MIMIC

960
00:37:16,397 --> 00:37:18,766
THE RIGHT DELIVERY OF DRUG TO 

961
00:37:18,766 --> 00:37:22,870
HUMAN LUNGS AND WHAT MAY BE THE 

962
00:37:22,870 --> 00:37:24,138
TRANSLATIONAL MODEL AND FACED 

963
00:37:24,138 --> 00:37:26,040
CHALLENGES IN ACHIEVING POTENCY 

964
00:37:26,040 --> 00:37:27,608
AND ACCEPTED MODELS.

965
00:37:27,608 --> 00:37:29,376
A SUBJECT THAT PERHAPS DESERVES 

966
00:37:29,376 --> 00:37:31,545
ITS OWN TALK.

967
00:37:31,545 --> 00:37:35,382
AND I TOOK A POSITION AT 

968
00:37:35,382 --> 00:37:39,620
ANALYTICA AND IT WAS A STORIED 

969
00:37:39,620 --> 00:37:44,258
COMPANY OF A FEW HUNDRED PEOPLE.

970
00:37:44,258 --> 00:37:46,727
AND THE MANUFACTURING FACILITY 

971
00:37:46,727 --> 00:37:54,001
IN THE QC DEPARTMENT AND WORKED 

972
00:37:54,001 --> 00:37:56,837
WITH THE THERAPEUTIC ON CMS FOR 

973
00:37:56,837 --> 00:37:58,205
CHEMISTRY, MANUFACTURING AND 

974
00:37:58,205 --> 00:37:58,606
CONTROLS.

975
00:37:58,606 --> 00:38:01,675
AS AN ACADEMIC INITIALLY 

976
00:38:01,675 --> 00:38:03,310
INDUSTRY SCIENCE I HAD NEVER 

977
00:38:03,310 --> 00:38:04,745
HEARD THE TERM BUT IF YOU WANT 

978
00:38:04,745 --> 00:38:10,084
TO BRING THE DRUG TO THE CLINIC 

979
00:38:10,084 --> 00:38:12,152
IT'S ADVISABLE TO THINK OF 

980
00:38:12,152 --> 00:38:14,254
PRACTICES AND GUIDELINES JEFF 

981
00:38:14,254 --> 00:38:23,030
TOUCHED UPON.

982
00:38:23,030 --> 00:38:24,632
I'LL TOUCH ON THIS NOW AND BEEN 

983
00:38:24,632 --> 00:38:25,499
DURING MY TALK.

984
00:38:25,499 --> 00:38:27,568
WHEN I FIRST SAW CARBON IT WAS 

985
00:38:27,568 --> 00:38:31,071
PRIMARILY THROUGH THE LENS OF MY

986
00:38:31,071 --> 00:38:34,775
EXPERIENCE AT ANALYTICA AND 

987
00:38:34,775 --> 00:38:38,278
TECHNOLOGY BY A CO-FOUNDER BUT 

988
00:38:38,278 --> 00:38:45,252
THAT'S A STORY BY ANOTHER TIME.

989
00:38:45,252 --> 00:38:48,288
CARBON IS TRYING TO CORRECT 

990
00:38:48,288 --> 00:38:49,657
CYSTIC FIBROSIS.

991
00:38:49,657 --> 00:38:52,092
THERE'S NO APPROVED GENE 

992
00:38:52,092 --> 00:38:55,396
THERAPIES OF ANY KIND FOR CYSTIC

993
00:38:55,396 --> 00:38:57,031
FIBROSIS AND WHILE IT'S 

994
00:38:57,031 --> 00:38:58,065
EXHILARATING IF YOU'RE NOT 

995
00:38:58,065 --> 00:38:59,233
TERRIFIED YOU PROBABLY HAVEN'T 

996
00:38:59,233 --> 00:39:04,672
THOUGHT IT THROUGH.

997
00:39:04,672 --> 00:39:05,172
CYSTIC FIBROSIS IS A 

998
00:39:05,172 --> 00:39:06,273
LIFE-THREATENING INHERITED 

999
00:39:06,273 --> 00:39:09,009
DISEASE WITH HIGH MORBIDITY AND 

1000
00:39:09,009 --> 00:39:10,644
MORTALITY AND AVERAGE AGE OF 

1001
00:39:10,644 --> 00:39:12,346
DEATH OF 40 YEARS OLD.

1002
00:39:12,346 --> 00:39:13,781
WHILE THERE'S SMALL MOLECULE 

1003
00:39:13,781 --> 00:39:14,682
DRUGS AVAILABLE NOT ALL PATIENTS

1004
00:39:14,682 --> 00:39:18,452
CAN USE THEM.

1005
00:39:18,452 --> 00:39:24,992
A CRITICAL SUBSET OF CF HAVE 

1006
00:39:24,992 --> 00:39:27,027
INTOLERANCE OR NON COMPLIANCE 

1007
00:39:27,027 --> 00:39:29,963
AND WILL TARGET 10,000 PATIENTS 

1008
00:39:29,963 --> 00:39:32,866
NOT CURRENTLY ELIGIBLE FOR 

1009
00:39:32,866 --> 00:39:41,675
STANDARD OF CARE DRUGS.

1010
00:39:41,675 --> 00:39:44,278
CARBON PLANS TO REFER TO AN 

1011
00:39:44,278 --> 00:39:46,213
APPROACH THAT DELIVERS GENETIC 

1012
00:39:46,213 --> 00:39:47,047
INFORMATION TO A PATIENT'S 

1013
00:39:47,047 --> 00:39:47,414
CELLS.

1014
00:39:47,414 --> 00:39:48,649
IF THE PATIENT'S DISEASE IS 

1015
00:39:48,649 --> 00:39:49,783
CAUSED BY MUTATION THAT RESULTS 

1016
00:39:49,783 --> 00:39:50,851
IN THE LACK OF FUNCTIONAL 

1017
00:39:50,851 --> 00:39:53,587
PROTEIN AS IS THE CASE FOR CF, 

1018
00:39:53,587 --> 00:39:55,956
THEN DELIVERING A NORMAL COPY OF

1019
00:39:55,956 --> 00:39:57,624
THE GENE COULD HAVE THERAPEUTIC 

1020
00:39:57,624 --> 00:39:59,359
OR CURATIVE EFFECTS.

1021
00:39:59,359 --> 00:40:02,262
THIS APPROACH CAN COME IN 

1022
00:40:02,262 --> 00:40:04,465
SEVERAL MODALITIES THROUGH LIPID

1023
00:40:04,465 --> 00:40:07,267
NANO PARTICLES OR VIRAL VECTORS.

1024
00:40:07,267 --> 00:40:09,636
VIRAL VECTORS ARE VIRUSES 

1025
00:40:09,636 --> 00:40:11,705
ENGINEERED TO DELIVER PAY LOADS 

1026
00:40:11,705 --> 00:40:14,274
WITHOUT REPLICATING.

1027
00:40:14,274 --> 00:40:18,779
AMONG THE MOST COMMON EMPLOYED 

1028
00:40:18,779 --> 00:40:21,915
ARE AAV AND LINDSEY VIRUS 

1029
00:40:21,915 --> 00:40:23,684
HOWEVER, VIRAL VECTORS CAN COME 

1030
00:40:23,684 --> 00:40:26,053
WITH SIGNIFICANT RISK AND 

1031
00:40:26,053 --> 00:40:27,354
LIMITATIONS INCLUDING TISSUE OFF

1032
00:40:27,354 --> 00:40:29,256
TARGET EFFECTS AND LIMITED PAY 

1033
00:40:29,256 --> 00:40:34,094
LOAD AND NEUTRALIZING OR 

1034
00:40:34,094 --> 00:40:36,430
DANGEROUS IMMUNOGENIC RESPONSES.

1035
00:40:36,430 --> 00:40:37,631
CARBON TECHNOLOGY SEEKS TO PUR 

1036
00:40:37,631 --> 00:40:40,134
PASS THE LIMITATION OF THE 

1037
00:40:40,134 --> 00:40:43,036
CURRENT STATE OF TECHNOLOGY BY 

1038
00:40:43,036 --> 00:40:45,639
TAKING ADVANTAGE OF THE 

1039
00:40:45,639 --> 00:40:48,408
UNEXPLORED LANDSCAPE AND THEY 

1040
00:40:48,408 --> 00:40:51,044
HOLD THE PROMISE OF GREATER -- 

1041
00:40:51,044 --> 00:40:51,712
SPECIFICITY AND GREATER GENETIC 

1042
00:40:51,712 --> 00:40:53,480
PAY LOAD.

1043
00:40:53,480 --> 00:40:56,250
IT BUILDS ON IMPROVED ASSETS OF 

1044
00:40:56,250 --> 00:41:01,922
GENE MODALITIES.

1045
00:41:01,922 --> 00:41:04,024
FOR EXAMPLE, WE CAN FIND WELL 

1046
00:41:04,024 --> 00:41:06,226
VETTED MANUFACTURING SAFETY 

1047
00:41:06,226 --> 00:41:09,229
PRACTICES TO OUR PLATFORM WHICH 

1048
00:41:09,229 --> 00:41:11,031
MAY INTERN DELIVER MEDICINES TO 

1049
00:41:11,031 --> 00:41:12,166
PATIENTS IN NEED.

1050
00:41:12,166 --> 00:41:14,434
AND IT HAS ITS ROOTS IN THE 

1051
00:41:14,434 --> 00:41:16,737
PIONEERING WORK OF OUR 

1052
00:41:16,737 --> 00:41:18,438
CO-FOUNDERS WHO YOU'LL HEAR TALK

1053
00:41:18,438 --> 00:41:24,444
SOON, DR. ENGLEHARDT AND DR. JAN

1054
00:41:24,444 --> 00:41:28,148
AND DELIVER A NORMAL COPY OF THE

1055
00:41:28,148 --> 00:41:29,516
GENE.

1056
00:41:29,516 --> 00:41:33,253
BEING ENGINEERED BY EVOLUTION 

1057
00:41:33,253 --> 00:41:37,324
FOR SPECIFICITY TO THE HUMAN 

1058
00:41:37,324 --> 00:41:40,127
LUNG IT STANDS POISED TO MAKE 

1059
00:41:40,127 --> 00:41:43,530
ITS MARK ON GENE THERAPY.

1060
00:41:43,530 --> 00:41:47,768
CARBON IS THE ONLY ONE THAT 

1061
00:41:47,768 --> 00:41:50,070
DELIVERS THE FULL LENGTH BUT 

1062
00:41:50,070 --> 00:41:52,940
BEING SUITED TO YOUR TARGET CAN 

1063
00:41:52,940 --> 00:41:54,341
COME WITH THE INABILITY TO USE 

1064
00:41:54,341 --> 00:41:59,079
SMALL PRODENTS.

1065
00:41:59,079 --> 00:42:05,719
 -- RODENTS.

1066
00:42:05,719 --> 00:42:10,057
AT CARBON OUR GOAL IS DELIVER 

1067
00:42:10,057 --> 00:42:18,565
AND THE LUNG IS A MARVELOUS 

1068
00:42:18,565 --> 00:42:20,100
DIVERSE ELEMENT AND THIS IS 

1069
00:42:20,100 --> 00:42:22,302
COMMON KNOWLEDGE TO THOSE IN THE

1070
00:42:22,302 --> 00:42:24,538
FILED BUT THE FIRST TIME I 

1071
00:42:24,538 --> 00:42:27,040
LOOKED AT THIS PICTURE FILLED ME

1072
00:42:27,040 --> 00:42:28,242
WITH WONDER AND ANGST, WHERE 

1073
00:42:28,242 --> 00:42:32,112
SHOULD WE DELIVER THE CELL OR 

1074
00:42:32,112 --> 00:42:36,316
HAVE AMELIORATED PROPERTIES?

1075
00:42:36,316 --> 00:42:38,685
CAN WE HAVE DELETERIOUS EFFECTS 

1076
00:42:38,685 --> 00:42:40,153
TO THE WRONG CELLS OR 

1077
00:42:40,153 --> 00:42:40,520
CONCENTRATIONS?

1078
00:42:40,520 --> 00:42:42,556
THESE ARE THE CHALLENGES AND 

1079
00:42:42,556 --> 00:42:45,192
CONSIDERATIONS WE MUST TRY TO 

1080
00:42:45,192 --> 00:42:46,960
ADDRESS AS WE CONTINUE TO BRING 

1081
00:42:46,960 --> 00:42:48,462
OUR MEDICINES TO CLIN INNING.

1082
00:42:48,462 --> 00:42:58,639
-- CLINIC.

1083
00:43:04,111 --> 00:43:07,881
SMALL RODENTS AND RATS ARE USED 

1084
00:43:07,881 --> 00:43:10,417
TO ASSESS EFFICACY.

1085
00:43:10,417 --> 00:43:18,425
AND THERE'S CHALLENGES BEHIND.

1086
00:43:18,425 --> 00:43:20,294
 NEB YOU'LLULIZING THE VECTOR 

1087
00:43:20,294 --> 00:43:21,795
AND HOW CAN WE MAKE SURE WE'RE 

1088
00:43:21,795 --> 00:43:25,299
MAKING THE SAFEST AND MOST 

1089
00:43:25,299 --> 00:43:25,899
EFFICACIOUS DRUGS POSSIBLE 

1090
00:43:25,899 --> 00:43:30,270
BEFORE MOVING INTO MORE COSTLY 

1091
00:43:30,270 --> 00:43:31,505
AND COMPLICATED MODELS SUCH AS 

1092
00:43:31,505 --> 00:43:33,974
LARGE ANIMAL STUDIES AND LOOK AT

1093
00:43:33,974 --> 00:43:34,608
EXISTING IN VITRO MODELS WE MAY 

1094
00:43:34,608 --> 00:43:44,785
LEVERAGE.

1095
00:43:46,553 --> 00:43:50,290
AND WE HAVE STEM LIKE CELLS WILL

1096
00:43:50,290 --> 00:43:51,024
DIFFERENTIATE INTO DIFFERENT 

1097
00:43:51,024 --> 00:43:53,126
CELL TYPE WHEN IT'S CLOSE TO AIR

1098
00:43:53,126 --> 00:43:53,860
ON ONE SIDE.

1099
00:43:53,860 --> 00:43:55,062
AS IT WOULD IN THE AIRWAY.

1100
00:43:55,062 --> 00:43:57,764
A WELL DOCUMENTED TECHNIQUE 

1101
00:43:57,764 --> 00:43:59,066
COMMONLY REFERRED TO AS 

1102
00:43:59,066 --> 00:44:03,036
INTERFACE OR ALI CULTURES, THIS 

1103
00:44:03,036 --> 00:44:09,910
COULD BE AP CAPTURE AND IDENTIFY

1104
00:44:09,910 --> 00:44:10,410
PROTEIN MARKERS.

1105
00:44:10,410 --> 00:44:13,981
THE IMAGES WERE PROVIDED BY 

1106
00:44:13,981 --> 00:44:17,384
PARTNERS AT THE CYSTIC FIBROSIS 

1107
00:44:17,384 --> 00:44:17,851
FOUNDATION.

1108
00:44:17,851 --> 00:44:21,955
AS AMAZING AS THIS IS IT 

1109
00:44:21,955 --> 00:44:24,224
PROVIDED ANOTHER MOMENT OF 

1110
00:44:24,224 --> 00:44:24,424
ANGST.

1111
00:44:24,424 --> 00:44:29,730
THROUGH THE CMC LENS 

1112
00:44:29,730 --> 00:44:32,299
CONSIDERATIONS AROSE IN THE 

1113
00:44:32,299 --> 00:44:32,532
PROJECT.

1114
00:44:32,532 --> 00:44:35,002
WELL THE WORK FLOWS BE ROBUST 

1115
00:44:35,002 --> 00:44:36,269
AND REPRODUCIBLE ENOUGH TO GUIDE

1116
00:44:36,269 --> 00:44:37,137
OUR PROCESSING?

1117
00:44:37,137 --> 00:44:38,972
CAN THEY BE ADAPTED TO PERFORM 

1118
00:44:38,972 --> 00:44:41,775
AT THE LEVEL REQUIRED TO SUPPORT

1119
00:44:41,775 --> 00:44:43,877
PHASE 1 TESTING?

1120
00:44:43,877 --> 00:44:48,915
CAN WE RELY ON SUPPLY OF 

1121
00:44:48,915 --> 00:44:51,051
RE-AGENTS SUCH AS TESTING GNP 

1122
00:44:51,051 --> 00:44:51,251
LOSS.

1123
00:44:51,251 --> 00:44:53,954
MOST THE WORK DONE IN THE SYSTEM

1124
00:44:53,954 --> 00:44:54,955
WAS WITH CELLS WHICH THEN GIVE 

1125
00:44:54,955 --> 00:44:56,256
RISE TO THE CONCERN OF 

1126
00:44:56,256 --> 00:45:02,362
VULNERABILITY AND AVAILABILITY.

1127
00:45:02,362 --> 00:45:08,335
HERE AGAIN I MUST GIVE CREDIT TO

1128
00:45:08,335 --> 00:45:13,407
A CO-FOUNDER AND THE CMTR CAN 

1129
00:45:13,407 --> 00:45:16,410
RECOVER FUNCTION IN THE CELL 

1130
00:45:16,410 --> 00:45:17,677
LINE DERIVED FROM THE 

1131
00:45:17,677 --> 00:45:18,412
PARTICIPANT LUNG.

1132
00:45:18,412 --> 00:45:20,047
AT CARBON WE HAVEN'T BEEN ABLE 

1133
00:45:20,047 --> 00:45:22,082
TO REPRODUCE THIS AND THIS 

1134
00:45:22,082 --> 00:45:24,384
BRINGS ME TO THE NEXT ATTRIBUTE.

1135
00:45:24,384 --> 00:45:28,321
THE ABILITY TO RECAPITULATE LUNG

1136
00:45:28,321 --> 00:45:31,058
CELL ACTIVITY.

1137
00:45:31,058 --> 00:45:36,930
PATIENTS WITH CF LACK FUNCTIONAL

1138
00:45:36,930 --> 00:45:37,531
CTR.

1139
00:45:37,531 --> 00:45:38,965
CONSEQUENTLY THERE'S NO EFFECT 

1140
00:45:38,965 --> 00:45:39,833
ON PATIENT CELLS.

1141
00:45:39,833 --> 00:45:42,702
THIS IS FUNCTION AND RECOVERY IS

1142
00:45:42,702 --> 00:45:43,970
WELL DOCUMENTED IN THE ALL RIGHT

1143
00:45:43,970 --> 00:45:45,472
AND CAN BE MODELLED BY ADAPTING 

1144
00:45:45,472 --> 00:45:49,943
THE TECHNIQUE TO THE DEVICE AND 

1145
00:45:49,943 --> 00:45:54,581
THE NAME FOR THE CHAMBER.

1146
00:45:54,581 --> 00:45:56,616
IT ALLOWS US TO DIRECT IONS IN 

1147
00:45:56,616 --> 00:45:58,218
THE PRESENCE OR ABSENCE OF THE 

1148
00:45:58,218 --> 00:45:59,052
THERAPEUTIC.

1149
00:45:59,052 --> 00:46:00,554
THE ABILITY TO ACCOMPLISH THIS 

1150
00:46:00,554 --> 00:46:01,822
USING ESTABLISHED HUMAN CELL 

1151
00:46:01,822 --> 00:46:04,324
LINE SHOULD NOT BE UNDER STATED.

1152
00:46:04,324 --> 00:46:05,692
THIS ALLOWS US TO CREATE LINES 

1153
00:46:05,692 --> 00:46:08,328
AND CELL BANKS AS NEEDED TO 

1154
00:46:08,328 --> 00:46:10,597
IMPROVE ROBUST REPRODUCIBLE 

1155
00:46:10,597 --> 00:46:12,699
RESULTS.

1156
00:46:12,699 --> 00:46:15,435
FORTUNATELY, GENETICALLY MODE 

1157
00:46:15,435 --> 00:46:19,940
MODIFIED CELL LINES TO 

1158
00:46:19,940 --> 00:46:20,640
CAPITULATE THE TARGET.

1159
00:46:20,640 --> 00:46:26,613
AT CARBON WE HAVE TAKEN THE NEXT

1160
00:46:26,613 --> 00:46:30,317
STEP AND SHOWN AND THE CELL LINE

1161
00:46:30,317 --> 00:46:35,055
IS RESPONSIVE TO STANDARD OF 

1162
00:46:35,055 --> 00:46:37,724
CARE THERAPEUTICS AND I'LL WALK 

1163
00:46:37,724 --> 00:46:39,459
THROUGH THE DATA FOR THE SAKE OF

1164
00:46:39,459 --> 00:46:41,461
THOSE NOT DOMAIN EXPERTS.

1165
00:46:41,461 --> 00:46:43,964
THE Y AXIS MEASURES THE 

1166
00:46:43,964 --> 00:46:47,033
MAGNITUDE OF THE SHORT CIRCUIT 

1167
00:46:47,033 --> 00:46:49,870
CURRENT SEPARATE THE BY LAYER OF

1168
00:46:49,870 --> 00:46:52,405
LUNG CELLS.

1169
00:46:52,405 --> 00:46:55,041
THE GRAY LINE REPRESENT 

1170
00:46:55,041 --> 00:46:55,942
REPLICATES OF CELLS.

1171
00:46:55,942 --> 00:46:57,811
WHEN NO CELLS ARE REPRESENTED 

1172
00:46:57,811 --> 00:47:01,314
WITH THE ACTIVITY THEY DO NOT 

1173
00:47:01,314 --> 00:47:01,515
STOP.

1174
00:47:01,515 --> 00:47:02,782
THE BLUE LINES REPRESENT THE 

1175
00:47:02,782 --> 00:47:05,485
CELL LINE TREATED WITH APPROVED 

1176
00:47:05,485 --> 00:47:05,785
DRUGS.

1177
00:47:05,785 --> 00:47:07,053
THOSE CELLS CLEARLY RESPOND TO 

1178
00:47:07,053 --> 00:47:09,489
THE STIMULUS AND RETURN TO 

1179
00:47:09,489 --> 00:47:11,057
BASELINE WHEN SPECIFIC 

1180
00:47:11,057 --> 00:47:17,564
INHIBITERS RETURN.

1181
00:47:17,564 --> 00:47:19,366
I'M HAPPY TO REPORT WE AT CARBON

1182
00:47:19,366 --> 00:47:22,536
CAN NOW GENERATE OUR OWN GENETIC

1183
00:47:22,536 --> 00:47:26,840
MEDICINE FOR CYSTIC FIBROSIS 

1184
00:47:26,840 --> 00:47:30,577
LEVERAGING THE TECHNIQUE WE 

1185
00:47:30,577 --> 00:47:32,913
DEMONSTRATED OUR VECTOR CAN 

1186
00:47:32,913 --> 00:47:35,982
TARGET A VARIETY OF AIRWAY CELL 

1187
00:47:35,982 --> 00:47:37,584
POPULATIONS RELEVANT FOR DISEASE

1188
00:47:37,584 --> 00:47:40,387
POPULATION AND DEMONSTRATE THE 

1189
00:47:40,387 --> 00:47:50,330
RECOVERY OF ACTIVITY COMPARABLE.

1190
00:47:50,330 --> 00:47:52,666
NATURE HAS MADE THE HUMAN CAPSID

1191
00:47:52,666 --> 00:47:55,035
HIGHLY SPECIFIC TO THE HUMAN AUR

1192
00:47:55,035 --> 00:47:55,202
WAY.

1193
00:47:55,202 --> 00:47:57,437
AN IDEAL AND UNPRECEDENTED 

1194
00:47:57,437 --> 00:47:59,072
CANDIDATE FOR A GENE THERAPY TO 

1195
00:47:59,072 --> 00:48:04,811
TREAT LUNG CASES AND THIS 

1196
00:48:04,811 --> 00:48:05,812
ESTABLISHES SAFETY AND EFFICACY 

1197
00:48:05,812 --> 00:48:08,415
IN NON-HUMAN ANIMAL MODELS.

1198
00:48:08,415 --> 00:48:11,651
WE CAN LEVERAGE A WELL VALIDATED

1199
00:48:11,651 --> 00:48:12,419
IN VITRO SYSTEM TO DEMONSTRATE 

1200
00:48:12,419 --> 00:48:13,820
DRUG EFFICACY.

1201
00:48:13,820 --> 00:48:16,022
A MAJOR CHALLENGE ON OUR PATH TO

1202
00:48:16,022 --> 00:48:18,258
IND AND ULTIMATELY TO THE CLINIC

1203
00:48:18,258 --> 00:48:20,427
IS ESTABLISHING A PSEUDO TOX KOL

1204
00:48:20,427 --> 00:48:22,562
BY MODEL TO ENSURE THE SAFETY OF

1205
00:48:22,562 --> 00:48:24,965
OUR DRUGS IN PATIENT.

1206
00:48:24,965 --> 00:48:26,433
TO ACCOMPLISH THIS WE'LL ATTEMPT

1207
00:48:26,433 --> 00:48:36,910
TO ADMINISTER THE CTG001 TO 

1208
00:48:46,886 --> 00:48:47,687
NONHUMAN PRIMATES AND I'D LIKE 

1209
00:48:47,687 --> 00:48:55,028
TO TAKE A MOMENT TO THANK OUR 

1210
00:48:55,028 --> 00:48:58,064
PARTNERS.

1211
00:48:58,064 --> 00:48:59,065
>> THANK YOU.

1212
00:48:59,065 --> 00:49:02,435
NEXT WE'LL HAVE THE PRESENTATION

1213
00:49:02,435 --> 00:49:12,746
FROM ERIN, PLEASE.

1214
00:49:27,093 --> 00:49:31,131
>> I'M ERIN HENNESSEY AT
BOSTON 

1215
00:49:31,131 --> 00:49:32,299
UNIVERSITY AND WE'RE THINKING 

1216
00:49:32,299 --> 00:49:42,108
HOW TO BEST SERVE PATIENTS WITH 

1217
00:49:42,108 --> 00:49:43,043
UNMET NEEDS AND RARE DISEASE 

1218
00:49:43,043 --> 00:49:45,512
POPULATIONS WHERE WE CANNOT DO 

1219
00:49:45,512 --> 00:49:46,346
POWERED CLINICAL TRIALS.

1220
00:49:46,346 --> 00:49:48,982
WE THINK ABOUT INHERENTLY 

1221
00:49:48,982 --> 00:49:50,917
VULNERABLE POPULATIONS LIKE KIDS

1222
00:49:50,917 --> 00:49:52,052
WITH PEDIATRIC DISEASES AND 

1223
00:49:52,052 --> 00:49:53,653
THINK ABOUT PATIENTS WITH SEVERE

1224
00:49:53,653 --> 00:49:56,523
DISEASE AND SPECIFICALLY WHEN 

1225
00:49:56,523 --> 00:49:58,124
THINKING ABOUT LUNG DISEASE, 

1226
00:49:58,124 --> 00:49:59,993
PATIENTS FOR WHOM A BIOPSY OR 

1227
00:49:59,993 --> 00:50:02,662
TISSUE COLLECTION WOULD BE 

1228
00:50:02,662 --> 00:50:03,229
HARMFUL OR DANGEROUS.

1229
00:50:03,229 --> 00:50:05,131
THE QUESTION REMAINS IS HOW CAN 

1230
00:50:05,131 --> 00:50:07,067
WE STUDY DISEASES IN THE 

1231
00:50:07,067 --> 00:50:14,474
VULNERABLE POPULATIONS?

1232
00:50:14,474 --> 00:50:21,114
WE LOOK TO INDUCED HEMATOPOIETIC

1233
00:50:21,114 --> 00:50:24,484
STEM CELLS OR IPSCs AND TAKE THE

1234
00:50:24,484 --> 00:50:26,486
FROM A PUNCH AND TURN BACK THE 

1235
00:50:26,486 --> 00:50:28,588
CLOCK TO REPROGRAM THEM TO A 

1236
00:50:28,588 --> 00:50:30,757
STATE MEANING THEY THEN HAVE THE

1237
00:50:30,757 --> 00:50:32,158
POTENTIAL TO BECOME PRETTY MUCH 

1238
00:50:32,158 --> 00:50:38,498
ANY CELL TYPE YOU CAN THINK OF.

1239
00:50:38,498 --> 00:50:40,133
AND WHERE WE ADD A TIME SERIES 

1240
00:50:40,133 --> 00:50:41,735
OF GROWTH FACTORS WE CAN LED 

1241
00:50:41,735 --> 00:50:50,009
THEM TO BE OUR CELLS OF INTEREST

1242
00:50:50,009 --> 00:50:52,545
AND WE CAN EASILY TAKE CELLS 

1243
00:50:52,545 --> 00:50:55,048
FROM ANY DONOR AND TURN THEM 

1244
00:50:55,048 --> 00:51:05,558
INTO IPSC CELLS AND CAN TURN 

1245
00:51:18,271 --> 00:51:20,140
BACK TIME TO A TIME BEFORE 

1246
00:51:20,140 --> 00:51:21,307
DISEASE PROGRESS AND CAN HONE IN

1247
00:51:21,307 --> 00:51:23,376
AND IDENTIFY WHAT AND WHEN AND 

1248
00:51:23,376 --> 00:51:29,048
WHERE THINGS WENT WRONG.

1249
00:51:29,048 --> 00:51:35,021
THERE'S VALUABLE INFORMATION 

1250
00:51:35,021 --> 00:51:36,022
STORES IN THE CELLS.

1251
00:51:36,022 --> 00:51:41,027
I'LL USE THIS TO STUDY DISEASE 

1252
00:51:41,027 --> 00:51:43,096
WITH UNMET NEEDS AND CONCENTRATE

1253
00:51:43,096 --> 00:51:46,499
ON CHLID BECAUSE THAT'S WHAT MY 

1254
00:51:46,499 --> 00:51:49,903
Ph.D. REASON IS ON BUT I ALSO 

1255
00:51:49,903 --> 00:51:53,640
THINK IT FITS THE BILL AND IS A 

1256
00:51:53,640 --> 00:51:55,542
RARE DISEASE THAT AFFECTS 1 IN 

1257
00:51:55,542 --> 00:51:58,711
10,000 BIRTHS IN THE U.S. AND 

1258
00:51:58,711 --> 00:51:59,846
AFFECTS A PEDIATRIC POPULATION.

1259
00:51:59,846 --> 00:52:02,248
FROM THE MAJORITY OF CASES ARE 

1260
00:52:02,248 --> 00:52:07,487
CAUSED BY MUTATIONS IN THE EBCA3

1261
00:52:07,487 --> 00:52:11,925
GENE WHOSE ASSOCIATED PROTEIN IS

1262
00:52:11,925 --> 00:52:12,992
INSTRUMENTAL IN THE PULMONARY 

1263
00:52:12,992 --> 00:52:16,930
SURFACTANT SO IT LEADS TO A 

1264
00:52:16,930 --> 00:52:20,366
SURFACTANT SUFFICIENCY THOUGHT 

1265
00:52:20,366 --> 00:52:20,934
TO BE DRIVING A DISEASE AND 

1266
00:52:20,934 --> 00:52:21,601
KNOWN DRIVER OF THE DISEASE AND 

1267
00:52:21,601 --> 00:52:26,506
HAVE EVIDENCE TO SUGGEST THERE'S

1268
00:52:26,506 --> 00:52:37,050
MORE TO IT THAN THAT I'M SHOWING

1269
00:52:40,420 --> 00:52:45,492
HISTOPATHOLOGY AND WE SEE THESE 

1270
00:52:45,492 --> 00:52:50,730
ORGANELLES AND THEY'RE 

1271
00:52:50,730 --> 00:52:52,365
SURFACTANT PRODUCTION TAKES 

1272
00:52:52,365 --> 00:52:55,235
PLACE AND HAVE MORPHOLOGY OF A 

1273
00:52:55,235 --> 00:52:57,070
FINGERPRINT OR RINGS OF I ATREE.

1274
00:52:57,070 --> 00:52:58,505
ON THE RIGHT I'M SHOWING 

1275
00:52:58,505 --> 00:53:01,975
PATHOLOGY FROM A PATIENT WITH 

1276
00:53:01,975 --> 00:53:11,951
ABC3 MUTATION AND THEAL -- 

1277
00:53:11,951 --> 00:53:20,727
ALVEOLAR SPACE HAVE LOST THE AND

1278
00:53:20,727 --> 00:53:21,628
IT'S ASSOCIATED WITH HIGH 

1279
00:53:21,628 --> 00:53:23,029
MORBIDITY AND MORTALITY BECAUSE 

1280
00:53:23,029 --> 00:53:24,664
THERE'S NO REAL TREATMENTS THAT 

1281
00:53:24,664 --> 00:53:25,865
ADDRESS IT.

1282
00:53:25,865 --> 00:53:28,568
CURRENTLY IT'S MANAGED WITH 

1283
00:53:28,568 --> 00:53:31,037
THINGS LIKE IMMUNOSUPPRESSANTS 

1284
00:53:31,037 --> 00:53:36,075
AND HIYDROXYCHLOROQUINE AND 

1285
00:53:36,075 --> 00:53:37,944
THERE'S AN URGENT NEED TO STUDY 

1286
00:53:37,944 --> 00:53:39,045
THE DISEASE FOR TREATMENT.

1287
00:53:39,045 --> 00:53:41,781
WE COULD USE PRIMARY PATIENT 

1288
00:53:41,781 --> 00:53:47,153
CELLS BUT THEY ARE DIFFICULT TO 

1289
00:53:47,153 --> 00:53:50,924
ACQUIRE AND WE WANT TO STUDY THE

1290
00:53:50,924 --> 00:53:51,991
INCEPTION OF THE DISEASE.

1291
00:53:51,991 --> 00:53:53,693
WHEN WE GET PRIMARY CELLS 

1292
00:53:53,693 --> 00:53:56,763
THEY'RE OFTEN FROM A DONOR WHOSE

1293
00:53:56,763 --> 00:53:58,898
DISEASE HAS ALREADY PROGRESSED 

1294
00:53:58,898 --> 00:54:00,833
SO YOU'RE NOT ABLE TO STUDY THE 

1295
00:54:00,833 --> 00:54:03,036
START OF THE DISEASE.

1296
00:54:03,036 --> 00:54:06,272
FOR THESE REASONS WE CHOOSE TO 

1297
00:54:06,272 --> 00:54:09,475
USE IPSC CELLS AND HOW DO WE GET

1298
00:54:09,475 --> 00:54:13,646
TO I22s.

1299
00:54:13,646 --> 00:54:19,018
HERE'S IMAGES OF THE MUTATION.

1300
00:54:19,018 --> 00:54:25,325
THE SCAN ON THE LEFT SHOWS 

1301
00:54:25,325 --> 00:54:27,360
PULMONARY REMODELLING AND 

1302
00:54:27,360 --> 00:54:33,733
THICKENING AND THE BLACK ARROW 

1303
00:54:33,733 --> 00:54:33,967
POINTS.

1304
00:54:33,967 --> 00:54:35,902
WE TOOK A BLOOD DRAW AND ISOLATE

1305
00:54:35,902 --> 00:54:39,772
THE CELLS WE NEEDED ANDRY 

1306
00:54:39,772 --> 00:54:43,142
CULTURED TO BECOME IPSC AND 

1307
00:54:43,142 --> 00:54:45,645
HERE'S THE CELLS.

1308
00:54:45,645 --> 00:54:49,449
WE KNOW THEY'RE IPSCs.

1309
00:54:49,449 --> 00:54:52,685
AND FOR THE PROCESS OF 

1310
00:54:52,685 --> 00:54:53,987
DIFFERENTIATION WE TURN THEM 

1311
00:54:53,987 --> 00:55:00,226
INTO AIA2s AND THE RED IS A 

1312
00:55:00,226 --> 00:55:05,431
REPORTER KNOCKED IN AND HAS 

1313
00:55:05,431 --> 00:55:07,300
ACCESS LIKE A WAY TO PURIFY THE 

1314
00:55:07,300 --> 00:55:10,803
CELLS AND WE KNOW THEY'RE 

1315
00:55:10,803 --> 00:55:17,677
SCOMBREFG THE PROGRAM-- EXPRESS 

1316
00:55:17,677 --> 00:55:20,446
HOW DO WE STUDY THE MUTATION?

1317
00:55:20,446 --> 00:55:23,416
THE GRAD STUDENT BEFORE ME 

1318
00:55:23,416 --> 00:55:24,651
APPROACHED THE QUESTION BY 

1319
00:55:24,651 --> 00:55:28,955
FUSING GFP THROUGH THE 

1320
00:55:28,955 --> 00:55:30,757
ENDOGENOUS LOCUS TO RAISE THE 

1321
00:55:30,757 --> 00:55:31,824
PROTEIN.

1322
00:55:31,824 --> 00:55:34,027
BEFORE SHE DID THIS IN ANY CELLS

1323
00:55:34,027 --> 00:55:36,029
FROM THE DONORS HE FIRST 

1324
00:55:36,029 --> 00:55:37,163
PERFORMED THE EXPERIMENT IN THE 

1325
00:55:37,163 --> 00:55:38,931
CELL LINES WE HAVE IN OUR CENTER

1326
00:55:38,931 --> 00:55:43,202
FROM A HEALTHY DONOR.

1327
00:55:43,202 --> 00:55:46,639
BECAUSE ABCA3 GOES TO THE 

1328
00:55:46,639 --> 00:55:50,176
LIMITING MEMBRANE WHAT WE EXPECT

1329
00:55:50,176 --> 00:55:53,813
TO SEE ARE GREEN CIRCLES AND 

1330
00:55:53,813 --> 00:55:55,014
WHEN WE DIFFERENTIATE THE CELLS 

1331
00:55:55,014 --> 00:55:56,916
AND LOOK AT THEM THAT'S WHAT WE 

1332
00:55:56,916 --> 00:55:59,819
SEE.

1333
00:55:59,819 --> 00:56:02,789
HERE I'M SHOWING YOU WHAT WE 

1334
00:56:02,789 --> 00:56:11,764
CALL AN ALVEOLAR SPHERE THAT 

1335
00:56:11,764 --> 00:56:12,932
HAVE GROWN TOGETHER AND WHAT'S 

1336
00:56:12,932 --> 00:56:15,234
COOL IS THEY'RE POLARIZED LIKE A

1337
00:56:15,234 --> 00:56:18,204
LUNG AND WE KNOW THE CELLS ARE 

1338
00:56:18,204 --> 00:56:21,974
ACTUALLY PUMPING SURFACTANT INTO

1339
00:56:21,974 --> 00:56:23,543
THE MIDDLE OF THE OPEN SPACE 

1340
00:56:23,543 --> 00:56:25,144
ACTING LIKE THE LUNG LUMEN.

1341
00:56:25,144 --> 00:56:26,979
BECAUSE IT'S FROM A HEALTHY 

1342
00:56:26,979 --> 00:56:30,683
DONOR WE SEE NICE JUICY CIRCLES 

1343
00:56:30,683 --> 00:56:32,385
WORKING HARD TO MAKE THE 

1344
00:56:32,385 --> 00:56:32,685
SURFACTANT.

1345
00:56:32,685 --> 00:56:37,924
HOW MIGHT THE IMAGE CHANGE IF 

1346
00:56:37,924 --> 00:56:40,727
THE IG2s STUDY THIS AND WE TOOK 

1347
00:56:40,727 --> 00:56:43,029
OUR HEALTHY DONOR LINE AND 

1348
00:56:43,029 --> 00:56:48,101
FUSION PROTEIN AND KNOCKED IN A 

1349
00:56:48,101 --> 00:56:49,135
HOMOZYGOUS WAY THE MUTATION.

1350
00:56:49,135 --> 00:56:54,874
IN THE MIDDLE WE SHOW IG2s WITH 

1351
00:56:54,874 --> 00:56:56,075
A TYPE 1 MUTATION AND THEY'RE 

1352
00:56:56,075 --> 00:56:57,744
KNOWN TO DISRUPT THE PRACTICING 

1353
00:56:57,744 --> 00:56:59,045
OF THE PROTEIN.

1354
00:56:59,045 --> 00:57:01,681
AS EXPECTED HERE, THE ABC IS 

1355
00:57:01,681 --> 00:57:03,916
MORE DIFFUSED AND NOT GETTING 

1356
00:57:03,916 --> 00:57:06,085
PROPERLY TRAFFICKED.

1357
00:57:06,085 --> 00:57:09,422
ON THE RIGHT I'M SHOWING CELLS 

1358
00:57:09,422 --> 00:57:16,462
WITH HOMOZYGOUS MUTATION WITH 

1359
00:57:16,462 --> 00:57:19,799
THE FUNCTION OF THE ABCA3 

1360
00:57:19,799 --> 00:57:25,138
PROTEIN AND WE SEE ABC3 LOCALIZE

1361
00:57:25,138 --> 00:57:30,977
TO NORC BODIES AND THEY'RE 

1362
00:57:30,977 --> 00:57:33,212
SMALLER AND MORE FROM THE 

1363
00:57:33,212 --> 00:57:34,747
EXPERIMENTS FROM A HEALTHY DONOR

1364
00:57:34,747 --> 00:57:38,251
WE CAN DO THE SAME TYPES OF 

1365
00:57:38,251 --> 00:57:40,686
EXPERIMENTS FROM OUR ILD DONOR.

1366
00:57:40,686 --> 00:57:44,123
WHAT WE DO IS CORRECT THE ABC3 

1367
00:57:44,123 --> 00:57:46,759
MUTATION THEY HAVE, WHICH IS 

1368
00:57:46,759 --> 00:57:51,297
EASIER TO DO IN A PETRI DISH 

1369
00:57:51,297 --> 00:57:53,366
THAN A PERSON AND WE END UP WITH

1370
00:57:53,366 --> 00:57:55,835
THE WILD TYPE CONTROL WITH ALL 

1371
00:57:55,835 --> 00:57:57,270
THE DONOR'S INFORMATION WITH 

1372
00:57:57,270 --> 00:58:01,808
ONLY ONE ABCA3 VARIANT CHANGE.

1373
00:58:01,808 --> 00:58:04,410
IN ADDITION TO MEASURING ANOTHER

1374
00:58:04,410 --> 00:58:05,978
BODY SIZE AND SURFACTANT 

1375
00:58:05,978 --> 00:58:08,281
PRODUCTION WE HAVE UNBIASSED 

1376
00:58:08,281 --> 00:58:09,348
WAYS TO ASK THE CELLS WHAT'S 

1377
00:58:09,348 --> 00:58:11,684
GOING ON AND WE DO THIS THROUGH 

1378
00:58:11,684 --> 00:58:12,318
OUR SEQUENCING.

1379
00:58:12,318 --> 00:58:14,554
I'M SHOWING DATA FROM THAT HERE.

1380
00:58:14,554 --> 00:58:17,657
THIS IS INFORMATION THAT YOU 

1381
00:58:17,657 --> 00:58:18,624
MIGHT FIND IN THE DISEASE AND A 

1382
00:58:18,624 --> 00:58:21,060
WAY TO LOOK AT THE PATHWAYS THAT

1383
00:58:21,060 --> 00:58:24,130
HAVE BEEN DISRUPTED OR PERTURBED

1384
00:58:24,130 --> 00:58:25,698
AND FURTHER DRIVING THE DISEASE.

1385
00:58:25,698 --> 00:58:29,902
SO SOME OF THESE PATHWAYS PLAY 

1386
00:58:29,902 --> 00:58:31,571
BE FIXABLE, IE DRUGGABLE.

1387
00:58:31,571 --> 00:58:32,738
FIRST I WANT TO DRAW YOUR 

1388
00:58:32,738 --> 00:58:34,807
ATTENTION TO THE UPREGULATED 

1389
00:58:34,807 --> 00:58:38,978
PATHWAYS IN THE MUTANT CELLS.

1390
00:58:38,978 --> 00:58:43,049
HERE WE'RE SEEING THINGS LIKE 

1391
00:58:43,049 --> 00:58:52,491
INTERFERON RESPONSES, APOPTOSIS 

1392
00:58:52,491 --> 00:58:54,927
AND THERE MAY BE MORE HAPPENING 

1393
00:58:54,927 --> 00:58:57,630
THAN JUST THE SURFACTANT 

1394
00:58:57,630 --> 00:58:58,664
DEFICIENCY ALONE.

1395
00:58:58,664 --> 00:59:01,100
WE THINK THE UP REGULATION OF 

1396
00:59:01,100 --> 00:59:03,903
INFLAMMATORY PATHWAYS MAY BE THE

1397
00:59:03,903 --> 00:59:06,806
RESULT OF ONE OF TWO THINGS, THE

1398
00:59:06,806 --> 00:59:08,708
MUTANT PROTEIN IS ACTING IN A 

1399
00:59:08,708 --> 00:59:11,878
WAY TO CAUSE THE REGULATIONS IN 

1400
00:59:11,878 --> 00:59:22,088
A MECHANISM OR B, THE 

1401
00:59:22,088 --> 00:59:22,855
INFLAMMA 

1402
00:59:22,855 --> 00:59:24,156
INFLAMMATION ARE UNHAPPY CELLS 

1403
00:59:24,156 --> 00:59:27,293
AND SOMETHING WE'RE WORKING TO 

1404
00:59:27,293 --> 00:59:34,834
UNDERSTAND.

1405
00:59:34,834 --> 00:59:37,236
SOMETHING WE KNOW ABOUT THE A2 

1406
00:59:37,236 --> 00:59:40,172
CELLS THE MAJOR PRODUCTION IS 

1407
00:59:40,172 --> 00:59:41,874
THE SURFACTANT PRODUCTION AND 

1408
00:59:41,874 --> 00:59:44,477
PLAY A ROLE AS A PROGENITOR CELL

1409
00:59:44,477 --> 00:59:49,649
AND HOW TO REPOPULATE THE 

1410
00:59:49,649 --> 00:59:50,182
ALVEOLI.

1411
00:59:50,182 --> 00:59:53,352
AND THE PATHWAYS INDICATE TO US 

1412
00:59:53,352 --> 00:59:54,887
THAT WITH THE DISEASE THE CELLS 

1413
00:59:54,887 --> 00:59:56,989
MAY BE LOSING SOME OF THEIR 

1414
00:59:56,989 --> 01:00:00,359
PROBETWEEN FOR POTENTIAL.

1415
01:00:00,359 --> 01:00:01,294
-- PROGENITOR POTENTIAL.

1416
01:00:01,294 --> 01:00:05,731
THE TWO CONCEPTS WE OFTEN TALK 

1417
01:00:05,731 --> 01:00:07,033
ABOUT IN DEVELOPMENTAL BIOLOGY 

1418
01:00:07,033 --> 01:00:09,502
IS NECESSARY AND SUFFICIENT.

1419
01:00:09,502 --> 01:00:11,570
IF AN EVENT IS NECESSARY WE KNOW

1420
01:00:11,570 --> 01:00:15,241
IT IS REQUIRED IN ORDER FOR THE 

1421
01:00:15,241 --> 01:00:15,675
OUTCOME TO OCCUR.

1422
01:00:15,675 --> 01:00:17,810
FOR EXAMPLE, WITH KNOW IF WE 

1423
01:00:17,810 --> 01:00:25,351
TAKE IG2s FROM A DONOR FROM THE 

1424
01:00:25,351 --> 01:00:26,919
ABCA3 MUTATION WITH ONLY ONE 

1425
01:00:26,919 --> 01:00:28,220
NUCLEOTIDE IS ENOUGH TO RESTORE 

1426
01:00:28,220 --> 01:00:31,524
THE FUNCTION OF THE CELLS.

1427
01:00:31,524 --> 01:00:33,259
 IN OTHER WORDS EVEN WITH ALL 

1428
01:00:33,259 --> 01:00:34,660
THE INFORMATION THE PATIENT HAS 

1429
01:00:34,660 --> 01:00:40,199
THERE'S ONE MUTATION NECESSARY 

1430
01:00:40,199 --> 01:00:43,035
TO CAUSE ALL -- AND THE EVENT 

1431
01:00:43,035 --> 01:00:46,038
ALONE IS ENOUGH TO GUARANTEE THE

1432
01:00:46,038 --> 01:00:46,272
OUTCOME.

1433
01:00:46,272 --> 01:00:50,776
FOR EXAMPLE, IF WE TAKE OUR IN 

1434
01:00:50,776 --> 01:00:55,014
HOUSE IPSC LINE FROM OUR HEALTHY

1435
01:00:55,014 --> 01:01:00,820
DONOR WITH ALL THEIR GENETIC 

1436
01:01:00,820 --> 01:01:03,022
INFORMATION WE HAVE END UP WITH 

1437
01:01:03,022 --> 01:01:04,724
REDUCED SURFACTANT PRODUCTION 

1438
01:01:04,724 --> 01:01:05,558
INDICATING ONE MUTATION IS 

1439
01:01:05,558 --> 01:01:09,028
SUFFICIENT TO CAUSE THE DISEASE.

1440
01:01:09,028 --> 01:01:11,030
SO IN DOING DISEASE RESEARCH 

1441
01:01:11,030 --> 01:01:16,602
THIS IS A POWERFUL INFORMATION 

1442
01:01:16,602 --> 01:01:18,637
TO HAVE AND ABLE TO UNDERSTAND 

1443
01:01:18,637 --> 01:01:23,376
IT BECAUSE YOU HAVE OUR IPSC 

1444
01:01:23,376 --> 01:01:23,976
CELL MODEL.

1445
01:01:23,976 --> 01:01:26,145
BRIEFLY I'LL TALK ABOUT THE MEAT

1446
01:01:26,145 --> 01:01:32,251
OF MY Ph.D. PROJECT AND WANT TO 

1447
01:01:32,251 --> 01:01:36,355
DEVELOP A THERAPEUTIC FOR ABCA3 

1448
01:01:36,355 --> 01:01:37,757
AND THINK GENE THERAPY COULD BE 

1449
01:01:37,757 --> 01:01:39,058
A GOOD AVENUE TO EXPLORE.

1450
01:01:39,058 --> 01:01:41,427
I KNOW I REFERENCED THE FACT WE 

1451
01:01:41,427 --> 01:01:42,962
THINK IT'S A MUTATION FOR GAIN 

1452
01:01:42,962 --> 01:01:44,730
OF FUNCTION BUT THERE'S PLENTY 

1453
01:01:44,730 --> 01:01:46,432
OF MUTATIONS WE KNOW OF AND 

1454
01:01:46,432 --> 01:01:47,600
DELETIONS AND PREMATURE STOPS 

1455
01:01:47,600 --> 01:01:49,735
THAT ARE LOSS OF FUNCTION.

1456
01:01:49,735 --> 01:01:56,308
SO ONE OF THE PILLARS OF A GOOD 

1457
01:01:56,308 --> 01:01:57,009
FWE

1458
01:01:57,009 --> 01:01:58,444
GENE THERAPY IS EFFICIENT AT 

1459
01:01:58,444 --> 01:01:59,512
GENE TRANSFER.

1460
01:01:59,512 --> 01:02:00,713
I'VE BEEN EXPLORING VIRAL 

1461
01:02:00,713 --> 01:02:04,917
OPTIONS FOR GENE THERAPY.

1462
01:02:04,917 --> 01:02:07,019
I'M SHOWING THE ACTUAL 

1463
01:02:07,019 --> 01:02:07,920
TECHNIQUE.

1464
01:02:07,920 --> 01:02:09,455
THE CELLS TYPICALLY GROW -- LIKE

1465
01:02:09,455 --> 01:02:13,292
I SHOWED BEFORE IN STEROIDS IN 

1466
01:02:13,292 --> 01:02:17,029
3-D MAKER GELS BUT CAN GROW THEM

1467
01:02:17,029 --> 01:02:19,598
IN AIR LIQUID INTERFACE, ALI, 

1468
01:02:19,598 --> 01:02:23,035
WHERE THEY'RE IN A MONOLAYER ON 

1469
01:02:23,035 --> 01:02:27,006
A TRANS MEMBRANE SO THE BASAL 

1470
01:02:27,006 --> 01:02:30,109
LATERAL SIDE ARE EXPOSED AND THE

1471
01:02:30,109 --> 01:02:31,444
OTHER EXPOSED TO AIR.

1472
01:02:31,444 --> 01:02:35,014
AND THEY MAY EXIST IN VIVO.

1473
01:02:35,014 --> 01:02:38,617
THERE'S A MORE RELEVANT CULTURE 

1474
01:02:38,617 --> 01:02:39,819
SYSTEM.

1475
01:02:39,819 --> 01:02:43,722
TO TEST VIRAL EFFICIENCIES AND 

1476
01:02:43,722 --> 01:02:47,026
IN TERMS OF VIRUSES I TRIED WERE

1477
01:02:47,026 --> 01:02:48,994
LENTI VIRUSES IN COLLABORATION 

1478
01:02:48,994 --> 01:02:51,897
WITH A GROUP AT UNIVERSITY OF 

1479
01:02:51,897 --> 01:02:52,098
IOWA.

1480
01:02:52,098 --> 01:02:55,034
WE TRIED A FEW PSEUDO TYPES OF 

1481
01:02:55,034 --> 01:02:57,036
LENTI VIRUSES WITH THE HOPES 

1482
01:02:57,036 --> 01:02:57,870
SOME WOULD BE MORE EFFICIENCY 

1483
01:02:57,870 --> 01:02:59,238
THAN OTHERS.

1484
01:02:59,238 --> 01:03:05,978
AND THE GENE I'M TRANSFERRING 

1485
01:03:05,978 --> 01:03:08,781
ARE GFP.

1486
01:03:08,781 --> 01:03:11,050
WE FOUND SOME PSEUDO KINDS BUT 

1487
01:03:11,050 --> 01:03:12,551
VERY LITTLE.

1488
01:03:12,551 --> 01:03:17,389
LOOKING TO THE GRAPH TO THE 

1489
01:03:17,389 --> 01:03:18,891
RIGHT AND THE GENE TRANSFER WAS 

1490
01:03:18,891 --> 01:03:19,792
LESS THAN 5%.

1491
01:03:19,792 --> 01:03:22,361
THOUGH WE DON'T KNOW EXACTLY HOW

1492
01:03:22,361 --> 01:03:23,629
EFFICIENT A VIRUS NEEDS TO BE IN

1493
01:03:23,629 --> 01:03:34,173
ORDER FOR THE GENE THERAPY AND 

1494
01:03:35,107 --> 01:03:37,176
I'M EXPLORING GENE THERAPY IN 

1495
01:03:37,176 --> 01:03:41,847
THE EXPERIMENT I'M TESTING AN 

1496
01:03:41,847 --> 01:03:48,154
TYPE CREATED BY SARAH WOOTEN'S 

1497
01:03:48,154 --> 01:03:50,422
GROUP SPECIFICALLY TROPIC FOR 

1498
01:03:50,422 --> 01:03:53,359
TYPE 2 CELLS AND ABLE TO GET UP 

1499
01:03:53,359 --> 01:03:56,762
TO 80% TRANSDUCTION THERAPY IN 

1500
01:03:56,762 --> 01:03:59,965
THE CELLS IN VITRO AND IN 

1501
01:03:59,965 --> 01:04:00,733
CULTURE AND IN VIVO.

1502
01:04:00,733 --> 01:04:02,968
AND IT'S BEEN PROVEN TO BE AN 

1503
01:04:02,968 --> 01:04:05,104
EFFICIENT VIRUS MORE SO THAN THE

1504
01:04:05,104 --> 01:04:09,341
LENTI VIRUSES OR ONLY ABLE TO 

1505
01:04:09,341 --> 01:04:11,143
GET 5% EFFICIENCY.

1506
01:04:11,143 --> 01:04:14,680
THE PROBLEM IS THE CARGO 

1507
01:04:14,680 --> 01:04:18,250
CAPACITY OR AAD IS SMALLER THAN 

1508
01:04:18,250 --> 01:04:23,789
THE SIZE OF THE GENE SO WHAT I'M

1509
01:04:23,789 --> 01:04:25,758
CHOOSING TO DO IS TO DELIVER THE

1510
01:04:25,758 --> 01:04:27,126
HALF SEPARATELY WHICH IS WHY IN 

1511
01:04:27,126 --> 01:04:31,397
THE EXPERIMENT I'M SHOWING HERE 

1512
01:04:31,397 --> 01:04:33,766
I'M TESTING THE DUAL DELIVERY 

1513
01:04:33,766 --> 01:04:38,337
MODEL AND SEE THEY'RE ABLE TO BE

1514
01:04:38,337 --> 01:04:39,905
TRANSDUCED WITH HIGH EFFICIENCY.

1515
01:04:39,905 --> 01:04:42,374
THESE LENTI VIRUSES AND SYSTEMS 

1516
01:04:42,374 --> 01:04:45,010
ARE A FEW OF THE THERAPEUTIC 

1517
01:04:45,010 --> 01:04:51,016
OPTIONS WE ARE INVESTIGATING FOR

1518
01:04:51,016 --> 01:04:53,052
ABCA3 THERAPIES RIGHT NOW.

1519
01:04:53,052 --> 01:04:55,688
IN THIS TALK, I HOPE I HAVE 

1520
01:04:55,688 --> 01:04:58,457
IMPRESSED UPON YOU THE UTILITY 

1521
01:04:58,457 --> 01:05:03,529
OF IPSCs AS A MODEL FOR 

1522
01:05:03,529 --> 01:05:04,230
VULNERABLE DISEASE POPULATION.

1523
01:05:04,230 --> 01:05:09,068
SOME TAKEAWAYS I HOPE ARE IPSC 

1524
01:05:09,068 --> 01:05:10,069
CELLS ARE PATIENT SPECIFIC.

1525
01:05:10,069 --> 01:05:12,171
WHEN YOU TAKE THEM FROM A DONOR 

1526
01:05:12,171 --> 01:05:14,039
NOT ONLY WILL THEY KWON TAIN 

1527
01:05:14,039 --> 01:05:14,907
YOUR MUTATION OF INTEREST AND 

1528
01:05:14,907 --> 01:05:17,409
THE GENETIC INFORMATION.

1529
01:05:17,409 --> 01:05:19,278
NUMBER TWO, THEY ARE EXPANDABLE 

1530
01:05:19,278 --> 01:05:22,181
IN CULTURE.

1531
01:05:22,181 --> 01:05:24,650
THEY'RE RENEWABLE AND NEARLY 

1532
01:05:24,650 --> 01:05:25,517
INEXHAUSTIBLE WHICH IS A GREAT 

1533
01:05:25,517 --> 01:05:26,785
RESOURCE TO HAVE AND FREEZE THEM

1534
01:05:26,785 --> 01:05:30,022
AND RETURN TO THEM YEARS LATER.

1535
01:05:30,022 --> 01:05:32,558
NUMBER THREE, THEY'RE A 

1536
01:05:32,558 --> 01:05:33,025
REDUCTIONIST MODEL.

1537
01:05:33,025 --> 01:05:34,860
THIS IS BOTH A PRO AND CON 

1538
01:05:34,860 --> 01:05:37,863
DEPENDING ON HOW YOU LOOK AT IT 

1539
01:05:37,863 --> 01:05:38,864
AND WHAT YOUR NEEDS ARE.

1540
01:05:38,864 --> 01:05:40,432
FOR MY PURPOSES IT'S BEEN 

1541
01:05:40,432 --> 01:05:43,035
HELPFUL TO BE ABLE TO STUDY THE 

1542
01:05:43,035 --> 01:05:48,307
DISEASE MANIFESTATIONS IN AD2 

1543
01:05:48,307 --> 01:05:51,810
ALONE BEFORE MOVING TO THE 

1544
01:05:51,810 --> 01:05:52,911
GREATER LUNG.

1545
01:05:52,911 --> 01:05:54,313
FOUR, THEIR EDITABLE AND CORRECT

1546
01:05:54,313 --> 01:05:57,416
THE PATIENT MUTATION OR KNOCK-IN

1547
01:05:57,416 --> 01:06:00,452
MUTATION WHICH GIVES US THE 

1548
01:06:00,452 --> 01:06:02,221
POWER OF CONTROLS.

1549
01:06:02,221 --> 01:06:05,057
AND NUMBER 5, DRUGGABLE AND USED

1550
01:06:05,057 --> 01:06:05,991
TO EXPLORE THERAPEUTIC OPTIONS 

1551
01:06:05,991 --> 01:06:07,092
LIKE THE GENE THERAPY I SHOWED 

1552
01:06:07,092 --> 01:06:09,428
YOU.

1553
01:06:09,428 --> 01:06:09,928
OKAY.

1554
01:06:09,928 --> 01:06:11,330
THANK YOU ALL VERY MUCH FOR 

1555
01:06:11,330 --> 01:06:12,064
LISTENING.

1556
01:06:12,064 --> 01:06:14,199
I'M NOT ABLE TO BE LIVE IN THE 

1557
01:06:14,199 --> 01:06:15,534
WORKSHOP THIS MORNING BUT PLEASE

1558
01:06:15,534 --> 01:06:17,069
DON'T HESITATE TO REACH OUT WITH

1559
01:06:17,069 --> 01:06:18,304
ANY QUESTIONS OR COMMENTS YOU 

1560
01:06:18,304 --> 01:06:18,570
MAY HAVE.

1561
01:06:18,570 --> 01:06:28,781
THANK YOU.

1562
01:06:28,781 --> 01:06:29,348
>> OKAY.

1563
01:06:29,348 --> 01:06:31,016
THANK YOU.

1564
01:06:31,016 --> 01:06:36,922
SO OUR NEXT TALK IS BY 

1565
01:06:36,922 --> 01:06:39,191
DR. BIRCSAK FROM MIMETAS.

1566
01:06:39,191 --> 01:06:39,892
>> THANK YOU FOR THE 

1567
01:06:39,892 --> 01:06:40,225
INTRODUCTION.

1568
01:06:40,225 --> 01:06:42,928
I WANT TO THANK THE ORGANIZERS 

1569
01:06:42,928 --> 01:06:44,129
FOR THE INVITATION TO PRESENT 

1570
01:06:44,129 --> 01:06:44,330
TODAY.

1571
01:06:44,330 --> 01:06:51,103
I'M EXCITE TO SHARE THE LATEST 

1572
01:06:51,103 --> 01:06:55,040
MEMETICS AND ORGANOID CHIP 

1573
01:06:55,040 --> 01:06:56,408
MODELS FOR MIMICKING DISEASE 

1574
01:06:56,408 --> 01:06:56,709
PROCESS.

1575
01:06:56,709 --> 01:06:58,610
HERE'S AN OVERVIEW OF OUR 

1576
01:06:58,610 --> 01:06:58,944
PRESENTATION.

1577
01:06:58,944 --> 01:07:03,682
FIRST WE'LL START OFF WITH AN 

1578
01:07:03,682 --> 01:07:06,385
INTRODUCTION TO MIMETAS AND MOVE

1579
01:07:06,385 --> 01:07:08,320
ON TO THE ORGAN CHIP SYSTEMS.

1580
01:07:08,320 --> 01:07:11,423
I WANT TO THANK ANTWAN IN 

1581
01:07:11,423 --> 01:07:11,657
ADVANCE.

1582
01:07:11,657 --> 01:07:13,959
I HAVE MULTIPLE TRANSITIONS IN 

1583
01:07:13,959 --> 01:07:16,261
THE SLIDES SO THANK YOU AND I'LL

1584
01:07:16,261 --> 01:07:17,529
BE KEEPING YOU ON YOUR TOES, I 

1585
01:07:17,529 --> 01:07:27,639
THINK.

1586
01:07:31,076 --> 01:07:32,745
MIMETAS IS A COMPANY WITH 

1587
01:07:32,745 --> 01:07:35,714
PROVIDES MODELS AND ORGANS ON A 

1588
01:07:35,714 --> 01:07:35,914
CHIP.

1589
01:07:35,914 --> 01:07:37,282
A MORE COMPLEX IN VITRO MODELS 

1590
01:07:37,282 --> 01:07:46,225
THAT AID TO GENERATE CRITICAL 

1591
01:07:46,225 --> 01:07:48,060
INFORMATION AND IT'S A GLOBAL 

1592
01:07:48,060 --> 01:07:48,494
COMPANY.

1593
01:07:48,494 --> 01:07:50,496
WE ARE HEADQUARTERS IN THE 

1594
01:07:50,496 --> 01:07:53,298
NETHERLANDS AND HAVE OFFICES IN 

1595
01:07:53,298 --> 01:07:54,299
JAPAN AS WELL AS IN THE UNITED 

1596
01:07:54,299 --> 01:07:55,033
STATES.

1597
01:07:55,033 --> 01:07:57,803
I'M BASED IN OUR OFFICE IN 

1598
01:07:57,803 --> 01:07:58,804
MARYLAND WHERE WE HAVE A SMALL 

1599
01:07:58,804 --> 01:08:08,180
RESEARCH AND DEVELOPMENT TEAM.

1600
01:08:08,180 --> 01:08:10,916
OUR ORGAN ON CHIP SYSTEMS COME 

1601
01:08:10,916 --> 01:08:13,018
WITH KEY BUILDING BLOCKS THAT 

1602
01:08:13,018 --> 01:08:14,853
ARE IMPORTANT FOR MODELLING 

1603
01:08:14,853 --> 01:08:15,120
TISSUE.

1604
01:08:15,120 --> 01:08:20,559
SO WE CAN MODEL EPITHELIUM, THE 

1605
01:08:20,559 --> 01:08:21,894
CONNECTIVE TISSUE, VASCULATURE, 

1606
01:08:21,894 --> 01:08:22,761
THE IMMUNE SYSTEM.

1607
01:08:22,761 --> 01:08:23,729
WE KNOW THEY'RE ALL IMPORTANT 

1608
01:08:23,729 --> 01:08:26,131
PARTS AND IT'S IMPORTANT TO KEEP

1609
01:08:26,131 --> 01:08:27,633
IN MIND WE CAN INCORPORATE ALL 

1610
01:08:27,633 --> 01:08:31,036
OF THEM BUT IT DEPENDS ON YOUR 

1611
01:08:31,036 --> 01:08:32,671
RESEARCH QUESTION WHETHER 

1612
01:08:32,671 --> 01:08:35,040
NECESSARILY SHOULD INCORPORATE 

1613
01:08:35,040 --> 01:08:37,176
ALL THOSE COMPONENTS INTO YOUR 

1614
01:08:37,176 --> 01:08:37,843
MICROPHYSIOLOGICAL SYSTEM.

1615
01:08:37,843 --> 01:08:42,714
AND I THINK WHAT SETS US A PART 

1616
01:08:42,714 --> 01:08:43,949
FROM OTHER MICROPHYSIOLOGICAL 

1617
01:08:43,949 --> 01:08:46,251
SYSTEMS IS THE FACT WE'RE A 

1618
01:08:46,251 --> 01:08:48,821
SCREENABLE PLATFORM.

1619
01:08:48,821 --> 01:08:51,023
WE CAN MODEL THESE TISSUES EIGHT

1620
01:08:51,023 --> 01:08:52,324
SIGNIFICANT SCALE AND WHAT 

1621
01:08:52,324 --> 01:08:55,394
ENABLES US TO DO THAT IS THE 

1622
01:08:55,394 --> 01:08:59,064
FACT THAT CHIPS ARE CONTAINED 

1623
01:08:59,064 --> 01:09:01,333
WITHIN A 384 FOOTPRINT.

1624
01:09:01,333 --> 01:09:04,036
WE'RE FITTING ON STANDARD 

1625
01:09:04,036 --> 01:09:06,038
LABORATORY MICROSCOPES, PLATE 

1626
01:09:06,038 --> 01:09:08,307
READERS, HIGH CONTENT 

1627
01:09:08,307 --> 01:09:11,043
MICROSCOPES, LIQUID HANDLERS YOU

1628
01:09:11,043 --> 01:09:13,912
CAN SEE ON THE RIGHT SIDE AND 

1629
01:09:13,912 --> 01:09:15,514
THE PLATFORM IS CALLED THE 

1630
01:09:15,514 --> 01:09:16,014
ORGANO PLATE.

1631
01:09:16,014 --> 01:09:18,517
WE'LL GO INTO MORE DETAIL SOON 

1632
01:09:18,517 --> 01:09:19,618
ABOUT THAT.

1633
01:09:19,618 --> 01:09:25,591
BUT THE FACT IT'S IN THE 

1634
01:09:25,591 --> 01:09:32,598
FOOTPRINT MEANS WE CAN USE AUTO 

1635
01:09:32,598 --> 01:09:34,967
MATED LIQUID HANDLING AND THIS 

1636
01:09:34,967 --> 01:09:36,668
ENABLES US TO BE ABLE TO CULTURE

1637
01:09:36,668 --> 01:09:38,871
TENS OF PLATES AT A TIME AND WIN

1638
01:09:38,871 --> 01:09:43,475
ONE PLATE WE HAVE ANYWHERE FROM 

1639
01:09:43,475 --> 01:09:45,944
40 TO 96 INDIVIDUAL TISSUES OR 

1640
01:09:45,944 --> 01:09:47,012
REPLICATES YOU CAN IMAGINE.

1641
01:09:47,012 --> 01:09:51,617
SO IT WOULD BE FEASIBLE AND 

1642
01:09:51,617 --> 01:09:54,586
WE'VE DONE THIS BEFORE TO SCREEN

1643
01:09:54,586 --> 01:09:55,888
THOUSANDS OF YOUR CHIPS TO 

1644
01:09:55,888 --> 01:09:56,388
POTENTIALLY FIND NOVEL 

1645
01:09:56,388 --> 01:10:04,630
COMPOUNDS.

1646
01:10:04,630 --> 01:10:11,103
HERE I'LL SHOW YOU A VIDEO TO 

1647
01:10:11,103 --> 01:10:18,644
GIVE YOU AN IDEA HOW IT PLATES.

1648
01:10:18,644 --> 01:10:21,046
THE BOTTOM HAS MICROSCOPE 

1649
01:10:21,046 --> 01:10:24,483
QUALITY GLASS AND SEE THE THREE 

1650
01:10:24,483 --> 01:10:27,019
CHANNELS IN PARALLEL.

1651
01:10:27,019 --> 01:10:32,457
WE CAN ADD IN OUR EXTRA CELLULAR

1652
01:10:32,457 --> 01:10:34,192
MATRIX GEL AND NO BARRIERS THAT 

1653
01:10:34,192 --> 01:10:36,595
SEPARATE THE CHANNEL.

1654
01:10:36,595 --> 01:10:39,331
WE HAVE THIS PHASED GUIDE AND 

1655
01:10:39,331 --> 01:10:41,667
CAPILLARY PRESSURE BARRIER THAT 

1656
01:10:41,667 --> 01:10:42,768
PREVENTS THE EXTRA CELLULAR 

1657
01:10:42,768 --> 01:10:44,102
MATRIX FROM SPILLING OVER.

1658
01:10:44,102 --> 01:10:45,837
ONCE THAT SOLIDIFIED WE CAN ADD 

1659
01:10:45,837 --> 01:10:49,341
CELLS AND MEDIA TO THE ADJACENT 

1660
01:10:49,341 --> 01:10:51,743
PERFUSION OR MEDIA CHANNELS AND 

1661
01:10:51,743 --> 01:10:53,178
WE CAN ACHIEVE PERFUSION BY 

1662
01:10:53,178 --> 01:10:54,880
PLACING THE PLATE ON A ROCKER 

1663
01:10:54,880 --> 01:10:59,685
SYSTEM DIRECTLY WITHIN THE 

1664
01:10:59,685 --> 01:11:03,221
INCUBATOR AND STACK MULTIPLE 

1665
01:11:03,221 --> 01:11:05,357
PLATES ON THE SYSTEM.

1666
01:11:05,357 --> 01:11:09,561
WHAT WE FIND OVER TIME IS THAT 

1667
01:11:09,561 --> 01:11:11,730
WHEN WE HAVE THE CELLS IN THE 

1668
01:11:11,730 --> 01:11:14,633
PROFUSION CHANNEL THEY'LL 

1669
01:11:14,633 --> 01:11:16,101
PROLIFERATE AND FORM THIS 3-D 

1670
01:11:16,101 --> 01:11:19,037
STRUCTURE IN THIS CASE MIMICKING

1671
01:11:19,037 --> 01:11:29,314
A BLOOD VESSEL.

1672
01:11:30,382 --> 01:11:31,817
THAT'S A BIT ON THE PLATFORM IN 

1673
01:11:31,817 --> 01:11:32,217
GENERAL.

1674
01:11:32,217 --> 01:11:35,020
NOW I'D LIKE TO GO INTO MORE 

1675
01:11:35,020 --> 01:11:39,024
DETAIL IN OUR AIRWAY ORGANOID 

1676
01:11:39,024 --> 01:11:49,234
CHIP SYSTEM.

1677
01:11:55,073 --> 01:11:59,044
WE'RE FOCUSSING  PRIMARILY ON 

1678
01:11:59,044 --> 01:12:00,445
THE LUNG.

1679
01:12:00,445 --> 01:12:02,648
WE CAN MODEL DIFFERENT DISEASE 

1680
01:12:02,648 --> 01:12:05,884
PLATFORM DISEASES WITH AN 

1681
01:12:05,884 --> 01:12:08,120
INTERNAL TRIGGER, BY CELLS THAT 

1682
01:12:08,120 --> 01:12:11,223
EXPRESS MAYBE A CERTAIN DISEASE 

1683
01:12:11,223 --> 01:12:17,896
MUTATION OR EXTERNAL TRIGGER BY 

1684
01:12:17,896 --> 01:12:18,897
APPLYING INFECTIOUS COMPONENTS 

1685
01:12:18,897 --> 01:12:20,232
LIKE VIRUSES AND THINGS LIKE 

1686
01:12:20,232 --> 01:12:20,499
THAT.

1687
01:12:20,499 --> 01:12:25,804
AND WE'RE ALSO CAPABLE OF HAVING

1688
01:12:25,804 --> 01:12:27,706
MULTIPLE READOUTS WITHIN ONE 

1689
01:12:27,706 --> 01:12:28,240
CHIP COLLECTING A LOT OF 

1690
01:12:28,240 --> 01:12:31,009
DIFFERENT DATA.

1691
01:12:31,009 --> 01:12:33,645
SO THE BOTTOM MADE OF THE 

1692
01:12:33,645 --> 01:12:35,914
MICROSCOPE QUALITY GLAD WE'RE 

1693
01:12:35,914 --> 01:12:39,017
ABLE TO PERFORM IMAGING AND 

1694
01:12:39,017 --> 01:12:42,421
CAPTURING REALLY IN SITU THE 3-D

1695
01:12:42,421 --> 01:12:43,689
STRUCTURE OF THE TISSUE.

1696
01:12:43,689 --> 01:12:46,191
CAN TO ELECTRICAL SENSING, 

1697
01:12:46,191 --> 01:12:47,325
EXPRESSION ANALYSIS AND THEN 

1698
01:12:47,325 --> 01:12:53,298
IT'S ALSO POSSIBLE TO SAMPLE THE

1699
01:12:53,298 --> 01:12:55,834
MEDIA FROM THOSE RESERVOIR WELLS

1700
01:12:55,834 --> 01:12:57,803
AND TO EVALUATE CYTOKINES THAT 

1701
01:12:57,803 --> 01:12:58,570
MAY HAVE BEEN RELEASED AND 

1702
01:12:58,570 --> 01:13:02,407
PRODUCED BY THE CULTURES.

1703
01:13:02,407 --> 01:13:06,111
AS A RESULT OF A DISEASE OR 

1704
01:13:06,111 --> 01:13:06,378
TREATMENT.

1705
01:13:06,378 --> 01:13:09,648
AND SO OUR LUNG ON A CHIP MODEL 

1706
01:13:09,648 --> 01:13:11,850
CAN BE USED ACROSS THE SPECTRUM 

1707
01:13:11,850 --> 01:13:14,519
I THINK ACROSS FROM DISCOVERY 

1708
01:13:14,519 --> 01:13:17,322
AND DEVELOPMENT FROM VERY EARLY 

1709
01:13:17,322 --> 01:13:20,425
ON IN DISCOVERY DECIPHERING THE 

1710
01:13:20,425 --> 01:13:20,959
DISEASE BIOLOGY THROUGH 

1711
01:13:20,959 --> 01:13:23,028
DERISKING AND ACCELERATING YOUR 

1712
01:13:23,028 --> 01:13:33,371
DRUG TO THE CLINIC.

1713
01:13:35,073 --> 01:13:36,341
I'M NOT GOING TO GO INTO A LOT 

1714
01:13:36,341 --> 01:13:39,377
OF DETAIL AND NOTICED IN OUR 

1715
01:13:39,377 --> 01:13:41,580
FIRST PRESENTATION BY ALFRED HE 

1716
01:13:41,580 --> 01:13:47,319
USED THE SIMILAR PICTOFWRGRAPHI 

1717
01:13:47,319 --> 01:13:48,854
WHICH IS GREAT TO SHOW YOU THE 

1718
01:13:48,854 --> 01:13:50,021
DIFFERENT AREAS AND PARTS OF THE

1719
01:13:50,021 --> 01:13:52,157
LUNG IF YOU'RE INTERESTED IN 

1720
01:13:52,157 --> 01:13:56,061
MODELLING ONE AREA YOU MAY WANT 

1721
01:13:56,061 --> 01:13:59,030
TO INCLUDE SPECIFIC CELL TYPES.

1722
01:13:59,030 --> 01:14:03,468
FOR EXAMPLE, AT MIMETAS WE'RE 

1723
01:14:03,468 --> 01:14:13,879
LOOK AT THE BRONCHULAR 

1724
01:14:14,379 --> 01:14:15,514
EPITHELIUM AND THE PRIMARY 

1725
01:14:15,514 --> 01:14:17,783
FUNCTION OF THE LUNG IS GAS 

1726
01:14:17,783 --> 01:14:20,318
EXCHANGE AND INVOLVED IN THE 

1727
01:14:20,318 --> 01:14:23,522
REGULATION OF BLOOD PH AND 

1728
01:14:23,522 --> 01:14:26,024
DEFENDING AGAINST PATHOGENS.

1729
01:14:26,024 --> 01:14:29,194
WE CAN ALSO SEE -- WE KNOW IT'S 

1730
01:14:29,194 --> 01:14:32,631
PARTICULARLY IMPORTANT TO BE 

1731
01:14:32,631 --> 01:14:34,499
ABLE TO MODEL LUNG FUNCTION IN 

1732
01:14:34,499 --> 01:14:37,836
VITRO AND LUNG DISEASES IN VITRO

1733
01:14:37,836 --> 01:14:42,474
BECAUSE CHRONIC RESPIRATORY 

1734
01:14:42,474 --> 01:14:44,910
DISEASES IS A LEADING CAUSE OF 

1735
01:14:44,910 --> 01:14:46,745
GLOBAL CANCER AND MORTALITY.

1736
01:14:46,745 --> 01:14:50,182
WE KNOW THERE'S AN YOU ARE FWENT

1737
01:14:50,182 --> 01:14:53,685
NEED TO BE ABLE ACCELERATE THE 

1738
01:14:53,685 --> 01:14:55,053
THERAPIES AND GET THEM KNOW 

1739
01:14:55,053 --> 01:14:56,755
PATIENTS AND PATIENTS IN NEED.

1740
01:14:56,755 --> 01:15:01,226
THE PRECLINICAL MODELS ARE JUST 

1741
01:15:01,226 --> 01:15:02,527
THEY'RE NOT GOOD ENOUGH AS JEFF 

1742
01:15:02,527 --> 01:15:04,863
INDICATED IN HIS TALK.

1743
01:15:04,863 --> 01:15:07,065
SO WE NEED TO KEEP IMPROVING OUR

1744
01:15:07,065 --> 01:15:10,602
MODELS SO THAT WE CAN GET THESE 

1745
01:15:10,602 --> 01:15:11,203
THERAPIES TO PATIENTS AS FAST 

1746
01:15:11,203 --> 01:15:21,379
POSSIBLE.

1747
01:15:22,280 --> 01:15:24,416
FIRST WE'RE GOING TO OUR LUNG ON

1748
01:15:24,416 --> 01:15:25,884
A CHIP MODEL WE USED 

1749
01:15:25,884 --> 01:15:28,486
PARTICULARLY FOR AN AIR 

1750
01:15:28,486 --> 01:15:28,753
EXPOSURE.

1751
01:15:28,753 --> 01:15:30,055
IMAGINE IT'S USED FOR A THERAPY 

1752
01:15:30,055 --> 01:15:33,058
THAT MAY BE INHALED, FOR 

1753
01:15:33,058 --> 01:15:33,625
EXAMPLE.

1754
01:15:33,625 --> 01:15:38,997
IN THE LEFT PANEL WE SEE WE HAVE

1755
01:15:38,997 --> 01:15:43,501
A PICTOGRAPH OF ONE OF OUR CHIPS

1756
01:15:43,501 --> 01:15:45,804
THE RED MEDIA CHANNEL IS WHERE 

1757
01:15:45,804 --> 01:15:48,106
THERE'S MEDIA PROFUSING BACK AND

1758
01:15:48,106 --> 01:15:51,009
FORTH IN A BI-DIRECTIONAL 

1759
01:15:51,009 --> 01:15:51,610
MANNER.

1760
01:15:51,610 --> 01:15:52,777
IN THE CENTER CHANNEL IN THE 

1761
01:15:52,777 --> 01:15:57,716
BLUE WE HAVE AN EXTRA CELLULAR 

1762
01:15:57,716 --> 01:16:00,151
MATRIX THAT FORM SOMEWHAT OF A 

1763
01:16:00,151 --> 01:16:00,452
SCAFFOLD.

1764
01:16:00,452 --> 01:16:05,056
IN THE CENTER OF THE SCAFFOLD WE

1765
01:16:05,056 --> 01:16:11,363
HAVE BASICALLY NRL A HOLD TO AP 

1766
01:16:11,363 --> 01:16:13,531
THE CELLS AND WHEN WE DO THAT 

1767
01:16:13,531 --> 01:16:18,970
THEY ADHERE IN A 3-D STRUCTURE 

1768
01:16:18,970 --> 01:16:20,739
TO THE EXTRA CELLULAR MATRIX 

1769
01:16:20,739 --> 01:16:21,006
SCAFFOLD.

1770
01:16:21,006 --> 01:16:26,778
WHAT WE'RE LOOKING AT IN THE TOP

1771
01:16:26,778 --> 01:16:28,513
IMAGE 2-D, TOP DOWN.

1772
01:16:28,513 --> 01:16:30,248
SOMETIMES IT'S DIFFICULT TO 

1773
01:16:30,248 --> 01:16:32,150
GRASP WHAT'S GOING ON IN A 3-D 

1774
01:16:32,150 --> 01:16:33,084
CULTURE.

1775
01:16:33,084 --> 01:16:35,487
WE ALSO TRY TO PROVIDE THE 

1776
01:16:35,487 --> 01:16:36,154
CROSS-SECTIONAL VIEWS.

1777
01:16:36,154 --> 01:16:38,290
THAT'S WHAT WE SEE IN THE BOTTOM

1778
01:16:38,290 --> 01:16:38,590
PANELS.

1779
01:16:38,590 --> 01:16:40,525
THERE'S TWO CROSS-SECTIONAL 

1780
01:16:40,525 --> 01:16:40,725
VIEWS.

1781
01:16:40,725 --> 01:16:46,264
THE ONE ON TOP IS WHAT THE 

1782
01:16:46,264 --> 01:16:48,133
CULTURE AFTER WE APPLIED THE 

1783
01:16:48,133 --> 01:16:50,101
CELLS TO THE EXTRA CELLULAR 

1784
01:16:50,101 --> 01:16:52,837
MATRIX SCAFFOLD AND THE WHITE 

1785
01:16:52,837 --> 01:16:54,572
CELLS ADHERE TO THE BLUE AND WE 

1786
01:16:54,572 --> 01:16:56,775
HAVE A MEDIA THAT WE FIRST USED 

1787
01:16:56,775 --> 01:16:59,044
WHEN APPLYING THEM TO THE 

1788
01:16:59,044 --> 01:17:00,378
CULTURE.

1789
01:17:00,378 --> 01:17:03,648
ONCE THE CELLS HAVE ADHERED AND 

1790
01:17:03,648 --> 01:17:07,018
PROLIFERATED TOE ENCOMPASS THE 

1791
01:17:07,018 --> 01:17:10,355
ENTIRE 3-D TUBULE STRUCTURE WE 

1792
01:17:10,355 --> 01:17:13,358
CAN REMOVE THE MEDIA AND DO WHAT

1793
01:17:13,358 --> 01:17:16,828
WE CALL AIR LIFT TO HAVE THIS 

1794
01:17:16,828 --> 01:17:18,630
AIR LIQUID INTERFACE SIMILAR TO 

1795
01:17:18,630 --> 01:17:20,465
WHAT PRESENTERS WERE TALKING 

1796
01:17:20,465 --> 01:17:21,700
ABOUT IN THE PREVIOUS TWO TALKS.

1797
01:17:21,700 --> 01:17:25,470
SO THE IDEA IS VERY SIMILAR IN 

1798
01:17:25,470 --> 01:17:28,073
THE FACT THAT YOU HAVE AIR ON 

1799
01:17:28,073 --> 01:17:30,775
ONE SIDE AND THEN MEDIA OR 

1800
01:17:30,775 --> 01:17:31,810
MIMICKING THE BLOOD COMPARTMENT 

1801
01:17:31,810 --> 01:17:34,179
ON THE OTHER SIDE.

1802
01:17:34,179 --> 01:17:40,585
THIS REALLY PUSHES THE CULTURES 

1803
01:17:40,585 --> 01:17:43,621
TO DIFFERENTIATE.

1804
01:17:43,621 --> 01:17:46,858
HERE WE HAVE SOME AMINO 

1805
01:17:46,858 --> 01:17:52,931
FLUORESCENT STAINING IMAGES OF 

1806
01:17:52,931 --> 01:17:54,165
OUR ORGANO PLATE ERROR IS WHAT 

1807
01:17:54,165 --> 01:17:55,734
WE'RE CALLING THIS.

1808
01:17:55,734 --> 01:17:57,936
WE CAN CULTURE THE CELLS OUT TO 

1809
01:17:57,936 --> 01:17:59,037
AT LEAST 35 DAYS.

1810
01:17:59,037 --> 01:18:00,438
WHAT WE SEE ON THE LEFT-HAND 

1811
01:18:00,438 --> 01:18:03,808
SIDE THE FIRST IMAGE IS A 2-D 

1812
01:18:03,808 --> 01:18:04,075
IMAGE.

1813
01:18:04,075 --> 01:18:05,643
BEING A 3-D CULTURE IT'S 

1814
01:18:05,643 --> 01:18:08,013
IMPORTANT TO KEEP IN MIND LIKE 

1815
01:18:08,013 --> 01:18:10,081
THE STEROIDS PROBABLY THE CASE 

1816
01:18:10,081 --> 01:18:14,786
IN THE PREVIOUS PRESENTATION IN 

1817
01:18:14,786 --> 01:18:17,255
ORDER TO CAPTURE THE 3-D 

1818
01:18:17,255 --> 01:18:19,691
INFORMATION YOU'RE USING A 

1819
01:18:19,691 --> 01:18:22,427
CONFOCAL MICROSCOPE TO Z STACK 

1820
01:18:22,427 --> 01:18:23,495
THROUGH THE HEIGHT OF THE 

1821
01:18:23,495 --> 01:18:25,630
CHANNEL AND MAKE SURE YOU'RE 

1822
01:18:25,630 --> 01:18:26,464
CAPTURING ALL INFORMATION ABOUT 

1823
01:18:26,464 --> 01:18:27,599
THE 3-D OF THE CULTURE.

1824
01:18:27,599 --> 01:18:31,036
AND WHAT YOU CAN DO WITH ALL 

1825
01:18:31,036 --> 01:18:34,506
THOSE IMAGES IS COMPRESS THEM 

1826
01:18:34,506 --> 01:18:36,975
INTO A MAXIMUM INTENSITY 

1827
01:18:36,975 --> 01:18:38,877
PROJECTION AND MAKE IT A 2-D 

1828
01:18:38,877 --> 01:18:39,077
IMAGE.

1829
01:18:39,077 --> 01:18:42,113
THAT'S WHAT WE'RE LOOKING AT ON 

1830
01:18:42,113 --> 01:18:48,353
THE BOTTOM THE INTENSITY OF THE 

1831
01:18:48,353 --> 01:18:53,525
3-D STRUCTURE AND SEE EXPRESSION

1832
01:18:53,525 --> 01:19:00,031
FOR THE  TUBULIN AND IF YOU 

1833
01:19:00,031 --> 01:19:03,001
ADVANCE ONE MORE TIME WE SEE THE

1834
01:19:03,001 --> 01:19:04,702
NEXT IMAGE ROTATING BITS.

1835
01:19:04,702 --> 01:19:07,105
WHAT YOU CAN DO WHEN YOU TAKE 

1836
01:19:07,105 --> 01:19:09,207
THE Z STACKS IS MAKE A 3-D 

1837
01:19:09,207 --> 01:19:10,875
RECONSTRUCTION AND THAT'S WHAT 

1838
01:19:10,875 --> 01:19:14,212
WE'RE SEEING HERE.

1839
01:19:14,212 --> 01:19:21,086
A ROTATING 3-D RECONSTRUCTION OF

1840
01:19:21,086 --> 01:19:26,524
THAT BRONCULAR TUBULE.

1841
01:19:26,524 --> 01:19:30,762
WE CAN ALSO SEE THE FUNCTIONING 

1842
01:19:30,762 --> 01:19:33,832
OF THE CILIATED CELLS.

1843
01:19:33,832 --> 01:19:36,234
IN OUR FAITH CONTRAST IMAGE WE 

1844
01:19:36,234 --> 01:19:39,237
CAN POTENTIALLY SEE BEATING AND 

1845
01:19:39,237 --> 01:19:41,706
MOVING OF THE CILIATED CELLS AND

1846
01:19:41,706 --> 01:19:43,441
ABLE TO QUANTIFY THIS AND SEE 

1847
01:19:43,441 --> 01:19:45,110
ANN INCREASE IN THE NUMBER OF 

1848
01:19:45,110 --> 01:19:55,553
CILIATED CELLS OVER TIME.

1849
01:19:57,021 --> 01:20:02,427
WE'RE ABLE TO QUANTIFY HYPER 

1850
01:20:02,427 --> 01:20:06,264
PLAY SHA OF THE CELLS AND -- 

1851
01:20:06,264 --> 01:20:09,067
HYPERPLASIA OF THE CELLS AND 

1852
01:20:09,067 --> 01:20:10,902
KNOWN IN INFLAMMATION AND TISSUE

1853
01:20:10,902 --> 01:20:13,505
REGENERATION AND CAN SEE THERE'S

1854
01:20:13,505 --> 01:20:16,341
AN INCREASE IN THE COMPRESSION 

1855
01:20:16,341 --> 01:20:20,512
OF MUC BY C AND DECREASED IN THE

1856
01:20:20,512 --> 01:20:24,649
EXPRESSION OF CYTOKERATIN 5 AND 

1857
01:20:24,649 --> 01:20:31,022
HAVE UP REGULATION IN THE  

1858
01:20:31,022 --> 01:20:39,631
GOBLET CELLS IN THE POPULATION.

1859
01:20:39,631 --> 01:20:44,636
I'LL MOVE TO OUR ORGAN ON A 

1860
01:20:44,636 --> 01:20:49,274
ORGAN ON A CHIP MODEL AND THIS 

1861
01:20:49,274 --> 01:20:50,074
IS SLIGHTLY DIFFERENT IN THE IN 

1862
01:20:50,074 --> 01:20:55,914
THE NEXT SLIDE I GO INTO A BIT 

1863
01:20:55,914 --> 01:21:02,387
MORE DETAIL SO WE'RE IN A 

1864
01:21:02,387 --> 01:21:03,721
SIMILAR CHIP LIKE STRUCTURE ON 

1865
01:21:03,721 --> 01:21:04,923
THE RIGHT AND LEFT SIDE.

1866
01:21:04,923 --> 01:21:06,391
IN PINK WE HAVE THE MEDIA 

1867
01:21:06,391 --> 01:21:11,062
CHANNELS WHERE IN THIS CASE WE 

1868
01:21:11,062 --> 01:21:14,299
HAVE ENDOTHELIAL CELL TUBULE 

1869
01:21:14,299 --> 01:21:15,066
GROWING ON BOTH SIDES.

1870
01:21:15,066 --> 01:21:16,701
WHAT'S DIFFERENT HERE IS WE 

1871
01:21:16,701 --> 01:21:18,169
DON'T HAVE AN OPEN CHANNEL.

1872
01:21:18,169 --> 01:21:20,171
INSTEAD WHAT WE HAVE IN THE 

1873
01:21:20,171 --> 01:21:24,209
MIDDLE IS A VASCULAR BED 

1874
01:21:24,209 --> 01:21:26,878
EMBEDDED WITHIN AN EXTRA 

1875
01:21:26,878 --> 01:21:27,879
CELLULAR MATRIX CELLS.

1876
01:21:27,879 --> 01:21:29,080
HERE THE CELLS ARE MIXED 

1877
01:21:29,080 --> 01:21:35,019
TOGETHER WITH FIBROBLASTS AND 

1878
01:21:35,019 --> 01:21:38,990
SPONTANEOUSLY FORM A  VASCULAR 

1879
01:21:38,990 --> 01:21:40,959
BED AND FORM A MONOLAYER 

1880
01:21:40,959 --> 01:21:43,728
DIRECTLY ON TOP OF THE VASCULAR 

1881
01:21:43,728 --> 01:21:54,272
BED THOSE LUNG EPITHELIAL CELLS.

1882
01:21:57,342 --> 01:21:58,843
WE KNOW THE CELLS ARE 

1883
01:21:58,843 --> 01:21:59,410
UNDIFFERENTIATED AND ONCE 

1884
01:21:59,410 --> 01:22:02,013
THEY'VE SUSTAINED THEMSELVES, IF

1885
01:22:02,013 --> 01:22:08,386
YOU CLICK ONE MORE TIME WE DO AN

1886
01:22:08,386 --> 01:22:18,596
AIR LIFT AND ACTING AS THE BASO 

1887
01:22:18,596 --> 01:22:20,198
COMPARTMENT AND WHERE WE ADDED 

1888
01:22:20,198 --> 01:22:22,000
THE LUNG EPITHELIAL CELLS IS 

1889
01:22:22,000 --> 01:22:23,001
WHERE THE AIR EXPOSURE IS 

1890
01:22:23,001 --> 01:22:29,173
OCCURRING.

1891
01:22:29,173 --> 01:22:30,875
THAT'S WHERE WE SEE THE 

1892
01:22:30,875 --> 01:22:41,152
DIFFERENTIATION.

1893
01:22:41,486 --> 01:22:43,054
HERE WE HAVE THE IMAGES OF THE 

1894
01:22:43,054 --> 01:22:46,257
TIME AND I CAN TELL YOU IT'S 

1895
01:22:46,257 --> 01:22:48,293
INCREASED DIFFERENTIATION IS 

1896
01:22:48,293 --> 01:22:53,464
DIFFICULT TO TELL BUT BY PHASED 

1897
01:22:53,464 --> 01:22:56,634
CONTRAST AND THE WAY WE KNOW 

1898
01:22:56,634 --> 01:22:58,369
THIS IS DEVELOPING WELL IS 

1899
01:22:58,369 --> 01:23:00,605
SEEING THE DARKENING OF 

1900
01:23:00,605 --> 01:23:02,340
CULTURING OVER TIME AND THE 

1901
01:23:02,340 --> 01:23:04,575
TUBULES IN THE ADJACENT CHANNELS

1902
01:23:04,575 --> 01:23:08,946
ARE REMAINING INTACT AND HOW 

1903
01:23:08,946 --> 01:23:09,580
THANK YOU KNOW THE CULTURE IS 

1904
01:23:09,580 --> 01:23:19,891
PROGRESSING WELL.

1905
01:23:20,158 --> 01:23:24,729
ANOTHER WAY TO EVALUATE THIS IS 

1906
01:23:24,729 --> 01:23:31,469
BY TRANS EPITHELIAL ELECTRICAL 

1907
01:23:31,469 --> 01:23:34,372
RESISTANCE AND DIP IN THE MEDIA 

1908
01:23:34,372 --> 01:23:38,276
WELLS AND CAN MEASURE THE TIER 

1909
01:23:38,276 --> 01:23:40,578
OVER TIME.

1910
01:23:40,578 --> 01:23:43,047
ACROSS MULTIPLE ITERATIONS WITH 

1911
01:23:43,047 --> 01:23:45,950
HE SEE ROBUSTLY OVER TIME THE 

1912
01:23:45,950 --> 01:23:50,288
TIERED VALUES CAN DECREASE WHICH

1913
01:23:50,288 --> 01:23:52,123
IS WHAT'S CONSISTENT WITH THE 

1914
01:23:52,123 --> 01:23:54,826
LITERATURE AND TO DO WITH THE 

1915
01:23:54,826 --> 01:23:55,326
DIFFERENTIATION PROCESS 

1916
01:23:55,326 --> 01:24:05,837
OCCURRING WITHIN THE CULTURES.

1917
01:24:08,639 --> 01:24:11,042
I HAVE A FEW IMMUNOSTAINING 

1918
01:24:11,042 --> 01:24:14,345
IMAGES AND PROJECTIONS AND 3-D 

1919
01:24:14,345 --> 01:24:14,712
RECONSTRUCTION.

1920
01:24:14,712 --> 01:24:19,050
SO WHAT WE SHOW HERE IS JUST 

1921
01:24:19,050 --> 01:24:21,886
SOME NICE BRONCHIAL EPITHELIAL 

1922
01:24:21,886 --> 01:24:27,058
ORGANIZATION AND DIFFERENT

1923
01:24:27,058 --> 01:24:30,428
DIFFERENTITION -- 

1924
01:24:30,428 --> 01:24:39,036
DIFFERENTIATION AND THE TUBULIN 

1925
01:24:39,036 --> 01:24:49,514
DEMONSTRATING THE CILIATED 

1926
01:24:56,788 --> 01:24:57,121
CELLS.

1927
01:24:57,121 --> 01:25:07,665
THAT'S THE LONG ORTHOGONAL VIEW.

1928
01:25:14,739 --> 01:25:20,111
I WANTED TO POINT OUT THE CY

1929
01:25:20,111 --> 01:25:24,148
CYTOKERATIN 5.

1930
01:25:24,148 --> 01:25:28,152
WE HAVE THE EXPRESSION OF THE 

1931
01:25:28,152 --> 01:25:28,319
5AC.

1932
01:25:28,319 --> 01:25:29,520
HERE WE ALSO EXPECT THAT WOULD 

1933
01:25:29,520 --> 01:25:32,089
BE KIND OF EXPRESSED MORE 

1934
01:25:32,089 --> 01:25:36,761
TOWARDS THE ATYPICAL SIDE AS 

1935
01:25:36,761 --> 01:25:38,329
WELL AND WHERE WE SEE SOME OF 

1936
01:25:38,329 --> 01:25:39,063
THE RED STAINING.

1937
01:25:39,063 --> 01:25:43,935
IF YOU CLICK ONE TIME WE HAVE A 

1938
01:25:43,935 --> 01:25:45,369
VIDEO MOVING THROUGH THE 

1939
01:25:45,369 --> 01:25:46,704
STAINING AND THROUGH THE DISEASE

1940
01:25:46,704 --> 01:25:47,038
STACKS.

1941
01:25:47,038 --> 01:25:57,582
THE FIRST IMAGE YOU SEE IS THE 

1942
01:26:00,384 --> 01:26:00,952
BOTTOM OF THE CULTURE.

1943
01:26:00,952 --> 01:26:05,423
WHAT'S IMPORTANT TO POINT OUT 

1944
01:26:05,423 --> 01:26:06,891
ABOUT THIS MODEL IS IT'S MORE 

1945
01:26:06,891 --> 01:26:09,060
COMPLEX AND HAS NOT ONLY THE 

1946
01:26:09,060 --> 01:26:10,928
EPITHELIUM AND ALSO PRESENT IS 

1947
01:26:10,928 --> 01:26:12,330
THE VASCULAR BED INDIRECT 

1948
01:26:12,330 --> 01:26:14,265
CONTACT WITH THE EPITHELIUM.

1949
01:26:14,265 --> 01:26:16,334
AND SO ON THE LEFT-HAND SIDE YOU

1950
01:26:16,334 --> 01:26:20,505
CAN SEE THOSE MORE ZOOMED OUT 

1951
01:26:20,505 --> 01:26:23,241
2-D IMAGES OF CD 31 

1952
01:26:23,241 --> 01:26:25,376
DEMONSTRATING THE VASCULATURE 

1953
01:26:25,376 --> 01:26:29,113
AND PRESENCE OF A ROBUST 

1954
01:26:29,113 --> 01:26:30,214
VASCULATURE BED AND IN THE 

1955
01:26:30,214 --> 01:26:32,483
CENTER CHANNEL WHERE THE 

1956
01:26:32,483 --> 01:26:33,284
EPITHELIUM ARE PRESENT AND THEN 

1957
01:26:33,284 --> 01:26:35,786
IF YOU MOVE TO THE CENTER IMAGES

1958
01:26:35,786 --> 01:26:39,957
WE CAN ACTUALLY SEE THAT 3-D 

1959
01:26:39,957 --> 01:26:41,859
RECONSTRUCTION AND VERY CLOSE 

1960
01:26:41,859 --> 01:26:45,730
ASSOCIATION OF THE EPITHELIUM 

1961
01:26:45,730 --> 01:26:53,738
WITH THE CD 31 VASCULAR BED.

1962
01:26:53,738 --> 01:26:56,474
YOU MAY BE WONDERING IS THE 

1963
01:26:56,474 --> 01:26:57,775
VASCULAR BED FUNCTIONAL?

1964
01:26:57,775 --> 01:26:59,944
AND IT ACTUALLY IS.

1965
01:26:59,944 --> 01:27:04,815
WE CAN EVALUATE THE 

1966
01:27:04,815 --> 01:27:09,620
PROFUSABILITY OF THE VASCULAR 

1967
01:27:09,620 --> 01:27:14,292
BED AND FLOW DYE AND EVALUATE 

1968
01:27:14,292 --> 01:27:18,696
THIS BY ADDING THE DYE TO THE 

1969
01:27:18,696 --> 01:27:20,464
MEDIA CHANNEL AND IF IT'S 

1970
01:27:20,464 --> 01:27:26,103
PROFUSABLE YOU'LL SEE WHAT YOU 

1971
01:27:26,103 --> 01:27:35,046
SEE HERE WHICH IS THE VASCULAR 

1972
01:27:35,046 --> 01:27:35,947
BED LIGHTING UP.

1973
01:27:35,947 --> 01:27:46,457
IT'S FUNCTIONAL IN BOTH CASES.

1974
01:27:47,425 --> 01:27:49,460
YOU CAN REPEAT IT OVER TIME.

1975
01:27:49,460 --> 01:27:51,762
MAYBE YOU WANT IT FLOW SOMETHING

1976
01:27:51,762 --> 01:27:55,066
THROUGH LIKE IMMUNE CELLS AND 

1977
01:27:55,066 --> 01:27:56,901
BEFORE YOU FLOW THROUGH SOME 

1978
01:27:56,901 --> 01:27:57,702
VERY APPRECIATION IMMUNE CELLS 

1979
01:27:57,702 --> 01:27:59,971
YOU HAVE PERHAPS YOU WANT TO 

1980
01:27:59,971 --> 01:28:02,440
UNDERSTAND IS THE VASCULAR BED 

1981
01:28:02,440 --> 01:28:04,408
PROFUSABLE AND CAN PERFORM THE 

1982
01:28:04,408 --> 01:28:05,309
ASSAY AND ONCE YOU'RE CONFIDENT 

1983
01:28:05,309 --> 01:28:08,479
YOU HAVE A PROFUSABLE BED YOU 

1984
01:28:08,479 --> 01:28:09,146
CAN START ADDING YOUR MORE 

1985
01:28:09,146 --> 01:28:19,056
APPRECIATION MATERIAL.

1986
01:28:19,056 --> 01:28:20,558
WE CAN UNDERSTAND THINGS LIKE 

1987
01:28:20,558 --> 01:28:22,526
VESSEL AREA AND TOTAL NUMBER OF 

1988
01:28:22,526 --> 01:28:23,060
JUNCTIONS.

1989
01:28:23,060 --> 01:28:24,895
IF YOU'RE INTERESTED IN 

1990
01:28:24,895 --> 01:28:27,064
UNDERSTANDING HOW COMPOUND WERE 

1991
01:28:27,064 --> 01:28:28,299
POTENTIALLY INFLUENCING THAT 

1992
01:28:28,299 --> 01:28:38,542
VASCULAR BED.

1993
01:28:39,210 --> 01:28:40,444
WE FLOWED THE DYE THROUGH BUT 

1994
01:28:40,444 --> 01:28:44,882
WERE ABLE TO FLOW THROUGH IMMUNE

1995
01:28:44,882 --> 01:28:47,051
CELLS WE USED AND LABELLED WITH 

1996
01:28:47,051 --> 01:28:47,718
CELL TRACKER.

1997
01:28:47,718 --> 01:28:48,419
IF YOU CLICK ONE TIME YOU'LL SEE

1998
01:28:48,419 --> 01:28:57,161
A VIDEO ON THE LEFT-HAND SIDE.

1999
01:28:57,161 --> 01:28:59,330
THE IMMUNE CELLS FLOW ACROSS THE

2000
01:28:59,330 --> 01:29:03,034
STOP.

2001
01:29:03,034 --> 01:29:13,177
-- TOP.

2002
01:29:20,685 --> 01:29:25,056
YOU SEE THE IMMUNE CELLS IN ONE 

2003
01:29:25,056 --> 01:29:28,392
DIRECTION ON THE TOP AND FLOWING

2004
01:29:28,392 --> 01:29:30,227
THROUGH THE VASCULAR BED AND ON 

2005
01:29:30,227 --> 01:29:34,031
THE RIGHT SIDE IS THE TIME LAPSE

2006
01:29:34,031 --> 01:29:37,068
IMAGING COMPLIED TOGETHER IN A 

2007
01:29:37,068 --> 01:29:38,869
SINGLE IMAGE.

2008
01:29:38,869 --> 01:29:40,705
EACH COLOR REPRESENTING A 

2009
01:29:40,705 --> 01:29:41,338
SPECIFIC TIME WHAT PATH THE 

2010
01:29:41,338 --> 01:29:45,810
IMMUNE CELL WAS TAKING.

2011
01:29:45,810 --> 01:29:47,678
FOR EXAMPLE, EARLY ON THOSE ARE 

2012
01:29:47,678 --> 01:29:48,846
MORE THE PURPLE BLUE.

2013
01:29:48,846 --> 01:29:50,915
YOU CAN SEE ESPECIALLY ON THE 

2014
01:29:50,915 --> 01:29:52,583
LEFT-HAND SIDE OF THE VASCULAR 

2015
01:29:52,583 --> 01:29:55,052
BED THERE WERE CELLS THAT TOOK 

2016
01:29:55,052 --> 01:29:57,655
THAT PATH AND LATER ON YOUR 

2017
01:29:57,655 --> 01:30:00,124
ACTUALLY MORE IN THE YELLOW 

2018
01:30:00,124 --> 01:30:01,625
WHITE AND SEE WHERE THE IMMUNE 

2019
01:30:01,625 --> 01:30:03,194
CELLS WERE LATER IN THE 

2020
01:30:03,194 --> 01:30:13,471
PROFUSION ASSAY.

2021
01:30:19,110 --> 01:30:23,314
I WANT TO DEMONSTRATE THE 

2022
01:30:23,314 --> 01:30:26,951
FEASIBILITY OF MODELLING 

2023
01:30:26,951 --> 01:30:27,752
PULMONARY DISEASE.

2024
01:30:27,752 --> 01:30:30,020
WE MODELLED PULMONARY FIBROSIS 

2025
01:30:30,020 --> 01:30:31,889
AND APPLIED A FIBROTIC COCKTAIL 

2026
01:30:31,889 --> 01:30:33,758
TO THE CULTURES AND EVALUATED 

2027
01:30:33,758 --> 01:30:37,194
THEIR BED ON THE VASCULAR BED 

2028
01:30:37,194 --> 01:30:38,763
PROFUSABILITY AND DOES A COUPLE 

2029
01:30:38,763 --> 01:30:41,298
TYPES OF EXPOSURE.

2030
01:30:41,298 --> 01:30:42,466
ON THE LEFT SIDE WE DIDN'T HAVE 

2031
01:30:42,466 --> 01:30:52,910
ANYTHING IN THE CULTURES.

2032
01:30:59,717 --> 01:31:02,419
AND ADDED DIRECT "ON TOP OF THE 

2033
01:31:02,419 --> 01:31:04,155
EPITHELIUM TO SEE WHAT THE 

2034
01:31:04,155 --> 01:31:05,756
DIFFERENCE IS BETWEEN THE TWO 

2035
01:31:05,756 --> 01:31:10,694
EXPOSURES ROOTS AND ON THE RIGHT

2036
01:31:10,694 --> 01:31:13,164
SIDE ADDED A FIBROSIS INHIBITOR 

2037
01:31:13,164 --> 01:31:16,333
TO SEE IF WE COULD PREVENT THE 

2038
01:31:16,333 --> 01:31:16,901
FIBROTIC EFFECT.

2039
01:31:16,901 --> 01:31:21,138
WHAT WE OBSERVED IN BOTH CASES 

2040
01:31:21,138 --> 01:31:23,040
IN THE EXPOSURE CONDITIONS WE 

2041
01:31:23,040 --> 01:31:26,143
SEE A LOSS OF VASCULAR BED 

2042
01:31:26,143 --> 01:31:28,979
PROFUSABILITY THAT WAS PREVENTED

2043
01:31:28,979 --> 01:31:31,148
BY THE PERFUSION OF THE 

2044
01:31:31,148 --> 01:31:41,325
INHIBITOR.

2045
01:31:54,605 --> 01:31:59,076
EYE  AND THERE'S A TRANSITION 

2046
01:31:59,076 --> 01:31:59,677
OCCURRING WHICH IS COMMON IN 

2047
01:31:59,677 --> 01:32:08,219
FIBROSIS.

2048
01:32:08,219 --> 01:32:12,256
I THINK IT WRAPS IT UP FOR MY 

2049
01:32:12,256 --> 01:32:14,258
UPDATE ON THE MIMETAS CHIP 

2050
01:32:14,258 --> 01:32:14,491
MODELS.

2051
01:32:14,491 --> 01:32:15,860
I WANTED TO EMPHASIZE AND LET 

2052
01:32:15,860 --> 01:32:18,229
YOU KNOW THAT AT MIMETAS WE HAVE

2053
01:32:18,229 --> 01:32:20,364
LOTS OF DIFFERENT SOLUTIONS FOR 

2054
01:32:20,364 --> 01:32:25,870
YOUR RESEARCH FROM DISCOVERY, 

2055
01:32:25,870 --> 01:32:27,972
LEED OPTIMIZATION AND SUPPORT.

2056
01:32:27,972 --> 01:32:30,808
WE OFFER THE PLATES AND FOLKS 

2057
01:32:30,808 --> 01:32:33,677
CAN BUY THEM THEMSELVES AND 

2058
01:32:33,677 --> 01:32:39,016
BRING THEM IN YOUR LABORATORY 

2059
01:32:39,016 --> 01:32:39,650
AND FAIRLY STRAIGHTFORWARD AND 

2060
01:32:39,650 --> 01:32:42,486
WE CAN WORK WITH YOU AND DO MORE

2061
01:32:42,486 --> 01:32:43,888
WORK ON OUR SIDE AND IF YOU 

2062
01:32:43,888 --> 01:32:47,057
PREFER NOT TO BRING IT IN HOUSE 

2063
01:32:47,057 --> 01:32:57,134
TO TO THAT.

2064
01:32:57,134 --> 01:32:58,836
I WANT TO THANK OUR COLLEAGUES 

2065
01:32:58,836 --> 01:33:03,274
AND THE WORK FUNDED BY THE EU 

2066
01:33:03,274 --> 01:33:04,141
GRANT AND THANK YOU FOR YOUR 

2067
01:33:04,141 --> 01:33:05,442
ATTENTION AND LOOK FORWARD TO 

2068
01:33:05,442 --> 01:33:07,044
THE PANEL DISCUSSION.

2069
01:33:07,044 --> 01:33:13,918
THANK YOU.

2070
01:33:13,918 --> 01:33:15,486
>> THANK YOU, KRISTIN.

2071
01:33:15,486 --> 01:33:16,287
GREAT TALK.

2072
01:33:16,287 --> 01:33:17,955
THE FINAL PRESENTATION WILL BE 

2073
01:33:17,955 --> 01:33:19,490
GIVEN BY JOHN ENGLEHARDT FROM 

2074
01:33:19,490 --> 01:33:21,759
THE UNIVERSITY OF IOWA.

2075
01:33:21,759 --> 01:33:22,826
JOHN, TAKE IT AWAY.

2076
01:33:22,826 --> 01:33:25,396
>> THANKS, JOHN.

2077
01:33:25,396 --> 01:33:26,563
I'M JOHN ENGLEHARDT CURRENTLY AT

2078
01:33:26,563 --> 01:33:28,565
THE UNIVERSITY OF IOWA.

2079
01:33:28,565 --> 01:33:30,567
WE'LL BE TRANSITIONING THIS 

2080
01:33:30,567 --> 01:33:33,337
SUMMER TO THE UNIVERSITY OF 

2081
01:33:33,337 --> 01:33:35,072
ALABAMA BIRMINGHAM.

2082
01:33:35,072 --> 01:33:40,311
THESE ARE MY DISCLOSURES.

2083
01:33:40,311 --> 01:33:42,947
ONE RELEVANT TO WHAT IS RELEVANT

2084
01:33:42,947 --> 01:33:43,147
TODAY.

2085
01:33:43,147 --> 01:33:45,916
I'LL TALK ABOUT THE UTILITY OF 

2086
01:33:45,916 --> 01:33:49,520
FERRETS TO MODEL LUNG DISEASE.

2087
01:33:49,520 --> 01:33:52,056
AND THE BIOLOGICAL BASE OF THAT 

2088
01:33:52,056 --> 01:33:54,258
CELL BIOLOGY IN THE LUNG AND HOW

2089
01:33:54,258 --> 01:33:56,894
THAT IMPACTS THE PROGRESSION OF 

2090
01:33:56,894 --> 01:33:58,295
CERTAIN DISEASES AND 

2091
01:33:58,295 --> 01:34:07,338
APPLICATIONS TO TEST THERAPIES.

2092
01:34:07,338 --> 01:34:10,341
WHAT YOU'VE WRITTEN HERE ARE 

2093
01:34:10,341 --> 01:34:14,979
DISEASE MODELS IN FERRET SOME 

2094
01:34:14,979 --> 01:34:15,946
CONSTRUCTED WITH THE NATIONAL 

2095
01:34:15,946 --> 01:34:20,484
RESOURCE I DIRECT AND SOME ARE 

2096
01:34:20,484 --> 01:34:21,151
ENVIRONMENTAL STIMULUS INJURY 

2097
01:34:21,151 --> 01:34:23,721
INCLUDING CYSTIC FIBROSIS, 

2098
01:34:23,721 --> 01:34:25,789
ASTHMA AND SMOKING MODELS HAVE 

2099
01:34:25,789 --> 01:34:29,994
BEEN VERY SUCCESSFUL AT THE 

2100
01:34:29,994 --> 01:34:40,371
UNIVERSITY OF ALABAMA.

2101
01:34:50,848 --> 01:34:55,052
 BRONCHITIS AND EMPHA SEE 345 

2102
01:34:55,052 --> 01:34:59,056
RARCHBD I'LL DISCUSS THE BIOLOGY

2103
01:34:59,056 --> 01:35:04,628
BETWEEN IPF OR GLIAL MODELS IN 

2104
01:35:04,628 --> 01:35:11,402
THE FERRET AND THERAPIES IN THE 

2105
01:35:11,402 --> 01:35:11,635
LATTER.

2106
01:35:11,635 --> 01:35:18,409
IT'S IMPORTANT WHEN COMPARING 

2107
01:35:18,409 --> 01:35:22,479
DIFFERENCES IN THE TYPES OF CELL

2108
01:35:22,479 --> 01:35:27,051
TYPES THAT SPAN THE AIRWAY, 

2109
01:35:27,051 --> 01:35:31,055
THEIR DISTRIBUTION AS WELL AS 

2110
01:35:31,055 --> 01:35:33,824
ANATOMICAL SITES.

2111
01:35:33,824 --> 01:35:39,063
MUCOSA GLANDS AND THE IONOCYTES 

2112
01:35:39,063 --> 01:35:44,668
THAT ARE ASSOCIATED WITH GLANDS 

2113
01:35:44,668 --> 01:35:46,937
EXPAND AND DELAYS NO CARTILAGE 

2114
01:35:46,937 --> 01:35:50,607
WIN THE INTERLOPIN AIRWAYS AND 

2115
01:35:50,607 --> 01:35:54,211
THE IONOCYTES ARE LOCATED ATHET 

2116
01:35:54,211 --> 01:35:56,580
PROXIMAL END TO THE TRACHEA AND 

2117
01:35:56,580 --> 01:35:59,450
RELEVANT TO DISEASES LIKE IPF 

2118
01:35:59,450 --> 01:36:07,057
AND DISTRIBUTION OF BASAL CELLS 

2119
01:36:07,057 --> 01:36:08,158
EXTEND TO THE TERMINAL 

2120
01:36:08,158 --> 01:36:09,259
BRONCHIOLES GOING TO THE AIRWAYS

2121
01:36:09,259 --> 01:36:11,161
IN RODENTS.

2122
01:36:11,161 --> 01:36:14,998
AND RODENTS DON'T HAVE 

2123
01:36:14,998 --> 01:36:20,504
RESPIRATORY BRONCHIOLES.

2124
01:36:20,504 --> 01:36:27,044
AND SO THESE DIFFERENCES IN CELL

2125
01:36:27,044 --> 01:36:31,048
BIOLOGY AFFECT PROGRESSION OF 

2126
01:36:31,048 --> 01:36:35,052
CYSTIC FIBROSIS IN THE TEE 

2127
01:36:35,052 --> 01:36:37,621
EFFECTIVE GENE IN THE FTR GENE.

2128
01:36:37,621 --> 01:36:43,026
THE RIGHT TWO PRANLS AND THE 

2129
01:36:43,026 --> 01:36:45,129
MUCOSA GLAND AND THE LUMEN OF 

2130
01:36:45,129 --> 01:36:47,030
THE AIRWAY AND THE MICE DON'T 

2131
01:36:47,030 --> 01:36:52,836
GET LUNG DISEASE.

2132
01:36:52,836 --> 01:36:54,538
THERE'S DIFFERENCES IN THE 

2133
01:36:54,538 --> 01:36:59,076
DISTAL CONDUCTING AIRWAYS AND 

2134
01:36:59,076 --> 01:37:01,145
RESPIRATORY BRONCHIOLES BETWEEN 

2135
01:37:01,145 --> 01:37:04,515
RODENTS AND HUMANS AND FERRETS 

2136
01:37:04,515 --> 01:37:05,883
AS WELL AS PIGS.

2137
01:37:05,883 --> 01:37:11,054
THE AREAS CONTAIN BASAL CELLS AS

2138
01:37:11,054 --> 01:37:22,032
WELL AS A CLUB SECRETSECRETATORS

2139
01:37:22,032 --> 01:37:23,667
AND SHOWN TO BE DIFFERENT 

2140
01:37:23,667 --> 01:37:28,305
BETWEEN RODENTS AND HUMANS 

2141
01:37:28,305 --> 01:37:35,045
INCLUDING AND THE CELL TYPES 

2142
01:37:35,045 --> 01:37:38,949
HAVE BEEN SHOWN TO HAVE 

2143
01:37:38,949 --> 01:37:49,493
SPECIALIZED PROGENITOR CAPACITY.

2144
01:37:56,166 --> 01:37:58,669
YOU SEE DOUBLE TRANSGENIC MICE 

2145
01:37:58,669 --> 01:38:01,838
ON THE LEFT AND THIS CAN BE 

2146
01:38:01,838 --> 01:38:05,375
STIMULATED WITH TAMOXIFEN TO 

2147
01:38:05,375 --> 01:38:07,711
INDUCE A RECORDER SWITCH 

2148
01:38:07,711 --> 01:38:11,048
SWITCHING TO GFP.

2149
01:38:11,048 --> 01:38:17,521
THIS IS THE ALPHA SMA GENE FATE 

2150
01:38:17,521 --> 01:38:21,225
MAPPING BLOOD VESSELS, VASCULAR 

2151
01:38:21,225 --> 01:38:22,159
SMOOTH MUSCLE AND SMOOTH MUSCLE 

2152
01:38:22,159 --> 01:38:28,332
IN THE DISTAL AIRWAY AT THE 

2153
01:38:28,332 --> 01:38:32,970
BRONCHIOLE ALVEOLAR JUNCTION AND

2154
01:38:32,970 --> 01:38:35,739
WHERE IN MICE A STEM CELL EXISTS

2155
01:38:35,739 --> 01:38:38,675
THAT REPAIRS THE DISTAL AIRWAY 

2156
01:38:38,675 --> 01:38:39,209
FOLLOWING INJURY.

2157
01:38:39,209 --> 01:38:46,116
THE SAME MODEL GENERATED WITHIN 

2158
01:38:46,116 --> 01:38:51,221
PER ITS AND THE DISTAL AIRWAY IS

2159
01:38:51,221 --> 01:39:01,398
WHERE THE TERMINAL BRONCHIOLE 

2160
01:39:01,398 --> 01:39:04,501
CELL EXISTS AND ONE THE DRIVER 

2161
01:39:04,501 --> 01:39:09,573
THE SMOOTH MUSCLE BELOW THE CLUB

2162
01:39:09,573 --> 01:39:11,041
CELLS VERY DIFFERENT PICTURE 

2163
01:39:11,041 --> 01:39:13,610
ANATOMICALLY AND THIS IMPACTS 

2164
01:39:13,610 --> 01:39:16,280
THE PROGRESSION OF PULMONARY 

2165
01:39:16,280 --> 01:39:16,847
FIBROSIS FOLLOWING MICE IN 

2166
01:39:16,847 --> 01:39:26,990
INJURY.

2167
01:39:44,041 --> 01:39:46,376
AND LOOKING AT THE RISK IN 

2168
01:39:46,376 --> 01:39:49,379
PULMONARY CARTILAGE AND ONE SEES

2169
01:39:49,379 --> 01:39:54,851
THE THREE LARGEST SPECIES, 

2170
01:39:54,851 --> 01:40:03,627
FERRET,  AND PIG ARE CLOSE TO 

2171
01:40:03,627 --> 01:40:05,128
HUMAN LUNG.

2172
01:40:05,128 --> 01:40:06,697
I'LL TALK BRIEFLY ABOUT THE 

2173
01:40:06,697 --> 01:40:08,565
INJURY MODEL IN THE FERRET IN 

2174
01:40:08,565 --> 01:40:11,968
WHICH WHAT WE'RE LOOKING AT HERE

2175
01:40:11,968 --> 01:40:17,307
IS SINGLE NUC SEQ OF EPITHELIAL 

2176
01:40:17,307 --> 01:40:21,211
CELLS AND SHOWING THE CELL TYPES

2177
01:40:21,211 --> 01:40:25,515
ONE CAN SEE FOLLOWING GLIOMYCIN 

2178
01:40:25,515 --> 01:40:27,284
INJURY AND THE CHANGES THAT 

2179
01:40:27,284 --> 01:40:31,822
OCCUR.

2180
01:40:31,822 --> 01:40:40,464
YOU CAN SEE TWO CELL TYPE AND 

2181
01:40:40,464 --> 01:40:44,668
BASALOID LIKE CELL POORLY MODELS

2182
01:40:44,668 --> 01:40:45,268
IN MICE COMPARED TO IPF IN 

2183
01:40:45,268 --> 01:40:53,810
HUMAN.

2184
01:40:53,810 --> 01:40:55,078
AND THERE'S A REPAIR IN THE 

2185
01:40:55,078 --> 01:40:57,547
DISTAL AIR SPACES.

2186
01:40:57,547 --> 01:41:03,019
THIS PHENOTYPE OF TP63 CARE 7 

2187
01:41:03,019 --> 01:41:05,922
POSITIVE MARK BY THE ARROWS DOES

2188
01:41:05,922 --> 01:41:08,058
NOT EXIST IN MOUSE LUNG AND IT'S

2189
01:41:08,058 --> 01:41:10,660
THOUGHT THIS MIGHT CONTRIBUTE TO

2190
01:41:10,660 --> 01:41:15,031
DIFFERENCES IN THE PERSISTENCE 

2191
01:41:15,031 --> 01:41:22,439
OF INJURY.

2192
01:41:22,439 --> 01:41:26,276
THIS IS THE RESULT OF FIBROSIS 

2193
01:41:26,276 --> 01:41:32,282
AND THE ALVEOLI AND THE PROXIMAL

2194
01:41:32,282 --> 01:41:35,051
AIRWAY WITH THE NOTCH SIGNALLING

2195
01:41:35,051 --> 01:41:37,654
AND HIGHLIGHTING THE BASALOID 

2196
01:41:37,654 --> 01:41:39,923
CELLS WITH THE YELLOW ARROWS AND

2197
01:41:39,923 --> 01:41:42,292
SHOWN WHAT THE NORMAL AIRWAY 

2198
01:41:42,292 --> 01:41:43,927
LOOKS LIKE ON THE PANEL A.

2199
01:41:43,927 --> 01:41:47,564
THAT'S A PORTION OF THE LUNG 

2200
01:41:47,564 --> 01:41:52,803
SECTION AND THE MORE DYSPLASTIC 

2201
01:41:52,803 --> 01:41:53,770
PROXIMALIZATION WHERE THE 

2202
01:41:53,770 --> 01:41:57,040
AIRWAYS INVADE THE AIR SPACES 

2203
01:41:57,040 --> 01:41:58,675
AND EVENTUALLY LEAD TO 

2204
01:41:58,675 --> 01:42:09,186
IRREVERSIBLE DAMAGE AND DEATH.

2205
01:42:19,029 --> 01:42:21,465
AND THE BRONCHIAL SMOOTH MUSCLE 

2206
01:42:21,465 --> 01:42:26,136
WE BELIEVE CONTRIBUTE TO 

2207
01:42:26,136 --> 01:42:30,207
MYOFIBROBLASTS AND THIS IS SHOWN

2208
01:42:30,207 --> 01:42:35,045
IN THE FERRET PRIOR TO BLEOMYCIN

2209
01:42:35,045 --> 01:42:38,648
INJURY AND SEE THE ASMA AND THE 

2210
01:42:38,648 --> 01:42:40,917
NEXT AIR I WANT TO TALK ABOUT 

2211
01:42:40,917 --> 01:42:43,820
HAS TO TO WITH UTILIZING THE 

2212
01:42:43,820 --> 01:42:46,890
FERRET MODEL FOR TESTING GENE 

2213
01:42:46,890 --> 01:42:49,426
THERAPIES OR RNA THERAPIES FOR 

2214
01:42:49,426 --> 01:42:50,427
CYSTIC FIBROSIS.

2215
01:42:50,427 --> 01:42:55,932
WE HEARD ABOUT THE CFTR CHANNEL 

2216
01:42:55,932 --> 01:42:58,201
WHICH TRANSPORTS BY CARBONATE 

2217
01:42:58,201 --> 01:42:58,835
AND CHLORIDE IN THE ABSENCE OF 

2218
01:42:58,835 --> 01:43:03,006
THE FUNCTION.

2219
01:43:03,006 --> 01:43:05,542
AND THE AIRWAY SURFACE FLUID 

2220
01:43:05,542 --> 01:43:12,249
BECOMES DEHIYDRDEHYDRATED THE P 

2221
01:43:12,249 --> 01:43:16,319
LOWER AND VISCOSITY IS CLEARED 

2222
01:43:16,319 --> 01:43:19,656
AND LEADS TO INFECTION.

2223
01:43:19,656 --> 01:43:23,894
THE CELL TYPES MARK IN THE BOX 

2224
01:43:23,894 --> 01:43:26,396
HERE HIGHLIGHT DIVERGENT LEVELS 

2225
01:43:26,396 --> 01:43:29,566
OF MRNA EXPRESSION AND PROTEIN 

2226
01:43:29,566 --> 01:43:33,036
EXPRESSION WITHIN THE CELLS FROM

2227
01:43:33,036 --> 01:43:38,108
GREATER 15% TO 90% EXPRESS CTFR.

2228
01:43:38,108 --> 01:43:41,244
IT'S UNCLEAR WHETHER OR NOT THE 

2229
01:43:41,244 --> 01:43:42,946
CFTR EXPRESSION FROM A GENE 

2230
01:43:42,946 --> 01:43:44,614
THERAPY STANDPOINT HAS TO 

2231
01:43:44,614 --> 01:43:51,021
RECAPITULATE THE ENDOGENOUS 

2232
01:43:51,021 --> 01:43:52,455
EXPRESSION PATTERN TO REVERSE 

2233
01:43:52,455 --> 01:43:52,689
DISEASE.

2234
01:43:52,689 --> 01:43:53,323
THAT'S ONE THING I'LL TOUCH ON 

2235
01:43:53,323 --> 01:44:03,433
HERE.

2236
01:44:06,970 --> 01:44:12,142
AND WE LOOKED AT THE KNOCKOUT 

2237
01:44:12,142 --> 01:44:16,680
511D IN THE ABSENCE OF THE FCFR 

2238
01:44:16,680 --> 01:44:19,583
MODULATOR THAT CAN CORRECTLY 

2239
01:44:19,583 --> 01:44:22,752
RESTORE GAITING OF THE MUTANT 

2240
01:44:22,752 --> 01:44:28,658
CHANNEL -- GATING OF THE MUTANT 

2241
01:44:28,658 --> 01:44:29,726
CHANNEL IS MORE USEFUL BECAUSE 

2242
01:44:29,726 --> 01:44:35,632
WE CAN TURN IT ON AND OFF AND 

2243
01:44:35,632 --> 01:44:39,636
HAVE IT RELEVANT TO GENE THERAPY

2244
01:44:39,636 --> 01:44:43,506
INCLUDING CAN IT REVERSE DISEASE

2245
01:44:43,506 --> 01:44:45,342
OR PROGRESS THE ONSET OF 

2246
01:44:45,342 --> 01:44:48,878
DISEASE.

2247
01:44:48,878 --> 01:44:51,348
NOW, THIS IS THE TYPICAL WORK 

2248
01:44:51,348 --> 01:44:53,917
FLOW WHEN MY GROUP INTERACTS 

2249
01:44:53,917 --> 01:44:55,752
WITH OTHERS FROM THE OTHER SIDE 

2250
01:44:55,752 --> 01:44:57,387
TO EVALUATE FERRETS.

2251
01:44:57,387 --> 01:45:02,892
SOME OF THE FIRST MOST IMPORTANT

2252
01:45:02,892 --> 01:45:05,261
QUESTIONS AND IF ONE'S 

2253
01:45:05,261 --> 01:45:07,030
CONSIDERING A VIRUS WHETHER OR 

2254
01:45:07,030 --> 01:45:11,701
NOT THEIR TROPISM DIFFERENCES 

2255
01:45:11,701 --> 01:45:13,370
BETWEEN HUMAN AND FERRET AND 

2256
01:45:13,370 --> 01:45:16,473
LESS CONCERN FOR LNPs.

2257
01:45:16,473 --> 01:45:18,508
WE THEN TYPICALLY MOVE TO 

2258
01:45:18,508 --> 01:45:23,046
REPORTER AND INTERFACE AND 

2259
01:45:23,046 --> 01:45:25,415
CULTURES THAT -- WHERE YOU CAN 

2260
01:45:25,415 --> 01:45:27,083
EXPRESS CREE AND DRIVE THE 

2261
01:45:27,083 --> 01:45:30,286
REPORTER EXPRESSION LIKE I 

2262
01:45:30,286 --> 01:45:33,056
SHOWED IN VIVO TO GFP AS A 

2263
01:45:33,056 --> 01:45:33,356
SENSOR.

2264
01:45:33,356 --> 01:45:36,092
AND THE NEXT LEVEL WOULD BE 

2265
01:45:36,092 --> 01:45:38,561
FUNCTIONAL CORRECTION USING A 

2266
01:45:38,561 --> 01:45:40,797
CFTR WITHIN THE VIRAL VECTOR OR 

2267
01:45:40,797 --> 01:45:44,634
MRNA AND LOOK FOR 

2268
01:45:44,634 --> 01:45:45,368
ELECTROPHYSIOLOGIC COLLECTION IN

2269
01:45:45,368 --> 01:45:50,774
AIR INTERFACE CULTURES.

2270
01:45:50,774 --> 01:45:53,810
THEN MOVING IN VIVO, I PREFER TO

2271
01:45:53,810 --> 01:45:58,448
USE THE UNEQUIVOCAL DETECTION 

2272
01:45:58,448 --> 01:46:00,550
USING THE CREE REPORTER AND ASK 

2273
01:46:00,550 --> 01:46:03,620
QUESTIONS WHAT CELL TYPES ARE 

2274
01:46:03,620 --> 01:46:04,220
TARGETED.

2275
01:46:04,220 --> 01:46:07,023
WHAT CELL TYPES USING PHENOTYPIC

2276
01:46:07,023 --> 01:46:09,726
MARKERS.

2277
01:46:09,726 --> 01:46:11,995
AND THEN THE MORE DIFFICULT 

2278
01:46:11,995 --> 01:46:15,031
QUESTION THEN MOVE IN VIVO INTO 

2279
01:46:15,031 --> 01:46:17,233
THE DISEASE CYSTIC FIBROSIS 

2280
01:46:17,233 --> 01:46:25,475
USING HUMAN CFTR AND THERE'S 

2281
01:46:25,475 --> 01:46:27,777
SHORTER TERM AND ASK FOR DISEASE

2282
01:46:27,777 --> 01:46:33,349
REVERSAL AND A CF FERRET WITH 

2283
01:46:33,349 --> 01:46:36,553
ROSEA TG WITH CREE ASKING THE 

2284
01:46:36,553 --> 01:46:37,987
VIRAL VECTOR OR mRNA CAN 

2285
01:46:37,987 --> 01:46:47,030
PENETRATE THE MUE CAS BARRIER.

2286
01:46:47,030 --> 01:46:49,365
I'LL DISCUSS TESTS WITH THE 

2287
01:46:49,365 --> 01:46:53,970
LIPID NANO PARTICLE.

2288
01:46:53,970 --> 01:46:56,539
SOME OF THE FIRST STUDIES WE DID

2289
01:46:56,539 --> 01:46:57,941
WERE IN AIR INTERFACE CULTURES 

2290
01:46:57,941 --> 01:47:01,277
AND SEE IN THE UPPER RIGHT HAND 

2291
01:47:01,277 --> 01:47:07,050
CHANNEL THE SWITCH OF CELLS TO 

2292
01:47:07,050 --> 01:47:09,152
CFTR POSITIVE AND THE ONES 

2293
01:47:09,152 --> 01:47:11,254
TARGETED IN THE BOTTOM PANEL.

2294
01:47:11,254 --> 01:47:14,524
YOU CAN SEE THIS IS MEASUREMENTS

2295
01:47:14,524 --> 01:47:20,530
WHERE THE BLUE WERE LOOKING AT A

2296
01:47:20,530 --> 01:47:25,301
COMPARISON BETWEEN A RELEVANT 

2297
01:47:25,301 --> 01:47:28,037
RNA AND COMPARED TO WILD TYPE 

2298
01:47:28,037 --> 01:47:28,371
UNTRANSFECTED.

2299
01:47:28,371 --> 01:47:31,040
YOU CAN SEE WITH THE HUMAN CFTR 

2300
01:47:31,040 --> 01:47:34,010
WHAT LOOKS AT THE BLUE ARROWS 

2301
01:47:34,010 --> 01:47:37,914
THE ACTIVATION OF CFTR IS ABOUT 

2302
01:47:37,914 --> 01:47:41,785
50% AND INHIBITION WITH THE CFTR

2303
01:47:41,785 --> 01:47:43,586
INHIBITOR THAT ACTS ON FERRET 

2304
01:47:43,586 --> 01:47:47,023
AND YOU CAN SEE THE LIPID NANO 

2305
01:47:47,023 --> 01:47:48,525
PARTICLE CORRECTS SHORT CIRCUIT 

2306
01:47:48,525 --> 01:47:51,027
CURRENT FROM 50% TO 75% 

2307
01:47:51,027 --> 01:47:55,031
APPROXIMATELY.

2308
01:47:55,031 --> 01:47:59,068
NEXT WE MOVED IN VIVO WITHIN 

2309
01:47:59,068 --> 01:48:03,039
FERRET, TRANSGENIC FERRET AND 

2310
01:48:03,039 --> 01:48:06,910
DEMONSTRATED THE TRANSFECTING 

2311
01:48:06,910 --> 01:48:07,110
CREE.

2312
01:48:07,110 --> 01:48:08,244
WE SEE THE HIGH LEVEL 

2313
01:48:08,244 --> 01:48:10,647
TRANSFECTION OF THE TRACHEA 

2314
01:48:10,647 --> 01:48:12,882
CONVERSION TO EGFP AND THE 

2315
01:48:12,882 --> 01:48:19,322
INTERLOPER AIRWAYS ON THE RIGHT.

2316
01:48:19,322 --> 01:48:26,095
AND -- INTERACHAL AIRWAYS AND 

2317
01:48:26,095 --> 01:48:34,470
SEE THE INTRALOBAR AIRWAY.

2318
01:48:34,470 --> 01:48:38,041
AND CAN TEST FOR TRANSFECTION OF

2319
01:48:38,041 --> 01:48:40,109
CELLS AND GUIDE THE PHENOTYPING.

2320
01:48:40,109 --> 01:48:42,178
WE'RE LOOKING AT A FERRET AND 

2321
01:48:42,178 --> 01:48:51,020
THE AIRWAY IN THE TRACHEA ON THE

2322
01:48:51,020 --> 01:48:53,556
TOP.

2323
01:48:53,556 --> 01:48:58,194
AND LOCALIZATION OF CFTC.

2324
01:48:58,194 --> 01:49:01,030
AND LOCALIZATION OF CFTR IS 

2325
01:49:01,030 --> 01:49:06,836
SHOWN ON THE RIGHT WITH A LUNAR 

2326
01:49:06,836 --> 01:49:07,070
VEHICLE.

2327
01:49:07,070 --> 01:49:12,041
THE YELLOW AIRS HAVE HIGH 

2328
01:49:12,041 --> 01:49:13,509
TRANSFECTION AND SEE THE 

2329
01:49:13,509 --> 01:49:14,844
POTENTIAL SECRETORY CELL IN THE 

2330
01:49:14,844 --> 01:49:16,980
CENTER AND ONCE THE INCREASED 

2331
01:49:16,980 --> 01:49:21,284
ABUNDANCE OF CFTC AT THE 

2332
01:49:21,284 --> 01:49:25,588
MEMBRANE AS CO-LOCALIZED WITH 

2333
01:49:25,588 --> 01:49:32,295
THE CELLS.

2334
01:49:32,295 --> 01:49:35,031
SO, WHAT CELL TYPES ARE 

2335
01:49:35,031 --> 01:49:35,331
TRANSFECTED?

2336
01:49:35,331 --> 01:49:38,835
SO IN THESE PANELS WE'RE SHOWING

2337
01:49:38,835 --> 01:49:42,572
TRANSFECTION WITH TOMATO MRNA 

2338
01:49:42,572 --> 01:49:46,009
AND LOCALIZATION WITH THE TOP 

2339
01:49:46,009 --> 01:49:47,043
PANELS.

2340
01:49:47,043 --> 01:49:51,681
ONE CAN SEE AND FOX J1 WHICH 

2341
01:49:51,681 --> 01:49:54,784
LABELS THE NUCLEUS OF CILIATED 

2342
01:49:54,784 --> 01:49:56,653
CELLS ARE HIGH TRANSFECTED.

2343
01:49:56,653 --> 01:49:59,088
ON THE BOTTOM PANEL YOU CAN SEE 

2344
01:49:59,088 --> 01:50:03,026
WITH THE YELLOW TWO ARROWS 

2345
01:50:03,026 --> 01:50:05,728
EXPRESSION OF TOMATO WITHIN 

2346
01:50:05,728 --> 01:50:08,064
SECRETORY CELLS EXPRESSING THE 

2347
01:50:08,064 --> 01:50:13,136
TOMATO AND MODIFIED AC.

2348
01:50:13,136 --> 01:50:16,572
THESE ARE OTHER EXAMPLES.

2349
01:50:16,572 --> 01:50:18,374
BASAL CELLS IN VIVO VERY FEW 

2350
01:50:18,374 --> 01:50:19,876
BASAL CELLS WERE TRANS VECTED 

2351
01:50:19,876 --> 01:50:24,781
MARK BY P63 ON THE LEFT.

2352
01:50:24,781 --> 01:50:34,257
ON THE RIGHT ARE THESE PULL MON 

2353
01:50:34,257 --> 01:50:34,524
IONOCYTES.

2354
01:50:34,524 --> 01:50:39,362
AND IF ONE HONES ON SECRETORY 

2355
01:50:39,362 --> 01:50:42,966
CELL MARK BY THE RED AND YELLOW 

2356
01:50:42,966 --> 01:50:44,133
MARK IN A TRANSFECTED SECRETORY 

2357
01:50:44,133 --> 01:50:54,243
CELL.

2358
01:50:58,247 --> 01:51:03,720
AND THIS IS CELL TYPE 

2359
01:51:03,720 --> 01:51:04,020
SPECIFICITY.

2360
01:51:04,020 --> 01:51:08,324
CAN THE mRNA CLEAR THE DEFECT?

2361
01:51:08,324 --> 01:51:10,159
THE FIRST THING WE NEEDED TO DO 

2362
01:51:10,159 --> 01:51:13,296
WAS TO DEVELOP AN ASSAY THAT WE 

2363
01:51:13,296 --> 01:51:15,431
FELT WAS ROBUST AND CAPABLE OF 

2364
01:51:15,431 --> 01:51:19,035
DETECTING CFTR IMPACT ON 

2365
01:51:19,035 --> 01:51:21,671
CLEARANCE.

2366
01:51:21,671 --> 01:51:29,712
WE USED PET CT AND AND WE SEE A 

2367
01:51:29,712 --> 01:51:31,047
DEPOSITION MOVING UP THE TRACHEA

2368
01:51:31,047 --> 01:51:36,819
AND ONE UP AND OFF AND ONE CAN 

2369
01:51:36,819 --> 01:51:38,988
QUANTIFY THIS USING THE RAMENT 

2370
01:51:38,988 --> 01:51:39,622
OF PERCENT THAT'S CHEERED AND 

2371
01:51:39,622 --> 01:51:41,758
WHAT ONE SEES ON THE BLUE LINE 

2372
01:51:41,758 --> 01:51:45,962
ARE BASELINE STUDY AND THEN 

2373
01:51:45,962 --> 01:51:48,998
PROGRESSIVELY WITH TIME OFF THE 

2374
01:51:48,998 --> 01:51:55,038
AIR 770 THE DE DECLINE WITHIN 

2375
01:51:55,038 --> 01:51:57,006
THE MUCOUS CLEARANCE AND THE 

2376
01:51:57,006 --> 01:51:58,808
GREEN LINE BOUNCES BACK UP TO 

2377
01:51:58,808 --> 01:52:00,009
WHAT IT WAS.

2378
01:52:00,009 --> 01:52:03,846
WE FELT CONFIDENT THAT THIS WAS 

2379
01:52:03,846 --> 01:52:06,382
INDEXING CFTR ACTIVITY AND THIS 

2380
01:52:06,382 --> 01:52:08,284
IS QUANTIFIED AS THE CLEARANCE 

2381
01:52:08,284 --> 01:52:08,818
RATE WITHIN THE FIRST SIX 

2382
01:52:08,818 --> 01:52:17,527
MINUTES.

2383
01:52:17,527 --> 01:52:19,362
THE QUESTION WAS DOES THE LIEU 

2384
01:52:19,362 --> 01:52:23,232
FLOOR CFTR CORRECT THE MCC 

2385
01:52:23,232 --> 01:52:30,540
DEFECT SO THE STUDY WAS TO FIRST

2386
01:52:30,540 --> 01:52:32,608
SCAN BASELINE MCC WITH CF 

2387
01:52:32,608 --> 01:52:36,579
FERRETS OFF THE MODULATOR AND 

2388
01:52:36,579 --> 01:52:38,314
THEN WHEN THEY REACHED A 

2389
01:52:38,314 --> 01:52:42,118
BASELINE OF 20 TO 40 TO TREAT 

2390
01:52:42,118 --> 01:52:45,855
WITH THE HUMAN CFTC.

2391
01:52:45,855 --> 01:52:46,656
AND CLEARANCE AFTER THAT.

2392
01:52:46,656 --> 01:52:49,892
WHAT WE'RE LOOKING AT THE HERE 

2393
01:52:49,892 --> 01:52:53,963
ARE BASELINE SCANS IN EIGHT 

2394
01:52:53,963 --> 01:52:57,366
ANIMALS FOLLOWED BY THE HUMAN 

2395
01:52:57,366 --> 01:52:58,134
LMP DELIVERY.

2396
01:52:58,134 --> 01:53:00,369
SEE SIGNIFICANT IMPROVEMENT AND 

2397
01:53:00,369 --> 01:53:03,039
CLEARANCE BOTH ON THE QUANTIFIED

2398
01:53:03,039 --> 01:53:06,509
ON THE LEFT.

2399
01:53:06,509 --> 01:53:10,646
AND ONE COMPARES THAT TO THE 

2400
01:53:10,646 --> 01:53:16,719
LUNAR TOMATO mRmRNA THERE WAS N 

2401
01:53:16,719 --> 01:53:19,889
SIGNIFICANT DELTA AND 

2402
01:53:19,889 --> 01:53:25,795
QUANTITIFQUANTI

2403
01:53:25,795 --> 01:53:27,130
QUANTITIFQUANTI

2404
01:53:27,130 --> 01:53:28,464
QUANTITIFQUANTI

2405
01:53:28,464 --> 01:53:29,098
QUANTITIFYING  -- QUANTIFYING 

2406
01:53:29,098 --> 01:53:32,101
THE DIFFERENCE IN TOMATO AND 

2407
01:53:32,101 --> 01:53:34,670
HERE EVALUATED BY NOVA AND HERE 

2408
01:53:34,670 --> 01:53:39,041
THE PAIRED ANALYSIS FOR THE TWO 

2409
01:53:39,041 --> 01:53:39,375
GROUPS.

2410
01:53:39,375 --> 01:53:43,045
SO THE LAST QUESTION BEFORE I 

2411
01:53:43,045 --> 01:53:47,016
SUM UP IS CAN THE LNP PENETRATE 

2412
01:53:47,016 --> 01:53:48,651
THE MUCOUS BARRIER.

2413
01:53:48,651 --> 01:53:50,953
THIS IS AN EXAMPLE OF A CF 

2414
01:53:50,953 --> 01:53:53,589
FERRET TAKEN OFF THE MODULATOR 

2415
01:53:53,589 --> 01:53:54,357
FOR FIVE MONTHS.

2416
01:53:54,357 --> 01:53:59,162
YOU CAN SEE ON THE RIGHT THIS 

2417
01:53:59,162 --> 01:54:00,596
MUCOUS BEARING DOWN ON THE 

2418
01:54:00,596 --> 01:54:03,032
SURFACE OF THE INTERLOPER 

2419
01:54:03,032 --> 01:54:07,103
CONDUCTING AIRWAY AND THIS IS 

2420
01:54:07,103 --> 01:54:10,239
FOLLOWING LUNAR CREE DELIVERY 

2421
01:54:10,239 --> 01:54:12,108
SWITCHING ON THE CFG REPORTER.

2422
01:54:12,108 --> 01:54:15,044
AND YOU CAN SEE AT LEAST IN THIS

2423
01:54:15,044 --> 01:54:17,580
EXAMPLE RIGHT HERE THERE'S 

2424
01:54:17,580 --> 01:54:20,249
PENETRATION TO THE LIPID TO 

2425
01:54:20,249 --> 01:54:21,384
TRANSFECT THE SURFACE AIRWAY 

2426
01:54:21,384 --> 01:54:27,390
EPITHELIUM.

2427
01:54:27,390 --> 01:54:29,225
TO SUMMARIZE, CF FERRETS AND 

2428
01:54:29,225 --> 01:54:32,395
OTHER FERRET MODELS CAN AID IN 

2429
01:54:32,395 --> 01:54:35,031
THE EVALUATION OF LUNG DISEASE 

2430
01:54:35,031 --> 01:54:36,566
PATHOPHYSIOLOGY AND THERAPIES.

2431
01:54:36,566 --> 01:54:39,402
THERE'S SPECIFIC ASPECTS OF THE 

2432
01:54:39,402 --> 01:54:42,004
FERRET LUNG AND CELL BIOLOGY AND

2433
01:54:42,004 --> 01:54:47,310
ANATOMY THAT MAKE IT A GOOD 

2434
01:54:47,310 --> 01:54:52,648
MODEL FOR LUNG DISEASES AND IT 

2435
01:54:52,648 --> 01:54:54,784
DELIVERS mRNA AND EFFECTIVE 

2436
01:54:54,784 --> 01:54:56,586
CLEARANCE DEFECT WITHIN CF 

2437
01:54:56,586 --> 01:54:58,487
FERRETS AND THE IMPROVED MCC 

2438
01:54:58,487 --> 01:55:03,025
WITH THE HIGHEST TRANSFECTION OF

2439
01:55:03,025 --> 01:55:04,860
CILIATED CELLS LOWERS IONOCYTES 

2440
01:55:04,860 --> 01:55:09,398
AND IT'S IMPORTANT BECAUSE THE 

2441
01:55:09,398 --> 01:55:13,002
PATTERN OF TRANSFECTION IS NOT 

2442
01:55:13,002 --> 01:55:18,074
IDENTICAL TO THE ENDOGENOUS 

2443
01:55:18,074 --> 01:55:20,142
EXPRESSION AND INDICATES OTHER 

2444
01:55:20,142 --> 01:55:22,778
CELL TYPES MAY BE ABLE TO PICK 

2445
01:55:22,778 --> 01:55:29,385
UP THE SLACK AND RECONSTITUTE 

2446
01:55:29,385 --> 01:55:30,920
AIRWAY CLEARANCE.

2447
01:55:30,920 --> 01:55:33,389
AND THEN I'LL SAY LASTLY THE 

2448
01:55:33,389 --> 01:55:39,028
STUDIES HELPED TO MOVE LUNAR 

2449
01:55:39,028 --> 01:55:40,863
THROUGH CLINICAL TRIALS AND 

2450
01:55:40,863 --> 01:55:42,331
WE'RE CURRENTLY UNDERGOING 

2451
01:55:42,331 --> 01:55:46,335
TRIALS ONE THE LNP.

2452
01:55:46,335 --> 01:55:49,071
I'D LIKE TO THANK THOSE IN MY 

2453
01:55:49,071 --> 01:55:51,040
LABORATORY AND THE TREMENDOUS 

2454
01:55:51,040 --> 01:55:53,476
NUMBER OF COLLABORATORS WORKING 

2455
01:55:53,476 --> 01:55:55,444
WITH THE LARGER ANIMAL MODEL IS 

2456
01:55:55,444 --> 01:55:55,978
DEFINITELY A TEAM SCIENCE.

2457
01:55:55,978 --> 01:56:04,287
SO THANK YOU.

2458
01:56:04,287 --> 01:56:05,521
>> THANK YOU.

2459
01:56:05,521 --> 01:56:06,589
I ALSO WANT TO TAKE THE 

2460
01:56:06,589 --> 01:56:08,658
OPPORTUNITY TO THANK OUR 

2461
01:56:08,658 --> 01:56:10,326
SPEAKERS FROM SESSION 1 SHOWING 

2462
01:56:10,326 --> 01:56:14,230
THE FOREGROUND FROM MODEL AND 

2463
01:56:14,230 --> 01:56:15,865
TOOL DEVELOPMENT.

2464
01:56:15,865 --> 01:56:17,767
AS PART OF OUR TRANSITION TO 

2465
01:56:17,767 --> 01:56:20,903
PANEL DISCUSSION HERE I WANT SO 

2466
01:56:20,903 --> 01:56:23,039
SHOW OUR EXPERIENCE IN THE SPACE

2467
01:56:23,039 --> 01:56:25,341
AND WHICH I THINK WE'LL FIND 

2468
01:56:25,341 --> 01:56:27,043
SYNERGIES WITH THE PRESENTATION 

2469
01:56:27,043 --> 01:56:37,420
WE HEARD IN SESSION 1.

2470
01:56:39,088 --> 01:56:45,394
WE'VE ENGINEERED LUNG TISSUE 

2471
01:56:45,394 --> 01:56:52,335
MODELS FOR TESTING.

2472
01:56:52,335 --> 01:56:54,370
A TOPIC WE'VE INVESTIGATED IS IN

2473
01:56:54,370 --> 01:56:55,571
THE INTERACTION.

2474
01:56:55,571 --> 01:56:57,340
THE LUNG IS ONE OF THE LARGEST 

2475
01:56:57,340 --> 01:56:58,274
INTERFACE BETWEEN TOUR BODY AND 

2476
01:56:58,274 --> 01:57:03,946
THE OUTSIDE ENVIRONMENT.

2477
01:57:03,946 --> 01:57:09,352
SO WE HAVE POLLUTANTS AND VIRUS 

2478
01:57:09,352 --> 01:57:12,922
AND IT'S BECOMING A ROBUST FOR 

2479
01:57:12,922 --> 01:57:15,024
STUDYING DISEASE MECHANISM AND 

2480
01:57:15,024 --> 01:57:19,562
FOR TESTING THERAPEUTICS.

2481
01:57:19,562 --> 01:57:21,397
HOWEVER, AND THERE'S BEEN ONE 

2482
01:57:21,397 --> 01:57:26,702
MAJOR LIMITATION IS MOST MODEL 

2483
01:57:26,702 --> 01:57:30,306
HAVE ETHICAL INCONFIRMATION AND 

2484
01:57:30,306 --> 01:57:39,014
WE PUT STEM CELLS INTO A SETTING

2485
01:57:39,014 --> 01:57:41,450
A

2486
01:57:41,450 --> 01:57:45,254
AND THEY'LL CAUSE A SURFACE WITH

2487
01:57:45,254 --> 01:57:46,956
CILIA CLEARANCE TO HAPPEN IN 

2488
01:57:46,956 --> 01:57:47,957
CLOSE INTERFACE WHICH BECOMES 

2489
01:57:47,957 --> 01:57:49,258
DIFFICULT TO ACCESS WHEN IT 

2490
01:57:49,258 --> 01:57:51,794
COMES TO INTRODUCING THE 

2491
01:57:51,794 --> 01:57:53,529
ENVIRONMENTAL THREATS LIKE 

2492
01:57:53,529 --> 01:57:55,297
POLLUTION OR VIRUS OR BACTERIA.

2493
01:57:55,297 --> 01:57:56,532
IN THE PAST FEW YEARS US AND 

2494
01:57:56,532 --> 01:57:59,068
OTHER GROUPS HAVE BEEN COMING UP

2495
01:57:59,068 --> 01:58:04,240
WITH STRATEGY TO REDUCE THE 

2496
01:58:04,240 --> 01:58:06,008
POLARITY AND MOST USE A STRATEGY

2497
01:58:06,008 --> 01:58:07,676
DRAWING SUPPORT FROM THE CULTURE

2498
01:58:07,676 --> 01:58:10,980
AT THE BEGINNING OR END OF THE 

2499
01:58:10,980 --> 01:58:11,914
DIFFERENTIATION WHICH SERVES AS 

2500
01:58:11,914 --> 01:58:15,985
AN EFFECTIVE STRATEGY TO REVERSE

2501
01:58:15,985 --> 01:58:17,653
POLARITY AND SIMILAR TO WHAT WE 

2502
01:58:17,653 --> 01:58:28,063
SEE IN THE NATIVE LUNG.

2503
01:58:31,100 --> 01:58:39,041
AND THE SURFACE I COAT WITH 

2504
01:58:39,041 --> 01:58:44,280
CILIA AND PADDLING IN A 

2505
01:58:44,280 --> 01:58:47,016
SYNCHRONIZED MATTER CAN BRAVE 

2506
01:58:47,016 --> 01:58:49,285
THE MOTILITY OF WHAT WE ENGINEER

2507
01:58:49,285 --> 01:58:51,387
AND WE CAN TRACK THE MOTILITY OF

2508
01:58:51,387 --> 01:58:57,393
THE ORGANOID AND CORRELATE THIS 

2509
01:58:57,393 --> 01:59:01,430
WITH CILIA MOTILITY AND GET 

2510
01:59:01,430 --> 01:59:11,974
CELLS FROM HEALTHY OR PCD CELLS 

2511
01:59:15,211 --> 01:59:16,946
AND CAN SERVE AS A SCREENING 

2512
01:59:16,946 --> 01:59:26,322
PLATFORM.

2513
01:59:26,322 --> 01:59:34,497
WE'RE MAKING MODELS MORE AUTO 

2514
01:59:34,497 --> 01:59:44,340
MATED AND YOU HAVE THIS ORGANOID

2515
01:59:44,340 --> 01:59:49,011
AND DRAW THE SPIRAL ON THE 

2516
01:59:49,011 --> 01:59:51,013
SURFACE UNTIL I BUMPS INTO A 

2517
01:59:51,013 --> 01:59:54,950
WALL OR ORGANOID AND DEVELOPED A

2518
01:59:54,950 --> 01:59:56,452
SWARM ANALYSIS LIKE WATCHING A 

2519
01:59:56,452 --> 01:59:59,054
GROUP OF INSECTS MOVING AROUND 

2520
01:59:59,054 --> 02:00:01,524
AND NOW WATCH ORGANOID MOVING 

2521
02:00:01,524 --> 02:00:04,627
AROUND POWERED BY CILIA AND TELL

2522
02:00:04,627 --> 02:00:06,896
THE DIFFERENCE BETWEEN THEY WILL

2523
02:00:06,896 --> 02:00:09,164
THE INDIVIDUAL VERSUS THE 

2524
02:00:09,164 --> 02:00:10,132
ORGANOID BEING TRIGGERED WITH 

2525
02:00:10,132 --> 02:00:20,376
AIR POLLUTION.

2526
02:00:21,477 --> 02:00:23,746
HE WE CAN COME UP WITH ASSAYS 

2527
02:00:23,746 --> 02:00:31,020
AND ALTER THE PIPELINE COMBINED 

2528
02:00:31,020 --> 02:00:37,893
WITH AUTO MATED IMAGING AND WE 

2529
02:00:37,893 --> 02:00:39,995
HAVE FUNCTION WITH WHOLE TISSUE 

2530
02:00:39,995 --> 02:00:40,296
THROUGHPUT.

2531
02:00:40,296 --> 02:00:44,767
FOR THE ORGANOIDS TO SPIN AND 

2532
02:00:44,767 --> 02:00:47,069
ROTATE IT HAS TO WORK IN A 

2533
02:00:47,069 --> 02:00:48,270
COORDINATING MANNER AND HAVE 

2534
02:00:48,270 --> 02:00:52,174
POLARITY WHICH ALLOWS US TO 

2535
02:00:52,174 --> 02:00:53,142
INTRODUCE POLLUTANT BACTERIA 

2536
02:00:53,142 --> 02:00:54,710
VIRUS FOR THERAPEUTICS WE INTEND

2537
02:00:54,710 --> 02:01:01,850
TO DELIVER INTO THE AIR SPACE.

2538
02:01:01,850 --> 02:01:07,022
WE HAVE TO BEAR IN MIND IT'S 

2539
02:01:07,022 --> 02:01:11,026
STILL RECAPITULATING WHAT IT 

2540
02:01:11,026 --> 02:01:15,030
LOOKS LIKE AND LOOK AT THE 

2541
02:01:15,030 --> 02:01:15,397
POLARITY.

2542
02:01:15,397 --> 02:01:19,134
WE SHOULD CONSIDER INTRODUCING 

2543
02:01:19,134 --> 02:01:22,705
THE BASAL STROMAL ENVIRONMENT 

2544
02:01:22,705 --> 02:01:24,873
AND THE MEMBRANE BETWEEN 

2545
02:01:24,873 --> 02:01:29,945
EPITHELIAL AND STROMAL CELLS.

2546
02:01:29,945 --> 02:01:32,748
THIS IS ECHOING THE PLATFORM WE 

2547
02:01:32,748 --> 02:01:35,084
THINK IT'S ESSENTIAL TO 

2548
02:01:35,084 --> 02:01:37,219
RECAPITULATE THE GEOMETRY OF THE

2549
02:01:37,219 --> 02:01:39,922
LUNG TISSUE AND IT'S CONSTANTLY 

2550
02:01:39,922 --> 02:01:44,259
BRANCHING TUBULES LEADING TO 

2551
02:01:44,259 --> 02:01:44,927
GENERATIONAL BRANCHES.

2552
02:01:44,927 --> 02:01:49,932
PUTTING MORE INSIGHT INTO HOW 

2553
02:01:49,932 --> 02:01:51,333
THE PATHOLOGY SPECIFY AROUND 

2554
02:01:51,333 --> 02:01:55,137
DIFFERENT PARTS OF THE 

2555
02:01:55,137 --> 02:02:03,045
RESPIRATORY TRACT.

2556
02:02:03,045 --> 02:02:07,049
WITH THIS IN MIND AND WHERE WE 

2557
02:02:07,049 --> 02:02:11,020
PUT STEM CELLS INSTEAD OF THE 

2558
02:02:11,020 --> 02:02:13,155
INDIVIDUAL CELLS AND RATHER 

2559
02:02:13,155 --> 02:02:16,659
EMBED THEM IN THE HYDROGEL IN A 

2560
02:02:16,659 --> 02:02:19,395
SPATIALLY CONSTRAINED MANNER.

2561
02:02:19,395 --> 02:02:21,397
WE CAN EXTRUDE THE CELL INTO 

2562
02:02:21,397 --> 02:02:26,468
ALIGN AND OVER TIME THE CELL CAN

2563
02:02:26,468 --> 02:02:27,069
SENSE THE PRESENCE OF 

2564
02:02:27,069 --> 02:02:30,439
SURROUNDING CELLS AND FORM THE 

2565
02:02:30,439 --> 02:02:31,040
TUBULE.

2566
02:02:31,040 --> 02:02:34,343
IF YOU MAKE THIS FROM AIRWAY 

2567
02:02:34,343 --> 02:02:36,278
STEM CELLS WE CAN GET ROBUST 

2568
02:02:36,278 --> 02:02:37,379
CLEARANCE WITH TYPICAL CELLS YOU

2569
02:02:37,379 --> 02:02:43,185
WOULD FIND IN THE AIRWAY.

2570
02:02:43,185 --> 02:02:50,893
AND WE CAN APPLY SIMILAR 

2571
02:02:50,893 --> 02:02:53,162
STRATEGIES AROUND USE THE CELL 

2572
02:02:53,162 --> 02:02:56,699
LINE WHICH YOU CAN SEE AND AGAIN

2573
02:02:56,699 --> 02:02:59,034
OVER ABOUT A MONTH OF CULTURE WE

2574
02:02:59,034 --> 02:03:01,737
CAN GENERATE FROM THE TUBULE OF 

2575
02:03:01,737 --> 02:03:05,607
THE TYPE 2 CELLS WITH THE 

2576
02:03:05,607 --> 02:03:07,543
INTERCONNECTED LUMEN.

2577
02:03:07,543 --> 02:03:09,845
OF CURSE I WANT TO THANK ALL THE

2578
02:03:09,845 --> 02:03:11,547
FOLKS IN THE LAB WHO MADE THE 

2579
02:03:11,547 --> 02:03:14,750
WORK POSSIBLE AND ALSO OUR 

2580
02:03:14,750 --> 02:03:15,317
AMAZING COLLABORATORS AND 

2581
02:03:15,317 --> 02:03:23,025
FUNDING AGENCIES.

2582
02:03:23,025 --> 02:03:25,260
WITH THAT I'D LIKE TO TRANSITION

2583
02:03:25,260 --> 02:03:26,729
TO OUR PANEL DISCUSSION.

2584
02:03:26,729 --> 02:03:29,231
WE HEARD OF DIFFERENT MODELS FOR

2585
02:03:29,231 --> 02:03:34,269
MIMICKING THE LUNG TISSUE IN 

2586
02:03:34,269 --> 02:03:44,813
HEALTH AND DISEASE AND CHIP AND 

2587
02:03:46,949 --> 02:03:48,250
ORGANOID MODELS AND THEY'VE BEEN

2588
02:03:48,250 --> 02:03:51,386
USED TO TACKLE DIFFERENT LUNG 

2589
02:03:51,386 --> 02:03:56,792
DISEASES FROM CYSTIC FIBROSIS 

2590
02:03:56,792 --> 02:04:03,065
AND LUNG DISEASE TO PULMONARY 

2591
02:04:03,065 --> 02:04:07,770
FIBROSIS AND GENE THERAPY.

2592
02:04:07,770 --> 02:04:09,238
NOW I'D LIKE TO TRANSITION TOE A

2593
02:04:09,238 --> 02:04:11,607
PANEL DISCUSSION AND TURN TO MY 

2594
02:04:11,607 --> 02:04:12,474
MODERATOR INTEREST SHERIDAN TO 

2595
02:04:12,474 --> 02:04:15,978
BRING THE FIRST QUESTION TO OUR 

2596
02:04:15,978 --> 02:04:16,345
PANEL.

2597
02:04:16,345 --> 02:04:18,080
WE CAN STOP SCREEN SHARING NOW.

2598
02:04:18,080 --> 02:04:18,380
THANK YOU.

2599
02:04:18,380 --> 02:04:19,381
>> THANK YOU, CHARLIE AND
THANK 

2600
02:04:19,381 --> 02:04:24,987
YOU TO ALL OF OUR SPEAKERS.

2601
02:04:24,987 --> 02:04:26,088
DR. MOFFITTS IS ALSO JOINING OUR

2602
02:04:26,088 --> 02:04:27,923
PANEL SO IF YOU HAVE ANY 

2603
02:04:27,923 --> 02:04:30,559
QUESTIONS FOR HIM, PLEASE FEEL 

2604
02:04:30,559 --> 02:04:31,860
FREE TO ASK THAT AS WELL.

2605
02:04:31,860 --> 02:04:34,563
AS A REMINDER USE THE Q&A
TOOL 

2606
02:04:34,563 --> 02:04:36,398
AT THE BOTTOM OF THE ZOOM SCREEN

2607
02:04:36,398 --> 02:04:39,468
TO ENTER ANY QUESTIONS YOU HAVE.

2608
02:04:39,468 --> 02:04:42,004
SO IN THE MEANTIME WHILE WE 

2609
02:04:42,004 --> 02:04:44,640
AWAIT SOME QUESTIONS I WILL GO 

2610
02:04:44,640 --> 02:04:45,674
AHEAD AND ASK THE FIRST 

2611
02:04:45,674 --> 02:04:54,082
QUESTION.

2612
02:04:54,082 --> 02:04:57,419
I LIKE AT MODELS AND THERE'S 

2613
02:04:57,419 --> 02:04:59,054
STRENGTHS AND WEAKNESSES WITH 

2614
02:04:59,054 --> 02:05:01,089
ALL MODELS.

2615
02:05:01,089 --> 02:05:03,859
IT'S HARD TO RECAPITULATE 

2616
02:05:03,859 --> 02:05:06,328
EXACTLY WHAT HAPPENS IN A NATIVE

2617
02:05:06,328 --> 02:05:08,564
TISSUE IN A GIVEN MODEL 

2618
02:05:08,564 --> 02:05:10,899
ESPECIALLY NATIVE HUMAN TISSUE.

2619
02:05:10,899 --> 02:05:13,268
SO WONDERING IF THE PANEL COULD 

2620
02:05:13,268 --> 02:05:16,338
COMMENT ON SOME OF THE STRENGTHS

2621
02:05:16,338 --> 02:05:17,706
AND WEAKNESSES OF THE MODELS 

2622
02:05:17,706 --> 02:05:23,045
THAT YOU HAVE EXPERIENCES 

2623
02:05:23,045 --> 02:05:30,586
WORKING WITH HOW THEY ULTIMATELY

2624
02:05:30,586 --> 02:05:31,486
MAY COMPLEMENT EACH OTHER.

2625
02:05:31,486 --> 02:05:33,589
I DON'T KNOW WHO WOULD LIKE TO 

2626
02:05:33,589 --> 02:05:35,090
GO FIRST.

2627
02:05:35,090 --> 02:05:45,500
>> I'LL START OFF FIRST.

2628
02:05:46,368 --> 02:05:48,203
WHAT'S DIFFICULT TO MODEL IS THE

2629
02:05:48,203 --> 02:05:51,773
VIRAL BASED THERAPY ANYTHING CAN

2630
02:05:51,773 --> 02:05:55,043
HAPPEN WITH THE CO-RECEPTORS.

2631
02:05:55,043 --> 02:06:00,048
THE BEST WAY TO RERISK -- DERISK

2632
02:06:00,048 --> 02:06:02,150
THAT IS LOOK AT THE CELL TYPES 

2633
02:06:02,150 --> 02:06:07,022
THAT ARE TRANSDUCED AND MAKE 

2634
02:06:07,022 --> 02:06:10,726
SURE THEY'RE SIMILAR AND THE 

2635
02:06:10,726 --> 02:06:12,995
BOCA VIRUS IS ONE THE FERRET 

2636
02:06:12,995 --> 02:06:14,429
DOESN'T MODEL THE TROPISM.

2637
02:06:14,429 --> 02:06:15,430
IT'S ONE OF THE REASON WE 

2638
02:06:15,430 --> 02:06:17,266
HAVEN'T DONE THE NCC EXPERIMENT 

2639
02:06:17,266 --> 02:06:24,740
IN THE FERRET BECAUSE THERE'S NO

2640
02:06:24,740 --> 02:06:35,017
TRANSDUCTION WITH THAT VIRUS.

2641
02:06:35,017 --> 02:06:39,755
BUT IT'S HYDROTROPIC AND YOU 

2642
02:06:39,755 --> 02:06:41,423
HAVE TO KNOW THE QUESTION YOU'RE

2643
02:06:41,423 --> 02:06:44,526
GOING TO ASK.

2644
02:06:44,526 --> 02:06:46,995
IF I CAN ASK ABOUT STEM CELL 

2645
02:06:46,995 --> 02:06:51,033
BIOLOGY IN THE LUNG OF MICE AND 

2646
02:06:51,033 --> 02:06:52,668
I'LL TWO TO THE MOUSE FIRST BUT 

2647
02:06:52,668 --> 02:06:55,237
EVENTUALLY THE QUESTIONS FOR MY 

2648
02:06:55,237 --> 02:06:58,340
RESEARCH TEND TO MOVE THROUGH AN

2649
02:06:58,340 --> 02:07:00,409
AREA THAT IS NOT ADEQUATELY 

2650
02:07:00,409 --> 02:07:03,211
MODELLED IN MICE AND WHY WE USE 

2651
02:07:03,211 --> 02:07:03,445
FERRETS.

2652
02:07:03,445 --> 02:07:05,814
>> I COMPLETELY AGREE,
JOHN.

2653
02:07:05,814 --> 02:07:08,350
I THINK SOME OF THE REALLY 

2654
02:07:08,350 --> 02:07:09,785
INTERESTING PARTS ABOUT THIS AND

2655
02:07:09,785 --> 02:07:13,088
I THINK FOLKS HAVE HEARD AROUND 

2656
02:07:13,088 --> 02:07:13,989
DIFFERENT THERAPEUTIC APPROACHES

2657
02:07:13,989 --> 02:07:16,425
THAT HAVE BEEN VALIDATED IN THE 

2658
02:07:16,425 --> 02:07:16,658
MODELS.

2659
02:07:16,658 --> 02:07:20,295
THOSE ARE GREAT FAST FOLLOWERS 

2660
02:07:20,295 --> 02:07:22,364
BUT WHAT HAPPENS IF YOU'RE 

2661
02:07:22,364 --> 02:07:23,465
LEVERAGING NEW MODALITIES?

2662
02:07:23,465 --> 02:07:25,033
YOU DON'T KNOW WHAT TO TRUST 

2663
02:07:25,033 --> 02:07:25,434
YET.

2664
02:07:25,434 --> 02:07:28,236
THIS IS WHERE THE STRENGTHS OF 

2665
02:07:28,236 --> 02:07:30,305
THE MULTIPLE MODELS COME INTO 

2666
02:07:30,305 --> 02:07:32,040
PLAY BECAUSE YOU START 

2667
02:07:32,040 --> 02:07:33,475
EVALUATING THE THERAPEUTIC 

2668
02:07:33,475 --> 02:07:35,344
HYPOTHESIS FROM RAIN ORTHOGONAL 

2669
02:07:35,344 --> 02:07:36,878
STANDPOINT AND IT'S ALMOST LIKE 

2670
02:07:36,878 --> 02:07:40,082
A WEIGHT OF EVIDENCE, OKAY, THE 

2671
02:07:40,082 --> 02:07:41,016
MODELS ARE LINING UP FOR WHAT'S 

2672
02:07:41,016 --> 02:07:41,616
EXPECTED.

2673
02:07:41,616 --> 02:07:43,051
I THINK THIS IS A REASONABLE 

2674
02:07:43,051 --> 02:07:44,853
SURROGATE FOR WHAT I'M TRYING TO

2675
02:07:44,853 --> 02:07:46,321
ACCOMPLISH.

2676
02:07:46,321 --> 02:07:48,757
THAT'S EASY IF YOU'RE FOLLOWING 

2677
02:07:48,757 --> 02:07:54,997
DOWN A PATHWAY ALREADY 

2678
02:07:54,997 --> 02:07:56,498
ENBLAZENED BUT YOU HAVE TO BAKE 

2679
02:07:56,498 --> 02:08:01,503
IN SAFEGUARDS IF YOU HAVE LESS 

2680
02:08:01,503 --> 02:08:03,038
CERTAINTY AROUND CLINICAL DOSE 

2681
02:08:03,038 --> 02:08:03,338
PROJECTION.

2682
02:08:03,338 --> 02:08:05,540
YOU MAY BE FACED WITH A 

2683
02:08:05,540 --> 02:08:07,009
SITUATION EVEN IF YOU DON'T HAVE

2684
02:08:07,009 --> 02:08:09,444
A SOLID IN VITRO MODEL YOU'VE 

2685
02:08:09,444 --> 02:08:12,314
HEARD ABOUT TODAY, YOU MAY BE 

2686
02:08:12,314 --> 02:08:13,882
FACED WITH A SITUATION WHERE YOU

2687
02:08:13,882 --> 02:08:15,417
HAVE TO GO LOW AND SLOW IN THE 

2688
02:08:15,417 --> 02:08:19,287
CLINIC THAT CAN PROTRACT 

2689
02:08:19,287 --> 02:08:20,722
CLINICAL DEVELOPMENT.

2690
02:08:20,722 --> 02:08:22,357
THE MORE YOU CAN LEVERAGE THE 

2691
02:08:22,357 --> 02:08:23,625
UNIQUE TOOLS TO FIGURE THINGS 

2692
02:08:23,625 --> 02:08:27,429
OUT THE BETTER OFF YOU'LL BE.

2693
02:08:27,429 --> 02:08:30,232
>> TO RESPOND TO THAT, I
ALSO 

2694
02:08:30,232 --> 02:08:33,468
WANT TO MAKE THE POINT THAT YOU 

2695
02:08:33,468 --> 02:08:35,504
CAN LEARN A LOT FROM THE MODEL 

2696
02:08:35,504 --> 02:08:41,943
THAT DOESN'T REPRODUCE THE HUMAN

2697
02:08:41,943 --> 02:08:43,945
DISEASE IF YOU ASK THE RIGHT 

2698
02:08:43,945 --> 02:08:44,446
QUESTIONS.

2699
02:08:44,446 --> 02:08:47,883
THE REASON I POINTED OUT THE 

2700
02:08:47,883 --> 02:08:50,352
AIRWAY SMOOTH MUSCLE AND HAVING 

2701
02:08:50,352 --> 02:08:51,319
THOSE GENERATE FIBROBLASTS 

2702
02:08:51,319 --> 02:08:53,789
DOESN'T HAPPEN IN THE MOUSE.

2703
02:08:53,789 --> 02:08:55,057
AND MULTIPLE PEOPLE HAVE 

2704
02:08:55,057 --> 02:08:56,691
REPORTED THAT.

2705
02:08:56,691 --> 02:08:57,492
I DON'T QUITE UNDERSTAND THE 

2706
02:08:57,492 --> 02:09:03,031
BIOLOGY AND I WANT TO BECAUSE IF

2707
02:09:03,031 --> 02:09:06,201
YOU UNDERSTOOD WHY GLIOMYACIN 

2708
02:09:06,201 --> 02:09:08,303
INJURY IN A MOUSE IS MORE 

2709
02:09:08,303 --> 02:09:10,906
REFERSABLE THAN A FERRET AND 

2710
02:09:10,906 --> 02:09:14,142
PULMONARY FIBROSIS IN HUMAN 

2711
02:09:14,142 --> 02:09:15,043
THERAPEUTICALLY YOU CAN 

2712
02:09:15,043 --> 02:09:21,450
POTENTIALLY UTILIZE THAT.

2713
02:09:21,450 --> 02:09:23,919
>> MAYBE I CAN GIVE MY TWO
CENT 

2714
02:09:23,919 --> 02:09:25,587
ON IS THE COMPLEX IN VITRO 

2715
02:09:25,587 --> 02:09:25,954
MODELS.

2716
02:09:25,954 --> 02:09:31,026
I THINK IT'S A NEW AND EXCITING 

2717
02:09:31,026 --> 02:09:33,462
SPACE AND PEOPLE ARE OFTEN 

2718
02:09:33,462 --> 02:09:36,565
EXCITED TO START USING THE 

2719
02:09:36,565 --> 02:09:38,333
MODELS BUT I ALWAYS CAUTION 

2720
02:09:38,333 --> 02:09:40,302
FOLKS AND SAY DON'T BRING THIS 

2721
02:09:40,302 --> 02:09:41,570
TECHNOLOGY INTO YOUR LAB UNLESS 

2722
02:09:41,570 --> 02:09:43,038
HAVE YOU A GAP.

2723
02:09:43,038 --> 02:09:53,415
UNLESS THERE'S A NEED.

2724
02:09:55,083 --> 02:09:56,618
IS THIS A NEED FOR ADDITIONAL 

2725
02:09:56,618 --> 02:09:59,421
CELL TYPES AND IS THE TARGET IN 

2726
02:09:59,421 --> 02:10:03,458
YOUR FIBROBLAST AND DOES IT 

2727
02:10:03,458 --> 02:10:07,062
CROSS TALK AND DO YOU NEED MEDIA

2728
02:10:07,062 --> 02:10:09,131
PERFUSION THROUGH OR SIT ENOUGH 

2729
02:10:09,131 --> 02:10:13,568
TO USE A 2-D CULTURE OR STEROID?

2730
02:10:13,568 --> 02:10:14,769
AND ON THE FLIP SIDE MAYBE 

2731
02:10:14,769 --> 02:10:16,404
YOU'RE ANIMAL MODELS DON'T 

2732
02:10:16,404 --> 02:10:19,274
EXPRESS THE TARGET AND THIS IS 

2733
02:10:19,274 --> 02:10:22,410
OFTEN THE CASE FOR SOME OF THE 

2734
02:10:22,410 --> 02:10:24,546
MORE NOVEL MODALITIES YOU MAY 

2735
02:10:24,546 --> 02:10:27,516
NEED A MORE HUMAN-BASED SYSTEM 

2736
02:10:27,516 --> 02:10:31,052
AND I THINK THAT'S WHERE I SEE 

2737
02:10:31,052 --> 02:10:32,521
THE COMPLEX OF THE IN VITRO 

2738
02:10:32,521 --> 02:10:34,789
MODELS AT THE FOREFRONT.

2739
02:10:34,789 --> 02:10:37,192
IT ALSO GOES BACK TO WHAT IS 

2740
02:10:37,192 --> 02:10:38,927
YOUR RESEARCH QUESTION AND 

2741
02:10:38,927 --> 02:10:43,031
THAT'S GOING TO HELP DEFINE WHAT

2742
02:10:43,031 --> 02:10:46,434
MODEL YOU CHOOSE.

2743
02:10:46,434 --> 02:10:51,039
>> WE COME FROM A TISSUE 

2744
02:10:51,039 --> 02:10:51,907
ENGINEER PERSPECTIVE AND THEN 

2745
02:10:51,907 --> 02:10:54,509
ONCE YOU HAVE THE MODEL IN 

2746
02:10:54,509 --> 02:10:55,744
PLACE, WHAT QUESTION ARE YOU 

2747
02:10:55,744 --> 02:10:58,780
GOING TO ASK USING THE MODELS 

2748
02:10:58,780 --> 02:10:59,948
AND WITH COLLABORATORS WE 

2749
02:10:59,948 --> 02:11:01,283
UNDERSTAND THE LUNG PATHOLOGY 

2750
02:11:01,283 --> 02:11:07,055
AND TRY TO FIND BEST MODELS TO 

2751
02:11:07,055 --> 02:11:12,594
ANSWER THE BEST QUESTION.

2752
02:11:12,594 --> 02:11:14,763
ALL RIGHT, WE'LL ENCOURAGE THE 

2753
02:11:14,763 --> 02:11:16,031
ATTENDEES TO POSE YOUR QUESTIONS

2754
02:11:16,031 --> 02:11:17,265
IN THE Q&A SESSION AND LOOKS

2755
02:11:17,265 --> 02:11:19,034
LIKE WE HAVE A COUPLE IN THE
Q&A

2756
02:11:19,034 --> 02:11:19,601
ALREADY.

2757
02:11:19,601 --> 02:11:21,403
THERE'S A QUESTION BEING ASKED 

2758
02:11:21,403 --> 02:11:24,673
TO ONE SPECIFIC SPEAKER AND 

2759
02:11:24,673 --> 02:11:27,042
WE'LL USUALLY ASK THE SPEAKER TO

2760
02:11:27,042 --> 02:11:32,113
ADDRESS THE QUESTION DIRECTLY 

2761
02:11:32,113 --> 02:11:33,882
AND HAVE IT MORE GENERAL TO THE 

2762
02:11:33,882 --> 02:11:34,249
PANEL.

2763
02:11:34,249 --> 02:11:36,151
I'LL BRING UP OUR NEXT QUESTION 

2764
02:11:36,151 --> 02:11:38,987
IS IT IMPORTANT TO TAKE A 

2765
02:11:38,987 --> 02:11:40,355
REDUCTIONIST APPROACH TO EARLY 

2766
02:11:40,355 --> 02:11:44,726
DRUG DISCOVERY SUCH AS FOCUS ON 

2767
02:11:44,726 --> 02:11:46,161
MODELS FOR HIGH THROUGHPUT 

2768
02:11:46,161 --> 02:11:48,563
SCREENING GIVEN THE COMPLEXITY 

2769
02:11:48,563 --> 02:11:49,164
OF THE LUNGS AND CELL TYPE OF 

2770
02:11:49,164 --> 02:11:59,040
THE EFFECT.

2771
02:11:59,040 --> 02:12:02,477
>> YEAH, IT'S REALLY
HELPFUL TO 

2772
02:12:02,477 --> 02:12:03,812
DO THAT AND BREAK IT DOWN.

2773
02:12:03,812 --> 02:12:05,714
YOU HAVE TO THINK ABOUT THE 

2774
02:12:05,714 --> 02:12:06,948
DISEASE YOU'RE TRYING TO TREAT 

2775
02:12:06,948 --> 02:12:09,217
AND HOW WELL YOU CHARACTERIZE 

2776
02:12:09,217 --> 02:12:11,019
THE BIOLOGY THAT YOU'RE TRYING 

2777
02:12:11,019 --> 02:12:13,288
TO INTERVENE WITH THE MODALITY.

2778
02:12:13,288 --> 02:12:15,957
I THINK THERE'S WAYS YOU CAN 

2779
02:12:15,957 --> 02:12:18,326
LEVERAGE A LOT OF THESE HIGHER 

2780
02:12:18,326 --> 02:12:20,362
THROUGHPUT TECHNOLOGIES TO TRY 

2781
02:12:20,362 --> 02:12:22,897
AND NARROW DOWN IN ON THE 

2782
02:12:22,897 --> 02:12:25,734
INTENDED BIOLOGY AND THE 

2783
02:12:25,734 --> 02:12:28,203
EFFICACY PROFILE AND YOU'LL BE 

2784
02:12:28,203 --> 02:12:30,205
FLYING BLIND THOUGH THE LARGER 

2785
02:12:30,205 --> 02:12:33,375
IN VIVO SAFETY CONSIDERATIONS 

2786
02:12:33,375 --> 02:12:34,709
PARTICULARLY AROUND FOR SMALL 

2787
02:12:34,709 --> 02:12:36,311
MOLECULE THAT MAY INCUR LATER ON

2788
02:12:36,311 --> 02:12:38,847
BUT THAT'S OKAY BECAUSE AS YOU 

2789
02:12:38,847 --> 02:12:40,749
START TO UNDERSTAND WHAT ARE THE

2790
02:12:40,749 --> 02:12:42,250
PRODUCT ATTRIBUTES THAT ARE VERY

2791
02:12:42,250 --> 02:12:43,518
BENEFICIAL FOR EFFICACY I THINK 

2792
02:12:43,518 --> 02:12:47,022
THAT'S HOW YOU CAN START TO 

2793
02:12:47,022 --> 02:12:49,457
STEER IN THAT DIRECTION.

2794
02:12:49,457 --> 02:12:52,060
REGARDLESS OF LUNG OR SLEEP OR 

2795
02:12:52,060 --> 02:12:54,529
ANY OTHER INDICATION IS YOUR 

2796
02:12:54,529 --> 02:12:56,064
AREA OF INTEREST.

2797
02:12:56,064 --> 02:12:57,666
KRISTIN, DO YOU AGREE WITH THAT?

2798
02:12:57,666 --> 02:12:59,501
>> I DO.

2799
02:12:59,501 --> 02:13:04,105
I THINK ESPECIALLY BEING FROM 

2800
02:13:04,105 --> 02:13:07,375
THE MORE HIGH THROUGHPUT SPACE 

2801
02:13:07,375 --> 02:13:09,511
SO WE ARE OFFERING THESE MORE 

2802
02:13:09,511 --> 02:13:10,912
COMPLEX MODELS IN THE HIGH 

2803
02:13:10,912 --> 02:13:13,682
THROUGHPUT CAPACITY SO THAT 

2804
02:13:13,682 --> 02:13:14,916
FOLKS CAN AS QUARTERLY AS 

2805
02:13:14,916 --> 02:13:17,085
POSSIBLE GET THE COMPLEXITY BUT 

2806
02:13:17,085 --> 02:13:19,654
THEN ALSO EARLY AND QUICKLY BE 

2807
02:13:19,654 --> 02:13:20,488
ABLE TO KIND OF UNDERSTAND 

2808
02:13:20,488 --> 02:13:22,891
WHETHER WE SHOULD BE PROGRESSING

2809
02:13:22,891 --> 02:13:27,028
THE MOLECULES THROUGH THE 

2810
02:13:27,028 --> 02:13:30,432
PIPELINE.

2811
02:13:30,432 --> 02:13:32,801
>> I'LL MAKE ONE COMMENT
THAT IT

2812
02:13:32,801 --> 02:13:36,137
DEPENDS WHAT YOUR THERAPEUTIC 

2813
02:13:36,137 --> 02:13:36,438
IS.

2814
02:13:36,438 --> 02:13:38,573
OF COURSE I GRAVITATE TO THINK 

2815
02:13:38,573 --> 02:13:41,242
ABOUT CYSTIC FIBROSIS BECAUSE IT

2816
02:13:41,242 --> 02:13:44,913
SPANS THE CHEMICAL SPACE AND 

2817
02:13:44,913 --> 02:13:48,016
BIOLOGICAL TO THE mRNA AND LNP 

2818
02:13:48,016 --> 02:13:50,185
WHICH IS STILL DIFFERENT.

2819
02:13:50,185 --> 02:13:55,023
I THINK SO IF YOU TALK THE SMALL

2820
02:13:55,023 --> 02:13:56,825
MOLECULES FOR THE SCREEN AND 

2821
02:13:56,825 --> 02:13:59,060
SCREEN IN THYROID CELLS IN THE 

2822
02:13:59,060 --> 02:14:01,730
ORIGINAL STUDIES THAT WERE DONE 

2823
02:14:01,730 --> 02:14:04,099
IS FINE BECAUSE THE TARGET OF 

2824
02:14:04,099 --> 02:14:06,768
THAT WAS THE ACTUAL GATING OF 

2825
02:14:06,768 --> 02:14:08,203
THE CFDR CHANNEL AND THERE'S 

2826
02:14:08,203 --> 02:14:09,938
WAYS TO EVALUATE THAT.

2827
02:14:09,938 --> 02:14:13,108
WHEN YOU THINK ABOUT A BIOLOGIC 

2828
02:14:13,108 --> 02:14:15,744
AND ALL THE VARIABLES HOW YOU'LL

2829
02:14:15,744 --> 02:14:18,813
CORRECT AIRWAY EPITHELIUM, THEN 

2830
02:14:18,813 --> 02:14:22,317
I GRAVITATE TO SOME AIR LIQUID 

2831
02:14:22,317 --> 02:14:25,420
INTERFACE TYPE OF SCREEN EVEN IF

2832
02:14:25,420 --> 02:14:27,555
IT'S SEMI HIGH THROUGHPUT WHICH 

2833
02:14:27,555 --> 02:14:35,029
VERTEX IS KNOWN FOR DOING.

2834
02:14:35,029 --> 02:14:38,099
>> GREAT, THANK YOU.

2835
02:14:38,099 --> 02:14:39,501
I'LL GO AHEAD AND ASK THE NEXT 

2836
02:14:39,501 --> 02:14:41,202
QUESTION.

2837
02:14:41,202 --> 02:14:42,837
THIS WAS DIRECTED TO DR. MOFFITT

2838
02:14:42,837 --> 02:14:44,506
DIRECTLY BUT I THINK IT MAY BE 

2839
02:14:44,506 --> 02:14:45,874
RELEVANT FOR THE OTHERS TO WEIGH

2840
02:14:45,874 --> 02:14:48,810
IN AS WELL IF THEY HAVE 

2841
02:14:48,810 --> 02:14:49,110
THOUGHTS.

2842
02:14:49,110 --> 02:14:51,179
SO THE QUESTION WAS ASKING IF 

2843
02:14:51,179 --> 02:14:53,648
YOU COULD COMMENT ON ANY OF THE 

2844
02:14:53,648 --> 02:14:57,452
PROCESSES, CHALLENGES, HURDLES 

2845
02:14:57,452 --> 02:15:03,024
IN DEVELOPING AND ADVANCING 

2846
02:15:03,024 --> 02:15:07,128
ALLOSTERIC MODULATORS.

2847
02:15:07,128 --> 02:15:11,766
>> OH, THAN AN EASY ONE.

2848
02:15:11,766 --> 02:15:13,501
AT HIGH A LEVEL IF YOUR FOCUSSED

2849
02:15:13,501 --> 02:15:17,272
ON THE SAFETY YOU'LL PERCEIVE 

2850
02:15:17,272 --> 02:15:18,907
WHETHER IT'S AN ALLOSTERIC 

2851
02:15:18,907 --> 02:15:20,975
MODULATOR OR NOT AND TRYING TO 

2852
02:15:20,975 --> 02:15:21,976
CHARACTERIZE THE SAFETY PROFILE 

2853
02:15:21,976 --> 02:15:27,015
AND TARGET ORIGINS OF SAFETY AND

2854
02:15:27,015 --> 02:15:31,853
SAFETY MARGIN AND EFFICACIOUS 

2855
02:15:31,853 --> 02:15:32,987
DOSE AND I THINK MORE WHERE 

2856
02:15:32,987 --> 02:15:35,023
WE'RE COMING FROM IS MORE ON THE

2857
02:15:35,023 --> 02:15:37,058
EFFICACY SIDE OF THINGS.

2858
02:15:37,058 --> 02:15:38,893
THAT'S WHERE YOU ARE HAVING A 

2859
02:15:38,893 --> 02:15:40,929
GOOD UNDERSTANDING OF THE 

2860
02:15:40,929 --> 02:15:43,031
BIOLOGY AND CHARACTERIZING THE 

2861
02:15:43,031 --> 02:15:53,541
OF THE CHANGE OF THE RECEPTOR 

2862
02:15:55,176 --> 02:16:02,484
WHY IMPACT BINDING AND HOW IT 

2863
02:16:02,484 --> 02:16:05,320
BEHAVES AND IN VIVO ANY SPECIES 

2864
02:16:05,320 --> 02:16:08,790
SELECTION BECAUSE THAT'S ONE OF 

2865
02:16:08,790 --> 02:16:10,725
THE IMPORTANT EARLY DRIVERS ONCE

2866
02:16:10,725 --> 02:16:13,628
YOU GET BEYOND THE IN VIVO 

2867
02:16:13,628 --> 02:16:14,028
CHARACTERIZATION.

2868
02:16:14,028 --> 02:16:15,029
I'LL TURN IT TO OTHERS FOR THEIR

2869
02:16:15,029 --> 02:16:24,005
THOUGHTS.

2870
02:16:24,005 --> 02:16:27,509
>> THE BIOLOGICAL
VARIABILITY AS

2871
02:16:27,509 --> 02:16:31,012
WITH PRO VIRUSES IS THEY ARE 

2872
02:16:31,012 --> 02:16:41,556
BIND TO GLYCOPROTEINS IN A WAY 

2873
02:16:42,690 --> 02:16:43,424
YOU WOULDN'T HAVE PREDICTED SO I

2874
02:16:43,424 --> 02:16:43,992
AGREE.

2875
02:16:43,992 --> 02:16:46,961
>> THANK YOU.

2876
02:16:46,961 --> 02:16:50,498
SO, WE'LL MOVE ON TO THE NEXT 

2877
02:16:50,498 --> 02:16:54,536
QUESTION FROM OUR Q&A, MANY
RARE

2878
02:16:54,536 --> 02:16:58,606
GENETIC LUNG DISEASE SUCH AS 

2879
02:16:58,606 --> 02:17:05,713
CFTR MUTATION HAVE VARIANTS AND 

2880
02:17:05,713 --> 02:17:07,549
WHAT ARE THE PANEL'S THOUGHTS 

2881
02:17:07,549 --> 02:17:11,719
HOW TO PRIORITIZE THE VARIANTS?

2882
02:17:11,719 --> 02:17:15,023
COULD THERAPY OVERLAP BETWEEN 

2883
02:17:15,023 --> 02:17:22,797
GENES?

2884
02:17:22,797 --> 02:17:23,765
>> YOU'LL GO.

2885
02:17:23,765 --> 02:17:34,242
A COMMENT FROM ASSIST  -- A 

2886
02:17:37,612 --> 02:17:40,214
CYSTIC FIBROSIS PATHWAY THERE'S 

2887
02:17:40,214 --> 02:17:41,149
NONSENSE MEDIATED DECAY.

2888
02:17:41,149 --> 02:17:45,520
IF YOU'RE GOING TO TARGET THAT 

2889
02:17:45,520 --> 02:17:46,387
PATHWAY SPECIFICALLY IT'S 

2890
02:17:46,387 --> 02:17:49,424
DIFFERENT THAN A CHANNEL 

2891
02:17:49,424 --> 02:17:50,224
MODULATOR ENHANCING TRAFFICKING 

2892
02:17:50,224 --> 02:17:56,965
OR ENHANCING ENGAGEMENT IN CFTR.

2893
02:17:56,965 --> 02:18:04,672
THE ADVANTAGE OF A GENE ADDITION

2894
02:18:04,672 --> 02:18:08,009
IS IT WILL CORRECT ALL MUTATIONS

2895
02:18:08,009 --> 02:18:11,212
BUT THE DISADVANTAGE REGARDLESS 

2896
02:18:11,212 --> 02:18:14,415
OF THE DISEASE YOU HAVE TO KNOW 

2897
02:18:14,415 --> 02:18:15,750
THE PATHOPHYSIOLOGY AND TARGET 

2898
02:18:15,750 --> 02:18:18,252
OF YOUR PROTEIN TO ANSWER THE 

2899
02:18:18,252 --> 02:18:19,687
QUESTION AND SEE IF IT'S ONLY 

2900
02:18:19,687 --> 02:18:21,189
NOW BEGINNING TO GET TO THE 

2901
02:18:21,189 --> 02:18:23,024
POINT WHERE ANSWERS ARE BECOMING

2902
02:18:23,024 --> 02:18:26,761
CLEAR ON THAT.

2903
02:18:26,761 --> 02:18:31,032
THE CFTR KNOCKING IN A GENE 

2904
02:18:31,032 --> 02:18:36,838
EDITING APPROACH KNOCK IN A 

2905
02:18:36,838 --> 02:18:39,340
LARGE AXONS FOR INTERON AND 

2906
02:18:39,340 --> 02:18:41,309
EVERYTHING DOWN STREAM IS A 

2907
02:18:41,309 --> 02:18:43,411
UNIVERSAL APPROACH TO GENE 

2908
02:18:43,411 --> 02:18:45,513
THERAPY TO TARGET MULTIPLE 

2909
02:18:45,513 --> 02:18:50,251
MUTATIONS.

2910
02:18:50,251 --> 02:18:51,419
>> MAYBE BUILDING ON THAT
FOR 

2911
02:18:51,419 --> 02:18:54,255
ONE SECOND THINKING ABOUT THE 

2912
02:18:54,255 --> 02:18:57,525
WAY YOU'RE TRYING TO THERAP

2913
02:18:57,525 --> 02:19:01,396
THERAPEUTICALLY APPROACH THE 

2914
02:19:01,396 --> 02:19:06,701
DISEASE AND A SHORT SWORD AND 

2915
02:19:06,701 --> 02:19:07,301
SHIELD MENTALITY BUT YOU MAY 

2916
02:19:07,301 --> 02:19:09,370
HAVE A TOXIC GAIN OF FUNCTION 

2917
02:19:09,370 --> 02:19:10,705
FROM THE VARIANT AND YOU NEED TO

2918
02:19:10,705 --> 02:19:14,008
FIND A WAY TO KNOCK THAT DOWN IN

2919
02:19:14,008 --> 02:19:18,146
COMBINATION WITH HOPEFULLY 

2920
02:19:18,146 --> 02:19:20,048
EXPRESSING OR GETTING THE 

2921
02:19:20,048 --> 02:19:20,848
ENDOGENOUS PROTEIN THERE WHETHER

2922
02:19:20,848 --> 02:19:24,152
IT'S A TRANSPORTER OR OTHERWISE 

2923
02:19:24,152 --> 02:19:25,920
TO FUNCTION CORRECTLY.

2924
02:19:25,920 --> 02:19:26,487
THERE'S DIFFERENT WAYS AND 

2925
02:19:26,487 --> 02:19:28,089
DIFFERENT SOLUTIONS TO THE 

2926
02:19:28,089 --> 02:19:28,322
PROBLEM.

2927
02:19:28,322 --> 02:19:30,725
YOU CAN APPRECIATE IF YOU START 

2928
02:19:30,725 --> 02:19:32,193
TAKING A DOUBLE APPROACH WHERE 

2929
02:19:32,193 --> 02:19:35,029
YOU NEED TO KNOCK DOWN AND OVER 

2930
02:19:35,029 --> 02:19:36,698
EXPRESS, THAT'S EFFECTIVELY LIKE

2931
02:19:36,698 --> 02:19:38,633
TWO DRUG DEVELOPMENT PROGRAMS 

2932
02:19:38,633 --> 02:19:40,068
WRAPPED INTO ONE.

2933
02:19:40,068 --> 02:19:45,506
WE  -- IT CAN GET COMPLICATED 

2934
02:19:45,506 --> 02:19:46,340
FAST AND ADDS TO THE COMPLEXITY 

2935
02:19:46,340 --> 02:19:47,208
OF IT.

2936
02:19:47,208 --> 02:19:51,512
I THINK THIS IS WHERE MODELS 

2937
02:19:51,512 --> 02:19:54,282
LIKE ALFRED AND KRISTIN TALKED 

2938
02:19:54,282 --> 02:19:57,218
ABOUT ARE SO IMPORTANT RATHER 

2939
02:19:57,218 --> 02:19:59,153
THAN TACKLING THE TOTALITY OF 

2940
02:19:59,153 --> 02:20:02,757
EVERYTHING IN AN IN VIVO MODEL 

2941
02:20:02,757 --> 02:20:06,928
PARS THEM OUT AND ASK TARGETED 

2942
02:20:06,928 --> 02:20:09,497
QUESTIONS THE IN VIVO MODELS AND

2943
02:20:09,497 --> 02:20:11,966
BUILD THAT BACK UP TO, LOOK, I 

2944
02:20:11,966 --> 02:20:15,036
THINK THIS IS THE RIGHT SOLUTION

2945
02:20:15,036 --> 02:20:20,641
FOR THIS DISEASE.

2946
02:20:20,641 --> 02:20:21,342
>> OKAY.

2947
02:20:21,342 --> 02:20:25,513
I WILL GO AHEAD AND ASK THE NEXT

2948
02:20:25,513 --> 02:20:25,780
QUESTION.

2949
02:20:25,780 --> 02:20:27,915
SO ARE WE LEARNING ANYTHING FROM

2950
02:20:27,915 --> 02:20:34,622
THE LUNG CANCER DRUG DEVELOPMENT

2951
02:20:34,622 --> 02:20:34,889
COMMUNITY?

2952
02:20:34,889 --> 02:20:37,358
GRANTED THE PATHOPHYSIOLOGY IS 

2953
02:20:37,358 --> 02:20:40,061
DIFFERENT BUT IMAGINE THERE ARE 

2954
02:20:40,061 --> 02:20:42,930
SIMILAR ISSUES WITH MODELS TO 

2955
02:20:42,930 --> 02:20:44,732
TEST THAT DON'T ADEQUATELY 

2956
02:20:44,732 --> 02:20:46,667
REPRESENT HUMAN DISEASE.

2957
02:20:46,667 --> 02:20:47,268
SO ANY THOUGHTS ON CANCER 

2958
02:20:47,268 --> 02:20:57,411
MODELS?

2959
02:20:58,980 --> 02:21:03,751
>> THAT'S A TOUGH ONE.

2960
02:21:03,751 --> 02:21:06,454
CYSTIC FIBROSIS.

2961
02:21:06,454 --> 02:21:08,456
THERE'S A LOT OF BIOLOGY WHEN WE

2962
02:21:08,456 --> 02:21:11,025
THINK OF STEM CELLS THAT 

2963
02:21:11,025 --> 02:21:11,826
REGENERATE THERE'S A LOT OF 

2964
02:21:11,826 --> 02:21:14,962
BIOLOGY SIMILAR TO CANCER THAT'S

2965
02:21:14,962 --> 02:21:16,564
GONE AWRY.

2966
02:21:16,564 --> 02:21:18,466
AND IN THE PULMONARY FIBROSIS I 

2967
02:21:18,466 --> 02:21:23,004
TALKED ABOUT THE FIBROBLASTS ARE

2968
02:21:23,004 --> 02:21:24,572
SECRETING MOLECULES THAT ARE 

2969
02:21:24,572 --> 02:21:27,508
DRIVING DYSPLASTIC REPAIR AND 

2970
02:21:27,508 --> 02:21:32,146
REGENERATION OF AIRWAY 

2971
02:21:32,146 --> 02:21:33,514
CONDUCTING CELLS IN THE AIR 

2972
02:21:33,514 --> 02:21:33,848
SPACE.

2973
02:21:33,848 --> 02:21:35,016
IT'S ALL ABOUT COMMUNICATION 

2974
02:21:35,016 --> 02:21:41,556
REPAIR AND RESOLVE OF AN INJURY 

2975
02:21:41,556 --> 02:21:42,723
RESPONSE AND IN THE DISEASES 

2976
02:21:42,723 --> 02:21:43,891
VERY CHRONIC SOME PROCESSES ARE 

2977
02:21:43,891 --> 02:21:50,131
RELATED TO PATHWAYS THAT GO AWRY

2978
02:21:50,131 --> 02:22:00,608
IN CANCER AND CROSS GENETIC 

2979
02:22:25,867 --> 02:22:31,038
SPACE IN CANCER OR GENETIC OR 

2980
02:22:31,038 --> 02:22:35,843
CHRONIC DISEASE IN THE LUNG.

2981
02:22:35,843 --> 02:22:38,913
>> WE'RE MODELLING
FIBROSIS AND 

2982
02:22:38,913 --> 02:22:41,816
WE SEE THAT AS JUST THE 

2983
02:22:41,816 --> 02:22:44,318
PRELIMINARY PROCESSES BEFORE 

2984
02:22:44,318 --> 02:22:45,519
LUNG CANCER DEVELOPMENT.

2985
02:22:45,519 --> 02:22:51,025
AND I THINK WE'RE STARTING TO 

2986
02:22:51,025 --> 02:22:55,029
WANT TO BE ABLE TO MODEL THAT 

2987
02:22:55,029 --> 02:22:57,231
THE PROGRESSION FROM FIBROSIS ON

2988
02:22:57,231 --> 02:23:00,301
A CHIP AND I AGREE THERE SHOULD 

2989
02:23:00,301 --> 02:23:02,803
BE MORE COLLABORATION BETWEEN 

2990
02:23:02,803 --> 02:23:06,507
THE FIELDS AND I HOPE WE CAN SEE

2991
02:23:06,507 --> 02:23:06,974
THAT.

2992
02:23:06,974 --> 02:23:08,809
>> THERE MAY ALSO BE
OPPORTUNITY

2993
02:23:08,809 --> 02:23:09,911
TO LOOK AT SOME OF THE 

2994
02:23:09,911 --> 02:23:11,879
COMPENSATORY CHANGES THAT HAPPEN

2995
02:23:11,879 --> 02:23:13,281
IN THE ONCOGENIC STATE.

2996
02:23:13,281 --> 02:23:14,749
WE TALKED ABOUT A NUMBER OF 

2997
02:23:14,749 --> 02:23:17,051
TRANSPORTERS THAT ARE ASSOCIATED

2998
02:23:17,051 --> 02:23:20,154
WITH DIFFERENT DISEASES AS WELL.

2999
02:23:20,154 --> 02:23:25,559
WE CERTAINLY KNOW FROM A 

3000
02:23:25,559 --> 02:23:26,227
MULTI-DRUG RESISTANT STANDPOINT 

3001
02:23:26,227 --> 02:23:33,634
MANY ARE UPRENREGULATED SPACE I 

3002
02:23:33,634 --> 02:23:35,469
THE TRANSPORTERS THEY ARE PLAY 

3003
02:23:35,469 --> 02:23:42,043
COME INTO PLAY FROM A MULTI-DRUG

3004
02:23:42,043 --> 02:23:43,010
RESISTANT STANDPOINT.

3005
02:23:43,010 --> 02:23:44,445
THAT'S NOT MY AREA OF EXPERTISE 

3006
02:23:44,445 --> 02:23:46,314
BUT I CAN SEE THE WHERE THE 

3007
02:23:46,314 --> 02:23:48,149
LEARNINGS IN THE BIOLOGY 

3008
02:23:48,149 --> 02:23:49,050
ULTIMATELY NEED TO BE ADDRESSED 

3009
02:23:49,050 --> 02:23:59,560
IN THE LUNG CANCER SPACE TOO.

3010
02:24:02,229 --> 02:24:07,001
>> I HAVE A QUESTION FOR
THE 

3011
02:24:07,001 --> 02:24:08,069
PANEL.

3012
02:24:08,069 --> 02:24:11,005
IT'S LISTENING TO THE DISCUSSION

3013
02:24:11,005 --> 02:24:15,443
IT BRINGS TO THE FORE ONE OF MY 

3014
02:24:15,443 --> 02:24:17,712
CONCERNS AROUND THE IN VITRO 

3015
02:24:17,712 --> 02:24:20,314
MODEL IS WE CAN'T ADEQUATELY 

3016
02:24:20,314 --> 02:24:23,017
MODEL THE EXTRA CELLULAR SPACE 

3017
02:24:23,017 --> 02:24:27,021
WITH THE MILIEU THE DRUG 

3018
02:24:27,021 --> 02:24:30,291
ENCOUNTERS WHEN IT'S BEING 

3019
02:24:30,291 --> 02:24:35,396
ADMINISTERED AND BRINGS TO MIND 

3020
02:24:35,396 --> 02:24:40,935
THE CONCERNS AROUND CAN TO TO 

3021
02:24:40,935 --> 02:24:51,479
BETTER MODEL THAT AND THE SECOND

3022
02:24:56,884 --> 02:25:00,921
QUESTION IN THE SESSION IS MAYBE

3023
02:25:00,921 --> 02:25:03,491
MORE DIRECTED TO JOHN IF WE ARE 

3024
02:25:03,491 --> 02:25:05,226
THINKING THAT THERE'S A 

3025
02:25:05,226 --> 02:25:06,861
REGENERATIVE ASPECT OF THIS AND 

3026
02:25:06,861 --> 02:25:11,966
WE'RE LOOKING AT A DISEASE THE 

3027
02:25:11,966 --> 02:25:13,834
CELLULAR ENVIRONMENT OF THAT IS 

3028
02:25:13,834 --> 02:25:15,936
NOT REALLY GOING TO BE SIMILAR 

3029
02:25:15,936 --> 02:25:22,043
TO WHAT WE'RE LOOKING AT IN A 

3030
02:25:22,043 --> 02:25:32,586
DISH AND IN AND MAY BE CELLULAR 

3031
02:25:33,054 --> 02:25:34,722
DAMAGE AND HOW DO YOU SEE 

3032
02:25:34,722 --> 02:25:36,290
REGENERATION WHEN WE DELIVER OR 

3033
02:25:36,290 --> 02:25:40,094
GENE, OUR DRUGS, ARE WE THINKING

3034
02:25:40,094 --> 02:25:43,030
THAT SOME OF THE EFFECTS NOT 

3035
02:25:43,030 --> 02:25:45,599
NECESSARILY EVEN ON THE CELLS 

3036
02:25:45,599 --> 02:25:49,236
ACTIVELY PARTICIPATING UP 

3037
02:25:49,236 --> 02:25:54,442
SECRETION BUT PERHAPS PERHAPS IF

3038
02:25:54,442 --> 02:25:56,077
WE INTRODUCE OUR BASAL CELLS AND

3039
02:25:56,077 --> 02:25:57,478
LAG TIME AND HAVE GENERATION OF 

3040
02:25:57,478 --> 02:26:01,248
LUNG AND THEN GOING TO SEE THE 

3041
02:26:01,248 --> 02:26:01,482
EFFECTS.

3042
02:26:01,482 --> 02:26:04,251
CAN YOU COMMENT ON ANY OF THOSE 

3043
02:26:04,251 --> 02:26:08,656
SOMEWHAT RANDOM QUESTIONS?

3044
02:26:08,656 --> 02:26:10,224
>> THERE'S A LOT TO UNPACK

3045
02:26:10,224 --> 02:26:10,491
THERE.

3046
02:26:10,491 --> 02:26:11,625
GETTING TO YOUR FIRST QUESTION 

3047
02:26:11,625 --> 02:26:15,029
IS HOW CAN YOU PREDICT WHAT IS 

3048
02:26:15,029 --> 02:26:17,898
GOING TO BE MOST RELEVANT IN 

3049
02:26:17,898 --> 02:26:18,999
VIVO AND I'D SAY YOU CAN'T 

3050
02:26:18,999 --> 02:26:20,301
ALWAYS PREDICT.

3051
02:26:20,301 --> 02:26:23,938
YOU START WITH -- WE TALKED 

3052
02:26:23,938 --> 02:26:29,176
ABOUT KRISTIN'S MODEL IN VITRO 

3053
02:26:29,176 --> 02:26:30,845
ON A CHIP MODEL IS INTRIGUING.

3054
02:26:30,845 --> 02:26:34,515
SAY YOU HAVE A SMALL DRUG AND 

3055
02:26:34,515 --> 02:26:39,186
YOU DO SOME PRELIMINARY STABLES 

3056
02:26:39,186 --> 02:26:45,426
IN VIVO AND CROSS THE 

3057
02:26:45,426 --> 02:26:48,529
VASCULATURE AND HOW TO IMPROVE 

3058
02:26:48,529 --> 02:26:51,031
THE PERMEABILITY START WITH A 

3059
02:26:51,031 --> 02:26:52,299
MODEL THAT IS EASY LIKE IN VITRO

3060
02:26:52,299 --> 02:26:57,238
OR EASIER THAN DOING A LOT OF 

3061
02:26:57,238 --> 02:27:00,074
ANIMALS IN VITRO ON A CHIP AND 

3062
02:27:00,074 --> 02:27:01,175
CAN SCREEN AND UNDERSTAND THE 

3063
02:27:01,175 --> 02:27:05,179
CHEMISTRY THAT NEEDS TO BE 

3064
02:27:05,179 --> 02:27:06,347
CHANGED AND THEN GO IN VIVO AND 

3065
02:27:06,347 --> 02:27:08,649
ASK SPECIFIC QUESTIONS ON A 

3066
02:27:08,649 --> 02:27:10,117
SUBSET OF WHAT YOU MIGHT HAVE 

3067
02:27:10,117 --> 02:27:11,051
SCREENED WHETHER OR NOT THE 

3068
02:27:11,051 --> 02:27:14,321
BIOLOGY IS RETAINED WHEN YOU 

3069
02:27:14,321 --> 02:27:16,657
ACROSS TO IN VIVO.

3070
02:27:16,657 --> 02:27:18,959
THE SECOND QUESTION ABOUT 

3071
02:27:18,959 --> 02:27:21,529
PROGENITOR CELLS, CERTAINLY ANY 

3072
02:27:21,529 --> 02:27:26,834
REMODELLED ORGAN IS GOING TO BE 

3073
02:27:26,834 --> 02:27:28,302
MORE DIFFICULT TO FROM A GENE 

3074
02:27:28,302 --> 02:27:29,436
THERAPY STANDPOINT.

3075
02:27:29,436 --> 02:27:30,938
IF YOU'RE TARGETING BASAL CELLS 

3076
02:27:30,938 --> 02:27:33,507
YOU NEED A GEN EDITING APPROACH.

3077
02:27:33,507 --> 02:27:36,210
IF YOU TAKE A VIRUS YOU STICK IT

3078
02:27:36,210 --> 02:27:38,679
IN AND BASAL CELL REGENERATES 

3079
02:27:38,679 --> 02:27:40,648
AND DIVIDES THE VIRUS UNLESS 

3080
02:27:40,648 --> 02:27:42,516
IT'S INTEGRATED IS GOING TO BE 

3081
02:27:42,516 --> 02:27:45,252
DILUTED AS THE REGENERATIVE 

3082
02:27:45,252 --> 02:27:46,086
PROCESS GOES ON.

3083
02:27:46,086 --> 02:27:50,124
HAVE YOU THE INTEGRATOR AND EDIT

3084
02:27:50,124 --> 02:27:51,025
THE MUTANT GENE.

3085
02:27:51,025 --> 02:27:54,795
AND REPAIRING THE REMODELLED 

3086
02:27:54,795 --> 02:27:56,564
LUNG OR WHATEVER YOUR TARGET IS 

3087
02:27:56,564 --> 02:28:01,735
WILL BE A LONGER PROCESS SOME 

3088
02:28:01,735 --> 02:28:03,571
WON'T REPAIR FROM MODELLING BUT 

3089
02:28:03,571 --> 02:28:07,942
FROM A GENE EDITING STANDPOINT 

3090
02:28:07,942 --> 02:28:10,311
THAT'S THE WAY TO GET THE CARGO 

3091
02:28:10,311 --> 02:28:11,879
TO THE RIGHT PLACE AND YOU TA 

3092
02:28:11,879 --> 02:28:13,280
YOU CAN ABOUT BASAL CELLS AS 

3093
02:28:13,280 --> 02:28:15,516
BEING THE TARGET IT'S PROBABLY 

3094
02:28:15,516 --> 02:28:18,953
1% OF BASAL CELLS ARE REALLY THE

3095
02:28:18,953 --> 02:28:19,987
ULTIMATE STEM CELL WILL PERSIST 

3096
02:28:19,987 --> 02:28:30,497
FOR THE LIFE OF AN INDIVIDUAL.

3097
02:28:33,567 --> 02:28:34,702
>> THANK YOU ALL.

3098
02:28:34,702 --> 02:28:37,338
THANK YOU TO THE PANEL FOR 

3099
02:28:37,338 --> 02:28:39,006
SHARING THEIR EXPERT OPINION AND

3100
02:28:39,006 --> 02:28:39,707
SHARING THEIR EXCITING RESEARCH 

3101
02:28:39,707 --> 02:28:40,174
WITH US.

3102
02:28:40,174 --> 02:28:42,209
I THINK WE'LL COME TO OUR LUNCH 

3103
02:28:42,209 --> 02:28:44,078
BREAK AND COME BACK AT 12:45 FOR

3104
02:28:44,078 --> 02:28:44,478
THE NEXT SESSION.

3105
02:28:44,478 --> 02:28:47,390
THANK YOU ALL.

3106
02:28:47,390 --> 02:28:48,791
>> HELLO AND THANK YOU ALL
FOR 

3107
02:28:48,791 --> 02:28:49,292
COMING BACK.

3108
02:28:49,292 --> 02:28:51,895
WE ARE GOING TO START THE SECOND

3109
02:28:51,895 --> 02:28:57,467
SESSION OF TODAY WHICH IS 

3110
02:28:57,467 --> 02:29:06,342
PARTNERING WITH REGULATORS FOR 

3111
02:29:06,342 --> 02:29:06,843
SUCCESSION AND NOW TO 

3112
02:29:06,843 --> 02:29:08,978
DR. MOFFITT.

3113
02:29:08,978 --> 02:29:10,914
>> IF THIS IMPORTANT WAS 

3114
02:29:10,914 --> 02:29:12,715
FOCUSSED TO DEVELOPING YOUR 

3115
02:29:12,715 --> 02:29:14,384
ASSETS TO HAVE THE PHARMACOLOGY 

3116
02:29:14,384 --> 02:29:16,419
AND SAFETY PROFILE, THIS 

3117
02:29:16,419 --> 02:29:18,488
AFTERNOON IS MORE FACING 

3118
02:29:18,488 --> 02:29:18,755
OUTWARDLY.

3119
02:29:18,755 --> 02:29:22,492
HOW DO YOU INTERACT WITH 

3120
02:29:22,492 --> 02:29:22,792
REGULATORS?

3121
02:29:22,792 --> 02:29:23,726
WHAT ARE THE EXPECTATIONS YOU 

3122
02:29:23,726 --> 02:29:26,262
HAVE FOR YOUR PROGRAMS AND HOW 

3123
02:29:26,262 --> 02:29:28,565
DO YOU NAVIGATE THAT PROCESS 

3124
02:29:28,565 --> 02:29:29,699
WHETHER YOU HAVE THE DRUG ABOUT 

3125
02:29:29,699 --> 02:29:32,402
ITSELF OR WORK TOGETHER WITH A 

3126
02:29:32,402 --> 02:29:35,505
DEVICE TO DELIVER THAT DRUG OR 

3127
02:29:35,505 --> 02:29:38,241
EVEN HAVE COMPANION DIAGNOSTICS 

3128
02:29:38,241 --> 02:29:39,509
YOU CAN THINK OF.

3129
02:29:39,509 --> 02:29:42,111
THERE'S REGULATORY COMPONENTS.

3130
02:29:42,111 --> 02:29:44,647
WE'LL HEAR A LITTLE BIT ABOUT 

3131
02:29:44,647 --> 02:29:45,882
THE OVER ALL REGULATORY PROCESS.

3132
02:29:45,882 --> 02:29:48,017
IN ADDITION WE'LL HEAR HOW YOU 

3133
02:29:48,017 --> 02:29:50,486
MARKET YOUR PROGRAM.

3134
02:29:50,486 --> 02:29:54,390
ESSENTIALLY WORKING WITH YOUR 

3135
02:29:54,390 --> 02:29:57,327
OWN INTERNAL PROGRAMS AND MAKE 

3136
02:29:57,327 --> 02:30:00,763
THE PACKAGE THAT LOOKS LIKE 

3137
02:30:00,763 --> 02:30:03,900
SOMETHING LIKE VENTURE CAPITAL 

3138
02:30:03,900 --> 02:30:06,469
OR PHARMA TECH COMPANIES MAY BE 

3139
02:30:06,469 --> 02:30:08,071
INTERESTED IN AROUND IP AND 

3140
02:30:08,071 --> 02:30:09,472
OTHER CONSIDERATIONS.

3141
02:30:09,472 --> 02:30:10,873
FINALLY, WE'LL TALK ABOUT 

3142
02:30:10,873 --> 02:30:12,342
GOVERNMENT REGULATORY MECHANISMS

3143
02:30:12,342 --> 02:30:14,877
THERE TO HELP INCENTIVIZE 

3144
02:30:14,877 --> 02:30:16,746
BUSINESS DEVELOPMENT AND GUIDE 

3145
02:30:16,746 --> 02:30:18,214
FOLKS THROUGH THE PROCESS GOING 

3146
02:30:18,214 --> 02:30:19,449
TO FORWARD.

3147
02:30:19,449 --> 02:30:20,883
SO, I THINK IT'S GOING TO TO BE 

3148
02:30:20,883 --> 02:30:22,185
A REALLY INTERESTING TALK ABOUT 

3149
02:30:22,185 --> 02:30:23,553
THE NEXT PHASE OF DRUG 

3150
02:30:23,553 --> 02:30:24,854
DEVELOPMENT AS YOU'RE TRYING TO 

3151
02:30:24,854 --> 02:30:27,357
GET THE WORD OUT AND INTERFACE 

3152
02:30:27,357 --> 02:30:37,767
WITH THE EXTERNAL WORLD.

3153
02:30:39,569 --> 02:30:41,004
THE FIRST SPEAKER IS DR. MOODY 

3154
02:30:41,004 --> 02:30:46,509
DIRECTOR OF REGULATORY AFARDZ 

3155
02:30:46,509 --> 02:30:54,150
 -- AFFAIRS IN RTI AND WORKED IN

3156
02:30:54,150 --> 02:30:56,819
NON-CLINICAL AREAS AND CMC AND A

3157
02:30:56,819 --> 02:30:58,488
WEALTH OF ADVICE COMING FROM HER

3158
02:30:58,488 --> 02:30:59,889
TODAY AROUND HOW TO NAVIGATE 

3159
02:30:59,889 --> 02:31:10,300
THAT REGULATORY PROCESS.

3160
02:31:11,968 --> 02:31:17,940
SECOND SPEAKER IS KEVIN 

3161
02:31:17,940 --> 02:31:22,211
WHITTLESEY AN EXPERT IN ALL 

3162
02:31:22,211 --> 02:31:22,945
PHASES.

3163
02:31:22,945 --> 02:31:25,782
HE'S WORKED FOR THE FDA AND 

3164
02:31:25,782 --> 02:31:27,317
WORKED FOR A COMPANY GETTING 

3165
02:31:27,317 --> 02:31:28,951
ASSETS PAST THE IND STAGE INTO 

3166
02:31:28,951 --> 02:31:30,486
DEVELOPMENT AND FINALLY HE'S 

3167
02:31:30,486 --> 02:31:33,222
BEEN A CONSULTANT FOR A NUMBER 

3168
02:31:33,222 --> 02:31:34,791
OF YEARLY YEARS PARTNERING WITH 

3169
02:31:34,791 --> 02:31:37,760
COMPANIES SMALL AND LARGE TO 

3170
02:31:37,760 --> 02:31:38,494
HELP THEM ADVANCE THEIR PRODUCTS

3171
02:31:38,494 --> 02:31:48,237
GOING FORWARD.

3172
02:31:48,237 --> 02:31:51,741
NEXT WE HAVE DR. CLARK AT THE 

3173
02:31:51,741 --> 02:31:54,510
UNIVERSITY OF KENTUCKY WORKING 

3174
02:31:54,510 --> 02:31:55,812
TO DEVELOP THERAPEUTIC 

3175
02:31:55,812 --> 02:31:58,481
PORTFOLIOS FOR FOLKS WITHIN THAT

3176
02:31:58,481 --> 02:32:00,483
ORGANIZATION AND DEVELOP 

3177
02:32:00,483 --> 02:32:03,586
BUSINESS MODELS AROUND HOW TO 

3178
02:32:03,586 --> 02:32:06,522
EXTERNALIZE THESE NEW 

3179
02:32:06,522 --> 02:32:07,924
TECHNOLOGIES.

3180
02:32:07,924 --> 02:32:12,595
DR. STEPHANIE DAVIS IS A SMALL 

3181
02:32:12,595 --> 02:32:14,497
BUSINESS PROGRAM COORDINATOR AT 

3182
02:32:14,497 --> 02:32:14,697
NHLBI.

3183
02:32:14,697 --> 02:32:16,766
SHE'S BEEN IN THIS ROLE A NUMBER

3184
02:32:16,766 --> 02:32:18,267
OF YEARS NOW AND HAD FORMALLY 

3185
02:32:18,267 --> 02:32:21,070
BEEN A POSTDOC AND IS A TRAINING

3186
02:32:21,070 --> 02:32:21,637
BY SCIENTIST.

3187
02:32:21,637 --> 02:32:24,073
SHE HAD INTERNED AT THE 

3188
02:32:24,073 --> 02:32:25,308
UNIVERSITY OF KENTUCKY PROGRAM 

3189
02:32:25,308 --> 02:32:25,608
AS WELL TOO.

3190
02:32:25,608 --> 02:32:28,211
SHE'LL BE SHARING SOME OF HER 

3191
02:32:28,211 --> 02:32:31,681
GUIDANCE AND INSIGHT AND FINALLY

3192
02:32:31,681 --> 02:32:36,953
DR. MIKE PECK WHO IS DIRECTOR OF

3193
02:32:36,953 --> 02:32:39,021
THE NHLBI CATALYZE PROGRAM AND 

3194
02:32:39,021 --> 02:32:40,323
WILL WALK US THROUGH KEY 

3195
02:32:40,323 --> 02:32:43,326
ACTIVITIES TO LEVERAGE AND THINK

3196
02:32:43,326 --> 02:32:46,195
ABOUT TO TRANSLATE OUR IDEAS 

3197
02:32:46,195 --> 02:32:48,364
INTO A COMMERCIAL OPPORTUNITY 

3198
02:32:48,364 --> 02:32:50,066
AND LEVERAGE THE GOVERNMENT 

3199
02:32:50,066 --> 02:32:50,500
OPPORTUNITIES THERE.

3200
02:32:50,500 --> 02:32:54,570
WITH THAT I'LL TURN IT OVER TO 

3201
02:32:54,570 --> 02:32:57,840
DR. MOODY.

3202
02:32:57,840 --> 02:32:58,474
>> GOOD AFTERNOON.

3203
02:32:58,474 --> 02:33:00,410
THANK YOU SO MUCH FOR JOINING 

3204
02:33:00,410 --> 02:33:02,478
US.

3205
02:33:02,478 --> 02:33:06,482
I'M GOING TO TO BE TALKING ABOUT

3206
02:33:06,482 --> 02:33:11,320
HOW TO PREPARE FOR PRE-IND 

3207
02:33:11,320 --> 02:33:12,255
MIDDLEWEIGHT THE FIRST MEETING 

3208
02:33:12,255 --> 02:33:14,490
YOU HAVE DURING DEVELOPMENT AND 

3209
02:33:14,490 --> 02:33:24,867
THEN GO INTO THE IND.

3210
02:33:26,569 --> 02:33:28,738
THE BIG QUESTION IS WHY DO I 

3211
02:33:28,738 --> 02:33:31,207
NEED A PRE-IND OR INTERACT 

3212
02:33:31,207 --> 02:33:31,874
MEETING WITH THE FDA.

3213
02:33:31,874 --> 02:33:34,477
THE ANSWER IS YOU DO NOT NEED TO

3214
02:33:34,477 --> 02:33:35,745
HAVE ONE.

3215
02:33:35,745 --> 02:33:38,147
THEY'RE NOT MANDATORY BY ANY 

3216
02:33:38,147 --> 02:33:39,649
MEANS BUT IT'S A GOOD IDEA 

3217
02:33:39,649 --> 02:33:41,150
ESPECIALLY IF YOU HAVE A NEW 

3218
02:33:41,150 --> 02:33:47,824
MOLECULAR ENTITY ORE A DRUG NOT 

3219
02:33:47,824 --> 02:33:52,328
PREVIOUSLY APPROVED TO TWO TO 

3220
02:33:52,328 --> 02:33:54,430
THE REVIEW TEAM AND GET TARGETED

3221
02:33:54,430 --> 02:33:56,933
ADVICE TO HELP YOU DEVELOP YOUR 

3222
02:33:56,933 --> 02:34:00,736
PRODUCT AND AVOID COSTLY 

3223
02:34:00,736 --> 02:34:01,003
MISTAKES.

3224
02:34:01,003 --> 02:34:03,940
AGAIN, JUST TO ESTABLISH A 

3225
02:34:03,940 --> 02:34:05,842
RAPPORT AND TRUST WITH THE FDA 

3226
02:34:05,842 --> 02:34:13,549
REVIEW DIVISION.

3227
02:34:13,549 --> 02:34:14,484
THE FDA GIVES A SPECIFIC 

3228
02:34:14,484 --> 02:34:17,487
PERSPECTIVE ON THE BENEFITS OF A

3229
02:34:17,487 --> 02:34:18,488
PRE-IND MEETING.

3230
02:34:18,488 --> 02:34:20,823
AND ONE OF THE MAIN THINGS THEY 

3231
02:34:20,823 --> 02:34:26,395
TELL YOU IS IT MIN MIEDZS -- 

3232
02:34:26,395 --> 02:34:27,296
MINIMIZES THE POTENTIAL FOR 

3233
02:34:27,296 --> 02:34:27,997
CLINICAL HOLD.

3234
02:34:27,997 --> 02:34:29,932
WHAT THAT DOES IS IT GIVES YOU 

3235
02:34:29,932 --> 02:34:31,167
THE OPPORTUNITY AGAIN TO GO OVER

3236
02:34:31,167 --> 02:34:35,071
THE BASICS OF ALL THREE OF YOUR 

3237
02:34:35,071 --> 02:34:40,042
BASIC PROGRAMS AND DISCUSS WITH 

3238
02:34:40,042 --> 02:34:42,578
THEM WHERE YOU ARE AND WHAT 

3239
02:34:42,578 --> 02:34:44,347
SAFETY YOU'LL BE PRESENTING AND 

3240
02:34:44,347 --> 02:34:45,748
IT WILL HELP MINIMIZE COST, 

3241
02:34:45,748 --> 02:34:50,486
AVOID UNNECESSARY STUDIES, AS 

3242
02:34:50,486 --> 02:34:58,995
WELL AS MANY OTHER THINGS.

3243
02:34:58,995 --> 02:35:01,664
WHAT IS A PRE-IND MEETING?

3244
02:35:01,664 --> 02:35:03,299
IT'S A MEETING CALLED A TYPE B 

3245
02:35:03,299 --> 02:35:04,000
MEETING.

3246
02:35:04,000 --> 02:35:08,771
IT'S A FORMAL MEETING WITH THE 

3247
02:35:08,771 --> 02:35:09,105
FDA.

3248
02:35:09,105 --> 02:35:11,841
ONCE YOU REQUEST THE MEETING THE

3249
02:35:11,841 --> 02:35:13,576
FDA WILL SCHEDULE THAT MEETING 

3250
02:35:13,576 --> 02:35:14,176
WITHIN 60 DAYS OF THE MEETING 

3251
02:35:14,176 --> 02:35:23,819
REQUEST.

3252
02:35:23,819 --> 02:35:25,354
YOU'LL HAVE TO SUBMIT A BRIEFING

3253
02:35:25,354 --> 02:35:26,589
DOCUMENT THAT PROVIDES THE 

3254
02:35:26,589 --> 02:35:30,126
QUESTIONS YOU WANT TO DISCUSS 

3255
02:35:30,126 --> 02:35:36,766
AND SUPPORTING INFORMATION TO 

3256
02:35:36,766 --> 02:35:38,501
GIVE THE FDA GUIDANCE ABOUT 

3257
02:35:38,501 --> 02:35:40,436
ANSWERING QUESTIONS.

3258
02:35:40,436 --> 02:35:44,574
IT'S GREAT TO HAVE A PLAN FOR A 

3259
02:35:44,574 --> 02:35:45,875
PRE-IND MEETING BEFORE THE IND 

3260
02:35:45,875 --> 02:35:46,509
SUBMISSION.

3261
02:35:46,509 --> 02:35:52,615
THIS GIVES YOU A CHANCE TO GET 

3262
02:35:52,615 --> 02:35:57,720
THINGS INFLATION -- IN PLACE IF 

3263
02:35:57,720 --> 02:35:59,055
YOU NEED TO.

3264
02:35:59,055 --> 02:35:59,989
IT'S GOOD TO HAVE PRELIMINARY 

3265
02:35:59,989 --> 02:36:05,661
CONCEPT AND SAFETY STUDIES DONE.

3266
02:36:05,661 --> 02:36:06,929
WHEN YOU HAVE DEVELOPED YOUR 

3267
02:36:06,929 --> 02:36:10,399
MANUFACTURING PROCESS YOU MAY 

3268
02:36:10,399 --> 02:36:13,202
NOT BE IN GNP MANUFACTURING YET 

3269
02:36:13,202 --> 02:36:15,471
BUT DEFINE THE MANUFACTURING 

3270
02:36:15,471 --> 02:36:17,974
PROCESS AND BEFORE YOU'RE 

3271
02:36:17,974 --> 02:36:21,110
DEFINITIVE GOP TOX STUDIES.

3272
02:36:21,110 --> 02:36:23,079
ONE OF THE MAIN THINGS THAT WILL

3273
02:36:23,079 --> 02:36:27,116
BE HELPFUL TO DISCUSS AT THE 

3274
02:36:27,116 --> 02:36:31,220
PRE-IND MEETING IS HOW YOU PLAN 

3275
02:36:31,220 --> 02:36:34,290
TO RUN THE GLP MEETINGS AND HOW 

3276
02:36:34,290 --> 02:36:38,494
THEY SUPPORT YOUR PHASE I 

3277
02:36:38,494 --> 02:36:42,498
PROTOCOL.

3278
02:36:42,498 --> 02:36:45,101
WHAT TOPIC SHOULD BE DISCUSSED 

3279
02:36:45,101 --> 02:36:46,836
SALT THE PRE-IND MEETING?

3280
02:36:46,836 --> 02:36:49,005
IT'S ABOUT THE DESIGN AND 

3281
02:36:49,005 --> 02:36:55,311
DURATION OF THE GLP TOX STUDIES.

3282
02:36:55,311 --> 02:36:58,481
ISSUES WITH WITH YOUR CMC AND 

3283
02:36:58,481 --> 02:37:03,719
DEVELOPING COMPARABILITY STUDIES

3284
02:37:03,719 --> 02:37:06,489
OR ANY SPECIFIC MANUFACTURING 

3285
02:37:06,489 --> 02:37:07,523
ISSUES YOU HAD CAN DISCUSS 

3286
02:37:07,523 --> 02:37:09,291
THEM -- WELL, YOU NEED TO HAVE 

3287
02:37:09,291 --> 02:37:12,728
YOUR PROTOCOL SYNOPSIS OR 

3288
02:37:12,728 --> 02:37:14,897
PROTOCOL AND WE RECOMMEND YOU 

3289
02:37:14,897 --> 02:37:18,534
HAVE A DRAFT PROTOCOL IN YOUR 

3290
02:37:18,534 --> 02:37:19,301
BRIEFING DOCUMENT FOR YOUR FIRST

3291
02:37:19,301 --> 02:37:24,306
IN MAN STUDIES.

3292
02:37:24,306 --> 02:37:26,475
AND DISCUSS YOUR REGIMENT AND 

3293
02:37:26,475 --> 02:37:28,277
DEVELOPMENT CHALLENGES YOU HAVE 

3294
02:37:28,277 --> 02:37:32,081
AND REALLY GO OVER ANY TOPICS 

3295
02:37:32,081 --> 02:37:33,382
THAT THERE'S NO REALLY AN FDA 

3296
02:37:33,382 --> 02:37:41,490
GUIDANCE FOR.

3297
02:37:41,490 --> 02:37:43,426
AN INTERACT MEETING CAN OCCUR 

3298
02:37:43,426 --> 02:37:45,327
BEFORE THE PRE IND MEETING.

3299
02:37:45,327 --> 02:37:50,499
IT'S A NEWER FORMAL MEETING THE 

3300
02:37:50,499 --> 02:37:51,000
FDA ESTABLISHED.

3301
02:37:51,000 --> 02:37:54,336
AND BASICALLY THE INTERACT 

3302
02:37:54,336 --> 02:37:56,806
MEETING IS SCHEDULED 75 DAYS 

3303
02:37:56,806 --> 02:37:58,541
BEFORE THE MEETING.

3304
02:37:58,541 --> 02:38:00,409
ONCE YOU SUBMIT THE MEETING 

3305
02:38:00,409 --> 02:38:02,945
REQUEST AND THE IMPORTANT POINT 

3306
02:38:02,945 --> 02:38:05,981
IS YOU'RE BRIEFING BOOK OR YOUR 

3307
02:38:05,981 --> 02:38:10,252
MEETING PACKAGE BACKGROUND NEEDS

3308
02:38:10,252 --> 02:38:12,188
TO BE SUBMITTED WITH THE MEETING

3309
02:38:12,188 --> 02:38:12,455
REQUEST.

3310
02:38:12,455 --> 02:38:15,591
SO WHEN DO YOU HAVE YOUR 

3311
02:38:15,591 --> 02:38:16,425
INTERACT MEETING?

3312
02:38:16,425 --> 02:38:18,427
IT'S EARLY IN THE DEVELOPMENT.

3313
02:38:18,427 --> 02:38:21,330
USUALLY AFTER YOU HAVE YOUR 

3314
02:38:21,330 --> 02:38:24,033
COMPOUND IDENTIFIED, YOU'VE GOT 

3315
02:38:24,033 --> 02:38:26,502
PRE CLINICAL PROOF OF CONCEPT OR

3316
02:38:26,502 --> 02:38:28,137
PILOT DATA AND AGAIN YOU HAVEN'T

3317
02:38:28,137 --> 02:38:33,342
DONE YOUR DEFINITIVE TOX STUDIES

3318
02:38:33,342 --> 02:38:36,045
BUT MAYBE YOU HAVE SOME ANIMAL 

3319
02:38:36,045 --> 02:38:41,150
STUDIES IMPORTANT TO DISCUSS AND

3320
02:38:41,150 --> 02:38:45,321
AGAIN MANUFACTURING PLAN 

3321
02:38:45,321 --> 02:38:52,328
STRATEGY FOR GOING FORWARD.

3322
02:38:52,328 --> 02:38:56,298
AGAIN, WHAT SHOULD BE DISCUSSED?

3323
02:38:56,298 --> 02:38:57,900
NOVEL PROGRAMS AND IT'S VERY 

3324
02:38:57,900 --> 02:39:00,536
IMPORTANT TO DISCUSS YOUR 

3325
02:39:00,536 --> 02:39:01,203
PRECLINICAL MODELS, THE 

3326
02:39:01,203 --> 02:39:03,973
APPROPRIATE PRECLINICAL MODELS 

3327
02:39:03,973 --> 02:39:08,410
OR THE TOXICOLOGY STUDIES FOR 

3328
02:39:08,410 --> 02:39:09,278
NOVEL PRODUCTS.

3329
02:39:09,278 --> 02:39:10,880
CMC REQUIREMENTS AND ANY 

3330
02:39:10,880 --> 02:39:14,283
SPECIFIC PLANS THAT YOU HAVE TO 

3331
02:39:14,283 --> 02:39:15,684
DEMONSTRATE PRODUCT SAFETY.

3332
02:39:15,684 --> 02:39:17,520
YOUR PROOF OF CONCEPT STUDY 

3333
02:39:17,520 --> 02:39:24,627
DESIGN.

3334
02:39:24,627 --> 02:39:25,561
YOUR RECOMMENDATIONS ABOUT THE 

3335
02:39:25,561 --> 02:39:28,264
FIRST IN HUMAN TRIAL.

3336
02:39:28,264 --> 02:39:31,267
USUALLY THAT'S NOT SOMETHING YOU

3337
02:39:31,267 --> 02:39:33,602
SPEND AS MUCH TIME ON IN AN 

3338
02:39:33,602 --> 02:39:34,503
INTERACT MEETING.

3339
02:39:34,503 --> 02:39:38,674
BUT ANYTHING THAT IF YOU WANT TO

3340
02:39:38,674 --> 02:39:40,609
USE ANY DATA COMING FROM OUTSIDE

3341
02:39:40,609 --> 02:39:42,812
THE U.S. YOU DISCUSS IT WITH THE

3342
02:39:42,812 --> 02:39:51,587
AGENCY.

3343
02:39:51,587 --> 02:39:53,155
AND YOU DON'T HAVE TO ANSWER THE

3344
02:39:53,155 --> 02:39:54,557
IN THE CHAT BUT SHOULD YOU 

3345
02:39:54,557 --> 02:39:59,094
SCHEDULE A PRE-IND MEETING WITH 

3346
02:39:59,094 --> 02:40:02,832
FDA AS EARLY IN DEVELOPMENT AS 

3347
02:40:02,832 --> 02:40:03,966
POSSIBLE AND PEOPLE THINK IT'S 

3348
02:40:03,966 --> 02:40:06,468
TRUE BUT IT'S FALSE.

3349
02:40:06,468 --> 02:40:13,175
YOU WANT TO SCHEDULE YOUR P

3350
02:40:13,175 --> 02:40:14,109
PRE-IND MEETING AND PROOF OF 

3351
02:40:14,109 --> 02:40:16,579
CONCEPT AND A YEAR BEFORE YOU'RE

3352
02:40:16,579 --> 02:40:24,620
PLANNING TO FILE YOUR IND.

3353
02:40:24,620 --> 02:40:26,288
IT'S REALLY IMPORTANT WHEN YOU 

3354
02:40:26,288 --> 02:40:31,861
GO TO THE FDA ABOUT HOW YOU ASK 

3355
02:40:31,861 --> 02:40:33,462
YOUR QUESTIONS AND THE FDA -- 

3356
02:40:33,462 --> 02:40:34,964
YOUR FDA QUESTION SHOULD BE 

3357
02:40:34,964 --> 02:40:42,471
HIGHLY SCRIPTED AND FORMAL.

3358
02:40:42,471 --> 02:40:44,840
HOW DO YOU ASK AN FDA QUESTION?

3359
02:40:44,840 --> 02:40:46,508
USUALLY QUESTIONS ARE TYPICALLY 

3360
02:40:46,508 --> 02:40:52,414
YES OR NO QUESTIONS AND YOU 

3361
02:40:52,414 --> 02:40:55,584
FRAME THEM AS DOES THE AGENCY 

3362
02:40:55,584 --> 02:40:55,784
AGREE?

3363
02:40:55,784 --> 02:40:59,955
SO YOU PRESENT WHAT YOUR 

3364
02:40:59,955 --> 02:41:01,924
STRATEGY IS AND THEN ASK THE FDA

3365
02:41:01,924 --> 02:41:03,359
IF THEY AGREE WITH YOUR 

3366
02:41:03,359 --> 02:41:08,430
STRATEGY, IF THEY THINK IT'S 

3367
02:41:08,430 --> 02:41:10,299
TRUE OR REASONABLE AND THE UNTO 

3368
02:41:10,299 --> 02:41:13,569
ACT MEETING YOU CAN HAVE LESS 

3369
02:41:13,569 --> 02:41:16,272
STRUCTURED QUESTIONS BUT FOR 

3370
02:41:16,272 --> 02:41:18,440
YOUR PRE-IND YOU REALLY NEED FOR

3371
02:41:18,440 --> 02:41:20,109
THEM TO BE MORE STRUCTURED.

3372
02:41:20,109 --> 02:41:21,377
FREQUENTLY THE FDA'S GOING TO 

3373
02:41:21,377 --> 02:41:24,647
SAY NO, WE DO NOT AGREE BUT 

3374
02:41:24,647 --> 02:41:28,484
DON'T STRESS BECAUSE OFTEN TIMES

3375
02:41:28,484 --> 02:41:31,453
WHEN THEY DON'T AGREE -- NOT 

3376
02:41:31,453 --> 02:41:36,792
OFTEN TIMES BUT THEY PROVIDE YOU

3377
02:41:36,792 --> 02:41:39,328
WITH ON WHY THEY DON'T AGREE AND

3378
02:41:39,328 --> 02:41:42,498
TRY TO MAKE SURE THEY UNDERSTAND

3379
02:41:42,498 --> 02:41:52,408
WHY THEY DON'T AGREE.

3380
02:41:52,408 --> 02:41:54,576
AND IF YOU DO CELEBRATE AND RUSH

3381
02:41:54,576 --> 02:42:01,517
TO THE NEXT THING.

3382
02:42:01,517 --> 02:42:04,286
AND YOU HAVE TO REMEMBER THE FDA

3383
02:42:04,286 --> 02:42:07,656
ARE NOT REGULATORY OR MANAGEMENT

3384
02:42:07,656 --> 02:42:08,157
CONSULTANTS.

3385
02:42:08,157 --> 02:42:12,227
YOU DO NOT GO TO THE FDA WITH 

3386
02:42:12,227 --> 02:42:15,798
BROAD OPEN ENDED QUESTIONS LIKE 

3387
02:42:15,798 --> 02:42:18,500
WHAT DOES THE FDA THINK OF OUR 

3388
02:42:18,500 --> 02:42:19,535
DEVELOPMENT PLAN.

3389
02:42:19,535 --> 02:42:21,337
THAT'S WAY TOO BROAD AND WAY TOO

3390
02:42:21,337 --> 02:42:22,071
OPEN ENDED.

3391
02:42:22,071 --> 02:42:27,343
WHAT YOU WANT TO DO IS YOU WANT 

3392
02:42:27,343 --> 02:42:29,545
TO SPEND TIME GOING THROUGH AND 

3393
02:42:29,545 --> 02:42:35,684
WHAT SPECIFICALLY YOUR QUESTION 

3394
02:42:35,684 --> 02:42:36,251
IS.

3395
02:42:36,251 --> 02:42:39,555
AND YOU CAN GET SPECIFIC 

3396
02:42:39,555 --> 02:42:40,789
FOCUSSED INFORMATION ON YOUR 

3397
02:42:40,789 --> 02:42:41,056
QUESTION.

3398
02:42:41,056 --> 02:42:42,324
BECAUSE IF YOU ASK A GENERAL 

3399
02:42:42,324 --> 02:42:44,526
QUESTION YOUR LIKELY TO GET A 

3400
02:42:44,526 --> 02:42:46,028
GENERAL RESPONSE OR LIKELY TO 

3401
02:42:46,028 --> 02:42:50,499
GET A RESPONSE BASED ON THE 

3402
02:42:50,499 --> 02:42:52,134
INFORMATION PROVIDED.

3403
02:42:52,134 --> 02:42:57,339
WE CAN'T ANSWER THIS QUESTION.

3404
02:42:57,339 --> 02:42:59,074
I RECOMMEND SPENDING A LOT OF 

3405
02:42:59,074 --> 02:43:04,113
TIME THINKING ABOUT THE QUESTION

3406
02:43:04,113 --> 02:43:10,486
YOU WANT TO ASK.

3407
02:43:10,486 --> 02:43:14,590
AND LIMIT QUESTIONS IN YOUR MAIN

3408
02:43:14,590 --> 02:43:21,330
REQUEST TO TEN.

3409
02:43:21,330 --> 02:43:24,633
AN USE QUESTIONS TO REALLY ADD 

3410
02:43:24,633 --> 02:43:26,468
THE NUMBER AND INCREASE THE 

3411
02:43:26,468 --> 02:43:31,306
NUMBER OF QUESTIONS.

3412
02:43:31,306 --> 02:43:33,609
IF YOU HAVE A ONE-TIME INFORMAL 

3413
02:43:33,609 --> 02:43:35,944
QUESTION, YOU CAN REACH OUT TO 

3414
02:43:35,944 --> 02:43:40,883
YOUR REGULATORY PROJECT MANAGER 

3415
02:43:40,883 --> 02:43:42,351
AND DISCUSS WHAT YOUR SPECIFIC 

3416
02:43:42,351 --> 02:43:44,153
ISSUES ARE AND THE REGULATORY 

3417
02:43:44,153 --> 02:43:45,421
PROJECT MANGE WILL GIVE YOU 

3418
02:43:45,421 --> 02:43:46,388
ADVICE ABOUT WHETHER YOU NEED A 

3419
02:43:46,388 --> 02:43:56,665
MEETING OR NOT.

3420
02:43:58,033 --> 02:43:59,401
AND ONE WEEK BEFORE THE MEETING 

3421
02:43:59,401 --> 02:44:03,605
THE FDA WILL USUALLY SEND YOU A 

3422
02:44:03,605 --> 02:44:06,208
PRELIMINARY RESPONSE, 24 TO 72 

3423
02:44:06,208 --> 02:44:06,475
HOURS.

3424
02:44:06,475 --> 02:44:08,210
I SAY USUALLY BUT NOW IT'S 

3425
02:44:08,210 --> 02:44:12,114
PRETTY MUCH ALL THE TIME.

3426
02:44:12,114 --> 02:44:16,552
THEY'RE GOING TO RESPOND TO YOUR

3427
02:44:16,552 --> 02:44:18,954
QUESTIONS IN THAT 24 TO 48 HOUR 

3428
02:44:18,954 --> 02:44:26,995
TIME FRAME BEFORE THE MEETING.

3429
02:44:26,995 --> 02:44:28,363
AND USE THE PRELIMINARY 

3430
02:44:28,363 --> 02:44:31,233
RESPONSES AND MAKE SURE THE FDA 

3431
02:44:31,233 --> 02:44:34,903
ANSWERED THE QUESTIONS TO THE 

3432
02:44:34,903 --> 02:44:36,038
POINT THAT THEY CAN UNDERSTAND 

3433
02:44:36,038 --> 02:44:42,211
THE FDA'S POINT.

3434
02:44:42,211 --> 02:44:45,347
AND IF THEY STOP ANSWERING TOE 

3435
02:44:45,347 --> 02:44:50,486
THIS TEAM'S SATISFACTION YOU CAN

3436
02:44:50,486 --> 02:44:51,987
CANCEL THE MEETING AND NOT HAVE 

3437
02:44:51,987 --> 02:44:52,554
THE MEETING.

3438
02:44:52,554 --> 02:44:53,689
IF FURTHER CLARIFICATION IS 

3439
02:44:53,689 --> 02:44:54,857
NEEDED FOR THE QUESTION USUALLY 

3440
02:44:54,857 --> 02:44:58,760
OUT OF YOUR 10 YOU MAY HAVE 

3441
02:44:58,760 --> 02:45:00,295
THREE OR FOUR QUESTIONS THAT 

3442
02:45:00,295 --> 02:45:00,863
NEED FURTHER CLARIFICATION.

3443
02:45:00,863 --> 02:45:05,234
WHAT YOU WANT TO DO IS GET THAT 

3444
02:45:05,234 --> 02:45:08,170
TO THE PROJECT MANAGER AND SAY 

3445
02:45:08,170 --> 02:45:10,372
OUR TEAM IS INTERESTED IN HAVING

3446
02:45:10,372 --> 02:45:16,678
A DISCUSSION ABOUT 3, 5 AND 7 

3447
02:45:16,678 --> 02:45:18,147
AND WANT NUMBER 7 TO BE THE 

3448
02:45:18,147 --> 02:45:28,657
PRIORITY QUESTION TO DISCUSS.

3449
02:45:33,395 --> 02:45:38,400
AND HOLD A MOCK MEET AND HAVE 

3450
02:45:38,400 --> 02:45:39,334
SOMEONE PLAY THE ROLE OF AN FDA 

3451
02:45:39,334 --> 02:45:39,601
REVIEWER.

3452
02:45:39,601 --> 02:45:42,004
[NO AUDIO]

3453
02:45:42,004 --> 02:45:52,247
OR APPLICANTS.

3454
02:45:53,949 --> 02:45:56,919
IT'S AN MORE MEETING AND THE RPM

3455
02:45:56,919 --> 02:45:57,920
CHAIRS THE MEETING.

3456
02:45:57,920 --> 02:45:59,755
THE FIRST FIVE MINUTES ARE OR 

3457
02:45:59,755 --> 02:46:00,989
INTRODUCTION TO THE FDA AND 

3458
02:46:00,989 --> 02:46:01,223
SPONSOR.

3459
02:46:01,223 --> 02:46:03,091
NO LONGER IT USED TO BE WHEN I 

3460
02:46:03,091 --> 02:46:06,061
FIRST GOT STARTED THE COMPANY 

3461
02:46:06,061 --> 02:46:07,696
GAVE INFORMATION ON THE COMPANY 

3462
02:46:07,696 --> 02:46:11,266
AND THINGS LIKE THAT NO LONGER 

3463
02:46:11,266 --> 02:46:14,136
DO WE DO THAT AND WE HAD BASIC 

3464
02:46:14,136 --> 02:46:15,571
INTRODUCTIONS AND THAT ALLOWS 

3465
02:46:15,571 --> 02:46:19,808
FOR THE NEXT 50 MINUTES FOR 

3466
02:46:19,808 --> 02:46:21,376
DISCUSSION OF THE QUESTIONS 

3467
02:46:21,376 --> 02:46:23,212
AGAIN IN THE ORDER YOU NOTIFIED 

3468
02:46:23,212 --> 02:46:33,622
THE RPM AS THE PRIORITY.

3469
02:46:39,294 --> 02:46:41,563
THE SUBJECT MATTER EXPERT CAN 

3470
02:46:41,563 --> 02:46:42,931
GIVE A BRIEF EXPOSITION OR 

3471
02:46:42,931 --> 02:46:45,334
BACKGROUND ON THE SPECIFIC 

3472
02:46:45,334 --> 02:46:48,870
QUESTION.

3473
02:46:48,870 --> 02:46:50,505
USUALLY THAT'S THE MOST 

3474
02:46:50,505 --> 02:46:52,507
INFORMATION OR EXPLANATORY 

3475
02:46:52,507 --> 02:46:53,408
INFORMATION YOU'LL HAVE.

3476
02:46:53,408 --> 02:46:59,681
AND LIST OF PRIMARY QUESTIONS 

3477
02:46:59,681 --> 02:47:00,682
FIRST THEN FIVE MINUTES BEFORE 

3478
02:47:00,682 --> 02:47:02,484
THE MEETING ENDS IT'S A GOOD 

3479
02:47:02,484 --> 02:47:06,054
IDEA FOR THE SPONSOR TO GIVE A 

3480
02:47:06,054 --> 02:47:08,457
SUMMARY STATEMENT OF BASICALLY 

3481
02:47:08,457 --> 02:47:09,758
THEIR UNDERSTANDING AND 

3482
02:47:09,758 --> 02:47:11,760
TAKEAWAYS AND ACTION ITEMS.

3483
02:47:11,760 --> 02:47:14,963
AND AGAIN IT'S REALLY IMPORTANT 

3484
02:47:14,963 --> 02:47:19,534
TO LISTEN AND TRY NOT TO ARGUE 

3485
02:47:19,534 --> 02:47:20,269
WITH THE FDA.

3486
02:47:20,269 --> 02:47:24,039
AUTHOR EASY TO TALK TO IN MY 

3487
02:47:24,039 --> 02:47:25,040
EXPERIENCE AND REALLY WANT TO 

3488
02:47:25,040 --> 02:47:26,375
HELP YOU SO YOU NEED TO GO INTO 

3489
02:47:26,375 --> 02:47:33,548
THE MEETING WITH THAT IDEA.

3490
02:47:33,548 --> 02:47:35,350
SO THE POST MEETING WRITE UP 

3491
02:47:35,350 --> 02:47:36,385
YOUR MEETING MINUTES IMMEDIATELY

3492
02:47:36,385 --> 02:47:37,486
AFTER THE MEETING AND CIRCULATE 

3493
02:47:37,486 --> 02:47:39,154
TO THE INTERNAL TEAM.

3494
02:47:39,154 --> 02:47:40,889
SO EVERYBODY ON THE TEAM SHOULD 

3495
02:47:40,889 --> 02:47:44,760
BE TAKING NOTES WHILE THE 

3496
02:47:44,760 --> 02:47:45,827
MEETING'S GOING ON.

3497
02:47:45,827 --> 02:47:47,596
YOU PUT YOUR NOTES TOGETHER AND 

3498
02:47:47,596 --> 02:47:51,366
MAKE SURE THAT YOU ALL ARE 

3499
02:47:51,366 --> 02:47:54,503
UNDERSTANDING WHAT YOU THINK 

3500
02:47:54,503 --> 02:48:05,013
YOU'VE AGREED WITH THE FDA I 

3501
02:48:08,183 --> 02:48:11,987
IT'S NOT THE FORM TO SEND THE 

3502
02:48:11,987 --> 02:48:13,722
MEETING MINUTES BUT IT USED TO 

3503
02:48:13,722 --> 02:48:16,591
BE AND THEY'LL SEND THEM 30 DAYS

3504
02:48:16,591 --> 02:48:17,726
WITHIN THE MEETING AND IT'S WEST

3505
02:48:17,726 --> 02:48:23,231
TO PUT YOUR TEAM TOGETHER, 

3506
02:48:23,231 --> 02:48:24,900
REVIEW THE FDA'S INFORMATION 

3507
02:48:24,900 --> 02:48:27,102
MAKE SURE THE INFORMATION THE 

3508
02:48:27,102 --> 02:48:28,603
FDA PROVIDED IS RELEVANT AND 

3509
02:48:28,603 --> 02:48:29,771
APPROPRIATE FOR WHAT YOU THINK 

3510
02:48:29,771 --> 02:48:36,178
YOU DISCUSSED AND WHAT YOU 

3511
02:48:36,178 --> 02:48:37,079
AGREED AND IF THERE'S ANYTHING 

3512
02:48:37,079 --> 02:48:39,348
YOU THINK IS NOT APPROPRIATE OR 

3513
02:48:39,348 --> 02:48:44,686
NOT EXACTLY RIGHT THE SPONSOR 

3514
02:48:44,686 --> 02:48:47,989
CAN SUBMIT A REQUEST FOR 

3515
02:48:47,989 --> 02:48:49,024
CLARIFICATION WITHIN 30 DAYS OF 

3516
02:48:49,024 --> 02:48:50,892
THE RECEIPT OF THE MEETING AND 

3517
02:48:50,892 --> 02:48:52,527
COME BACK TO YOU WITH RESPONSES 

3518
02:48:52,527 --> 02:49:02,571
TO YOUR CLARIFICATION.

3519
02:49:02,571 --> 02:49:09,811
AND WE'LL PREPARE THE FDA SMICKS

3520
02:49:09,811 --> 02:49:10,579
WHAT'S AN IND?

3521
02:49:10,579 --> 02:49:12,647
AN IND IS A DOCUMENT YOU SUBMIT 

3522
02:49:12,647 --> 02:49:14,116
TO THE AGENCY TO ALLOW YOU TO GO

3523
02:49:14,116 --> 02:49:18,787
INTO CLINICAL STUDIES.

3524
02:49:18,787 --> 02:49:20,322
THERE'S THREE MAIN PARTS TO THE 

3525
02:49:20,322 --> 02:49:20,889
IND.

3526
02:49:20,889 --> 02:49:22,290
THE NON-CLINICAL INFORMATION, 

3527
02:49:22,290 --> 02:49:26,495
WHICH IS THE RESULTS OF YOUR 

3528
02:49:26,495 --> 02:49:29,498
PRE-CLINICAL DEVELOPMENT PROGRAM

3529
02:49:29,498 --> 02:49:31,933
INCLUDING YOUR GLP TOXICOLOGY 

3530
02:49:31,933 --> 02:49:32,167
STUDIES.

3531
02:49:32,167 --> 02:49:33,602
THE OVER ALL GOAL OF THAT IS TO 

3532
02:49:33,602 --> 02:49:41,243
ASSURE YOU HAVE ADEQUATE SAFETY 

3533
02:49:41,243 --> 02:49:44,546
AND THE CHEMISTRY CONTROL WHICH 

3534
02:49:44,546 --> 02:49:46,915
IS INFORMATION ON THE DETAILS OF

3535
02:49:46,915 --> 02:49:49,651
YOUR DRUG SUBSTANCE OR API SOME 

3536
02:49:49,651 --> 02:49:50,819
CALL IT AND THE DRUG PRODUCT 

3537
02:49:50,819 --> 02:49:59,161
MANUFACTURING.

3538
02:49:59,161 --> 02:50:02,130
YOU CAN MAKE AND TEST YOUR 

3539
02:50:02,130 --> 02:50:05,267
PRODUCT USING GOOD MANUFACTURING

3540
02:50:05,267 --> 02:50:06,468
PRACTICE AND A BRIDGE BETWEEN 

3541
02:50:06,468 --> 02:50:08,904
THE MATERIAL YOU USED IN YOUR 

3542
02:50:08,904 --> 02:50:15,343
NON-CLINICAL GLP STUDIES TO THE 

3543
02:50:15,343 --> 02:50:17,012
MATERIAL YOU'RE GOING TO USE IN 

3544
02:50:17,012 --> 02:50:18,313
YOUR CLINICAL TRIALS.

3545
02:50:18,313 --> 02:50:19,414
IT DOESN'T HAVE TO BE EXACTLY 

3546
02:50:19,414 --> 02:50:20,682
THE SAME BUT THE FDA WILL REVIEW

3547
02:50:20,682 --> 02:50:27,722
THE TATE FROM THE NON-CLINICAL 

3548
02:50:27,722 --> 02:50:30,358
MATERIAL AND CLINICAL MATERIAL 

3549
02:50:30,358 --> 02:50:32,861
TO MAKE SURE YOU HAVE COVERAGE.

3550
02:50:32,861 --> 02:50:36,031
INCLUDING THE PROTOCOL FOR THE 

3551
02:50:36,031 --> 02:50:37,766
PHASE 1 CLINICAL TRIAL AND 

3552
02:50:37,766 --> 02:50:38,467
INVESTIGATOR BROCHURE.

3553
02:50:38,467 --> 02:50:42,370
YOU NEED TO REASSURE THE FDA THE

3554
02:50:42,370 --> 02:50:45,373
PROPOSED STUDIES WILL BE SAFE, 

3555
02:50:45,373 --> 02:50:53,982
THEY'RE SCIENTIFICALLY VALID AND

3556
02:50:53,982 --> 02:50:56,685
NOT EXCESSIVELY BURDENSOME AND 

3557
02:50:56,685 --> 02:51:03,825
GOOD CONTENT AND FORMAT FOR THE 

3558
02:51:03,825 --> 02:51:04,860
GUIDANCE DOCUMENT AND STILL VOLE

3559
02:51:04,860 --> 02:51:15,437
RANT AND APPROPRIATE NOW.

3560
02:51:15,437 --> 02:51:17,706
THERE'S AN INTRODUCTORY FORM OF 

3561
02:51:17,706 --> 02:51:21,343
THE 1571 IN YOUR COVER LETTER.

3562
02:51:21,343 --> 02:51:22,477
THAT'S THE ADMINISTRATIVE 

3563
02:51:22,477 --> 02:51:22,777
INFORMATION.

3564
02:51:22,777 --> 02:51:28,383
THEN YOU PROVIDE AN INTRODUCTORY

3565
02:51:28,383 --> 02:51:30,819
SUMMARY AND WHAT YOU WANT TO GET

3566
02:51:30,819 --> 02:51:32,554
ACROSS TO THE AGENCY.

3567
02:51:32,554 --> 02:51:33,755
THEN THERE'S THE GENERAL 

3568
02:51:33,755 --> 02:51:34,990
INVESTIGATIONAL PLAN AND THAT'S 

3569
02:51:34,990 --> 02:51:36,925
USUALLY ONE TO TWO PAGES AND THE

3570
02:51:36,925 --> 02:51:39,227
PURPOSE IS TO LET THE AGENCY 

3571
02:51:39,227 --> 02:51:42,964
KNOW WHAT YOU'RE GOING TO DO.

3572
02:51:42,964 --> 02:51:43,932
NON CLINICALLY AND CLINICALLY 

3573
02:51:43,932 --> 02:51:54,543
FROM THE NEXT YEAR REALLY.

3574
02:51:54,543 --> 02:51:56,177
PROVIDE YOUR PROTOCOL AND 

3575
02:51:56,177 --> 02:51:58,113
STARTED SIX TO 12 MONTHS BEFORE 

3576
02:51:58,113 --> 02:52:02,484
YOUR IND AND I LIKE TO INCLUDE A

3577
02:52:02,484 --> 02:52:05,420
DRAFT PROTOCOL IN THE PRE-IND 

3578
02:52:05,420 --> 02:52:06,054
BRIEFING DOCUMENT BECAUSE THE 

3579
02:52:06,054 --> 02:52:07,989
FDA VEEP FOR QUESTIONS, EVEN FOR

3580
02:52:07,989 --> 02:52:13,562
THINGS IRE IN YOUR PROTOCOL YOU 

3581
02:52:13,562 --> 02:52:15,964
MAY NOT HAVE ASKED A QUESTION 

3582
02:52:15,964 --> 02:52:18,833
OFTEN THE AGENCY NOTICES A FLAG 

3583
02:52:18,833 --> 02:52:21,570
THEY'LL GIVE YOU ADVICE ON 

3584
02:52:21,570 --> 02:52:23,772
SPECIFIC INFORMATION IN YOUR 

3585
02:52:23,772 --> 02:52:25,674
PROTOCOL THAT MAYBE YOU DID NOT 

3586
02:52:25,674 --> 02:52:32,981
HAVE THE QUESTION FOR THE PR

3587
02:52:32,981 --> 02:52:35,550
PRE-IND THEN YOU WANT THE 

3588
02:52:35,550 --> 02:52:36,751
INVESTIGATOR'S BROCHURE AND 

3589
02:52:36,751 --> 02:52:38,219
START THAT NINE MONTHS BEFORE 

3590
02:52:38,219 --> 02:52:44,059
YOUR IND SUBMISSION AND IT'S 

3591
02:52:44,059 --> 02:52:46,494
REALLY TO COVER IN SUMMARY FORM 

3592
02:52:46,494 --> 02:52:51,366
THE INVESTIGATORS CAN UNDERSTAND

3593
02:52:51,366 --> 02:52:57,806
THE INFORMATION KWLUN CAL 

3594
02:52:57,806 --> 02:52:59,608
INFORMATION YOU THINK IS 

3595
02:52:59,608 --> 02:53:02,477
IMPORTANT FOR THE INVESTIGATOR 

3596
02:53:02,477 --> 02:53:12,721
TO UNDERSTAND.

3597
02:53:18,560 --> 02:53:22,497
INCLUDE INVESTIGATOR ON THE DATA

3598
02:53:22,497 --> 02:53:26,234
ON FORM 1572 AND PRESENT THE CVs

3599
02:53:26,234 --> 02:53:27,569
OF YOUR INVESTIGATORS.

3600
02:53:27,569 --> 02:53:31,673
THE IND DATA AND PROVIDE AN IND 

3601
02:53:31,673 --> 02:53:34,075
ROSTER SO THE AGENCY KNOWS WHO 

3602
02:53:34,075 --> 02:53:36,344
THE IRB WAS THAT REVIEWED YOUR 

3603
02:53:36,344 --> 02:53:39,681
CLINICAL PROTOCOL.

3604
02:53:39,681 --> 02:53:41,683
YOU CAN PROVIDE YOUR INFORMED 

3605
02:53:41,683 --> 02:53:46,521
CONSENT IF THERE'S SOMETHING 

3606
02:53:46,521 --> 02:53:51,393
SPECIFIC OR IF YOU HAVE A 

3607
02:53:51,393 --> 02:53:53,328
DIFFICULT PROTOCOL OR PATIENT 

3608
02:53:53,328 --> 02:53:54,929
POPULATION, YOU MAY WANT TO 

3609
02:53:54,929 --> 02:53:56,398
INCLUDE YOUR INFORMED CONSENT 

3610
02:53:56,398 --> 02:53:57,365
FORM AND ASK THE FDA FOR 

3611
02:53:57,365 --> 02:54:07,542
FEEDBACK.

3612
02:54:09,010 --> 02:54:11,680
IF YOU DO A LITERATURE SEARCH IF

3613
02:54:11,680 --> 02:54:13,615
THERE'S RELEVANCE TO INCLUDE ANY

3614
02:54:13,615 --> 02:54:14,516
INFORMATION ON YOUR PRODUCT OR 

3615
02:54:14,516 --> 02:54:21,189
VERY SIMILAR PRODUCTS.

3616
02:54:21,189 --> 02:54:26,761
YOU HAVE TO SUBMIT THE FORM 3674

3617
02:54:26,761 --> 02:54:28,797
SAYING YOU'LL REGISTER YOUR 

3618
02:54:28,797 --> 02:54:30,832
STUDY WITH CLINICALTRIALS.gov.

3619
02:54:30,832 --> 02:54:35,203
MOST THE TIME YOUR PHASE I 

3620
02:54:35,203 --> 02:54:36,971
PROTOCOL IS INCLUDED BUT LOOK AT

3621
02:54:36,971 --> 02:54:40,442
THE FORM AND MAKE SURE THAT 

3622
02:54:40,442 --> 02:54:47,716
YOU'RE PHASE 1 IS EXCLUDED.

3623
02:54:47,716 --> 02:54:50,852
 A RISK ASSESSMENT STATEMENT AND

3624
02:54:50,852 --> 02:54:53,221
BASIC INFORMATION ON LABELLING 

3625
02:54:53,221 --> 02:54:58,493
AND INCLUDE THE COVER SHEET IF 

3626
02:54:58,493 --> 02:55:04,199
IT'S RELEVANT BUT IT'S USUALLY 

3627
02:55:04,199 --> 02:55:05,567
NOT NEEDED.

3628
02:55:05,567 --> 02:55:11,272
SO NOW FOR ALL COMMERCIAL 

3629
02:55:11,272 --> 02:55:13,041
SUBMISSIONS, WHICH ARE MOST IND 

3630
02:55:13,041 --> 02:55:16,678
SUBMISSIONS, THE IND HAS TO BE 

3631
02:55:16,678 --> 02:55:18,146
SUBMITTED IN THE ELECTRONIC 

3632
02:55:18,146 --> 02:55:20,982
COMMON DOCUMENT FORMAT.

3633
02:55:20,982 --> 02:55:26,187
THIS IS THE INFORMATION THERE'S 

3634
02:55:26,187 --> 02:55:29,290
FIVE MODULES IN THE ECTD.

3635
02:55:29,290 --> 02:55:30,492
MODULE 1 IS ADMINISTRATIVE THE 

3636
02:55:30,492 --> 02:55:34,262
COVER LETTER AND 1571 GOES AND 

3637
02:55:34,262 --> 02:55:38,466
INTRODUCTORY STATEMENT AND 

3638
02:55:38,466 --> 02:55:39,400
INVESTIGATOR BROCHURES AND 

3639
02:55:39,400 --> 02:55:45,373
INCLUDES SUMMARIES FOR 

3640
02:55:45,373 --> 02:55:50,278
NON-CLINICAL CMC AND CLINICAL 

3641
02:55:50,278 --> 02:55:53,414
SUMMARY IF APPLICABLE BUT OFTEN 

3642
02:55:53,414 --> 02:55:59,821
FOR PHASE I IT'S NOT AND MODULE 

3643
02:55:59,821 --> 02:56:03,324
4 IS THE STUDY REPORT AND MODULE

3644
02:56:03,324 --> 02:56:06,461
5 ARE STUDY REPORTS.

3645
02:56:06,461 --> 02:56:09,130
IT'S IMPORTANT TO GET REGULATORY

3646
02:56:09,130 --> 02:56:12,267
ASSISTANCE NOW BECAUSE NOT ONLY 

3647
02:56:12,267 --> 02:56:19,941
IS THERE SPECIFIC FORMAT FOR THE

3648
02:56:19,941 --> 02:56:27,382
ECTD YOU CAN GET WITH RTI AND WE

3649
02:56:27,382 --> 02:56:37,625
CAN HELP YOU.

3650
02:56:41,763 --> 02:56:42,497
WE'RE GOING TO TALK ABOUT 

3651
02:56:42,497 --> 02:56:43,965
PROGRAMS FOR EXPIRED CONDITIONS 

3652
02:56:43,965 --> 02:56:44,999
AND THERE'S FAST TRACK, WHICH IS

3653
02:56:44,999 --> 02:56:50,605
WHEN YOU HAVE NON-CLINICAL OR 

3654
02:56:50,605 --> 02:56:52,674
CLINICAL DATA THAT POTENTIALLY 

3655
02:56:52,674 --> 02:56:56,911
ADDRESS AN UNMET MEDICAL NEED.

3656
02:56:56,911 --> 02:56:58,479
ALSO WHEN YOUR PRODUCT'S BEEN 

3657
02:56:58,479 --> 02:57:00,048
DESIGNATED AS A QUALIFIED 

3658
02:57:00,048 --> 02:57:05,019
INFECTIOUS DISEASE PRODUCT.

3659
02:57:05,019 --> 02:57:13,628
YOU CAN SUBMIT AND THE PRE-IND 

3660
02:57:13,628 --> 02:57:15,763
MEETING AND IT GRANTS YOU 

3661
02:57:15,763 --> 02:57:18,466
ACTIACTION

3662
02:57:18,466 --> 02:57:20,635
S EXPEDITE ROLLING REVIEW WHICH 

3663
02:57:20,635 --> 02:57:24,572
IS A REVIEW WHERE YOU SUBMIT 

3664
02:57:24,572 --> 02:57:30,478
PORTIONS OF YOUR MARKETING 

3665
02:57:30,478 --> 02:57:36,651
APPLICATION PERIODICALLY AND 

3666
02:57:36,651 --> 02:57:40,421
THEN THERE'S BREAKTHROUGH AND 

3667
02:57:40,421 --> 02:57:42,290
IT'S HARDER TO GET A 

3668
02:57:42,290 --> 02:57:43,558
BREAKTHROUGH INDICATION.

3669
02:57:43,558 --> 02:57:46,327
IT'S WHERE YOU PRESENT 

3670
02:57:46,327 --> 02:57:47,662
PRELIMINARY CLINICAL EVIDENCE 

3671
02:57:47,662 --> 02:57:49,831
THAT DEMONSTRATES THIS IS 

3672
02:57:49,831 --> 02:57:52,567
IMPORTANT -- THAT DEMONSTRATES 

3673
02:57:52,567 --> 02:57:55,303
SUBSTANTIAL IMPROVEMENT ON A 

3674
02:57:55,303 --> 02:57:56,604
CLINICALLY SIGNIFICANT END POINT

3675
02:57:56,604 --> 02:58:02,243
OVER AVAILABLE THERAPIES.

3676
02:58:02,243 --> 02:58:04,579
SUBMIT THE APPLICATION WITH OR 

3677
02:58:04,579 --> 02:58:06,981
WITHOUT THE IND BUT BEFORE THE 

3678
02:58:06,981 --> 02:58:09,851
END OF PHASE II AND GRANTS YOU 

3679
02:58:09,851 --> 02:58:15,023
INTENSIVE GUIDANCE AND ROLLING 

3680
02:58:15,023 --> 02:58:18,559
REVIEW.

3681
02:58:18,559 --> 02:58:22,897
SO SOME TALK HOME PEARLS, DO 

3682
02:58:22,897 --> 02:58:24,599
ENGAGE THE TEAM EARLY AND 

3683
02:58:24,599 --> 02:58:26,467
CONSIDER AN INTERACT MEETING AND

3684
02:58:26,467 --> 02:58:31,806
PRE-IND MEETING IF YOUR PRODUCT 

3685
02:58:31,806 --> 02:58:32,707
IS NOVEL.

3686
02:58:32,707 --> 02:58:39,213
FOR ME FAMILIARIZE YOUR GUIDANCE

3687
02:58:39,213 --> 02:58:40,815
AND START NINE TO 12 MONTHS 

3688
02:58:40,815 --> 02:58:41,449
BEFORE SUBMISSION AND EARLIER IF

3689
02:58:41,449 --> 02:58:47,789
YOU CAN.

3690
02:58:47,789 --> 02:58:49,557
DON'T ASK THE FDA OPEN ENDED 

3691
02:58:49,557 --> 02:58:50,258
QUESTIONS.

3692
02:58:50,258 --> 02:58:51,392
ASSUME INTERACTIONS WITH THE FDA

3693
02:58:51,392 --> 02:58:57,231
ARE IN FORMAL.

3694
02:58:57,231 --> 02:58:58,900
TREATS THEM AS FORMAL.

3695
02:58:58,900 --> 02:59:04,505
HAVE YOUR QUESTIONS WORDED 

3696
02:59:04,505 --> 02:59:14,749
APPROPRIA

3697
02:59:20,488 --> 02:59:23,057
APPROPRIATELY AND IT'S A BEST TO

3698
02:59:23,057 --> 02:59:27,095
KNOW THE ANSWERS NOW BEFORE YOUR

3699
02:59:27,095 --> 02:59:30,465
IND THAN IT IS TO GET TO YOUR 

3700
02:59:30,465 --> 02:59:40,408
IND CLINICAL.

3701
02:59:40,408 --> 02:59:41,275
AND THERE'S DIFFERENT TYPES OF 

3702
02:59:41,275 --> 02:59:44,045
FDA MEETINGS AND ABCD AND 

3703
02:59:44,045 --> 02:59:44,312
INTERACT.

3704
02:59:44,312 --> 02:59:46,147
WE'RE NOT GOING TO GO THROUGH 

3705
02:59:46,147 --> 02:59:46,347
THOSE.

3706
02:59:46,347 --> 02:59:50,918
THOSE ARE THE FORMAL MEETINGS.

3707
02:59:50,918 --> 02:59:54,622
WE CAN DISCUSS THOSE AT A LATER 

3708
02:59:54,622 --> 02:59:57,392
POINT IN TIME.

3709
02:59:57,392 --> 03:00:00,194
AND HO DO YOU SUBMIT AN FDA 

3710
03:00:00,194 --> 03:00:00,795
MEETING REQUEST?

3711
03:00:00,795 --> 03:00:01,863
WE'RE FOT GOING TO GO THROUGH 

3712
03:00:01,863 --> 03:00:06,467
THE SPECIFICS OF THAT BUT IT'S 

3713
03:00:06,467 --> 03:00:09,270
VERY SCRIPTED AND IN THE FORMAL 

3714
03:00:09,270 --> 03:00:10,471
MEETINGS BETWEEN THE FDA 

3715
03:00:10,471 --> 03:00:16,611
SPONSORS AND/OR APPLICANTS.

3716
03:00:16,611 --> 03:00:18,046
THE INFORMATION THAT GOES IN A 

3717
03:00:18,046 --> 03:00:21,549
BRIEFLY BOOK YOU ASKED FOR A 

3718
03:00:21,549 --> 03:00:21,816
TEMPLATE.

3719
03:00:21,816 --> 03:00:23,117
WE CAN PROVIDE YOU WITH ONE IF 

3720
03:00:23,117 --> 03:00:25,086
YOU DON'T HAVE ONE.

3721
03:00:25,086 --> 03:00:28,022
THERE'S NO SPECIFIC FDA GUIDANCE

3722
03:00:28,022 --> 03:00:31,159
ON YOUR BRIEFING BOOK BUT WE CAN

3723
03:00:31,159 --> 03:00:33,394
PROVIDE YOU WITH FEEDBACK.

3724
03:00:33,394 --> 03:00:35,229
FINALIZE QUESTIONS.

3725
03:00:35,229 --> 03:00:36,631
THE BACKGROUND INFORMATION MEDED

3726
03:00:36,631 --> 03:00:39,634
FOR THE FDA TO ANSWER YOUR 

3727
03:00:39,634 --> 03:00:39,901
QUESTIONS.

3728
03:00:39,901 --> 03:00:45,673
YOUR DATA SHOULD BE SUMMARIZED 

3729
03:00:45,673 --> 03:00:46,507
AND SUCCINCT.

3730
03:00:46,507 --> 03:00:47,708
REMEMBER THEY HAVE I ASHORT 

3731
03:00:47,708 --> 03:00:49,077
PERIOD OF TIME TO REVIEW YOUR 

3732
03:00:49,077 --> 03:00:50,244
INFORMATION SO YOU WA IN THE TO 

3733
03:00:50,244 --> 03:00:52,246
MAKE SURE IT'S SUMMARIZED AND 

3734
03:00:52,246 --> 03:00:54,715
VERY SUCCINCT.

3735
03:00:54,715 --> 03:01:00,755
AGAIN, AFTER YOU SUBMIT YOUR 

3736
03:01:00,755 --> 03:01:03,691
BRIEFING BOOK, YOU CANNOT 

3737
03:01:03,691 --> 03:01:06,494
PROVIDE NEW INFORMATION TO THE 

3738
03:01:06,494 --> 03:01:16,938
AGENCY DURING THE MEETING.

3739
03:01:21,809 --> 03:01:26,781
DON'T PANIC IT DOESN'T MEAN THE 

3740
03:01:26,781 --> 03:01:29,150
FDA THINK THERE ARE MAJOR ISSUES

3741
03:01:29,150 --> 03:01:30,785
THEY ARE CAN'T ADDRESS IN THE 

3742
03:01:30,785 --> 03:01:33,387
WRITTEN RESPONSE AND GIVEN THE 

3743
03:01:33,387 --> 03:01:39,026
DETAILED WRIT ENRESPONSE -- 

3744
03:01:39,026 --> 03:01:43,264
WRITTEN RESPONSE AND THE SILVER 

3745
03:01:43,264 --> 03:01:44,866
LINING IS YOU GET IT 30 DAYS 

3746
03:01:44,866 --> 03:01:45,833
EARLIER FAN IF YOU HAD A FACE TO

3747
03:01:45,833 --> 03:01:54,809
FACE MEETING.

3748
03:01:54,809 --> 03:01:58,212
THE INFORMATION YOU PUT IN THE 

3749
03:01:58,212 --> 03:01:59,647
PROTOCOL IS IN THE GUIDANCE 

3750
03:01:59,647 --> 03:02:01,082
DOCUMENT BUT YOU PROVIDE THE 

3751
03:02:01,082 --> 03:02:04,018
TITLE PAGE AND OBJECTIVES AND 

3752
03:02:04,018 --> 03:02:06,254
STUDY DESIGN.

3753
03:02:06,254 --> 03:02:08,523
THE STUDY PROCEDURES AND YOUR 

3754
03:02:08,523 --> 03:02:13,761
SAFETY REPORTING AND MONITORING 

3755
03:02:13,761 --> 03:02:17,131
AND REPORTING YOUR STATISTICAL 

3756
03:02:17,131 --> 03:02:21,035
ANALYSIS PLAN AND ETHICS AND 

3757
03:02:21,035 --> 03:02:21,802
CONSIDERATIONS FOR QUALITY 

3758
03:02:21,802 --> 03:02:23,905
ASSURANCE AND INFORMING ON DATA 

3759
03:02:23,905 --> 03:02:27,975
MANAGEMENT AND MANAGING AND 

3760
03:02:27,975 --> 03:02:30,478
AUDITING AND YOUR INFORMED 

3761
03:02:30,478 --> 03:02:35,283
CONSENT IS OPTIONAL.

3762
03:02:35,283 --> 03:02:36,918
SO THE INFORMATION THAT GOES IN 

3763
03:02:36,918 --> 03:02:40,154
THE TOP SECTION OF YOUR IND IS 

3764
03:02:40,154 --> 03:02:41,689
THE DESCRIPTION OF THE 

3765
03:02:41,689 --> 03:02:42,490
PHARMACOLOGIC EFFECTS AND 

3766
03:02:42,490 --> 03:02:44,659
MECHANISMS OF THE ACTIONS IN 

3767
03:02:44,659 --> 03:02:51,933
YOUR ANIMAL STUDIES AND DATA.

3768
03:02:51,933 --> 03:02:53,267
YOU INCLUDE AN INTEGRATED 

3769
03:02:53,267 --> 03:02:55,536
SUMMARY OF YOUR TOXICOLOGY WHICH

3770
03:02:55,536 --> 03:02:58,439
IS A BRIEF DESCRIPTION OF THE 

3771
03:02:58,439 --> 03:02:59,974
TRIALS AND ANY DEVIATIONS OR 

3772
03:02:59,974 --> 03:03:02,476
ANYTHING LIKE THAT.

3773
03:03:02,476 --> 03:03:06,981
YOU PROVIDE A TOXICOLOGY FULL 

3774
03:03:06,981 --> 03:03:09,383
TABULATION IS FOR EACH 

3775
03:03:09,383 --> 03:03:11,886
TOXICOLOGY STUDY YOU PROVIDE A 

3776
03:03:11,886 --> 03:03:13,521
FULL TABULATION OF THE DATA 

3777
03:03:13,521 --> 03:03:23,631
SUITABLE FOR DETAILED REVIEW.

3778
03:03:23,631 --> 03:03:25,399
INFORMATION ON YOUR DRUG 

3779
03:03:25,399 --> 03:03:26,467
SUBSTANCE.

3780
03:03:26,467 --> 03:03:31,606
IT'S THE CHEMICAL, PHYSICAL, 

3781
03:03:31,606 --> 03:03:33,374
BIOLOGICAL CHARACTERISTICS AND 

3782
03:03:33,374 --> 03:03:37,245
FLOW CHART FOR YOUR 

3783
03:03:37,245 --> 03:03:38,379
MANUFACTURING PROCESS YOUR 

3784
03:03:38,379 --> 03:03:45,386
LIMITS AND ANALYTICAL 

3785
03:03:45,386 --> 03:03:50,925
SPECIFICATIONS YOU WANT TO 

3786
03:03:50,925 --> 03:03:53,694
EXPERIENCE SUPPORT FROM YOUR 

3787
03:03:53,694 --> 03:03:56,497
CDMO EARLY ON AND THE DRUG 

3788
03:03:56,497 --> 03:03:57,798
PRODUCT INCLUDE THE COMPONENT 

3789
03:03:57,798 --> 03:04:02,403
USED IN THE FORMERLATION.

3790
03:04:02,403 --> 03:04:04,338
THE QUANTITATIVE COMPOSITION AND

3791
03:04:04,338 --> 03:04:08,309
NAME AND ADDRESS AND 

3792
03:04:08,309 --> 03:04:10,144
MANUFACTURING PROCESS AND 

3793
03:04:10,144 --> 03:04:13,214
ACCEPTABLE LIMITS WITH 

3794
03:04:13,214 --> 03:04:14,949
SPECIFICATIONS AND ANALYTICAL 

3795
03:04:14,949 --> 03:04:15,316
METHOD.

3796
03:04:15,316 --> 03:04:16,517
INFORMATION ON THE STABILITY OF 

3797
03:04:16,517 --> 03:04:17,652
THE DRUG PRODUCT AND PLACEBO IF 

3798
03:04:17,652 --> 03:04:28,062
YOU'RE USING A PLACEBO.

3799
03:04:35,870 --> 03:04:39,307
>> AFTER YOU SUBMIT THE
IND TOTE

3800
03:04:39,307 --> 03:04:40,574
FDA, FDA HAS 03 DAYS TO 

3801
03:04:40,574 --> 03:04:45,780
ACKNOWLEDGE AND REVIEW RECEIPT 

3802
03:04:45,780 --> 03:04:49,317
AND BEFORE THEY PUT YOU ON A 

3803
03:04:49,317 --> 03:04:50,117
CLINICAL HOLD.

3804
03:04:50,117 --> 03:04:51,752
IF THEY DON'T HEAR FROM THE FDA 

3805
03:04:51,752 --> 03:04:53,154
WITHIN 30 DAYS YOU CAN PROCEED 

3806
03:04:53,154 --> 03:04:54,422
WITH YOUR STUDY, YOUR CLINICAL 

3807
03:04:54,422 --> 03:05:02,096
STUDY.

3808
03:05:02,096 --> 03:05:10,037
IT'S BEST PRACTICE TO SAY IT'S 

3809
03:05:10,037 --> 03:05:16,077
BEEN 30 DAYS AND AFTER THE 30 

3810
03:05:16,077 --> 03:05:26,253
DAYS WITH COMMENTS AND YOU NEED 

3811
03:05:26,253 --> 03:05:28,789
TO ACKNOWLEDGE.

3812
03:05:28,789 --> 03:05:34,328
AND WE HAVE A SHORT PERIOD OF 

3813
03:05:34,328 --> 03:05:43,137
TIME AND SO ACCELERATED APPROVAL

3814
03:05:43,137 --> 03:05:46,040
AND PRIORITY REVIEW AND 

3815
03:05:46,040 --> 03:05:47,308
ACCELERATE APPROVAL IS BASICALLY

3816
03:05:47,308 --> 03:05:51,345
A DRUG THAT PROVIDES THE 

3817
03:05:51,345 --> 03:05:54,482
MEANINGFUL ADVANTAGE OF 

3818
03:05:54,482 --> 03:05:56,317
AVAILABLE THERAPIES AND 

3819
03:05:56,317 --> 03:06:01,455
DEMONSTRATE END POINT AND THAT'S

3820
03:06:01,455 --> 03:06:11,999
REALLY IMPORTANT AND ACCELERATED

3821
03:06:17,872 --> 03:06:22,209
APPROVAL AND 50USH8LY WITH 

3822
03:06:22,209 --> 03:06:24,512
ONCOLOGY AND HIGH RISK, HIGH 

3823
03:06:24,512 --> 03:06:27,081
REWARD AND POST APPROVAL YOU 

3824
03:06:27,081 --> 03:06:30,551
HAVE TO VAL TATE THE SIR GAT END

3825
03:06:30,551 --> 03:06:39,326
POINT USED DURING DEVELOPMENT.

3826
03:06:39,326 --> 03:06:40,661
AND YOU NEED SUCH MONTHS RATHER 

3827
03:06:40,661 --> 03:06:50,471
THAN A SOW MONTH REVIEW.

3828
03:06:50,471 --> 03:06:52,973
>> CARAMELA THANK YOU FOR
THE 

3829
03:06:52,973 --> 03:06:54,175
WONDERFUL OVERVIEW.

3830
03:06:54,175 --> 03:06:55,409
I THOUGHT IT WAS FANTASTIC.

3831
03:06:55,409 --> 03:06:57,478
A REMINDER TO FOLKS, PLEASE 

3832
03:06:57,478 --> 03:07:01,549
UPLOAD QUESTIONS FOR CARAMELA IN

3833
03:07:01,549 --> 03:07:02,683
THE Q&A SECTION AND WE'LL
COME 

3834
03:07:02,683 --> 03:07:03,250
BACK TO THAT.

3835
03:07:03,250 --> 03:07:08,856
WITH THAT I'LL TURN IT OVER TO 

3836
03:07:08,856 --> 03:07:09,757
DR. KEVIN WHITTLESEY.

3837
03:07:09,757 --> 03:07:12,526
>> THANK YOU FOR BEING
HERE AND 

3838
03:07:12,526 --> 03:07:23,070
THANKS FOR THE ORG -- ORGANIZER 

3839
03:07:25,639 --> 03:07:29,977
AROUND CONSULTANT AND WORKED IN 

3840
03:07:29,977 --> 03:07:31,312
PRODUCT DEVELOPMENT.

3841
03:07:31,312 --> 03:07:37,585
I WENT WANT TO TALK ABOUT 

3842
03:07:37,585 --> 03:07:38,486
AEROSOL DEVELOPMENT AND IT'S 

3843
03:07:38,486 --> 03:07:39,787
IMPORTANT CONSIDERATIONS AS A 

3844
03:07:39,787 --> 03:07:40,354
PRODUCT FOR DELIVERY TO THE 

3845
03:07:40,354 --> 03:07:49,363
AIRWAY.

3846
03:07:49,363 --> 03:07:52,366
AND IN SETTING YOURSELF UP AND 

3847
03:07:52,366 --> 03:07:53,968
SHE GAVE A NICE OVERVIEW OF THE 

3848
03:07:53,968 --> 03:07:57,404
AND TALK ABOUT THE DELIVERY 

3849
03:07:57,404 --> 03:08:04,512
DEVICE CONSIDERATIONS AND HOW IT

3850
03:08:04,512 --> 03:08:06,180
INTEGRATES WITH THE EXPOSURE AND

3851
03:08:06,180 --> 03:08:10,317
MY PRIMARY SPACE IS IN GENE 

3852
03:08:10,317 --> 03:08:10,651
THERAPIES.

3853
03:08:10,651 --> 03:08:11,519
THERE'S KEY DIFFERENCES I'LL 

3854
03:08:11,519 --> 03:08:21,896
WORK ON HIGHLIGHTING.

3855
03:08:26,600 --> 03:08:32,473
SO THINKING ABOUT SOME OF THE 

3856
03:08:32,473 --> 03:08:33,707
CONSIDERATIONS FOR AEROSOL 

3857
03:08:33,707 --> 03:08:37,111
DEVELOPMENT IN TERMS OF THE 

3858
03:08:37,111 --> 03:08:38,012
THERAPEUTIC MODALITIES SAY SMALL

3859
03:08:38,012 --> 03:08:39,813
MOLECULE DRUG AND GENE THERAPY 

3860
03:08:39,813 --> 03:08:47,888
PRODUCTS OR OTHER BIOLOGICALS.

3861
03:08:47,888 --> 03:08:58,365
AND BY CONTRACT IN A GENES 

3862
03:09:01,302 --> 03:09:02,603
THERAPY THE DEVELOPMENT 

3863
03:09:02,603 --> 03:09:05,272
NECESSARY IN ENSURING AN 

3864
03:09:05,272 --> 03:09:07,441
ACCURATE AND EFFECTIVE DOSE THE 

3865
03:09:07,441 --> 03:09:12,980
ONUS IS HIGHER FOR GENE THERAPY 

3866
03:09:12,980 --> 03:09:20,220
PRODUCT.

3867
03:09:20,220 --> 03:09:25,192
AND WE CONTRAST AND OTHER 

3868
03:09:25,192 --> 03:09:26,994
BIOLOGICS CAN BE ADVERSELY 

3869
03:09:26,994 --> 03:09:31,365
AFFECTED BY THE.

3870
03:09:31,365 --> 03:09:38,472
NEBULIZATION PROCESS AND MUST BE

3871
03:09:38,472 --> 03:09:38,706
TESTED.

3872
03:09:38,706 --> 03:09:44,545
AND REPEAT DOSE ON A WEEKLY OR 

3873
03:09:44,545 --> 03:09:52,486
SOMETIMES DAILY BASIS AND FOR A 

3874
03:09:52,486 --> 03:09:54,088
SINGLE ADMINISTRATION THE 

3875
03:09:54,088 --> 03:09:56,056
PATIENTS ARE ABLE TO RECEIVING 

3876
03:09:56,056 --> 03:09:57,391
ARE CRITICALLY IMPORTANT AND 

3877
03:09:57,391 --> 03:09:58,459
ANYTHING THE FDA PUTS A LOT OF 

3878
03:09:58,459 --> 03:10:00,294
EMPHASIS ON.

3879
03:10:00,294 --> 03:10:02,463
AND FINALLY COST IN THE SMALL 

3880
03:10:02,463 --> 03:10:05,566
MOLECULES CANNOT BE MASS 

3881
03:10:05,566 --> 03:10:07,001
PRODUCED IN LARGE QUANTITIES 

3882
03:10:07,001 --> 03:10:08,202
EFFECTIVELY AND OTHER BIOLOGICS 

3883
03:10:08,202 --> 03:10:12,106
CAN BE DIFFICULT AND EXPENSIVE 

3884
03:10:12,106 --> 03:10:14,141
TO PRODUCE.

3885
03:10:14,141 --> 03:10:18,779
THE PRODUCT LOST DURING THE 

3886
03:10:18,779 --> 03:10:19,546
AEROSOLIZATION PROCESS 

3887
03:10:19,546 --> 03:10:21,415
ULTIMATELY BECOMES THE COST OF 

3888
03:10:21,415 --> 03:10:22,683
THE PRODUCT IF YOU'RE MARKETING 

3889
03:10:22,683 --> 03:10:32,893
THE PRODUCT.

3890
03:10:37,531 --> 03:10:39,400
IN THINKING OF SELECTION AND 

3891
03:10:39,400 --> 03:10:41,135
IMPORTANT TO THINK THROUGH THE 

3892
03:10:41,135 --> 03:10:45,873
RIGHT QUESTIONS IN TERMS OF 

3893
03:10:45,873 --> 03:10:50,477
SELECTING THE DEVICE TO MOVE 

3894
03:10:50,477 --> 03:10:51,679
FORWARD WITH.

3895
03:10:51,679 --> 03:10:54,748
FOR RESPIRATORY DISEASES ARE YOU

3896
03:10:54,748 --> 03:10:56,684
LOOKING TO TARGET MORE OF THE 

3897
03:10:56,684 --> 03:10:58,819
UPPER AIRWAYS VERSUS THE LOWER 

3898
03:10:58,819 --> 03:11:00,220
MORE DISTAL AIRWAYS?

3899
03:11:00,220 --> 03:11:03,190
AND SO WHAT IS YOUR TARGET SITE 

3900
03:11:03,190 --> 03:11:05,125
FOR DEPOSITION NECESSARY FOR 

3901
03:11:05,125 --> 03:11:06,894
YOUR ACTUAL THERAPEUTIC PRODUCT.

3902
03:11:06,894 --> 03:11:10,130
AND THEN WHAT TECHNOLOGY DO YOU 

3903
03:11:10,130 --> 03:11:10,464
SELECT?

3904
03:11:10,464 --> 03:11:14,134
THE THREE CORE TECHNOLOGIES 

3905
03:11:14,134 --> 03:11:19,740
INCORPORATED INTO JET NEBULIZER 

3906
03:11:19,740 --> 03:11:20,974
AND VIBRATING MESH AND IT'S 

3907
03:11:20,974 --> 03:11:22,242
IMPORTANT TO THINK ABOUT WHAT 

3908
03:11:22,242 --> 03:11:25,813
TECHNOLOGY YOU'RE USING AND THE 

3909
03:11:25,813 --> 03:11:26,580
SUITABILITY FOR YOUR PARTICULAR 

3910
03:11:26,580 --> 03:11:27,981
DRUG PRODUCT.

3911
03:11:27,981 --> 03:11:31,285
FOR A FEW EXAMPLES JET 

3912
03:11:31,285 --> 03:11:34,154
NEBULIZERS SUBJECT THE DRUG TO 

3913
03:11:34,154 --> 03:11:35,456
NEB YOU'LL I'DATION FOR TIMES 

3914
03:11:35,456 --> 03:11:42,463
OVER AND OVER WHICH CAN BE 

3915
03:11:42,463 --> 03:11:45,399
DESTABILIZING AND INCREASE THE 

3916
03:11:45,399 --> 03:11:46,500
RESERVOIR AFFECTING THE PRODUCT 

3917
03:11:46,500 --> 03:11:48,669
OR IF THE PATIENT'S IMMUNE 

3918
03:11:48,669 --> 03:11:51,205
RESPONSE AS THE CONCENTRATION OF

3919
03:11:51,205 --> 03:11:54,041
THE BUFFER SOLUTION INCREASES 

3920
03:11:54,041 --> 03:11:56,076
AND LASTLY SHEER FORCES DIFFER 

3921
03:11:56,076 --> 03:12:01,582
BETWEEN NEBULIZER AND MAY NEED 

3922
03:12:01,582 --> 03:12:03,383
TO BE TESTED TO WHAT 

3923
03:12:03,383 --> 03:12:06,353
TECHNOLOGIES ARE COMPATIBLE TO 

3924
03:12:06,353 --> 03:12:08,288
THE THERAPEUTIC PRODUCT AND NEB 

3925
03:12:08,288 --> 03:12:14,461
YOU'LLNEBULIZERS HAVE DIFFERENT 

3926
03:12:14,461 --> 03:12:15,562
CAPACITIES DEPENDING ON HOW CON 

3927
03:12:15,562 --> 03:12:17,030
OF CONCENTRATED THE SOLUTION AND

3928
03:12:17,030 --> 03:12:24,972
DEPENDS ON THE VOLUME 

3929
03:12:24,972 --> 03:12:26,507
ADMINISTERED WHERE THERE'S 

3930
03:12:26,507 --> 03:12:27,441
PATIENT COMPLIANCES THAT MAY 

3931
03:12:27,441 --> 03:12:34,014
NEED TO BE INCLUDED.

3932
03:12:34,014 --> 03:12:36,617
TAKING THIS INTO THE 

3933
03:12:36,617 --> 03:12:38,619
TECHNOLOGIES SHOWN AND ULTRA 

3934
03:12:38,619 --> 03:12:42,022
SONIC VIBRATING MESH WHICH I'VE 

3935
03:12:42,022 --> 03:12:43,190
REFERENCED AND THEY ARE HAVE 

3936
03:12:43,190 --> 03:12:44,725
SLIGHTLY DIFFERENT REQUIREMENTS 

3937
03:12:44,725 --> 03:12:46,527
IN TERMS OF THE CLINICAL SITE 

3938
03:12:46,527 --> 03:12:48,562
ALL THREE REQUIRE ELECTRICITY 

3939
03:12:48,562 --> 03:12:53,033
BUT A JET NEB YOU'LL I'DER 

3940
03:12:53,033 --> 03:12:57,437
REQUIRE THE EXPRESSED SOURCE AND

3941
03:12:57,437 --> 03:12:58,539
SEEMS STRAIGHTFORWARD BUT 

3942
03:12:58,539 --> 03:13:00,174
IMPORTANT TO UNDERSTAND MAYBE A 

3943
03:13:00,174 --> 03:13:01,341
RARE DISEASE IN LESS DEVELOPED 

3944
03:13:01,341 --> 03:13:01,875
AREAS WHERE THERE'S 

3945
03:13:01,875 --> 03:13:02,509
INFRASTRUCTURE CHALLENGES AN 

3946
03:13:02,509 --> 03:13:04,511
ENSURE YOU HAVE ACCESS TO THE 

3947
03:13:04,511 --> 03:13:05,112
RESOURCES NECESSARY AT THE 

3948
03:13:05,112 --> 03:13:14,988
INTENDED CLINICAL SITE.

3949
03:13:14,988 --> 03:13:25,265
AND THERE'S HETEROGENEITY AND 

3950
03:13:25,265 --> 03:13:27,134
INDICATORS OF THE BOTTOM 

3951
03:13:27,134 --> 03:13:28,635
RESERVOIR IS WHERE THE EXPRESSED

3952
03:13:28,635 --> 03:13:32,339
AIR SOURCE COMES IN AND THAT 

3953
03:13:32,339 --> 03:13:34,708
THEN GENERATES THE VENTURI 

3954
03:13:34,708 --> 03:13:35,709
EFFECT WHICH DRAWS FLUID FROM 

3955
03:13:35,709 --> 03:13:39,446
THE SURFACE INTO THE AIR STREAM 

3956
03:13:39,446 --> 03:13:43,383
THEN AEROSOLIZED.

3957
03:13:43,383 --> 03:13:46,286
THE BAFFLE IS WHERE LARGER 

3958
03:13:46,286 --> 03:13:52,426
PARTICLES ARE DEFLECTED TO BE 

3959
03:13:52,426 --> 03:13:54,494
RE-NEBULIZED IN THE EFFECT FOR 

3960
03:13:54,494 --> 03:13:55,262
RE.

3961
03:13:55,262 --> 03:13:56,897
YOU'LLIZATION MANY TIMES IN THE 

3962
03:13:56,897 --> 03:13:59,166
JET NEBULIZER.

3963
03:13:59,166 --> 03:14:02,035
THEY HAVE THE BROADEST RANGE OF 

3964
03:14:02,035 --> 03:14:03,303
PARTICLE SIDES WITHIN THE 

3965
03:14:03,303 --> 03:14:03,837
AEROSOL GENERATED.

3966
03:14:03,837 --> 03:14:06,440
THAT CAN BE TAILORED BASED ON A 

3967
03:14:06,440 --> 03:14:09,877
NUMBER OF PROPERTIES BUT HAVE A 

3968
03:14:09,877 --> 03:14:11,812
MORE DIVERSE RANGE BY CONTRAST 

3969
03:14:11,812 --> 03:14:14,481
AND ULTRA SONIC HAVE MORE 

3970
03:14:14,481 --> 03:14:15,382
CONSISTENT SIZED PARTICLES THAT 

3971
03:14:15,382 --> 03:14:18,018
SIT AT THE BOTTOM OF THE 

3972
03:14:18,018 --> 03:14:22,522
RESERVOIR AND GENERATES 

3973
03:14:22,522 --> 03:14:24,958
ULTRASOUND AND AEROSOL ON THAT 

3974
03:14:24,958 --> 03:14:29,196
AND THE VIBRATING MESH HAS A 

3975
03:14:29,196 --> 03:14:30,998
PHYSICAL SCREEN WITH HOLES 

3976
03:14:30,998 --> 03:14:33,267
PUNCHED IN IT AND THEY DEFINE 

3977
03:14:33,267 --> 03:14:35,936
THE PARTICLE SIZE AND 

3978
03:14:35,936 --> 03:14:39,439
SUBSEQUENTLY HAS THE SIZE 

3979
03:14:39,439 --> 03:14:49,917
DISTRIBUTION OF THE SIZES.

3980
03:14:49,917 --> 03:14:51,785
AND IN TERMS OF THE ADDITIONAL 

3981
03:14:51,785 --> 03:14:55,689
SHEER FORCES AND ULTRA SONIC 

3982
03:14:55,689 --> 03:14:57,958
GENERATES HEAT IN THE REDS 

3983
03:14:57,958 --> 03:15:02,162
VOYEUR AND NOT FOR THERMAL 

3984
03:15:02,162 --> 03:15:05,399
PRODUCTS BECAUSE OF THE 

3985
03:15:05,399 --> 03:15:08,735
SOLUTION.

3986
03:15:08,735 --> 03:15:10,504
VIBRATING MESH AND THE RESERVOIR

3987
03:15:10,504 --> 03:15:11,838
SITS OVER THE TOP AND THE 

3988
03:15:11,838 --> 03:15:13,874
SOLUTION IS THEREFORE ONLY 

3989
03:15:13,874 --> 03:15:16,510
NEBULIZED ONCE PASSING THROUGH 

3990
03:15:16,510 --> 03:15:18,045
THE SCREEN WHICH MAY BE 

3991
03:15:18,045 --> 03:15:19,012
ADVANTAGEOUS TO SOME PRODUCTS.

3992
03:15:19,012 --> 03:15:22,516
IN TERMS OF WHAT'S GOING ON IN 

3993
03:15:22,516 --> 03:15:25,252
THE RESERVOIR, JET AND ULTRA 

3994
03:15:25,252 --> 03:15:32,059
SONIC NEBULIZERS ARE SUBJECT TO 

3995
03:15:32,059 --> 03:15:33,260
EVAPORATIVE LOSS IN THE SOLUTION

3996
03:15:33,260 --> 03:15:42,502
ACROSS THE VOLUME OF THE BOTTOM 

3997
03:15:42,502 --> 03:15:49,609
AND THERE'S HIGH OSMOLALITY 

3998
03:15:49,609 --> 03:15:50,644
CHANGE AND SEE DIFFERENCES IN 

3999
03:15:50,644 --> 03:16:01,121
TERMS OF THE PRODUCT THAT'S 

4000
03:16:04,992 --> 03:16:06,126
EXPENSIVE TO MANUFACTURING AND 

4001
03:16:06,126 --> 03:16:09,196
JET AND ULTRA SONIC EXPERIENCE A

4002
03:16:09,196 --> 03:16:12,733
LOSS WHICH IS IN TERMS OF COST 

4003
03:16:12,733 --> 03:16:18,338
OF GOODS AND WHAT THAT DOES IN 

4004
03:16:18,338 --> 03:16:28,715
TERMS OF THE PROGRAM.

4005
03:16:40,627 --> 03:16:43,430
AND THERE'S IMPORTANT OSMALALITY

4006
03:16:43,430 --> 03:16:49,970
AND AFFECT THE AEROSOL.

4007
03:16:49,970 --> 03:16:57,110
IN THE CASE OF JET THE PARTICLE 

4008
03:16:57,110 --> 03:17:02,949
SIZE AND DISTRIBUTION TO TARGET 

4009
03:17:02,949 --> 03:17:10,791
THE AEROSOL YOU NEED TO CONSIDER

4010
03:17:10,791 --> 03:17:20,767
THESE OF THE THERAPEUTICS.

4011
03:17:20,767 --> 03:17:23,403
SO NOW IN THINKING IN TERMS OF 

4012
03:17:23,403 --> 03:17:25,639
HOW YOU ARE GOING TO DELIVER 

4013
03:17:25,639 --> 03:17:33,213
YOUR AEROSOL NON CLINICALLY.

4014
03:17:33,213 --> 03:17:34,514
THERE'S EXPOSURE SYSTEMS FOR 

4015
03:17:34,514 --> 03:17:36,016
DIFFERENT ANIMALS AND ANIMAL 

4016
03:17:36,016 --> 03:17:38,185
MELDS AND IN THE LEFT AND MIDDLE

4017
03:17:38,185 --> 03:17:40,787
ARE DELIVERY SYSTEMS FOR SMALL 

4018
03:17:40,787 --> 03:17:46,326
ANIMAL FOR RODENTS.

4019
03:17:46,326 --> 03:17:55,569
WHAT YOU CAN APPRECIATE AND 

4020
03:17:55,569 --> 03:17:56,703
INHALED THROUGH THEIR NESS AND 

4021
03:17:56,703 --> 03:17:58,171
MOUTH.

4022
03:17:58,171 --> 03:18:00,040
THAT'S QUITE DIFFERENT FROM THE 

4023
03:18:00,040 --> 03:18:03,176
WAY A PATIENT WILL EXPERIENCE 

4024
03:18:03,176 --> 03:18:05,812
AEROSOL WHEREBY THEY'RE 

4025
03:18:05,812 --> 03:18:06,847
TYPICALLY BREATHING OFF THE 

4026
03:18:06,847 --> 03:18:09,082
MOUTH PIECE OF A NEBULIZER SO IN

4027
03:18:09,082 --> 03:18:10,717
THE CASE OF A SMALL ANIMAL 

4028
03:18:10,717 --> 03:18:15,822
YOU'RE PRESENTING LESS MATERIAL 

4029
03:18:15,822 --> 03:18:21,461
GOING TO INTO  WHICH NEEDS TO BE

4030
03:18:21,461 --> 03:18:31,671
CONSIDERED.

4031
03:18:37,277 --> 03:18:39,446
AND THE NHP BREATHING THROUGH 

4032
03:18:39,446 --> 03:18:41,148
BOTH THE NOSE AND THE MOUTH 

4033
03:18:41,148 --> 03:18:42,916
LEADING TO POTENTIALLY MORE 

4034
03:18:42,916 --> 03:18:46,853
PRODUCT BEING EXPOSED TO THE 

4035
03:18:46,853 --> 03:18:47,921
UPPER FROM THE LOWER AIRWAYS 

4036
03:18:47,921 --> 03:18:49,689
FROM THE CLINICAL SETTING.

4037
03:18:49,689 --> 03:18:50,490
IT NEEDS TO BE CONSIDERED IN 

4038
03:18:50,490 --> 03:18:56,363
TERMS OF WHAT IMPACTS THE 

4039
03:18:56,363 --> 03:19:00,000
DIFFERENCES MAY HAVE IN TERMS OF

4040
03:19:00,000 --> 03:19:02,502
THE SAFETY AND EFFICACY 

4041
03:19:02,502 --> 03:19:12,712
ASSESSMENT.

4042
03:19:15,382 --> 03:19:20,554
AND THEY CAN BE USEFUL IN 

4043
03:19:20,554 --> 03:19:27,460
DEMONSTRATE WITH EFFICACY AND 

4044
03:19:27,460 --> 03:19:32,399
DELIVERY REPRESENTATIVE OF THE 

4045
03:19:32,399 --> 03:19:33,300
CLINICAL SETTING IS DIFFICULT 

4046
03:19:33,300 --> 03:19:37,671
AND DEMONSTRATING THEY'RE 

4047
03:19:37,671 --> 03:19:39,439
INTENDED CLINICAL USE OF THE 

4048
03:19:39,439 --> 03:19:40,073
PRODUCT.

4049
03:19:40,073 --> 03:19:42,242
WITH SMALL ANIMALS INHALATION 

4050
03:19:42,242 --> 03:19:43,577
RESULTS IN THE LARGE FRACTION OF

4051
03:19:43,577 --> 03:19:45,445
THE DOSE IN THE UPPER AIRWAYS 

4052
03:19:45,445 --> 03:19:48,448
WHICH MAY BE IMPACTFUL IN 

4053
03:19:48,448 --> 03:19:51,251
ASSESSMENT OF SAFETY AND 

4054
03:19:51,251 --> 03:19:51,885
EFFICACY IN THE MODEL.

4055
03:19:51,885 --> 03:19:53,987
AND A COMMON ROUTE OF 

4056
03:19:53,987 --> 03:19:57,557
ADMINISTRATION IN MICE AND RATS 

4057
03:19:57,557 --> 03:20:02,495
IS WHEREBY LIQUID IS DIRECTLY IN

4058
03:20:02,495 --> 03:20:04,164
THE TRACHEA AND GETS IN THE 

4059
03:20:04,164 --> 03:20:08,535
LOWER AIRWAYS OPPOSED TO THE 

4060
03:20:08,535 --> 03:20:10,303
NASAL INHALATION BUT CAN STILL 

4061
03:20:10,303 --> 03:20:11,938
BE USEFUL IN DEMONSTRATION OF 

4062
03:20:11,938 --> 03:20:13,373
EFFICACY OF THE PRODUCT.

4063
03:20:13,373 --> 03:20:15,475
IT'S AN DIFFERENCE BETWEEN THE 

4064
03:20:15,475 --> 03:20:16,710
CLINICAL PRESENTATION THAT NEED 

4065
03:20:16,710 --> 03:20:17,410
TO BE CONSIDERED AND 

4066
03:20:17,410 --> 03:20:18,511
APPROPRIATELY JUSTIFIED TO THE 

4067
03:20:18,511 --> 03:20:20,313
FDA.

4068
03:20:20,313 --> 03:20:24,818
LARGE ANIMALS ESPECIALLY LARGE 

4069
03:20:24,818 --> 03:20:26,253
HUMAN PRIMATES DO NEED THE 

4070
03:20:26,253 --> 03:20:28,054
CLINICAL DEVICE BUT THEY ARE 

4071
03:20:28,054 --> 03:20:29,789
CANNOT BE TRAINED TO BREATHE 

4072
03:20:29,789 --> 03:20:31,191
THROUGH A CLINICAL MOUTH PIECE 

4073
03:20:31,191 --> 03:20:34,394
YOU HAVE TO USE THE PEDIATRIC 

4074
03:20:34,394 --> 03:20:35,996
MASK WHICH ALSO RESULTS IN 

4075
03:20:35,996 --> 03:20:37,631
SIGNIFICANT LOSS OF THE 

4076
03:20:37,631 --> 03:20:39,065
DELIVERED DOSE AND DIFFERENT 

4077
03:20:39,065 --> 03:20:40,100
PRESENTATION THAT WOULD BE USED 

4078
03:20:40,100 --> 03:20:41,534
CLINICALLY AND ALTERNATIVE IS 

4079
03:20:41,534 --> 03:20:44,738
THE ANIMALS CAN BE -- YOU CAN 

4080
03:20:44,738 --> 03:20:52,345
DELIVER TO THE ANIMALS USING AN 

4081
03:20:52,345 --> 03:20:56,049
ENDOTRACHEAL TUBE AND USE THE 

4082
03:20:56,049 --> 03:20:57,684
DEVICE DELIVERED TO THE LOWER 

4083
03:20:57,684 --> 03:21:01,421
AIRWAYS AND THESE ARE 

4084
03:21:01,421 --> 03:21:03,490
ANESTHETIZED ANIMALS AN IT MAY 

4085
03:21:03,490 --> 03:21:04,958
LEAD TO A DIFFERENT 

4086
03:21:04,958 --> 03:21:06,293
UNDERSTANDING OF THE SAFETY AND 

4087
03:21:06,293 --> 03:21:08,561
EFFICACY OF THE PRODUCT AND 

4088
03:21:08,561 --> 03:21:09,229
DELIVERED DOSE.

4089
03:21:09,229 --> 03:21:10,697
THESE ARE REALLY IMPORTANT 

4090
03:21:10,697 --> 03:21:14,868
QUESTIONS TO PUT FORWARD TO THE 

4091
03:21:14,868 --> 03:21:15,935
FDA.

4092
03:21:15,935 --> 03:21:17,671
CARAMELA TOUCHED ON THIS IN 

4093
03:21:17,671 --> 03:21:22,742
TERMS OF A PRE-IND QUESTION 

4094
03:21:22,742 --> 03:21:27,480
SEEKING THE APPROPRIATENESS OF 

4095
03:21:27,480 --> 03:21:29,349
AN ANIMAL MODEL AND SAY HERE'S 

4096
03:21:29,349 --> 03:21:30,483
WHAT WE'RE THINKING.

4097
03:21:30,483 --> 03:21:31,818
WE UNDERSTAND THE DIFFERENCES 

4098
03:21:31,818 --> 03:21:33,553
FROM OUR INTENDED CLINICAL USE 

4099
03:21:33,553 --> 03:21:36,723
AND WHY WE THINK IT'S AN 

4100
03:21:36,723 --> 03:21:38,325
APPROPRIATE MODEL AND DOES THE 

4101
03:21:38,325 --> 03:21:40,694
FDA AGREE.

4102
03:21:40,694 --> 03:21:42,329
IT'S COMMON AND CAN BE A USEFUL 

4103
03:21:42,329 --> 03:21:43,196
THING TO DO BEFORE YOU GO DOWN 

4104
03:21:43,196 --> 03:21:51,771
THE WRONG PATH.

4105
03:21:51,771 --> 03:21:53,606
YOU WANTED TO TALK ABOUT 

4106
03:21:53,606 --> 03:21:54,641
DELIVERY DEVICE TESTING.

4107
03:21:54,641 --> 03:21:57,243
IT'S IMPORTANT YOU CHARACTERIZE 

4108
03:21:57,243 --> 03:21:58,244
YOUR PRODUCT AND ONE OF THE 

4109
03:21:58,244 --> 03:22:00,947
POINTS I MENTIONED EARLIER ON 

4110
03:22:00,947 --> 03:22:02,382
WAS ESPECIALLY WITH THE GENE 

4111
03:22:02,382 --> 03:22:04,851
THERAPY PRODUCT AND LOOK AT THE 

4112
03:22:04,851 --> 03:22:05,919
INTENDED DELIVERED DOSE IS.

4113
03:22:05,919 --> 03:22:09,122
THIS IS USUALLY DONE AS 

4114
03:22:09,122 --> 03:22:10,490
EMPIRICAL LABORATORY BENCH 

4115
03:22:10,490 --> 03:22:12,225
TESTING WITH YOUR AEROSOL IN 

4116
03:22:12,225 --> 03:22:14,294
SOME CASES COUPLED TO IN VIVO 

4117
03:22:14,294 --> 03:22:15,495
STUDIES AS WELL BUT SOME 

4118
03:22:15,495 --> 03:22:16,696
CONSIDERATIONS INCORPORATED 

4119
03:22:16,696 --> 03:22:17,964
FIRSTLY WHAT ARE THE PROPERTIES 

4120
03:22:17,964 --> 03:22:23,970
OF THE AEROSOL AND KEY 

4121
03:22:23,970 --> 03:22:27,507
PARAMETERS ASSESSED AND THE 

4122
03:22:27,507 --> 03:22:28,575
DYNAMIC DIAMETER AND THE 

4123
03:22:28,575 --> 03:22:31,111
STANDARD DEVIATION AND FRACTION.

4124
03:22:31,111 --> 03:22:33,346
THIS IS CHARACTERIZING THE 

4125
03:22:33,346 --> 03:22:39,919
DISTRIBUTION OF SIZE PARTICLES 

4126
03:22:39,919 --> 03:22:44,591
AND IF THEY NEED TONE LESS THAN 

4127
03:22:44,591 --> 03:22:46,526
FIVE MICRONS TO BE INHALED IN 

4128
03:22:46,526 --> 03:22:49,095
THE LUNGS AND WHAT FRACTION OF 

4129
03:22:49,095 --> 03:22:54,267
YOUR PRODUCT IS WITHIN THE 

4130
03:22:54,267 --> 03:22:56,736
RANGE.

4131
03:22:56,736 --> 03:22:58,772
ARE THERE CONCENTRATIONS 

4132
03:22:58,772 --> 03:23:01,841
INSTABILITY OR BIOLOGICAL 

4133
03:23:01,841 --> 03:23:03,910
STABILITY OF YOUR PRODUCT IN 

4134
03:23:03,910 --> 03:23:05,111
TERMS OF STRAIGHT OUT OF THE 

4135
03:23:05,111 --> 03:23:06,646
VIAL FROM THE MANUFACTURER 

4136
03:23:06,646 --> 03:23:10,417
VERSUS GOING TO THROUGH THE 

4137
03:23:10,417 --> 03:23:13,553
NEBULIZATION PROCESS AND 

4138
03:23:13,553 --> 03:23:14,487
ASSESSED AND THAT'S AN IMPORTANT

4139
03:23:14,487 --> 03:23:15,588
ASPECT TO UNDERSTAND.

4140
03:23:15,588 --> 03:23:16,890
AND THE DELIVERED DOSE PRESENTED

4141
03:23:16,890 --> 03:23:22,495
TO THE PATIENT AIRWAY?

4142
03:23:22,495 --> 03:23:32,572
THE.

4143
03:23:46,052 --> 03:23:48,855
AND WHAT YOU'LL SEE IN THE CUP 

4144
03:23:48,855 --> 03:23:50,523
OF THE NEBULIZER AND TYPICALLY 

4145
03:23:50,523 --> 03:23:53,993
THE FDA ASKS TO COLLECT BENCH 

4146
03:23:53,993 --> 03:23:56,029
TESTING DATA AND UNDER 

4147
03:23:56,029 --> 03:23:56,629
WORST-CASE SCENARIO EXPECTED 

4148
03:23:56,629 --> 03:23:58,064
DURING THE TRIAL AND IF 

4149
03:23:58,064 --> 03:23:59,566
SOMETHING GOES WRONG DURING THE 

4150
03:23:59,566 --> 03:23:59,933
ADMINISTRATION.

4151
03:23:59,933 --> 03:24:04,471
A FEW EXAMPLES MIGHT BE A THEY 

4152
03:24:04,471 --> 03:24:06,105
ARE.

4153
03:24:06,105 --> 03:24:07,340
 -- A DELAY AND THERE'S SOME 

4154
03:24:07,340 --> 03:24:12,412
THEY ARE IN TERMS OF LOGISTICS 

4155
03:24:12,412 --> 03:24:16,049
BEFORE THE PATIENT CAN START 

4156
03:24:16,049 --> 03:24:20,119
TAKING THE NEBULIZED DOSE AND 

4157
03:24:20,119 --> 03:24:22,055
HOW LONG CAN IT TAKE APPROPRIATE

4158
03:24:22,055 --> 03:24:23,923
CONCENTRATION AND ENSURE THERE'S

4159
03:24:23,923 --> 03:24:27,060
NO COMPATIBILITY ISSUES?

4160
03:24:27,060 --> 03:24:29,996
ADDITIONALLY, MAYBE THE PATIENT 

4161
03:24:29,996 --> 03:24:32,832
IF IT'S RESPIRATORY DISEASE 

4162
03:24:32,832 --> 03:24:35,835
PERHAPS THEY'LL EXPERIENCE A 

4163
03:24:35,835 --> 03:24:36,469
PULMONARY EXACERBATION AND THE 

4164
03:24:36,469 --> 03:24:39,038
CLINICIAN NEEDS TO INTERRUPT THE

4165
03:24:39,038 --> 03:24:46,246
NEB YOU'LL I'D -- NEBULIZER AND 

4166
03:24:46,246 --> 03:24:56,789
RESUME DOES IT BEHAVE THE SAME?

4167
03:25:04,497 --> 03:25:09,435
YOU NEED TO UNDERSTAND BASED ON 

4168
03:25:09,435 --> 03:25:11,170
THE PATIENT POPULATION WHAT ARE 

4169
03:25:11,170 --> 03:25:13,039
THIS THINGS THAT CAN GO WRONG 

4170
03:25:13,039 --> 03:25:16,910
AND DO WE NEED TO MODEL 

4171
03:25:16,910 --> 03:25:17,610
NON-CLINICALLY AND ASSESS OUR 

4172
03:25:17,610 --> 03:25:19,512
PRODUCT WILL PERFORM AS 

4173
03:25:19,512 --> 03:25:21,347
EXPECTED?

4174
03:25:21,347 --> 03:25:23,650
SO THIS TOPIC OF TESTING IS 

4175
03:25:23,650 --> 03:25:25,618
SOMETHING THAT IS APPROPRIATE TO

4176
03:25:25,618 --> 03:25:31,057
TALK ABOUT WITH THE FDA AS PART 

4177
03:25:31,057 --> 03:25:33,526
OF THE PRE-IND MEETING AND LAID 

4178
03:25:33,526 --> 03:25:37,363
OUT A CLEAR PLAN TO HEAR ALL THE

4179
03:25:37,363 --> 03:25:38,064
THINGS WE'RE PLANNING TO DO AND 

4180
03:25:38,064 --> 03:25:39,832
HOW WE'RE PLANNING TO ASSESS 

4181
03:25:39,832 --> 03:25:43,703
THEM AND DOES THE FDA AGREE IT 

4182
03:25:43,703 --> 03:25:45,204
CHARACTERIZES THE AEROSOL FOR 

4183
03:25:45,204 --> 03:25:54,814
OUR INTENDED TARGET POPULATION.

4184
03:25:54,814 --> 03:25:57,483
LAST THING I WANT TO TOUCH ON 

4185
03:25:57,483 --> 03:25:59,052
ARE TO SHOW THE MEANS BY WHICH 

4186
03:25:59,052 --> 03:26:01,120
THE ASSESSMENTS ARE CONDUCTED?

4187
03:26:01,120 --> 03:26:04,090
SO SHOWN ON THE LEFT IS A 

4188
03:26:04,090 --> 03:26:07,860
CASCADE IMPACTER AND USED TO 

4189
03:26:07,860 --> 03:26:08,861
CHARACTERIZE AEROSOLS.

4190
03:26:08,861 --> 03:26:13,700
YOU SEE ON THE OTHER SIDE OF THE

4191
03:26:13,700 --> 03:26:17,670
LID A SERIES OF SILVER METAL 

4192
03:26:17,670 --> 03:26:20,707
SCREENS WITH INCREASINGLY FINE 

4193
03:26:20,707 --> 03:26:24,844
MESH FROM LEFT TO RIGHT COARSEST

4194
03:26:24,844 --> 03:26:27,413
TO THE FINEST AND AEROSOLS FLOAT

4195
03:26:27,413 --> 03:26:30,316
IN ON THE LEFT AND FROM THE LEFT

4196
03:26:30,316 --> 03:26:32,151
SIDE AND AS THE AEROSOL GOES 

4197
03:26:32,151 --> 03:26:34,487
THROUGH THE CLAIM BERS THE 

4198
03:26:34,487 --> 03:26:36,089
PARTICLES OF THAT SIZE ARE 

4199
03:26:36,089 --> 03:26:39,492
RETAINED AND CAN BE CAPTURED ON 

4200
03:26:39,492 --> 03:26:43,830
FILTERS FOR SUBSEQUENT ANALYSIS.

4201
03:26:43,830 --> 03:26:46,733
IT'S AN ACTUAL PHYSICAL 

4202
03:26:46,733 --> 03:26:47,800
COLLECTION AND DETERMINE HOW 

4203
03:26:47,800 --> 03:26:49,469
MUCH OF YOUR PRODUCT ARE IN THE 

4204
03:26:49,469 --> 03:26:51,504
SIZE RANGE PARTICLES WITHIN THE 

4205
03:26:51,504 --> 03:26:51,971
CHAUM BERS.

4206
03:26:51,971 --> 03:26:53,373
AND WHAT PERCENTAGE TO 

4207
03:26:53,373 --> 03:26:56,709
UNDERSTAND WHAT FRACTION OF YOUR

4208
03:26:56,709 --> 03:27:00,079
DOSE IS WITHIN THE AEROSOL AND 

4209
03:27:00,079 --> 03:27:02,015
CHARACTERIZATION AND BY CONTRAST

4210
03:27:02,015 --> 03:27:05,318
IS THE TESTING SCHEME USING A 

4211
03:27:05,318 --> 03:27:07,420
LASER DETECTION METHOD WHEREBY 

4212
03:27:07,420 --> 03:27:09,222
YOU USE LASER TO MEASURE IN REAL

4213
03:27:09,222 --> 03:27:10,456
TIME THE DISTRIBUTION OF 

4214
03:27:10,456 --> 03:27:11,324
PARTICLES GENERATED BY AN 

4215
03:27:11,324 --> 03:27:15,495
AEROSOL.

4216
03:27:15,495 --> 03:27:18,531
IT'S MORE DIFFICULT TO COLLECT 

4217
03:27:18,531 --> 03:27:20,700
BUT HIGH THROUGHPUT AND 

4218
03:27:20,700 --> 03:27:20,967
ACCURATE.

4219
03:27:20,967 --> 03:27:24,037
THESE ARE TWO COMMON METHODS 

4220
03:27:24,037 --> 03:27:27,607
USED TO CHARACTERIZE AEROSOLS IN

4221
03:27:27,607 --> 03:27:29,942
UNDERSTANDING WHAT IS YOUR 

4222
03:27:29,942 --> 03:27:33,479
PRODUCT LOOKING LIKE AND HOW IS 

4223
03:27:33,479 --> 03:27:36,382
IT PERFORMING.

4224
03:27:36,382 --> 03:27:38,985
TO WRAP UP A COUPLE KEY POINTS 

4225
03:27:38,985 --> 03:27:41,988
IS SELECTION OF YOUR NEBULIZER 

4226
03:27:41,988 --> 03:27:43,790
TECHNOLOGY IS IMPORTANT AND 

4227
03:27:43,790 --> 03:27:45,491
NEEDS TON THE MOST APPROPRIATE 

4228
03:27:45,491 --> 03:27:47,260
FOR YOUR TARGET INDICATION AND 

4229
03:27:47,260 --> 03:27:48,327
FOR YOUR THERAPEUTIC PRODUCT AND

4230
03:27:48,327 --> 03:27:52,598
THAT'S INFLUENCED BY A NUMBER OF

4231
03:27:52,598 --> 03:27:54,500
CONSIDERATIONS.

4232
03:27:54,500 --> 03:27:57,003
ANIMAL MODELS EXHIBIT 

4233
03:27:57,003 --> 03:27:57,603
SIGNIFICANT INDICATIONS FOR 

4234
03:27:57,603 --> 03:27:58,504
AEROSOL DELIVERY AND IT'S 

4235
03:27:58,504 --> 03:27:59,839
IMPORTANT TO UNDERSTAND WHAT 

4236
03:27:59,839 --> 03:28:02,075
THEY ARE AND THINK ABOUT THE 

4237
03:28:02,075 --> 03:28:03,509
APPROPRIATE SCIENTIFIC 

4238
03:28:03,509 --> 03:28:05,144
JUSTIFICATION FOR UTILIZATION OF

4239
03:28:05,144 --> 03:28:06,946
THE RIGHT ANIMAL AND GET 

4240
03:28:06,946 --> 03:28:08,815
AGREEMENT FROM THE FDA AROUND 

4241
03:28:08,815 --> 03:28:11,951
YOUR RATIONALE AND ANIMAL MODELS

4242
03:28:11,951 --> 03:28:14,620
AS APPROPRIATE.

4243
03:28:14,620 --> 03:28:20,293
 DETERMINE STARTING CLINICAL 

4244
03:28:20,293 --> 03:28:21,494
DOSE TO ENSURE PATIENTS ARE 

4245
03:28:21,494 --> 03:28:26,065
GOING TO SEE NOT ONLY SAFE BUT 

4246
03:28:26,065 --> 03:28:27,333
EFFICACIOUS DOSE AND CONSIDER 

4247
03:28:27,333 --> 03:28:28,367
DEVELOPMENT PLAN TIME LINE AND 

4248
03:28:28,367 --> 03:28:33,406
WHEN TO SEEK INPUT ON YOUR PLAN 

4249
03:28:33,406 --> 03:28:34,707
FROM THE FDA AND THANK YOU FOR 

4250
03:28:34,707 --> 03:28:35,341
YOUR TAKES AND LOOK FORWARD TO 

4251
03:28:35,341 --> 03:28:41,314
THE DISCUSSION.

4252
03:28:41,314 --> 03:28:42,482
>> THANK YOU.

4253
03:28:42,482 --> 03:28:45,985
A GREAT SUMMARY OF A DETAILED 

4254
03:28:45,985 --> 03:28:47,353
APPROACH OVER YEARS OF 

4255
03:28:47,353 --> 03:28:52,091
EXPERIENCE AND WORKING WITH 

4256
03:28:52,091 --> 03:28:52,825
REGULATORS DIRECTLY.

4257
03:28:52,825 --> 03:28:54,460
IF THERE'S QUESTIONS FOR KEVIN 

4258
03:28:54,460 --> 03:28:56,429
BEFORE YOU E-MAIL HIM PERSONALLY

4259
03:28:56,429 --> 03:28:57,396
ASK HIM IN YOUR DISCUSSION AT 

4260
03:28:57,396 --> 03:28:58,231
THE END OF TODAY AND E-MAIL HIM 

4261
03:28:58,231 --> 03:29:04,036
LATER.

4262
03:29:04,036 --> 03:29:07,740
NEXT UP IS DR. HOLLY CLARK 

4263
03:29:07,740 --> 03:29:12,678
>> GOOD AFTERNOON MY NAME
IS 

4264
03:29:12,678 --> 03:29:15,481
HOLLY CLARK AND I'M U.K. 

4265
03:29:15,481 --> 03:29:19,318
INNOVATE AT THE UNIVERSITY OF 

4266
03:29:19,318 --> 03:29:20,920
KENTUCKY AND WE THINK AHEAD TO 

4267
03:29:20,920 --> 03:29:24,824
MAKE A PRODUCT OR PATIENT FROM 

4268
03:29:24,824 --> 03:29:26,058
YOUR INNOVATION.

4269
03:29:26,058 --> 03:29:30,329
AND YOU'VE HEARD A LOT OF VERY 

4270
03:29:30,329 --> 03:29:32,231
VALUABLE INFORMING ABOUT THE 

4271
03:29:32,231 --> 03:29:34,934
DRUG DEVELOPMENT PROCESS AND TOX

4272
03:29:34,934 --> 03:29:37,603
STUDIES AND REGULATORY MODELS 

4273
03:29:37,603 --> 03:29:39,739
AND REQUIREMENTED AND MORE AND 

4274
03:29:39,739 --> 03:29:41,340
TO HAVE THE NEW THERAPEUTIC EN 

4275
03:29:41,340 --> 03:29:43,576
TORE FOR A NEW PATIENT YOU NEED 

4276
03:29:43,576 --> 03:29:45,011
TO THINK OF THE 

4277
03:29:45,011 --> 03:29:48,181
COMMERCIALIZATION PROCESS.

4278
03:29:48,181 --> 03:29:50,483
SO, IT'S ALSO OTHER WISE KNOWN 

4279
03:29:50,483 --> 03:29:52,985
AS WHY AND WHEN TO CONTACT THE 

4280
03:29:52,985 --> 03:30:00,426
TECHNOLOGY TRANSFER OFFICE.

4281
03:30:00,426 --> 03:30:02,895
THE FIRST QUESTION IS THE WHY.

4282
03:30:02,895 --> 03:30:04,630
I USE THE SLIDE WHEN I'M TALKING

4283
03:30:04,630 --> 03:30:07,667
TO FACULTY AND OTHER RESEARCHERS

4284
03:30:07,667 --> 03:30:09,135
ACROSS CAMPUS AND I HEAR I TO MY

4285
03:30:09,135 --> 03:30:11,003
RESEARCH AND I PUBLISH AND 

4286
03:30:11,003 --> 03:30:12,672
PRESENT IT AND GO TO CONFERENCES

4287
03:30:12,672 --> 03:30:18,477
BUT WHY SHOULD I DO THIS 

4288
03:30:18,477 --> 03:30:19,245
INTELLECTUAL PROPERTY 

4289
03:30:19,245 --> 03:30:20,179
COMMERCIALIZATION THING?

4290
03:30:20,179 --> 03:30:21,547
PROTECTING YOUR INNOVATION IS 

4291
03:30:21,547 --> 03:30:24,250
NECESSARY TO GET YOUR NEW 

4292
03:30:24,250 --> 03:30:25,351
THERAPEUTIC TO A PATIENT.

4293
03:30:25,351 --> 03:30:28,554
OF COURSE THERE'S MUCH VALUE TO 

4294
03:30:28,554 --> 03:30:29,856
YOUR UNIVERSITY OR RESEARCH 

4295
03:30:29,856 --> 03:30:33,259
INSTITUTION AND THE INNOVATOR IN

4296
03:30:33,259 --> 03:30:34,293
THE PROCESS.

4297
03:30:34,293 --> 03:30:37,730
IT'S GETTING NEW UNIVERSITY OR 

4298
03:30:37,730 --> 03:30:39,432
RESEARCH INNOVATIONS ON THE 

4299
03:30:39,432 --> 03:30:40,800
MARKET.

4300
03:30:40,800 --> 03:30:42,001
NEW PRODUCTS TO IMPROVE QUALITY 

4301
03:30:42,001 --> 03:30:45,705
OF LIFE FOR A PATIENT.

4302
03:30:45,705 --> 03:30:47,273
IF YOUR TALKING ABOUT STARTING A

4303
03:30:47,273 --> 03:30:49,442
COMPANY AND BUILDING LOCALLY 

4304
03:30:49,442 --> 03:30:54,480
IT'S ECONOMIC DEVELOPMENT.

4305
03:30:54,480 --> 03:30:57,516
AND RESEARCH INSTITUTION THAT 

4306
03:30:57,516 --> 03:31:02,488
HAS A ROBUST PROGRAM IN THIS 

4307
03:31:02,488 --> 03:31:05,992
AREA CAN ATTRACT INNOVATIVE 

4308
03:31:05,992 --> 03:31:08,060
FACULTY AND RETAIN STUDENTS AND 

4309
03:31:08,060 --> 03:31:10,496
POSTTOCK.

4310
03:31:10,496 --> 03:31:13,432
WITH PROMOTE INDUSTRIAL 

4311
03:31:13,432 --> 03:31:14,500
RELATIONS TO LICENSE NOVE RATION

4312
03:31:14,500 --> 03:31:19,338
AND SUPPORTING NEW RESEARCH AND 

4313
03:31:19,338 --> 03:31:20,873
THE RIGS GOES TO THE PERSONAL 

4314
03:31:20,873 --> 03:31:26,512
SIDE TO THE INNOVATORS.

4315
03:31:26,512 --> 03:31:29,749
PERSONAL SATISFACTION OF THE 

4316
03:31:29,749 --> 03:31:31,083
DISCOVERY OF A PATIENT HELPING A

4317
03:31:31,083 --> 03:31:32,285
PATIENT.

4318
03:31:32,285 --> 03:31:33,886
I ALSO WANT TO MENTION MOST 

4319
03:31:33,886 --> 03:31:37,023
INSTITUTIONS, IF NOT ALL, OUR 

4320
03:31:37,023 --> 03:31:38,457
INSTITUTION AND OTHERS DO SHARE 

4321
03:31:38,457 --> 03:31:41,727
LICENSE INCOME WITH INNOVATORS 

4322
03:31:41,727 --> 03:31:42,161
DIRECTLY.

4323
03:31:42,161 --> 03:31:47,633
THERE'S VALUE IN KNOWING YOUR 

4324
03:31:47,633 --> 03:31:54,507
INNOVATION GOT OUT THERE AND THE

4325
03:31:54,507 --> 03:31:55,508
INITIAL INNOVATION RAND 

4326
03:31:55,508 --> 03:31:57,143
TECHNOLOGY TRANSFER PROCESS IS 

4327
03:31:57,143 --> 03:31:58,077
SUMMED UP ON THE SLIDE.

4328
03:31:58,077 --> 03:32:00,279
FIRST, GIVE US A CALL.

4329
03:32:00,279 --> 03:32:03,149
TALK TO YOUR TECH TRANSFER 

4330
03:32:03,149 --> 03:32:03,382
OFFICE.

4331
03:32:03,382 --> 03:32:05,818
SUBMIT A DESCRIPTION OF YOUR 

4332
03:32:05,818 --> 03:32:07,853
INNOVATION THROUGH WHATEVER 

4333
03:32:07,853 --> 03:32:08,587
PORTAL SYSTEM PROCESS YOU'RE 

4334
03:32:08,587 --> 03:32:09,555
INSTITUTION USES.

4335
03:32:09,555 --> 03:32:15,561
WE HAVE AN ONLINE PROGRAM 

4336
03:32:15,561 --> 03:32:20,666
PORTAL.

4337
03:32:20,666 --> 03:32:22,468
I USUAL HAVE A NOTE SAYING 

4338
03:32:22,468 --> 03:32:23,703
BEFORE YOU PUBLISH OR PRESENT.

4339
03:32:23,703 --> 03:32:28,407
SO IF YOU'RE TAKING NOTES RIGHT 

4340
03:32:28,407 --> 03:32:31,777
NOW, PLEASE WRITE DOWN CONTACT 

4341
03:32:31,777 --> 03:32:34,113
MY TECH TRANSFER OFFICE BEFORE I

4342
03:32:34,113 --> 03:32:36,949
PRESENT AND YOU'LL SEE WHY IN A 

4343
03:32:36,949 --> 03:32:37,883
FEW SLIDE.

4344
03:32:37,883 --> 03:32:40,686
THAT'S THE FIRST PART.

4345
03:32:40,686 --> 03:32:41,554
LET US KNOW ABOUT YOUR 

4346
03:32:41,554 --> 03:32:42,088
INNOVATION.

4347
03:32:42,088 --> 03:32:43,889
AS SOON AS WE KNOW ABOUT YOUR 

4348
03:32:43,889 --> 03:32:44,657
INNOVATION WE WELL LEARN MORE 

4349
03:32:44,657 --> 03:32:47,159
AND LIKE TO SEE DATA AND LIKE TO

4350
03:32:47,159 --> 03:32:47,493
LEARN.

4351
03:32:47,493 --> 03:32:56,369
WE'LL THEN DO A PRETTY 

4352
03:32:56,369 --> 03:32:57,036
MARKETABILITY ASSESSMENT TO SEE 

4353
03:32:57,036 --> 03:33:03,609
HOW TO PROTECT THE INNOVATION 

4354
03:33:03,609 --> 03:33:06,212
AND INTELLECTUAL PROPERTY.

4355
03:33:06,212 --> 03:33:15,287
WE'RE   NONPROFIT AND NEED TO GO

4356
03:33:15,287 --> 03:33:15,821
FORWARD TO CREATE A NEW 

4357
03:33:15,821 --> 03:33:25,498
THERAPEUTIC OR DRUG.

4358
03:33:25,498 --> 03:33:28,234
WE WANT TO PROTECT THE 

4359
03:33:28,234 --> 03:33:29,035
INTELLECTUAL PROPERTY.

4360
03:33:29,035 --> 03:33:33,439
THIS SLIDE SUMMARIZES THE FOYER 

4361
03:33:33,439 --> 03:33:34,707
PLAIN WAYS INTELLECTUAL PROPERTY

4362
03:33:34,707 --> 03:33:37,009
IS PROTECTED FOR RESEARCH 

4363
03:33:37,009 --> 03:33:38,477
INSTITUTIONS.

4364
03:33:38,477 --> 03:33:44,550
WE FOCUS ON PATENT COPYRIGHT AND

4365
03:33:44,550 --> 03:33:47,319
TRADEMARK AND I PUT TRADE SECRET

4366
03:33:47,319 --> 03:33:49,622
TO THE SIDE BECAUSE THEY'RE 

4367
03:33:49,622 --> 03:33:49,855
SECRET.

4368
03:33:49,855 --> 03:33:52,258
SOME ARE USED AND THEY'RE MEANT 

4369
03:33:52,258 --> 03:34:00,099
TO BE CONFIDENT CONFIDENTIAL AND

4370
03:34:00,099 --> 03:34:01,967
THAT'S FOR FUNDING WHERE WE WANT

4371
03:34:01,967 --> 03:34:04,703
TO DISSEMINATE RESULTS BROADLY 

4372
03:34:04,703 --> 03:34:06,472
TO THE PUBLIC.

4373
03:34:06,472 --> 03:34:08,741
WE DON'T TEND TO DO TRADE 

4374
03:34:08,741 --> 03:34:10,242
SECRETS SO I'LL FOCUS ON PATENTS

4375
03:34:10,242 --> 03:34:11,944
AND WHAT DOES IT GET YOU IF YOU 

4376
03:34:11,944 --> 03:34:16,182
HAVE AN ISSUED PATENT?

4377
03:34:16,182 --> 03:34:18,451
THE RIGHT TO EXCLUDE USING A 

4378
03:34:18,451 --> 03:34:20,119
CLAIMED INVENTION FOR A 

4379
03:34:20,119 --> 03:34:23,322
CERTAINLY PERIOD OF TIME, A 20 

4380
03:34:23,322 --> 03:34:26,692
YEAR PERIOD AND TRANSLATES TO A 

4381
03:34:26,692 --> 03:34:34,500
PERIOD OF EXCLUSIVITY.

4382
03:34:34,500 --> 03:34:38,904
I MENTIONED IT EXCLUDES OTHERS 

4383
03:34:38,904 --> 03:34:41,507
BUT IT WAS SET UP TO ENCOURAGE 

4384
03:34:41,507 --> 03:34:45,544
INNOVATION AND ENCOURAGES 

4385
03:34:45,544 --> 03:34:47,279
THE DISCLOSURE OF KNOWLEDGE 

4386
03:34:47,279 --> 03:34:49,982
BECAUSE PRAT ENTS ARE PUBLISHED 

4387
03:34:49,982 --> 03:34:52,885
YOU CAN SEE THE LATEST AND EST 

4388
03:34:52,885 --> 03:34:54,487
IN UPCOMING INNOVATIONS IN THE 

4389
03:34:54,487 --> 03:34:57,623
FIELD AND HOW YOU MIGHT FURTHER 

4390
03:34:57,623 --> 03:34:58,757
INNOVATE IN THE FIELD.

4391
03:34:58,757 --> 03:35:01,360
MOST IMPORTANTLY FOR THE 

4392
03:35:01,360 --> 03:35:04,096
PURPOSES OF COMMERCIALIZATION 

4393
03:35:04,096 --> 03:35:05,164
PATENTS PROTECTION INTELLECTUAL 

4394
03:35:05,164 --> 03:35:05,831
PROPERTY PROTECTION IS USUALLY 

4395
03:35:05,831 --> 03:35:08,434
THE BEST WAY TO BENEFIT THE 

4396
03:35:08,434 --> 03:35:10,202
PUBLIC HEALTH BECAUSE OF THAT 

4397
03:35:10,202 --> 03:35:11,604
RIGHT TO EXCLUDE OTHERS FROM 

4398
03:35:11,604 --> 03:35:13,606
MAKING, AND SELLING.

4399
03:35:13,606 --> 03:35:15,441
THAT ABILITY SO EXCLUSIVITY AND 

4400
03:35:15,441 --> 03:35:18,511
A COMPANY PARTNER IS GOING TO 

4401
03:35:18,511 --> 03:35:21,013
WANT EXCLUSIVITY TO KNOW WHEN 

4402
03:35:21,013 --> 03:35:22,581
THEY'RE PUTTING IN MILLIONS OF 

4403
03:35:22,581 --> 03:35:25,451
DOLLARS TO DEVELOP A NEW 

4404
03:35:25,451 --> 03:35:26,485
THERAPEUTIC, THERE'S AN A PERIOD

4405
03:35:26,485 --> 03:35:36,729
OF EXCLUSIVY.

4406
03:35:37,429 --> 03:35:42,268
VERY QUICKLY WHAT CAN BE PR

4407
03:35:42,268 --> 03:35:43,335
PATENTED?

4408
03:35:43,335 --> 03:35:44,737
RATE.

4409
03:35:44,737 --> 03:35:51,544
I'LL FOCUS ON THE SLIDE 

4410
03:35:51,544 --> 03:35:54,280
COMPOSITIONAL MATTER YOUR NEW 

4411
03:35:54,280 --> 03:35:56,115
THERAPEUTIC OR BIOLOGIC AND THE 

4412
03:35:56,115 --> 03:35:57,383
NEW INNOVATION AND METHOD OF 

4413
03:35:57,383 --> 03:35:57,883
TREATMENT.

4414
03:35:57,883 --> 03:35:59,919
YOU PLAY HAVE A NEW INNOVATION.

4415
03:35:59,919 --> 03:36:04,623
YOU MAY BE REPURPOSING A NEW 

4416
03:36:04,623 --> 03:36:07,159
DRUG AND IT MIGHT BE REPURPOSING

4417
03:36:07,159 --> 03:36:08,928
A NEW DRUG FOR A NEW INDICATION.

4418
03:36:08,928 --> 03:36:11,130
IT WOULD BE A METHOD OF NEW 

4419
03:36:11,130 --> 03:36:20,873
TREATMENT FOR A NEW DRUG.

4420
03:36:20,873 --> 03:36:23,042
I MENTIONED THE VAECHLT PROCESS 

4421
03:36:23,042 --> 03:36:24,643
IN YOUR UNIVERSITY TECH TRANSFER

4422
03:36:24,643 --> 03:36:25,311
OFFICE.

4423
03:36:25,311 --> 03:36:27,446
IT'S IMPORTANT TO HAVE THEM 

4424
03:36:27,446 --> 03:36:30,282
CONDUCT A VERY THOROUGH PATENT 

4425
03:36:30,282 --> 03:36:31,250
ABILITY ASSESSMENT.

4426
03:36:31,250 --> 03:36:32,117
YOU CERTAINLY ARE EXPERTS IN 

4427
03:36:32,117 --> 03:36:33,285
YOUR FIELD AND KNOW THE 

4428
03:36:33,285 --> 03:36:33,586
LITERATURE.

4429
03:36:33,586 --> 03:36:38,490
BUT PUBLISHED PAPERS DO NOT 

4430
03:36:38,490 --> 03:36:40,326
INCLUDE EVERYTHING MOST 

4431
03:36:40,326 --> 03:36:41,594
COMPANIES DON'T PUBLISH ON THEIR

4432
03:36:41,594 --> 03:36:42,528
LEAD DRUG.

4433
03:36:42,528 --> 03:36:45,030
INSTEAD THEY CERTAINLY ARE GOING

4434
03:36:45,030 --> 03:36:45,598
TO PROTECT THEM.

4435
03:36:45,598 --> 03:36:49,568
SO TECH TRANSFER OFFICES WILL 

4436
03:36:49,568 --> 03:36:52,238
SEARCH BOTH THE U.S. AND WORLD 

4437
03:36:52,238 --> 03:36:54,473
WIDE PATENT DATA OFFICES AND 

4438
03:36:54,473 --> 03:36:55,808
WHEN THEY FIND SOMETHING CLOSE 

4439
03:36:55,808 --> 03:36:57,243
THEY'LL LET YOU KNOW.

4440
03:36:57,243 --> 03:36:59,144
WE ENGAGE THE INNOVATORS.

4441
03:36:59,144 --> 03:37:01,380
IT'S A TIME AND COST SAVING WITH

4442
03:37:01,380 --> 03:37:03,215
PAT IN THE PROSECUTION AND CAN 

4443
03:37:03,215 --> 03:37:06,852
HELP US GUIDE THE INNOVATION AND

4444
03:37:06,852 --> 03:37:08,320
HOW THE CLAIMS WILL BE 

4445
03:37:08,320 --> 03:37:08,621
STRUCTURED.

4446
03:37:08,621 --> 03:37:09,555
THE EARLIER THE BETTER.

4447
03:37:09,555 --> 03:37:11,890
THIS WILL BE A THEME OF MY TALK.

4448
03:37:11,890 --> 03:37:13,826
THE EARLIER THE BETTER YOU TALK 

4449
03:37:13,826 --> 03:37:15,327
TO YOUR TECH TRANSFER OFFICE IS 

4450
03:37:15,327 --> 03:37:16,362
THE BETTER.

4451
03:37:16,362 --> 03:37:19,398
GIVE YOUR TECH TRANSFER OFFICE 

4452
03:37:19,398 --> 03:37:21,567
TIME TO CONDUCT A THOROUGH 

4453
03:37:21,567 --> 03:37:29,408
ASSESSMENT.

4454
03:37:29,408 --> 03:37:32,278
WE'VE DONE AN ASSESSMENT AND IT 

4455
03:37:32,278 --> 03:37:35,047
LOOKS LIKE WE'LL HAVE ROOM TO 

4456
03:37:35,047 --> 03:37:36,081
GAIN CLAIMS AND LOOKS LIKE 

4457
03:37:36,081 --> 03:37:40,152
THERE'S AN AREA WHAT WE FOUND IS

4458
03:37:40,152 --> 03:37:43,455
CERTAINLY NEW, USEFUL, OFFICE.

4459
03:37:43,455 --> 03:37:46,525
BECAUSE IT'S THE INVENTION MAY 

4460
03:37:46,525 --> 03:37:48,761
BE PATENTABLE DOESN'T MEAN 

4461
03:37:48,761 --> 03:37:50,963
ANYONE WILL PICK UP THE PRODUCT.

4462
03:37:50,963 --> 03:37:54,600
WE ALSO LOOK IN THE MARKET.

4463
03:37:54,600 --> 03:37:59,672
WHAT'S THE MARKET POTENTIAL AND 

4464
03:37:59,672 --> 03:38:03,008
BETTER THAN WHAT IS OUT THERE 

4465
03:38:03,008 --> 03:38:05,844
AND THIS IS SOMETHING THE TECH 

4466
03:38:05,844 --> 03:38:08,013
TRANSFER OFFICE WILL WORK WITH 

4467
03:38:08,013 --> 03:38:09,548
YOU ON.

4468
03:38:09,548 --> 03:38:11,784
THE THIRD PART OF THE ASSESSMENT

4469
03:38:11,784 --> 03:38:13,218
PROCESS IS THE COMMERCIALIZATION

4470
03:38:13,218 --> 03:38:13,419
PART.

4471
03:38:13,419 --> 03:38:20,559
AS I MENTIONED BEFORE, AND WE 

4472
03:38:20,559 --> 03:38:22,494
DON'T PUT THINGS IN A BOTTLE AND

4473
03:38:22,494 --> 03:38:25,431
MARKET AND SELL TO THE PUBLIC 

4474
03:38:25,431 --> 03:38:25,831
FOR PUBLIC HEALTH.

4475
03:38:25,831 --> 03:38:27,833
OUR GOAL IS TO HAVE THE 

4476
03:38:27,833 --> 03:38:30,135
TECHNOLOGY DEVELOPED TO BENEFIT 

4477
03:38:30,135 --> 03:38:31,970
THE PUBLIC AND TO FIND AN 

4478
03:38:31,970 --> 03:38:35,240
INDUSTRY PARTNER TO HELP DEVELOP

4479
03:38:35,240 --> 03:38:37,042
THAT TECHNOLOGY.

4480
03:38:37,042 --> 03:38:38,143
THERE ARE MANY TIMES OF 

4481
03:38:38,143 --> 03:38:40,312
COMMERCIALIZATION PATHWAYS THAT 

4482
03:38:40,312 --> 03:38:42,481
CAN LEAD TO PARTNERSHIP.

4483
03:38:42,481 --> 03:38:44,717
LIKE I SAID IT MIGHT BE WE HAVE 

4484
03:38:44,717 --> 03:38:47,786
A PARTNER THAT WANTS TO HELP 

4485
03:38:47,786 --> 03:38:49,021
SPONSOR DEVELOPMENT OF A 

4486
03:38:49,021 --> 03:38:49,922
TECHNOLOGY GOING FORWARD AND 

4487
03:38:49,922 --> 03:38:57,563
RESEARCH IN A LAB.

4488
03:38:57,563 --> 03:38:58,263
LICENSING.

4489
03:38:58,263 --> 03:38:59,164
WE HAVE PROGRAMS THAT START NEW 

4490
03:38:59,164 --> 03:39:05,504
COMPANIES.

4491
03:39:05,504 --> 03:39:08,507
THIS SLIDE TALKS ABOUT 

4492
03:39:08,507 --> 03:39:09,141
COMMERCIALIZATION PATHWAYS 

4493
03:39:09,141 --> 03:39:10,909
FINDING A COMMERCIALIZATION 

4494
03:39:10,909 --> 03:39:11,477
PARTNER.

4495
03:39:11,477 --> 03:39:13,712
WHEN I STARTED IN TECH TRANSFER 

4496
03:39:13,712 --> 03:39:16,749
FOR A NEW THERAPEUTIC THERE WERE

4497
03:39:16,749 --> 03:39:17,783
LICENSING TO ESTABLISH A COMPANY

4498
03:39:17,783 --> 03:39:18,417
AND POTENTIALLY STARTING A NEW 

4499
03:39:18,417 --> 03:39:25,290
COMPANY.

4500
03:39:25,290 --> 03:39:26,592
THERE'S ADVANTAGES TO WORKING 

4501
03:39:26,592 --> 03:39:29,094
WITH A BIG PHARMA OR ESTABLISHED

4502
03:39:29,094 --> 03:39:30,729
BIO TECH AND THEY'RE WELL FUNDED

4503
03:39:30,729 --> 03:39:33,232
AND BEEN THERE, DONE THAT.

4504
03:39:33,232 --> 03:39:35,167
THEY HAVE THE PROCESS ALREADY 

4505
03:39:35,167 --> 03:39:35,801
THERE, HOWEVER, THERE ARE THINGS

4506
03:39:35,801 --> 03:39:46,011
TO CONSIDER.

4507
03:39:49,615 --> 03:39:51,550
I'VE HEARD FROM THE PARTNERS, 

4508
03:39:51,550 --> 03:39:53,719
THE BIG PLAYERS IT'S AN 

4509
03:39:53,719 --> 03:39:54,787
INTERESTING MECHANISM AND 

4510
03:39:54,787 --> 03:39:57,456
INTRIGUING DATA BUT CALL US WHEN

4511
03:39:57,456 --> 03:40:00,325
IT'S IN PHASE II RAND IT'S HARD 

4512
03:40:00,325 --> 03:40:02,494
TO GET THERE.

4513
03:40:02,494 --> 03:40:06,165
YOU MAY THINK I KNOW MY 

4514
03:40:06,165 --> 03:40:07,566
TECHNOLOGY THE POST AND WILL 

4515
03:40:07,566 --> 03:40:09,902
START A COMPANY.

4516
03:40:09,902 --> 03:40:12,438
OUR COMPANIES HAVE PROGRAMS TO 

4517
03:40:12,438 --> 03:40:14,339
HELP AND HAVE ENTREPRENEURSHIP 

4518
03:40:14,339 --> 03:40:15,941
TRAINING AND HELP WITH FUNDING 

4519
03:40:15,941 --> 03:40:18,110
AND YOU'LL HEAR THE NEXT TALK IS

4520
03:40:18,110 --> 03:40:24,783
ABOUT SOME FUNDING IN THAT AREA 

4521
03:40:24,783 --> 03:40:29,087
YOU NEED TO CONSIDER YOUR AND 

4522
03:40:29,087 --> 03:40:30,289
STARTING A START UP PLAY BE 

4523
03:40:30,289 --> 03:40:33,125
DIFFICULT FOR A ONE-PRODUCT TYPE

4524
03:40:33,125 --> 03:40:35,661
OF TECHNOLOGY OR MAYBE A SMALL 

4525
03:40:35,661 --> 03:40:36,094
MARKET.

4526
03:40:36,094 --> 03:40:38,130
IT'S TIME.

4527
03:40:38,130 --> 03:40:39,031
IT'S FUNDING.

4528
03:40:39,031 --> 03:40:40,599
FUNDING -- THERE ARE CHALLENGES.

4529
03:40:40,599 --> 03:40:42,501
TIME AND FUNDING.

4530
03:40:42,501 --> 03:40:43,836
SO THINGS TO CONSIDER.

4531
03:40:43,836 --> 03:40:48,407
OVER THE PAST DECADE THOUGH A 

4532
03:40:48,407 --> 03:40:49,675
NEW COMMERCIALIZATION PATH HAS 

4533
03:40:49,675 --> 03:40:51,577
ARISEN WHICH IS GOOD FOR NEW 

4534
03:40:51,577 --> 03:40:54,480
THERAPEUTIC TECHNOLOGIES.

4535
03:40:54,480 --> 03:40:57,015
AND IT'S THE VENTURE BUILDER OR 

4536
03:40:57,015 --> 03:41:01,687
VENTURE STUDIO.

4537
03:41:01,687 --> 03:41:03,322
THESE ARE VENTURE CAPITAL 

4538
03:41:03,322 --> 03:41:05,824
ACTUALLY STARTING NEW START UPS 

4539
03:41:05,824 --> 03:41:09,561
BUT HAVE SAID, HMM, MAYBE WE CAN

4540
03:41:09,561 --> 03:41:11,096
REDUCE OUR RISK BY AN 

4541
03:41:11,096 --> 03:41:12,130
INTERESTING ACADEMIC TECHNOLOGY 

4542
03:41:12,130 --> 03:41:14,366
AND LICENSE IT AND BUILD THE 

4543
03:41:14,366 --> 03:41:15,567
COMPANY FROM WITHIN BECAUSE WE 

4544
03:41:15,567 --> 03:41:17,202
HAVE THE FUND AND CAN HAVE A 

4545
03:41:17,202 --> 03:41:17,703
MANAGEMENT TEAM.

4546
03:41:17,703 --> 03:41:19,171
WE HAVE THE EXPERTISE.

4547
03:41:19,171 --> 03:41:21,540
AND THEY WORK WITH THE 

4548
03:41:21,540 --> 03:41:22,741
INSTITUTION, WITH THEY ARE 

4549
03:41:22,741 --> 03:41:24,776
LICENSE THE TECHNOLOGY, WORK 

4550
03:41:24,776 --> 03:41:27,779
WITH THE INNOVATOR AND HELP 

4551
03:41:27,779 --> 03:41:31,617
DEVELOP IT SO IT'S MORE 

4552
03:41:31,617 --> 03:41:33,552
ATTRACTIVE FOR THE BIGGER 

4553
03:41:33,552 --> 03:41:39,892
PLAYERS.

4554
03:41:39,892 --> 03:41:42,494
A PATENT EXCLUDE OTHERS FROM 

4555
03:41:42,494 --> 03:41:44,997
USING AND SELLING AND LICENSE IT

4556
03:41:44,997 --> 03:41:45,697
THROUGH AN AGREEMENT.

4557
03:41:45,697 --> 03:41:48,834
IT'S A PERMISSION SLIP AND 

4558
03:41:48,834 --> 03:41:50,936
IMPORT THE INVENTION.

4559
03:41:50,936 --> 03:41:53,105
THEY CAN BE EXCLUSIVE TO ONE 

4560
03:41:53,105 --> 03:41:54,339
COMPANY OR NON-EXCLUSIVE IF YOU 

4561
03:41:54,339 --> 03:41:56,108
HAVE A PLATFORM TECHNOLOGY AND 

4562
03:41:56,108 --> 03:41:58,810
WANT TO HAVE SEVERAL LICENSEES 

4563
03:41:58,810 --> 03:42:00,812
AND IT MAY BE NON-EXCLUSIVE.

4564
03:42:00,812 --> 03:42:04,049
THERE'S A LOT I CAN GET INTO 

4565
03:42:04,049 --> 03:42:05,784
WITH HOW A LICENSE WORKS BUT THE

4566
03:42:05,784 --> 03:42:07,486
MAJOR BENEFITS TO DOING A 

4567
03:42:07,486 --> 03:42:09,488
LICENSE IS YOU'RE FINDING A 

4568
03:42:09,488 --> 03:42:13,625
PARTNER AND YOU'RE WORKING ONE A

4569
03:42:13,625 --> 03:42:17,462
PARTNER TO GET THE PRODUCT 

4570
03:42:17,462 --> 03:42:17,729
DEVELOPED.

4571
03:42:17,729 --> 03:42:19,331
THERE MAY BE RESEARCH SHARED 

4572
03:42:19,331 --> 03:42:20,599
RESOURCES AND MAY BE SPONSORED 

4573
03:42:20,599 --> 03:42:23,068
RESEARCH A COMPANY MAY SAY WE'RE

4574
03:42:23,068 --> 03:42:24,670
GOING TO LICENSE THE TECHNOLOGY 

4575
03:42:24,670 --> 03:42:25,871
AND SPONSOR RESEARCH IN THE LAB 

4576
03:42:25,871 --> 03:42:27,839
TO DEVELOP IT FURTHER.

4577
03:42:27,839 --> 03:42:31,977
IN TERMS OF INCOME, THE 

4578
03:42:31,977 --> 03:42:33,078
UNIVERSITY CAN RECOUP THE 

4579
03:42:33,078 --> 03:42:33,812
INTELLECTUAL PROPERTY COST.

4580
03:42:33,812 --> 03:42:35,714
THERE MAY BE ROYALTIES OR OTHER 

4581
03:42:35,714 --> 03:42:37,115
PAYMENTS FOR DEVELOPMENT.

4582
03:42:37,115 --> 03:42:38,216
THERE'S A LOT THAT CAN COME FROM

4583
03:42:38,216 --> 03:42:48,694
THE LICENSE OF AN INVENTION.

4584
03:42:51,163 --> 03:42:53,298
ANOTHER REASON TO CONTACT THE 

4585
03:42:53,298 --> 03:42:55,267
TECH TRANSFER OFFICE WE DO A LOT

4586
03:42:55,267 --> 03:42:57,336
OF OTHER THINGS.

4587
03:42:57,336 --> 03:43:00,172
A LOT OF OTHER AGREEMENTS.

4588
03:43:00,172 --> 03:43:02,307
PROTECTING THE EXCHANGE OF 

4589
03:43:02,307 --> 03:43:02,608
INFORMATION.

4590
03:43:02,608 --> 03:43:04,343
IT'S VERY IMPORTANT AMONG 

4591
03:43:04,343 --> 03:43:05,477
RESEARCH INSTITUTIONS AND 

4592
03:43:05,477 --> 03:43:07,245
COLLABORATORS TO SHARE DATA AND 

4593
03:43:07,245 --> 03:43:08,614
INFORMATION AND MATERIALS AND WE

4594
03:43:08,614 --> 03:43:12,451
CAN HANDLE ALL THAT AGREEMENT TO

4595
03:43:12,451 --> 03:43:14,119
PROTECT YOU IN THAT SHARING.

4596
03:43:14,119 --> 03:43:14,853
WHEN THERE'S A JOINT TECHNOLOGY 

4597
03:43:14,853 --> 03:43:19,458
WE WORK IT OUT WITH THE OTHER 

4598
03:43:19,458 --> 03:43:21,193
COLLABORATING INSTITUTION TO 

4599
03:43:21,193 --> 03:43:22,861
COME UP WITH AN AGREEMENT WHICH 

4600
03:43:22,861 --> 03:43:28,033
SETS THE TERMS OF THE AGREEMENT 

4601
03:43:28,033 --> 03:43:30,502
AND HOW A JOINT TECHNOLOGY 

4602
03:43:30,502 --> 03:43:34,873
WESTBOUND  -- WILL BE PROTECTED 

4603
03:43:34,873 --> 03:43:35,307
AND COMMERCIALIZED.

4604
03:43:35,307 --> 03:43:38,410
COMING BACK TO WHEN SHOULD YOU 

4605
03:43:38,410 --> 03:43:40,846
CONTACT MY TECH TRANSFER OFFICE?

4606
03:43:40,846 --> 03:43:41,113
EARLY.

4607
03:43:41,113 --> 03:43:43,849
WE LIKE TO SAY IT'S NEVER TOO 

4608
03:43:43,849 --> 03:43:45,784
EARLY BEFORE YOU PUBLISH OR 

4609
03:43:45,784 --> 03:43:47,085
PRESENT HOWEVER IT CAN BE TOO 

4610
03:43:47,085 --> 03:43:47,285
LATE.

4611
03:43:47,285 --> 03:43:52,257
YOU WANT TO MAKE SURE YOU 

4612
03:43:52,257 --> 03:43:54,059
PROTECT WORLDWIDE PATENT RIGHTS.

4613
03:43:54,059 --> 03:43:55,861
CONTACT US FOR TRANSFERS OF 

4614
03:43:55,861 --> 03:43:56,728
MATERIALS, CONFIDENTIAL 

4615
03:43:56,728 --> 03:44:02,200
INFORMATION, CONNECTIONS WITH 

4616
03:44:02,200 --> 03:44:03,735
RESEARCHERS -- REGULATORY AND 

4617
03:44:03,735 --> 03:44:09,307
OTHER PARTNERS.

4618
03:44:09,307 --> 03:44:19,151
I GET INNOVATORS WHO ARE WRITING

4619
03:44:19,151 --> 03:44:19,985
 FUNDING OPPORTUNITIES, I WILL

4620
03:44:19,985 --> 03:44:24,856
MENTION A FEW IN A MOMENT AND

4621
03:44:24,856 --> 03:44:28,627
LETTERS OF MENTION, NEXT SLIDE,

4622
03:44:28,627 --> 03:44:29,528
PLEASE.

4623
03:44:29,528 --> 03:44:30,162
NEXT SLIDE, PLEASE.

4624
03:44:30,162 --> 03:44:31,096
VERY QUICKLY I WOULD LIKE TO

4625
03:44:31,096 --> 03:44:36,468
INTRODUCE YOU TO OUR OFFICE, UK

4626
03:44:36,468 --> 03:44:36,735
INNOVATE.

4627
03:44:36,735 --> 03:44:39,838
ACTUALLY NEXT SLIDE, GO,A HEAD.

4628
03:44:39,838 --> 03:44:40,806
-- GO,A HEAD.

4629
03:44:40,806 --> 03:44:43,542
WE ARE A BIT UNIQUE IN THAT ALL

4630
03:44:43,542 --> 03:44:45,644
AREAS OF COMMERCIALIZATION AND

4631
03:44:45,644 --> 03:44:47,979
ENTREPRENEURSHIP ARE LOCATED

4632
03:44:47,979 --> 03:44:53,118
UNDER 1 ROOF, THAT'S UKINNOVATE,

4633
03:44:53,118 --> 03:44:54,419
THE COMMERCIALIZATION PROCESS IS

4634
03:44:54,419 --> 03:44:55,587
UNDER OUR COMMERCIALIZATION

4635
03:44:55,587 --> 03:44:57,489
PILLAR.

4636
03:44:57,489 --> 03:44:59,458
OUR INNOVATION TRAINING AND

4637
03:44:59,458 --> 03:45:01,026
ENTREPRENEURSHIP TRAINING IS

4638
03:45:01,026 --> 03:45:02,427
UNDER OUR ECOSYSTEM PILLAR WHERE

4639
03:45:02,427 --> 03:45:07,632
WE HELP TEACH OUR INNOVATORS

4640
03:45:07,632 --> 03:45:08,900
ABOUT THIS PROCESS.

4641
03:45:08,900 --> 03:45:11,837
INNOVATION CONNECT IS OUR

4642
03:45:11,837 --> 03:45:12,437
INDUSTRY PARTNERSHIP'S GROUP.

4643
03:45:12,437 --> 03:45:14,740
THIS IS A GROWING AREA AND I'M

4644
03:45:14,740 --> 03:45:17,242
HAPPY TO SAY UK'S IN THE LEAD IN

4645
03:45:17,242 --> 03:45:21,947
THIS AREA AND INNOVATIONS THAT

4646
03:45:21,947 --> 03:45:23,415
HAVE SOCIETAL IMPACT AND EXIST

4647
03:45:23,415 --> 03:45:28,120
WITH AND FOR THE COMMUNITY FOR

4648
03:45:28,120 --> 03:45:31,022
THE PUBLIC BENEFIT AND NEXT

4649
03:45:31,022 --> 03:45:31,790
SLIDE, PLEASE.

4650
03:45:31,790 --> 03:45:33,091
AND JUST HIGHLIGHTING A FEW

4651
03:45:33,091 --> 03:45:34,726
PROGRAMS, I MENTIONED FUNDING,

4652
03:45:34,726 --> 03:45:37,963
UK IS PART OF THE MIDSOUTH HUB

4653
03:45:37,963 --> 03:45:45,570
WHICH IS A 4 STATE HUB FOR BOTH

4654
03:45:45,570 --> 03:45:47,939
NSF I-CORP AND RESEARCH AND

4655
03:45:47,939 --> 03:45:49,007
EVALUATION HUB.

4656
03:45:49,007 --> 03:45:51,009
IT'S A FUNDING PROGRAM FOCUSED

4657
03:45:51,009 --> 03:45:51,810
ON PRODUCT DEVELOPMENT.

4658
03:45:51,810 --> 03:45:53,512
WE HAVE OTHER PROGRAMS THAT ARE

4659
03:45:53,512 --> 03:45:56,114
ON THIS SLIDE, IN BLUE IS A

4660
03:45:56,114 --> 03:45:57,449
BRAND NEW, WE JUST ROLLED THAT

4661
03:45:57,449 --> 03:45:57,616
OUT.

4662
03:45:57,616 --> 03:46:02,721
IT'S A NEOF THE  -- NETWORK AND

4663
03:46:02,721 --> 03:46:04,156
TRAINING FOR ANGEL INVESTORS TO

4664
03:46:04,156 --> 03:46:06,258
INVEST IN OUR START-UP

4665
03:46:06,258 --> 03:46:06,925
COMPANIES.

4666
03:46:06,925 --> 03:46:07,959
NEXT SLIDE, PLEASE.

4667
03:46:07,959 --> 03:46:09,861
WITH THAT, THANK YOU VERY MUCH.

4668
03:46:09,861 --> 03:46:11,830
ONCE AGAIN I'M HOLLI AND THIS IS

4669
03:46:11,830 --> 03:46:14,966
WHERE TO FIND ME.

4670
03:46:14,966 --> 03:46:17,736
>> HOLLY, THANKS SO MUCH
IF ARE

4671
03:46:17,736 --> 03:46:18,170
THAT WONDERFUL TALK.

4672
03:46:18,170 --> 03:46:20,038
WORKING WITH TECH TRANSFER

4673
03:46:20,038 --> 03:46:21,306
OFFICES, I CAN UNDERSCORE HOW

4674
03:46:21,306 --> 03:46:22,374
IMPORTANT IT IS THAT INDUSTRY

4675
03:46:22,374 --> 03:46:24,009
LOVES TO WORK WITH FOLKS THAT

4676
03:46:24,009 --> 03:46:25,710
ARE REALLY ORGANIZED, HAVE THEIR

4677
03:46:25,710 --> 03:46:26,912
ACTS TOGETHER AND KNOW EXACTLY

4678
03:46:26,912 --> 03:46:28,079
HOW TO COMMUNICATE WITH US

4679
03:46:28,079 --> 03:46:29,481
BECAUSE OF WHAT WE'RE LOOKING

4680
03:46:29,481 --> 03:46:31,449
FOR, SO THANKS FOR THAT GREAT

4681
03:46:31,449 --> 03:46:32,851
OVERVIEW THEY THINK REALLY

4682
03:46:32,851 --> 03:46:34,886
CAPTURES IT.

4683
03:46:34,886 --> 03:46:35,153
THANK YOU.

4684
03:46:35,153 --> 03:46:39,424
ALL RIGHT, SO NEXT UP IS

4685
03:46:39,424 --> 03:46:40,826
DR. STEPHANIE DAVIS.

4686
03:46:40,826 --> 03:46:41,393
>> THANK YOU JEFF.

4687
03:46:41,393 --> 03:46:43,328
IF YOU COULD ADVANCE TO THE NEXT

4688
03:46:43,328 --> 03:46:43,895
SLIDE.

4689
03:46:43,895 --> 03:46:45,831
I WILL TRY TO ZIP THROUGH THESE

4690
03:46:45,831 --> 03:46:47,465
WITH MY COLLEAGUE DR. MIKE PECK,

4691
03:46:47,465 --> 03:46:48,633
NEXT SLIDE, THERE WE GO.

4692
03:46:48,633 --> 03:46:51,136
SO WE'RE GOING TO BE COVERING 2

4693
03:46:51,136 --> 03:46:54,406
PROGRAMS THAT CAN HELP FUND

4694
03:46:54,406 --> 03:46:55,874
TRANSLATIONAL TECHNOLOGIES IN

4695
03:46:55,874 --> 03:46:57,809
THE HEART, LUNG, BLOOD AND SLEEP

4696
03:46:57,809 --> 03:46:58,043
SPACE.

4697
03:46:58,043 --> 03:47:00,445
I WILL TALK ABOUT THE SMALL

4698
03:47:00,445 --> 03:47:03,915
BUSINESS, ALSO KNOWN AS THE

4699
03:47:03,915 --> 03:47:06,418
SBIR/STTR PROGRAMS AND DR. PECK

4700
03:47:06,418 --> 03:47:07,519
WILL COVER CATALYZE.

4701
03:47:07,519 --> 03:47:10,522
SO WE WILL NEXT THIS EFFICIENT.

4702
03:47:10,522 --> 03:47:11,323
NEXT SLIDE, PLEASE.

4703
03:47:11,323 --> 03:47:14,259
NEXT SLIDE, PLEASE. 

4704
03:47:14,259 --> 03:47:15,360
THERE WE GO.

4705
03:47:15,360 --> 03:47:17,562
SO IT WAS A GREAT THING THAT

4706
03:47:17,562 --> 03:47:18,496
DR. CLARK WENT BEFORE US

4707
03:47:18,496 --> 03:47:21,900
BECAUSE, YOU KNOW IF YOU ARE AN

4708
03:47:21,900 --> 03:47:23,101
ACADEMIC RESEARCHER YOUR TECH

4709
03:47:23,101 --> 03:47:25,003
TRANSFER OFFICE WILL BE A GREAT

4710
03:47:25,003 --> 03:47:27,105
RESOURCE FOR ANYTHING HAVE TO DO

4711
03:47:27,105 --> 03:47:28,874
FOR APPLYING FOR SMALL BUSINESS

4712
03:47:28,874 --> 03:47:29,107
FUNDING.

4713
03:47:29,107 --> 03:47:30,242
SO FOR THOSE WHO ARE UNFAMILIAR

4714
03:47:30,242 --> 03:47:33,945
WITH THE PROGRAMS, THE SBIR, TCR

4715
03:47:33,945 --> 03:47:35,347
PROGRAMS MAKE UP THE AMERICA'S

4716
03:47:35,347 --> 03:47:37,616
SEED FUND OR THE SMALL BUSINESS

4717
03:47:37,616 --> 03:47:38,316
PROGRAM.

4718
03:47:38,316 --> 03:47:41,386
AT THE NIH WE SET ASIDE 1.4

4719
03:47:41,386 --> 03:47:43,321
BILLION DOLLARS TOWARDS FUNDING

4720
03:47:43,321 --> 03:47:44,389
EARLY STAGE TECHNOLOGIES THAT

4721
03:47:44,389 --> 03:47:45,857
CAN HELP ADVANCE THE MISSION OF

4722
03:47:45,857 --> 03:47:46,491
THE NIH.

4723
03:47:46,491 --> 03:47:47,192
OH, GO BACK.

4724
03:47:47,192 --> 03:47:48,693
THANK YOU.

4725
03:47:48,693 --> 03:47:50,862
SO THESE PROGRAMS CONSIST OF THE

4726
03:47:50,862 --> 03:47:52,464
SMALL BUSINESS INNOVATION

4727
03:47:52,464 --> 03:47:53,531
RESEARCH, OR SBIR, WHICH THE IS

4728
03:47:53,531 --> 03:47:55,267
PARENT PROGRAM AND THIS IS

4729
03:47:55,267 --> 03:47:56,101
DESIGNED TO SUPPORT SMALL

4730
03:47:56,101 --> 03:47:57,168
BUSINESS CONCERNS THAT ARE

4731
03:47:57,168 --> 03:47:58,236
DEVELOPING NEW TECHNOLOGIES FOR

4732
03:47:58,236 --> 03:47:59,971
THE POTENTIAL FOR COMMERCIAL

4733
03:47:59,971 --> 03:48:01,606
ARIZATION, AND THE SISTER

4734
03:48:01,606 --> 03:48:02,540
PROGRAM, THE STTR PROGRAM.

4735
03:48:02,540 --> 03:48:05,477
THIS IS VERY SIMILAR TO THE

4736
03:48:05,477 --> 03:48:06,811
SBIR, WITH 1 MAJOR DIFFERENCE IN

4737
03:48:06,811 --> 03:48:10,048
TERMS OF ITS GOAL AND THAT IS TO

4738
03:48:10,048 --> 03:48:11,249
FACILITATE PARTNERSHIPS BETWEEN

4739
03:48:11,249 --> 03:48:14,552
ACADEMIC INSTITUTIONS AND SMALL

4740
03:48:14,552 --> 03:48:14,853
BUSINESSES.

4741
03:48:14,853 --> 03:48:15,053
OKAY.

4742
03:48:15,053 --> 03:48:23,528
JUST GO TO THE NEXT SLIDE.

4743
03:48:23,528 --> 03:48:25,330
THERE WE G. BOTH OF THESE

4744
03:48:25,330 --> 03:48:26,564
PROGRAMS HAVE A SIMILAR GOAL.

4745
03:48:26,564 --> 03:48:28,867
AND THROUGH BOTH OF THESE

4746
03:48:28,867 --> 03:48:30,602
PROGRAMS, THE NHIERARCHIES H IS

4747
03:48:30,602 --> 03:48:31,670
STAGED AS THE EARLY CAPITAL FOR

4748
03:48:31,670 --> 03:48:35,707
STAGES IN THE U.S., SO RO-1,

4749
03:48:35,707 --> 03:48:38,743
R23S, OTHER BASIC GRANTS TEND TO

4750
03:48:38,743 --> 03:48:40,145
FUND SMALL BUSINESS SCIENCE,

4751
03:48:40,145 --> 03:48:41,947
SMALL BUSINESS FUNDS WILL FUND

4752
03:48:41,947 --> 03:48:43,581
EVERYTHING FROM EARLY STAGE,

4753
03:48:43,581 --> 03:48:45,016
PROOF OF CONCEPT, TECHNOLOGICAL

4754
03:48:45,016 --> 03:48:46,551
DEVELOPMENT ALL THE WAY UP TO

4755
03:48:46,551 --> 03:48:47,085
SCALE AND MANUFACTURING.

4756
03:48:47,085 --> 03:48:50,021
AND WE ARE THERE TO FUND THESE

4757
03:48:50,021 --> 03:48:51,389
INNOVATIONS WHEN PRIVATE

4758
03:48:51,389 --> 03:48:54,259
INVESTORS SUCH AS ANGEL

4759
03:48:54,259 --> 03:48:59,698
INVESTORS, VC TIRMS AND PART

4760
03:48:59,698 --> 03:49:02,133
MERS ARE NOT TO RISK TOO MUCH.

4761
03:49:02,133 --> 03:49:04,269
SO OUR GOAL IS TO RISK ENOUGH

4762
03:49:04,269 --> 03:49:06,705
THAT THEY ARE ATTRACTED FOR

4763
03:49:06,705 --> 03:49:07,439
THESE PRIVATE INVESTORS.

4764
03:49:07,439 --> 03:49:11,409
WHEN YOU APPLY FOR AN NIH GRANT,

4765
03:49:11,409 --> 03:49:12,377
IT'S NONDILUTIVE, WE'RE NOT

4766
03:49:12,377 --> 03:49:14,312
TAKING MONEY OUT OF YOUR COMPANY

4767
03:49:14,312 --> 03:49:16,314
AND IT'S A LOAN THAT YOU DON'T

4768
03:49:16,314 --> 03:49:17,382
HAVE TO PAY BACK.

4769
03:49:17,382 --> 03:49:18,984
AND WHEN YOU GET THESE GRANTS

4770
03:49:18,984 --> 03:49:22,520
YOU HAVE TO GO THROUGH THE

4771
03:49:22,520 --> 03:49:23,788
RIGOROUS PEER REVIEW PROCESS AS

4772
03:49:23,788 --> 03:49:33,298
YOU WOULD WITH ANYTHING ELSE AT

4773
03:49:33,298 --> 03:49:34,566
THE NIH. 

4774
03:49:34,566 --> 03:49:38,570
NEXT SLIDE, PLEASE.

4775
03:49:38,570 --> 03:49:40,872
SO AS I MENTIONED THE SMALL

4776
03:49:40,872 --> 03:49:43,875
BUSINESS PROGRAMS ARE MADE UP OF

4777
03:49:43,875 --> 03:49:45,343
SBIR AND STTR GRANTS AND THEY

4778
03:49:45,343 --> 03:49:46,845
ARE MORE SIMILAR THAN THEY ARE

4779
03:49:46,845 --> 03:49:48,747
DIFFERENT BUT THERE ARE A FEW

4780
03:49:48,747 --> 03:49:50,415
KEY DIFFERENCES.

4781
03:49:50,415 --> 03:49:51,916
SO SBIR GRANTS DON'T REQUIRE

4782
03:49:51,916 --> 03:49:53,752
PARTNERING WITH BUT THEY CAN

4783
03:49:53,752 --> 03:49:55,754
ALLOW FOR PARTNERING TO OCCUR,

4784
03:49:55,754 --> 03:49:57,155
THERE ARE GUIDELINES WHERE FOR

4785
03:49:57,155 --> 03:49:58,456
EARLY STAGE GRANTS ALSO KNOWN AS

4786
03:49:58,456 --> 03:50:00,392
PHASE 1S ISSUES THE COMPANY CAN

4787
03:50:00,392 --> 03:50:05,030
OUTSOURCE UP TO 33% OF THE

4788
03:50:05,030 --> 03:50:06,164
BUDGET AND 50% OF THE BUDGET IN

4789
03:50:06,164 --> 03:50:08,733
PHASE 2 AND THERE IS A

4790
03:50:08,733 --> 03:50:10,468
REQUIREMENT FOR AN SBIR ON THE

4791
03:50:10,468 --> 03:50:13,338
GRANT TO BE PRIMARILY EMPLOYED

4792
03:50:13,338 --> 03:50:14,806
BY THE BUSINESS AT THE TIME OF

4793
03:50:14,806 --> 03:50:16,107
THE AWARD.

4794
03:50:16,107 --> 03:50:17,842
SO THE STTR, IT'S THE PRIME

4795
03:50:17,842 --> 03:50:21,212
OPTION FOR EARLY STAGE ACADEMIC

4796
03:50:21,212 --> 03:50:22,013
SPIN-OUTS A-BECAUSE THERE'S A

4797
03:50:22,013 --> 03:50:23,548
REQUIREMENT TO HAVE A RESEARCH

4798
03:50:23,548 --> 03:50:26,117
INSTITUTION PARTNER WITH THE

4799
03:50:26,117 --> 03:50:27,052
SMALL BUSINESS THAT THAT

4800
03:50:27,052 --> 03:50:28,119
RESEARCH INSTITUTION AND PARTNER

4801
03:50:28,119 --> 03:50:30,221
WILL BE A SMALL ORDER RECIPIENT.

4802
03:50:30,221 --> 03:50:31,122
THERE ARE MINIMUM WORK

4803
03:50:31,122 --> 03:50:32,290
REQUIREMENTS OR BUDGET

4804
03:50:32,290 --> 03:50:33,224
REQUIREMENTS FOR BOTH THE SMALL

4805
03:50:33,224 --> 03:50:34,392
BUSINESS AND RESEARCH

4806
03:50:34,392 --> 03:50:35,794
INSTITUTION PARTNER AND MOST

4807
03:50:35,794 --> 03:50:37,395
IMPORTANTLY, THE PI CAN BE

4808
03:50:37,395 --> 03:50:39,230
EMPLOYED EITHER BY THE RESEARCH

4809
03:50:39,230 --> 03:50:40,498
INSTITUTION OR BY THE SMALL

4810
03:50:40,498 --> 03:50:40,932
BUSINESS.

4811
03:50:40,932 --> 03:50:43,835
SO IF YOU'RE A FULL-TIME FACULTY

4812
03:50:43,835 --> 03:50:45,703
MEMBER WITH A COMPANY, STTR A

4813
03:50:45,703 --> 03:50:47,372
GREAT OPTION FOR YOU BUT IN BOTH

4814
03:50:47,372 --> 03:50:51,409
CASES THE AWARD IS MADE TO SMALL

4815
03:50:51,409 --> 03:50:56,314
BUSINESSES.

4816
03:50:56,314 --> 03:50:57,215
NEXT SLIDE.

4817
03:50:57,215 --> 03:50:59,284
SO THERE ARE ELIGIBILITY

4818
03:50:59,284 --> 03:51:01,119
CRITERIA THAT NEED TO BE MADE

4819
03:51:01,119 --> 03:51:03,221
THROUGH THE TIME OF THEARS WARDS

4820
03:51:03,221 --> 03:51:05,390
BUT IN SUMMARY IN ORDER FOR YOUR

4821
03:51:05,390 --> 03:51:07,258
COMPANY TO BE ELIGIBLE FOR A

4822
03:51:07,258 --> 03:51:09,594
GRANT, IT MUST BE ORGANIZE AS A

4823
03:51:09,594 --> 03:51:11,563
U.S. BUSINESS, SMALLER THAN 500

4824
03:51:11,563 --> 03:51:12,497
EMPLOYEES, ALTHOUGH MOST OF THE

4825
03:51:12,497 --> 03:51:13,565
COMPANIES WE FUND ARE MUCH

4826
03:51:13,565 --> 03:51:14,632
SMALLER THAN THIS AND THE WORK

4827
03:51:14,632 --> 03:51:16,201
ON THE PROJECT MUST BE DONE

4828
03:51:16,201 --> 03:51:19,737
WITHIN THE U.S. WITH A FEW VERY

4829
03:51:19,737 --> 03:51:20,872
CASE BY CASE EXEMPTIONS.

4830
03:51:20,872 --> 03:51:25,310
SO THE COMPANY MUST BE AT LEAST

4831
03:51:25,310 --> 03:51:27,946
50% U.S. OWNED AND INDEPENDENTLY

4832
03:51:27,946 --> 03:51:29,247
OPERATED SO KEEP IN MIND ALL

4833
03:51:29,247 --> 03:51:30,248
THESE THINGS NEED TO BE MET AT

4834
03:51:30,248 --> 03:51:32,917
THE TIME OF THE AWARD, BUT DUE

4835
03:51:32,917 --> 03:51:36,254
TO THE SBIR AND STTR POLICY

4836
03:51:36,254 --> 03:51:37,755
DIRECTIVE, ALL ORGANIZATIONS

4837
03:51:37,755 --> 03:51:39,357
THAT RECEIVE GRANTS NEED TO

4838
03:51:39,357 --> 03:51:40,692
ADHERE TO THIS CRITERIA.

4839
03:51:40,692 --> 03:51:41,159
NEXT SLIDE, PLEASE.

4840
03:51:41,159 --> 03:51:42,794
SO AS I MENTIONED THERE ARE

4841
03:51:42,794 --> 03:51:43,628
DIFFERENT PHASES OF SMALL

4842
03:51:43,628 --> 03:51:44,462
BUSINESS GRANTS AND THESE ARE

4843
03:51:44,462 --> 03:51:47,332
NOT THE SAME AS CLINICAL TRIAL

4844
03:51:47,332 --> 03:51:47,732
PHASES.

4845
03:51:47,732 --> 03:51:50,201
THEY HAVE SAID THEY REFER TO

4846
03:51:50,201 --> 03:51:50,969
STAGE OF DEVELOP.

4847
03:51:50,969 --> 03:51:52,003
SO EARLY STAGE INVESTIGATORS

4848
03:51:52,003 --> 03:51:54,239
TEND TO COME IN WITH A PHASE 1

4849
03:51:54,239 --> 03:51:55,840
GRANT AND THIS IS A SMALL GRANT

4850
03:51:55,840 --> 03:51:57,609
THAT SUPPORTS PROOF OF CONCEPT

4851
03:51:57,609 --> 03:52:00,512
WORK, USUALLY IT'S AROUND

4852
03:52:00,512 --> 03:52:01,546
$314,000 TOTAL COSTS, AND

4853
03:52:01,546 --> 03:52:03,848
BETWEEN 1 AND 2 YEARS IN LENGTH.

4854
03:52:03,848 --> 03:52:05,583
AND ONCE THE PHASE 1 IS DONE,

4855
03:52:05,583 --> 03:52:06,985
THE COMPANY CAN APPLY FOR A

4856
03:52:06,985 --> 03:52:10,054
PHASE 2 GRANT WHICH SUPPORTS

4857
03:52:10,054 --> 03:52:11,956
MORE EFFICACY ORIENTED WORK AND

4858
03:52:11,956 --> 03:52:13,658
MORE EXTENSIVE R&D
ACTIVITIES

4859
03:52:13,658 --> 03:52:15,059
COMPARED TO THE PHASE 1.

4860
03:52:15,059 --> 03:52:17,061
SO THESE GRANTS CAN BE BETWEEN 1

4861
03:52:17,061 --> 03:52:20,231
AND 3 YEARS LONG AND USUALLY

4862
03:52:20,231 --> 03:52:22,133
THEY'RE A LITTLE BIT OVER

4863
03:52:22,133 --> 03:52:23,067
$2 MILLION TOTAL COST.

4864
03:52:23,067 --> 03:52:24,369
THERE ARE ADDITIONAL OPTIONS, SO

4865
03:52:24,369 --> 03:52:26,237
IF A COMPANY WANTS TO APPLY FOR

4866
03:52:26,237 --> 03:52:28,540
BOTH THE PHASE 1 AND PHASE 2

4867
03:52:28,540 --> 03:52:29,674
SIMULTANEOUSLY, THEY CAN DO IT

4868
03:52:29,674 --> 03:52:32,243
FAST TRACK AND ALSO FOR SBIR

4869
03:52:32,243 --> 03:52:33,511
APPLICANTS, IF A COMPANY HAS

4870
03:52:33,511 --> 03:52:35,346
ALREADY DONE WHAT WOULD AMOUNT

4871
03:52:35,346 --> 03:52:36,748
TO A PHASE 1 GRANT, THEY CAN DO

4872
03:52:36,748 --> 03:52:38,183
A DIRECT TO PHASE 2.

4873
03:52:38,183 --> 03:52:40,952
AND SOME INSTITUTES AND CENTERS

4874
03:52:40,952 --> 03:52:43,121
INCLUDING THE NHLBI OFFER

4875
03:52:43,121 --> 03:52:43,821
ADDITIONAL FUNDING OPPORTUNITIES

4876
03:52:43,821 --> 03:52:44,789
BEYOND THE PHASE 2.

4877
03:52:44,789 --> 03:52:48,193
HOWEVER, WE DON'T HAVE PHASE 3

4878
03:52:48,193 --> 03:52:48,726
AWARDS.

4879
03:52:48,726 --> 03:52:54,532
SOME  AGENCIES WILL ACCESS THESE

4880
03:52:54,532 --> 03:52:55,934
WHERE THE BUYER ACCESSES THE

4881
03:52:55,934 --> 03:52:57,669
INFORMATION, WE DON'T DO THAT.

4882
03:52:57,669 --> 03:52:59,737
WE HAVE THE IDEA THAT BEYOND

4883
03:52:59,737 --> 03:53:04,175
THESE FUNDING OPPORTUNITIES THE

4884
03:53:04,175 --> 03:53:07,278
COMPANY WILL BE SUPPORTED BY A

4885
03:53:07,278 --> 03:53:08,346
STRATEGIC PARTNER OR SUPPORTED

4886
03:53:08,346 --> 03:53:09,914
BY PRIVATE FUNDS AND WON'T NEED

4887
03:53:09,914 --> 03:53:13,418
US ANYMORE.

4888
03:53:13,418 --> 03:53:14,185
NEXT SLIDE.

4889
03:53:14,185 --> 03:53:16,254
SO I RECOMMEND IN ANYBODY HAS

4890
03:53:16,254 --> 03:53:17,555
QUESTIONS ABOUT PUTTING TOGETHER

4891
03:53:17,555 --> 03:53:19,624
AN APPLICATION, THE NIH SEED

4892
03:53:19,624 --> 03:53:21,926
OFFICE WEBSITE HAS A LOT OF

4893
03:53:21,926 --> 03:53:24,996
REALLY GREAT RESOURCES AND THESE

4894
03:53:24,996 --> 03:53:26,931
INCLUDE THE SF424 APPLICATION

4895
03:53:26,931 --> 03:53:29,234
MANUEL FOR SMALL BUSINESS

4896
03:53:29,234 --> 03:53:30,568
GRANTS, ANTIINFORM SET AND

4897
03:53:30,568 --> 03:53:32,136
SAMPLE APPLICATIONS FROM REAL

4898
03:53:32,136 --> 03:53:33,705
LIFE SMALL BUSINESS GRANTEES.

4899
03:53:33,705 --> 03:53:36,107
I ALSO RECOMMEND THAT YOU LOOK

4900
03:53:36,107 --> 03:53:37,575
THROUGH STATE RESOURCES, THROUGH

4901
03:53:37,575 --> 03:53:40,345
THE SBA FAST PROGRAM, AND OTHER

4902
03:53:40,345 --> 03:53:41,746
RESOURCES THAT HOLLY SHOUTED OUT

4903
03:53:41,746 --> 03:53:43,715
TO IN HER PRESENTATION, IF YOU

4904
03:53:43,715 --> 03:53:45,950
HAVE ANY NIH FUNDED ACADEMIC

4905
03:53:45,950 --> 03:53:47,452
PROOF OF CONCEPT PROGRAMS, SUCH

4906
03:53:47,452 --> 03:53:49,554
AS THE REACH PROGRAM, WHICH RUNS

4907
03:53:49,554 --> 03:53:51,289
THE MIDSOUTH HUB, I WOULD

4908
03:53:51,289 --> 03:53:52,323
STRONGLY RECOMMEND TAKE 8

4909
03:53:52,323 --> 03:53:54,025
HOURSING ADVANTAGE OF THOSE TO

4910
03:53:54,025 --> 03:53:55,560
HELP MAKE YOURSELF MORE

4911
03:53:55,560 --> 03:53:58,263
COMPETITIVE. 

4912
03:53:58,263 --> 03:53:59,764
NEXT SLIDE, PLEASE.

4913
03:53:59,764 --> 03:54:01,633
>> SO THE NHLBI SMALL
BUSINESS

4914
03:54:01,633 --> 03:54:04,235
PROGRAM, WE HAVE APPROXIMATELY

4915
03:54:04,235 --> 03:54:06,638
$136 MILLION EVERY YEAR IN

4916
03:54:06,638 --> 03:54:08,139
NONDILUTED FUNDING TO SUPPORT

4917
03:54:08,139 --> 03:54:14,679
OVER 300 ACTIVE  PROJECTS THAT

4918
03:54:14,679 --> 03:54:17,415
ARE MEANT TO PREVENT, TREAT AND

4919
03:54:17,415 --> 03:54:18,583
DIAGNOSE LUNG DISEASES, SO AS

4920
03:54:18,583 --> 03:54:19,751
YOU SEE BY THIS PIE CHART WE

4921
03:54:19,751 --> 03:54:21,152
HAVE A LOT OF TECHNOLOGIES WE

4922
03:54:21,152 --> 03:54:22,820
FUND AND THIS BREAK DOWN VARIES

4923
03:54:22,820 --> 03:54:27,358
FROM YEAR TO YEAR BUT ABOUT 33%

4924
03:54:27,358 --> 03:54:29,560
OF OUR PORTFOLIO CONSISTS OF

4925
03:54:29,560 --> 03:54:31,429
THERAPEUTICS BOTH SMALL

4926
03:54:31,429 --> 03:54:33,331
MOLECULES AND BIOLOGICS.

4927
03:54:33,331 --> 03:54:33,931
NEXT SLIDE, PLEASE.

4928
03:54:33,931 --> 03:54:37,101
SO HERE IS JUST A BRIEF SAMPLING

4929
03:54:37,101 --> 03:54:39,437
OF DISEASES THAT FALL WITHIN THE

4930
03:54:39,437 --> 03:54:42,373
NHLBI MISSION SPACE.  THIS IS

4931
03:54:42,373 --> 03:54:43,574
NOT AN EXHAUSTIVE LIST THOUGH SO

4932
03:54:43,574 --> 03:54:45,643
IF YOU HAVE A CONDITION THAT'S

4933
03:54:45,643 --> 03:54:47,679
NOT LISTED HERE AND YOU'RE NOT

4934
03:54:47,679 --> 03:54:49,314
SURE WHETHER IT'S WITH OUR

4935
03:54:49,314 --> 03:54:50,815
MISSIONS BASE, WE STRONGLY

4936
03:54:50,815 --> 03:54:51,949
RECOMMEND REACHING OUT TO US

4937
03:54:51,949 --> 03:54:54,786
FIRST AND IF IT DOESN'T WORK

4938
03:54:54,786 --> 03:54:58,289
WITH US, WE CAN DIRECT YOU TO

4939
03:54:58,289 --> 03:54:59,691
THE INSTITUTION OR BEST FIT.

4940
03:54:59,691 --> 03:55:02,193
SO I WILL NOT GO INTO ALL THE

4941
03:55:02,193 --> 03:55:03,227
DETAILTHIS SLIDE BUT THIS

4942
03:55:03,227 --> 03:55:04,896
PROVIDES A BRIEF SUMMARY OF WHAT

4943
03:55:04,896 --> 03:55:07,231
MY OFFICE, THE INNOVATION AND

4944
03:55:07,231 --> 03:55:09,534
COMMERCIALIZATION OFFICE DOES SO

4945
03:55:09,534 --> 03:55:10,702
OUR OFFICE PROVIDES SUPPORT IN

4946
03:55:10,702 --> 03:55:12,370
THE FORM OF APPLICATION SERVICES

4947
03:55:12,370 --> 03:55:15,373
SO THIS CAN INCLUDE OUTREACH AND

4948
03:55:15,373 --> 03:55:18,643
EDUCATION EVENTS, PREAPPLICATION

4949
03:55:18,643 --> 03:55:19,344
CONSULTS, AND ASSISTANCE

4950
03:55:19,344 --> 03:55:22,480
PROGRAMS TO HELP COMPANIES

4951
03:55:22,480 --> 03:55:23,247
BECOME COMPETITIVE APPLICANTS.

4952
03:55:23,247 --> 03:55:28,319
WE OFFER SMALL BUSINESS GRANTS,

4953
03:55:28,319 --> 03:55:30,288
PHASE 1 AND PHASE 2 AND

4954
03:55:30,288 --> 03:55:31,222
ADDITIONAL FUNDING BEYOND PHASE

4955
03:55:31,222 --> 03:55:33,758
2 AND FOR AWARDEES THEY HAVE A

4956
03:55:33,758 --> 03:55:35,526
LOT OF DIFFERENT RESOURCES THEY

4957
03:55:35,526 --> 03:55:37,862
CAN TAKE ADVANTAGE OF FOR THEIR

4958
03:55:37,862 --> 03:55:38,663
PRODUCT DEVELOPMENT JOURNEY.

4959
03:55:38,663 --> 03:55:41,766
SO THESE CAN INCLUDE CONSULTS

4960
03:55:41,766 --> 03:55:42,834
FROM OUR PART-TIME ENTREPRENEUR

4961
03:55:42,834 --> 03:55:44,268
AND RESIDENT WHO IS CAN PROVIDE

4962
03:55:44,268 --> 03:55:46,671
FEEDBACK IN AREAS SUCH AS

4963
03:55:46,671 --> 03:55:48,639
INTELLECTUAL PROPERTY,

4964
03:55:48,639 --> 03:55:50,074
REGULATORY AFFAIRS,

4965
03:55:50,074 --> 03:55:50,708
REIMBURSEMENT, HEALTH ECONOMICS

4966
03:55:50,708 --> 03:55:51,909
CLINICAL TRIALS AND FUNDRAISING

4967
03:55:51,909 --> 03:55:55,179
AND THESE EE Rs HELP US PUT ON

4968
03:55:55,179 --> 03:55:59,684
WEBINARS AND EDUCATIONAL EVENTS.

4969
03:55:59,684 --> 03:56:01,953
SO WE HAVE ADDITIONAL

4970
03:56:01,953 --> 03:56:03,121
ADMINISTRATIVE SUPPLEMENTS SUCH

4971
03:56:03,121 --> 03:56:05,022
AS RECRUITING NEW TALENT TO

4972
03:56:05,022 --> 03:56:05,790
ENHANCE THE DIVERSITY OF THE

4973
03:56:05,790 --> 03:56:08,359
PROGRAM, WE HAVE A SPECIAL

4974
03:56:08,359 --> 03:56:09,260
DIVERSITY SUPPLEMENT PROGRAM

4975
03:56:09,260 --> 03:56:10,428
SPECIFICALLY FOR SMALL BUSINESS

4976
03:56:10,428 --> 03:56:10,795
GRANTS.

4977
03:56:10,795 --> 03:56:15,199
WE SUPPORT COMPANIES THROUGH THE

4978
03:56:15,199 --> 03:56:17,535
ICHOR PROGRAM WHICH HELPS WITH

4979
03:56:17,535 --> 03:56:20,438
DORPHY, WE PROVIDE TECHNICAL AND

4980
03:56:20,438 --> 03:56:21,005
BUSINESS ASSISTANCE RELATED

4981
03:56:21,005 --> 03:56:21,706
ACTIVITIES AND BEYOND THE PHASE

4982
03:56:21,706 --> 03:56:23,908
2 WE OFFER SPECIAL AWARDS

4983
03:56:23,908 --> 03:56:25,243
THROUGH THE COMMERCIALIZATION

4984
03:56:25,243 --> 03:56:29,147
READINESS PILOT, AWARD PROGRAM.

4985
03:56:29,147 --> 03:56:31,916
WE ALSO HAVE A LOT OF NEW

4986
03:56:31,916 --> 03:56:33,451
ACTIVITIES, WE'RE DOING AS PART

4987
03:56:33,451 --> 03:56:35,653
OF OUR INVESTOR INITIATIVES.

4988
03:56:35,653 --> 03:56:38,689
SO THIS CAN INCLUDE COMPANIES TO

4989
03:56:38,689 --> 03:56:40,658
PRESENT AT SHOWCASE EVENTS WHERE

4990
03:56:40,658 --> 03:56:42,827
THEY'RE COACHED BY OUR EIR TEAM

4991
03:56:42,827 --> 03:56:45,730
SO THEY CAN PUT TOGETHER A GREAT

4992
03:56:45,730 --> 03:56:46,697
PRESENTATION THAT ATTRACTS

4993
03:56:46,697 --> 03:56:48,499
INVESTORS IN ATTENDANCE, WE HAVE

4994
03:56:48,499 --> 03:56:51,169
ALSO RECENTLY DEVELOPED AN AI

4995
03:56:51,169 --> 03:56:52,437
POWERED PARTNERING PLATFORM TO

4996
03:56:52,437 --> 03:56:54,772
BETTER HELP OUR AWARDYS CONNECT

4997
03:56:54,772 --> 03:56:56,774
WITH POTENTIAL INVESTORS AND

4998
03:56:56,774 --> 03:56:57,742
COLLABORATORS AND STRATEGIC

4999
03:56:57,742 --> 03:57:04,549
PARTNERS. 

5000
03:57:04,549 --> 03:57:04,982
NEXT SLIDE, PLEASE.

5001
03:57:04,982 --> 03:57:08,152
HERE IS THE TARGETING

5002
03:57:08,152 --> 03:57:11,823
OPPORTUNITIES THAT THE NHLBI

5003
03:57:11,823 --> 03:57:14,492
FOCUSES ON, MOST OF THESE COME

5004
03:57:14,492 --> 03:57:15,893
IN AS OMNIBUS SOLICITATIONS BUT

5005
03:57:15,893 --> 03:57:17,428
THERE ARE A FEW SELECTED

5006
03:57:17,428 --> 03:57:19,430
OPPORTUNITIES THAT HAVE SPECIAL

5007
03:57:19,430 --> 03:57:22,200
DUE DATES OR SPECIAL REVIEW

5008
03:57:22,200 --> 03:57:24,268
CRITERIA OR IN SOME CASES SET

5009
03:57:24,268 --> 03:57:25,102
ASIDE FUNDS.

5010
03:57:25,102 --> 03:57:26,737
SO HERE'S EXAMPLES OF SPECIAL

5011
03:57:26,737 --> 03:57:27,605
FUNDING OPPORTUNITY WE SUPPORT,

5012
03:57:27,605 --> 03:57:33,644
SO WE HAVE OUR SPECIAL PHASE 2

5013
03:57:33,644 --> 03:57:36,180
IIB-BRIDGE AND SMALL MARKET

5014
03:57:36,180 --> 03:57:39,083
RFAs WHICH PROVIDE FUNDING FOR

5015
03:57:39,083 --> 03:57:40,017
COMMERCIALIZATION, THE

5016
03:57:40,017 --> 03:57:41,118
COMMERCIALIZATION PILOT

5017
03:57:41,118 --> 03:57:42,587
READINESS AWARDS WHICH CAN

5018
03:57:42,587 --> 03:57:44,555
PROVIDE UP TO $500,000 IN

5019
03:57:44,555 --> 03:57:47,492
EXPANSE PENSS TO SUPPORT LATE

5020
03:57:47,492 --> 03:57:49,894
STAGE RND AND TECHNICAL

5021
03:57:49,894 --> 03:57:50,495
ASSISTANCE.

5022
03:57:50,495 --> 03:57:52,096
FOR COMPANIES DEVELOPING

5023
03:57:52,096 --> 03:57:53,598
TECHNOLOGYINGS FOCUSED ON PANE

5024
03:57:53,598 --> 03:57:56,400
MANAGEMENT WE PARTICIPATE IN THE

5025
03:57:56,400 --> 03:57:58,269
HEAL INITIATIVE RFAs, TODAY WE

5026
03:57:58,269 --> 03:58:00,104
SUPPORTED 3 PROJECTS TOTAL 2 OF

5027
03:58:00,104 --> 03:58:02,406
WHICH FOCUSED ON PROJECTS IN THE

5028
03:58:02,406 --> 03:58:03,708
LUNG DISEASES MISSION SPACE.

5029
03:58:03,708 --> 03:58:05,543
IF YOU'RE DEVELOPING ANYTHING

5030
03:58:05,543 --> 03:58:08,713
FOCUSED ON HEALTH DISPARITIES OR

5031
03:58:08,713 --> 03:58:09,747
TREATING MINORITY HEALTH

5032
03:58:09,747 --> 03:58:12,216
PATIENTS WITH HEART, LUNG, BLOOD

5033
03:58:12,216 --> 03:58:14,385
AND SLEEP DISORDERS, WE STRONGLY

5034
03:58:14,385 --> 03:58:16,254
RECOMMEND LIVING INTO THE

5035
03:58:16,254 --> 03:58:17,855
HEALTHY LIVING RNA AND FINALLY

5036
03:58:17,855 --> 03:58:19,390
WE HAVE 4 NOTICES OF FUNDING

5037
03:58:19,390 --> 03:58:22,360
OPPORTUNITY THAT SERVE AS AN

5038
03:58:22,360 --> 03:58:24,295
OMNIBUS SET FOR OUR NEW

5039
03:58:24,295 --> 03:58:25,229
ENTREPRENEURS AND EARLY STAGE

5040
03:58:25,229 --> 03:58:26,731
INVESTIGATORS WHO WANT TO SERVE

5041
03:58:26,731 --> 03:58:30,601
AS PIs ON SMALL BUSINESS

5042
03:58:30,601 --> 03:58:35,339
GRANTS.

5043
03:58:35,339 --> 03:58:35,840
NEXT SLIDE, PLEASE.

5044
03:58:35,840 --> 03:58:38,009
AND EACH IF YOU'RE NOT APPLYING

5045
03:58:38,009 --> 03:58:43,414
FOR THE OPPORTUNITIES AND GOING

5046
03:58:43,414 --> 03:58:46,150
THROUGH THE OMNIBUS, WE

5047
03:58:46,150 --> 03:58:48,586
RECOMMEND LOOKING AT THIS FOR

5048
03:58:48,586 --> 03:58:50,688
PRIORITIZING HIGH AREAS OF

5049
03:58:50,688 --> 03:58:51,889
SCIENTIFIC ACTIVITY.

5050
03:58:51,889 --> 03:58:54,992
YOU CAN PUT OMNIBUS BUT IF PUT

5051
03:58:54,992 --> 03:58:57,428
THE NOCI IN THE PAGE 4, YOU CAN

5052
03:58:57,428 --> 03:58:58,829
GET THE APPLICATION TAGGED AS

5053
03:58:58,829 --> 03:59:00,831
BEING A PRIORITY AREA, SO HAVE

5054
03:59:00,831 --> 03:59:03,768
YOU A PROPOSAL THAT RELATES TO 1

5055
03:59:03,768 --> 03:59:05,636
OF THESE NOCIs, WE RECOMMEND

5056
03:59:05,636 --> 03:59:11,008
APPLYING THROUGH IT. 

5057
03:59:11,008 --> 03:59:11,442
NEXT SLIDE, PLEASE.

5058
03:59:11,442 --> 03:59:12,743
ALL RIGHT AND SO IF YOU HAVE ANY

5059
03:59:12,743 --> 03:59:14,045
QUESTIONS OR WANT TO FIND OUT

5060
03:59:14,045 --> 03:59:14,946
ANYTHING ABOUT THE SMALL

5061
03:59:14,946 --> 03:59:17,682
BUSINESS PROGRAM AND

5062
03:59:17,682 --> 03:59:18,649
COMMERCIALIZATION OFFICE, I

5063
03:59:18,649 --> 03:59:23,588
RECOMMEND FOLLOWING US ON X,

5064
03:59:23,588 --> 03:59:24,522
VISITING OUR WEBSITE OR IF HAVE

5065
03:59:24,522 --> 03:59:26,257
YOU ANY QUESTIONS YOU CAN REACH

5066
03:59:26,257 --> 03:59:34,799
OUT TO US AT NHLBISCOAR

5067
03:59:34,799 --> 03:59:38,302
SBIR@MAIL.NIH .GOV.

5068
03:59:38,302 --> 03:59:40,905
AND WITH THAT I WILL TURN IT

5069
03:59:40,905 --> 03:59:44,542
OVER TO MY COLLEAGUE DR. PECK TO

5070
03:59:44,542 --> 03:59:44,942
DISCUSS CATALYZE.

5071
03:59:44,942 --> 03:59:46,978
>> I THINK I AM THE LAST
SPEAKER

5072
03:59:46,978 --> 03:59:48,879
IF THIS SESSION SO I WILL TRY TO

5073
03:59:48,879 --> 03:59:49,880
BE SUCCINCT.

5074
03:59:49,880 --> 03:59:52,583
SO WE TOLD YOU ABOUT THE

5075
03:59:52,583 --> 03:59:53,417
SBIR/STTR, WHERE YOU HAVE TO

5076
03:59:53,417 --> 03:59:57,154
HAVE A SMALL BUSINESS TO APPLY,

5077
03:59:57,154 --> 04:00:00,091
CATALYZE SUPPORTS ALL U.S. BASED

5078
04:00:00,091 --> 04:00:05,262
ORGANIZATIONS, FOR PROFIT,

5079
04:00:05,262 --> 04:00:05,529
NONPROFIT.

5080
04:00:05,529 --> 04:00:06,464
PREACADEMIC, ALL OF IT.

5081
04:00:06,464 --> 04:00:08,099
SO WE'RE TAKING A SLIGHTLY

5082
04:00:08,099 --> 04:00:10,301
DIFFERENT APPROACH THAN THE SBIR

5083
04:00:10,301 --> 04:00:12,637
AND OTHER FUNDING PROGRAMS AT

5084
04:00:12,637 --> 04:00:13,938
THE NHLBI, SO INSTEAD OF GIVING

5085
04:00:13,938 --> 04:00:16,907
YOU MONEY AND FINDING ONCE A

5086
04:00:16,907 --> 04:00:18,643
YEAR, WE BECOME A PARTNER AND

5087
04:00:18,643 --> 04:00:20,845
EXTENSION OF YOUR TEAM THROUGH

5088
04:00:20,845 --> 04:00:21,912
OUR RECORDING CENTER AND HELP

5089
04:00:21,912 --> 04:00:22,947
YOU ACHIEVE WHAT YOU'RE TRYING

5090
04:00:22,947 --> 04:00:24,915
TO DO OR LET THE TECHNOLOGY FAIL

5091
04:00:24,915 --> 04:00:28,552
WITH WRAP AROUND SUPPORT WITH

5092
04:00:28,552 --> 04:00:31,188
EXPERTS AND REALLY 3 KEY AREAS,

5093
04:00:31,188 --> 04:00:32,957
EXPERTISE WHEN YOU NEED IT IN

5094
04:00:32,957 --> 04:00:34,892
TECHNICAL AND BUSINESS, AS

5095
04:00:34,892 --> 04:00:37,428
CARMEN MENTIONED THERE'S A

5096
04:00:37,428 --> 04:00:40,164
REGULATORY PART THAT COMES WITH

5097
04:00:40,164 --> 04:00:41,999
THE PROGRAM, AND VALIDATION OFF

5098
04:00:41,999 --> 04:00:43,968
THE BASIC RO-1 OR R35 AWARD, ALL

5099
04:00:43,968 --> 04:00:46,303
THE WAY UP TO MAYBE IN BETWEEN

5100
04:00:46,303 --> 04:00:50,207
SBIR PHASE 2, AND 2 A PHASE 2 B,

5101
04:00:50,207 --> 04:00:54,578
FOR THE STUDIES. 

5102
04:00:54,578 --> 04:00:55,279
NEXT SLIDE, PLEASE.

5103
04:00:55,279 --> 04:00:58,182
AND WE'RE REALLY HERE FOR THE

5104
04:00:58,182 --> 04:00:59,550
ENTIRE NHLBI COMMUNITY, SO HEART

5105
04:00:59,550 --> 04:01:01,485
LUNG, BLOOD, SLEEP AND 5

5106
04:01:01,485 --> 04:01:03,220
DIFFERENT TRACTS OF TECHNOLOGY,

5107
04:01:03,220 --> 04:01:04,622
I GUESS MOST RELEVANT TO THIS

5108
04:01:04,622 --> 04:01:07,391
TALK WOULD BE THE FIRST 2 ROWS

5109
04:01:07,391 --> 04:01:08,426
OF THE THERAPEUTICS BUT THEN

5110
04:01:08,426 --> 04:01:12,129
ALSO WE DO HAVE SOME COME IN FOR

5111
04:01:12,129 --> 04:01:13,531
ENABLING TECHNOLOGY AND

5112
04:01:13,531 --> 04:01:14,231
TRANSFORMATIVE PLATFORMS I WILL

5113
04:01:14,231 --> 04:01:15,366
WALK YOU THROUGH AND AGAINOT

5114
04:01:15,366 --> 04:01:17,768
LEFT SIDE ARE WHAT WE FOUND

5115
04:01:17,768 --> 04:01:19,503
THROUGH, WHAT WE CALL THE

5116
04:01:19,503 --> 04:01:20,304
PRODUCT DEFINITION, GRANT

5117
04:01:20,304 --> 04:01:21,072
FUNDING PROGRAM AND ON THE RIGHT

5118
04:01:21,072 --> 04:01:22,540
SIDE OF THE BLUE, THE

5119
04:01:22,540 --> 04:01:24,408
PRECLINICAL WE HAVE A STAND

5120
04:01:24,408 --> 04:01:25,242
ALONE PRECLINICAL SERVICES

5121
04:01:25,242 --> 04:01:26,877
PROGRAM TO SUPPORT TARGETED

5122
04:01:26,877 --> 04:01:28,279
ACTIVITIES IN THIS BUCKET TO GET

5123
04:01:28,279 --> 04:01:34,685
YOU TO THE FDA, TO

5124
04:01:34,685 --> 04:01:40,558
[INDISCERNIBLE].

5125
04:01:40,558 --> 04:01:42,860
NEXT SLIDE.

5126
04:01:42,860 --> 04:01:46,197
SO WE HAVE SUITE AS A STAND

5127
04:01:46,197 --> 04:01:47,932
ALONE TECHNOLOGY, AND

5128
04:01:47,932 --> 04:01:58,476
INFORMATIVE PLATFORM, AND PHASE

5129
04:01:58,476 --> 04:02:03,080
2, WE HAVE GENE THERAPY TEAM FOR

5130
04:02:03,080 --> 04:02:03,914
DEVELOPING NEW TARGETING

5131
04:02:03,914 --> 04:02:06,183
VEHICLES FOR THE TEAM DELIVERY

5132
04:02:06,183 --> 04:02:08,586
FOR EXAMPLE, IN THE MIDDLE, THIS

5133
04:02:08,586 --> 04:02:10,855
TALK OR THE SMALL MOLECULE

5134
04:02:10,855 --> 04:02:14,125
BIOLOGICS SO WE HAVE A BI-PHASIC

5135
04:02:14,125 --> 04:02:17,728
R33 AND THIS IS FOR SMALL

5136
04:02:17,728 --> 04:02:18,395
MOLECULES, BIOLOGICS, GENE

5137
04:02:18,395 --> 04:02:20,030
THERAPY AND ALSO THIS NEW THIS

5138
04:02:20,030 --> 04:02:21,999
YEAR AND NEW IN ROUND ARE FOR

5139
04:02:21,999 --> 04:02:23,634
ACCOMMODATION PROD UBTS THAT

5140
04:02:23,634 --> 04:02:25,469
HAVE A COMPONENT AND THEN FOR A

5141
04:02:25,469 --> 04:02:27,138
SLIGHTLY LATER STAGE PROJECTS WE

5142
04:02:27,138 --> 04:02:29,607
HAVE A STAND ALONE R33 PROGRAM.

5143
04:02:29,607 --> 04:02:32,643
ALL OF THE DIRECT COSTS ARE

5144
04:02:32,643 --> 04:02:34,712
LISTED HERE AND AGAIN THE MAIN

5145
04:02:34,712 --> 04:02:35,546
DIFFERENTIATOR BETWEEN THIS

5146
04:02:35,546 --> 04:02:36,514
PROGRAM AND WHAT WAS MENTIONED

5147
04:02:36,514 --> 04:02:41,418
IS THAT WE DON'T HAVE THE SAME

5148
04:02:41,418 --> 04:02:42,686
PERCENT REQUIREMENTS OR THE

5149
04:02:42,686 --> 04:02:44,355
ORGANIZE IEGZ REQUIREMENTS OF

5150
04:02:44,355 --> 04:02:45,089
THE SMALL BUSINESS PROGRAM

5151
04:02:45,089 --> 04:02:47,858
REQUIRES AND WE DO ALLOW YOU TO

5152
04:02:47,858 --> 04:02:52,129
OUTSOURCE MUCH OF YOUR WORK IF

5153
04:02:52,129 --> 04:02:57,034
IT'S JUSTIFIED TO CONTRACT

5154
04:02:57,034 --> 04:02:57,401
ORGANIZATIONS.

5155
04:02:57,401 --> 04:02:58,102
NEXT SLIDE, PLEASE.

5156
04:02:58,102 --> 04:03:00,037
SO BAKED INTO THESE PROJECTS ARE

5157
04:03:00,037 --> 04:03:04,041
A FOCUS ON PROJECT MANAGEMENT,

5158
04:03:04,041 --> 04:03:06,310
MILESTONES AND TIMELINES AND THE

5159
04:03:06,310 --> 04:03:08,245
IMPORTANCE OF HOLLY MENTIONED

5160
04:03:08,245 --> 04:03:09,280
INTELLECTUAL PROPERTY BUT THEN

5161
04:03:09,280 --> 04:03:11,115
ALSO A FOCUS EVEN AT THE

5162
04:03:11,115 --> 04:03:12,082
APPLICATION STAGE THAT YOU MUST

5163
04:03:12,082 --> 04:03:14,585
HAVE SOME SORT OF REGULATORY

5164
04:03:14,585 --> 04:03:15,786
STRATEGY, DEVELOPED AND THAT

5165
04:03:15,786 --> 04:03:18,289
COULD BE AS BASIC AS WE WILL GO,

5166
04:03:18,289 --> 04:03:20,491
THIS IS A DRUG THAT HAS TO GET

5167
04:03:20,491 --> 04:03:23,894
AN IND AND IF YOU HAVE A MORE

5168
04:03:23,894 --> 04:03:25,429
MATURE PROJECT, WE HAVE TO HAVE

5169
04:03:25,429 --> 04:03:30,067
MORE DETAIL ABOUT WHAT THAT

5170
04:03:30,067 --> 04:03:30,501
ENTAILS.

5171
04:03:30,501 --> 04:03:32,870
FOR THE R33, WE HAVE A MATCHING

5172
04:03:32,870 --> 04:03:35,172
FUNDS EXECATION AS WELL AS

5173
04:03:35,172 --> 04:03:38,876
ACRAILTOR PARTNER AND AGAIN THE

5174
04:03:38,876 --> 04:03:40,144
KENTUCKY ORGANIZATION WOULD BE

5175
04:03:40,144 --> 04:03:41,812
AN EXAMPLE OF AN ACCELERATOR

5176
04:03:41,812 --> 04:03:43,113
PARTNER AND THE GOAL OF THIS IS

5177
04:03:43,113 --> 04:03:45,583
TO KIND OF HELP ACADEMIC

5178
04:03:45,583 --> 04:03:47,651
INVESTIGATORS CHANGE THEIR FOCUS

5179
04:03:47,651 --> 04:03:50,287
FROM THAT RO-1 HYPOTHESIS DRIVEN

5180
04:03:50,287 --> 04:03:51,889
RESEARCH TO NOW, WORK TOWARD

5181
04:03:51,889 --> 04:03:53,624
PRODUCT DEVELOPMENT AND GETTING

5182
04:03:53,624 --> 04:03:54,792
THEM FOCUSED ON ACCELERATING

5183
04:03:54,792 --> 04:03:58,529
THIS PROJECT TO BECOME A

5184
04:03:58,529 --> 04:04:02,766
THERAPY.

5185
04:04:02,766 --> 04:04:03,334
NEXT SLIDE.

5186
04:04:03,334 --> 04:04:04,835
SO IN ADDITION TO THE GRANT

5187
04:04:04,835 --> 04:04:06,403
FINISHED BE SITE, WE ARE A

5188
04:04:06,403 --> 04:04:07,338
PRECLINICAL SERVICES PROGRAM

5189
04:04:07,338 --> 04:04:10,975
WITH YOU APPLY THROUGH OUR

5190
04:04:10,975 --> 04:04:12,309
CODING CENTER, RTI, FOR A MORE

5191
04:04:12,309 --> 04:04:14,378
TARGETED POINT TO GET YOU TO

5192
04:04:14,378 --> 04:04:17,147
AIAN POINT TO FINALIZE YOUR YOUR

5193
04:04:17,147 --> 04:04:19,250
IND OR GET YOU DATA TO THEN

5194
04:04:19,250 --> 04:04:21,585
APPLY FOR AN SPWITH IRPHASE 2

5195
04:04:21,585 --> 04:04:22,853
AND REALLY WHERE THE ENTRY POINT

5196
04:04:22,853 --> 04:04:25,189
OF THIS IS FOR THE DRUG OF

5197
04:04:25,189 --> 04:04:26,790
THERAPEUTICS IS, YOU MAY NOT

5198
04:04:26,790 --> 04:04:28,559
HAVE HAD YOUR PREIND YET BUT YOU

5199
04:04:28,559 --> 04:04:30,527
SHOULD HAVE ALL YOUR DATA TO

5200
04:04:30,527 --> 04:04:32,296
SUPPORT A PREIND AND WE CAN

5201
04:04:32,296 --> 04:04:34,398
SUPPORT THE PREIND THROUGH OUR

5202
04:04:34,398 --> 04:04:35,766
REGULATORY SERVICES AND THEN

5203
04:04:35,766 --> 04:04:37,935
ONCE THAT'S COMPLETE, WE WILL

5204
04:04:37,935 --> 04:04:40,938
INVEST IN THOSE SCALE TO THE GMP

5205
04:04:40,938 --> 04:04:45,943
QUALITY MATERIALS FOR THE IND

5206
04:04:45,943 --> 04:04:46,510
ENABLING TECHNOLOGIES.

5207
04:04:46,510 --> 04:04:48,913
AND AGAIN WE'RE NOT SUPPORTING

5208
04:04:48,913 --> 04:04:49,880
THE ENTIRE PRECLINICAL PROGRAM,

5209
04:04:49,880 --> 04:04:51,815
WE COME IN AND IDENTIFY KEY

5210
04:04:51,815 --> 04:04:53,751
ACTIVITIES THAT WILL MAKE YOUR

5211
04:04:53,751 --> 04:04:55,152
-- DERISK YOUR TECHNOLOGY AND

5212
04:04:55,152 --> 04:04:56,687
MAKING IT MORE COMPETITIVE FOR

5213
04:04:56,687 --> 04:04:57,621
TRADITIONAL GRANT FUNDING OR IF

5214
04:04:57,621 --> 04:04:59,690
YOU HAVE AN INVESTOR IN MIND WHO

5215
04:04:59,690 --> 04:05:02,660
NEEDS TO SEE CERTAIN DATA IN

5216
04:05:02,660 --> 04:05:03,861
ORDER TO INVEST, WE ALSO

5217
04:05:03,861 --> 04:05:06,363
EVALUATE THOSE TYPE OF

5218
04:05:06,363 --> 04:05:11,435
APPLICATIONS, TOO.

5219
04:05:11,435 --> 04:05:11,869
NEXT SLIDE.

5220
04:05:11,869 --> 04:05:14,705
GREAT SO THIS A PARTNERSHIP

5221
04:05:14,705 --> 04:05:16,807
BETWEEN MYSELF, AND I SIT ACROSS

5222
04:05:16,807 --> 04:05:20,144
ALL THE DIVISIONS OF THE NHLBI

5223
04:05:20,144 --> 04:05:21,979
ISSUES THE PROGRAMMATICS SUBJECT

5224
04:05:21,979 --> 04:05:23,514
MATTER EXPERT WITHIN THE NHLBI

5225
04:05:23,514 --> 04:05:26,350
AND THEN WE ALSO HAVE STEF AND

5226
04:05:26,350 --> 04:05:28,752
HER TEAM IN THE INNOVATION

5227
04:05:28,752 --> 04:05:30,821
OFFICE SUPPORT THESE

5228
04:05:30,821 --> 04:05:34,959
INVESTIGATORS AND THIS IS WITHIN

5229
04:05:34,959 --> 04:05:36,694
PARTNERSHIP WITH THE

5230
04:05:36,694 --> 04:05:38,495
COORDINATING CENTER, THE RTI

5231
04:05:38,495 --> 04:05:40,030
WHICH HAS THE INFRASTRUCTURE TO

5232
04:05:40,030 --> 04:05:41,498
SUPPORT A BROADER ARRAY OF

5233
04:05:41,498 --> 04:05:42,099
TECHNOLOGY TYPES.

5234
04:05:42,099 --> 04:05:50,908
SO WE CAN GO TO THE NEXT LIED.

5235
04:05:50,908 --> 04:05:52,676
AND REALLY THIS IS A TEAM OF

5236
04:05:52,676 --> 04:05:57,881
EXPERTS, EACH  TEAM IS ASSIGNED,

5237
04:05:57,881 --> 04:05:59,984
A PROGRAM MANAGER, OR A EXPERT

5238
04:05:59,984 --> 04:06:01,151
AND THEN HAVE YOU ACCESS WHEN

5239
04:06:01,151 --> 04:06:03,988
YOU NEED IT, AND THEN ALSO WE

5240
04:06:03,988 --> 04:06:06,323
HAVE A THE LARGE ARRAY OF

5241
04:06:06,323 --> 04:06:07,491
INVESTIGATORS, SOME ARE HARD

5242
04:06:07,491 --> 04:06:08,325
CORE ACADEMICS AND THEY KOANT

5243
04:06:08,325 --> 04:06:10,160
WANT TO START A COMPANY, THEY

5244
04:06:10,160 --> 04:06:12,796
JUST WANT THE TECHNOLOGY

5245
04:06:12,796 --> 04:06:14,298
DEVELOPED, WE HAVE SOME

5246
04:06:14,298 --> 04:06:15,366
ACADEMICS WHO WANT TO START A

5247
04:06:15,366 --> 04:06:16,433
BUSINESS BUT THEY DON'T KNOW

5248
04:06:16,433 --> 04:06:20,070
HOW, AND THEN WE HAVE

5249
04:06:20,070 --> 04:06:20,704
START-AUTOPSIES WHO UNDERSTAND

5250
04:06:20,704 --> 04:06:22,139
THE START UP BUT DON'T

5251
04:06:22,139 --> 04:06:23,540
UNDERSTAND THE NEXT STAGES OF

5252
04:06:23,540 --> 04:06:24,808
COMPANY DEVELOPMENT SO WE WORK

5253
04:06:24,808 --> 04:06:29,246
ACROSS THIS FULL GROUP OF TEAMS

5254
04:06:29,246 --> 04:06:30,647
TO HELP THEM MEET THEM WHERE

5255
04:06:30,647 --> 04:06:31,815
THEY'RE AT AND GET THEM TO WHERE

5256
04:06:31,815 --> 04:06:36,954
THEY WANT TO GO AS FAR AS THE

5257
04:06:36,954 --> 04:06:38,222
ENTREPRENEURIAL POSITION GOES.

5258
04:06:38,222 --> 04:06:39,390
AND THE PI IS A GROUP WE BRING

5259
04:06:39,390 --> 04:06:40,924
TO THE STABLE AND THE NEXT SLIDE

5260
04:06:40,924 --> 04:06:42,326
SHOWS WHAT WE -- THE

5261
04:06:42,326 --> 04:06:43,360
EXPECTATIONS AND THE OFFER OF

5262
04:06:43,360 --> 04:06:45,329
WHAT WE PROVIDE TO YOU.

5263
04:06:45,329 --> 04:06:47,398
SO EVEN AS EARLY AS POST PEER

5264
04:06:47,398 --> 04:06:49,600
REVIEW, WE WORK WITH THE

5265
04:06:49,600 --> 04:06:51,101
INVESTIGATORS TO RIGHT SIZE

5266
04:06:51,101 --> 04:06:51,969
MILESTONES, IF THERE'S SOMETHING

5267
04:06:51,969 --> 04:06:53,470
MISSING WE KNOW THE FDA WILL

5268
04:06:53,470 --> 04:06:55,539
WANT DOWN THE ROAD, WE BAKE

5269
04:06:55,539 --> 04:06:57,174
THOSE IN BEFORE WE FUND YOU.

5270
04:06:57,174 --> 04:06:58,142
AT THE KICKOFF MEETING WE

5271
04:06:58,142 --> 04:06:59,043
DEVELOP A PRODUCT DEVELOPMENT

5272
04:06:59,043 --> 04:07:01,578
PLAN WHICH I THINK IS PART OF

5273
04:07:01,578 --> 04:07:03,347
TOMORROW'S TALK, AND THEN, LONG

5274
04:07:03,347 --> 04:07:05,082
YOUR JOURNEY, WE MEET WITH YOU

5275
04:07:05,082 --> 04:07:09,753
QUARTERLY TO GO OVER ANY

5276
04:07:09,753 --> 04:07:10,387
TECHNICAL CHALLENGES, MILESTONE

5277
04:07:10,387 --> 04:07:11,989
REVIEW AND THEN YOU ALSO HAVE

5278
04:07:11,989 --> 04:07:15,359
ACCESS TO OUR TECHNICAL TEAM

5279
04:07:15,359 --> 04:07:17,361
ADHOC TO THEN IF YOU HAVE, YOU

5280
04:07:17,361 --> 04:07:20,197
KNOW, NEED TO SPEAK WITH OUR

5281
04:07:20,197 --> 04:07:23,967
MEDICINAL CHEMIST ABOUT YOUR

5282
04:07:23,967 --> 04:07:24,501
STRATEGY FOR STREAMING

5283
04:07:24,501 --> 04:07:25,969
MOLECULINGS OR IF YOU HAVE AN

5284
04:07:25,969 --> 04:07:27,271
ASSAY THAT'S NOT WORKING OUT WE

5285
04:07:27,271 --> 04:07:29,740
CAN TRY TO HELP TO IDENTIFY A

5286
04:07:29,740 --> 04:07:31,775
STRATEGY TO IDENTIFY AND GET

5287
04:07:31,775 --> 04:07:35,179
AROUND THE RISK THAT YOU'RE

5288
04:07:35,179 --> 04:07:37,181
HAVING.

5289
04:07:37,181 --> 04:07:46,457
NEXT SLIDE.

5290
04:07:46,457 --> 04:07:46,690
PLEASE.

5291
04:07:46,690 --> 04:07:48,792
AND WE ALSO ARE A STAND ALONE

5292
04:07:48,792 --> 04:07:49,726
PROGRAM, SO IF HAVE YOU SOME

5293
04:07:49,726 --> 04:07:51,695
DATA AND YOU WANT A GAP

5294
04:07:51,695 --> 04:07:52,896
ASSESSMENT TO SAY WHAT DO YOU

5295
04:07:52,896 --> 04:07:53,697
THINK I HAVE?

5296
04:07:53,697 --> 04:07:56,533
WHAT DO YOU THINK THE FDA WILL

5297
04:07:56,533 --> 04:07:56,900
NEED?

5298
04:07:56,900 --> 04:07:58,635
AS CARMEN MENTIONED WE CAN HELP

5299
04:07:58,635 --> 04:08:01,238
WITH THAT, AND WE CAN DO GAP

5300
04:08:01,238 --> 04:08:02,973
ASSESSMENT AND WE CAN HELP

5301
04:08:02,973 --> 04:08:06,610
PREPARE THE I90 AND HELP ENGAGE

5302
04:08:06,610 --> 04:08:11,081
WITH THE FDA AND IT'S A FULL

5303
04:08:11,081 --> 04:08:12,249
SPECTRUM OF RESOURCES, YOU NOW

5304
04:08:12,249 --> 04:08:14,551
HAVE TO HAVE CURRENT ACTIVE

5305
04:08:14,551 --> 04:08:18,155
AWARD, HOWEVER, IF YOU HAVE A

5306
04:08:18,155 --> 04:08:19,156
CATALYZE AWARD, REGULATORY

5307
04:08:19,156 --> 04:08:20,824
SERVICES COME AS A PART OF THE

5308
04:08:20,824 --> 04:08:22,693
PACKAGING, YOU DO NOT NEED TO

5309
04:08:22,693 --> 04:08:23,427
APPLY SEPARATELY FROM THAT.

5310
04:08:23,427 --> 04:08:25,829
AND AGAIN THIS IS TO GET YOU TO

5311
04:08:25,829 --> 04:08:27,831
THE IND AND HAND YOU OFF TO

5312
04:08:27,831 --> 04:08:29,133
OTHERS TO TAKE YOU THROUGH THE

5313
04:08:29,133 --> 04:08:33,770
CLINICAL TRIALS BECAUSE WE HAVE

5314
04:08:33,770 --> 04:08:35,506
ADDITIONAL FUNDING FOR THAT

5315
04:08:35,506 --> 04:08:39,409
STAGE OF DEVELOPMENT.

5316
04:08:39,409 --> 04:08:40,544
NEXT SLIDE.

5317
04:08:40,544 --> 04:08:40,777
GREAT.

5318
04:08:40,777 --> 04:08:43,180
SO UPPER LEFT HAND SIDE,

5319
04:08:43,180 --> 04:08:44,781
CATALYZE, THERE IS A SIGN UP

5320
04:08:44,781 --> 04:08:48,719
THERE WHERE YOU CAN GET OUR NEWS

5321
04:08:48,719 --> 04:08:49,386
LETTERS AND E-MAILS.

5322
04:08:49,386 --> 04:08:50,921
AGAIN, REACH OUT TO US IF YOU

5323
04:08:50,921 --> 04:08:52,856
HAVE AN IDEA OR WANT TO GET

5324
04:08:52,856 --> 04:08:54,057
FEEDBACK ON MORE DETAILS OF THE

5325
04:08:54,057 --> 04:08:54,858
PROGRAM.

5326
04:08:54,858 --> 04:08:59,263
WE ALSO HAVE A VERY ROBUST FAQ

5327
04:08:59,263 --> 04:09:01,265
PAGE AND WE ALSO SEND OUT

5328
04:09:01,265 --> 04:09:02,533
QUARTERLY NEWS LETTERS SO IF YOU

5329
04:09:02,533 --> 04:09:03,967
WANT TO GET THOSE, REACH OUT TO

5330
04:09:03,967 --> 04:09:05,602
US OR SIGN UP ON THE WEBSITE.

5331
04:09:05,602 --> 04:09:14,044
I THINK THAT'S MY LAST SLIDE.

5332
04:09:14,044 --> 04:09:14,378
>> AWESOME.

5333
04:09:14,378 --> 04:09:14,912
THANKS SO MUCH MIKE.

5334
04:09:14,912 --> 04:09:16,413
THAT WAS A FANTASTIC OVERVIEW,

5335
04:09:16,413 --> 04:09:18,448
SO MAYBE I COULD INVITE OUR

5336
04:09:18,448 --> 04:09:19,283
SPEAKERS FROM THE SESSION TO

5337
04:09:19,283 --> 04:09:22,152
COME BACK ON CAMERA, AND I THINK

5338
04:09:22,152 --> 04:09:24,021
YOU KNOW JUST TO KIND OF SUM UP

5339
04:09:24,021 --> 04:09:25,556
THAT SESSION, I KNOW WE COVERED

5340
04:09:25,556 --> 04:09:28,192
A LOT OF TERRITORY HERE FROM

5341
04:09:28,192 --> 04:09:29,860
REGULATORY ALL THE WAY UP TO

5342
04:09:29,860 --> 04:09:32,062
GENERATING FUNDING BUT I THINK

5343
04:09:32,062 --> 04:09:32,763
IT UNDERSCORES SOMETHING HOLLY

5344
04:09:32,763 --> 04:09:35,399
BROUGHT UP, THERE ARE A LOT OF

5345
04:09:35,399 --> 04:09:36,033
ONGOING CONCURRENT ACTIVITIES

5346
04:09:36,033 --> 04:09:37,201
THAT HAVE TO HAPPEN DURING THIS

5347
04:09:37,201 --> 04:09:39,036
PHASE IN ORDER TO BE SUCCESSFUL

5348
04:09:39,036 --> 04:09:41,238
AND THEN TRANSITION YOUR IDEA

5349
04:09:41,238 --> 04:09:42,306
INTO AN ASSET GOING FORWARD.

5350
04:09:42,306 --> 04:09:44,708
SO I THINK THAT'S REALLY WHAT

5351
04:09:44,708 --> 04:09:46,009
WE'RE TRYING TO CAPTURE IN THIS

5352
04:09:46,009 --> 04:09:46,210
GROUP.

5353
04:09:46,210 --> 04:09:48,979
SO MAYBE TO KIND OF KICKOFF ANY

5354
04:09:48,979 --> 04:09:50,814
-- FIRST OF ALL TO INVITE

5355
04:09:50,814 --> 04:09:51,882
EVERYBODY TO SEND THEIR

5356
04:09:51,882 --> 04:09:54,952
QUESTIONS TO THE Q&A
LINK,IME

5357
04:09:54,952 --> 04:09:56,587
HAPPY TO SHARE THOSE WITH THE

5358
04:09:56,587 --> 04:10:00,257
SPEAKERS AND ASK THEM, BUT MAYBE

5359
04:10:00,257 --> 04:10:02,593
TO KIND OF KICK THINGS OFF, I

5360
04:10:02,593 --> 04:10:04,328
CAN ASK STEPHAN EXPE MIKE WHAT

5361
04:10:04,328 --> 04:10:05,596
ARE THE ATTRIBUTES YOU'VE SEEN

5362
04:10:05,596 --> 04:10:06,663
FOR THESE DIFFERENT SUBMISSIONS

5363
04:10:06,663 --> 04:10:08,732
THAT ARE LIKE ASSOCIATE WIDE

5364
04:10:08,732 --> 04:10:09,967
HIGH QUALITY SUBMISSIONS.

5365
04:10:09,967 --> 04:10:11,535
I MEAN, WHAT'S THAT PHENOTYPE OR

5366
04:10:11,535 --> 04:10:12,803
ATTRIBUTES THAT YOU'RE LOOKING

5367
04:10:12,803 --> 04:10:15,572
FOR THAT HAVE BEEN HISTORICALLY

5368
04:10:15,572 --> 04:10:23,880
SUCCESSFUL IN THIS SPACE?

5369
04:10:23,880 --> 04:10:24,648
>> YEAH, ABSOLUTELY.

5370
04:10:24,648 --> 04:10:26,149
SOPHISTICATED THERE ARE SEVERAL

5371
04:10:26,149 --> 04:10:27,251
AREAS OF MERITORIOUS

5372
04:10:27,251 --> 04:10:28,485
APPLICATIONS WE SEE THROUGH THE

5373
04:10:28,485 --> 04:10:29,186
SMALL BUSINESS PROGRAM.

5374
04:10:29,186 --> 04:10:30,153
ONE OF THE BIGGEST THINGS I SEE

5375
04:10:30,153 --> 04:10:32,256
THAT I WOULD SAY IS ASSOCIATED

5376
04:10:32,256 --> 04:10:34,091
WITH HIGH SCORING APPLICATIONS

5377
04:10:34,091 --> 04:10:36,627
IS TEAMS THAT DID THEIR HOME

5378
04:10:36,627 --> 04:10:38,161
WORK, ESPECIALLY EVEN IF IT'S AN

5379
04:10:38,161 --> 04:10:39,329
EARLY STAGE COMPANY, OR A NEW

5380
04:10:39,329 --> 04:10:41,198
SPIN OUT FROM AN ACADEMIC

5381
04:10:41,198 --> 04:10:43,133
INSTITUTION, WE WANT YOU TO BE

5382
04:10:43,133 --> 04:10:45,202
COMING IN WITH A GOOD IDEA ABOUT

5383
04:10:45,202 --> 04:10:47,170
WHAT YOUR UNMET MEDICAL NEED IS

5384
04:10:47,170 --> 04:10:49,106
AND HOW YOUR PROPOSED SOLUTION

5385
04:10:49,106 --> 04:10:51,108
WILL MEET IT AND ALSO WHAT'S

5386
04:10:51,108 --> 04:10:52,376
INNOVATIVE ABOUT YOUR PROPOSED

5387
04:10:52,376 --> 04:10:52,709
SOLUTION.

5388
04:10:52,709 --> 04:10:55,012
SO IF YOU HAVE SOMETHING THAT IS

5389
04:10:55,012 --> 04:10:56,413
SIMILAR TO OTHER DEVICES OR

5390
04:10:56,413 --> 04:10:58,181
DRUGS ON THE MARKET, WHAT MAKE

5391
04:10:58,181 --> 04:10:59,916
ITS BETTER?

5392
04:10:59,916 --> 04:11:01,385
IS IT SAFER?

5393
04:11:01,385 --> 04:11:02,286
EASIER TO USE?

5394
04:11:02,286 --> 04:11:03,253
LOWER COST TO MANUFACTURE?

5395
04:11:03,253 --> 04:11:05,455
THOSE ARE THINGS THAT IT'S

5396
04:11:05,455 --> 04:11:06,456
IMPORTANT TO EMPHASIZE.

5397
04:11:06,456 --> 04:11:07,524
YOU KNOW 1 OF THE OTHER THINGS I

5398
04:11:07,524 --> 04:11:11,662
SEE A LOT IS IF YOU ARE USED TO

5399
04:11:11,662 --> 04:11:14,531
WRITING RO-1S, THE THING TO KEEP

5400
04:11:14,531 --> 04:11:16,900
IN MIND WITH THESE PHASE 1 SMALL

5401
04:11:16,900 --> 04:11:19,670
BUSINESS GRANTS IS IT'S A MUCH

5402
04:11:19,670 --> 04:11:21,538
SMALLER GRANT, 1-2 YEARS, A FEW

5403
04:11:21,538 --> 04:11:23,140
THOUSAND THOUSAND DOLLARS, I

5404
04:11:23,140 --> 04:11:25,842
HAVE SEEN PIs GET TOO

5405
04:11:25,842 --> 04:11:27,344
AMBITIOUS AND GET DINGED ON

5406
04:11:27,344 --> 04:11:29,913
REVIEW ON APPROACH.

5407
04:11:29,913 --> 04:11:31,748
SO PHASE 1 IS JUST PROOF OF

5408
04:11:31,748 --> 04:11:32,949
CONCEPT, DON'T PUT TOO MUCH,

5409
04:11:32,949 --> 04:11:34,351
THERE ARE OTHER PHASES FOR THAT

5410
04:11:34,351 --> 04:11:35,719
AND FINALLY NOT HAVING THE

5411
04:11:35,719 --> 04:11:36,453
APPROPRIATE EXPERTISE.

5412
04:11:36,453 --> 04:11:37,921
SO IF YOU DON'T HAVE EXPERTISE

5413
04:11:37,921 --> 04:11:38,955
IN A PARTICULAR AREA THAT'S

5414
04:11:38,955 --> 04:11:40,557
NECESSARY FOR YOUR PROJECT, CALL

5415
04:11:40,557 --> 04:11:43,660
UP 1 OF YOUR COLLABORATORS, FIND

5416
04:11:43,660 --> 04:11:46,730
SOMEONE ELSE TO PROVIDE ADVISORY

5417
04:11:46,730 --> 04:11:48,031
SERVICES, SO IT'S OFTEN, THERE'S

5418
04:11:48,031 --> 04:11:50,133
A BIG FOCUS ON THE TEAM NOT JUST

5419
04:11:50,133 --> 04:11:54,905
THE CONTACT PI FOR THESE TYPES

5420
04:11:54,905 --> 04:11:55,539
OF APPLICATIONS.

5421
04:11:55,539 --> 04:11:56,873
>> IN ADDITION TO ALL
THOSE

5422
04:11:56,873 --> 04:11:58,642
THINGS WHICH ARE TRUE FOR THE

5423
04:11:58,642 --> 04:12:00,277
CATALYZE PROGRAM, HAVING CLEAR

5424
04:12:00,277 --> 04:12:00,977
MEASURABLE MILESTONES FOR

5425
04:12:00,977 --> 04:12:03,613
CATARISE IS REALLY ESSENTIAL SO

5426
04:12:03,613 --> 04:12:06,316
THAT WE UNDERSTAND WHAT YOU

5427
04:12:06,316 --> 04:12:07,517
DETERMINED IS YOUR SUCCESS OR

5428
04:12:07,517 --> 04:12:11,388
FAILURE, AND THEN TIE TO THAT IS

5429
04:12:11,388 --> 04:12:13,457
GETTING OFTEN TIMES THE BEST

5430
04:12:13,457 --> 04:12:14,991
APPLICATIONS THAT I HAVE SPOKEN

5431
04:12:14,991 --> 04:12:16,059
TO US BEFORE HAND SO THEY

5432
04:12:16,059 --> 04:12:17,394
UNDERSTAND WHAT THE EXPECTATIONS

5433
04:12:17,394 --> 04:12:23,266
ARE EVEN BEFORE THE REVIEW

5434
04:12:23,266 --> 04:12:23,767
HAPPENS.

5435
04:12:23,767 --> 04:12:26,136
>> I THINK THAT'S GREAT
FEEDBACK

5436
04:12:26,136 --> 04:12:27,371
THERE.

5437
04:12:27,371 --> 04:12:29,106
YOU KNOW, WHAT ARE SOME MAYBE

5438
04:12:29,106 --> 04:12:30,307
JUST KIND OF WORKING ALONG WITH

5439
04:12:30,307 --> 04:12:32,609
THAT, TOO, WHAT ARE THE TYPICAL

5440
04:12:32,609 --> 04:12:33,777
MISTAKES YOU SEE FOLKS MAKING

5441
04:12:33,777 --> 04:12:34,978
WITH THE SUBMISSION THAT THEY

5442
04:12:34,978 --> 04:12:38,482
SHOULD TRY AND AVOID.

5443
04:12:38,482 --> 04:12:40,751
>> I WILL JUST REITERATE,
A LOT

5444
04:12:40,751 --> 04:12:42,686
OF TEAMS ARE OVERLY AMBITIOUS

5445
04:12:42,686 --> 04:12:46,990
ESPECIALLY IF WE ARE A 3 OR 2

5446
04:12:46,990 --> 04:12:48,125
YEAR PEER PROGRAM, AND SAY

5447
04:12:48,125 --> 04:12:49,626
YOU'RE GOING FROM SCREENING UP

5448
04:12:49,626 --> 04:12:50,827
TO YOUR LEAD MOLECULE ALL THE

5449
04:12:50,827 --> 04:12:54,531
WAY UP TO PREIND, OR IND, YOU

5450
04:12:54,531 --> 04:12:57,868
AGAIN, YOU GOT A LOT OF OVERLY

5451
04:12:57,868 --> 04:12:59,302
AMBITIOUS APPLICATIONS SO HAVING

5452
04:12:59,302 --> 04:13:00,103
REALISTIC TARGETS IN MIND IS

5453
04:13:00,103 --> 04:13:02,372
SOMETHING WE CAN GIVE YOU

5454
04:13:02,372 --> 04:13:03,306
FEEDBACK ON BEFORE HAND BUT IF

5455
04:13:03,306 --> 04:13:05,075
YOU COME IN KIND OF COLD, YOU

5456
04:13:05,075 --> 04:13:06,610
MAY ASSUME THAT THAT'S -- YOU

5457
04:13:06,610 --> 04:13:10,814
CAN ACTUALLY DO IT AND WE

5458
04:13:10,814 --> 04:13:11,848
ACTUALLY TERMINATE AWARDS EARLY

5459
04:13:11,848 --> 04:13:13,383
AND THAT'S 1 OF THE KIND OF

5460
04:13:13,383 --> 04:13:14,785
REOCCURRING PROBLEMS WITH THE 1

5461
04:13:14,785 --> 04:13:15,786
THAT HAVE DETERMINED IS THEY ARE

5462
04:13:15,786 --> 04:13:18,054
ABLE TO MEET THE MILESTONES

5463
04:13:18,054 --> 04:13:21,725
BECAUSE THEY SET THE BAR

5464
04:13:21,725 --> 04:13:22,426
UNREALISTICALLY.

5465
04:13:22,426 --> 04:13:23,593
NYEAH, I DEFINITELY AGREE WITH

5466
04:13:23,593 --> 04:13:26,997
WHAT MIKE SAID, YOU KNOW, A LOT

5467
04:13:26,997 --> 04:13:28,965
OF PEOPLE SEE BEING AMBITIOUS AS

5468
04:13:28,965 --> 04:13:30,534
A GOOD THING AND MANY CASES IT

5469
04:13:30,534 --> 04:13:32,769
IS, BUT NOT FOR THESE TYPES OF

5470
04:13:32,769 --> 04:13:33,036
PROJECTS.

5471
04:13:33,036 --> 04:13:34,171
THE REVIEWERS ARE LOOKING FOR A

5472
04:13:34,171 --> 04:13:35,105
TEAM THAT NOT ONLY KNOW WHAT IS

5473
04:13:35,105 --> 04:13:37,774
THEY WANT TO DO BUT ALSO IS

5474
04:13:37,774 --> 04:13:39,075
REALISTIC ABOUT THEIR

5475
04:13:39,075 --> 04:13:40,010
LIMITATIONS BOG BUDGETAR EXPE

5476
04:13:40,010 --> 04:13:41,845
TIME RELATED ALSO IN TERMS OF

5477
04:13:41,845 --> 04:13:43,013
THE BAND WIDTH OF THE TEAM.

5478
04:13:43,013 --> 04:13:45,449
SOME OF THE OTHER THINGS I SEE

5479
04:13:45,449 --> 04:13:47,884
ARE -- AND THESE ARE MORE

5480
04:13:47,884 --> 04:13:49,186
ADMINISTRATIVE MISTAKES BUT IT

5481
04:13:49,186 --> 04:13:50,320
HAPPENS A LOT.

5482
04:13:50,320 --> 04:13:51,955
SO FOR EXAMPLE, IF YOU'RE DOING

5483
04:13:51,955 --> 04:13:53,657
A HUMAN SUBJECT STUDY, IT'S

5484
04:13:53,657 --> 04:13:54,391
IMPORTANT WHETHER YOU KNOW

5485
04:13:54,391 --> 04:13:56,293
WHETHER IT'S FALLS UNDER THE NIH

5486
04:13:56,293 --> 04:13:57,127
DEFINITION OF A CLINICAL TRIAL

5487
04:13:57,127 --> 04:13:58,528
EMPLOY SO WE ALWAYS RECOMMEND

5488
04:13:58,528 --> 04:13:59,896
REACHING OUT TO I PROGRAM

5489
04:13:59,896 --> 04:14:01,198
OFFICER AT LEAST 1 MONTH IN

5490
04:14:01,198 --> 04:14:02,599
ADVANCE TO DETERMINE YOU A-KNOW

5491
04:14:02,599 --> 04:14:04,234
WHAT YOUR STUDY IS CLASSIFIED AS

5492
04:14:04,234 --> 04:14:05,969
AND THAT YOU'RE APPLYING TO

5493
04:14:05,969 --> 04:14:07,137
RIGHT NOTICE OF FUNDING

5494
04:14:07,137 --> 04:14:07,938
OPPORTUNITY.

5495
04:14:07,938 --> 04:14:09,206
FOR A SMALL BUSINESS GRANTS IN

5496
04:14:09,206 --> 04:14:11,208
PARTICULAR, THEY ARE THE OMNIBUS

5497
04:14:11,208 --> 04:14:12,609
GRANTS ARE SEPARATED INTO

5498
04:14:12,609 --> 04:14:15,245
CLINICAL TRIAL REQUIRED AND

5499
04:14:15,245 --> 04:14:16,713
CLINICAL TRIAL NOT ALLOWED SO

5500
04:14:16,713 --> 04:14:20,183
YOU DON'T WANT YOUR GRANT TO END

5501
04:14:20,183 --> 04:14:21,985
UP AS A MISCLASSIFIED CLINICAL

5502
04:14:21,985 --> 04:14:22,686
TRIAL.

5503
04:14:22,686 --> 04:14:23,653
ANOTHER THING IS IT'S REALLY

5504
04:14:23,653 --> 04:14:24,955
IMPORTANT TO READ THE INTRUKSS

5505
04:14:24,955 --> 04:14:27,390
SO THINGS LIKE THE PAGE LIMITS,

5506
04:14:27,390 --> 04:14:30,494
LOOKING OVER THE SF424, IT'S NOT

5507
04:14:30,494 --> 04:14:32,062
THE MOST INTERESTING DOCUMENT,

5508
04:14:32,062 --> 04:14:33,263
BUT TRUST ME WHEN I SAY THIS IT

5509
04:14:33,263 --> 04:14:34,531
IS 1 YOU WANT TO KEEP ON

5510
04:14:34,531 --> 04:14:36,166
MANDATORY BECAUSE A LOT OF YOUR

5511
04:14:36,166 --> 04:14:37,767
MOST COMMON ANSWERS WILL BE IN

5512
04:14:37,767 --> 04:14:41,404
THAT DOCUMENT.

5513
04:14:41,404 --> 04:14:41,705
>> GREAT.

5514
04:14:41,705 --> 04:14:43,306
I THINK WE WILL JUMP AROUND A

5515
04:14:43,306 --> 04:14:43,473
BIT.

5516
04:14:43,473 --> 04:14:44,941
THE NEXT QUESTION IS FOR KEVIN

5517
04:14:44,941 --> 04:14:47,878
THAT CAME FROM ONLINE HERE.

5518
04:14:47,878 --> 04:14:49,746
HOW DOES 1 MAKE A DECISION

5519
04:14:49,746 --> 04:14:51,681
BETWEEN THE DPI, THE DRUG

5520
04:14:51,681 --> 04:14:53,216
PRODUCT, I ASSUME FORMULATION

5521
04:14:53,216 --> 04:14:55,452
AND AEROSOL OR BOTH?

5522
04:14:55,452 --> 04:14:58,221
AS IT'S COSTLY TO DO DIFFERENT

5523
04:14:58,221 --> 04:14:58,622
FORMULATIONS.

5524
04:14:58,622 --> 04:15:02,359
WHAT DATA WOULD HELP TO MAKE

5525
04:15:02,359 --> 04:15:03,793
THOSE DECISIONS?

5526
04:15:03,793 --> 04:15:05,395
>> WOW, WE COULD SPEND THE
REST

5527
04:15:05,395 --> 04:15:07,931
OF THE DAY ON THIS TOPIC

5528
04:15:07,931 --> 04:15:08,632
ACTUALLY.

5529
04:15:08,632 --> 04:15:09,232
[LAUGHTER]

5530
04:15:09,232 --> 04:15:11,134
I MEAN, IT'S A VERY REAL

5531
04:15:11,134 --> 04:15:12,335
QUESTION AND THE FORMULATION

5532
04:15:12,335 --> 04:15:14,070
WORK IS SO HARD, IT IS SO

5533
04:15:14,070 --> 04:15:18,475
EXPENSIVE, IT CAN BE SO TIME

5534
04:15:18,475 --> 04:15:21,311
CONSUMING AND I DON'T THINK -- I

5535
04:15:21,311 --> 04:15:23,380
DON'T THINK I HAVE A GOOD ANSWER

5536
04:15:23,380 --> 04:15:27,183
THERE OTHER THAN DON'T LET THE

5537
04:15:27,183 --> 04:15:28,451
[INDISCERNIBLE] GOOD THAT VERY

5538
04:15:28,451 --> 04:15:31,788
COMMONLY FORMULATION WORK IS

5539
04:15:31,788 --> 04:15:33,156
NECESSARILY ITERATIVE.

5540
04:15:33,156 --> 04:15:34,591
APPROACHES THAT I'VE WORKED SORT

5541
04:15:34,591 --> 04:15:37,494
OF WITH MYSELF AND CLIENTS ON IS

5542
04:15:37,494 --> 04:15:39,329
INITIALLY DON'T REINVENT THE

5543
04:15:39,329 --> 04:15:39,863
WHEEL.

5544
04:15:39,863 --> 04:15:42,265
IF THERE IS GOOD PRECEDENT OUT

5545
04:15:42,265 --> 04:15:43,567
THERE OF, OH, GREAT, HERE'S

5546
04:15:43,567 --> 04:15:46,770
SOMEBODY THAT HAS USED

5547
04:15:46,770 --> 04:15:47,837
X-FORMULATION BUFFER WITH A

5548
04:15:47,837 --> 04:15:48,772
PRODUCT THAT LOOKS SIMILAR TO

5549
04:15:48,772 --> 04:15:51,074
MINE, THAT WOULD BE A REALLY

5550
04:15:51,074 --> 04:15:52,142
GOOD PLACE TO START AND IF

5551
04:15:52,142 --> 04:15:53,343
YOU'RE GETTING THE PROPERTIES

5552
04:15:53,343 --> 04:15:58,415
THAT YOU NEEDED, YOU ARE GETTING

5553
04:15:58,415 --> 04:15:59,182
REASONABLE REASONABLE PARTICLE

5554
04:15:59,182 --> 04:16:00,850
DISTRIBUTION, IF YOU'RE GETTING

5555
04:16:00,850 --> 04:16:05,855
REASONABLE  PRODUCT IN THE RIGHT

5556
04:16:05,855 --> 04:16:06,189
RANGE, GREAT.

5557
04:16:06,189 --> 04:16:08,992
BUT YOU NEED TO BE PREPARED FOR

5558
04:16:08,992 --> 04:16:10,860
-- YOU TALKED ABOUT CMC, EARLIER

5559
04:16:10,860 --> 04:16:12,162
TODAY, IF YOU'RE FOLKS ARE

5560
04:16:12,162 --> 04:16:13,129
LOOKING AT STABILITY, IF IT

5561
04:16:13,129 --> 04:16:14,297
TURNS OUT THAT STABILITY IS NOT

5562
04:16:14,297 --> 04:16:16,066
GREAT IN YOUR FORMULATION BUFFER

5563
04:16:16,066 --> 04:16:17,133
THAT MIGHT BE A SITUATION WHERE

5564
04:16:17,133 --> 04:16:18,935
YOU NEED TO GO BACK AND LOOK AT

5565
04:16:18,935 --> 04:16:19,869
IT AND UNFORTUNATELY IF THAT'S

5566
04:16:19,869 --> 04:16:24,074
WHAT YOU NEED DO, IT WILL

5567
04:16:24,074 --> 04:16:26,176
REQUIRE RETREADING SOME OF THE

5568
04:16:26,176 --> 04:16:30,680
SAME GROUND , SO NECESSARILY

5569
04:16:30,680 --> 04:16:32,148
PRODUCT DEVELOPMENT IS NOT

5570
04:16:32,148 --> 04:16:34,784
LINEAR IT'S ITERATIVE AND

5571
04:16:34,784 --> 04:16:35,952
UNFORTUNATELY THAT'S THE REALITY

5572
04:16:35,952 --> 04:16:37,454
WE WORK IN.

5573
04:16:37,454 --> 04:16:39,055
AEROSOL PRODUCTS ARE COMPLICATED

5574
04:16:39,055 --> 04:16:40,790
AND EXPENSIVE AND THAT'S THE

5575
04:16:40,790 --> 04:16:42,959
REALITY BUT IT GIVES YOU THE

5576
04:16:42,959 --> 04:16:45,328
ABILITY TO GO AFTER DISEASES

5577
04:16:45,328 --> 04:16:48,398
THAT YOU CAN'T WITH OTHER FORMS

5578
04:16:48,398 --> 04:16:51,768
OF RESPIRAIGZ SO I'M SORRY BUT

5579
04:16:51,768 --> 04:16:52,335
IT'S THE ANSWER.

5580
04:16:52,335 --> 04:16:53,370
>> IT'S A GOOD 1.

5581
04:16:53,370 --> 04:16:56,506
SO OUR NEXT QUESTION IS FOR

5582
04:16:56,506 --> 04:16:56,773
CARMELLA.

5583
04:16:56,773 --> 04:16:57,807
YOU CAN IMAGINE A LOT OF FOLKS

5584
04:16:57,807 --> 04:16:59,809
ON THIS CALL MAY BE INTERESTED

5585
04:16:59,809 --> 04:17:03,713
WITH THEIR FIRST REGULATORY IRPT

5586
04:17:03,713 --> 04:17:05,682
ACTION BETWEEN AN INTERACT

5587
04:17:05,682 --> 04:17:07,217
MEETING AND THEY APPLY AND A LOT

5588
04:17:07,217 --> 04:17:08,985
OF FOLKS GET SHOT DOWN TO HAVE

5589
04:17:08,985 --> 04:17:11,621
THAT MEETING SO MAYBE YOU COULD

5590
04:17:11,621 --> 04:17:12,622
TALK AROUND WHY FOLKS GET SHOT

5591
04:17:12,622 --> 04:17:14,157
DOWN FOR THAT MEETING AND THEN

5592
04:17:14,157 --> 04:17:15,792
NOT TO DESPAIR IN HOW THEY CAN

5593
04:17:15,792 --> 04:17:17,327
KIND OF SHIFT AND WHAT THEIR

5594
04:17:17,327 --> 04:17:21,765
NEXT STEPS ARE GOING FORWARD.

5595
04:17:21,765 --> 04:17:25,235
>> WELL THE INTERACT
MEETING IS

5596
04:17:25,235 --> 04:17:30,874
REALLY -- IT'S GOT STARTED IN

5597
04:17:30,874 --> 04:17:35,578
SELLING GENE THERAPY. 

5598
04:17:35,578 --> 04:17:38,348
AND REALLY LOOKING AT NEW AND

5599
04:17:38,348 --> 04:17:39,282
VERY INNOVATIVE TECHNOLOGIES,

5600
04:17:39,282 --> 04:17:42,786
TECHNOLOGIES WHERE THERE WEREN'T

5601
04:17:42,786 --> 04:17:45,388
GOOD GUIDANCE DOCUMENTS

5602
04:17:45,388 --> 04:17:49,959
AVAILABLE FOR REVIEW AND SO,

5603
04:17:49,959 --> 04:17:53,530
IT'S REALLY TRANSFERRED OVER

5604
04:17:53,530 --> 04:17:56,332
INTO ANYTHING UNDER CDER OR CBER

5605
04:17:56,332 --> 04:18:00,070
NOW, BUT THAT'S WHAT YOU NEED TO

5606
04:18:00,070 --> 04:18:02,505
CONSIDER IS THAT USUALLY IF IT'S

5607
04:18:02,505 --> 04:18:04,708
REALLY NEW, REALLY INMODEL

5608
04:18:04,708 --> 04:18:07,744
CITIZENIVATIVE, REALLY, THERE'S

5609
04:18:07,744 --> 04:18:09,679
NO SPECIFICKIFIEDANCE DOCUMS OUT

5610
04:18:09,679 --> 04:18:12,148
THERE, THEN YOU MIGHT GET SHUT

5611
04:18:12,148 --> 04:18:12,382
DOWN.

5612
04:18:12,382 --> 04:18:14,384
THE OTHER -- I MEAN, OR YOU

5613
04:18:14,384 --> 04:18:17,554
WOULD GET A MEETING.

5614
04:18:17,554 --> 04:18:21,624
THE OTHER THINGS THAT ARE OFTEN

5615
04:18:21,624 --> 04:18:25,862
TIMES SELECTED IF YOU HAVE SAY

5616
04:18:25,862 --> 04:18:29,499
AN ISSUE WITH YOUR ANIMAL MODEL

5617
04:18:29,499 --> 04:18:32,936
OR YOUR END POINT, THEN USUALLY,

5618
04:18:32,936 --> 04:18:35,338
IF THERE'S NOT A GOOD ANIMAL

5619
04:18:35,338 --> 04:18:37,407
MODEL AND YOU'RE COMING UP WITH

5620
04:18:37,407 --> 04:18:39,876
A NEW ANIMAL MODEL OR A NEW

5621
04:18:39,876 --> 04:18:41,144
STRATEGY, THEN USUALLY THOSE ARE

5622
04:18:41,144 --> 04:18:49,819
THE KINDS OF THINGS THAT GET

5623
04:18:49,819 --> 04:18:52,355
SELECTED FOR AN INTERACT

5624
04:18:52,355 --> 04:18:52,889
MEETING.

5625
04:18:52,889 --> 04:18:53,189
HOW'S THAT?

5626
04:18:53,189 --> 04:18:54,524
>> THAT IS I THINK A GREAT
PLACE

5627
04:18:54,524 --> 04:18:56,826
TO START.

5628
04:18:56,826 --> 04:18:57,327
THANKS CARMELLA.

5629
04:18:57,327 --> 04:18:59,028
SO GOING BACK TO SOME OF THE

5630
04:18:59,028 --> 04:19:00,730
QUESTIONS ONLINE, I THINK THE

5631
04:19:00,730 --> 04:19:03,133
NEXT QUESTION'S FOR MIKE, A

5632
04:19:03,133 --> 04:19:07,771
QUESTION HERE IS FOR CATALYZE,

5633
04:19:07,771 --> 04:19:09,739
DOES NHLBI REQUIRE THAT THE

5634
04:19:09,739 --> 04:19:11,708
START-UP HAS ALREADY LICENSED

5635
04:19:11,708 --> 04:19:11,941
THE IP?

5636
04:19:11,941 --> 04:19:13,810
USUALLY THIS IS OWNED BY THE

5637
04:19:13,810 --> 04:19:16,012
UNIVERSITY AND OURS REQUIRES THE

5638
04:19:16,012 --> 04:19:17,947
START-UP COMPANY TO BE QUITE FAR

5639
04:19:17,947 --> 04:19:19,716
ALONG BEFORE THEY WILL LICENSE

5640
04:19:19,716 --> 04:19:21,050
IT TO THEM?

5641
04:19:21,050 --> 04:19:22,418
>> SO, THERE'S NO
REQUIREMENT TO

5642
04:19:22,418 --> 04:19:24,087
HAVE IP AT THE TIME OF

5643
04:19:24,087 --> 04:19:25,789
APPLICATION, WHAT WE'RE LOOKING

5644
04:19:25,789 --> 04:19:29,292
FOR IS INDICATION IN THAT IP

5645
04:19:29,292 --> 04:19:30,693
STRATEGY THAT MAYBE YOU SPOKEN

5646
04:19:30,693 --> 04:19:32,629
TO THE TECH TRANSFER OFFICE OR

5647
04:19:32,629 --> 04:19:34,330
HAVE YOU SOME LINE OF

5648
04:19:34,330 --> 04:19:35,732
COMMUNICATION SO THAT THEY'RE

5649
04:19:35,732 --> 04:19:36,833
AWARE THAT YOU ACTUALLY WANT

5650
04:19:36,833 --> 04:19:38,101
THEIR TECHNOLOGY AND THAT'S WHAT

5651
04:19:38,101 --> 04:19:39,135
WE'RE REALLY LOOKING FOR AND

5652
04:19:39,135 --> 04:19:40,270
THEN ONCE YOU'RE IN THE PROGRAM,

5653
04:19:40,270 --> 04:19:42,238
WE WILL WORK WITH YOU AND INSURE

5654
04:19:42,238 --> 04:19:44,274
THAT THE RIGHT PEOPLE ARE AT THE

5655
04:19:44,274 --> 04:19:45,809
MEETINGS, WHEN WE KIND OF

5656
04:19:45,809 --> 04:19:49,179
DISCUSS THE TECHNOLOGY BEING

5657
04:19:49,179 --> 04:19:49,445
DEVELOPED.

5658
04:19:49,445 --> 04:19:51,147
THE OTHER PART I SHOULD NOTE IS

5659
04:19:51,147 --> 04:19:53,850
THAT IF YOU ARE DOING ANY KIND

5660
04:19:53,850 --> 04:19:55,752
OF RESEARCH ON A -- SOMETHING

5661
04:19:55,752 --> 04:19:57,253
THAT'S IP PROTECTED LIKE A

5662
04:19:57,253 --> 04:19:59,322
COMPOUND FOR EVEN AN OFF LABEL

5663
04:19:59,322 --> 04:20:00,523
USE, WE DO EXPECT YOU TO HAVE A

5664
04:20:00,523 --> 04:20:02,258
LETTER OF SUPPORT FROM THE IP

5665
04:20:02,258 --> 04:20:04,460
HOLDER OF THAT MOLECULE OR

5666
04:20:04,460 --> 04:20:10,900
COMPOUND AT THE TIME OF

5667
04:20:10,900 --> 04:20:11,868
APPLICATION.

5668
04:20:11,868 --> 04:20:12,268
>> THANKS MIKE.

5669
04:20:12,268 --> 04:20:13,336
SO, I THINK THE LAST QUESTION

5670
04:20:13,336 --> 04:20:15,471
WE'RE GOING TO TAKE FOR THE SAKE

5671
04:20:15,471 --> 04:20:17,507
OF TIME WILL PROBABLY BE

5672
04:20:17,507 --> 04:20:19,676
SOMETHING KEVIN AND I MIGHT

5673
04:20:19,676 --> 04:20:21,077
SPLIT BUT I'LL LET HIM TAKE THIS

5674
04:20:21,077 --> 04:20:21,778
1 FIRST.

5675
04:20:21,778 --> 04:20:23,179
THE QUESTION IS CONCERNING A

5676
04:20:23,179 --> 04:20:27,150
PEPTIDE THAT WILL BE

5677
04:20:27,150 --> 04:20:28,785
ADMINISTERED INTRA FECALLY,

5678
04:20:28,785 --> 04:20:30,887
WOULD THE PANELISTS SUGGIEST ANY

5679
04:20:30,887 --> 04:20:33,656
INVITRO OR IN VIVO IMMUNOLOGICAL

5680
04:20:33,656 --> 04:20:36,259
PRECLINICAL TESTING TO EVALUATE

5681
04:20:36,259 --> 04:20:36,492
SAFETY?

5682
04:20:36,492 --> 04:20:41,865
YOU WANT TO TAKE THAT 1 KEVIN?

5683
04:20:41,865 --> 04:20:44,367
>> THAT WAS A VERY
DIFFICULT

5684
04:20:44,367 --> 04:20:47,303
QUESTION TO GET INTO WITHOUT

5685
04:20:47,303 --> 04:20:48,238
KNOWING THE SPECIFIC INDICATION

5686
04:20:48,238 --> 04:20:52,075
THAT -- SO THAT'S REALLY

5687
04:20:52,075 --> 04:20:52,342
IMPORTANT.

5688
04:20:52,342 --> 04:20:56,679
I MEAN REALLY IT AMOUNTS TO A

5689
04:20:56,679 --> 04:20:57,714
RISK BENEFIT ASSESSMENT.

5690
04:20:57,714 --> 04:20:59,482
SO VERY CALMLY, SO INTRA FECAL

5691
04:20:59,482 --> 04:21:01,217
IS AN INTERESTING 1, I CERTAINLY

5692
04:21:01,217 --> 04:21:04,120
HAVE SEEN THE FDA BE ACCEPTING

5693
04:21:04,120 --> 04:21:05,755
OF IND ENABLING PACKAGES THAT

5694
04:21:05,755 --> 04:21:08,024
DID NOT NECESSARILY REQUIRE

5695
04:21:08,024 --> 04:21:10,093
LARGE ANIMAL MODELS IN CONTRAST,

5696
04:21:10,093 --> 04:21:12,061
IF IT'S AN INJECTION COMMONLY,

5697
04:21:12,061 --> 04:21:19,435
YOU WILL NEED A LARGE ANIMAL

5698
04:21:19,435 --> 04:21:21,537
MODEL, SO AND WHAT IS THE

5699
04:21:21,537 --> 04:21:24,474
MECHANISM OF ACTION, HOW DOES IT

5700
04:21:24,474 --> 04:21:26,643
INTERACT WITH, IS THERE

5701
04:21:26,643 --> 04:21:28,044
SUFFICIENT HOMOLOGY THAT

5702
04:21:28,044 --> 04:21:29,112
INTERACTS WITH THE SPECIES

5703
04:21:29,112 --> 04:21:30,647
APPROPRIATELY, THERE ARE A LOT

5704
04:21:30,647 --> 04:21:31,681
OF DIFFERENT CONSIDERATIONS

5705
04:21:31,681 --> 04:21:32,582
AROUND THIS.

5706
04:21:32,582 --> 04:21:37,487
NHEY, KEVIN, I HAVE TO CUT YOU

5707
04:21:37,487 --> 04:21:43,126
OFF THEIR 1 SECOND, I MISSPOKE,

5708
04:21:43,126 --> 04:21:46,496
AND KEVIN, THANK YOU.

5709
04:21:46,496 --> 04:21:48,398
INTRA TRACHEALLY.

5710
04:21:48,398 --> 04:21:49,532
I'M SORRY.

5711
04:21:49,532 --> 04:21:51,200
>> OH, OKAY.

5712
04:21:51,200 --> 04:21:53,836
>> IT MAKES MORE SENSE FOR
OUR

5713
04:21:53,836 --> 04:21:55,972
DISCUSSION. 

5714
04:21:55,972 --> 04:22:00,176
>> THIS 1'S AN
OUTLIAISONNER, SO

5715
04:22:00,176 --> 04:22:02,512
AEROSOL VERSUS INTRA TRACHEAL

5716
04:22:02,512 --> 04:22:03,079
INSTALLATION.

5717
04:22:03,079 --> 04:22:03,379
>> YEAH.

5718
04:22:03,379 --> 04:22:05,648
>> SO THIS IS AN
INTERESTING

5719
04:22:05,648 --> 04:22:07,116
QUESTION, SO INTRA TRACHEAL

5720
04:22:07,116 --> 04:22:10,453
INSTALLATION HAS BEEN USED VERY

5721
04:22:10,453 --> 04:22:13,189
SUCCESSFULLY FOR IN SMALL ANIMAL

5722
04:22:13,189 --> 04:22:14,557
EFFICACY MODELS WHERE YOU'RE

5723
04:22:14,557 --> 04:22:16,192
LOOKING TO SHOW BIOLOGICAL

5724
04:22:16,192 --> 04:22:17,393
ACTIVITY OF YOUR PRODUCT, WHICH

5725
04:22:17,393 --> 04:22:20,029
CAN BE REALLY USEFUL PROOF OF

5726
04:22:20,029 --> 04:22:22,265
PRINCIPLE, GETTING FROM THE --

5727
04:22:22,265 --> 04:22:24,867
EXTRAPOLATING FROM THAT TO AN

5728
04:22:24,867 --> 04:22:26,836
AEROSOLIZED DOSE CAN BE QUITE

5729
04:22:26,836 --> 04:22:27,904
CHALLENGING, SO, I THINK IT

5730
04:22:27,904 --> 04:22:29,672
THINK IT WOULD BE GREAT, VERY

5731
04:22:29,672 --> 04:22:31,975
USEFUL TO DEMON TRAIT EFFICACY.

5732
04:22:31,975 --> 04:22:33,376
THERE WON'T NECESSARILY BE A BIG

5733
04:22:33,376 --> 04:22:36,546
TRANSITION TO MOVE FROM THAT TO

5734
04:22:36,546 --> 04:22:38,614
DEVELOPMENT OF YOUR AEROSOL AND

5735
04:22:38,614 --> 04:22:40,883
COLLECTING APPROPRIATE SAFETY

5736
04:22:40,883 --> 04:22:43,786
DATA IN AN HAN MALFOR WHICH YOU

5737
04:22:43,786 --> 04:22:46,222
CAN DELIVER AEROSOL.

5738
04:22:46,222 --> 04:22:48,157
SO INTRA TRACHEA INSTALLATION,

5739
04:22:48,157 --> 04:22:50,793
IN AND OF ITSELF WILL BE

5740
04:22:50,793 --> 04:22:52,095
DIFFICULT FOR SINGULAR IND

5741
04:22:52,095 --> 04:22:53,930
ENABLING, IT WOULD BE IND

5742
04:22:53,930 --> 04:22:55,798
SUPPORTED AND WOULD NEED TO BE

5743
04:22:55,798 --> 04:22:58,234
PAIRED WITH SOME OTHER STUDY

5744
04:22:58,234 --> 04:23:01,004
SAYS ASSUMING AEROSOL WILL BE

5745
04:23:01,004 --> 04:23:02,038
INTENDED CLINICAL ROUTE.

5746
04:23:02,038 --> 04:23:03,439
>> CARMELA DO YOU HAVE
ANYTHING

5747
04:23:03,439 --> 04:23:13,850
TO ADD TO THAT FROM YOUR

5748
04:23:18,054 --> 04:23:18,287
PERSPECTIVE?

5749
04:23:18,287 --> 04:23:20,256
>> RIGHT, BASICALLY IT'S
WHAT WE

5750
04:23:20,256 --> 04:23:26,295
TRY TO TELL IS TO START OUT

5751
04:23:26,295 --> 04:23:28,131
WITH, YOU KNOW THE ROUTE THAT

5752
04:23:28,131 --> 04:23:29,065
YOU'RE GOING TO GO WITH

5753
04:23:29,065 --> 04:23:37,073
APPROXIMATE YOU   -- IF YOU KNOT

5754
04:23:37,073 --> 04:23:37,673
THAT IS.

5755
04:23:37,673 --> 04:23:39,675
AND WHAT I WANT TO BRING UP IN

5756
04:23:39,675 --> 04:23:41,110
THE PRODUCT SYSTEM THAT REMEMBER

5757
04:23:41,110 --> 04:23:45,815
BY PHASE 2, YOU HAVE TO HAVE

5758
04:23:45,815 --> 04:23:48,651
YOUR DEVICE DESIGN ESTABLISHED

5759
04:23:48,651 --> 04:23:55,191
AND IF YOU ARE GOING TO HAVE A

5760
04:23:55,191 --> 04:23:57,894
NEBUEALIZED PRODUCT, YOU HAVE TO

5761
04:23:57,894 --> 04:24:03,866
INCLUDE THE SPECIFIC NEBULIZER

5762
04:24:03,866 --> 04:24:04,567
IN YOUR LABEL.

5763
04:24:04,567 --> 04:24:04,867
>> GREAT.

5764
04:24:04,867 --> 04:24:06,235
THANK YOU VERY MUCH.

5765
04:24:06,235 --> 04:24:08,037
FOLKS, I REALLY APPRECIATE THE

5766
04:24:08,037 --> 04:24:10,740
PANEL'S FEEDBACK HERE.

5767
04:24:10,740 --> 04:24:11,574
I'M SORRY, COULD PROBABLY KEEP

5768
04:24:11,574 --> 04:24:13,109
GOING BUT TO STAY ON SCHEDULE, I

5769
04:24:13,109 --> 04:24:14,377
THINK WE WILL CALL IT THERE AND

5770
04:24:14,377 --> 04:24:18,114
MAYBE I CAN TURN IT BACK OVER

5771
04:24:18,114 --> 04:24:19,549
TOMORROW FOR OUR TRANSITION TO

5772
04:24:19,549 --> 04:24:22,585
THE NEXT PHASE. 

5773
04:24:22,585 --> 04:24:23,319
THANK YOU.

5774
04:24:23,319 --> 04:24:24,287
NTHANK YOU ALL.

5775
04:24:24,287 --> 04:24:27,723
SO WE HAVE ABOUT 20 MINUTES LEFT

5776
04:24:27,723 --> 04:24:29,492
IN THE AGENDA FOR A NETWORKING

5777
04:24:29,492 --> 04:24:29,725
BREAK.

5778
04:24:29,725 --> 04:24:33,463
AND SO THIS IS AN OPPORTUNITY

5779
04:24:33,463 --> 04:24:34,530
FOR INDIVIDUALS ONLINE TO FEEL

5780
04:24:34,530 --> 04:24:38,668
FREE TO COME ON CAMERA AND CHAT

5781
04:24:38,668 --> 04:24:42,105
WITH ANY OF THE PANELISTS, WHO

5782
04:24:42,105 --> 04:24:43,506
SPOKE TODAY OR ANY OF THE

5783
04:24:43,506 --> 04:24:45,441
INDIVIDUALS IF THE MEETING AND

5784
04:24:45,441 --> 04:24:52,281
THEN WE WILL BEGIN SESSION 3,

5785
04:24:52,281 --> 04:24:53,478
WHICH IS DEFINING THE TARGET

5786
04:24:53,478 --> 04:24:55,842
PROFILE AT PROMPTLY 3:00 P.M.
EASTERN TIME.

5787
04:24:55,842 --> 04:25:00,313
DEFINING THE PRODUCT

5788
04:25:00,313 --> 04:25:03,049
PROFILE AND DR. ZIADY AND

5789
04:25:03,049 --> 04:25:04,217
DR. WEST WILL BE YOUR MODERATORS

5790
04:25:04,217 --> 04:25:07,087
AND I WILL TURN IT OVER TO THEM.

5791
04:25:07,087 --> 04:25:09,322
>> HI, EVERYBODY, JUST A
BIT

5792
04:25:09,322 --> 04:25:11,758
ABOUT MYSELF AND THEN JAMES CAN

5793
04:25:11,758 --> 04:25:12,325
GO.

5794
04:25:12,325 --> 04:25:18,765
I'M A PROPESESSOR AT SEN

5795
04:25:18,765 --> 04:25:19,065
 CINCINNATI

5796
04:25:19,065 --> 04:25:21,167
CHILDREN'S MEDICAL CENTER AND

5797
04:25:21,167 --> 04:25:24,137
HOSPITAL, I'M JUST A Ph.D., I

5798
04:25:24,137 --> 04:25:25,372
HAVE TAKEN QUITE A FEW BUSINESS

5799
04:25:25,372 --> 04:25:27,173
CLASSES AND I WAS ASKED TO DO

5800
04:25:27,173 --> 04:25:32,979
THIS BECAUSE OF MY BACKGROUND IN

5801
04:25:32,979 --> 04:25:34,781
SOME COMMERCIALIZATION

5802
04:25:34,781 --> 04:25:35,815
DEVELOPMENT AND I'VE BEEN

5803
04:25:35,815 --> 04:25:38,585
INVENTOR ON A NUMBER OF PATENTS

5804
04:25:38,585 --> 04:25:41,221
GRANTED AND SOME PROVISIONAL

5805
04:25:41,221 --> 04:25:43,056
PATENTS THAT ARE PENTINE REGIMEN

5806
04:25:43,056 --> 04:25:44,224
NOTHING TECHNOLOGIES THAT WE'VE

5807
04:25:44,224 --> 04:25:46,426
WORKED WITH AT CHILDREN'S, EMORY

5808
04:25:46,426 --> 04:25:47,927
AND CASE WESTERN SO WE'LL CHAT

5809
04:25:47,927 --> 04:25:49,663
ABOUT THAT IN MY LITTLE 10

5810
04:25:49,663 --> 04:25:53,099
MINUTE SPEECH.

5811
04:25:53,099 --> 04:25:56,436
>> THANK YOU AND I'M JAIPS
WEST,

5812
04:25:56,436 --> 04:25:57,671
PROFESSOR AT VANDERBILT.

5813
04:25:57,671 --> 04:25:59,439
I'M FAIRLY SURE I WAS ASKED

5814
04:25:59,439 --> 04:26:01,875
BECAUSE I HAVE GOTTEN A FAIR

5815
04:26:01,875 --> 04:26:03,943
NUMBER OF DRUGS THROUGH FROM THE

5816
04:26:03,943 --> 04:26:05,979
CLINIC TO THE BENCH AND HAD A

5817
04:26:05,979 --> 04:26:07,614
LOT OF INDUSTRY COLLABORATIONS

5818
04:26:07,614 --> 04:26:09,082
SO HAVE A FAIR AMOUNT OF

5819
04:26:09,082 --> 04:26:14,854
EXPERIENCE IN THIS SPACE AS

5820
04:26:14,854 --> 04:26:15,088
WELL.

5821
04:26:15,088 --> 04:26:17,257
>> I THINK THE SESSION WAS
PUT

5822
04:26:17,257 --> 04:26:18,825
TOGETHER PRETTY NICELY.

5823
04:26:18,825 --> 04:26:22,362
IT WILL EXPOSE THE VIEWERS TO A

5824
04:26:22,362 --> 04:26:24,731
MIX OF ENTREPRENEURS THAT HAVE

5825
04:26:24,731 --> 04:26:27,534
ACTUALLY BEEN IN THE SMALL

5826
04:26:27,534 --> 04:26:29,035
BUSINESS PLACE AND YOU KNOW

5827
04:26:29,035 --> 04:26:33,440
BASICALLY WORKED ON TAKING THEIR

5828
04:26:33,440 --> 04:26:35,208
DRUGS TO MARKET AND CREATING

5829
04:26:35,208 --> 04:26:37,110
SMALL COMPANIES AND SORT OF

5830
04:26:37,110 --> 04:26:38,712
BRINGING THAT TO MARKET AND THEN

5831
04:26:38,712 --> 04:26:42,382
WE'LL ALSO HEAR FROM A COUPLE OF

5832
04:26:42,382 --> 04:26:44,751
SPEAKERS ON FUNDING AND SUPPORT

5833
04:26:44,751 --> 04:26:46,252
AND VENTURE CAPITAL SUPPORT FOR

5834
04:26:46,252 --> 04:26:49,189
THIS WORK, SO I THINK OUR FIRST

5835
04:26:49,189 --> 04:26:54,027
TALK WILL BE BY DR. HOLTZMAN

5836
04:26:54,027 --> 04:26:59,332
WHO'S FOUNDER AND PRESIDENT AT

5837
04:26:59,332 --> 04:27:05,171
NUPEAK AND THEN WE WILL HEAR

5838
04:27:05,171 --> 04:27:06,840
FROM ENRIQUE SHIAO, FROM THE

5839
04:27:06,840 --> 04:27:09,909
HEAD OF SEARCH AND EVALUATION

5840
04:27:09,909 --> 04:27:13,346
AND THEN LUCY LIU AT LONG WOOD

5841
04:27:13,346 --> 04:27:17,217
FUND AND THEN WE WILL WRAP UP

5842
04:27:17,217 --> 04:27:23,022
WITH STEPHEN BENOIT FROM MDI

5843
04:27:23,022 --> 04:27:23,389
THERAPEUTICS.

5844
04:27:23,389 --> 04:27:26,659
>> OKAY, I'M ON, I GUESS.

5845
04:27:26,659 --> 04:27:27,961
FIRST LET ME THANK THE

5846
04:27:27,961 --> 04:27:31,030
ORGANIZERS AND THE INTRODUCERS

5847
04:27:31,030 --> 04:27:31,698
FOR THE OPPORTUNITY.

5848
04:27:31,698 --> 04:27:33,700
IT'S BEEN A TERRIFIC MEETING FOR

5849
04:27:33,700 --> 04:27:37,203
ME SO FAR AND I APPRECIATE BEING

5850
04:27:37,203 --> 04:27:37,804
PART OF IT.

5851
04:27:37,804 --> 04:27:39,739
MY TALK'S GOING TO BE A BIT

5852
04:27:39,739 --> 04:27:42,208
DIFFERENT I THINK THAN SOME OF

5853
04:27:42,208 --> 04:27:43,343
THE 1S YOU'VE HEARD PREVIOUSLY

5854
04:27:43,343 --> 04:27:45,445
AND I WILL TRY TO CONVEY THE

5855
04:27:45,445 --> 04:27:47,747
VIEW I HAVE FROM, YOU KNOW OUR

5856
04:27:47,747 --> 04:27:51,484
YOWRNY IF YOU WILL FROM INITIAL

5857
04:27:51,484 --> 04:27:54,687
DISCOVERY TO WHERE WE'RE AT NOW

5858
04:27:54,687 --> 04:27:55,421
WHICH IS ESSENTIALLY PREIND

5859
04:27:55,421 --> 04:27:58,324
STAGE. 

5860
04:27:58,324 --> 04:27:59,325
SO NEXT SLIDE.

5861
04:27:59,325 --> 04:28:00,727
FOR SOMETHING A LITTLE

5862
04:28:00,727 --> 04:28:01,761
DIFFERENT, FIRST I JUST WANT TO

5863
04:28:01,761 --> 04:28:03,263
INTRODUCE WHO I AM AND WHERE I'M

5864
04:28:03,263 --> 04:28:05,231
AT AND THEN I WILL SPEND THE

5865
04:28:05,231 --> 04:28:08,067
MAIORITY OF MY TEAM IN A MORE

5866
04:28:08,067 --> 04:28:09,369
GENERAL MODE TO TRY AND GIVE YOU

5867
04:28:09,369 --> 04:28:12,005
THINGS THAT MIGHT BE HELPFUL.

5868
04:28:12,005 --> 04:28:13,706
SO THE FIRST POINT, I WANT TO

5869
04:28:13,706 --> 04:28:18,211
MAKE IS THIS IS THE WAY I DID IT

5870
04:28:18,211 --> 04:28:19,512
AND THOSE OF YOU IN THIS

5871
04:28:19,512 --> 04:28:21,047
AUDIENCE WHO HAVE A BIT MORE

5872
04:28:21,047 --> 04:28:24,017
YEARS ON THEM WILL REMEMBER

5873
04:28:24,017 --> 04:28:28,488
FRANK SINAT RA, THE ORIGINAL

5874
04:28:28,488 --> 04:28:30,323
HEAD OF THE BOARD WHO HAD THE

5875
04:28:30,323 --> 04:28:32,325
FAMOUS SONG, I DID IT MY WAY AND

5876
04:28:32,325 --> 04:28:33,893
I DID, BUT I WANT TO MAKE MY

5877
04:28:33,893 --> 04:28:36,763
POINT THAT IN SPITE OF ALL THE

5878
04:28:36,763 --> 04:28:37,630
SUGGESTIONS AND RECOMMENDATIONS

5879
04:28:37,630 --> 04:28:38,464
AND TEMPLATES THAT YOU WILL SEE,

5880
04:28:38,464 --> 04:28:42,669
YOU WILL HAVE TO DO IT YOUR WAY.

5881
04:28:42,669 --> 04:28:44,103
EACH PROJECT IS DIFFERENT AND A

5882
04:28:44,103 --> 04:28:45,138
LOT OF THE PERSPECTIVE HERE WILL

5883
04:28:45,138 --> 04:28:46,339
BE BASED ON MY EXPERIENCE BUT

5884
04:28:46,339 --> 04:28:48,842
YOU WILL HAVE TO FIND SOMETHING

5885
04:28:48,842 --> 04:28:50,476
THAT'S MORE TAILORED TO YOUR

5886
04:28:50,476 --> 04:28:51,544
PROJECT, SO IF YOU DON'T

5887
04:28:51,544 --> 04:28:52,912
REMEMBER ANYTHING ELSE I SAID,

5888
04:28:52,912 --> 04:28:54,347
REMEMBER THAT ADVICE IS GREAT,

5889
04:28:54,347 --> 04:28:55,982
BUT IT'S REALLY UP TO YOU TO BE

5890
04:28:55,982 --> 04:29:00,019
THE BEST PERSON TO FIGURE IT

5891
04:29:00,019 --> 04:29:01,354
OUT.

5892
04:29:01,354 --> 04:29:01,821
NEXT SLIDE.

5893
04:29:01,821 --> 04:29:07,193
AND TO DO THAT, YOU WILL NEED A

5894
04:29:07,193 --> 04:29:08,828
TEAM OF PEOPLE AND FIRST TO

5895
04:29:08,828 --> 04:29:11,564
THANK THESE GUYS AND TO SHOW YOU

5896
04:29:11,564 --> 04:29:13,967
THAT THAT EXISTS IN MY CASE.

5897
04:29:13,967 --> 04:29:16,169
I'VE TAKEN A HYBRID APPROACH, SO

5898
04:29:16,169 --> 04:29:17,570
ON THE LEFT SIDE YOU WILL SEE

5899
04:29:17,570 --> 04:29:20,640
THE TEAM AT WASH-U AND ST. LOUIS

5900
04:29:20,640 --> 04:29:22,909
IN THE RIGHT SIDE YOU WILL SEE

5901
04:29:22,909 --> 04:29:24,444
THE TEAM AT NUPEAK THERAPEUTICS

5902
04:29:24,444 --> 04:29:25,945
THAT I STARTED IN 2012 EMPLOY

5903
04:29:25,945 --> 04:29:27,914
YOU CAN SEE THAT BOTH HAVE GROWN

5904
04:29:27,914 --> 04:29:29,215
INTO A VERY EXTENSIVE CAST TO

5905
04:29:29,215 --> 04:29:32,385
COVER WHAT I CALL THE FULL

5906
04:29:32,385 --> 04:29:37,924
SPECTRUM DRUG  DISCOVERY AND

5907
04:29:37,924 --> 04:29:39,192
DEVELOPMENT TO THE PROGRAM.

5908
04:29:39,192 --> 04:29:42,595
SO THE LEFT SIDE OF THE SCREEN

5909
04:29:42,595 --> 04:29:43,296
IS DISCOVERY EMECHANISM AND

5910
04:29:43,296 --> 04:29:44,564
SCIENCE AND THE RIGHT SIDE OF

5911
04:29:44,564 --> 04:29:46,065
THE TEAM IS RESPONSIBLE FOR

5912
04:29:46,065 --> 04:29:47,700
BRINGING IT INTO THE

5913
04:29:47,700 --> 04:29:49,402
COMMERCIALIZATION PROCESS AND

5914
04:29:49,402 --> 04:29:50,570
I'M FAMOUSLY KNOWN FOR NOT

5915
04:29:50,570 --> 04:29:51,771
KNOWING ENOUGH ABOUT EITHER SO

5916
04:29:51,771 --> 04:29:53,907
BOTH SIDES ARE ABLE TO CRITICIZE

5917
04:29:53,907 --> 04:29:57,577
ME ON A PRETTY SIGNIFICANT BASIS

5918
04:29:57,577 --> 04:29:58,978
BUT IN GENERAL, THIS KIND OF

5919
04:29:58,978 --> 04:30:01,047
APPROACH HAS WORKED WELL FOR ME.

5920
04:30:01,047 --> 04:30:02,682
I DON'T RECOMMEND IT TO

5921
04:30:02,682 --> 04:30:04,183
EVERYONE, BUT IT DOES ALLOW YOU

5922
04:30:04,183 --> 04:30:06,920
TO COVER THE FULL GAMUT OF THE

5923
04:30:06,920 --> 04:30:09,956
APPROACHES WHICH HAS BEEN

5924
04:30:09,956 --> 04:30:12,191
HELPFUL.

5925
04:30:12,191 --> 04:30:15,561
NEXT SLIDE. 

5926
04:30:15,561 --> 04:30:16,429
NEXT SLIDE, PLEASE.

5927
04:30:16,429 --> 04:30:17,530
SO JUST 1 WORD ABOUT WHAT I'M

5928
04:30:17,530 --> 04:30:19,732
DOING AND THEN I WILL GO INTO A

5929
04:30:19,732 --> 04:30:20,867
MORE GENERAL MODE.

5930
04:30:20,867 --> 04:30:22,635
SO THIS CARTOON SYMBOLIZES MY

5931
04:30:22,635 --> 04:30:24,370
LIFE OVER THE LAST DECADE, AND

5932
04:30:24,370 --> 04:30:25,672
THE APPROACH THAT WE'VE TAKEN TO

5933
04:30:25,672 --> 04:30:29,409
FIGURE OUT A NEW DRUG FOR

5934
04:30:29,409 --> 04:30:31,444
PREDOMINANTLY COMMON LUNG

5935
04:30:31,444 --> 04:30:34,514
DISEASES AS AMONG COPD AND IT

5936
04:30:34,514 --> 04:30:35,248
JUST DEPICTS THE NOTION THAT I

5937
04:30:35,248 --> 04:30:36,649
WILL MAKE TO YOU SEVERAL TIMES

5938
04:30:36,649 --> 04:30:40,286
THAT WE'VE GONE AFTER NEW

5939
04:30:40,286 --> 04:30:42,255
CELLULAR AND MOLECULAR TRARGETS

5940
04:30:42,255 --> 04:30:44,090
IN EPITHELIAL CELLS AND STRESS

5941
04:30:44,090 --> 04:30:45,491
KINASES AND WE STARTED OFF A

5942
04:30:45,491 --> 04:30:47,527
SCREAM FROM THE LEFT TO RIGHT

5943
04:30:47,527 --> 04:30:48,928
WHERE THERE'S INITIAL INJURY AND

5944
04:30:48,928 --> 04:30:53,566
THEN STEM CELLS ARE REPROGRAMMED

5945
04:30:53,566 --> 04:30:57,103
TO CAUSE GROWTH, EXCESS,

5946
04:30:57,103 --> 04:30:58,037
ACTIVATION, INFLAMMATION, MUCUS

5947
04:30:58,037 --> 04:30:59,672
PRODUCTION WHICH ARE ALL

5948
04:30:59,672 --> 04:31:01,040
HALLMARKS OF CHRONIC LUNG DEC

5949
04:31:01,040 --> 04:31:03,009
AND WE DEVELOPED A SPECIFIC

5950
04:31:03,009 --> 04:31:05,178
KINASE INHIBITOR TO TRY TO DOWN

5951
04:31:05,178 --> 04:31:06,112
REGULATE THAT PROCESS AND

5952
04:31:06,112 --> 04:31:08,648
THEREBY FIX THE DEC.

5953
04:31:08,648 --> 04:31:10,483
SO THAT'S THE PERSPECTIVE I'M

5954
04:31:10,483 --> 04:31:11,784
COMING FROM, SMALL MOLECULE DRUG

5955
04:31:11,784 --> 04:31:13,052
DEVELOPER WORLD BUT I WANT TO

5956
04:31:13,052 --> 04:31:15,221
TAKE WHAT I'VE LEARNED THERE,

5957
04:31:15,221 --> 04:31:16,756
NOT TELL YOU ABOUT ALL THE

5958
04:31:16,756 --> 04:31:17,757
DETAILS OF THIS, COMPARED TO

5959
04:31:17,757 --> 04:31:19,492
SOME OF THE SPEAKERS, BUT TRY TO

5960
04:31:19,492 --> 04:31:23,629
TAKE WHAT I'VE LEARNED THERE AND

5961
04:31:23,629 --> 04:31:25,031
PROVIDE THE PEARLS FROM THAT IN

5962
04:31:25,031 --> 04:31:28,735
A MORE GENERAL CONTEXT.

5963
04:31:28,735 --> 04:31:29,168
NEXT SLIDE.

5964
04:31:29,168 --> 04:31:31,938
SO TO DO THAT, I WILL RUN

5965
04:31:31,938 --> 04:31:33,573
THROUGH PRETTY QUICKLY 5-POINTS

5966
04:31:33,573 --> 04:31:36,843
LISTED HERE, AS WHAT I CALL THE

5967
04:31:36,843 --> 04:31:38,044
BIG PICTURE MILESTONES THAT GOT

5968
04:31:38,044 --> 04:31:39,145
US AND HOPEFULLY CAN GET YOU

5969
04:31:39,145 --> 04:31:41,614
FROM THE BEGINNING TO THE END OF

5970
04:31:41,614 --> 04:31:42,782
THIS PATHWAY.

5971
04:31:42,782 --> 04:31:43,950
SO FIRST WE'LL DISCOVER, YOU

5972
04:31:43,950 --> 04:31:46,052
KNOW THE NEW TARGET, SMALL

5973
04:31:46,052 --> 04:31:49,055
MOLECULE, THEN THE DRUG, THEN

5974
04:31:49,055 --> 04:31:49,922
THE REGULATORY PROCESS, THEN THE

5975
04:31:49,922 --> 04:31:53,459
TRIALS AND THEN THE PAYMENT

5976
04:31:53,459 --> 04:31:53,960
STORY.

5977
04:31:53,960 --> 04:31:56,729
SO INCOME SLIDE. 

5978
04:31:56,729 --> 04:32:00,333
SO WE WILL START WITH FIRST 1

5979
04:32:00,333 --> 04:32:03,403
WHICH IS AS A MENTIONED TO YOU

5980
04:32:03,403 --> 04:32:04,704
WE DID FOR OURSELVES IN THE WAY

5981
04:32:04,704 --> 04:32:06,239
OF STEM CELLS AND KINASES AND

5982
04:32:06,239 --> 04:32:08,608
NOW TRY TO TAKE YOU A LITTLE BIT

5983
04:32:08,608 --> 04:32:12,311
THROUGH SOME OF THE PRIORITIES

5984
04:32:12,311 --> 04:32:13,813
AND HELPFUL GUIDES, HOPEFULLY IN

5985
04:32:13,813 --> 04:32:19,352
DOING THAT FOR YOU ALL.

5986
04:32:19,352 --> 04:32:19,952
NEXT SLIDE.

5987
04:32:19,952 --> 04:32:21,020
NEXT SLIDE. 

5988
04:32:21,020 --> 04:32:23,623
THERE WE GO.

5989
04:32:23,623 --> 04:32:24,991
SO THE FIRST THING TO SAY IN

5990
04:32:24,991 --> 04:32:27,527
GETTING A NEW TARGET IS THAT IT

5991
04:32:27,527 --> 04:32:27,994
SHOULD BE NEW.

5992
04:32:27,994 --> 04:32:32,865
SO I WANT TO EMPHASIZE THAT NOT

5993
04:32:32,865 --> 04:32:34,233
TO DO WHAT'S BEEN DONE BEFORE

5994
04:32:34,233 --> 04:32:36,669
BUT TO DO SOMETHING ENTIRELY

5995
04:32:36,669 --> 04:32:37,537
DIFFERENT AND NOVEL.

5996
04:32:37,537 --> 04:32:39,739
THIS WILL BE PROJECT SPECIFIC,

5997
04:32:39,739 --> 04:32:41,641
AS I MENTIONED WE'RE ON TO STEM

5998
04:32:41,641 --> 04:32:43,676
CELLS AND KINASES BUT IT'S GOING

5999
04:32:43,676 --> 04:32:45,545
TO BE DIFFERENT FOR YOU.

6000
04:32:45,545 --> 04:32:47,013
IN SPITE OF THAT DIFFERENCE

6001
04:32:47,013 --> 04:32:48,948
THERE ARE DIFFERENT TIPS IN

6002
04:32:48,948 --> 04:32:51,017
GENERAL, I FELT TO LOOK AT WHAT

6003
04:32:51,017 --> 04:32:51,918
WE'VE DONE.

6004
04:32:51,918 --> 04:32:55,154
FIRST AS I'M UNDERLINING HERE,

6005
04:32:55,154 --> 04:32:56,522
AIM FOR NOVELTY, PARTICULARLY ON

6006
04:32:56,522 --> 04:32:58,224
THE SCIENCE SIDE BUT ALSO ON THE

6007
04:32:58,224 --> 04:32:59,592
TRANSLATION OF THAT INTO THE

6008
04:32:59,592 --> 04:33:01,427
COMMERCIAL END.

6009
04:33:01,427 --> 04:33:04,597
SO YOU WANT NEW SCIENCE AND A

6010
04:33:04,597 --> 04:33:06,199
NEW INDICATION AS YOUR BEST

6011
04:33:06,199 --> 04:33:08,201
STRATEGY, I THINK TO BE

6012
04:33:08,201 --> 04:33:08,935
INNOVATIVE AND APPEALING AND TO

6013
04:33:08,935 --> 04:33:11,871
MAKE IT TO THE GOALPOST.

6014
04:33:11,871 --> 04:33:14,240
AND 2 OTHER POINTS I WANT TO

6015
04:33:14,240 --> 04:33:16,709
EMPHASIZE HERE, 1 EARLY ON TO

6016
04:33:16,709 --> 04:33:17,977
VALIDATE THESE TARGETS IN

6017
04:33:17,977 --> 04:33:20,480
PRECLINICAL MODELS THAT ARE HIGH

6018
04:33:20,480 --> 04:33:21,747
FIDELITY AND THAT MEANS BEING

6019
04:33:21,747 --> 04:33:24,984
ABLE TO TRACK THE TARGETS,

6020
04:33:24,984 --> 04:33:26,419
APPROPRIATELY AND HOPEFULLY, IN

6021
04:33:26,419 --> 04:33:29,255
MOST CASES KNOCK OUT THE TARGET

6022
04:33:29,255 --> 04:33:30,423
BOTH EXVIVO AND IN VIVO, TO BE

6023
04:33:30,423 --> 04:33:32,825
SURE THAT YOU HAVE THE RIGHT

6024
04:33:32,825 --> 04:33:33,059
TARGET.

6025
04:33:33,059 --> 04:33:37,330
AND I CAN TELL YOU, I KNOW 90

6026
04:33:37,330 --> 04:33:39,298
TIMES OUT OF A HUNDRED, THE

6027
04:33:39,298 --> 04:33:40,700
PROJECTS I FEEL HAVE NOT DONE

6028
04:33:40,700 --> 04:33:42,535
THAT, SO EARLY ON, YOU WANT THAT

6029
04:33:42,535 --> 04:33:44,270
INFORMATION AND THEN SECONDLY,

6030
04:33:44,270 --> 04:33:46,105
ALSO RARELY DONE, TRY TO

6031
04:33:46,105 --> 04:33:48,174
VALIDATE IT ALSO IN THE HUMAN

6032
04:33:48,174 --> 04:33:48,608
MODEL.

6033
04:33:48,608 --> 04:33:51,010
BOTH IN CELLULAR MOLECULAR

6034
04:33:51,010 --> 04:33:52,545
MODELS AND ALSO IN CLINICAL

6035
04:33:52,545 --> 04:33:53,846
SAMPLES THAT ARE RELEVANT TO YOU

6036
04:33:53,846 --> 04:33:55,381
WHAT YOU'RE TRYING TO DO.

6037
04:33:55,381 --> 04:33:57,116
AGAIN, YOU WANT TO TRACK THAT

6038
04:33:57,116 --> 04:33:59,886
CAREFULLY, SO YOU KNOW IF YOUR

6039
04:33:59,886 --> 04:34:02,155
TARGET IS REPRESENTATIVE AND

6040
04:34:02,155 --> 04:34:03,289
FUNCTIONAL AND ALSO WHEN

6041
04:34:03,289 --> 04:34:05,491
POSSIBLE, TRY TO KNOCK IT OUT AT

6042
04:34:05,491 --> 04:34:08,261
LEAST IN A CELL MODEL TO MAKE

6043
04:34:08,261 --> 04:34:15,234
SURE IT'S VALID.

6044
04:34:15,234 --> 04:34:18,437
NEXT SLIDE.

6045
04:34:18,437 --> 04:34:19,205
THERE WE GO.

6046
04:34:19,205 --> 04:34:21,440
SO STEP 2, ONCE YOU HAVE A CELL

6047
04:34:21,440 --> 04:34:22,875
OF TARGET AND INTEREST, THEN OF

6048
04:34:22,875 --> 04:34:25,378
COURSE YOU HAVE TO DISCOVER AND

6049
04:34:25,378 --> 04:34:27,079
OPTIMIZE A DRUG CANDIDATE TO HIT

6050
04:34:27,079 --> 04:34:27,713
THAT TARGET.

6051
04:34:27,713 --> 04:34:28,281
NEXT SLIDE.

6052
04:34:28,281 --> 04:34:31,083
SO 2 WORDS OF ADVICE HERE, FIRST

6053
04:34:31,083 --> 04:34:32,151
IN THE PRECLINICAL SPACE AND

6054
04:34:32,151 --> 04:34:34,153
THEN IN THE CLENICAL SPACE.

6055
04:34:34,153 --> 04:34:34,854
IN THE PRECLINICAL SPACE AFTER

6056
04:34:34,854 --> 04:34:36,622
YOU HAVE YOUR TARGET, IN THE

6057
04:34:36,622 --> 04:34:38,824
CASE OF SMALL MOLECULES THAT

6058
04:34:38,824 --> 04:34:41,093
MEANS STRUCTURE BASED DRUG

6059
04:34:41,093 --> 04:34:44,564
DESIGN NOWADAYS AIDED BY AI

6060
04:34:44,564 --> 04:34:45,598
METHODS, EDITORIALLY SPEAKING AI

6061
04:34:45,598 --> 04:34:47,066
METHODS ARE NOT SUFFICIENT IN

6062
04:34:47,066 --> 04:34:50,903
THEMSELVES, DON'T BELIEVE THAT

6063
04:34:50,903 --> 04:34:53,139
ADVERTISING, BUT YOU KNOW A LINK

6064
04:34:53,139 --> 04:34:55,074
TO CONVENTIONAL TECHNIQUES CAN

6065
04:34:55,074 --> 04:34:55,775
BE VERY VALUABLE.

6066
04:34:55,775 --> 04:34:57,310
THE END POINTS THERE THAT I

6067
04:34:57,310 --> 04:35:00,580
WANTED TO EMPHASIZE, VERY

6068
04:35:00,580 --> 04:35:03,749
POTENT, VERY SAFE, VERY

6069
04:35:03,749 --> 04:35:05,351
SELECTIVE, AND HOPEFULLY

6070
04:35:05,351 --> 04:35:09,789
DEVELOPED IN A HIGH FIDELITY

6071
04:35:09,789 --> 04:35:10,856
CELLULAR MODEL SYSTEM.

6072
04:35:10,856 --> 04:35:12,225
THOSE PIECES OF INFORMATION WILL

6073
04:35:12,225 --> 04:35:13,626
BE VERY VALUABLE TO YOU AS YOU

6074
04:35:13,626 --> 04:35:15,494
GO THROUGH THE NEXT STEPS I WILL

6075
04:35:15,494 --> 04:35:16,229
SHOW YOU.

6076
04:35:16,229 --> 04:35:17,063
ONCE YOU'VE GOTTEN THROUGH THAT,

6077
04:35:17,063 --> 04:35:21,267
YOU NEED TO MAKE IT PROPERLY,

6078
04:35:21,267 --> 04:35:22,535
FORMULATE IT PROPERLY, AGAIN

6079
04:35:22,535 --> 04:35:24,503
THINKING ABOUT SAFETY AND

6080
04:35:24,503 --> 04:35:26,205
SPECIFICITY AND SELECTIVITY AS

6081
04:35:26,205 --> 04:35:29,075
KEY END POINTS TO SUCCESS.

6082
04:35:29,075 --> 04:35:32,111
THIRDLY, IN THIS PROCESS, TALKED

6083
04:35:32,111 --> 04:35:33,646
A LITTLE BIT ABOUT THIS BUT YOU

6084
04:35:33,646 --> 04:35:36,382
WANT A MECHANISM OF ACTION, IT'S

6085
04:35:36,382 --> 04:35:37,650
UNABLE HOW MANY DRUG CANS GO

6086
04:35:37,650 --> 04:35:39,085
FORWARD WITH NO KNOWLEDGE OF

6087
04:35:39,085 --> 04:35:39,352
MECHANISM.

6088
04:35:39,352 --> 04:35:41,387
YOU WANT INSTEAD TO HAVE A

6089
04:35:41,387 --> 04:35:42,922
MECHANISM THAT SHOWS POTENCY,

6090
04:35:42,922 --> 04:35:44,824
AGAIN, SAFETY, AND AGAIN,

6091
04:35:44,824 --> 04:35:45,691
SCIENTIFIC NOVELTY.

6092
04:35:45,691 --> 04:35:47,526
THAT WILL ALL SERVICE YOU VERY

6093
04:35:47,526 --> 04:35:49,595
WELL AS YOU ANY THROUGH THIS

6094
04:35:49,595 --> 04:35:50,396
PROCESS.

6095
04:35:50,396 --> 04:35:51,697
AND THEN LASTLY THE END POINT OF

6096
04:35:51,697 --> 04:35:53,666
THIS IS PROOF OF CONCEPT AND

6097
04:35:53,666 --> 04:35:54,500
PRECLINICAL AND CLENICAL

6098
04:35:54,500 --> 04:35:59,171
GUIDANCE AND ONCE AGAIN, I WOULD

6099
04:35:59,171 --> 04:36:00,539
EMPHASIZE, POTENCY, SAFETY,

6100
04:36:00,539 --> 04:36:02,141
VALIDATE YOUR TARGET, MAKE SURE

6101
04:36:02,141 --> 04:36:05,044
THAT IT'S HIT, ENGAGED AND

6102
04:36:05,044 --> 04:36:06,912
BLOCKED AND ALL THAT IS BEST

6103
04:36:06,912 --> 04:36:10,283
DONE IN A HIGH FIDELITY

6104
04:36:10,283 --> 04:36:10,816
PRECLINICAL ANIMAL MODEL.

6105
04:36:10,816 --> 04:36:11,951
THERE AGAIN A LOT OF MILES USED

6106
04:36:11,951 --> 04:36:14,387
THAT ARE NOT WHAT I WOULD CALL

6107
04:36:14,387 --> 04:36:17,690
HIGH FIDELITY TO THE CLINICAL

6108
04:36:17,690 --> 04:36:19,058
CONDITION.

6109
04:36:19,058 --> 04:36:21,927
NEXT SLIDE.

6110
04:36:21,927 --> 04:36:25,264
SO THAT'S THE CLINICAL END, THE

6111
04:36:25,264 --> 04:36:26,932
PRECLINICAL PART TO THIS STORY,

6112
04:36:26,932 --> 04:36:28,200
VERY SIMILAR TO WHAT I SHUOF THE

6113
04:36:28,200 --> 04:36:29,702
SHOWED YOU.

6114
04:36:29,702 --> 04:36:30,970
AGAIN, DATA PROJECT SPECIFIC,

6115
04:36:30,970 --> 04:36:33,372
JUST AS OTHER DATA BUT AGAIN,

6116
04:36:33,372 --> 04:36:34,607
GENERAL GUIDE LINES THAT COULD

6117
04:36:34,607 --> 04:36:37,076
BE HELPFUL EMPLOY ONCE IMEAN, I

6118
04:36:37,076 --> 04:36:39,245
EMPHASIZE 3 THINGS, FIRST AGAIN

6119
04:36:39,245 --> 04:36:41,113
AIM FOR NOVELTY IN PARTICULAR

6120
04:36:41,113 --> 04:36:42,715
RELEVANCE, YOU KNOW THE

6121
04:36:42,715 --> 04:36:45,017
INDICATION THAT YOU'RE WORKING

6122
04:36:45,017 --> 04:36:47,153
TOWARDS, I THINK THE NOVEL

6123
04:36:47,153 --> 04:36:52,925
ASPECT IS EXTREMELY USEFUL FOR

6124
04:36:52,925 --> 04:36:54,193
MANY REASONS, AS WE'LL ROLL OUT

6125
04:36:54,193 --> 04:36:56,162
HERE IN THE NEXT FEW SLIDES.

6126
04:36:56,162 --> 04:36:57,663
AGAIN, I THINK IT'S IMPORTANT TO

6127
04:36:57,663 --> 04:36:59,365
VALIDATE WHAT YOU FIND IN

6128
04:36:59,365 --> 04:37:00,032
CLINICAL SAMPLES.

6129
04:37:00,032 --> 04:37:03,235
MAKE SURE THE TARGET'S THERE,

6130
04:37:03,235 --> 04:37:04,036
IT'S UPREGULATED, POTENTIALLY IN

6131
04:37:04,036 --> 04:37:07,073
YOUR DISEASE AND ALSO, I REALLY

6132
04:37:07,073 --> 04:37:09,241
WANT TO EMPHASIZE IS THAT WHEN

6133
04:37:09,241 --> 04:37:11,110
YOU'RE DOING THIS, CONSIDER THIS

6134
04:37:11,110 --> 04:37:12,878
AS EXTENDING IT INTO THE

6135
04:37:12,878 --> 04:37:14,246
DEVELOPMENT OF BIOMARKERS AND

6136
04:37:14,246 --> 04:37:16,115
YOU'LL NEED THOSE BIOMARKERS FOR

6137
04:37:16,115 --> 04:37:18,617
PATIENT SELECTION AND MONITORING

6138
04:37:18,617 --> 04:37:20,686
FOR PROOF OF CONCEPT AND EARLY

6139
04:37:20,686 --> 04:37:23,322
CLINICAL PHASE TRIALS, AND TO

6140
04:37:23,322 --> 04:37:25,524
DEMONSTRATE ONCE AGAIN THAT YOU

6141
04:37:25,524 --> 04:37:26,926
HAVE TARGET ENGAGEMENT AND

6142
04:37:26,926 --> 04:37:28,561
BLOCKADE OR PROMOTION, OR

6143
04:37:28,561 --> 04:37:29,362
WHATEVER THE END POINT HAPPENED

6144
04:37:29,362 --> 04:37:31,364
TO BE BUT THOSE BIOMARKERS AS I

6145
04:37:31,364 --> 04:37:32,898
WILL SHOW YOU IN JUST A SECOND,

6146
04:37:32,898 --> 04:37:34,533
CAN BE VERY USEFUL IN DESIGNING

6147
04:37:34,533 --> 04:37:35,401
THE CLINICAL TRIAL AND FOR

6148
04:37:35,401 --> 04:37:39,338
PROVING THAT YOU HAVE SOMETHING

6149
04:37:39,338 --> 04:37:40,740
USEFUL.

6150
04:37:40,740 --> 04:37:41,841
NEXT SLIDE.

6151
04:37:41,841 --> 04:37:43,142
SO AFTER DISCOVERING THE TARGET

6152
04:37:43,142 --> 04:37:45,845
AND DISCOVERING THE DRUG TO

6153
04:37:45,845 --> 04:37:47,012
BLOCK IT, YOU GET THE

6154
04:37:47,012 --> 04:37:49,348
OPPORTUNITY OF RUNNING THROUGH

6155
04:37:49,348 --> 04:37:50,850
THE MANUFACTURING AND SAFETY

6156
04:37:50,850 --> 04:37:52,852
WHAT I CALL GAUNTLET TO IND

6157
04:37:52,852 --> 04:37:54,854
APPROVAL AND PART OF THAT IND

6158
04:37:54,854 --> 04:37:56,689
PROCESS WE'LL DISCUSS IN JUST A

6159
04:37:56,689 --> 04:37:58,457
SECOND IS DESIGNING AND THEN  

6160
04:37:58,457 --> 04:38:02,294
PERFORMING YOUR CLINICAL TRIALS.

6161
04:38:02,294 --> 04:38:02,862
NEXT SLIDE.

6162
04:38:02,862 --> 04:38:06,132
SO HERE I JUST TRIED TO DISTILL

6163
04:38:06,132 --> 04:38:08,234
DOWN AS I CHECK LIST.

6164
04:38:08,234 --> 04:38:10,169
IT WAS DIFFICULT FOR ME TO GET

6165
04:38:10,169 --> 04:38:11,904
THIS LIST IN ORDER OVER THE

6166
04:38:11,904 --> 04:38:13,406
YEARS AND NOW I WOULD LIKE TO

6167
04:38:13,406 --> 04:38:15,408
SHARE IT WITH THE WORLD IN TERMS

6168
04:38:15,408 --> 04:38:16,809
OF DOING THE MANUFACTURING AND

6169
04:38:16,809 --> 04:38:17,443
GETTING THE SAFETY DONE.

6170
04:38:17,443 --> 04:38:19,044
I KNOW SOME OF THIS HAS BEEN

6171
04:38:19,044 --> 04:38:21,680
TALKED ABOUT ALREADY IN TERMS OF

6172
04:38:21,680 --> 04:38:24,383
THE IND PROCESS, BUT IN SIMPLE

6173
04:38:24,383 --> 04:38:26,752
TERMS, CMC FIRST OF ALL, YOU'RE

6174
04:38:26,752 --> 04:38:28,687
GOING TO WAIT ON MANUFACTURE,

6175
04:38:28,687 --> 04:38:32,558
YOU'LL GOING TO WAIT ON GMP AND

6176
04:38:32,558 --> 04:38:38,597
JOHN GMP, SO YOU CAN DO GLP AND

6177
04:38:38,597 --> 04:38:41,567
NONGLP TRIALS, BE CAREFUL HOW

6178
04:38:41,567 --> 04:38:43,903
YOU DO THIS AND CHECK WITH

6179
04:38:43,903 --> 04:38:46,205
EXPERTS BEFORE, BECAUSE IT'S A

6180
04:38:46,205 --> 04:38:47,573
TRICKY BUSINESS COMING FROM 1

6181
04:38:47,573 --> 04:38:48,307
ACADEMIC BACKGROUND AND THEN YOU

6182
04:38:48,307 --> 04:38:50,509
WANT TO GO ON AND DO THE

6183
04:38:50,509 --> 04:38:53,179
TOXICOLOGY, BOTH INVITRO AND IN

6184
04:38:53,179 --> 04:38:54,914
VIVO, THIS HASN'T BEEN SAID

6185
04:38:54,914 --> 04:38:56,749
BEFORE, THE INVITRO IS USEFUL

6186
04:38:56,749 --> 04:38:57,917
RELATIVELY CHEAP AND FOOF THE

6187
04:38:57,917 --> 04:39:01,454
AND INFORMATIVE AND THEN YOU

6188
04:39:01,454 --> 04:39:07,259
MOVE TO TOX IN VIVO USING ANIMAL

6189
04:39:07,259 --> 04:39:11,564
MODEL, USUALLY 2 SPECIES, SO

6190
04:39:11,564 --> 04:39:14,133
MTD-DRF, IN 2 SPECS, RAT AND DOG

6191
04:39:14,133 --> 04:39:20,105
IS THE GO TO, AND THEN YOU MOVE

6192
04:39:20,105 --> 04:39:22,341
TO GLP TO TOCKSITY TO WRAP UP

6193
04:39:22,341 --> 04:39:23,709
WHAT THE FDA WILL WANT.

6194
04:39:23,709 --> 04:39:24,944
I JUST WANT TO EMPHASIZE HERE --

6195
04:39:24,944 --> 04:39:28,814
OOPS CAN YOU GO BACK.

6196
04:39:28,814 --> 04:39:30,349
GO -- OKAY, WHERE ARE WE GOING.

6197
04:39:30,349 --> 04:39:33,552
SO JUST TO EMPHASIZE HERE, THE

6198
04:39:33,552 --> 04:39:35,754
PK ANALYSIS I BELIEVE IS VERY

6199
04:39:35,754 --> 04:39:36,856
USEFUL UNDER THESE CONDITIONS,

6200
04:39:36,856 --> 04:39:38,491
YOU KNOW TO DETERMINE WHAT KIND

6201
04:39:38,491 --> 04:39:40,326
OF DOZING, WHEN YOU DOSE, HOW

6202
04:39:40,326 --> 04:39:41,760
YOU DOSE, HOW MUCH YOU DOSE, AND

6203
04:39:41,760 --> 04:39:44,864
A LOT OF TIMES PEOPLE JUMP INTO

6204
04:39:44,864 --> 04:39:46,031
TOXICITY WITHOUT HAVING THE FULL

6205
04:39:46,031 --> 04:39:48,767
KNOWLEDGE OF THAT DATA IN A FULL

6206
04:39:48,767 --> 04:39:49,602
RAINCHL OF SPECIES IN OUR CASE

6207
04:39:49,602 --> 04:39:52,571
AND I BELIEVE IN OTHERS THAT

6208
04:39:52,571 --> 04:39:55,174
WILL BE VERY USEFUL TO YOU AS

6209
04:39:55,174 --> 04:39:56,475
YOU MARCH THROUGH DESIGN HOW TO

6210
04:39:56,475 --> 04:39:57,643
SET UP THOSE STUDIES TO MAKE

6211
04:39:57,643 --> 04:39:59,345
SURE YOU DON'T NEED TO RUN THEM

6212
04:39:59,345 --> 04:40:00,980
2 AND 3 TIMES.

6213
04:40:00,980 --> 04:40:02,381
AND THE OTHER THING I WANT TO

6214
04:40:02,381 --> 04:40:03,449
EMPHASIZE ALTHOUGH I THINK THIS

6215
04:40:03,449 --> 04:40:04,517
WAS TALKED ABOUT SIGNIFICANTLY

6216
04:40:04,517 --> 04:40:07,052
IN THE LAST SESSION IS THE RULES

6217
04:40:07,052 --> 04:40:10,856
DIFFER, THIS LIST CHANGES FOR

6218
04:40:10,856 --> 04:40:11,824
INHALED VERSUS SYSTEMIC DELIVERY

6219
04:40:11,824 --> 04:40:15,327
AND WE WON'T HAVE THE SAME TO

6220
04:40:15,327 --> 04:40:17,162
DISCUSS THAT EXTENSIVELY, BUT

6221
04:40:17,162 --> 04:40:18,931
THE INHALED IS A TREMENDOUS

6222
04:40:18,931 --> 04:40:20,199
ADVANTAGE IF YOU CAN TAKE IT BUT

6223
04:40:20,199 --> 04:40:24,169
IT DOES HAVE A DIFFERENT SET OF

6224
04:40:24,169 --> 04:40:26,539
PROBLEMS AND ISSUES COMPARED TO

6225
04:40:26,539 --> 04:40:32,244
SYSTEMIC DELIVERY.

6226
04:40:32,244 --> 04:40:32,978
NEXT SLIDE.

6227
04:40:32,978 --> 04:40:35,281
AND THEN, YOU WANT TO TAKE ALL

6228
04:40:35,281 --> 04:40:38,984
OF THAT INFORMATION ON YOUR

6229
04:40:38,984 --> 04:40:40,986
PHARMACOLOGY MANUFACTURING AND

6230
04:40:40,986 --> 04:40:43,188
INTOXICOLOGY AND RUN THROUGH IN

6231
04:40:43,188 --> 04:40:44,790
TERMS OF THE SPECTRUM WE'RE

6232
04:40:44,790 --> 04:40:46,392
LOOKING AT TODAY WHAT I WOULD

6233
04:40:46,392 --> 04:40:51,430
CONSIDER PHASE 1 A AND B AND 2 A

6234
04:40:51,430 --> 04:40:54,300
CLINICAL TRIALS.

6235
04:40:54,300 --> 04:40:54,867
NEXT SLIDE.

6236
04:40:54,867 --> 04:40:58,170
SO SOME OF THIS YOU ALREADY

6237
04:40:58,170 --> 04:41:02,141
HEARD BUT PHASE 1 A AND B ARE

6238
04:41:02,141 --> 04:41:03,042
ASCENDING, MULTILE ASCENDING

6239
04:41:03,042 --> 04:41:04,677
DOSE TRIALS AND PHASE 2 IS THE

6240
04:41:04,677 --> 04:41:06,178
SAIOF THE AND EFFICACY TRIAL.

6241
04:41:06,178 --> 04:41:07,279
I WANT TO EMPHASIZE COUPLE

6242
04:41:07,279 --> 04:41:09,181
THINGS HERE WHERE I THINK A LOT

6243
04:41:09,181 --> 04:41:11,750
OF PROJECT VS RUN INTO TROUBLE.

6244
04:41:11,750 --> 04:41:14,887
SO FIRST IN THIS PHASE 1 AND

6245
04:41:14,887 --> 04:41:16,722
PHASE B FREQUENTLY DESIGNED FOR

6246
04:41:16,722 --> 04:41:18,757
SAFETY, BUT IT'S AN OPPORTUNITY

6247
04:41:18,757 --> 04:41:20,693
EARLY ON TO INCLUDE A PATIENT

6248
04:41:20,693 --> 04:41:22,695
ARM FOR AN EARLY READ OUT BOTH A

6249
04:41:22,695 --> 04:41:24,396
AND B.

6250
04:41:24,396 --> 04:41:25,564
AND THAT'S TERRIFICALLY VALUABLE

6251
04:41:25,564 --> 04:41:27,866
BECAUSE IT CAN GIVE YOU FIRST OF

6252
04:41:27,866 --> 04:41:29,535
ALL SOME EVIDENCE THAT YOU ARE

6253
04:41:29,535 --> 04:41:31,470
HITTING YOUR TARGET, YOUR

6254
04:41:31,470 --> 04:41:32,871
TARGET'S ENGAGED AND BLOCKED AND

6255
04:41:32,871 --> 04:41:34,840
ALSO IF YOU'RE REALLY LUCKY AND

6256
04:41:34,840 --> 04:41:36,909
YOU'VE DONE YOUR BIOMARKERS

6257
04:41:36,909 --> 04:41:38,210
PROPERLY, IT MIGHT GIVE YOU

6258
04:41:38,210 --> 04:41:40,379
EARLY EVIDENT THAT YOUR APPROACH

6259
04:41:40,379 --> 04:41:41,547
IS WORKING.

6260
04:41:41,547 --> 04:41:43,582
SAME THING IN 2A, BE CAREFUL

6261
04:41:43,582 --> 04:41:45,317
ABOUT TRYING TO INCLUDE SOME

6262
04:41:45,317 --> 04:41:46,952
EVIDENCE OF VALIDATION FOR

6263
04:41:46,952 --> 04:41:51,590
THERAPEUTIC EFFECTIVENESS OR AT

6264
04:41:51,590 --> 04:41:52,391
LEAST BIOMARKER ENGAGEMENT.

6265
04:41:52,391 --> 04:41:55,794
SO THE PS HERE IS BE THOUGHTFUL

6266
04:41:55,794 --> 04:41:57,029
ON PATIENT SELECTION.

6267
04:41:57,029 --> 04:41:58,097
THINK ABOUT VALIDATION BASED ON

6268
04:41:58,097 --> 04:42:01,300
THE SCIENCE YOU HAVE AND NOT ON

6269
04:42:01,300 --> 04:42:02,034
CONVENTIONAL, TRADITIONAL MODELS

6270
04:42:02,034 --> 04:42:03,435
THAT HAVE BEEN TRIED OVER AND

6271
04:42:03,435 --> 04:42:04,737
OVER AND HAVE GENERALLY FAILED

6272
04:42:04,737 --> 04:42:08,507
IN THE SAME DEC.

6273
04:42:08,507 --> 04:42:09,274
-- DISEASE.

6274
04:42:09,274 --> 04:42:10,142
NEXT SLIDE.

6275
04:42:10,142 --> 04:42:12,745
LASTLY, BUT NOT LEASTLY OF

6276
04:42:12,745 --> 04:42:15,447
COURSE, IS PAIN FOR ALL THESE

6277
04:42:15,447 --> 04:42:16,982
THING -- PAYING FOR ALL THESE

6278
04:42:16,982 --> 04:42:18,951
THINGS, SINCE THE PRICE IS

6279
04:42:18,951 --> 04:42:20,486
SIGNIFICANTLY HIGHER THAN THE

6280
04:42:20,486 --> 04:42:21,520
KINDS OF USUAL PATHOGENESIS

6281
04:42:21,520 --> 04:42:23,322
RESEARCH THAT YOU DO OR THAT I

6282
04:42:23,322 --> 04:42:27,926
DO IN ACADEMIA.

6283
04:42:27,926 --> 04:42:28,360
NEXT SLIDE.

6284
04:42:28,360 --> 04:42:29,895
SO FIRST THE WAY I DID IT AND

6285
04:42:29,895 --> 04:42:31,063
THEN I WILL JUST TOUCH ON THE

6286
04:42:31,063 --> 04:42:32,731
WAY A LOT OF OTHER IT IS DO IT,

6287
04:42:32,731 --> 04:42:33,799
I'VE GONE THROUGH THE

6288
04:42:33,799 --> 04:42:37,202
GOVERNMENT, SO I DORPHED THE

6289
04:42:37,202 --> 04:42:40,005
TARGET WITH RO-1, COULD ADD R35

6290
04:42:40,005 --> 04:42:41,273
GRANTS, I'VE DISCOVERED THE

6291
04:42:41,273 --> 04:42:42,074
CANDIDATE AGAIN WITH ANOTHER

6292
04:42:42,074 --> 04:42:45,144
SERIES OF GRANT AND THEN

6293
04:42:45,144 --> 04:42:46,378
TRANSITIONED INTO STTR,

6294
04:42:46,378 --> 04:42:49,014
FOUNDATION AND D.O.D. GRAPTS, I

6295
04:42:49,014 --> 04:42:50,649
RUN THE GAUNTLET WITH THOSE

6296
04:42:50,649 --> 04:42:52,184
GRANTS AT A HIGHER LEVEL AND I

6297
04:42:52,184 --> 04:42:53,752
PLAN ON DO THE CLINICAL TRIALS

6298
04:42:53,752 --> 04:42:55,120
WITH THE CLINICAL TRIALS AWARDS

6299
04:42:55,120 --> 04:42:56,422
FROM THE SAME AGENCIES, SO

6300
04:42:56,422 --> 04:42:59,391
THAT'S HOW I DID IT, THAT WAS MY

6301
04:42:59,391 --> 04:43:01,960
WAY BUT THERE'S AS YOU KNOW

6302
04:43:01,960 --> 04:43:05,330
SIGNIFICANT DELAYS, DOWN SIDES

6303
04:43:05,330 --> 04:43:07,066
AND PROBLEMS WITH THAT SOLE

6304
04:43:07,066 --> 04:43:07,800
GOVERNMENT FUNDING APPROACH

6305
04:43:07,800 --> 04:43:09,001
EMPLOY SO THEN THERE'S THE

6306
04:43:09,001 --> 04:43:09,535
PRIVATE WORLD.

6307
04:43:09,535 --> 04:43:10,536
NEXT SLIDE.

6308
04:43:10,536 --> 04:43:13,138
I WILL JUST HAVE 1 SLIDE ON THIS

6309
04:43:13,138 --> 04:43:18,811
FROM WHAT I'VE GLEANED FROM MY

6310
04:43:18,811 --> 04:43:21,680
DISCUSSIONS WITH VARIOUS VCs

6311
04:43:21,680 --> 04:43:23,282
AND PRIVATE INVESTORS, JUST A

6312
04:43:23,282 --> 04:43:24,917
QUICK LIST YOU COULD USE OR NOT

6313
04:43:24,917 --> 04:43:26,785
USE, TO GUIDE YOU HERE, SO FIRST

6314
04:43:26,785 --> 04:43:29,555
THEY'RE LOOKING FOR HIGH

6315
04:43:29,555 --> 04:43:30,823
QUALITY, DIFFERENT TECHNOLOGY,

6316
04:43:30,823 --> 04:43:32,224
ASSETS, HIGH LINE PLATFORM,

6317
04:43:32,224 --> 04:43:33,659
THAT'S ALL YOU GET OUT OF THE

6318
04:43:33,659 --> 04:43:35,627
FIRST COUPLE SLIDES I SHOWED

6319
04:43:35,627 --> 04:43:35,961
YOU.

6320
04:43:35,961 --> 04:43:37,763
THEY WANT COMPELLING DATA, THEY

6321
04:43:37,763 --> 04:43:38,964
WANT NEAR TERM CATALYSTS SO

6322
04:43:38,964 --> 04:43:40,599
THEY'RE IN THERE TO INCREASE

6323
04:43:40,599 --> 04:43:40,799
VALUE.

6324
04:43:40,799 --> 04:43:44,770
EVERYBODY WANTS TO GET TO HELP

6325
04:43:44,770 --> 04:43:46,505
PATIENTS THEY SAY BUT THEY'RE

6326
04:43:46,505 --> 04:43:47,806
ALSO LOOKING FOR CATALYST TO

6327
04:43:47,806 --> 04:43:49,808
RAISE THE VALUE OF YOUR PROJECT.

6328
04:43:49,808 --> 04:43:51,744
ONCE AGAIN, THE TIPS I GAVE YOU

6329
04:43:51,744 --> 04:43:53,245
WILL PROVIDE THAT INFORMATION.

6330
04:43:53,245 --> 04:43:55,347
STRONG IP, BE SURE YOU WORK OUT

6331
04:43:55,347 --> 04:44:00,185
CAREFULLY A MARKET STRATEGY AND

6332
04:44:00,185 --> 04:44:00,486
OPPORTUNITY.

6333
04:44:00,486 --> 04:44:02,387
DEFINITELY HAVE AN EXPERIENCED

6334
04:44:02,387 --> 04:44:03,088
MANAGEMENT TEAM, HOPEFULLY

6335
04:44:03,088 --> 04:44:04,156
PEOPLE WHO HAVE BEEN THERE AND

6336
04:44:04,156 --> 04:44:05,090
DONE THIS BEFORE EMPLOY THEY

6337
04:44:05,090 --> 04:44:06,525
LOVE TO SEE STAGES OF

6338
04:44:06,525 --> 04:44:11,764
DEVELOPMENT, IF YOU'RE NOT A G

6339
04:44:11,764 --> 04:44:14,266
ANTT LOVER ALREADY, YOU WILL

6340
04:44:14,266 --> 04:44:15,567
NEED TO BECOME 1 AND CONVINCE

6341
04:44:15,567 --> 04:44:16,602
PEOPLE OVER AND OVER THAT YOU'RE

6342
04:44:16,602 --> 04:44:18,137
ON TRACK AND THEY CAN TRACK YOU.

6343
04:44:18,137 --> 04:44:22,207
YOU WANT TO HAVE STRATEGIC

6344
04:44:22,207 --> 04:44:24,710
INTERESTS AND IDENTIFIABLE EXITS

6345
04:44:24,710 --> 04:44:26,378
IN ORDER FOR THEM TO SEE THE

6346
04:44:26,378 --> 04:44:27,880
VALUE AND INVESTMENT AND YOU

6347
04:44:27,880 --> 04:44:30,015
WOULD LIKE TO SHOW SOME SUPPORT

6348
04:44:30,015 --> 04:44:32,351
FROM LEADING INVESTORS.

6349
04:44:32,351 --> 04:44:33,852
THESE GUYS MOVE, NO OFFENSE

6350
04:44:33,852 --> 04:44:35,254
INTENDED HERE FOR ANYONE ON THE

6351
04:44:35,254 --> 04:44:39,057
CALL, THEY MOVE AS A HEARD, IT'S

6352
04:44:39,057 --> 04:44:40,425
A PENGUIN MENTALITY, 1

6353
04:44:40,425 --> 04:44:42,728
INVESTMENT FOLLOWS THE NEXT

6354
04:44:42,728 --> 04:44:44,630
INVESTMENT PERSON, SO IT'S GOOD

6355
04:44:44,630 --> 04:44:46,498
FOR YOU TO INCLUDE AT LEAST AS

6356
04:44:46,498 --> 04:44:49,802
BEST YOU CAN, SOME EVIDENCE OF

6357
04:44:49,802 --> 04:44:51,270
SUPPORT IN INVESTORS WHO SEEM TO

6358
04:44:51,270 --> 04:44:55,240
KNOW SOMETHING.

6359
04:44:55,240 --> 04:44:56,809
NEXT SLIDE.

6360
04:44:56,809 --> 04:44:58,477
ONE MORE.

6361
04:44:58,477 --> 04:44:59,178
THANK YOU.

6362
04:44:59,178 --> 04:45:02,181
SO TO RECAP, THAT WAS A LOT, A

6363
04:45:02,181 --> 04:45:03,715
LOT OF MILEAGE, A LOT OF ISSUES,

6364
04:45:03,715 --> 04:45:05,184
A LOT OF DETAILS.

6365
04:45:05,184 --> 04:45:07,419
SO RETURNING BACK TO THE BIG

6366
04:45:07,419 --> 04:45:09,254
PICTURE TO SOME UP, I'VE TRIED

6367
04:45:09,254 --> 04:45:10,989
TO GIVE YOU 5-POINTS HERE ALONG

6368
04:45:10,989 --> 04:45:14,159
THE  JOURNEY THAT ARE KEY TO

6369
04:45:14,159 --> 04:45:16,728
GETTING TO THE GOAL, DORPHING

6370
04:45:16,728 --> 04:45:17,963
NEW CANDIDATE, DISCOVERING NEW

6371
04:45:17,963 --> 04:45:20,699
DRUG FOR THE CANDIDATE,

6372
04:45:20,699 --> 04:45:22,768
TRANSLATING THOSE FINDINGS INTO

6373
04:45:22,768 --> 04:45:24,903
MANUFACTURING AND SAFETY,

6374
04:45:24,903 --> 04:45:26,572
TRANSLATING THAT INFORMATION AND

6375
04:45:26,572 --> 04:45:28,040
ALL OF THAT INFORMATION ACTUALLY

6376
04:45:28,040 --> 04:45:30,209
INTO A PROPERLY DESIGNED PHASE

6377
04:45:30,209 --> 04:45:32,711
1-2 TRIALS AND HAVING SOME CLUES

6378
04:45:32,711 --> 04:45:35,447
AT LEAST INTO HOW TO FUND IT ALL

6379
04:45:35,447 --> 04:45:36,715
IN AN EFFICIENT WAY.

6380
04:45:36,715 --> 04:45:38,684
AND I WILL STOP THERE AND TURN

6381
04:45:38,684 --> 04:45:46,859
IT OVER TO THE NEXT PERSON. 

6382
04:45:46,859 --> 04:45:50,696
THANK YOU. 

6383
04:45:50,696 --> 04:45:51,496
>> THANKS DR. HOLTZMAN.

6384
04:45:51,496 --> 04:45:55,100
NEXT WE WILL HAVE D

6385
04:45:55,100 --> 04:45:56,935
R. ENRIQUE SHIAO WHO HEADS THE

6386
04:45:56,935 --> 04:45:58,203
SEARCH AND EVALUATION VENTURE

6387
04:45:58,203 --> 04:45:59,838
GROUP AT THE CF FOUNDATION WHICH

6388
04:45:59,838 --> 04:46:02,574
HAS BEEN LOOKING INTO 1 TIME

6389
04:46:02,574 --> 04:46:07,145
CURES AND OTHER THERAPIES FOR

6390
04:46:07,145 --> 04:46:07,646
CYSTIC FIBROSIS.

6391
04:46:07,646 --> 04:46:08,881
>> HI, EVERYONE EEXSEATED
TO BE

6392
04:46:08,881 --> 04:46:10,649
AS PART OF THIS PANEL AND IN

6393
04:46:10,649 --> 04:46:12,050
GENERAL AS PART OF THIS

6394
04:46:12,050 --> 04:46:12,584
WORKSHOP.

6395
04:46:12,584 --> 04:46:14,152
THANKS AGAIN TO THE ORGANIZERS

6396
04:46:14,152 --> 04:46:17,656
FOR PUTTING THIS ALTOGETHER AND

6397
04:46:17,656 --> 04:46:20,225
DR. WEST AND DR. ZIADY FOR

6398
04:46:20,225 --> 04:46:22,561
MODERATING THE PANEL.

6399
04:46:22,561 --> 04:46:25,264
YOU'VE ALREADY HEARD ABOUT CF

6400
04:46:25,264 --> 04:46:26,632
TODAY, SO YOU'VE HEARD

6401
04:46:26,632 --> 04:46:28,734
INFORMATION AND KEY ADVANCES

6402
04:46:28,734 --> 04:46:30,469
AROUND MAN MALMODELS AND SOME

6403
04:46:30,469 --> 04:46:32,437
ASPECTS OF WHAT COULD CONSITUTE

6404
04:46:32,437 --> 04:46:33,839
A TARGET PROFILE IN CF AND I

6405
04:46:33,839 --> 04:46:36,575
WANT TO SPEND TIME TO INTRODUCE

6406
04:46:36,575 --> 04:46:37,509
THE CYSTIC PHOBEROSIS FOUNDATION

6407
04:46:37,509 --> 04:46:40,045
AND HOW YOU CAN PARTNER FROM

6408
04:46:40,045 --> 04:46:40,879
EARLY STAGE ACADEMIC WORK ALL

6409
04:46:40,879 --> 04:46:42,881
THE WAY TO THE CLINIC AND THEN

6410
04:46:42,881 --> 04:46:46,919
TO THE APPROVED PRODUCT.

6411
04:46:46,919 --> 04:46:48,020
NEXT SLIDE.

6412
04:46:48,020 --> 04:46:50,155
SO THE CF FOUNDATION HAS BEEN

6413
04:46:50,155 --> 04:46:51,290
AROUND FOR ALMOST 70 YEARS NOW.

6414
04:46:51,290 --> 04:46:52,724
WE WERE FIRST ESTABLISHED IN

6415
04:46:52,724 --> 04:46:55,027
1955 AT A TIME WHEN CF WAS

6416
04:46:55,027 --> 04:46:56,762
ALMOST SOLELY A PEDIATRIC

6417
04:46:56,762 --> 04:46:56,995
DISEASE.

6418
04:46:56,995 --> 04:46:58,797
TODAY BECAUSE OF TREATMENTS

6419
04:46:58,797 --> 04:47:00,465
AVAILABLE AND THE STANDARD OF

6420
04:47:00,465 --> 04:47:02,301
CARE ASSOCIATE WIDE CF, MANY

6421
04:47:02,301 --> 04:47:03,769
PEOPLE ARE LIVING INTO THEIR

6422
04:47:03,769 --> 04:47:05,470
LATE 50S AND BEYOND AND NEARLY

6423
04:47:05,470 --> 04:47:09,074
60% OF THE PEOPLE WITH CF ARE

6424
04:47:09,074 --> 04:47:09,474
NOW ADULTS.

6425
04:47:09,474 --> 04:47:11,410
IN TERMS OF MISSION OF THE CF

6426
04:47:11,410 --> 04:47:13,679
FOUNDATION IT IS TO CURE CF AND

6427
04:47:13,679 --> 04:47:16,415
PROVIDE ALL PEOPLE WITH CF THE

6428
04:47:16,415 --> 04:47:17,816
OPPORTUNITY TO LEAD LONG

6429
04:47:17,816 --> 04:47:18,951
FULFILLING LIVES BY FUNDING

6430
04:47:18,951 --> 04:47:20,752
RESEARCH AND DRUG DEVELOPMENT,

6431
04:47:20,752 --> 04:47:22,454
PARTNERING WITH THE CF COMMUNITY

6432
04:47:22,454 --> 04:47:24,756
AND ADVANCING HIGH QUALITY

6433
04:47:24,756 --> 04:47:26,124
SPECIALIZED CARE.

6434
04:47:26,124 --> 04:47:27,559
NEARLY ALL OF THE 16 APPROVED

6435
04:47:27,559 --> 04:47:30,762
THERAPIES HAVE BEEN APPROVED FOR

6436
04:47:30,762 --> 04:47:31,697
CYSTIC FIBROSIS WERE DEVELOPED

6437
04:47:31,697 --> 04:47:32,664
WITH THE FOUNDATION'S SUPPORT

6438
04:47:32,664 --> 04:47:35,567
AND TODAY THERE ARE MORE THAN

6439
04:47:35,567 --> 04:47:37,536
THE PIPE LENGTH TO ADDRESS THE

6440
04:47:37,536 --> 04:47:43,475
COST AND COMPLICATIONS OF THE

6441
04:47:43,475 --> 04:47:43,709
DISEASE.

6442
04:47:43,709 --> 04:47:44,476
NEXT SLIDE.

6443
04:47:44,476 --> 04:47:47,646
AND THEN, MORE BROADLY, WE ARE

6444
04:47:47,646 --> 04:47:50,849
IN A UNIQUE POSITION TO RISK AND

6445
04:47:50,849 --> 04:47:54,686
DECELERATE PROGRAMS THANKS TO

6446
04:47:54,686 --> 04:47:55,520
OUR WORK THROUGH PHILANTHROPY

6447
04:47:55,520 --> 04:47:56,822
AND OUR WORK AT THE COMMUNITY.

6448
04:47:56,822 --> 04:47:59,791
WE PLAN TO INVEST $3.5 MILLION

6449
04:47:59,791 --> 04:48:05,163
IN RND OVER THE NEXT FEW YEARS,

6450
04:48:05,163 --> 04:48:09,568
WE ARE NETWORK OF 100 + CENTERS

6451
04:48:09,568 --> 04:48:11,937
AND 47 MUSCULAR DYSTROPHY FTEs

6452
04:48:11,937 --> 04:48:14,439
AND BEST IN CLASS ASSAYS AND THE

6453
04:48:14,439 --> 04:48:15,907
LARGEST DATA BANK OF PATIENT

6454
04:48:15,907 --> 04:48:17,609
DERIVED CELLS AS WELL.

6455
04:48:17,609 --> 04:48:20,412
AND THEN WE'VE ALSO INVESTED IN

6456
04:48:20,412 --> 04:48:21,947
CF ANIMAL MODELS SOME OF WHICH

6457
04:48:21,947 --> 04:48:24,950
YOU HEARD TODAY, RODENTS,

6458
04:48:24,950 --> 04:48:27,953
FERRETS, PIG, SHEEP, AND ALSO

6459
04:48:27,953 --> 04:48:33,125
HAVE OUR BEST IN CLASS PATIENT

6460
04:48:33,125 --> 04:48:33,859
REGISTRY CAPTURING 90% OF THE

6461
04:48:33,859 --> 04:48:38,530
PATIENTS IN THE U.S., 90 + SITES

6462
04:48:38,530 --> 04:48:40,365
IN CF FOCUSED CLINICAL TRIALS

6463
04:48:40,365 --> 04:48:41,233
AND NETWORK, IN-HOUSE SCIENTIFIC

6464
04:48:41,233 --> 04:48:44,636
TEAM AND GLOBAL NETWORK OF

6465
04:48:44,636 --> 04:48:45,637
KOLs AND CLINICAL RESEARCH

6466
04:48:45,637 --> 04:48:47,105
DESIGN AND SUPPORT TEAM.

6467
04:48:47,105 --> 04:48:47,606
NEXT SLIDE.

6468
04:48:47,606 --> 04:48:49,374
IN TERMS OF HOW WE PARTNER WITH

6469
04:48:49,374 --> 04:48:51,109
TREY, WE HAVE,A PROACHS TO

6470
04:48:51,109 --> 04:48:52,511
INVEST IN CF PROGRAMS.

6471
04:48:52,511 --> 04:48:54,946
IT CAN BE AT PRODUCT BASES WITH

6472
04:48:54,946 --> 04:48:56,548
PRODUCT SPECIFIC ROALT GRAIMENTS

6473
04:48:56,548 --> 04:48:57,849
FOR LATE STAGE CLINICAL OR

6474
04:48:57,849 --> 04:49:00,819
COMMERCIAL PRODUCTS WE CAN ALSO

6475
04:49:00,819 --> 04:49:03,822
DO PREFERRED EQUITY SEED THROUGH

6476
04:49:03,822 --> 04:49:05,190
PRE-IPO, WHICH IS THE MOST

6477
04:49:05,190 --> 04:49:06,792
COMMON EARLY STAGE COMPANIES, WE

6478
04:49:06,792 --> 04:49:09,928
CAN ALSO DO COMMON EQUITY IN

6479
04:49:09,928 --> 04:49:10,662
PUBLIC COMPANIES, CONVERTIBLE

6480
04:49:10,662 --> 04:49:12,464
NOTES AND SAVES FOR BRIDGE

6481
04:49:12,464 --> 04:49:14,866
FUNDING AND HYBRID INVESTMENT

6482
04:49:14,866 --> 04:49:15,834
COMBINING APPROACH ANDS THEN

6483
04:49:15,834 --> 04:49:18,437
WE'RE ALSO LOOKING FOR

6484
04:49:18,437 --> 04:49:18,770
PARTNERSHIPS.

6485
04:49:18,770 --> 04:49:21,840
WE WORK OR WE CAN DO LIMITED

6486
04:49:21,840 --> 04:49:23,809
PARTNERSHIP INVESTMENTS, ALONG

6487
04:49:23,809 --> 04:49:25,410
THE INCUBATORS, NEW COMPANY

6488
04:49:25,410 --> 04:49:26,278
INFORMATION FOR SPECIFIC

6489
04:49:26,278 --> 04:49:29,247
APPLICATIONS AND THEN WORK IN

6490
04:49:29,247 --> 04:49:30,315
ACCELERATORS OR INCUBATORS TO

6491
04:49:30,315 --> 04:49:32,384
BRING NEW TECHNOLOGIES TO THE

6492
04:49:32,384 --> 04:49:33,452
CYSTIC FIBROSIS AND THE EXAMPLE

6493
04:49:33,452 --> 04:49:36,288
HERE IS TO THE GOLDEN TICKET WE

6494
04:49:36,288 --> 04:49:39,658
SPONSOR AT THE LABS AT BERKELEY.

6495
04:49:39,658 --> 04:49:40,425
NEXT SLIDE, PLEASE.

6496
04:49:40,425 --> 04:49:42,694
IN TERMS OF THE AREAS OF FOCUS,

6497
04:49:42,694 --> 04:49:45,030
ESPECIALENTIALLY IN THE GENETIC

6498
04:49:45,030 --> 04:49:46,364
THERAPIES, THE CYSTIC FIBROSIS

6499
04:49:46,364 --> 04:49:48,066
FOUNDATION IS REALLY LOOKING TO

6500
04:49:48,066 --> 04:49:50,769
ADDED TO THE NEED FOR THERAPIES

6501
04:49:50,769 --> 04:49:53,905
THAT MAY NEED HELP WITH

6502
04:49:53,905 --> 04:49:54,506
CYSTIC FIBROSIS, ESPECIALLY

6503
04:49:54,506 --> 04:49:56,241
THOSE THAT CANNOT TOLERATE

6504
04:49:56,241 --> 04:49:57,542
EXISTING TREEPS FOR EXAMPLE

6505
04:49:57,542 --> 04:49:59,211
MODULATORS AND WE ALSO BELIEVE

6506
04:49:59,211 --> 04:50:00,612
THAT GENETIC BASED THERAPIES ARE

6507
04:50:00,612 --> 04:50:01,780
KEY IN DEVELOPING A CURE FOR THE

6508
04:50:01,780 --> 04:50:03,882
DEC EMPLOY SO WHEN IT COMES TO

6509
04:50:03,882 --> 04:50:05,717
GENETIC MEDICINE THERE ARE 3 KEY

6510
04:50:05,717 --> 04:50:06,918
BUCKET WE'RE LOOKING FOR, THE

6511
04:50:06,918 --> 04:50:08,820
FIRST 1 IS PROTEIN RESTORATION.

6512
04:50:08,820 --> 04:50:11,223
WE'RE LOOKING FOR IMPROVEMENTS

6513
04:50:11,223 --> 04:50:12,190
OVER EXISTING RNA TECHNOLOGIES

6514
04:50:12,190 --> 04:50:13,458
THAT ARE CURRENTLY IN THE

6515
04:50:13,458 --> 04:50:14,292
CLINIC.

6516
04:50:14,292 --> 04:50:15,460
SO WE'RE LOOKING FOR EXAMPLE,

6517
04:50:15,460 --> 04:50:16,428
FOR OPPORTUNITIES THAT ALLOW US

6518
04:50:16,428 --> 04:50:18,964
TO READ THOSE APPROACHES

6519
04:50:18,964 --> 04:50:20,565
TARGETING THE LUNG, SYSTEMIC

6520
04:50:20,565 --> 04:50:22,067
APPROACHES THAT TARGET THE

6521
04:50:22,067 --> 04:50:24,836
LIVER, IMPROVE DURATION OR TIME

6522
04:50:24,836 --> 04:50:27,939
TO REDOSE, IN TERMS OF GENE

6523
04:50:27,939 --> 04:50:28,974
DELIVERY, WE RECOGNIZE THAT THE

6524
04:50:28,974 --> 04:50:32,511
LUNG IS HARD TO ACCESS AS YOU

6525
04:50:32,511 --> 04:50:33,612
HEARD TODAY, SO WE'RE LOOKING

6526
04:50:33,612 --> 04:50:36,348
FOR TECHNOLOGIES THAT CAN DO 1

6527
04:50:36,348 --> 04:50:38,950
OR MORE OF THE FOLLOWING TARGETS

6528
04:50:38,950 --> 04:50:40,519
ORIGINS, AS I MENTIONED LUNG IS

6529
04:50:40,519 --> 04:50:42,154
WHERE THE HIGHEST UNMET NEED IS

6530
04:50:42,154 --> 04:50:44,089
BUT WE'RE ALSO LOOKING AT

6531
04:50:44,089 --> 04:50:47,092
PANCREAS AND THE GI TRACT AND

6532
04:50:47,092 --> 04:50:48,727
THEN WITHIN THOSE ORGANS WE ARE

6533
04:50:48,727 --> 04:50:49,594
LOOKING AT SPECIFIC CELL TYPES

6534
04:50:49,594 --> 04:50:50,862
AND THEN WE'RE LOOKING FOR

6535
04:50:50,862 --> 04:50:52,264
TECHNOLOGIES THAT ALLOW US TO

6536
04:50:52,264 --> 04:50:54,499
READ THOSE OR DELIVER CARGO

6537
04:50:54,499 --> 04:50:55,734
SYSTEMICALLY AS WELL.

6538
04:50:55,734 --> 04:50:57,502
AND FINALLY FOR GENE EDITING WE

6539
04:50:57,502 --> 04:50:59,771
ARE LOOKING FOR APPROACHES TO

6540
04:50:59,771 --> 04:51:03,642
ALLOW US TO INSERT GENES INTO

6541
04:51:03,642 --> 04:51:05,911
RESTORE GENE FUNCTION AND THEN

6542
04:51:05,911 --> 04:51:07,913
MAKE EDITS WITH LITTLE

6543
04:51:07,913 --> 04:51:10,582
OFF-TARGETSMENT NEXT SLIDE,

6544
04:51:10,582 --> 04:51:10,849
PLEASE.

6545
04:51:10,849 --> 04:51:13,218
AND THEN THIS TERMS OF OFTEN

6546
04:51:13,218 --> 04:51:14,319
TIMES WITH THE DISEASE, THIS

6547
04:51:14,319 --> 04:51:16,054
MIGHT NOT BE THE KEY FOCUS OF

6548
04:51:16,054 --> 04:51:17,989
THE MEETING TODAY BUT I DID WANT

6549
04:51:17,989 --> 04:51:19,858
TO JUST TOUCH ON THIS AREA SINCE

6550
04:51:19,858 --> 04:51:23,061
IT IS AN AREA WHERE WE CONTINUE

6551
04:51:23,061 --> 04:51:27,432
TO MAKE INVESTMENTS,

6552
04:51:27,432 --> 04:51:28,800
SPECIFICALLY FOR SUITABLE

6553
04:51:28,800 --> 04:51:30,969
DIAGNOSING OF PATIENTS, WE ARE

6554
04:51:30,969 --> 04:51:36,274
LOOKING FOR IMPROVEMENTS FOR THE

6555
04:51:36,274 --> 04:51:42,247
TREATMENT OF CHRONIC

6556
04:51:42,247 --> 04:51:44,549
PSEUDONOAMON AS, AND ALSO

6557
04:51:44,549 --> 04:51:50,088
PULMONARY INFECS ASSOCIATE WIDE

6558
04:51:50,088 --> 04:51:51,656
BURKHOLDERRIA SPECS AND ALSO

6559
04:51:51,656 --> 04:51:53,124
METHODS FOR PULMONARY METHODS

6560
04:51:53,124 --> 04:51:57,996
THAT DON'T RELY ON SPUTUM WITH

6561
04:51:57,996 --> 04:52:00,265
PSEUDOMONAS SCREENINGS AND THEN

6562
04:52:00,265 --> 04:52:00,932
FOR OTHER DIAGNOSE.

6563
04:52:00,932 --> 04:52:03,034
WE'RE ALSO LOOKING FOR MAJOR

6564
04:52:03,034 --> 04:52:05,203
IMPROVE AMS IN TREATMENTS FOR

6565
04:52:05,203 --> 04:52:06,471
OTHER COMMON PATHOGENS AFFECTING

6566
04:52:06,471 --> 04:52:07,639
PEOPLE WITH CYSTIC FIBROSIS. 

6567
04:52:07,639 --> 04:52:10,542
NEXT SLIDE, PLEASE.

6568
04:52:10,542 --> 04:52:12,711
AND THEN HERE, I JUST ALSO

6569
04:52:12,711 --> 04:52:13,878
WANTED TO TOUCH REALLY BRIEFLY

6570
04:52:13,878 --> 04:52:16,948
ON MANY OF THE CHALLENGES THAT

6571
04:52:16,948 --> 04:52:18,683
WE HEARD MENTIONED TODAY AROUND

6572
04:52:18,683 --> 04:52:23,255
FUND XG AROUND MOVING A COMPANY

6573
04:52:23,255 --> 04:52:25,156
FORWARD, REALLY AMPLIFIED IN THE

6574
04:52:25,156 --> 04:52:26,758
CASE AND THE ANTIMICROBIAL

6575
04:52:26,758 --> 04:52:27,726
PROJECTS WHICH HAS AS I

6576
04:52:27,726 --> 04:52:29,027
MENTIONED MAYBE NOT BEEN THE

6577
04:52:29,027 --> 04:52:30,762
FOCUS TODAY BUT I WANT TO PLUG

6578
04:52:30,762 --> 04:52:32,764
THIS CONTINUES TO BE AN

6579
04:52:32,764 --> 04:52:34,332
IMPORTANT AREA FOR US AND HERE

6580
04:52:34,332 --> 04:52:35,634
WE PARTNERED CLOSELY WITH THE

6581
04:52:35,634 --> 04:52:37,502
NIH AND OTHER PROGRAMS FOR

6582
04:52:37,502 --> 04:52:38,336
EXAMPLE, THAT STEPHANIE DAVIS

6583
04:52:38,336 --> 04:52:40,538
MENTIONED AND OTHER PUBLIC

6584
04:52:40,538 --> 04:52:42,607
ENTITIES TO MOVE THERAPIES IN

6585
04:52:42,607 --> 04:52:43,508
THIS SPACE FURTHER.

6586
04:52:43,508 --> 04:52:51,683
NEXT SLIDE, PLEASE.

6587
04:52:51,683 --> 04:52:52,984
AND THEN BEYOND THIS DIRECT WAYS

6588
04:52:52,984 --> 04:52:54,853
IN WHICH WE ENGAGE COMPANIES WE

6589
04:52:54,853 --> 04:52:56,021
ALSO PROVIDE CLEAR GUIDANCE WHEN

6590
04:52:56,021 --> 04:52:58,523
IT COMES TO THE PRECLINICAL OR

6591
04:52:58,523 --> 04:52:59,557
EARLY STAGE DEVELOPMENT AND I

6592
04:52:59,557 --> 04:53:06,131
WANTED TO SHOW EXAMPLES FROM MY

6593
04:53:06,131 --> 04:53:07,165
COLLEAGUES, [INDISCERNIBLE] FROM

6594
04:53:07,165 --> 04:53:09,100
THE FOUNDATION, AS WELL AS JOHN

6595
04:53:09,100 --> 04:53:12,404
WHO IS HERE TODAY AS WELL.

6596
04:53:12,404 --> 04:53:13,872
NEXT SLIDE, PLEASE.

6597
04:53:13,872 --> 04:53:15,440
THIS IS AN SAMPLE DOCUMENT THAT

6598
04:53:15,440 --> 04:53:16,675
WE PROVIDE COMPANIES IN THE

6599
04:53:16,675 --> 04:53:17,742
MEDICINE SPACE.

6600
04:53:17,742 --> 04:53:18,576
IT DETAILS DIFFERENT ASSPEBTS

6601
04:53:18,576 --> 04:53:20,278
THAT ARE IMPORTANT WHEN YOU'RE

6602
04:53:20,278 --> 04:53:23,448
THINKING ABOUT FOR EXAMPLE,

6603
04:53:23,448 --> 04:53:26,685
HERE, A PRECLINICAL PACKAGE OR A

6604
04:53:26,685 --> 04:53:27,385
TRANSIENT THERAPY FOR REPEAT

6605
04:53:27,385 --> 04:53:28,486
DOSING WHAT ARE THE

6606
04:53:28,486 --> 04:53:29,921
CHARACTERISTICS YOU HAVE TO

6607
04:53:29,921 --> 04:53:33,291
THINK ABOUT AND WHAT ARE SOME OF

6608
04:53:33,291 --> 04:53:35,627
THE KEY AREAS WHERE SPECIFIC

6609
04:53:35,627 --> 04:53:37,362
ASSAYS THAT WERE DEVELOPED OR

6610
04:53:37,362 --> 04:53:39,264
THAT ARE DEVELOPED COULD HELP

6611
04:53:39,264 --> 04:53:40,465
YOU ANSWER SOME OF THE OPEN

6612
04:53:40,465 --> 04:53:40,832
QUESTIONS HERE.

6613
04:53:40,832 --> 04:53:46,171
NEXT SLIDE, PLEASE.

6614
04:53:46,171 --> 04:53:48,039
AS I MENTIONED AS WELL, WE ALSO

6615
04:53:48,039 --> 04:53:49,207
PROVIDE GUIDANCE IN TERMS OF

6616
04:53:49,207 --> 04:53:51,876
WHAT ARE THE KEY IN VIVO MODELS

6617
04:53:51,876 --> 04:53:53,945
THAT -- AS WELL AS CELL MODELS

6618
04:53:53,945 --> 04:53:55,413
IT CAN USE TO VALIDATE

6619
04:53:55,413 --> 04:53:56,414
TECHNOLOGIES, SOME OF WHICH WE

6620
04:53:56,414 --> 04:54:00,452
HAVE IN OUR LABS IN BOSTON, AND

6621
04:54:00,452 --> 04:54:03,455
THEN SOME OF THE MODELS THAT WE

6622
04:54:03,455 --> 04:54:05,657
HAVE THROUGH OUR PARTNERS, AND

6623
04:54:05,657 --> 04:54:07,292
THEN WE'RE HAPPY ALWAYS TO WALK

6624
04:54:07,292 --> 04:54:08,560
COMPANIES THROUGH WHAT ARE THE

6625
04:54:08,560 --> 04:54:10,095
DIFFERENT MODELS OUT THERE,

6626
04:54:10,095 --> 04:54:11,363
WHICH 1 IS MOST APPROPRIATE FOR

6627
04:54:11,363 --> 04:54:12,430
TECHNOLOGY THAT YOU'RE

6628
04:54:12,430 --> 04:54:13,598
DEVELOPING, AND WHICH 1S ARE

6629
04:54:13,598 --> 04:54:15,367
CRUCIAL WHEN YOU'RE THINKING

6630
04:54:15,367 --> 04:54:17,736
ABOUT PREPARING YOUR IND

6631
04:54:17,736 --> 04:54:20,572
PACKAGE. 

6632
04:54:20,572 --> 04:54:21,239
NEXT SLIDE, PLEASE.

6633
04:54:21,239 --> 04:54:23,208
AND THEN THE NEXT 3 SLIDES

6634
04:54:23,208 --> 04:54:26,044
REALLY JUST FOCUS ON SOME OF THE

6635
04:54:26,044 --> 04:54:27,846
KEY ASSAYS THAT WE'VE DEVELOPED,

6636
04:54:27,846 --> 04:54:31,282
BUT THEN, WE ALSO THINK OUR CREW

6637
04:54:31,282 --> 04:54:32,484
[INDISCERNIBLE] WHEN YOU'RE

6638
04:54:32,484 --> 04:54:34,285
PREPARING THAT PACKAGE FOR THE

6639
04:54:34,285 --> 04:54:37,188
FDA AND ALSO FOR THE NETWORK TO

6640
04:54:37,188 --> 04:54:38,690
ASSESS THE VALIDITY OF YOUR

6641
04:54:38,690 --> 04:54:38,990
TECHNOLOGY.

6642
04:54:38,990 --> 04:54:40,125
SO I WON'T WALK YOU THROUGH ALL

6643
04:54:40,125 --> 04:54:41,292
OF THIS BUT THIS IS JUST AN

6644
04:54:41,292 --> 04:54:43,294
EXAMPLE OF THE DOCUMS THAT WE'RE

6645
04:54:43,294 --> 04:54:44,729
HAPPY TO PROVIDE OUR PARTNERS,

6646
04:54:44,729 --> 04:54:47,832
IF YOU'RE THINKING ABOUT THE

6647
04:54:47,832 --> 04:54:48,900
PPP AND CYSTIC FIBROSIS.

6648
04:54:48,900 --> 04:54:54,239
WE CAN SKIP TO THE NEXT SLIDE

6649
04:54:54,239 --> 04:54:59,177
AND THE NEXT. 

6650
04:54:59,177 --> 04:54:59,878
NEXT SLIDE.

6651
04:54:59,878 --> 04:55:02,847
AND NEXT SLIDE, PLEASE.

6652
04:55:02,847 --> 04:55:05,116
AND THEN, FINALLY BECAUSE THE

6653
04:55:05,116 --> 04:55:07,051
TITLE OF THIS WORKSHOP IS FROM

6654
04:55:07,051 --> 04:55:08,686
CONCEPT TO CLINIC, I DID WANT TO

6655
04:55:08,686 --> 04:55:10,388
TOUCH ON ACADEMIC FUNDING

6656
04:55:10,388 --> 04:55:12,357
OPPORTUNITIES, WE HAVE A

6657
04:55:12,357 --> 04:55:15,960
PROLIFIC PORTFOLIO AND

6658
04:55:15,960 --> 04:55:17,095
MECHANISMS THAT CAN EXPLORE

6659
04:55:17,095 --> 04:55:18,663
PROGRAMS THAT CAN LEAD TO

6660
04:55:18,663 --> 04:55:19,063
COMPANY IDEAS.

6661
04:55:19,063 --> 04:55:21,366
IF YOU GO TO THE NEXT SLIDE.

6662
04:55:21,366 --> 04:55:23,101
SO OUR STANDARD FUNDED

6663
04:55:23,101 --> 04:55:26,604
MECHANISMS ARE RESEARCH GRANT, 3

6664
04:55:26,604 --> 04:55:29,073
YEARS, 150 K DIRECT PER YEE,

6665
04:55:29,073 --> 04:55:31,176
PLUS INDIRECT, PRELIMINARY DATA

6666
04:55:31,176 --> 04:55:31,676
REQUIRED.

6667
04:55:31,676 --> 04:55:31,976
NEXT SLIDE.

6668
04:55:31,976 --> 04:55:36,080
WE ALSO HAVE PILOT SIMULATED

6669
04:55:36,080 --> 04:55:43,021
GRANTS, 2 YEARS, 50 K PER YEAR,

6670
04:55:43,021 --> 04:55:44,422
PLUS [INDISCERNIBLE] PERCENT

6671
04:55:44,422 --> 04:55:44,656
DIRECT.

6672
04:55:44,656 --> 04:55:46,591
THAT'S NOT REQUIRED FOR THAT.

6673
04:55:46,591 --> 04:55:48,827
AND ALSO OFFER POST DOCTORAL

6674
04:55:48,827 --> 04:55:51,763
FELLOWSHIPS, 2 YEAR DURATION, 76

6675
04:55:51,763 --> 04:55:55,800
K IN YEAR 1 AND 76 IN YEAR 2.

6676
04:55:55,800 --> 04:55:58,670
NEXT YEAR AND THEN FINALLY WE

6677
04:55:58,670 --> 04:56:00,338
HAVE POST DOC FACULTY TRANSITION

6678
04:56:00,338 --> 04:56:03,174
AWARDS SIMILAR TO K99S, FUNDING

6679
04:56:03,174 --> 04:56:05,543
POST THE BOACT DOC PHASE AND THE

6680
04:56:05,543 --> 04:56:06,644
FACULTY PHASE.

6681
04:56:06,644 --> 04:56:08,279
APPLICATIONS ARE ACCEPTED IN

6682
04:56:08,279 --> 04:56:10,448
DECEMBER ONLY BUT IN GENERAL OUR

6683
04:56:10,448 --> 04:56:13,518
APPLICATION CYCLES ARE IN MAY

6684
04:56:13,518 --> 04:56:17,188
AND DECEMBER.

6685
04:56:17,188 --> 04:56:17,889
NEXT SLIDE.

6686
04:56:17,889 --> 04:56:20,158
AND THEN FINALLY WHEN IT COMES

6687
04:56:20,158 --> 04:56:23,161
TO PATH CHECK HERE WE HAVE

6688
04:56:23,161 --> 04:56:24,829
SPECIAL FUNDING MECHANISMS, FOR

6689
04:56:24,829 --> 04:56:26,698
EXAMPLE, OUR GOAL SUPPORT

6690
04:56:26,698 --> 04:56:27,398
INTEGRATED MULTIINVESTIGATOR

6691
04:56:27,398 --> 04:56:29,133
RESEARCH PROBABLES WITH THE

6692
04:56:29,133 --> 04:56:29,901
WELL-DEFINED CENTER OF FOCUS,

6693
04:56:29,901 --> 04:56:32,637
THIS IS UP TO 3 YEARS WITH

6694
04:56:32,637 --> 04:56:36,007
1 MILLION DIRECT PLUS 12%

6695
04:56:36,007 --> 04:56:37,842
INDIRECT FUNDING, RECEIPT DATE

6696
04:56:37,842 --> 04:56:40,078
IS JULY 29th.

6697
04:56:40,078 --> 04:56:40,278
NEXT.

6698
04:56:40,278 --> 04:56:42,213
AND THEN WE ALSO HAVE THE PIE O

6699
04:56:42,213 --> 04:56:44,182
NEAR AWARDS WITH THE GOAL TO

6700
04:56:44,182 --> 04:56:44,716
SUPPORT PARADIGM SHIFT IN

6701
04:56:44,716 --> 04:56:46,618
TECHNOLOGIES THAT HAVE THE

6702
04:56:46,618 --> 04:56:48,353
POTENTIAL TO CURE CF AT THE

6703
04:56:48,353 --> 04:56:50,755
CELLULAR OR THE GENETIC LEVEL

6704
04:56:50,755 --> 04:56:53,658
AFTER 3 YEARS AND FUNDING AS

6705
04:56:53,658 --> 04:56:55,960
AGREED UPON PLUS, 12% INDIRECT

6706
04:56:55,960 --> 04:56:56,995
ROLLING BASIS RECEIVE DATE AND

6707
04:56:56,995 --> 04:56:58,863
THIS 1 IS SIMILAR TO SOME OF THE

6708
04:56:58,863 --> 04:57:01,165
DISCUSSION WE HEARD IN THE PAST

6709
04:57:01,165 --> 04:57:02,133
PANEL AROUND RO-1S AND HOW

6710
04:57:02,133 --> 04:57:03,501
THEY'RE BEING USED TO THINK

6711
04:57:03,501 --> 04:57:07,939
ABOUT SOME OF THIS NEW

6712
04:57:07,939 --> 04:57:10,375
THERAPIES.

6713
04:57:10,375 --> 04:57:11,609
NEXT SLIDE.

6714
04:57:11,609 --> 04:57:14,178
SO YEAH, IN GENERAL, THE CFF IS

6715
04:57:14,178 --> 04:57:16,447
HERE REALLY TO PROVIDE

6716
04:57:16,447 --> 04:57:20,552
RESOURCES, THANK YOU SO MUCH

6717
04:57:20,552 --> 04:57:22,053
DR. HOLTZMAN, FOR MAKING THAT

6718
04:57:22,053 --> 04:57:24,222
PATH TOWARDS A THERAPEUTIC AND

6719
04:57:24,222 --> 04:57:25,523
WE'RE REALLY HOPING HERE TO BE

6720
04:57:25,523 --> 04:57:29,260
ABLE TO PROVIDE THE RESOURCES

6721
04:57:29,260 --> 04:57:30,795
THAT OUTLINED AS PEOPLE ARE

6722
04:57:30,795 --> 04:57:32,597
THINKING ABOUT CYSTIC FIBROSIS

6723
04:57:32,597 --> 04:57:34,365
AS AN INDICATION AND THEN IF YOU

6724
04:57:34,365 --> 04:57:35,700
ARE LOOKING FOR ADDITIONAL

6725
04:57:35,700 --> 04:57:37,268
INFORMATION, FEEL FREE TO

6726
04:57:37,268 --> 04:57:39,137
CONTACT ME OR VISIT THESE 2

6727
04:57:39,137 --> 04:57:39,337
SITES.

6728
04:57:39,337 --> 04:57:45,610
THANK YOU VERY MUCH. 

6729
04:57:45,610 --> 04:57:53,585
>> THANKS DR. SHIAO.

6730
04:57:53,585 --> 04:57:57,021
>> SO OUR NEXT SPEAKER
WILL BE

6731
04:57:57,021 --> 04:57:59,190
LUCY LIU, FROM THE LONG WOOD

6732
04:57:59,190 --> 04:58:01,125
FUND IN BOSTON AND SHE WILL

6733
04:58:01,125 --> 04:58:02,393
SHARE THEIR PRESENCE WITH

6734
04:58:02,393 --> 04:58:05,263
VENTURE CAPITAL THERE AND THE

6735
04:58:05,263 --> 04:58:06,564
STEPS THAT PEOPLE GO THROUGH TO

6736
04:58:06,564 --> 04:58:11,202
INTERACT WITH THEM AND THE

6737
04:58:11,202 --> 04:58:13,137
INVESTMENTS THEY MAKE WITH NEW

6738
04:58:13,137 --> 04:58:13,538
PATIENTS.

6739
04:58:13,538 --> 04:58:15,239
>> THANK YOU SO MUCH FOR
THE

6740
04:58:15,239 --> 04:58:16,941
MODERATORS AND ALSO FOR THE

6741
04:58:16,941 --> 04:58:19,243
OPPORTUNITY TO SPEAK WITH

6742
04:58:19,243 --> 04:58:19,944
EVERYONE HERE TODAY.

6743
04:58:19,944 --> 04:58:22,313
WOULD LOVE TO PROVIDE AN

6744
04:58:22,313 --> 04:58:24,382
OVERVIEW OF HOW EARLY STAGE

6745
04:58:24,382 --> 04:58:27,885
VENTURE PERSPECTIVE, WE LOOK AT

6746
04:58:27,885 --> 04:58:33,257
BOTH  ASSETS TO START A COMPANY

6747
04:58:33,257 --> 04:58:35,793
AND IF A COMPANY EXISTS AROUND

6748
04:58:35,793 --> 04:58:36,728
YOUR PARTICULAR TECHNOLOGY, THE

6749
04:58:36,728 --> 04:58:39,130
LINES WE HAVE AT EARLY STAGE

6750
04:58:39,130 --> 04:58:40,865
INVESTORS AND THINKING ABOUT

6751
04:58:40,865 --> 04:58:41,699
INVESTING OPPORTUNITIES THAT

6752
04:58:41,699 --> 04:58:44,135
WILL EVENTUALLY BECOME A DRUG,

6753
04:58:44,135 --> 04:58:47,138
SO I HAVE A FEW SLIDES HERE TO

6754
04:58:47,138 --> 04:58:48,606
HELP CONTEXTUALIZE MY

6755
04:58:48,606 --> 04:58:49,440
BACKGROUND, LONG BACKGROUND AND

6756
04:58:49,440 --> 04:58:52,110
HOW WE VOW THE WORD AS VENTURE

6757
04:58:52,110 --> 04:58:53,444
INVESTORS SO OFTEN TIME FIST YOU

6758
04:58:53,444 --> 04:58:56,414
INTERACTED WITH ANY OF US, THE

6759
04:58:56,414 --> 04:58:58,449
INTERACTIONS ARE LIKELY A LITTLE

6760
04:58:58,449 --> 04:58:58,883
BIT [INDISCERNIBLE].

6761
04:58:58,883 --> 04:59:04,489
SO ON THE NEXT SLIDE, PLEASE,

6762
04:59:04,489 --> 04:59:05,623
WANTED TO HIGHLIGHT HOW WE VIEW

6763
04:59:05,623 --> 04:59:08,393
THE WORLD WHICH IS THAT WE PRIDE

6764
04:59:08,393 --> 04:59:09,794
OURSELVES ON BUILDING BIOTECHS

6765
04:59:09,794 --> 04:59:12,697
THAT CREATE DRUGS THAT HAVE A

6766
04:59:12,697 --> 04:59:14,666
LASTING IMPACT AND COVER HIGH

6767
04:59:14,666 --> 04:59:14,966
UNMET NEEDS.

6768
04:59:14,966 --> 04:59:17,301
WHAT I AM SHOWING ARE A FEW

6769
04:59:17,301 --> 04:59:18,603
EXAMPLES OF COMPANIES THAT ALONG

6770
04:59:18,603 --> 04:59:20,038
WITH PARTNER VS STARTED.

6771
04:59:20,038 --> 04:59:26,944
ALL OF THESE STARTED WITH AN

6772
04:59:26,944 --> 04:59:28,513
[INDISCERNIBLE] LAB AND WITH 10

6773
04:59:28,513 --> 04:59:30,415
+ YEARS OR MORE THAN THAT AND

6774
04:59:30,415 --> 04:59:33,284
MILLIONS IF NOT BILLIONS IN

6775
04:59:33,284 --> 04:59:35,553
INVESTMENTS, THESE IDEA ANDS

6776
04:59:35,553 --> 04:59:38,056
CONCEPTS HAVE TURNED INTO OVER

6777
04:59:38,056 --> 04:59:40,258
20 DEFINITE MARKETED PRODUCTS,

6778
04:59:40,258 --> 04:59:41,926
AND ADDITIONAL THERAPIES AND

6779
04:59:41,926 --> 04:59:42,593
LATE STAGE DEVELOPMENT.

6780
04:59:42,593 --> 04:59:43,761
OT BOTTOM HERE HIGHLIGHTING A

6781
04:59:43,761 --> 04:59:47,432
FEW OF THE DRUGS THAT ALONG LONG

6782
04:59:47,432 --> 04:59:49,000
WOOD COMPANIES HAVE DEVELOPED

6783
04:59:49,000 --> 04:59:51,903
AND YOU KNOW 20 YEARS AGO

6784
04:59:51,903 --> 04:59:56,541
STARTED OUT AS A EXPERIMENT AND

6785
04:59:56,541 --> 04:59:57,575
EVENTUALLY IN A UNIVERSITY

6786
04:59:57,575 --> 04:59:57,842
SOMEWHERE.

6787
04:59:57,842 --> 05:00:02,113
SO ON THE NEXT SLIDE, PLEASE, 1

6788
05:00:02,113 --> 05:00:03,648
THING THAT I THINK WHAT NEEDS TO

6789
05:00:03,648 --> 05:00:06,617
BE QUITE CLEAR AND WHAT WE THINK

6790
05:00:06,617 --> 05:00:09,087
ABOUT DEEPLY IS THAT DRUG

6791
05:00:09,087 --> 05:00:10,822
DEVELOPMENT IS VERY EXPENSIVE IS

6792
05:00:10,822 --> 05:00:12,757
TAKES A VERY LONG TIME.

6793
05:00:12,757 --> 05:00:14,392
SO WE ALWAYS THINK ABOUT THE

6794
05:00:14,392 --> 05:00:16,661
PARTNERS THAT WE WILL HAVE TO

6795
05:00:16,661 --> 05:00:20,198
GET US THERE, GENERALLY IN TERMS

6796
05:00:20,198 --> 05:00:21,799
OF BIOTECH VENTURE CAPITAL,

6797
05:00:21,799 --> 05:00:25,603
WE'RE SPLIT INTO EARLY STAGE

6798
05:00:25,603 --> 05:00:27,138
GROWTH STAGE AND THEN

6799
05:00:27,138 --> 05:00:29,307
ESSENTIALLY GOING TO PUBLIC

6800
05:00:29,307 --> 05:00:31,142
INVESTORS AND THINGS GOING

6801
05:00:31,142 --> 05:00:32,610
BEYOND THERE.

6802
05:00:32,610 --> 05:00:34,812
AS EARLY STAGE INVESTORS, WE

6803
05:00:34,812 --> 05:00:36,047
THINK DEEPLY ABOUT THE PARTNERS

6804
05:00:36,047 --> 05:00:37,982
THAT HELP US GET THERE, I HAVE

6805
05:00:37,982 --> 05:00:39,851
LISTED SOME EXAMPLES HERE OF CO

6806
05:00:39,851 --> 05:00:41,586
INVESTORS THAT HAVE A SIMILAR

6807
05:00:41,586 --> 05:00:43,621
MIND SET TO US.

6808
05:00:43,621 --> 05:00:47,058
SO REALLY ON THE INVESTORS

6809
05:00:47,058 --> 05:00:49,427
GENERALLY TEND TO FLOCK

6810
05:00:49,427 --> 05:00:49,694
TOGETHER.

6811
05:00:49,694 --> 05:00:51,162
IT'S TRUE BECAUSE AT A VERY

6812
05:00:51,162 --> 05:00:52,764
EARLY STAGE OF THE COMPANY,

6813
05:00:52,764 --> 05:00:53,931
THERE WILL BE MANY DIRECTIONS

6814
05:00:53,931 --> 05:00:55,566
FOR THE TECHNOLOGY OR FOR THE

6815
05:00:55,566 --> 05:00:57,602
PLATFORM TO GO.

6816
05:00:57,602 --> 05:01:02,373
AND THOSE EARLY DECISIONS HELP

6817
05:01:02,373 --> 05:01:04,542
REALLY CRAFT THE PATH THAT YOUR

6818
05:01:04,542 --> 05:01:05,643
MOLECULE OR THAT YOUR PLATFORM

6819
05:01:05,643 --> 05:01:08,079
WILL TAKE TO DRIVE IT TOWARDS

6820
05:01:08,079 --> 05:01:08,546
SOMETHING MEANINGFUL.

6821
05:01:08,546 --> 05:01:11,149
AND THEN AT THE SAME TIME, WE'RE

6822
05:01:11,149 --> 05:01:13,217
VERY MUCH AWARE THAT EARLY

6823
05:01:13,217 --> 05:01:14,252
STAGES OF DEVELOPMENT, SMALL

6824
05:01:14,252 --> 05:01:16,954
COMPANY, YOU CAN GET STARTED ON

6825
05:01:16,954 --> 05:01:19,757
PRECLINICAL STUDIES, MAYBE YOU

6826
05:01:19,757 --> 05:01:21,125
CAN GO THROUGH YOUR EARLY PHASE

6827
05:01:21,125 --> 05:01:24,629
1 OR PHASE 2, BUT THEN GETTING

6828
05:01:24,629 --> 05:01:26,030
TO LATE STAGE DEVELOPMENT, IT'S

6829
05:01:26,030 --> 05:01:27,265
IMPORTANT THAT MOST OF THE TIME,

6830
05:01:27,265 --> 05:01:28,399
JUST GIVEN THE NUMBER OF

6831
05:01:28,399 --> 05:01:30,568
PATIENTS THAT YOU NEED TO

6832
05:01:30,568 --> 05:01:32,103
RECRUIT AND THE DURATION OF

6833
05:01:32,103 --> 05:01:34,172
CLINICAL STUDIES, THERE NEEDS TO

6834
05:01:34,172 --> 05:01:35,673
BE A STRATEGIC PARTNER SO IN

6835
05:01:35,673 --> 05:01:39,210
THIS CASE A PHARMA OR OTHER TO

6836
05:01:39,210 --> 05:01:40,812
REALLY HELP FINANCE THE REST OF

6837
05:01:40,812 --> 05:01:44,315
THE STUDIES AND THEN EVENTUALLY

6838
05:01:44,315 --> 05:01:45,216
BRING IT TO COMMERCIALIZATION.

6839
05:01:45,216 --> 05:01:49,453
AND THEN IN THE LAST BUCKET,

6840
05:01:49,453 --> 05:01:50,621
SCIENCE IS ALWAYS UNPREDICTABLE

6841
05:01:50,621 --> 05:01:54,792
AND WE LIKE TO WORK WITH

6842
05:01:54,792 --> 05:01:56,327
ACADEMICS THAT HELP REALLY SHAPE

6843
05:01:56,327 --> 05:01:57,728
OUR COMPANIES THAT WE BUILD AND

6844
05:01:57,728 --> 05:02:00,565
ALSO PROVIDE THE INITIAL

6845
05:02:00,565 --> 05:02:02,233
CATALYST THAT START A PARTICULAR

6846
05:02:02,233 --> 05:02:03,100
CONCEPT, SO JUST HIGHLIGHTING

6847
05:02:03,100 --> 05:02:05,469
SOME OF THE INSTITUTES THAT

6848
05:02:05,469 --> 05:02:08,239
WE'VE WORKED WITH, AND ALSO

6849
05:02:08,239 --> 05:02:10,641
WHERE LONG WOOD FOLKS HAVE

6850
05:02:10,641 --> 05:02:12,810
TRAINED TO REALLY START AND STEP

6851
05:02:12,810 --> 05:02:16,147
1 AS A CONCEPT TO BUILD A

6852
05:02:16,147 --> 05:02:22,687
COMPANY AROUND. 

6853
05:02:22,687 --> 05:02:23,287
NEXT SLIDE, PLEASE.

6854
05:02:23,287 --> 05:02:26,591
SO TO GIVE YOU A SENSE OF THE

6855
05:02:26,591 --> 05:02:28,659
TEAM AND THIS -- THE TEAM THAT

6856
05:02:28,659 --> 05:02:32,797
WE CAPTURE AT LONG WOOD IS QUITE

6857
05:02:32,797 --> 05:02:34,632
SIMILAR IN PROFILE TO OTHER

6858
05:02:34,632 --> 05:02:37,869
TEAMS THAT YOU WILL FIND OTHER

6859
05:02:37,869 --> 05:02:40,037
COMPANY CREATION FIRMS AROUND

6860
05:02:40,037 --> 05:02:41,539
THE COUNTRY AND SO WHAT I'M

6861
05:02:41,539 --> 05:02:42,840
HIGHLIGHTING HERE IS THAT WE'RE

6862
05:02:42,840 --> 05:02:44,008
GENERALLY A VERY SMALL TEAM.

6863
05:02:44,008 --> 05:02:46,344
THERE'S 7 OF US IN THE

6864
05:02:46,344 --> 05:02:47,044
INVESTMENT TEAM, FIRST OFF WEST

6865
05:02:47,044 --> 05:02:50,348
FALL IS A CONFOUNDER AND OUR

6866
05:02:50,348 --> 05:02:51,849
GENERAL PARTNER, AND HE IS

6867
05:02:51,849 --> 05:02:53,251
BEHIND A NUMBER OF COMPANIES

6868
05:02:53,251 --> 05:02:57,288
WHERE HE WAS THE INITIAL

6869
05:02:57,288 --> 05:02:59,257
CONFOUNDER WITH AN ACADEMIC AND

6870
05:02:59,257 --> 05:03:00,858
ALSO THE INITIAL CEO TO HELP

6871
05:03:00,858 --> 05:03:03,561
BUILD OUT THESE COMPANIES.

6872
05:03:03,561 --> 05:03:06,831
AND THEN, THOSE ADDITIONAL TEAM

6873
05:03:06,831 --> 05:03:09,400
MEMBERS, ALEX WHO'S 1 OF THE

6874
05:03:09,400 --> 05:03:10,968
PARTNERS, AND 1 OF THE VENTURE

6875
05:03:10,968 --> 05:03:12,403
PARTNERS AND THE REST OF US ON

6876
05:03:12,403 --> 05:03:13,437
THE INVESTMENT TEAM, WE ALL WORK

6877
05:03:13,437 --> 05:03:14,872
TOGETHER TO CRAFT AREA ARES OF

6878
05:03:14,872 --> 05:03:18,609
FOCUS THAT WE LIKE TO DUE A DEEP

6879
05:03:18,609 --> 05:03:20,244
DIVE ON AND THEN START WORKING

6880
05:03:20,244 --> 05:03:23,314
AS A SMALLER GROUP ON COMPANY

6881
05:03:23,314 --> 05:03:24,181
CREATION.

6882
05:03:24,181 --> 05:03:27,051
SO IF WE -- IF YOU EVER SEE AN

6883
05:03:27,051 --> 05:03:29,153
E-MAIL FROM 1 OF US REACHING OUT

6884
05:03:29,153 --> 05:03:30,354
TO YOU TO TALK ABOUT A

6885
05:03:30,354 --> 05:03:31,389
PARTICULAR OPPORTUNITY OR TO GET

6886
05:03:31,389 --> 05:03:32,323
YOUR THOUGHTS ON AN AREA WHERE

6887
05:03:32,323 --> 05:03:35,192
WE WOULD LIKE TO GET A LITTLE

6888
05:03:35,192 --> 05:03:38,663
BIT DEEPER INFORMATION ON,

6889
05:03:38,663 --> 05:03:39,397
YOU'LL PROBABLY KNOW THAT IT'S

6890
05:03:39,397 --> 05:03:40,798
GENERALLY FOR US TO EVALUATE

6891
05:03:40,798 --> 05:03:43,968
WHETHER WE WANT TO START A

6892
05:03:43,968 --> 05:03:45,569
COMPANY IN A PARTICULAR AREA AND

6893
05:03:45,569 --> 05:03:47,071
WHETHER YOU WOULD HAVE

6894
05:03:47,071 --> 05:03:49,807
INTERESTING TECHNOLOGIES THAT WE

6895
05:03:49,807 --> 05:03:52,076
SHOULD BE DISCUSSING TO START

6896
05:03:52,076 --> 05:03:52,743
THESE COMPANIES.

6897
05:03:52,743 --> 05:03:55,479
AND SO 1 THING THEY WANT TO

6898
05:03:55,479 --> 05:03:57,448
POINT OUT IS THAT AS EARLY STAGE

6899
05:03:57,448 --> 05:03:59,083
COMPANY CREATORS AND INVESTORS,

6900
05:03:59,083 --> 05:04:01,285
ALL OF US PRIDE OURSELVES ON

6901
05:04:01,285 --> 05:04:02,987
BEING SCIENTISTS AT SOME POINT,

6902
05:04:02,987 --> 05:04:05,890
MANY OF US, YOU WILL SEE ON THE

6903
05:04:05,890 --> 05:04:09,427
TEAM HERE, HAVE PH Ds OR POST

6904
05:04:09,427 --> 05:04:10,328
DOCS OR BOTH.

6905
05:04:10,328 --> 05:04:11,629
AND REALLY PRY OURSELVES ON

6906
05:04:11,629 --> 05:04:14,165
BEING ABLE TO, IF WE NEEDED TO,

6907
05:04:14,165 --> 05:04:14,765
ALTHOUGH, GENERALLY PROBABLY

6908
05:04:14,765 --> 05:04:16,701
DON'T WANT US THERE, GO BACK ON

6909
05:04:16,701 --> 05:04:18,869
THE BENCH AND RUN AN EXPERIMENT

6910
05:04:18,869 --> 05:04:22,740
OR AT LEAST DESIGN, EXPERIMENTS

6911
05:04:22,740 --> 05:04:24,875
FOR CROs TO BE ABLE TO DO.

6912
05:04:24,875 --> 05:04:27,912
SO WE REALLY STRADDLE BOTH

6913
05:04:27,912 --> 05:04:30,281
SCIENTIFIC ASPECT OF EARLY STAGE

6914
05:04:30,281 --> 05:04:32,249
EXPERIMENTS THAT WE THINK WILL

6915
05:04:32,249 --> 05:04:34,719
GENERATE VALUE, TO VALIDATE THE

6916
05:04:34,719 --> 05:04:35,886
TECHNOLOGY AS WELL AS THE

6917
05:04:35,886 --> 05:04:37,621
BUSINESS SIDE OF THINGS TO MAKE

6918
05:04:37,621 --> 05:04:39,890
SURE THAT THE COMPANY IS BUILT

6919
05:04:39,890 --> 05:04:45,997
ON A SUSTAINABLE BASIS FOR THE

6920
05:04:45,997 --> 05:04:46,263
LONG-TERM.

6921
05:04:46,263 --> 05:04:50,167
SO ON THE NEXT SLIDE, NEXT

6922
05:04:50,167 --> 05:04:51,369
SLIDE, PLEASE, I WOULD LIKE TO

6923
05:04:51,369 --> 05:04:53,871
GIVE YOU A ERROR HIGH LEVEL VIEW

6924
05:04:53,871 --> 05:04:55,873
OF IF YOU HAVE AN ASSET, OR IF

6925
05:04:55,873 --> 05:04:58,376
YOU HAVE A PLATFORM TECHNOLOGY,

6926
05:04:58,376 --> 05:05:01,112
HOW IF YOU BRING IT TO A BIOTECH

6927
05:05:01,112 --> 05:05:02,747
VEBTURE FUND, HOW THEY WOULD

6928
05:05:02,747 --> 05:05:05,149
EVALUATE YOUR ASSET OR

6929
05:05:05,149 --> 05:05:05,449
TECHNOLOGY.

6930
05:05:05,449 --> 05:05:07,785
THIS OF COURSE IS NOT APPLICABLE

6931
05:05:07,785 --> 05:05:08,986
TO EVERY SINGLE TYPE OF

6932
05:05:08,986 --> 05:05:09,920
TECHNOLOGY BUT JUST TO IMIF YOU

6933
05:05:09,920 --> 05:05:13,457
A SENSE OF HOW WE VIEW

6934
05:05:13,457 --> 05:05:14,158
EVERYTHING, THERE'S DEPENDING ON

6935
05:05:14,158 --> 05:05:16,927
THE STAIM THAT YOU'RE AT,

6936
05:05:16,927 --> 05:05:18,095
ASSUMING PRECLINICAL, THE

6937
05:05:18,095 --> 05:05:20,931
BUCKETS THAT WE CARE A LOT ABOUT

6938
05:05:20,931 --> 05:05:23,167
FOR 1 IS, IF THERE IS A

6939
05:05:23,167 --> 05:05:24,535
PARTICULAR ASSET, IT'S REALLY

6940
05:05:24,535 --> 05:05:27,138
THE DISEASE BIOLOGY, AND JUST

6941
05:05:27,138 --> 05:05:30,107
KIND OF GOING THROUGH HOW

6942
05:05:30,107 --> 05:05:31,842
BIOTECH THINKS ABOUT THINGS, 1

6943
05:05:31,842 --> 05:05:33,978
THING IS OF COURSE IS THE DEC

6944
05:05:33,978 --> 05:05:35,913
WELL DEFINED AND BY THAT I MEAN,

6945
05:05:35,913 --> 05:05:38,182
HAVE THERE BEEN PRIOR STUDIES

6946
05:05:38,182 --> 05:05:40,651
AROUND THIS PARTICULAR DISEASE,

6947
05:05:40,651 --> 05:05:42,319
DO WE UNDERSTAND THE BLACK AND

6948
05:05:42,319 --> 05:05:44,488
WHITE NATURE OF HOW DO YOU KNOW

6949
05:05:44,488 --> 05:05:48,659
IF YOU'RE SICK, WHAT'S THE

6950
05:05:48,659 --> 05:05:50,361
DEMARCATION BETWEEN HAVING I

6951
05:05:50,361 --> 05:05:52,263
GUESS FULLY HAVING A PARTICULAR

6952
05:05:52,263 --> 05:05:54,098
DISEASE VERSUS NOT, AND THEN

6953
05:05:54,098 --> 05:05:56,200
HAVING DIFFERENT TYPES OF

6954
05:05:56,200 --> 05:05:58,702
CONVICTION TO PERHAPS STRATIFY

6955
05:05:58,702 --> 05:06:00,871
YOUR PATIENT POPULATION SO YOU

6956
05:06:00,871 --> 05:06:02,173
CAN -- YOUR DRUG COULD BE ARK

6957
05:06:02,173 --> 05:06:03,574
PLIED TO THE PATIENTS THAT WILL

6958
05:06:03,574 --> 05:06:07,978
MOST LIKELY SHOW A RESPONSE, SO,

6959
05:06:07,978 --> 05:06:09,747
IN THAT CASE WE OFTEN THINK

6960
05:06:09,747 --> 05:06:10,948
ABOUT HUMAN GENETICS OR IF

6961
05:06:10,948 --> 05:06:13,784
THERE'S OTHER WAYS FOR US TO

6962
05:06:13,784 --> 05:06:17,421
PARSE PATIENTS INTO DIFFERENT

6963
05:06:17,421 --> 05:06:17,721
CATEGORIES.

6964
05:06:17,721 --> 05:06:19,123
ONE THING IN ADDITION TO THAT

6965
05:06:19,123 --> 05:06:21,358
JUST GOING BEYOND DEC BIOLOGY IS

6966
05:06:21,358 --> 05:06:24,528
UNDERSTANDING IF THERE'S A GOOD

6967
05:06:24,528 --> 05:06:26,030
MODEL, WHETHER INVITRO OR IN

6968
05:06:26,030 --> 05:06:27,631
VIVO, FOR US TO BETTER

6969
05:06:27,631 --> 05:06:31,869
UNDERSTAND THE DISEASE AND

6970
05:06:31,869 --> 05:06:33,637
WHETHER OR NOT YOUR KRUG IF IT

6971
05:06:33,637 --> 05:06:35,139
WORKS IN A DISH, IT WILL WORK IN

6972
05:06:35,139 --> 05:06:35,873
A PERSON.

6973
05:06:35,873 --> 05:06:37,074
THAT'S OF COURSE A VERY HIGH BAR

6974
05:06:37,074 --> 05:06:39,844
AND I WILL SAY AS A PRIOR

6975
05:06:39,844 --> 05:06:40,945
TRAINED NEUROSCIENTIST, THERE'S

6976
05:06:40,945 --> 05:06:43,981
VERY FEW GOOD MODELS OF

6977
05:06:43,981 --> 05:06:44,815
NEURODEGENERATIVE DISEASE FOR

6978
05:06:44,815 --> 05:06:47,885
EXAMPLE, BUT IN THE CASE OF

6979
05:06:47,885 --> 05:06:49,987
CYSTIC FIBROSIS, THERE'S A

6980
05:06:49,987 --> 05:06:51,122
FANTASTIC INVITRO MODEL DISEASE

6981
05:06:51,122 --> 05:06:53,858
THAT REALLY GIVES YOU INSIGHT

6982
05:06:53,858 --> 05:06:55,826
INTO HOW A DRUG COULD WORK AND

6983
05:06:55,826 --> 05:06:58,129
YOU KNOW THAT OF COURSE DRIVES A

6984
05:06:58,129 --> 05:07:01,298
LOT OF WHERE YOU SEE MORE DRUG

6985
05:07:01,298 --> 05:07:04,568
DISCOVERY FOR AREAS OF UNMET

6986
05:07:04,568 --> 05:07:04,768
NEED.

6987
05:07:04,768 --> 05:07:10,708
AND THEN, IN  TERMS OF ADDITIONL

6988
05:07:10,708 --> 05:07:12,910
THOUGHTS, THE WAY THAT EARLY

6989
05:07:12,910 --> 05:07:15,012
BIOTECH VENTURE GROUPS THINK IS

6990
05:07:15,012 --> 05:07:16,247
REALLY ON THE CLINICAL

6991
05:07:16,247 --> 05:07:18,716
DEVELOPMENT WHICH THEN BUILDS

6992
05:07:18,716 --> 05:07:21,185
INTO THE LAST ROW HERE WHICH IS

6993
05:07:21,185 --> 05:07:21,785
COMPANY BUILD.

6994
05:07:21,785 --> 05:07:23,721
ONE ASPECT IS THAT THESE

6995
05:07:23,721 --> 05:07:25,456
COMPANIES, YOU RAISE VERY SMALL

6996
05:07:25,456 --> 05:07:26,790
AMOUNTS OF CAPITAL ASK THEN YOU

6997
05:07:26,790 --> 05:07:28,159
END UP RAISING MORE AND MORE

6998
05:07:28,159 --> 05:07:30,127
MONEY AS YOU'RE CLOSER TO THE

6999
05:07:30,127 --> 05:07:32,463
CLINIC OR YOU'RE ALREADY IN THE

7000
05:07:32,463 --> 05:07:32,696
CLINIC.

7001
05:07:32,696 --> 05:07:33,731
AND SO, 1 THING THAT YOU WILL

7002
05:07:33,731 --> 05:07:36,400
PROBABLY NOTICE IS THAT THERE'S

7003
05:07:36,400 --> 05:07:39,570
FEWER BIOTECHS WORKING ON VERY,

7004
05:07:39,570 --> 05:07:40,838
VERY LARGE INDICATIONS, WHICH IN

7005
05:07:40,838 --> 05:07:43,140
A SENSE IS KIND OF A MAJOR

7006
05:07:43,140 --> 05:07:44,675
LIMITATION THAT THE FIELD HASN'T

7007
05:07:44,675 --> 05:07:48,112
REALLY  SOLVED.

7008
05:07:48,112 --> 05:07:50,414
SO VERY FEW BIOTECHS WORKING ON

7009
05:07:50,414 --> 05:07:51,682
HEART FAILURE FOR EXAMPLE, GIVEN

7010
05:07:51,682 --> 05:07:53,984
THAT IT'S A VERY LARGE AND

7011
05:07:53,984 --> 05:07:54,852
HETEROGENEOUS POPULATION.

7012
05:07:54,852 --> 05:07:56,020
IT WILL TAKE MANY YEARS ISSUES

7013
05:07:56,020 --> 05:07:58,656
IT'S DIFFICULT TO GET THAT FIRST

7014
05:07:58,656 --> 05:07:59,456
PROOF OF CONCEPT.

7015
05:07:59,456 --> 05:08:05,529
AND THEN, AT THE SAME TIME ASIDE

7016
05:08:05,529 --> 05:08:07,364
FROM CLINICAL DEVELOPMENT WE DO

7017
05:08:07,364 --> 05:08:09,466
WANT TO HAVE A VIEW ON WHAT DOES

7018
05:08:09,466 --> 05:08:10,534
SUCCESS LOOK LIKE, WHAT IS A

7019
05:08:10,534 --> 05:08:12,636
MARKER YOU NEED TO REACH OR WHAT

7020
05:08:12,636 --> 05:08:17,007
IS SOMETHING THAT CAN TELL YOU

7021
05:08:17,007 --> 05:08:19,076
OR HELP YOU, PUSH YOU TOWARDS

7022
05:08:19,076 --> 05:08:20,477
THE LIKELIHOOD OF SUCCESS

7023
05:08:20,477 --> 05:08:22,413
TOWARDS THE POSITIVE END.

7024
05:08:22,413 --> 05:08:25,282
AND THEN, AT THE SAME TIME,

7025
05:08:25,282 --> 05:08:26,717
COMPETITION, OF COURSE, THIS IS

7026
05:08:26,717 --> 05:08:29,386
SOMETHING THAT'S A LITTLE BIT

7027
05:08:29,386 --> 05:08:32,690
MORE DIFFICULT TO STRICTLY

7028
05:08:32,690 --> 05:08:36,060
QUANTIFY BUT 1 ASPECT IS FOR

7029
05:08:36,060 --> 05:08:39,296
EXAMPLE, STANDARD OF CARE AND

7030
05:08:39,296 --> 05:08:42,032
WHETHER EVERY SINGLE TREATMENT

7031
05:08:42,032 --> 05:08:45,402
FOR EXAMPLE, IS ALREADY ALL

7032
05:08:45,402 --> 05:08:47,438
PATENT, COST PENNY SAYS, MAYBE

7033
05:08:47,438 --> 05:08:49,206
THERE'S SOME UNMET NEED LEFT BUT

7034
05:08:49,206 --> 05:08:50,174
NOT TERRIBLY MUCH, YOU CAN

7035
05:08:50,174 --> 05:08:53,677
IMAGINE HOW IF THAT'S A NO

7036
05:08:53,677 --> 05:08:56,013
ACROSS THE BOARD, OR A YES

7037
05:08:56,013 --> 05:08:57,715
ACROSS THE BOARD, IT BECOMES

7038
05:08:57,715 --> 05:08:59,717
MUCH MORE DIFFICULT FOR EARLY

7039
05:08:59,717 --> 05:09:02,553
STAGE INVESTORS TO GET EXCITED.

7040
05:09:02,553 --> 05:09:04,822
AND THEN, EVERYTHING ELSE KIND

7041
05:09:04,822 --> 05:09:07,091
OF GOES INTO THE ADDRESSABLE

7042
05:09:07,091 --> 05:09:09,727
MARKET AND THE PRICE OF YOUR

7043
05:09:09,727 --> 05:09:11,095
POTENTIAL DRUG DOWN THE ROAD AND

7044
05:09:11,095 --> 05:09:15,299
HOW MANY PEOPLE IT WILL HELP AND

7045
05:09:15,299 --> 05:09:17,234
SO, REALLY PUTTING EVERYTHING

7046
05:09:17,234 --> 05:09:19,937
TOGETHER, YOU WILL SEE THAT

7047
05:09:19,937 --> 05:09:25,542
THERE IS MAYBE A PARTICULAR

7048
05:09:25,542 --> 05:09:27,244
NICHE THAT BIOTECH INVESTORS

7049
05:09:27,244 --> 05:09:29,747
FOCUS ON AND SO FOR ANY ASSET OR

7050
05:09:29,747 --> 05:09:31,715
PLATFORM TECHNOLOGY THAT YOU MAY

7051
05:09:31,715 --> 05:09:35,886
BE DEVELOPING, YOU CAN SEE HOW

7052
05:09:35,886 --> 05:09:38,789
THERE'S PERHAPS A WAY TO EITHER

7053
05:09:38,789 --> 05:09:39,957
TAYLOR THE RESEARCH OR WEAVE

7054
05:09:39,957 --> 05:09:40,924
YOUR STORY.

7055
05:09:40,924 --> 05:09:44,128
SO THERE'S MULTIPLE YESS FOR THE

7056
05:09:44,128 --> 05:09:46,697
FIRST FEW BUCKETS, AND AS WELL

7057
05:09:46,697 --> 05:09:48,666
GETTING A SENSE OF HOW MANY

7058
05:09:48,666 --> 05:09:50,367
YEARS IT WILL TAKE FOR YOUR

7059
05:09:50,367 --> 05:09:55,839
PARTICULAR PROGRAM TO GET TO A

7060
05:09:55,839 --> 05:09:57,808
STAGE THAT VALIDATES SOMEONE'S

7061
05:09:57,808 --> 05:09:58,108
INVESTMENT.

7062
05:09:58,108 --> 05:10:01,312
AND YOU KNOW ON THE RIGHT HAND

7063
05:10:01,312 --> 05:10:03,614
SIDE JUST KIND OF HIGHLIGHTING

7064
05:10:03,614 --> 05:10:06,383
THAT IF THERE'S MULTIPLE NOs

7065
05:10:06,383 --> 05:10:09,987
OF A LIMITING INTEREST AND THEN,

7066
05:10:09,987 --> 05:10:11,922
IF THE ANSWERS, I DON'T KNOW

7067
05:10:11,922 --> 05:10:14,792
THAT ALSO MAKES THINGS A LITTLE

7068
05:10:14,792 --> 05:10:16,794
BIT MORE DIFFICULT.

7069
05:10:16,794 --> 05:10:18,295
BUT IN SPEAKING WITH VENTURE

7070
05:10:18,295 --> 05:10:20,864
CREATION TYPE OF FUNDS AS OPPOSE

7071
05:10:20,864 --> 05:10:24,068
TO JUST VENTURE INVESTORS,

7072
05:10:24,068 --> 05:10:29,206
GENERALLY, IF THE TECHNOLOGY IS

7073
05:10:29,206 --> 05:10:30,808
VERY INTERESTING, AND COULD

7074
05:10:30,808 --> 05:10:31,542
UNLOCK SOMETHING SPECIAL,

7075
05:10:31,542 --> 05:10:33,210
VENTURE GROUPS WOULD LIKE TO

7076
05:10:33,210 --> 05:10:35,045
HAVE JUST AN OPEN BRAINSTORM

7077
05:10:35,045 --> 05:10:38,515
TYPE OF CONVERSATION TO

7078
05:10:38,515 --> 05:10:39,683
UNDERSTAND WHERE YOUR TECHNOLOGY

7079
05:10:39,683 --> 05:10:42,086
COULD BE APPLIED SO IT'S

7080
05:10:42,086 --> 05:10:43,487
DEFINITELY, YOU NEED TO DO ALL

7081
05:10:43,487 --> 05:10:44,888
THIS WORK YOURSELF AHEAD OF TIME

7082
05:10:44,888 --> 05:10:46,724
BEFORE TALKING TO SOMEONE, IT'S

7083
05:10:46,724 --> 05:10:48,826
REALLY A -- LET'S BRAINSTORM AND

7084
05:10:48,826 --> 05:10:51,095
FIGURE OUT WHERE THEIR COULD BE

7085
05:10:51,095 --> 05:10:54,598
APPLICATIONS AND SO, THOSE OF US

7086
05:10:54,598 --> 05:10:57,668
AT LONG WOOD AND IN GENERAL MOST

7087
05:10:57,668 --> 05:10:58,736
CREATION VENTURE FUNDS ARE HAPPY

7088
05:10:58,736 --> 05:11:00,637
TO HAVE THAT CONVERSATION JUST

7089
05:11:00,637 --> 05:11:01,605
AS A BRAINSTORM BECAUSE AT THE

7090
05:11:01,605 --> 05:11:03,574
SAME TIME FOR US, IF SOMETHING'S

7091
05:11:03,574 --> 05:11:04,875
NOT READY NOW IT DOESN'T MEAN

7092
05:11:04,875 --> 05:11:07,044
THAT IT'S NEVER GOING TO BE

7093
05:11:07,044 --> 05:11:07,945
READY.

7094
05:11:07,945 --> 05:11:11,181
EVERYTHING IS TIMING DEPENDENT.

7095
05:11:11,181 --> 05:11:15,185
SO WITH ALL OF THAT SAID, NEXT

7096
05:11:15,185 --> 05:11:16,120
SLIDE, PLEASE.

7097
05:11:16,120 --> 05:11:21,625
I WANT TO GIVE JUST A VIEW INTO

7098
05:11:21,625 --> 05:11:23,160
HOW WE'VE APPLIED THIS IN THE

7099
05:11:23,160 --> 05:11:23,460
PAST.

7100
05:11:23,460 --> 05:11:26,764
SO, TO GIVE YOU A TANGIBLE

7101
05:11:26,764 --> 05:11:30,134
EXAMPLE, THERE'S I THINK SEVERAL

7102
05:11:30,134 --> 05:11:31,268
PEOPLE FROM CARBON BIOSCIENCES

7103
05:11:31,268 --> 05:11:34,805
HERE, THIS A COMPANY THAT WE

7104
05:11:34,805 --> 05:11:38,342
BUILT 2 YEARS AGO CONFOUNDED,

7105
05:11:38,342 --> 05:11:41,578
PROFESSORS JOHN INGLE HATTER AND

7106
05:11:41,578 --> 05:11:42,646
ROCK COTTON AND TOO KIND OF

7107
05:11:42,646 --> 05:11:44,047
BRING YOU ALL THE WAY BACK.

7108
05:11:44,047 --> 05:11:46,283
ONE OF THE AREAS WE HAD FOCUSED

7109
05:11:46,283 --> 05:11:49,553
ON, MAYBE 3 YEARS AGO, WAS

7110
05:11:49,553 --> 05:11:53,490
WANTING TO START AN EXPLORATION

7111
05:11:53,490 --> 05:11:55,559
AROUND GENE DELIVERY OR EFFORTS

7112
05:11:55,559 --> 05:11:59,363
AROUND HOW 1 DELIVERS A GENE.

7113
05:11:59,363 --> 05:12:01,565
THIS QUICKLY FOR US SORTED INTO

7114
05:12:01,565 --> 05:12:02,866
A FEW DIFFERENT CATEGORIES, SO

7115
05:12:02,866 --> 05:12:04,668
IN THE WORLD OF GENE DELIVERY,

7116
05:12:04,668 --> 05:12:07,271
THERE'S OF COURSE VIRAL,

7117
05:12:07,271 --> 05:12:10,140
NONVIRAL, THERE'S ALSO THINKING

7118
05:12:10,140 --> 05:12:13,877
DEEPLY ABOUT WHETHER IT'S THE

7119
05:12:13,877 --> 05:12:16,513
CALCIT OR ENVELOPE OR THE CARGO

7120
05:12:16,513 --> 05:12:16,780
ITSELF.

7121
05:12:16,780 --> 05:12:18,615
AND GENERALLY FOR A NEW COMPANY,

7122
05:12:18,615 --> 05:12:22,986
WE LIKE TO ARE 1 AREA OF

7123
05:12:22,986 --> 05:12:24,521
INNOVATION THAT WE REALLY FOCUS

7124
05:12:24,521 --> 05:12:26,657
ON WHERE EVERYTHING ELSE KIND OF

7125
05:12:26,657 --> 05:12:30,127
AT AS MORE MAYBE TRADITIONAL OR

7126
05:12:30,127 --> 05:12:31,161
STANDARD OR WELL-VALIDATED.

7127
05:12:31,161 --> 05:12:35,032
SO IN PARSING OUT THE WORLD INTO

7128
05:12:35,032 --> 05:12:37,768
THESE 4 CATEGORIES, WE STARTED

7129
05:12:37,768 --> 05:12:39,503
THINKING ABOUT, NOT JUST WHO WE

7130
05:12:39,503 --> 05:12:42,072
COULD TALK TO TO DISCOVER MORE

7131
05:12:42,072 --> 05:12:44,208
ABOUT THESE TECHNOLOGIES AND

7132
05:12:44,208 --> 05:12:48,078
APPROACHES, BUT MORE SO ON IF WE

7133
05:12:48,078 --> 05:12:52,216
HAD THE IDEAL, WHETHER IT'S

7134
05:12:52,216 --> 05:12:55,652
CARGO OR ENVELOPE OR VIRAL OR

7135
05:12:55,652 --> 05:12:58,121
NONVIRAL APPROACH, WHAT IS A

7136
05:12:58,121 --> 05:13:00,390
DISEASE THAT COULD BE APPLICABLE

7137
05:13:00,390 --> 05:13:01,992
HERE WHERE WE WOULD FIT MANY OF

7138
05:13:01,992 --> 05:13:04,895
THE CRITERIA ON THE PRIOR SLIDE

7139
05:13:04,895 --> 05:13:05,696
THAT I MENTIONED.

7140
05:13:05,696 --> 05:13:08,565
WHERE THE DISEASE IS WELL

7141
05:13:08,565 --> 05:13:09,833
UNDERSTOOD, THERE'S BIOMARKER OR

7142
05:13:09,833 --> 05:13:11,635
NATURAL HISTORY OR OTHER METRICS

7143
05:13:11,635 --> 05:13:14,404
FOR US TO KNOW WHAT GOOD LOOKS

7144
05:13:14,404 --> 05:13:17,207
LIKE AND THEN AT THE SAME TIME

7145
05:13:17,207 --> 05:13:20,644
HAS A HIGH UNMET NEED FOR US TO

7146
05:13:20,644 --> 05:13:22,379
REALLY BE ABLE TO FIND PATIENTS

7147
05:13:22,379 --> 05:13:25,449
IN A REASONABLE AMOUNT OF TIME

7148
05:13:25,449 --> 05:13:26,984
TO SHOW SIGNS OF EFFICACY.

7149
05:13:26,984 --> 05:13:30,287
SO KIND OF MOVING THROUGH THE

7150
05:13:30,287 --> 05:13:35,893
EVALUATION AND SPEAKING WITH A

7151
05:13:35,893 --> 05:13:37,528
NUMBER OF SCIENTISTS WORKING IN

7152
05:13:37,528 --> 05:13:40,297
DIFFERENT AREAS OF BIOLOGY, WE

7153
05:13:40,297 --> 05:13:47,938
TRIANGULATED ON 2 THINGS. 

7154
05:13:47,938 --> 05:13:49,773
WE TRIANGULATED ON THE

7155
05:13:49,773 --> 05:13:52,943
[INDISCERNIBLE] TECHNOLOGY BEING

7156
05:13:52,943 --> 05:13:53,977
DEVELOPED AT THE ENGLAND

7157
05:13:53,977 --> 05:13:57,915
JOURNALLE HEART LAB AND THIS WAS

7158
05:13:57,915 --> 05:13:58,749
ALSO GEARED TOWARDS

7159
05:13:58,749 --> 05:13:59,116
CYSTIC FIBROSIS.

7160
05:13:59,116 --> 05:14:00,050
THIS IS EXCITING FOR US BECAUSE

7161
05:14:00,050 --> 05:14:04,388
IN THE WORLD OF THINKING ABOUT

7162
05:14:04,388 --> 05:14:05,222
CYSTIC FIBROSIS AS ENRIQUE JUST

7163
05:14:05,222 --> 05:14:06,523
MENTIONED IN THE PRIOR PANEL

7164
05:14:06,523 --> 05:14:09,459
THIS, 

7165
05:14:09,459 --> 05:14:13,664
THIS IS A DISEASE THAT HAS VERY

7166
05:14:13,664 --> 05:14:15,699
WELL UNDERSTOOD GENETICS,

7167
05:14:15,699 --> 05:14:17,034
INVITRO MODELING THAT GIVES YOU

7168
05:14:17,034 --> 05:14:19,202
A SENSE OF WHAT SUCCESS COULD

7169
05:14:19,202 --> 05:14:22,806
LOOK LIKE AND THERE'S ALSO A

7170
05:14:22,806 --> 05:14:26,443
DEEP UNDERSTANDING OF YEEN

7171
05:14:26,443 --> 05:14:28,512
THERAPY AND WHAT BOCA VIRUSES

7172
05:14:28,512 --> 05:14:30,681
WHICH HAS A LARGER

7173
05:14:30,681 --> 05:14:32,616
CARBOCAPACKITY COULD OPEN UP FOR

7174
05:14:32,616 --> 05:14:34,318
DISEASE WHERE EXISTING

7175
05:14:34,318 --> 05:14:37,487
APPROACHES HAVE VERY SMALL

7176
05:14:37,487 --> 05:14:38,622
TRUNCATED VERSIONS OF THE

7177
05:14:38,622 --> 05:14:39,990
CYSTIC FIBROSIS GENE BEING

7178
05:14:39,990 --> 05:14:42,526
DELIVERED WHICH HAS ITS JUST

7179
05:14:42,526 --> 05:14:44,227
BIOLOGICAL LIMITATIONS GIVEN

7180
05:14:44,227 --> 05:14:46,863
THAT IT'S SYNTHETIC TO AN

7181
05:14:46,863 --> 05:14:47,097
EXTENT.

7182
05:14:47,097 --> 05:14:51,234
AND SO ALL OF THESE THINGS

7183
05:14:51,234 --> 05:14:53,136
CULMINATED IN US GETTING EXCITED

7184
05:14:53,136 --> 05:14:55,906
ABOUT A PRIMARY ARCS SET AND

7185
05:14:55,906 --> 05:14:57,741
THEN AT THE SAME TIME WE ALWAYS

7186
05:14:57,741 --> 05:15:01,111
KNOWING THAT IN EARLY STAGE

7187
05:15:01,111 --> 05:15:02,613
THERE'S A HIGH FAILURE RATE WE

7188
05:15:02,613 --> 05:15:04,514
WANT THERE TO BE A PLATFORM

7189
05:15:04,514 --> 05:15:07,117
TECHNOLOGY THAT ALLOWS US TO

7190
05:15:07,117 --> 05:15:10,120
BUILD MULTIPLE OTHER PROGRAMS

7191
05:15:10,120 --> 05:15:11,555
THAT HAD A SIMILAR THESEIS

7192
05:15:11,555 --> 05:15:14,057
AROUND IT SO THAT'S WHAT ALLOWED

7193
05:15:14,057 --> 05:15:16,693
US TO BRING IN TECHNOLOGIES FROM

7194
05:15:16,693 --> 05:15:19,196
ROB COTTON, WHERE THERE'S A

7195
05:15:19,196 --> 05:15:21,164
NUMBER OF DIFFERENT PARVE O

7196
05:15:21,164 --> 05:15:22,933
VIRUSES THAT HAVE SIMILAR

7197
05:15:22,933 --> 05:15:24,334
FEATURES TO BOCA VIRUS THAT CAN

7198
05:15:24,334 --> 05:15:25,669
ALL BE MANUFACTURED IN A SIMILAR

7199
05:15:25,669 --> 05:15:28,739
WAY EMPLOY SO, IN ALL OF THIS,

7200
05:15:28,739 --> 05:15:32,242
WE ENDED UP DECIDING TO GO

7201
05:15:32,242 --> 05:15:34,177
FORWARD WITH A COMPANY BUILD

7202
05:15:34,177 --> 05:15:35,512
EMPLOY BUILD -- FIND A GROUP OF

7203
05:15:35,512 --> 05:15:37,214
INVESTOR WHO IS ARE LIKE MINDED

7204
05:15:37,214 --> 05:15:43,820
AND SEE THE VALUE IN THIS NOVEL

7205
05:15:43,820 --> 05:15:45,989
WAY OF APPROACHING GENE DELIVERY

7206
05:15:45,989 --> 05:15:49,493
BUT STILL MAINTAINING THE

7207
05:15:49,493 --> 05:15:51,061
SIMILAR STRUCTURE OF HOW THINGS

7208
05:15:51,061 --> 05:15:52,496
WILL BE DONE.

7209
05:15:52,496 --> 05:15:53,196
FOR EXAMPLE, MANUFACTURING

7210
05:15:53,196 --> 05:15:56,266
REMAINS NEARLY THE SAME AS

7211
05:15:56,266 --> 05:15:57,868
EXISTING PRIOR APPROACHES AND

7212
05:15:57,868 --> 05:16:00,804
THESE REALLY MATTER FOR TALKING

7213
05:16:00,804 --> 05:16:02,005
TO PHARMAS AND OTHER GROUPS WHO

7214
05:16:02,005 --> 05:16:05,942
WILL HELP WITH THAT LATER STAGE

7215
05:16:05,942 --> 05:16:08,378
DEVELOPMENT AND

7216
05:16:08,378 --> 05:16:10,280
COMMERCIALIZATION OF A POTENTIAL

7217
05:16:10,280 --> 05:16:10,547
PRODUCT.

7218
05:16:10,547 --> 05:16:12,949
SO YOU KNOW, THIS IS OF COURSE

7219
05:16:12,949 --> 05:16:15,352
MANY MONTHS OF WORK FROM A

7220
05:16:15,352 --> 05:16:16,453
COMPRESSED AND CONDENSED INTO A

7221
05:16:16,453 --> 05:16:18,789
SINGLE SLIDE BUT HOPEFULFULLY

7222
05:16:18,789 --> 05:16:19,956
THAT'S TANGIBLE VIEWING TO HOW

7223
05:16:19,956 --> 05:16:23,326
COMPANIES REALLY GET STARTED AND

7224
05:16:23,326 --> 05:16:25,195
1 APPROACH FROM, YOU KNOW THE

7225
05:16:25,195 --> 05:16:27,597
VENTURE SIDE OF HOW WE START

7226
05:16:27,597 --> 05:16:29,533
COMPANIES WHICH COULD BE A

7227
05:16:29,533 --> 05:16:31,568
LITTLE BIT DIFFERENT THAN HOW

7228
05:16:31,568 --> 05:16:34,171
OTHER GROUPS OR OTHER TYPES OF

7229
05:16:34,171 --> 05:16:38,642
COMPANY CREATION KIND OF OCCURS.

7230
05:16:38,642 --> 05:16:43,780
SO NEXT SLIDE, PLEASE.

7231
05:16:43,780 --> 05:16:45,549
TO REALLY WRAP IT UP, I WANT TO

7232
05:16:45,549 --> 05:16:50,587
JUST HIGHLIGHT THAT IN THE WORLD

7233
05:16:50,587 --> 05:16:52,689
OF NEW CO CREATION FROM A

7234
05:16:52,689 --> 05:16:53,757
VENTURE PERSPECTIVE, I'M JUST

7235
05:16:53,757 --> 05:16:55,158
HIGHLIGHTING THE COMPANIES THAT

7236
05:16:55,158 --> 05:16:56,893
WE HAVE INVESTED IN AND THAT WE

7237
05:16:56,893 --> 05:16:59,162
HAVE BUILT, THE 1S THAT HAS THE

7238
05:16:59,162 --> 05:17:01,832
GREEN BOX ON THE BOTTOM OR 1S

7239
05:17:01,832 --> 05:17:02,966
THAT WE'VE CONFOUNDED, I THINK

7240
05:17:02,966 --> 05:17:04,701
IN NEARLY ALL OF THESE CASES,

7241
05:17:04,701 --> 05:17:10,540
WITH AN ACADEMIC FOUNDER AS THE

7242
05:17:10,540 --> 05:17:11,408
SCIENTISTS, KEY SCIENTISTS

7243
05:17:11,408 --> 05:17:14,411
BEHIND THESE TECHNOLOGIES AND

7244
05:17:14,411 --> 05:17:15,479
THAT'S ESSENTIALLY THE MAIORITY

7245
05:17:15,479 --> 05:17:17,614
OF WHAT WE DO, WE LOOK FOR

7246
05:17:17,614 --> 05:17:19,750
EXCITING NOVEL BIOLOGY OR

7247
05:17:19,750 --> 05:17:22,486
EXCITING NEW TECHNOLOGY THAT CAN

7248
05:17:22,486 --> 05:17:24,488
SERVE TO TRANSFORM A PARTICULAR

7249
05:17:24,488 --> 05:17:27,190
AREA OR UNLOCK SOMETHING UNIQUE

7250
05:17:27,190 --> 05:17:30,594
TO GIVE US AN ANGLE INTO THE

7251
05:17:30,594 --> 05:17:33,430
MARKET AND YOU KNOW I THINK FOR

7252
05:17:33,430 --> 05:17:36,500
US, WHEN WE SEEK OUT TO MEET AND

7253
05:17:36,500 --> 05:17:38,168
TALK TO DIFFERENT WHETHER IT'S

7254
05:17:38,168 --> 05:17:40,003
PROFESSORS OR LICENSING GROUPS

7255
05:17:40,003 --> 05:17:41,538
OR OTHERS, IT'S REALLY JUST A

7256
05:17:41,538 --> 05:17:43,306
BETTER UNDERSTAND THE STATE OF

7257
05:17:43,306 --> 05:17:46,143
THE CURRENT FIELD AND ALSO TO

7258
05:17:46,143 --> 05:17:48,211
GATHER A GROUP OF LIKE MINDED

7259
05:17:48,211 --> 05:17:50,313
SCIENTISTS WITH A SIMILAR GOAL

7260
05:17:50,313 --> 05:17:51,815
IN MIND, TO BRAINSTORM AND SEE

7261
05:17:51,815 --> 05:17:54,484
IF THERE'S SOMETHING THAT WE CAN

7262
05:17:54,484 --> 05:17:55,852
BUILD TOGETHER.

7263
05:17:55,852 --> 05:17:59,556
SO HOPEFULLY, THAT GIVES YOU A

7264
05:17:59,556 --> 05:18:01,491
SENSE OF HOW, AT LEAST MOST OF

7265
05:18:01,491 --> 05:18:04,227
US AT LONG WOOD AND EARLY STAGE

7266
05:18:04,227 --> 05:18:05,595
VENTURE CREATION FIRMS THINK

7267
05:18:05,595 --> 05:18:08,398
ABOUT TECHNOLOGIES AND THINK

7268
05:18:08,398 --> 05:18:13,236
ABOUT COMPANY CREATION AND SOME

7269
05:18:13,236 --> 05:18:15,238
EXAMPLES OF COMPANIES WE'VE

7270
05:18:15,238 --> 05:18:18,742
BUILT IN THE PAST THAT ARE BEEN

7271
05:18:18,742 --> 05:18:19,943
SUCCESSFUL AND BROUGHT DRUGS TO

7272
05:18:19,943 --> 05:18:20,210
PATIENTS.

7273
05:18:20,210 --> 05:18:22,245
AND IF ANY OF YOU WOULD LIKE TO

7274
05:18:22,245 --> 05:18:23,513
TALK ABOUT COMPANY CREATION OR

7275
05:18:23,513 --> 05:18:24,681
TALK ABOUT YOUR TECHNOLOGY

7276
05:18:24,681 --> 05:18:26,183
YOU'RE DEVELOPING WHETHER

7277
05:18:26,183 --> 05:18:27,884
THERE'S IP BEHIND IT, MAYBE A

7278
05:18:27,884 --> 05:18:30,787
PAPER ABOUT TO BE PUBLISHED,

7279
05:18:30,787 --> 05:18:40,964
PLEASE REACH OUT, WOULD LOVE TO

7280
05:18:40,964 --> 05:18:43,700
HAVE THAT CONVERSATION.

7281
05:18:43,700 --> 05:18:45,101
>> THANK YOU VERY MUCH.

7282
05:18:45,101 --> 05:18:46,837
THE FINAL SPEAK OF THIS SERIES

7283
05:18:46,837 --> 05:18:51,575
WILL BE DR. BENOIT, HE HAS WITH

7284
05:18:51,575 --> 05:18:53,877
MDI THERAPEUTICS HE WILL SPEAK

7285
05:18:53,877 --> 05:18:55,278
ON START-AUTOPSIES AND COMPANIES

7286
05:18:55,278 --> 05:18:56,880
IN THE PHARMACEUTICAL

7287
05:18:56,880 --> 05:18:57,347
INDUSTRIES.

7288
05:18:57,347 --> 05:18:57,714
>> THANK YOU.

7289
05:18:57,714 --> 05:18:58,181
APPRECIATE IT.

7290
05:18:58,181 --> 05:18:59,983
HOPEFULLY YOU CAN ALL HEAR ME

7291
05:18:59,983 --> 05:19:00,483
WELL ENOUGH.

7292
05:19:00,483 --> 05:19:03,353
ALSO HAPPY TO HAVE MY HONORARY

7293
05:19:03,353 --> 05:19:04,654
DOCTORAL ISSUED AGAIN, TODAY,

7294
05:19:04,654 --> 05:19:05,355
THAT'S GOOD.

7295
05:19:05,355 --> 05:19:07,157
I'M STILL HAPPY JUST TO BE ABLE

7296
05:19:07,157 --> 05:19:10,126
TO SPELL Ph.D., SO, STILL

7297
05:19:10,126 --> 05:19:16,633
WORKING FROM THE BUSINESS SIDE.

7298
05:19:16,633 --> 05:19:17,968
ALONG WITH MY COLLEAGUES FROM

7299
05:19:17,968 --> 05:19:20,971
UNIVERSITY OF MICHIGAN. 

7300
05:19:20,971 --> 05:19:21,671
NEXT SLIDE, PLEASE.

7301
05:19:21,671 --> 05:19:24,207
I WAS ASKED TO SORT OF GIVE YOU

7302
05:19:24,207 --> 05:19:33,683
A SENSE FOR HOW WE COMBINE BOTH

7303
05:19:33,683 --> 05:19:35,185
SEED CAPITAL, NIH FUNDING AND

7304
05:19:35,185 --> 05:19:36,486
VENTURE FUNDING TO GET OUR FIRST

7305
05:19:36,486 --> 05:19:38,989
ASSET INTO THE CLINIC AND SO,IME

7306
05:19:38,989 --> 05:19:40,490
GOING TO KIND OF START AT THE

7307
05:19:40,490 --> 05:19:42,292
BACK END WHICH GIVES YOU A QUICK

7308
05:19:42,292 --> 05:19:45,996
IDEA, WE'RE DEVELOPING A SMALL

7309
05:19:45,996 --> 05:19:50,300
MOLECULE FOR INTERSTITIAL LUNG

7310
05:19:50,300 --> 05:19:54,671
DISEASE BROADLY, IT'S A PI-1

7311
05:19:54,671 --> 05:19:59,042
INHIBITOR, PLAZ MIDS

7312
05:19:59,042 --> 05:20:00,410
MIDINHIBITTOR 1 INHIBITORS AND

7313
05:20:00,410 --> 05:20:02,279
LISTENING TO DR. HOLTZMAN AND

7314
05:20:02,279 --> 05:20:03,146
OTHERS HERE TODAY THINKING ABOUT

7315
05:20:03,146 --> 05:20:05,282
SOME OF THE KEY MODELS WE REALLY

7316
05:20:05,282 --> 05:20:09,452
WANTED TO DEMONSTRATE THAT WE

7317
05:20:09,452 --> 05:20:11,988
HAD EFFICACY AND SORT OF THE

7318
05:20:11,988 --> 05:20:13,957
STANDARD OF CARE, GOLD STANDARD

7319
05:20:13,957 --> 05:20:15,225
MODEL AGAINST THE STANDARD OF

7320
05:20:15,225 --> 05:20:18,962
CARE AND SOME OF THE PRIOR

7321
05:20:18,962 --> 05:20:20,230
COMPOUNDS THAT HAVE BEEN

7322
05:20:20,230 --> 05:20:22,198
DEVELOPED AND TAKEN INTO THE

7323
05:20:22,198 --> 05:20:24,501
CLINIC, BUT NOT ADVANCED AND WE

7324
05:20:24,501 --> 05:20:28,338
THINK WE HAVE INSIGHTS INTO WHY

7325
05:20:28,338 --> 05:20:29,639
THOSE COMPOUNDS WERE NOT

7326
05:20:29,639 --> 05:20:30,707
SUCCESSFUL, ONCE THEY MOVED INTO

7327
05:20:30,707 --> 05:20:33,343
THE CLINIC AND IT HAS TO DO A

7328
05:20:33,343 --> 05:20:36,613
LOT WITH THE SCREEN AND WHETHER

7329
05:20:36,613 --> 05:20:42,252
THOSE COMPOUNDS WERE OR WERE NOT

7330
05:20:42,252 --> 05:20:43,320
BOUND WITH [INDISCERNIBLE].

7331
05:20:43,320 --> 05:20:48,191
SO WE FOCUSED ON INITIALLY

7332
05:20:48,191 --> 05:20:53,163
WITHIN SCLERODERMA AS THE

7333
05:20:53,163 --> 05:20:55,732
PRIMARY INDICATION BUT REALLY

7334
05:20:55,732 --> 05:20:58,034
AND I'LL SAY A -- WELL, 2 SIDES

7335
05:20:58,034 --> 05:21:00,337
OF THE SAME COIN LOOKING AT

7336
05:21:00,337 --> 05:21:02,272
INTERSTITIAL LUNG DISEASE, NOT

7337
05:21:02,272 --> 05:21:04,474
ONLY WITHIN THE SCLERODERMA

7338
05:21:04,474 --> 05:21:05,475
POPULATION BUT AS MANY OF YOU

7339
05:21:05,475 --> 05:21:07,344
WILL KNOW ON THE CALL THOSE

7340
05:21:07,344 --> 05:21:09,579
COMPOUNDS ARE BEING USED TO

7341
05:21:09,579 --> 05:21:12,382
TREAT IDIO PATHIC PULMONARY

7342
05:21:12,382 --> 05:21:14,217
FIBROSIS AND SO WE'VE GENERATED

7343
05:21:14,217 --> 05:21:17,387
QUITE A BIT OF DATA FROM THAT

7344
05:21:17,387 --> 05:21:19,456
STANDPOINT AS WELL AND THEN

7345
05:21:19,456 --> 05:21:20,323
INITIATED PHASE 1 HERE EARLIER

7346
05:21:20,323 --> 05:21:22,959
THIS YEAR AND WE'RE IN THE

7347
05:21:22,959 --> 05:21:29,199
PROCESS OF RUNNING THAT STUDY.

7348
05:21:29,199 --> 05:21:30,700
SO WE'VE COMBINED ABOUT, WELL

7349
05:21:30,700 --> 05:21:32,235
NOT ONLY OUR SEED BUT NIH

7350
05:21:32,235 --> 05:21:36,006
FUNDING AS WELL AS A SERIES

7351
05:21:36,006 --> 05:21:37,040
A-ROUTE TO ALTOGETHER ABOUT 13

7352
05:21:37,040 --> 05:21:39,909
AND HALF MILLION TO GET THE

7353
05:21:39,909 --> 05:21:42,312
PRODUCT THROUGH THE IND ENABLING

7354
05:21:42,312 --> 05:21:46,716
STUDIES AND INTO THE CLINIC AND

7355
05:21:46,716 --> 05:21:48,651
SO WE'RE FOCUSED REALLY IN ON

7356
05:21:48,651 --> 05:21:51,421
THAT STAGE OF THE DEVELOPMENT

7357
05:21:51,421 --> 05:21:51,654
PROCESS.

7358
05:21:51,654 --> 05:22:01,998
NEXT  SLIDE, PLEASE.

7359
05:22:02,432 --> 05:22:04,567
AT LEAST FROM OUR STANDPOINT THE

7360
05:22:04,567 --> 05:22:05,802
FOUNDERS WORK WAS ALL BEING DONE

7361
05:22:05,802 --> 05:22:09,572
AT THE UNIVERSITY OF MICHIGAN,

7362
05:22:09,572 --> 05:22:11,174
PRIMARILY DR. DAN LAWRENCE AND

7363
05:22:11,174 --> 05:22:11,674
HIS LAB.

7364
05:22:11,674 --> 05:22:14,344
AND I HAPPEN TO BE INTRODUCED TO

7365
05:22:14,344 --> 05:22:17,180
HIM BACK IN LATE 2017 SO A LOT

7366
05:22:17,180 --> 05:22:19,382
OF THIS EARLY FOUNDATIONAL WORK

7367
05:22:19,382 --> 05:22:20,950
THAT HE HAD DONE WAS BEING

7368
05:22:20,950 --> 05:22:23,286
PULLED TOGETHER BUT IT REALLY

7369
05:22:23,286 --> 05:22:26,656
WASN'T ENOUGH TO BE ABLE TO TAKE

7370
05:22:26,656 --> 05:22:27,957
-- THERE WASN'T ENOUGH DATA TO

7371
05:22:27,957 --> 05:22:31,995
TAKE A STORY TO A VENTURE GROUP

7372
05:22:31,995 --> 05:22:34,497
OR EVEN PROBABLY EVEN SUPPORT A

7373
05:22:34,497 --> 05:22:36,633
LOT OF LATER STAGE NIH FUNDING.

7374
05:22:36,633 --> 05:22:39,969
AND SO WE NEEDED TO WALK VERY

7375
05:22:39,969 --> 05:22:42,505
SLOWLY, IF YOU WILL, AND RAISE

7376
05:22:42,505 --> 05:22:44,474
THE SMALL AMOUNT OF MONEY AND

7377
05:22:44,474 --> 05:22:45,775
FRIENDS AND FAMILY AS WELL AS

7378
05:22:45,775 --> 05:22:48,878
THE UNIVERSITY OF MICHIGAN

7379
05:22:48,878 --> 05:22:50,013
BIOMEDICAL VENTURES FUND TO HELP

7380
05:22:50,013 --> 05:22:53,483
US GET OFF THE GROUND AND START

7381
05:22:53,483 --> 05:22:55,151
TO DERISK THE ASSET AND THEN

7382
05:22:55,151 --> 05:23:00,857
THAT ENABLED US TO GO FOR A

7383
05:23:00,857 --> 05:23:04,727
COUPLE OF R43 SBIR GRANTS AND

7384
05:23:04,727 --> 05:23:07,197
THEN, THOSE DATA WERE SUFFICIENT

7385
05:23:07,197 --> 05:23:11,701
TO NOT ONLY DERISK THE ASSET

7386
05:23:11,701 --> 05:23:13,203
FURTHER BUT ATTRACT A VENTURE

7387
05:23:13,203 --> 05:23:20,577
ROUND OF FINANCING IN 22 OR LATE

7388
05:23:20,577 --> 05:23:21,144
FEBRUARY OF 2022.

7389
05:23:21,144 --> 05:23:23,913
AND THEN THOSE ARE THE FUNDS

7390
05:23:23,913 --> 05:23:24,814
THAT WE'VE BEEN CAPITALIZING ON

7391
05:23:24,814 --> 05:23:26,616
TO GET TO THE POINT THAT WE'RE

7392
05:23:26,616 --> 05:23:29,319
CURRENTLY AT AND WILL BE RAISING

7393
05:23:29,319 --> 05:23:34,557
ANOTHER ROUND HERE IN THE FIRST

7394
05:23:34,557 --> 05:23:43,133
HALF OF 2025 TO CONTINUE THE

7395
05:23:43,133 --> 05:23:44,067
DEVELOPMENT PROGRAM.

7396
05:23:44,067 --> 05:23:49,105
THE FIRST PHASE 1 OF THE SBIRs

7397
05:23:49,105 --> 05:23:51,474
ALLOWED THE COMPANY TO BRING

7398
05:23:51,474 --> 05:23:53,977
MYSELF ON AND A COLLEAGUE OF

7399
05:23:53,977 --> 05:23:57,280
MINE WHO'S HAD A LOT OF

7400
05:23:57,280 --> 05:23:57,814
EXPERIENCE IN PRECLINICAL

7401
05:23:57,814 --> 05:23:59,716
DEVELOPMENT AND WE WERE ABLE TO

7402
05:23:59,716 --> 05:24:02,118
GET SOME OF THE INITIAL GENE

7403
05:24:02,118 --> 05:24:04,320
TALKS DONE, ACTUALLY

7404
05:24:04,320 --> 05:24:05,588
DR. HOLTZMAN HAD A NICE SUMMARY

7405
05:24:05,588 --> 05:24:06,756
CHART THERE AND WE KIND OF

7406
05:24:06,756 --> 05:24:10,560
TICKED DOWN THROUGH ALL OF THOSE

7407
05:24:10,560 --> 05:24:13,930
IND ENABLING STUDIES AND EARLY

7408
05:24:13,930 --> 05:24:17,233
ASSESSMENT PROGRAMS IN ADDITION

7409
05:24:17,233 --> 05:24:20,603
TO IN PARALLELLING ON THE CMC

7410
05:24:20,603 --> 05:24:23,573
ACTIVITIES WHICH ARE QUITE

7411
05:24:23,573 --> 05:24:26,509
CRITICAL, AND WE HAD A GOOD TEAM

7412
05:24:26,509 --> 05:24:30,213
TO HELP US BE ABLE TO DO THAT

7413
05:24:30,213 --> 05:24:30,713
WORK.

7414
05:24:30,713 --> 05:24:36,753
AND THAT REALLY -- SO WITH THE

7415
05:24:36,753 --> 05:24:38,388
R43 FUNDS, THE FRIENDS AND

7416
05:24:38,388 --> 05:24:39,522
FAMILY FUNDING, WE WERE THEN

7417
05:24:39,522 --> 05:24:42,358
IEBL TO ALSO BRING ON A BROADER

7418
05:24:42,358 --> 05:24:44,360
TEAM WHERE WE HAD THE CMC

7419
05:24:44,360 --> 05:24:51,668
EXPERTISE, WE HAD THE CLINICAL

7420
05:24:51,668 --> 05:24:54,904
AND CLIN OPS AND REGULATORY

7421
05:24:54,904 --> 05:24:56,973
STRATEGY AND DNPK, AS WELL AS

7422
05:24:56,973 --> 05:24:58,041
PRODUCT MANAGEMENT SOY IT WAS A

7423
05:24:58,041 --> 05:24:59,542
GROUP OF INDIVIDUALS THAT HAD

7424
05:24:59,542 --> 05:25:00,944
WORKED TOGETHER BEFORE, COULD

7425
05:25:00,944 --> 05:25:02,812
ALL MOVE ASSETS THROUGHENTIOUS

7426
05:25:02,812 --> 05:25:04,480
FICIENTLY, ALMOST EVERYONE IN

7427
05:25:04,480 --> 05:25:06,015
THE GROUP HAD SOME APPROXIMATE

7428
05:25:06,015 --> 05:25:08,051
NOT ALL OF THEM HAD SOME LARGE

7429
05:25:08,051 --> 05:25:10,520
PHARMA EXPERIENCE AS WELL AS

7430
05:25:10,520 --> 05:25:11,955
TIME IN A VENTURE FUNDED

7431
05:25:11,955 --> 05:25:12,222
ACTIVITY.

7432
05:25:12,222 --> 05:25:15,391
SO, YOU HAD A LOT OF FOLKS THAT

7433
05:25:15,391 --> 05:25:17,627
UNDERSTOOD HOW TO MOVE THE

7434
05:25:17,627 --> 05:25:22,465
PROGRAM FORWARD ON A FAIRLY

7435
05:25:22,465 --> 05:25:30,673
CAPITAL EFFICIENT BASES.

7436
05:25:30,673 --> 05:25:31,241
NEXT SLIDE.

7437
05:25:31,241 --> 05:25:39,148
THANKS SO THIS AND THE NEXT

7438
05:25:39,148 --> 05:25:41,084
SLIDE ARE JUST 2 EXTRACTS IF YOU

7439
05:25:41,084 --> 05:25:42,652
WILL OF WAWE WERE FOCUSED ON IN

7440
05:25:42,652 --> 05:25:44,020
THE PHASE 1 FUNDING WHICH AS YOU

7441
05:25:44,020 --> 05:25:45,755
KNOW WAS A COUPLE HUNDRED

7442
05:25:45,755 --> 05:25:47,557
THOUSAND DOLLARS, SO IT'S ENOUGH

7443
05:25:47,557 --> 05:25:51,160
TO MAKE PROGRESS BUT NOT A LOT

7444
05:25:51,160 --> 05:25:53,029
OF PROGRESS AND THEN THE PHASE 2

7445
05:25:53,029 --> 05:25:54,764
AWARDS WE WERE FORTUNATE ENOUGH

7446
05:25:54,764 --> 05:25:56,399
TO GENERATE THE RIGHT KIND OF

7447
05:25:56,399 --> 05:25:59,035
DATA AND RESULTS IN PHASE 1,

7448
05:25:59,035 --> 05:26:04,674
DERISK THE PROGRAM, FURTHER AND

7449
05:26:04,674 --> 05:26:07,076
GENERATE DATA THAT ACTUALLY ALSO

7450
05:26:07,076 --> 05:26:09,379
WAS SUPPORTIVE OF THE SERIES A

7451
05:26:09,379 --> 05:26:11,714
FINANCING WHICH ALLOWED US TO

7452
05:26:11,714 --> 05:26:16,252
BRING ABOUT $9 MILLION IN, SO WE

7453
05:26:16,252 --> 05:26:24,594
HAD A COMBINATION OF AN R43 AND

7454
05:26:24,594 --> 05:26:27,964
44 GRANT IN THE NIIAMS DIVISION

7455
05:26:27,964 --> 05:26:30,133
AND REALLY FOCUSED IN ON

7456
05:26:30,133 --> 05:26:33,870
RHEUMATOLOGY WELL AS AS -- NEXT

7457
05:26:33,870 --> 05:26:35,138
SLIDE, PLEASE.

7458
05:26:35,138 --> 05:26:37,907
AS WELL AS NHLBI AND SO WE WERE

7459
05:26:37,907 --> 05:26:39,542
ABLE TO FIGURE OUT WHAT PIECES

7460
05:26:39,542 --> 05:26:46,215
OF THE TOTAL DEVELOPMENT PROGRAM

7461
05:26:46,215 --> 05:26:47,350
WARRANTED FUNDING THROUGH THESE

7462
05:26:47,350 --> 05:26:49,619
GRANT MECHANISMS AND ALL OF

7463
05:26:49,619 --> 05:26:51,154
WHICH WAS TIED TOGETHER WITH THE

7464
05:26:51,154 --> 05:26:55,291
AIM OF MOVING THE ASSET TOWARDS

7465
05:26:55,291 --> 05:26:58,761
COMPLETING SUCCESSFULLY THE IND

7466
05:26:58,761 --> 05:27:00,263
ENABLING ACTIVITIES, GETTING

7467
05:27:00,263 --> 05:27:01,931
FEEDBACK FROM FDA RELATIVELY

7468
05:27:01,931 --> 05:27:07,070
EARLY ON IN THE PROCESS, THROUGH

7469
05:27:07,070 --> 05:27:09,706
PREIND PACKAGE AND THEN FILING

7470
05:27:09,706 --> 05:27:14,043
AN IND IN MARCH, EARLIER OF THIS

7471
05:27:14,043 --> 05:27:16,312
YEAR, AND SO, FROM OUR

7472
05:27:16,312 --> 05:27:17,380
STANDPOINT OTHER THAN SORT OF

7473
05:27:17,380 --> 05:27:20,616
THE TIME DELAYS WHICH I THINK

7474
05:27:20,616 --> 05:27:21,451
DR. HOLTZMAN ALLUDED TO AS

7475
05:27:21,451 --> 05:27:22,485
YOU'RE GOING THROUGH THE GRANTS

7476
05:27:22,485 --> 05:27:23,686
AND YOU DON'T GET EVERY 1 OF

7477
05:27:23,686 --> 05:27:28,424
THEM AS ALL OF YOU KNOW,

7478
05:27:28,424 --> 05:27:30,493
AUTHORIZED FOR FUNDING ON

7479
05:27:30,493 --> 05:27:34,063
EXACTLY YOUR SCHEDULE, BUT

7480
05:27:34,063 --> 05:27:37,333
INDEED, THE DATA WERE EVENTUALLY

7481
05:27:37,333 --> 05:27:38,735
COMPELLING ENOUGH TO WOR RANT

7482
05:27:38,735 --> 05:27:42,004
THE FUNDING BOTH AT THE NIH

7483
05:27:42,004 --> 05:27:45,441
LEVEL AS WELL AS AT THE VENTURE

7484
05:27:45,441 --> 05:27:48,478
LEVEL CAPITAL AND WE WERE ABLE

7485
05:27:48,478 --> 05:27:51,581
TO MOVE FORWARD WITH A MAY

7486
05:27:51,581 --> 05:27:53,783
PROCEED AUTHORIZATION FROM FDA

7487
05:27:53,783 --> 05:27:56,152
AND WE'RE FOCUSED IN NOW ON

7488
05:27:56,152 --> 05:27:57,520
EXECUTING THAT FIRST PHASE OF

7489
05:27:57,520 --> 05:28:05,161
THE PHASE 1 PROGRAMS. 

7490
05:28:05,161 --> 05:28:07,463
NEXT SLIDE, PLEASE.

7491
05:28:07,463 --> 05:28:10,867
SO DR. HOLTZMAN'S RIGHT,

7492
05:28:10,867 --> 05:28:12,101
[INDISCERNIBLE], GO THROUGH THE

7493
05:28:12,101 --> 05:28:13,836
LINES HERE BUT JUST TO GIVE YOU

7494
05:28:13,836 --> 05:28:15,938
AN IDEA OF SORT OF THE LEVEL OF

7495
05:28:15,938 --> 05:28:17,573
DETAIL THAT YOU TRY TRACKING

7496
05:28:17,573 --> 05:28:20,410
FROM, AND REALLY WORKING FROM

7497
05:28:20,410 --> 05:28:22,578
THE BACK END OF WHEN YOU HOPE TO

7498
05:28:22,578 --> 05:28:24,347
GET TO WHATEVER THAT PARTICULAR

7499
05:28:24,347 --> 05:28:27,984
MILESTONE IS, AND THEN YOU START

7500
05:28:27,984 --> 05:28:29,652
TO MOVE FORWARD TO THE PRIMARY

7501
05:28:29,652 --> 05:28:30,953
ACTIVITIES THAT HAVE TO GET

7502
05:28:30,953 --> 05:28:33,589
DONE, EARLY ON AND YOU CERTAINLY

7503
05:28:33,589 --> 05:28:35,725
START TO IDENTIFY WHERE YOUR

7504
05:28:35,725 --> 05:28:41,731
GAPS ARE AND I THINK 1 OF MY

7505
05:28:41,731 --> 05:28:44,367
CO-PRESENTERS HERE BEING SCIENCE

7506
05:28:44,367 --> 05:28:45,968
IS UNPREDICTABLE, WELL

7507
05:28:45,968 --> 05:28:46,702
DEVELOPMENT IS UNPREICTIBLE AS

7508
05:28:46,702 --> 05:28:51,874
WELL, AND WHO COULD HAVE

7509
05:28:51,874 --> 05:28:53,042
PREDICTED COVID INTERRUPTING THE

7510
05:28:53,042 --> 05:28:53,276
PATHWAY.

7511
05:28:53,276 --> 05:28:57,580
SO TRYING TO WORK THROUGH A

7512
05:28:57,580 --> 05:28:59,182
PANDEMIC, GLOBAL PANDEMIC ALONG

7513
05:28:59,182 --> 05:29:00,817
WITH KEEPING THE PROGRAM

7514
05:29:00,817 --> 05:29:06,055
ON-TIME, FINDING A WAY TO GET

7515
05:29:06,055 --> 05:29:07,857
INTO THE CUE FOR VARIOUS,

7516
05:29:07,857 --> 05:29:09,659
COMPLETION OF THE VARIOUS IND

7517
05:29:09,659 --> 05:29:16,098
ENABLING STUDIES HAVING TO

7518
05:29:16,098 --> 05:29:18,501
SWITCH THE SECOND NONRODENT

7519
05:29:18,501 --> 05:29:20,570
SPECIES TO NONHUMAN PRIMATES TO

7520
05:29:20,570 --> 05:29:24,874
GINNY PIGGINGS AS COVID DEMANDS

7521
05:29:24,874 --> 05:29:27,410
ON NHP AVAILABILITY AND

7522
05:29:27,410 --> 05:29:28,578
UTILIZATION WERE DRIVING THE

7523
05:29:28,578 --> 05:29:30,746
PRICE UP ABOUT 5 OR 6 TIMES, IF

7524
05:29:30,746 --> 05:29:34,450
YOU COULD EVEN FIND YOUR WAY

7525
05:29:34,450 --> 05:29:37,587
INTO A CRO QFOR THAT SPECIES.

7526
05:29:37,587 --> 05:29:43,025
A LOT OF PIECES KEEP MOVING

7527
05:29:43,025 --> 05:29:44,427
FORWARD AND SO, AS SIMPLISTIC AS

7528
05:29:44,427 --> 05:29:46,496
THE CHART IS, IT DOES HELP YOU

7529
05:29:46,496 --> 05:29:49,999
KEEP EVERYONE ON TRACK AND KEEP

7530
05:29:49,999 --> 05:29:54,604
YOU FOCUSED IN ON WHEN WHERE THE

7531
05:29:54,604 --> 05:29:56,539
KEY GAPS ARE AND WHEN YOUR

7532
05:29:56,539 --> 05:29:59,208
TIMELINES ARE GOING TO BE MET

7533
05:29:59,208 --> 05:30:03,312
DOWN THE ROAD.

7534
05:30:03,312 --> 05:30:03,880
NEXT SLIDE.

7535
05:30:03,880 --> 05:30:05,915
YEAH, SO THIS IS JUST SORT OF

7536
05:30:05,915 --> 05:30:07,083
THE EMERGING PROFILE, IT'S

7537
05:30:07,083 --> 05:30:08,918
REALLY JUST A SUMMARY OF WHERE

7538
05:30:08,918 --> 05:30:10,753
WE ARE RIGHT NOW.

7539
05:30:10,753 --> 05:30:14,056
SO, I THINK AS I JOINED THIS

7540
05:30:14,056 --> 05:30:16,492
SESSION HERE WITH DR. HOLTZMAN,

7541
05:30:16,492 --> 05:30:20,830
HE HIGHLIGHTED A LOT OF THESE AS

7542
05:30:20,830 --> 05:30:22,098
HAVING VERY CLEAR TARGET, CLEAR

7543
05:30:22,098 --> 05:30:25,601
IN OUR CASE INHIBITION ACTIVITY

7544
05:30:25,601 --> 05:30:28,404
BEING ABLE TO SEE ENGAGEMENT,

7545
05:30:28,404 --> 05:30:31,707
NOT ONLY IN THE PRIMARY ANIMAL

7546
05:30:31,707 --> 05:30:35,344
MODELS FOR EFFICACY AS WELL AS

7547
05:30:35,344 --> 05:30:41,484
SAFETY, BUT ALSO IN THE EARLY

7548
05:30:41,484 --> 05:30:46,088
STAGES OF PHASE 1 AND WE'VE GOT

7549
05:30:46,088 --> 05:30:50,092
A FAIRLY HIGH THRESHOLD OF TOP

7550
05:30:50,092 --> 05:30:53,629
DOZE THAT WILL BE EVALUATED

7551
05:30:53,629 --> 05:30:56,499
RELATIVE TO WHAT WE THINK THE

7552
05:30:56,499 --> 05:30:58,000
EXPOSURE REQUIRED FOR EFFICACY

7553
05:30:58,000 --> 05:31:00,836
WILL BE, SO, OBVIOUSLY ONCE WE

7554
05:31:00,836 --> 05:31:02,438
UNBLIND THE STUDY AND SEE WHAT

7555
05:31:02,438 --> 05:31:03,706
THOSE DATA ACTUALLY LOOK LIKE,

7556
05:31:03,706 --> 05:31:07,076
WE WILL KNOW HOW CLOSE WE WERE

7557
05:31:07,076 --> 05:31:09,278
TO BEING ON TRACK BUT WE THINK

7558
05:31:09,278 --> 05:31:13,149
WE HAVE A PRETTY GOOD MARNLIN TO

7559
05:31:13,149 --> 05:31:13,416
EVALUATE.

7560
05:31:13,416 --> 05:31:23,893
AND THEN IN OUR CASE, SINCE

7561
05:31:24,727 --> 05:31:26,362
HAVING PATIENTS THAT ARE NULL

7562
05:31:26,362 --> 05:31:30,333
FOR P A I 1 HAVE BEEN ASSOCIATE

7563
05:31:30,333 --> 05:31:31,067
WIDE BLEEDING DISORDERS EVEN

7564
05:31:31,067 --> 05:31:35,237
THOUGH WE'RE NOT DEVELOPING A

7565
05:31:35,237 --> 05:31:36,539
COMPOUND THAT ACTUALLY KNOCKS

7566
05:31:36,539 --> 05:31:38,975
OUT ALL P A I ACTIVITY, WE'RE

7567
05:31:38,975 --> 05:31:44,246
MORE OF A CHARACTERIZED AS A

7568
05:31:44,246 --> 05:31:50,319
RESTAT LIKE EFFECT WHERE YOU

7569
05:31:50,319 --> 05:31:51,520
RETURN DYSFUNCTIONAL LEVELS OF P

7570
05:31:51,520 --> 05:31:54,924
A I 1 DOWN TO NORMAL LEVELS BUT

7571
05:31:54,924 --> 05:31:56,926
EVERYONE ALWAYS ASKS YOU FOR

7572
05:31:56,926 --> 05:32:00,229
DATA ABOUT BLEEDING RISK AND SO

7573
05:32:00,229 --> 05:32:03,299
WE'VE HAD TO CONSTANTLY GENERATE

7574
05:32:03,299 --> 05:32:06,168
THAT NOT ONLY IN VIVO BUT EXVIVO

7575
05:32:06,168 --> 05:32:08,137
AND CONTINUE TO LOOK AT THAT.

7576
05:32:08,137 --> 05:32:09,805
IT'S NOT ACTUALLY INTERESTINGLY

7577
05:32:09,805 --> 05:32:12,441
ENOUGH AN ISSUE FROM THE FDA

7578
05:32:12,441 --> 05:32:14,043
STANDPOINT THAT HAS BEEN A

7579
05:32:14,043 --> 05:32:16,579
CONCERN AS WE'VE GONE THROUGH

7580
05:32:16,579 --> 05:32:20,483
THE INITIAL IND SUBMISSION, BUT

7581
05:32:20,483 --> 05:32:22,551
IT IS A CONSTANT QUESTION FROM

7582
05:32:22,551 --> 05:32:25,788
ALL OF THOSE POTENTIAL STRATEGIC

7583
05:32:25,788 --> 05:32:30,760
PARTNERS DOWN THE ROAD THAT AS

7584
05:32:30,760 --> 05:32:32,461
DR. LIU MENTIONED, YOU WILL

7585
05:32:32,461 --> 05:32:34,096
DEFINITELY NEED AS YOU ADVANCE

7586
05:32:34,096 --> 05:32:38,167
THE COMPOUND INTO LATER STAGES.

7587
05:32:38,167 --> 05:32:41,237
NEXT SLIDE.

7588
05:32:41,237 --> 05:32:42,705
OKAY, SO, SUMMARY, ALSO MY YOB

7589
05:32:42,705 --> 05:32:44,340
CORPS IS BEING THE LAST

7590
05:32:44,340 --> 05:32:46,308
PRESENTER IS TO GIVE THE GIFT OF

7591
05:32:46,308 --> 05:32:48,644
TIME TO THE MODERATORS WHO ARE

7592
05:32:48,644 --> 05:32:50,379
GOING TO CONVENE THE PANEL

7593
05:32:50,379 --> 05:32:51,647
AFTERWARDS AND JUST SUMMARIZE.

7594
05:32:51,647 --> 05:32:53,983
SO WE WERE ABLE TO REALLY PARLAY

7595
05:32:53,983 --> 05:32:58,054
THE FIRST 4 AND HALF MILLION, 4

7596
05:32:58,054 --> 05:33:01,991
OF WHICH WAS NIH PROVIDED

7597
05:33:01,991 --> 05:33:04,527
THROUGH R43 AND R44 GRANTS TO

7598
05:33:04,527 --> 05:33:06,462
GENERATE THE KIND OF DATA THAT

7599
05:33:06,462 --> 05:33:08,197
WOULD BE NEEDED NOT ONLY TO

7600
05:33:08,197 --> 05:33:09,899
RERISK THE ASSET FROM AN

7601
05:33:09,899 --> 05:33:12,368
INVESTOR STAND POINT WHICH WAS

7602
05:33:12,368 --> 05:33:15,171
HELPFUL IN ATTRACTING OUR SERIES

7603
05:33:15,171 --> 05:33:17,807
8 INITIAL ROUND OF FINANCING BUT

7604
05:33:17,807 --> 05:33:24,380
ALSO BEING ABLE TO DELIVER THE

7605
05:33:24,380 --> 05:33:26,449
IND PACKAGE THAT WAS SUFFICIENT

7606
05:33:26,449 --> 05:33:27,049
FOR FDA ABNORMAL GLUCOSE

7607
05:33:27,049 --> 05:33:28,451
TOLERANCE RISATION TO ADVANCE

7608
05:33:28,451 --> 05:33:29,652
THE PROGRAM.

7609
05:33:29,652 --> 05:33:32,288
SO COMING OUT OF COMPLETION HERE

7610
05:33:32,288 --> 05:33:35,124
OF THE PHASE 1 A AND 1 B ARE THE

7611
05:33:35,124 --> 05:33:38,994
SAD AND MAD FACES OF THE EARLY

7612
05:33:38,994 --> 05:33:40,629
CLINICAL STUDIES AND HEALTHY

7613
05:33:40,629 --> 05:33:44,233
VOLUNTEERS, WE'LL BE LOOKING AT

7614
05:33:44,233 --> 05:33:46,068
A PHASE 2A PROGRAM IN PATIENTS

7615
05:33:46,068 --> 05:33:47,703
OVER ABOUT A 6 MONTH PERIOD OF

7616
05:33:47,703 --> 05:33:49,004
TIME GENERATING THE DATA THAT

7617
05:33:49,004 --> 05:33:53,943
CAN  DERISK THE ASSET TOWARD THE

7618
05:33:53,943 --> 05:33:54,977
NEXT ROUND OF INVESTMENT.

7619
05:33:54,977 --> 05:33:55,311
ALL RIGHT.

7620
05:33:55,311 --> 05:33:58,481
SO I THINK THAT'S IT FOR MY

7621
05:33:58,481 --> 05:33:58,814
PRESENTATION.

7622
05:33:58,814 --> 05:34:00,216
AND HAPPY TO ANSWER QUESTIONS AS

7623
05:34:00,216 --> 05:34:06,756
WE GO THROUGH THE PANEL

7624
05:34:06,756 --> 05:34:07,056
DISCUSSION.

7625
05:34:07,056 --> 05:34:09,492
>> YEAH, I THINK WE WILL
DO THE

7626
05:34:09,492 --> 05:34:11,560
QUESTIONS DURING THE PANEL

7627
05:34:11,560 --> 05:34:11,927
DISCUSSION.

7628
05:34:11,927 --> 05:34:13,395
THAT'S GREAT. 

7629
05:34:13,395 --> 05:34:14,196
>> THANK YOU.

7630
05:34:14,196 --> 05:34:16,732
>> THANK YOU DR. BENOIT,

7631
05:34:16,732 --> 05:34:18,000
HONORARY DOCTOR.

7632
05:34:18,000 --> 05:34:19,068
APPRECIATE THAT.

7633
05:34:19,068 --> 05:34:23,272
IT WILL GET ME A PAID DINNER

7634
05:34:23,272 --> 05:34:23,706
TONIGHT.

7635
05:34:23,706 --> 05:34:24,840
[LAUGHTER]

7636
05:34:24,840 --> 05:34:26,342
>> SO I THINK THAT THE
ORDER

7637
05:34:26,342 --> 05:34:28,310
THAT THEY HAVE US GOING IN IS ME

7638
05:34:28,310 --> 05:34:32,047
AND THEN JAMES, I WAS GOING TO

7639
05:34:32,047 --> 05:34:34,116
GO VERY BRIEF, MUCH EARLIER IN

7640
05:34:34,116 --> 05:34:36,318
AND IT'S LETTING ME DO MY KRID

7641
05:34:36,318 --> 05:34:37,620
VIDEO BUT THAT'S FINE, YOU DON'T

7642
05:34:37,620 --> 05:34:41,991
NEED TO SEE ME, ANYWAY IT'LL

7643
05:34:41,991 --> 05:34:45,394
PROBABLY BE BETTER FOR -- THERE

7644
05:34:45,394 --> 05:34:46,128
WE GO.

7645
05:34:46,128 --> 05:34:47,496
IT MAY ACTUALLY BE BETTER TO

7646
05:34:47,496 --> 05:34:48,464
JUST HAVE IT OFF.

7647
05:34:48,464 --> 05:34:52,101
WE WILL SEE HOW STABLE IT IS.

7648
05:34:52,101 --> 05:34:57,273
SO THIS IS JAMES' IT WE CAN LOAD

7649
05:34:57,273 --> 05:34:59,041
THE 1 THAT SAYS THE AUDIO ON THE

7650
05:34:59,041 --> 05:35:06,882
END OF IT, PLEASE.

7651
05:35:06,882 --> 05:35:17,393
SO YOU'RE GETTING A PREVIEW OF

7652
05:35:36,045 --> 05:35:37,046
JAMES THERE.

7653
05:35:37,046 --> 05:35:42,818
JAMES DO YOU MIND JUMPING IN?

7654
05:35:42,818 --> 05:35:43,219
>> MY PLEASURE.

7655
05:35:43,219 --> 05:35:50,459
>> THANK YOU SO MUCH,
LET'S PUT

7656
05:35:50,459 --> 05:35:55,164
JAMES UP AND WE WILL FIND

7657
05:35:55,164 --> 05:35:55,531
DR. ZIADY'S.

7658
05:35:55,531 --> 05:36:01,136
>> SAME THING, BACKWARDS
FAST.

7659
05:36:01,136 --> 05:36:02,771
ALL RIGHT, SO, THE TALK I'M

7660
05:36:02,771 --> 05:36:04,740
GOING TO GIVE IS I WOULD SAY A

7661
05:36:04,740 --> 05:36:06,008
BRIEF THING, IS I THOUGHT IT

7662
05:36:06,008 --> 05:36:07,676
WOULD BE ENTERTAINING TO SEE A

7663
05:36:07,676 --> 05:36:12,147
LOT OF CONCEPTS THESE GUYS

7664
05:36:12,147 --> 05:36:16,685
TALKED ABOUT BUT, FOR A PROJECT

7665
05:36:16,685 --> 05:36:17,186
THAT HAD PROBLEMS.

7666
05:36:17,186 --> 05:36:21,156
I'VE GOT A LOT OF PROJECTS BUT I

7667
05:36:21,156 --> 05:36:23,092
THOUGHT THIS 1 HAD MORE

7668
05:36:23,092 --> 05:36:25,060
EDUCATIONAL CONTENT.

7669
05:36:25,060 --> 05:36:26,962
NEXT SLIDE.

7670
05:36:26,962 --> 05:36:28,564
SO, THE DISCLOSURES SORT OF

7671
05:36:28,564 --> 05:36:30,432
START OFF SO NHLBI FUNDED PART

7672
05:36:30,432 --> 05:36:32,301
OF THIS BUT THERE'S 4 DEFINITE

7673
05:36:32,301 --> 05:36:34,370
COMPANIES LISTED HERE, HERE ON

7674
05:36:34,370 --> 05:36:36,906
GSK'S LUNG DECS DIVISION,

7675
05:36:36,906 --> 05:36:38,040
[INDISCERNIBLE] AND ALL 4 OF

7676
05:36:38,040 --> 05:36:39,909
THOSE HAVE NO LONGER EXIST,

7677
05:36:39,909 --> 05:36:43,145
AFTER HELPING ME WITH THIS

7678
05:36:43,145 --> 05:36:45,281
PROJECT SO FAR NHLBI HAS

7679
05:36:45,281 --> 05:36:46,515
SURVIVED COLLABORATING WITH ME

7680
05:36:46,515 --> 05:36:50,119
BUT WE'LL SEE.

7681
05:36:50,119 --> 05:36:50,920
NEXT SLIDE.

7682
05:36:50,920 --> 05:36:54,089
SO THIS ISN'T EXACTLY EVERYBODY

7683
05:36:54,089 --> 05:36:55,824
ELSE PUT IT BUT CLOSE ENOUGH,

7684
05:36:55,824 --> 05:36:57,359
THERE'S A BUNCH OF THINGS YOU

7685
05:36:57,359 --> 05:36:58,327
WANT FOR YOUR IDEAL TARGET

7686
05:36:58,327 --> 05:36:59,828
PROFILE EMPLOY YOU WANT TO

7687
05:36:59,828 --> 05:37:01,363
TARGET THE MOLECULAR DRIVERS OF

7688
05:37:01,363 --> 05:37:02,798
DEC, YOU WANT LIMITED OFFTARGET

7689
05:37:02,798 --> 05:37:03,432
CENTER FOR EXCELLENCE ON AGINGS,

7690
05:37:03,432 --> 05:37:04,600
YOU WANT A THEREFOREY METHOD

7691
05:37:04,600 --> 05:37:05,868
THAT'S VIABLE AND YOU WANT THE

7692
05:37:05,868 --> 05:37:07,970
WHOLE THING TO BE VIABLE AND

7693
05:37:07,970 --> 05:37:11,140
REASONABLE TO MAKE PROTECTIVE

7694
05:37:11,140 --> 05:37:14,910
IP.

7695
05:37:14,910 --> 05:37:22,551
NEXT SLIDE. 

7696
05:37:22,551 --> 05:37:23,052
NEXT SLIDE.

7697
05:37:23,052 --> 05:37:33,295
SO 1 OF THE DECS I STUDY,

7698
05:37:33,295 --> 05:37:34,063
PULMONARY ARTERIAL HYPERTENSION,

7699
05:37:34,063 --> 05:37:36,632
IT'S A NICE TARGET, THERE'S A

7700
05:37:36,632 --> 05:37:37,800
PATHWAY ACE 2 WHICH LOOKS LIKE

7701
05:37:37,800 --> 05:37:41,036
IT HITS ALL OF THE BMPR2 DOWN

7702
05:37:41,036 --> 05:37:46,709
STREAM OW YOU MIGHT IMAGINE IF

7703
05:37:46,709 --> 05:37:49,078
BMPR2 IS PENETRATED YOU COULD

7704
05:37:49,078 --> 05:37:50,112
TREAT PATES WITH ACE 2.

7705
05:37:50,112 --> 05:37:50,746
NEXT SLIDE.

7706
05:37:50,746 --> 05:37:53,048
SO THERE'S A SMALL COMPANY

7707
05:37:53,048 --> 05:37:58,053
CALLED APIRON OUT OF GERMANY OR

7708
05:37:58,053 --> 05:37:59,555
AUSTRIA THAT HAD PATENTS ON

7709
05:37:59,555 --> 05:38:01,991
THEM, WE GOT TO GIVE THEM TO

7710
05:38:01,991 --> 05:38:03,192
MUTATIONS IN MICE, AND THEY

7711
05:38:03,192 --> 05:38:05,361
WORKED GREAT IN OUR HANDS, SOME

7712
05:38:05,361 --> 05:38:08,364
OTHER GROUPS TRIED IT IN OTHER

7713
05:38:08,364 --> 05:38:09,898
MODELS, OF PULL MONITORRORY

7714
05:38:09,898 --> 05:38:11,533
HYPERTENSION, IT WORKED GREAT

7715
05:38:11,533 --> 05:38:13,135
THERE, PRECLINICAL DATA IN MOST

7716
05:38:13,135 --> 05:38:18,340
MODELS BUT IT DID GRIT FOR

7717
05:38:18,340 --> 05:38:21,810
PULMONARY HYPERTENSION, SO THEY

7718
05:38:21,810 --> 05:38:23,479
SOLD THIS FOR GREAT PILES OF

7719
05:38:23,479 --> 05:38:23,679
MONEY.

7720
05:38:23,679 --> 05:38:25,581
SO SO FAR THIS LOOKS GREAT.

7721
05:38:25,581 --> 05:38:26,982
LOOKS LIKE IT SHOULD BE HITTING

7722
05:38:26,982 --> 05:38:29,718
THE FOUNDATION OF THE DISEASE,

7723
05:38:29,718 --> 05:38:32,221
LIMITED OFF TARGET EFFECTS

7724
05:38:32,221 --> 05:38:34,623
BECAUSE THE H2 IS FAIRLY ALREADY

7725
05:38:34,623 --> 05:38:35,791
A BIOLOGICAL PRODUCT AND IT'S

7726
05:38:35,791 --> 05:38:41,463
WELL KNOWN WHAT IT DID AND IT'S

7727
05:38:41,463 --> 05:38:42,731
GOT PREDICTABLE IP.

7728
05:38:42,731 --> 05:38:44,900
SO GSK GETS BACK AHOLD OF ME AND

7729
05:38:44,900 --> 05:38:47,202
WANTS A FIRST IN MAN TRIAL OF

7730
05:38:47,202 --> 05:38:48,737
ACE 2 IN MEL MONITORARY

7731
05:38:48,737 --> 05:38:50,806
HYPERTENSION AND WE DID IT AND

7732
05:38:50,806 --> 05:38:55,577
WE PUBLISH IT, IT LOOKS GREAT.

7733
05:38:55,577 --> 05:38:56,578
NEXT SLIDE.

7734
05:38:56,578 --> 05:38:58,080
THE -- I'M NOT GOING TO SHOW ALL

7735
05:38:58,080 --> 05:39:00,549
THIS STUFF BUT THE CLINICAL END

7736
05:39:00,549 --> 05:39:03,452
POINTS WERE WONDERFUL, IT

7737
05:39:03,452 --> 05:39:04,520
DROPPED THE PULMONARY PRESSURES,

7738
05:39:04,520 --> 05:39:08,057
HIT ALL THE RIGHT MOLECULAREND

7739
05:39:08,057 --> 05:39:08,624
POINTS.

7740
05:39:08,624 --> 05:39:12,294
THE HUMAN TRIALS, THE HUMAN CLIN

7741
05:39:12,294 --> 05:39:15,397
TRIALS LOOK LIKE, IT LOOKS SAFE.

7742
05:39:15,397 --> 05:39:17,166
NEXT SLIDE EMPLOY BUT IT'S

7743
05:39:17,166 --> 05:39:20,502
ACTUALLY TERRIBLE, IT'S TERRIBLE

7744
05:39:20,502 --> 05:39:22,304
BECAUSE THE ACTUAL DRUG, FIRST

7745
05:39:22,304 --> 05:39:24,740
OFF IT'S A SHORT HALF LIFE AND

7746
05:39:24,740 --> 05:39:25,441
REQUIRES DIRECT IV

7747
05:39:25,441 --> 05:39:27,042
ADMINISTRATION AND SO YOU CAN

7748
05:39:27,042 --> 05:39:28,343
WORK AROUND THAT BUT THAT'S A

7749
05:39:28,343 --> 05:39:33,949
HARD DRUG TO COMMERCIALIZE.

7750
05:39:33,949 --> 05:39:35,784
BUT SECONDLY EVEN GSK HAD

7751
05:39:35,784 --> 05:39:37,486
DIFFICULTY MAKE THANKSGIVING

7752
05:39:37,486 --> 05:39:39,655
THING, THE FUNCTIONAL

7753
05:39:39,655 --> 05:39:40,889
REQUIREMENTS MAKE POST

7754
05:39:40,889 --> 05:39:42,257
TRANSLATIONAL MODIIVESS, IT CAN

7755
05:39:42,257 --> 05:39:45,727
BE ONLY MADE IN MAMMALIAN CELLS,

7756
05:39:45,727 --> 05:39:48,564
IT WAS INSANELY DIFFICULT TO

7757
05:39:48,564 --> 05:39:50,065
MAKE, AND GSK BARELY HAD ENOUGH

7758
05:39:50,065 --> 05:39:53,135
FOR EVEN A SMALL TRIAL.

7759
05:39:53,135 --> 05:39:54,069
NEXT SLIDE.

7760
05:39:54,069 --> 05:39:55,304
HOWEVER, NO PROBLEM, RIGHT?

7761
05:39:55,304 --> 05:39:56,839
WE KNOW HOW THIS PATHWAY WORKS

7762
05:39:56,839 --> 05:39:59,441
WE KNOW HOW IT WORKS IS WAS LIKE

7763
05:39:59,441 --> 05:40:03,278
3000 PAPERS THAT SAY THIS, IS H2

7764
05:40:03,278 --> 05:40:05,547
CONVERTS TO [INDISCERNIBLE], AND

7765
05:40:05,547 --> 05:40:08,383
MASS 1 AND IT'S' 7 PEPTIDE,

7766
05:40:08,383 --> 05:40:11,253
SHOULD BE SUPER EASY TO MAKE,

7767
05:40:11,253 --> 05:40:14,389
IT'S ALWAYS UNSTABLE BUT GSK HAS

7768
05:40:14,389 --> 05:40:16,091
TONS OF PROPRIETARY TECHNOLOGIES

7769
05:40:16,091 --> 05:40:20,229
TO STABILIZE IT.

7770
05:40:20,229 --> 05:40:20,562
NEXT SLIDE.

7771
05:40:20,562 --> 05:40:22,064
GLAKS O DOES THAT AND THEY GIVE

7772
05:40:22,064 --> 05:40:24,066
US THE DRUG AND IT DOESN'T WORK.

7773
05:40:24,066 --> 05:40:26,235
WE TRY IT IN THE SAME MOUSE

7774
05:40:26,235 --> 05:40:29,071
MODELS AND IT DOESN'T DO WHAT

7775
05:40:29,071 --> 05:40:29,671
ACE 2 DID.

7776
05:40:29,671 --> 05:40:32,307
AND I WORRY THAT THE STUFF THEY

7777
05:40:32,307 --> 05:40:33,909
MODIFIED TO STABILIZE IT MADE IT

7778
05:40:33,909 --> 05:40:35,811
NO LONGER HIT THE RECEPTOR BUT

7779
05:40:35,811 --> 05:40:37,746
IT DOESN'T MATTER BECAUSE GSK

7780
05:40:37,746 --> 05:40:39,314
SHUT DOWN THEIR LUNG DISEASES

7781
05:40:39,314 --> 05:40:40,649
DIVISION, NOT RELATED TO THIS

7782
05:40:40,649 --> 05:40:45,888
AND THEY GAVE THE PATENT BACK TO

7783
05:40:45,888 --> 05:40:46,788
APIRON.

7784
05:40:46,788 --> 05:40:48,023
NEXT SLIDE.

7785
05:40:48,023 --> 05:40:50,125
SO APIRON, THE GUIDES THAT DID

7786
05:40:50,125 --> 05:40:55,864
APPEARON MADE A NEW COMPANY

7787
05:40:55,864 --> 05:40:58,233
CALLED ALTEROUS, AND INSTEAD OF

7788
05:40:58,233 --> 05:41:08,777
MAKING A -- TRYING TO STABILIZE

7789
05:41:09,778 --> 05:41:11,346
ANG 1-7, AND BY BLOCKING THESE

7790
05:41:11,346 --> 05:41:12,381
PROTEIN COMPLEX YAIG YAISS YOU

7791
05:41:12,381 --> 05:41:13,715
CAN MAKE IT GO WAY UP.

7792
05:41:13,715 --> 05:41:17,085
THIS IS JUST A FIGURE SHOWING

7793
05:41:17,085 --> 05:41:20,689
THAT ACE 2 BRINGS ANG 1-7 UP AS

7794
05:41:20,689 --> 05:41:22,191
A RELATOR IN BLOOD AND THE

7795
05:41:22,191 --> 05:41:23,926
COMBINATION OF THEIR DRUG IMETS

7796
05:41:23,926 --> 05:41:27,095
US UP TO 460 EMPLOY SO WE CAN

7797
05:41:27,095 --> 05:41:29,665
REPRODUCE THE THING WITH SMALL

7798
05:41:29,665 --> 05:41:36,004
MOLECULE INHIBITORS.

7799
05:41:36,004 --> 05:41:36,572
NEXT SLIDE.

7800
05:41:36,572 --> 05:41:37,973
NEXT SLIDE AND IT STILL DOESN'T

7801
05:41:37,973 --> 05:41:38,173
WORK.

7802
05:41:38,173 --> 05:41:39,708
IN FACT WHEN WE INCREASE THE

7803
05:41:39,708 --> 05:41:43,946
DOSE TO GIVE IT EVEN MORE ANG

7804
05:41:43,946 --> 05:41:45,247
1-7 THE PULMONARY HYPERTENSION

7805
05:41:45,247 --> 05:41:45,847
GETS WORSE.

7806
05:41:45,847 --> 05:41:48,617
NEXT SLIDE.

7807
05:41:48,617 --> 05:41:54,923
AND OH, ALTASD E R-- ALTERRAS

7808
05:41:54,923 --> 05:41:58,594
WENT OUT OF BUSINESS.

7809
05:41:58,594 --> 05:41:59,895
MAYBE HOW ACE 2 IS WORKING HAS

7810
05:41:59,895 --> 05:42:00,996
NOTHING TO DO WITH THIS.

7811
05:42:00,996 --> 05:42:01,597
NEXT SLIDE.

7812
05:42:01,597 --> 05:42:03,532
SO AT THIS POINT WHAT LOOKED

7813
05:42:03,532 --> 05:42:05,100
GOOD TO BEGIN WITH NOW LOOKS

7814
05:42:05,100 --> 05:42:05,567
LIKE A DISASTER.

7815
05:42:05,567 --> 05:42:07,536
WE DON'T HAVE A DELIVERY METHOD,

7816
05:42:07,536 --> 05:42:09,238
WE CAN'T MAKE IT A REASONABLE

7817
05:42:09,238 --> 05:42:10,305
PRICE, WE'RE NOT EVEN SURE WE

7818
05:42:10,305 --> 05:42:13,175
HAVE THE RIGHT TARGET.

7819
05:42:13,175 --> 05:42:14,843
KEEP GOING.

7820
05:42:14,843 --> 05:42:16,478
BUT WE KNOCKED OUT THE MASS

7821
05:42:16,478 --> 05:42:17,679
RECEPTOR IN MICE AND WHEN YOU

7822
05:42:17,679 --> 05:42:19,915
KNOCK OUT THE MASS RECEPTOR IN

7823
05:42:19,915 --> 05:42:22,184
MICE, ACE 2 NO LONGER WORKS SO

7824
05:42:22,184 --> 05:42:25,721
MASS IS THE RIGHT RECEPTOR.

7825
05:42:25,721 --> 05:42:26,555
NEXT SLIDE.

7826
05:42:26,555 --> 05:42:27,823
MOST MASS RECEPTOR AGONISTS THAT

7827
05:42:27,823 --> 05:42:29,591
ARE OUT THERE AREN'T ACTUALLY

7828
05:42:29,591 --> 05:42:30,993
MASS RECEPTOR AGONISTS BUT

7829
05:42:30,993 --> 05:42:34,062
THERE'S A COMPANY CALLED ARENA

7830
05:42:34,062 --> 05:42:35,230
PHARMACEUTICALS THAT HAD A

7831
05:42:35,230 --> 05:42:36,832
CLEVER CHEMIST THAT DID TONS OF

7832
05:42:36,832 --> 05:42:40,335
NICE WORK AND FOUND AN ACTUAL

7833
05:42:40,335 --> 05:42:41,637
MASS RECEPTOR AGONIST AND IT

7834
05:42:41,637 --> 05:42:42,771
WORKED GREAT, JUST AS WELL AS

7835
05:42:42,771 --> 05:42:44,906
ACE 2.

7836
05:42:44,906 --> 05:42:45,407
NEXT SLIDE.

7837
05:42:45,407 --> 05:42:49,111
BUT THEY WENT OUT OF BUSINESS.

7838
05:42:49,111 --> 05:42:50,045
[LAUGHTER]

7839
05:42:50,045 --> 05:42:51,480
AND THEIR PATENT WAS BOUGHT BY

7840
05:42:51,480 --> 05:42:52,748
PFIZER WHICH IS ABOUT WHERE WE

7841
05:42:52,748 --> 05:42:57,119
STOP NOW .

7842
05:42:57,119 --> 05:42:57,653
NEXT SLIDE.

7843
05:42:57,653 --> 05:42:59,755
SO GOOD NEWS IS THAT IT ALL

7844
05:42:59,755 --> 05:43:01,623
LOOKS PRETTY GOOD, TARGET

7845
05:43:01,623 --> 05:43:02,891
PROFILEETS MOLECULAR DRIVERS OF

7846
05:43:02,891 --> 05:43:04,760
DISEASE, LIMITED OFF TARGET

7847
05:43:04,760 --> 05:43:05,827
EFFECTS, THEREFOREY METHODS

7848
05:43:05,827 --> 05:43:07,796
VIABLE, IT'S SMALL MOLECULE,

7849
05:43:07,796 --> 05:43:10,565
THAT'S EXTREMELY STABLE AND

7850
05:43:10,565 --> 05:43:12,567
SUBCUTANEOUS, REASONABLE PRICE

7851
05:43:12,567 --> 05:43:15,103
TO MAKE, SMALL MOLECULE DRUG,

7852
05:43:15,103 --> 05:43:17,205
PROTECTABLE IP, AND THERE

7853
05:43:17,205 --> 05:43:19,875
EXISTING PATENTS ON THE THING.

7854
05:43:19,875 --> 05:43:21,677
NEXT SLIDE.

7855
05:43:21,677 --> 05:43:21,943
[LAUGHTER]

7856
05:43:21,943 --> 05:43:22,944
AND SO, THE LAST PROBLEM YOU

7857
05:43:22,944 --> 05:43:25,814
HAVE IS IF THE IP OWNER IS

7858
05:43:25,814 --> 05:43:26,615
INTERESTED IN THE INDICATION O

7859
05:43:26,615 --> 05:43:29,217
I'M GOING TO SEE IF I CAN'T GET

7860
05:43:29,217 --> 05:43:30,619
AHOLD WITH PFIZER AND SEE IF

7861
05:43:30,619 --> 05:43:31,720
THEY'RE INTERESTED IN PURSUING

7862
05:43:31,720 --> 05:43:33,188
THE PROJECT AND THE REASON FOR

7863
05:43:33,188 --> 05:43:34,823
THAT IS, IT'S LOVELY AND WOULD

7864
05:43:34,823 --> 05:43:36,325
GIVE ME A NICE PARTNER TO

7865
05:43:36,325 --> 05:43:37,626
PROCEED BUT IF NOT THERE'S A FEW

7866
05:43:37,626 --> 05:43:39,728
PROBLEMS WITH THE DRUG, IT'S A

7867
05:43:39,728 --> 05:43:41,596
GREAT DRUG BUT IT COULD BE MADE

7868
05:43:41,596 --> 05:43:43,765
MORE SOLUBLE FOR FEW THINGS TO

7869
05:43:43,765 --> 05:43:46,101
IMPROVE IT AND IF PFIZER'S NOT

7870
05:43:46,101 --> 05:43:47,102
INTERESTED, WE WILL REENGINEER

7871
05:43:47,102 --> 05:43:51,506
AND PROCEED FROM THERE.

7872
05:43:51,506 --> 05:43:52,307
NEXT SLIDE.

7873
05:43:52,307 --> 05:43:53,241
SOPHISTICATED REALLY A KEY TAKE

7874
05:43:53,241 --> 05:43:56,144
AWAY HERE IS THAT ACHIEVING A

7875
05:43:56,144 --> 05:43:57,479
TARGET PROFILE, SOMETIMES IT MAY

7876
05:43:57,479 --> 05:44:01,016
LOOK GOOD IN THE BEGINNING BUT

7877
05:44:01,016 --> 05:44:02,150
IT SOMETIMES REQUIRING WORKING

7878
05:44:02,150 --> 05:44:04,453
EITHER BECAUSE THE ORIGINAL

7879
05:44:04,453 --> 05:44:06,922
DIRECT PATHWAY YOU'RE USING JUST

7880
05:44:06,922 --> 05:44:09,024
TURNS OUT NOT TO BE VIABLE OR

7881
05:44:09,024 --> 05:44:12,194
BECAUSE IT TURNS OUT WHAT THE

7882
05:44:12,194 --> 05:44:13,495
LITERATURE ACCEPTS AS TRUE ISN'T

7883
05:44:13,495 --> 05:44:15,030
WHEN YOU START LOOKING AT IT OR

7884
05:44:15,030 --> 05:44:19,668
ANY OTHER VARIETY OF REASON WHY

7885
05:44:19,668 --> 05:44:22,704
YOU COULD HAVE TROUBLE SELLING

7886
05:44:22,704 --> 05:44:22,904
THIS.

7887
05:44:22,904 --> 05:44:24,306
ONE THINK THIS I WON'T TALK A

7888
05:44:24,306 --> 05:44:25,874
LOT ABOUT IS GETTING FUNDING FOR

7889
05:44:25,874 --> 05:44:27,275
THIS AND THERE'S A LOT OF

7890
05:44:27,275 --> 05:44:29,144
DIFFERENT FUNDING SOURCES FOR

7891
05:44:29,144 --> 05:44:29,478
THIS.

7892
05:44:29,478 --> 05:44:31,613
NHLBI PAID FOR THIS WITH RO-1S

7893
05:44:31,613 --> 05:44:32,514
AND PROJECT GRANTS.

7894
05:44:32,514 --> 05:44:34,116
WE HAD SMALL BUSINESS

7895
05:44:34,116 --> 05:44:36,017
COLLABORATIONS WITH THE

7896
05:44:36,017 --> 05:44:38,320
VENTUREICALTAL AND SMALL

7897
05:44:38,320 --> 05:44:43,325
BUSINESS GRANTS, IS SBIRS AND

7898
05:44:43,325 --> 05:44:44,760
STTRS, SO THERE'S A LOT OF WAYS

7899
05:44:44,760 --> 05:44:46,495
CAN YOU PAY FOR THIS SORT OF

7900
05:44:46,495 --> 05:44:46,828
THING.

7901
05:44:46,828 --> 05:44:50,265
I GUESS WHAT I'M  SAYING IS IS

7902
05:44:50,265 --> 05:44:50,999
THAT MY EXPERIENCE IN THIS

7903
05:44:50,999 --> 05:44:52,834
PROJECT THAT HAS HAD A LOT OF

7904
05:44:52,834 --> 05:44:55,737
LUMPS IN IT, IS THAT IT REALLY

7905
05:44:55,737 --> 05:44:57,539
IS A CONFIRMATION ANECDOTALLY OF

7906
05:44:57,539 --> 05:45:00,108
ALL THE OTHER STUFF THE GUYS

7907
05:45:00,108 --> 05:45:00,575
HAVE TALKED ABOUT.

7908
05:45:00,575 --> 05:45:03,612
AND I THINK THAT'S EVERYTHING I

7909
05:45:03,612 --> 05:45:05,514
HAVE TO SAY.

7910
05:45:05,514 --> 05:45:09,551
IF I MY CO MODERATOR IS READY?

7911
05:45:09,551 --> 05:45:20,095
>> YEAH, I THINK THEY HAVE
IT.

7912
05:45:31,206 --> 05:45:32,374
LET'S SEE.

7913
05:45:32,374 --> 05:45:34,609
AND I WANTED TO LOOK THEA THE

7914
05:45:34,609 --> 05:45:36,144
HURDLES, AND JAMES AS WELL AS WE

7915
05:45:36,144 --> 05:45:37,679
ARE AND OTHER SPEAKERS ON THE

7916
05:45:37,679 --> 05:45:41,716
PANEL AND DR. HOLTZMAN AND

7917
05:45:41,716 --> 05:45:45,153
DR. BENOIT, THAT YOU KNOW IN

7918
05:45:45,153 --> 05:45:46,788
DEVELOPMENT AND DEFINING THE

7919
05:45:46,788 --> 05:45:48,056
TARGET PRODUCTS AND GETTING THEM

7920
05:45:48,056 --> 05:45:49,691
TO MARKET, WE ARE STILL SORT OF

7921
05:45:49,691 --> 05:45:51,760
AN EARLY STAGES OF TRYING TO GET

7922
05:45:51,760 --> 05:45:54,329
A FEW PRODUCTS TO MARKET, 1 OF

7923
05:45:54,329 --> 05:45:55,564
THEM'S GENE THERAPY WHICH I

7924
05:45:55,564 --> 05:45:59,201
WON'T TALK ABOUT TODAY AND THE

7925
05:45:59,201 --> 05:46:01,303
OTHER IS A LAB TEST FOR

7926
05:46:01,303 --> 05:46:02,571
BIOMARKERS OF DISEASE, WHICH IS

7927
05:46:02,571 --> 05:46:03,405
TURNING INTO AN INTERESTING

7928
05:46:03,405 --> 05:46:05,173
PROJECT BUT I WANTED TO FOCUS

7929
05:46:05,173 --> 05:46:06,875
ON, YOU HEARD A LOT TODAY ABOUT

7930
05:46:06,875 --> 05:46:08,210
-- COULD I HAVE THE NEXT SLIDE

7931
05:46:08,210 --> 05:46:09,744
-- I WANT TO FOCUS ON THE

7932
05:46:09,744 --> 05:46:11,179
HURDLES BECAUSE YOU HEARD A LOT

7933
05:46:11,179 --> 05:46:12,681
ABOUT -- THESE ARE MY

7934
05:46:12,681 --> 05:46:13,548
DISCLOSURES HERE.

7935
05:46:13,548 --> 05:46:15,383
THIS IS OTHER PROJECTS HAVE BEEN

7936
05:46:15,383 --> 05:46:19,187
FUNDED IN THE LAB BY THE NHLBI

7937
05:46:19,187 --> 05:46:20,288
AND CF FOUNDATION.

7938
05:46:20,288 --> 05:46:23,658
WE RECENTLY GOT AN SBIR GRANT

7939
05:46:23,658 --> 05:46:25,160
ASK WE'VE GOTTEN SBIR FUNDING

7940
05:46:25,160 --> 05:46:27,028
BEFORE FOR THE GENE THERAPY WORK

7941
05:46:27,028 --> 05:46:29,097
THAT WORK ON TODAY WAS SUPPORTED

7942
05:46:29,097 --> 05:46:32,934
BY CATALYZE SO THE PROGRAM THAT

7943
05:46:32,934 --> 05:46:34,803
MIKE AND LAURA SPOKE ABOUT

7944
05:46:34,803 --> 05:46:35,837
EARLIER, FANTASTIC PROGRAM AND

7945
05:46:35,837 --> 05:46:36,905
SOMETHING THAT IN MY OPINION

7946
05:46:36,905 --> 05:46:39,007
EVERYONE WHO WANTS TO INNOVATE

7947
05:46:39,007 --> 05:46:40,642
AND REALLY SORT OF REDUCE TO

7948
05:46:40,642 --> 05:46:42,477
PRACTICE WHAT THEY'RE DOING, AND

7949
05:46:42,477 --> 05:46:44,112
THEN NOT JUST HAVE VISION BUT

7950
05:46:44,112 --> 05:46:46,147
TURN IT INTO SOMETHING SHOULD

7951
05:46:46,147 --> 05:46:46,982
REALLY INTERACT AND CROSS PATHS

7952
05:46:46,982 --> 05:46:49,651
WITH THIS PROGRAM AT THE NIH

7953
05:46:49,651 --> 05:46:50,118
IT'S REALLY AMAZING.

7954
05:46:50,118 --> 05:46:54,022
WE'RE WORKING WITH A COMPANY

7955
05:46:54,022 --> 05:46:55,357
CALLED GENEXYS, JUST TO DISCLOSE

7956
05:46:55,357 --> 05:46:57,792
THAT AND I'M AN INVENTOR ON A

7957
05:46:57,792 --> 05:46:59,928
NUMBER OF PATENTS I WILL DISCUSS

7958
05:46:59,928 --> 05:47:00,262
TODAY.

7959
05:47:00,262 --> 05:47:02,030
SO NEXT SLIDE PLEASE.

7960
05:47:02,030 --> 05:47:04,432
I REALLY WANT TO FOCUS ON THE

7961
05:47:04,432 --> 05:47:04,733
CHALLENGES.

7962
05:47:04,733 --> 05:47:07,035
YOU HEARD A LOT ABOUT BALANCING

7963
05:47:07,035 --> 05:47:07,836
VISION AND FEASIBILITY.

7964
05:47:07,836 --> 05:47:10,305
I THINK THAT'S IMPORTANTOT

7965
05:47:10,305 --> 05:47:12,841
SCIENTIFIC LEVEL, VISION IS NOT

7966
05:47:12,841 --> 05:47:15,443
IN SHORT SUPPLY, SO I THINK

7967
05:47:15,443 --> 05:47:16,845
GENERALLY, YOU DON'T GO INTO

7968
05:47:16,845 --> 05:47:18,547
SCIENCE IF YOU'RE NOT A

7969
05:47:18,547 --> 05:47:19,948
VISIONARY KIND OF PERSON THAT

7970
05:47:19,948 --> 05:47:22,017
YOU KNOW WANTED TO WIN THE NOBEL

7971
05:47:22,017 --> 05:47:23,318
FOR CURING ALL DISEASE AT SOME

7972
05:47:23,318 --> 05:47:23,518
POINT.

7973
05:47:23,518 --> 05:47:25,420
I THINK THAT THAT'S THE WAY A

7974
05:47:25,420 --> 05:47:27,589
LOT OF PEOPLE WHO DO BASIC

7975
05:47:27,589 --> 05:47:28,523
SCIENCE OR TRANSLATIONAL SCIENCE

7976
05:47:28,523 --> 05:47:30,125
ARE BUILT TO BEGIN WITH.

7977
05:47:30,125 --> 05:47:32,127
SO VISION IS, I DON'T THINK IN A

7978
05:47:32,127 --> 05:47:33,528
LOT OF SHORT SUPPLY BUT YOU

7979
05:47:33,528 --> 05:47:37,165
REALLY ARE TO COUPLE THAT WITH

7980
05:47:37,165 --> 05:47:41,202
FEASIBILITY AND THAT REALLY IS

7981
05:47:41,202 --> 05:47:42,203
THE -- QUITE IMPORTANT.

7982
05:47:42,203 --> 05:47:43,338
YOU HEARD QUITE A BIT ABOUT THAT

7983
05:47:43,338 --> 05:47:45,006
TODAY, AND YOU HAVE TO MAKE SURE

7984
05:47:45,006 --> 05:47:46,174
YOU CAN SCALE UP WHATEVER YOU'RE

7985
05:47:46,174 --> 05:47:48,276
MAKING, YOU ARE DOING, IF YOU'RE

7986
05:47:48,276 --> 05:47:50,345
MAKING A PRODUCT LIKE A SMALL

7987
05:47:50,345 --> 05:47:53,315
MOLECULE OR A GENE THERAPY

7988
05:47:53,315 --> 05:47:55,750
VECTOR, HAS TO BE SCALABLE, HAS

7989
05:47:55,750 --> 05:47:57,185
TO BE REPRODUCIBLY MADE, HAS TO

7990
05:47:57,185 --> 05:47:58,954
BE TABLE, YOU CAN STORE IT, A

7991
05:47:58,954 --> 05:48:02,924
LOT OF THINGS GO INTO, YOU KNOW

7992
05:48:02,924 --> 05:48:06,294
DEFINING THE PRODUCT ISSUES THE

7993
05:48:06,294 --> 05:48:07,829
TARGET PRODUCT, PART OF WHAT

7994
05:48:07,829 --> 05:48:09,531
GOES INTO DEFINING THAT IS CAN

7995
05:48:09,531 --> 05:48:11,333
YOU ACTUALLY MAKE SOMETHING THAT

7996
05:48:11,333 --> 05:48:13,535
WILL END UP BEING A PRODUCT

7997
05:48:13,535 --> 05:48:16,938
THAT'S VIABLE AND NOT JUST

7998
05:48:16,938 --> 05:48:17,973
SOMETHING THAT AFFECTS A PATHWAY

7999
05:48:17,973 --> 05:48:19,841
IN A CELL LINE IN A SINGLE

8000
05:48:19,841 --> 05:48:20,141
EXPERIMENT.

8001
05:48:20,141 --> 05:48:21,376
IT HAS TO BE SOMETHING THAT CAN

8002
05:48:21,376 --> 05:48:23,678
BE KEPT, YOU KNOW ON THE SHELF

8003
05:48:23,678 --> 05:48:26,648
IN A BOTTLE OR PILL AND

8004
05:48:26,648 --> 05:48:28,216
DELIVERED AND WORK STABLY AND

8005
05:48:28,216 --> 05:48:29,451
NOT BE TOXIC.

8006
05:48:29,451 --> 05:48:31,419
THERE'S A LOST THINGS THAT CAN

8007
05:48:31,419 --> 05:48:36,024
INFORM SELECT, YOU KNOW DEFINING

8008
05:48:36,024 --> 05:48:36,491
THE TARGET PRODUCT.

8009
05:48:36,491 --> 05:48:38,159
SO I THINK YOU HEARD A LOT ABOUT

8010
05:48:38,159 --> 05:48:39,861
THASHES I WANTED TO SORT OF

8011
05:48:39,861 --> 05:48:42,664
TOUCH A LITTLE BIT ON SOME OF

8012
05:48:42,664 --> 05:48:43,965
THE SHORT COMINGS, OR

8013
05:48:43,965 --> 05:48:47,802
LIMITATIONS THAT YOU RUN INTO,

8014
05:48:47,802 --> 05:48:50,639
FOR US ESPECIALLY, YOU KNOW

8015
05:48:50,639 --> 05:48:53,408
ACADEMIA, WHILE A LOT OF THESE

8016
05:48:53,408 --> 05:48:55,744
INNOVATIVE IDEAS DO SORT OF

8017
05:48:55,744 --> 05:48:57,846
PERKULATE IN ACADEMIA, HISTORY

8018
05:48:57,846 --> 05:48:59,347
OF ACADEMICS INTERACTING WITH

8019
05:48:59,347 --> 05:49:00,181
SPIN-OFFS OR COLLABORATING WITH

8020
05:49:00,181 --> 05:49:02,183
INDUSTRY AND COMING UP WITH

8021
05:49:02,183 --> 05:49:03,485
AMAZING THERAPIES, AND A LOST

8022
05:49:03,485 --> 05:49:04,786
THE GREAT THERAPIES OUT THERE

8023
05:49:04,786 --> 05:49:06,421
RIGHT NOW FOR CF, WHICH IS 1

8024
05:49:06,421 --> 05:49:07,956
THING WE WILL TALK ABOUT TODAY,

8025
05:49:07,956 --> 05:49:09,557
1 OF THE GREATEST THERAPIES THAT

8026
05:49:09,557 --> 05:49:11,226
HAVE BEEN THE MODULATORS

8027
05:49:11,226 --> 05:49:16,564
BASICALLY WERE DEVELOPED USING

8028
05:49:16,564 --> 05:49:18,500
BASIC SCIENCE ASSAYS LIKE

8029
05:49:18,500 --> 05:49:20,301
[INDISCERNIBLE] CHAMBER

8030
05:49:20,301 --> 05:49:21,236
MEASUREMENTS AND CHLORIDE

8031
05:49:21,236 --> 05:49:22,637
FUNCTION TESTS FOR ACTIVATION OF

8032
05:49:22,637 --> 05:49:24,272
THE CHANNELS FOR SMALL

8033
05:49:24,272 --> 05:49:24,539
MOLECULES.

8034
05:49:24,539 --> 05:49:26,341
SO BASIC SCIENCE PLAYED A BIG

8035
05:49:26,341 --> 05:49:29,411
ROLE BUT ACADEMIC SETTINGS,

8036
05:49:29,411 --> 05:49:32,914
FIRST OF ALL HAVE SOME SHORT

8037
05:49:32,914 --> 05:49:33,248
COMINGS.

8038
05:49:33,248 --> 05:49:35,250
ONE IS THERE'S MORE OF A PUSH TO

8039
05:49:35,250 --> 05:49:37,218
INVENTION RATHER THAN A PULL IN

8040
05:49:37,218 --> 05:49:39,087
RESPONSE TO A PREDEFINED

8041
05:49:39,087 --> 05:49:40,722
VALIDATED USER NEED, SO, I THINK

8042
05:49:40,722 --> 05:49:42,691
YOU HEARD THAT IN A COUPLE OF

8043
05:49:42,691 --> 05:49:44,859
TALKS TODAY, THAT HAVING A VERY

8044
05:49:44,859 --> 05:49:48,797
DEFINED NEED AND AN IMPORTANT

8045
05:49:48,797 --> 05:49:51,166
MARKET SPACE, AND THAT HAS A

8046
05:49:51,166 --> 05:49:52,567
DESCENT MARKET SHARE, SO THAT

8047
05:49:52,567 --> 05:49:54,235
INVESTORS WILL BE INTERESTED IN

8048
05:49:54,235 --> 05:49:56,504
INVESTING IN IT, THAT CAN BE

8049
05:49:56,504 --> 05:50:01,009
PROTECTED, IS AN IMPORTANT PART

8050
05:50:01,009 --> 05:50:04,412
OF DESIGNING YOUR TARGET

8051
05:50:04,412 --> 05:50:04,779
PRODUCT.

8052
05:50:04,779 --> 05:50:06,514
I THINK IT'S -- YOU REALLY HAVE

8053
05:50:06,514 --> 05:50:08,650
TO THINK ABOUT A LOT ABOUT

8054
05:50:08,650 --> 05:50:09,918
FORMALIZING THE ENVIRONMENTS OF

8055
05:50:09,918 --> 05:50:12,887
DEFINING THE PRODUCT IN A FORMAL

8056
05:50:12,887 --> 05:50:14,089
WAY EMPLOY THAT'S PROBABLY WHERE

8057
05:50:14,089 --> 05:50:17,158
SOME OF THE FRICTION OCCURS IN

8058
05:50:17,158 --> 05:50:20,795
ACADEMIA, THE WAYS THAT WE ARE

8059
05:50:20,795 --> 05:50:24,065
TRAINED AND THINK ABOUT DEFINING

8060
05:50:24,065 --> 05:50:24,999
EXPERIMENTS AND CONTROLS, ARE

8061
05:50:24,999 --> 05:50:27,969
IMPORTANT AND NECESSARY, BUT

8062
05:50:27,969 --> 05:50:29,137
THERE'S ALSO STANDARD OPERATING

8063
05:50:29,137 --> 05:50:30,205
PROCEDURES THAT CAN BE VERY

8064
05:50:30,205 --> 05:50:32,173
DETAILED AND HAVE TO BE VERY

8065
05:50:32,173 --> 05:50:34,008
MODULAR SO THAT THEY CAN BE

8066
05:50:34,008 --> 05:50:35,777
EASILY CARRIED OUT IN DIFFERENT

8067
05:50:35,777 --> 05:50:37,145
LABORATORIES AND DIFFERENT

8068
05:50:37,145 --> 05:50:38,179
SETTINGS AND THERE'S NOT SORT OF

8069
05:50:38,179 --> 05:50:39,314
CENTER CENTER FOR EXCELLENCE ON

8070
05:50:39,314 --> 05:50:41,082
AGINGS AND WHAT HAVE YOU THAT

8071
05:50:41,082 --> 05:50:42,016
HAPPENS IN ACADEMICS AND

8072
05:50:42,016 --> 05:50:43,585
RESEARCH HAVE TO BE CONSIDERED

8073
05:50:43,585 --> 05:50:45,587
AND REALLY QUITE A LOT OF TIME

8074
05:50:45,587 --> 05:50:49,224
AND MONEY TO SORT OF NAIL DOWN.

8075
05:50:49,224 --> 05:50:51,259
I THINK ALSO FROM THE VERY EARLY

8076
05:50:51,259 --> 05:50:52,794
POINT AND YOU'VE HEARD A BUNCH

8077
05:50:52,794 --> 05:50:54,295
OF TALKS ABOUT THIS TODAY AS

8078
05:50:54,295 --> 05:50:56,931
WELL, YOU HAVE TO THINK ABOUT

8079
05:50:56,931 --> 05:51:00,969
REGULATORY AND REIMBURSEMENT

8080
05:51:00,969 --> 05:51:02,403
CONSIDERATIONS, AND IN DESIGNING

8081
05:51:02,403 --> 05:51:05,106
OR DEFINING YOUR TARGET

8082
05:51:05,106 --> 05:51:06,741
PRODUCTS, EVEN THOUGH, YOU KNOW

8083
05:51:06,741 --> 05:51:08,510
YOU HAVE A SCIENTIFIC QUESTION

8084
05:51:08,510 --> 05:51:09,544
AND SCIENTIFIC PROBLEM, YOU'RE

8085
05:51:09,544 --> 05:51:11,312
TRYING TO SOLVE LIKE JAMES JUST

8086
05:51:11,312 --> 05:51:13,515
SHOWED, YOU REALLY WANT TO BE

8087
05:51:13,515 --> 05:51:15,483
THINKING ABOUT WHAT THE

8088
05:51:15,483 --> 05:51:16,918
REGULATORY AND THE REIMBURSEMENT

8089
05:51:16,918 --> 05:51:18,286
LOOKS FOR THAT, LOOKS LIKE FOR

8090
05:51:18,286 --> 05:51:19,354
THAT, VERY EARLY ON WHICH IS

8091
05:51:19,354 --> 05:51:22,757
SOMETHING WE'RE NOT TRAINED TO

8092
05:51:22,757 --> 05:51:24,526
DO AND SO, -- SO YOU SORT OF

8093
05:51:24,526 --> 05:51:28,696
HAVE TO THINK BACKWARDS FROM HOW

8094
05:51:28,696 --> 05:51:30,198
SAFE WILL THIS BE?

8095
05:51:30,198 --> 05:51:31,566
WHAT KIND OF REIMBURSEMENT WILL

8096
05:51:31,566 --> 05:51:32,033
THERE BE?

8097
05:51:32,033 --> 05:51:34,269
WHAT KIND OF COVERAGE?

8098
05:51:34,269 --> 05:51:37,405
, THENEY NEED AND SAFETY PROFILE

8099
05:51:37,405 --> 05:51:39,908
OF THIS, SORT OF YOU START THERE

8100
05:51:39,908 --> 05:51:40,875
AND WORK YOURSELF BACK TO WHAT

8101
05:51:40,875 --> 05:51:42,377
THE PRODUCT IS GOING TO BE.

8102
05:51:42,377 --> 05:51:46,214
IF YOU START WITH SOMETHING THAT

8103
05:51:46,214 --> 05:51:46,915
MISSES THESE CONSIDERATIONS, YOU

8104
05:51:46,915 --> 05:51:48,783
CAN SORT OF CATCH UP ON THEM

8105
05:51:48,783 --> 05:51:50,418
LATER ON BUT YOU COULD RUN INTO

8106
05:51:50,418 --> 05:51:51,786
TROUBLE WHERE YOU COULD FIND

8107
05:51:51,786 --> 05:51:54,055
YOURSELF FAR OFF THE MARK AND

8108
05:51:54,055 --> 05:52:01,796
THEN YOUR PRODUCT SORT OF FAILS,

8109
05:52:01,796 --> 05:52:02,831
NEXT SLIDE, PLEASE.

8110
05:52:02,831 --> 05:52:05,066
IT'S REALLY THESE 2 SLIDES ON

8111
05:52:05,066 --> 05:52:05,967
THE TRANSFORMATIONS, THE

8112
05:52:05,967 --> 05:52:07,502
TRANSFORMATIONS FOR US THE

8113
05:52:07,502 --> 05:52:09,370
BIGGEST CHALLENGES HAVE BEEN

8114
05:52:09,370 --> 05:52:11,206
INSTITUTIONAL 1S, SORT OF IN THE

8115
05:52:11,206 --> 05:52:14,909
ROOM AT LEAST FOR US IS THAT

8116
05:52:14,909 --> 05:52:16,411
INSTITUTIONS AND ACADEMIC

8117
05:52:16,411 --> 05:52:17,011
INSTITUTIONSENTIOUS SPECIALLY

8118
05:52:17,011 --> 05:52:24,352
ARE NOT BUILT TO TAKE HIGH RISK.

8119
05:52:24,352 --> 05:52:25,720
YOU KNOW INCREMENTAL MOTIVATION

8120
05:52:25,720 --> 05:52:27,589
IS REWARDED BECAUSE THAT'S WHERE

8121
05:52:27,589 --> 05:52:29,357
THE FUNDING AS BEEN GEARED OVER

8122
05:52:29,357 --> 05:52:33,328
THE LAST 30 YEAR ANDS THAT'S

8123
05:52:33,328 --> 05:52:40,902
CHANGING IN THE DISCUSSIONS WITH

8124
05:52:40,902 --> 05:52:42,170
MARA, AND CHANGES WILL NOT BE

8125
05:52:42,170 --> 05:52:44,672
WILL ONLY THINGS THAT ARE

8126
05:52:44,672 --> 05:52:55,183
CONSIDERED FOR THESE BREAD AND

8127
05:52:56,384 --> 05:52:58,253
BUTTER GRANTS AND RO-1 GRANTS

8128
05:52:58,253 --> 05:53:01,189
WHICH ARE TYPICALLY NOT THAT

8129
05:53:01,189 --> 05:53:02,490
RISKY AND SO THE BETTER THE

8130
05:53:02,490 --> 05:53:05,793
RANKING OF AN INSTITUTION, THE

8131
05:53:05,793 --> 05:53:07,328
YOU KNOW THAT THERE IS A

8132
05:53:07,328 --> 05:53:08,129
LIKELIHOOD THAT THAT INSTITUTION

8133
05:53:08,129 --> 05:53:09,797
WILL NOT BE THE BEST PLACE TO

8134
05:53:09,797 --> 05:53:12,867
INNOVATE AND NOT BE THE BEST

8135
05:53:12,867 --> 05:53:14,202
PLACE TO SUPPORT INNOVATION AND

8136
05:53:14,202 --> 05:53:15,336
I'M TALKING ABOUT INSTITUTIONAL

8137
05:53:15,336 --> 05:53:18,072
SUPPORT SO I'M TALKING ABOUT

8138
05:53:18,072 --> 05:53:20,708
THINGS LIKE SUPPORT WITHIN

8139
05:53:20,708 --> 05:53:25,179
DIVISIONS AND DEPARTMENTS IN

8140
05:53:25,179 --> 05:53:27,248
TERMS OF YOU KNOW SEED FUNDING,

8141
05:53:27,248 --> 05:53:28,683
START UP FUNDING AND INTERNAL

8142
05:53:28,683 --> 05:53:30,652
FUNDING WHICH CAN HELP A LOT

8143
05:53:30,652 --> 05:53:32,420
ESPECIALLY WHEN YOU GET AN IDEA

8144
05:53:32,420 --> 05:53:34,789
HERE OR THERE WHETHER THE SEED

8145
05:53:34,789 --> 05:53:36,357
FUNDING AND WHICH IS USUALLY

8146
05:53:36,357 --> 05:53:37,692
AVAILABLE AT THE INCREMENTAL

8147
05:53:37,692 --> 05:53:38,960
FOLKS SO THEY BRING IN MORE

8148
05:53:38,960 --> 05:53:41,162
GRANTS OR WHETHER YOU CAN GO FOR

8149
05:53:41,162 --> 05:53:42,697
MOVING TECHNOLOGY AND A LOT OF

8150
05:53:42,697 --> 05:53:52,206
TIMES THAT'S A CHALLENGE, AND

8151
05:53:52,206 --> 05:53:56,277
SO, ANOTHER THING IS IDENTIFYING

8152
05:53:56,277 --> 05:53:56,678
TARGET.

8153
05:53:56,678 --> 05:53:57,712
I THINK PEOPLE DON'T THINK ABOUT

8154
05:53:57,712 --> 05:53:58,646
THAT ENOUGH, AND YOU HAVE TO

8155
05:53:58,646 --> 05:53:59,747
THINK ABOUT IT EARLY ON AND IF

8156
05:53:59,747 --> 05:54:01,249
YOU DON'T HAVE A PARTNER THAT

8157
05:54:01,249 --> 05:54:06,020
WILL HELP YOU COMMERCIALIZE A

8158
05:54:06,020 --> 05:54:07,288
PRODUCT, NO MATTER WHAT THE

8159
05:54:07,288 --> 05:54:10,959
TARGET OF YOUR PRODUCT, IF IT'S

8160
05:54:10,959 --> 05:54:11,726
NOT SOMETHING COMMERCIALIZATION

8161
05:54:11,726 --> 05:54:12,727
PARTNERS ARE NOT INTERESTED IN,

8162
05:54:12,727 --> 05:54:15,263
AND I THINK YOU HAVE TO BE

8163
05:54:15,263 --> 05:54:16,764
REALLY GOOD AT INTERDISCIPLINARY

8164
05:54:16,764 --> 05:54:19,033
COORDINATION SO YOU WILL HAVE TO

8165
05:54:19,033 --> 05:54:20,668
WORK TOGETHER WITH RESEARCH AND

8166
05:54:20,668 --> 05:54:21,803
DEVELOPMENT FOLKS, YOU HAVE TO

8167
05:54:21,803 --> 05:54:23,204
THINK ABOUT MARKETING, YOU HAVE

8168
05:54:23,204 --> 05:54:24,539
TO THINK ABOUT MANUFACTURING AND

8169
05:54:24,539 --> 05:54:29,477
SCALE UP, YOU HAVE TO THINK

8170
05:54:29,477 --> 05:54:30,979
ABOUT PRIORITIES ALIGNING AND

8171
05:54:30,979 --> 05:54:33,481
YOU KNOW THAT'S COMPLEX TO DO.

8172
05:54:33,481 --> 05:54:39,554
AND SO IN MY OPINION, AND I

8173
05:54:39,554 --> 05:54:40,722
THINK MOST INVENTORS AND START

8174
05:54:40,722 --> 05:54:43,424
UP FOLKS ON HERE WOULD AGREE

8175
05:54:43,424 --> 05:54:48,162
THAT A LOT OF SUCCESS OF THESE

8176
05:54:48,162 --> 05:54:49,530
TECHNOLOGIES AND PICKING THE

8177
05:54:49,530 --> 05:54:51,833
RIGHT TARGET DOES HINGE ON A

8178
05:54:51,833 --> 05:54:54,135
SAVVY INVENTOR AND YOU HAVE TO

8179
05:54:54,135 --> 05:54:57,171
BE -- SUCCESS IS VERY SAVVY

8180
05:54:57,171 --> 05:54:58,206
INVENTOR-DEPENDENT WHICH I THINK

8181
05:54:58,206 --> 05:54:59,941
MISSES A LOT OF OPPORTUNITIES SO

8182
05:54:59,941 --> 05:55:01,242
THERE'S A LOT OF TIEWPTS THAT

8183
05:55:01,242 --> 05:55:02,744
ARE MISSED I THINK FOR PEOPLE

8184
05:55:02,744 --> 05:55:03,911
THAT AREN'T SAVVY THAT CAN'T

8185
05:55:03,911 --> 05:55:06,748
DEAL WITH THE POLITICS, THAT

8186
05:55:06,748 --> 05:55:09,484
CAN'T DEAL WITH THE MACHINATIONS

8187
05:55:09,484 --> 05:55:11,052
OF ACADEMIA AND WHAT IT REWARDS.

8188
05:55:11,052 --> 05:55:13,121
AND I THINK A LOT OF REALLY GOOD

8189
05:55:13,121 --> 05:55:14,856
IDEAS DO SORT OF FALL BY THE WAY

8190
05:55:14,856 --> 05:55:16,257
SIDE BECAUSE THEY'RE NOT SORT OF

8191
05:55:16,257 --> 05:55:19,227
-- THEY DON'T HAVE CHAMPIONS IN

8192
05:55:19,227 --> 05:55:21,095
PLACES WHERE INNOVATION IS SORT

8193
05:55:21,095 --> 05:55:22,463
OF TOUTED AS SOMETHING PEOPLE

8194
05:55:22,463 --> 05:55:24,866
ARE INTERESTED IN BUT REALLY

8195
05:55:24,866 --> 05:55:25,900
ACADEMICALLY SPEAKS THESE

8196
05:55:25,900 --> 05:55:27,535
INSTITUTIONS ARE NOT THAT

8197
05:55:27,535 --> 05:55:28,469
HEAVILY INVESTED.

8198
05:55:28,469 --> 05:55:29,771
NOW THERE ARE EXCEPTIONS TO

8199
05:55:29,771 --> 05:55:31,773
THAT, YOU KNOW I USED TO WORK AT

8200
05:55:31,773 --> 05:55:33,608
EMORY AND EMORY IS A PLACE THAT

8201
05:55:33,608 --> 05:55:36,144
YOU KNOW CAME UP WITH THE

8202
05:55:36,144 --> 05:55:38,312
REPLACEMENT FOR AZT AND THEY SAW

8203
05:55:38,312 --> 05:55:40,181
WHAT MOVING THE NEEDLE COULD

8204
05:55:40,181 --> 05:55:45,019
REALLY BE, AND SO, YOU KNOW THAT

8205
05:55:45,019 --> 05:55:47,121
WAS -- THAT WAS A PLACE WHERE

8206
05:55:47,121 --> 05:55:48,456
INNOVATION WAS REALLY HEAVILY

8207
05:55:48,456 --> 05:55:48,723
SUPPORTED.

8208
05:55:48,723 --> 05:55:50,391
WE WILL GO TO THE NEXT SLIDE.

8209
05:55:50,391 --> 05:55:52,293
SO A COUPLE TRY TO MOVE A LITTLE

8210
05:55:52,293 --> 05:55:57,765
FASTER HERE TO START THE PANEL

8211
05:55:57,765 --> 05:55:59,667
DISCUSSION, BUT TELL YOU ABOUT

8212
05:55:59,667 --> 05:56:01,636
THE BIOMARKERS WE'RE WORKING ON.

8213
05:56:01,636 --> 05:56:03,971
NEXT SLIDE, PLEASE.

8214
05:56:03,971 --> 05:56:09,010
SO THIS IS AN ATTEMPT TO TROY TO

8215
05:56:09,010 --> 05:56:12,080
GET AT THE [INDISCERNIBLE]

8216
05:56:12,080 --> 05:56:15,049
INDUSTRY IN CF LUNG DECLINE, SO

8217
05:56:15,049 --> 05:56:16,417
IN CF YOU HAVE THIS YEEN THAT

8218
05:56:16,417 --> 05:56:17,752
LEADS TO THE LUNG DISEASE, AND

8219
05:56:17,752 --> 05:56:22,557
IF YOU GO TO NEXT CLICK, PLEASE.

8220
05:56:22,557 --> 05:56:23,357
NEXT.

8221
05:56:23,357 --> 05:56:24,692
SO THIS IS AN EXAMPLE OF WHAT A

8222
05:56:24,692 --> 05:56:26,627
PATIENT LOOKS LIKE AND YOU CAN

8223
05:56:26,627 --> 05:56:29,697
SEE THAT THERE FEV1 LUNG

8224
05:56:29,697 --> 05:56:31,766
FUNCTION IS BECOMING MORE

8225
05:56:31,766 --> 05:56:32,934
ERRATIC WITH TIME AND IT'S HARD

8226
05:56:32,934 --> 05:56:33,801
TO PREDICT THAT WHEN YOU DON'T

8227
05:56:33,801 --> 05:56:35,970
KNOW WHAT THE REST OF THE

8228
05:56:35,970 --> 05:56:37,839
TRACINGS LOOK LEAK AND SO, NEXT

8229
05:56:37,839 --> 05:56:42,643
SLIDE, PLEASE, SO PRESENTLY USED

8230
05:56:42,643 --> 05:56:43,044
BIOMARKERS, NEXT.

8231
05:56:43,044 --> 05:56:44,712
I DIDN'T REALIZE THIS HAD SO

8232
05:56:44,712 --> 05:56:47,815
MANY -- ARE ALL LAGGING

8233
05:56:47,815 --> 05:56:49,383
INDICATORS AND WILL TELL YOU

8234
05:56:49,383 --> 05:56:50,585
ONCE IT'S BEEN ESTABLISHED SO

8235
05:56:50,585 --> 05:56:52,987
IT'S DIFFICULT TO REVERSE.

8236
05:56:52,987 --> 05:56:54,789
SO NEXT SLIDE.

8237
05:56:54,789 --> 05:56:55,857
AND THERE'S GOOD EVIDENCE THAT

8238
05:56:55,857 --> 05:56:57,525
THERE CONTINUES TO BE LUNG

8239
05:56:57,525 --> 05:56:58,493
FUNCTION DEDEADLINE SO THE

8240
05:56:58,493 --> 05:57:00,094
LIGHTER TRACE THAT'S ON TOP

8241
05:57:00,094 --> 05:57:01,963
THERE, THE GRAY TRACE IS SORT

8242
05:57:01,963 --> 05:57:04,365
OF, YOU CAN SEE LUNG FUNCTION

8243
05:57:04,365 --> 05:57:05,733
DECLINE THERE AND TRAJECTORY

8244
05:57:05,733 --> 05:57:07,568
LOOKS THE SAME AS NONTREATED

8245
05:57:07,568 --> 05:57:11,372
FOLKS IN THE BLACK TRACING,

8246
05:57:11,372 --> 05:57:13,708
EXACERBATIONS ARE DOWN BUT HIGH

8247
05:57:13,708 --> 05:57:14,742
EFFICACY MODULATORS, LUNG

8248
05:57:14,742 --> 05:57:15,710
FUNCTION DELINES IN CF AND

8249
05:57:15,710 --> 05:57:17,445
THAT'S FOR PEOPLE WHO CAN

8250
05:57:17,445 --> 05:57:20,047
TOLERATE THESE NEW MODULATORS.

8251
05:57:20,047 --> 05:57:22,783
SO THERE'S A NEED TO TRY TO --

8252
05:57:22,783 --> 05:57:25,553
THERE'S GOOD EVIDENCE THAT IF

8253
05:57:25,553 --> 05:57:28,422
YOU IMPROVE, -- IF YOU DO MORE

8254
05:57:28,422 --> 05:57:29,824
MONITORING FOR FOLKS, ON THE

8255
05:57:29,824 --> 05:57:31,125
LEFT-HAND SIDE HERE, CAN YOU SEE

8256
05:57:31,125 --> 05:57:36,097
THE IMPROVEMENT IN FEV1 SCORES.

8257
05:57:36,097 --> 05:57:38,666
IF YOU MONITOR PEOPLE WITHIN 28

8258
05:57:38,666 --> 05:57:40,501
DAYS, REPEAT VISITS RATHER THAN

8259
05:57:40,501 --> 05:57:43,938
THE 3 MONTHS REPEAT VISITS, IF

8260
05:57:43,938 --> 05:57:46,774
THEY FALL A CERTAIN PERCENTAGE,

8261
05:57:46,774 --> 05:57:48,042
IF YOU SEE THEM ONCE OVERALL

8262
05:57:48,042 --> 05:57:49,377
STRATEGY 4 WEEKS YOU THIS IS THE

8263
05:57:49,377 --> 05:57:51,479
IMPROVEMENT YOU CAN GET OF

8264
05:57:51,479 --> 05:57:53,714
CONVENTIONAL THERAPY OF CF

8265
05:57:53,714 --> 05:57:59,387
WITHOUT MODULATED THERAPY.

8266
05:57:59,387 --> 05:57:59,854
NEXT, PLEASE.

8267
05:57:59,854 --> 05:58:01,689
I THINK WE CAN ANY TO THE NEXT

8268
05:58:01,689 --> 05:58:01,956
1.

8269
05:58:01,956 --> 05:58:03,457
WHAT WE DEVELOPED IN THE LAB WAS

8270
05:58:03,457 --> 05:58:04,959
TO USE A HIGH THROUGH PUT

8271
05:58:04,959 --> 05:58:06,160
PROCEED I DON'T MEANIC APPROACH,

8272
05:58:06,160 --> 05:58:08,262
WHICH LOOKED AT 10S OF THOUSANDS

8273
05:58:08,262 --> 05:58:09,664
OF PROTEINS AND ACTUALLY FOUND

8274
05:58:09,664 --> 05:58:11,566
ABOUT 1100 PROTEINS THAT WERE

8275
05:58:11,566 --> 05:58:13,568
DEFINITE IN THE BLOOD THAT CAN

8276
05:58:13,568 --> 05:58:15,736
PREDICT WHAT HAPPENS WITH LUNG

8277
05:58:15,736 --> 05:58:16,204
FUNCTION.

8278
05:58:16,204 --> 05:58:17,071
COMPLEX, LUNG FUNCTION IS NOT 1

8279
05:58:17,071 --> 05:58:18,606
OF THESE THINGS WHERE YOU HAVE 1

8280
05:58:18,606 --> 05:58:19,540
MARKER THAT TELLS WHAT YOU'S

8281
05:58:19,540 --> 05:58:20,641
GOING ON WITH IT AND SO IF YOU

8282
05:58:20,641 --> 05:58:25,012
GO TO THE NEXT SLIDE, PLEASE.

8283
05:58:25,012 --> 05:58:28,883
THIS MAX SPEC DATA GENERATED

8284
05:58:28,883 --> 05:58:29,951
BASICALLY 474 MARKERS WHICH IS

8285
05:58:29,951 --> 05:58:31,686
WE CAN ONLY DO MY MASS SPEC

8286
05:58:31,686 --> 05:58:33,421
BECAUSE YOU CAN'T DO SOMETHING

8287
05:58:33,421 --> 05:58:35,022
LIKE A LUMEN EXPECTATIONS WHICH

8288
05:58:35,022 --> 05:58:40,194
IS LIMITED TO 8-12 BIOMARKERS OR

8289
05:58:40,194 --> 05:58:43,531
SO, BUT IT GENERATED 478 MARKERS

8290
05:58:43,531 --> 05:58:46,267
IN BLOOD THAT IF YOU LOOK AT IN

8291
05:58:46,267 --> 05:58:49,770
PRINCIPAL DEPUTY PAL COMPONENT

8292
05:58:49,770 --> 05:58:51,138
ANALYSIS COULD SEGGREGATE SEVERE

8293
05:58:51,138 --> 05:58:56,744
CF POPULATION AND LUNG FUNCTION

8294
05:58:56,744 --> 05:58:59,647
DECLINE PROGRESSION OVER THE

8295
05:58:59,647 --> 05:59:02,216
NEXT 3-24 MONTHS AND IT DID THAT

8296
05:59:02,216 --> 05:59:03,150
DURING STABLE DISEASE, SO THIS

8297
05:59:03,150 --> 05:59:04,819
IS NOT WHEN THEY'RE EXACERBATING

8298
05:59:04,819 --> 05:59:07,121
OR WHEN THEY'RE IN THE HOSPITAL

8299
05:59:07,121 --> 05:59:08,990
AND MEASURING WHICH MOST

8300
05:59:08,990 --> 05:59:10,057
BIOMARKERS DEVELOP FOR CFR AND

8301
05:59:10,057 --> 05:59:11,692
THIS IS WHEN THEY'RE IN STABLE

8302
05:59:11,692 --> 05:59:12,860
DISEASE AND THERE'S NO ISSUE.

8303
05:59:12,860 --> 05:59:15,529
AND YOU CAN SEE ON THE RIGHT

8304
05:59:15,529 --> 05:59:19,200
THERE, THAT OUR FIRST GENERATION

8305
05:59:19,200 --> 05:59:21,602
TEST FOR FIBROSIS, AND CF AND

8306
05:59:21,602 --> 05:59:25,172
OTHER DISEASES ACTUALLY HAS GOT

8307
05:59:25,172 --> 05:59:28,042
AN AUC OF 8.4, WHICH WE'RE NOW

8308
05:59:28,042 --> 05:59:29,143
DEVELOPING AND WE'RE VALIDATING

8309
05:59:29,143 --> 05:59:31,078
IN THE LARGE NUMBER OF SAMPLES

8310
05:59:31,078 --> 05:59:33,214
RIGHT NOW AND GETTING READY TO

8311
05:59:33,214 --> 05:59:36,917
GO BACK TOCCATAALIZE. 

8312
05:59:36,917 --> 05:59:37,485
NEXT SLIDE, PLEASE.

8313
05:59:37,485 --> 05:59:41,922
I THINK WE CAN SKIP THIS 1.

8314
05:59:41,922 --> 05:59:43,491
IT'S A SUMMARY OF WHAT THE

8315
05:59:43,491 --> 05:59:44,659
TECHNOLOGY LOOKS LIKE.

8316
05:59:44,659 --> 05:59:45,693
NEXT SLIDE, PLEASE.

8317
05:59:45,693 --> 05:59:46,961
I THINK JUST THE KEY TAKE ARK

8318
05:59:46,961 --> 05:59:51,032
WAYS FOR ME WOULD BE BALANCE,

8319
05:59:51,032 --> 05:59:51,599
VISION WITH FEASIBILITY.

8320
05:59:51,599 --> 05:59:53,467
THINK ABOUT WHAT IS FEASIBLE

8321
05:59:53,467 --> 05:59:54,735
BECAUSE THAT WILL EFFECT THE

8322
05:59:54,735 --> 05:59:58,439
PRODUCT THAT YOU END UP MAKING,

8323
05:59:58,439 --> 06:00:00,308
I THINK IT'S -- YOU HAVE TO BE

8324
06:00:00,308 --> 06:00:01,275
GOOD AT BUILDING GOOD

8325
06:00:01,275 --> 06:00:02,310
COLLABORATIONS AND THEY HAVE TO

8326
06:00:02,310 --> 06:00:04,178
BE STRONG AND YOU ALSO HAVE TO

8327
06:00:04,178 --> 06:00:07,048
HAVE DISCUSSIONS THAT ARE VERY

8328
06:00:07,048 --> 06:00:07,548
TRANSPARENT, SOMETIMES

8329
06:00:07,548 --> 06:00:09,016
COLLABORATIONS DON'T GO IN THE

8330
06:00:09,016 --> 06:00:10,084
RIGHT DIRECTION AND WHAT

8331
06:00:10,084 --> 06:00:11,819
SOMEBODY'S PROVIDING TO THE TEAM

8332
06:00:11,819 --> 06:00:13,988
IS NOT ACTUALLY HELPING SOLVE

8333
06:00:13,988 --> 06:00:16,057
THE PROBLEM OR MAKE THE PRODUCT

8334
06:00:16,057 --> 06:00:18,793
BETTER OR IMPROVE THE PRODUCT OR

8335
06:00:18,793 --> 06:00:20,328
SCALE IT UP OR MARKET IT OR WHAT

8336
06:00:20,328 --> 06:00:23,064
HAVE YOU AND YOU HAVE TO EITHER,

8337
06:00:23,064 --> 06:00:25,132
YOU KNOW ADJUST OR FIND OTHER

8338
06:00:25,132 --> 06:00:25,466
COLLABORATORS.

8339
06:00:25,466 --> 06:00:27,468
SO IT IS SOMETHING THAT YOU HAVE

8340
06:00:27,468 --> 06:00:29,236
TO BE VERY GOOD AT DOING AND YOU

8341
06:00:29,236 --> 06:00:31,906
CAN'T JUST BE STUCK IN THE SAME

8342
06:00:31,906 --> 06:00:38,212
SORT OF MODE OF  IGNORING WHAT

8343
06:00:38,212 --> 06:00:40,781
THE TARGET IS RATHER THAN JUST

8344
06:00:40,781 --> 06:00:42,983
TO BE NICE IN A SENSE AND I

8345
06:00:42,983 --> 06:00:44,618
THINK ALSO BE VERY CONSCIOUS OF

8346
06:00:44,618 --> 06:00:46,053
THE INSTITUTIONAL CAPACITY WHICH

8347
06:00:46,053 --> 06:00:51,125
IS ALWAYS A CHAL  CHALLENGE ANDY

8348
06:00:51,125 --> 06:00:52,093
AGAIN BE TERRIBLE FOR

8349
06:00:52,093 --> 06:00:52,727
TRANSLATION EMPLOY SO YOU HAVE

8350
06:00:52,727 --> 06:00:55,763
TO BE A BIT SAVVY AND I THINK MY

8351
06:00:55,763 --> 06:00:57,331
ONLY NOTE WOULD BE IF THE NIH

8352
06:00:57,331 --> 06:00:59,834
CAME UP WITH MORE PROGRAMS LIKE

8353
06:00:59,834 --> 06:01:04,105
CATALYZE WHICH ACTUALLY

8354
06:01:04,105 --> 06:01:04,839
INCENTIVIZE INSTITUTIONS TO

8355
06:01:04,839 --> 06:01:06,107
REWARD, YOU KNOW HIGH RISK

8356
06:01:06,107 --> 06:01:07,541
INNOVATION, I THINK THAT THAT

8357
06:01:07,541 --> 06:01:09,543
WOULD SHIFT THE CULTURE IN THESE

8358
06:01:09,543 --> 06:01:10,177
ACADEMIC CENTERS BECAUSE REALLY

8359
06:01:10,177 --> 06:01:14,048
THEY JUST FOLLOW THE MONEY AND

8360
06:01:14,048 --> 06:01:15,616
THAT WOULD REALLY HAVE A BIG

8361
06:01:15,616 --> 06:01:18,219
IMPACT ON CATCHING A LOT OF THIS

8362
06:01:18,219 --> 06:01:19,186
MISSED OPPORTUNITY I THINK THAT

8363
06:01:19,186 --> 06:01:20,454
FALLS BY THE WAY SIDE BECAUSE

8364
06:01:20,454 --> 06:01:22,056
PEOPLE DON'T GO THROUGH THE

8365
06:01:22,056 --> 06:01:23,657
TROUBLE OR THEY'RE NOT SAVVY

8366
06:01:23,657 --> 06:01:24,692
ENOUGH TO NAVIGATE THE

8367
06:01:24,692 --> 06:01:25,893
ENVIRONMENT TO KEEP THEIR

8368
06:01:25,893 --> 06:01:26,594
TECHNOLOGIES ALIVE.

8369
06:01:26,594 --> 06:01:27,628
THAT'S IT FOR ME.

8370
06:01:27,628 --> 06:01:36,837
SORRY IT TOOK A BIT LONG.

8371
06:01:36,837 --> 06:01:37,204
>> ALL RIGHT.

8372
06:01:37,204 --> 06:01:38,639
>> WE WILL HAVE THE
PANELISTS

8373
06:01:38,639 --> 06:01:44,712
COME ON THE CAMERA, WE WILL

8374
06:01:44,712 --> 06:01:45,045
SPOTLIGHT YOU.

8375
06:01:45,045 --> 06:01:45,880
>> THANK YOU VERY MUCH,

8376
06:01:45,880 --> 06:01:46,313
EVERYONE.

8377
06:01:46,313 --> 06:01:48,682
I THINK WE HAVE A REALLY

8378
06:01:48,682 --> 06:01:49,350
CONSISTENT MESSAGE HERE ALTHOUGH

8379
06:01:49,350 --> 06:01:51,685
DIFFERENT FROM THE DIFFERENT

8380
06:01:51,685 --> 06:01:53,654
PEOPLE.

8381
06:01:53,654 --> 06:01:55,055
WE DON'T HAVE ANY QUESTIONS FROM

8382
06:01:55,055 --> 06:01:58,959
THE AUDIENCE YET BUT I THOUGHT I

8383
06:01:58,959 --> 06:02:02,263
WOULD START BY ASKING INTERNAL

8384
06:02:02,263 --> 06:02:03,397
QUESTION WHICH IS WHAT DO YOU

8385
06:02:03,397 --> 06:02:05,332
THINK THE POINT AT WHICH OF

8386
06:02:05,332 --> 06:02:06,700
HAD-SO RESEARCHERS ARE PRETTY

8387
06:02:06,700 --> 06:02:08,569
GOOD AT COMING UP WITH TARGETS

8388
06:02:08,569 --> 06:02:10,471
BUT THEY'RE OFTEN BAD WITH ALL

8389
06:02:10,471 --> 06:02:11,439
OF THE REST OF IT.

8390
06:02:11,439 --> 06:02:13,741
WHAT DO YOU THINK THE -- WHAT

8391
06:02:13,741 --> 06:02:15,276
WOULD YOUR ADVICE BE TO SOMEBODY

8392
06:02:15,276 --> 06:02:16,677
WHO'S GOT SOMETHING THAT LOOKS

8393
06:02:16,677 --> 06:02:18,078
GOOD IN THEIR MOUSE OR WHATEVER

8394
06:02:18,078 --> 06:02:20,014
AND IS THINKING ABOUT TRYING TO

8395
06:02:20,014 --> 06:02:21,882
PUSH IT FURTHER?

8396
06:02:21,882 --> 06:02:25,920
AND IT'S FOR ANYBODY WHO THINKS

8397
06:02:25,920 --> 06:02:28,456
IT'S WORTH ANSWERING?

8398
06:02:28,456 --> 06:02:33,794
>> HIRE A GOOD CHEMIST.

8399
06:02:33,794 --> 06:02:34,695
>> [LAUGHTER]

8400
06:02:34,695 --> 06:02:38,966
>> THAT'S AN EXCELLENT
ANSWER,

8401
06:02:38,966 --> 06:02:39,233
ACTUALLY.

8402
06:02:39,233 --> 06:02:40,935
>> I THINK MAYBE ON THAT
FRONT,

8403
06:02:40,935 --> 06:02:42,803
IF THERE'S A GOOD TARGET

8404
06:02:42,803 --> 06:02:44,004
ESPECIALLY IF IT'S A NOVEL

8405
06:02:44,004 --> 06:02:47,708
TARGET THAT IS THE FIRST TIME

8406
06:02:47,708 --> 06:02:48,876
THAT ANYONE'S DISCOVERED THE

8407
06:02:48,876 --> 06:02:52,213
MECHANISM, A HUGE PART OF IT IS

8408
06:02:52,213 --> 06:02:54,281
REALLY SHOWING THAT IT CAN BE

8409
06:02:54,281 --> 06:02:55,583
REPRODUCED IN A DIFFERENT LAB,

8410
06:02:55,583 --> 06:02:58,419
THAT'S MAYBE A BIG AREA THAT

8411
06:02:58,419 --> 06:02:59,587
PREVENTS OTHERS FROM REALLY

8412
06:02:59,587 --> 06:03:03,457
INVESTING EARLY ON IN BIOLOGY IF

8413
06:03:03,457 --> 06:03:04,859
THE BIOLOGY'S ONLY EVER BEEN

8414
06:03:04,859 --> 06:03:07,194
DISCOVERED OR WORKED ON IN 1

8415
06:03:07,194 --> 06:03:07,628
LAB.

8416
06:03:07,628 --> 06:03:09,063
IT REALLY MAKES YOU OPEN UP THE

8417
06:03:09,063 --> 06:03:12,066
QUESTION OF JUST FROM INVESTORS,

8418
06:03:12,066 --> 06:03:14,702
YOU'RE NOT ON THE GROUND ALL THE

8419
06:03:14,702 --> 06:03:15,970
TIME TALKING WITH EVERYONE, IS

8420
06:03:15,970 --> 06:03:17,771
THAT THERE'S A VERY DIFFICULT

8421
06:03:17,771 --> 06:03:21,108
TECHNIQUE TO GET THERE, IS IT

8422
06:03:21,108 --> 06:03:23,077
THAT THE SCIENTIST IS LESS

8423
06:03:23,077 --> 06:03:24,245
LABRATIVE, THOSE ARE ALL THINGS

8424
06:03:24,245 --> 06:03:25,412
THAT PEOPLE CARE ABOUT QUITE A

8425
06:03:25,412 --> 06:03:34,321
LOT, JUST IN ADDITION TO THE

8426
06:03:34,321 --> 06:03:36,390
SCIENCE AND FROM THE PERSPECTIVE

8427
06:03:36,390 --> 06:03:38,025
OF HOW AND VENTURE CAPITAL YOU

8428
06:03:38,025 --> 06:03:39,593
RISK A TECHNOLOGY THAT SOMEBODY

8429
06:03:39,593 --> 06:03:41,195
COMES TO YOU WITH AND HOW DO YOU

8430
06:03:41,195 --> 06:03:42,830
LEVERAGE AND SORT OF BALANCE

8431
06:03:42,830 --> 06:03:43,864
THEIR VISION AGAINST THE REALITY

8432
06:03:43,864 --> 06:03:45,833
OF WHAT COULD BE ACHIEVED AND

8433
06:03:45,833 --> 06:03:47,668
HOW DO YOU ACTUALLY MAKE SURE

8434
06:03:47,668 --> 06:03:52,740
YOU'RE RIGHT AND IT'S NOT THEM

8435
06:03:52,740 --> 06:03:53,307
THAT'S RIGHT.

8436
06:03:53,307 --> 06:03:54,575
>> I WILL START, I THINK
FROM

8437
06:03:54,575 --> 06:03:56,010
OUR POINT OF VIEW, WE'RE VERY

8438
06:03:56,010 --> 06:03:58,779
LUCKY WE HAVE OUR LABS IN

8439
06:03:58,779 --> 06:03:59,947
BOSTON, WITH ABOUT 50 PEOPLE, SO

8440
06:03:59,947 --> 06:04:02,950
WE'RE ABLE TO REALLY BRING IN

8441
06:04:02,950 --> 06:04:04,318
TECHNOLOGIES, AND THAT'S NOT

8442
06:04:04,318 --> 06:04:06,287
ONLY BENEFICIAL FOR US, BUT IT'S

8443
06:04:06,287 --> 06:04:08,556
BENEFICIAL FOR THE COMPANY, TOO,

8444
06:04:08,556 --> 06:04:12,793
BECAUSE WE ACT AS EXTERNAL

8445
06:04:12,793 --> 06:04:13,494
VALIDATION CAN TECHNOLOGY, AND

8446
06:04:13,494 --> 06:04:15,696
AT THE SAME TIME THEY CAN SEE

8447
06:04:15,696 --> 06:04:16,964
EARLY ON, IS THE TECHNOLOGY

8448
06:04:16,964 --> 06:04:19,767
GOING TO WORK IN CYSTIC FIBROSIS

8449
06:04:19,767 --> 06:04:21,402
MAYBE IT WON'T FOR CF AND

8450
06:04:21,402 --> 06:04:23,470
PROVIDE THAT DATA EARLY ON IN

8451
06:04:23,470 --> 06:04:25,005
THE PROCESS, AND THEN FROM OUR

8452
06:04:25,005 --> 06:04:26,907
SIDE, WE CAN SAY, OKAY THIS

8453
06:04:26,907 --> 06:04:28,742
WORKS WELL, HERE, LET'S MOVE

8454
06:04:28,742 --> 06:04:30,244
FORWARD, AND WE CAN GET EXCITED

8455
06:04:30,244 --> 06:04:39,653
ABOUT IT, AND HELP THEM. 

8456
06:04:39,653 --> 06:04:50,097
NGREAT RESOURCE TO HAVE --

8457
06:04:55,002 --> 06:04:56,470
>> SO ON THAT FRONT IN THE

8458
06:04:56,470 --> 06:04:57,504
PROCESS OF DECIDING WHETHER OR

8459
06:04:57,504 --> 06:04:59,373
NOT WE WANT TO START THE COMPANY

8460
06:04:59,373 --> 06:05:01,308
OR TECHNOLOGY OR ASSET, WE

8461
06:05:01,308 --> 06:05:02,810
GENERALLY HAVE EXPERIMENTS THAT

8462
06:05:02,810 --> 06:05:05,713
WE WOULD LIKE ACADEMIC TO  

8463
06:05:05,713 --> 06:05:08,716
PERFORM, WE DESIGNED EXPERIMENTS

8464
06:05:08,716 --> 06:05:10,117
GENERALLY, THERE ARE 1S THAT FOR

8465
06:05:10,117 --> 06:05:13,554
IN THE ACADEMIC WORLD BE LIKE

8466
06:05:13,554 --> 06:05:15,589
SUPPLEMENTAL FIGURE, 25, BUT FOR

8467
06:05:15,589 --> 06:05:16,857
US VERY IMPORTANT TO VALIDATE

8468
06:05:16,857 --> 06:05:20,194
IT, AND SO IF ALL THAT KIND OF

8469
06:05:20,194 --> 06:05:21,528
COMES BACK POSITIVE, OR IN THE

8470
06:05:21,528 --> 06:05:24,431
DIRECTION OF WE THINK MAKES

8471
06:05:24,431 --> 06:05:26,133
SENSE THAT HELPS GREATLY

8472
06:05:26,133 --> 06:05:27,167
VALIDATE THE TECHNOLOGY THAT'S

8473
06:05:27,167 --> 06:05:28,268
WORKING IN A WAY THAT WE'RE

8474
06:05:28,268 --> 06:05:29,570
SEEING THINGS AND HOW WE'RE

8475
06:05:29,570 --> 06:05:32,039
DESIGNING IT AND YOU'RE GETTING

8476
06:05:32,039 --> 06:05:32,406
IT LIVE.

8477
06:05:32,406 --> 06:05:36,477
JUST TO HAVE THAT EXTRA

8478
06:05:36,477 --> 06:05:36,810
CONFIRMATION.

8479
06:05:36,810 --> 06:05:41,115
>> AND THEN I THINK TO ADD

8480
06:05:41,115 --> 06:05:42,716
BEYOND CFF, WE SEE FORESIGHT

8481
06:05:42,716 --> 06:05:44,551
LABS TO CALL THEM, THEY'RE

8482
06:05:44,551 --> 06:05:45,953
MOVING ALSO TOWARDS A MODEL

8483
06:05:45,953 --> 06:05:47,154
WHERE THEY'RE ALSO ABLE TO

8484
06:05:47,154 --> 06:05:48,122
VALIDATE SOME OF THE

8485
06:05:48,122 --> 06:05:49,823
TECHNOLOGIES AND THEN IF YOU

8486
06:05:49,823 --> 06:05:52,326
TALK TO STRATEGIC PLANEGEYICS,

8487
06:05:52,326 --> 06:05:53,927
FOR EXAMPLE, WHERE IT WAS

8488
06:05:53,927 --> 06:05:55,062
BEFORE, THERE WAS ALSO CAPACITY

8489
06:05:55,062 --> 06:05:57,998
TO BE ABLE TO VALIDATE

8490
06:05:57,998 --> 06:06:00,234
TECHNOLOGIES, OR TO CO DEVELOP

8491
06:06:00,234 --> 06:06:02,770
ONCE A TARGET IS FOUND BRINGING

8492
06:06:02,770 --> 06:06:04,238
IN THE CHEMIST FROM BIOTO KIND

8493
06:06:04,238 --> 06:06:07,007
OF WORK ON THE TARGET

8494
06:06:07,007 --> 06:06:09,209
SPECIFICALLY WHILE THE SAME TIME

8495
06:06:09,209 --> 06:06:11,378
ENGAGING THE PI WHO MIGHT OR

8496
06:06:11,378 --> 06:06:13,113
MIGHT NOT WANT TO BE SUPER

8497
06:06:13,113 --> 06:06:15,716
INVOLVED IN IT, BUT THERE'S

8498
06:06:15,716 --> 06:06:18,519
DIFFERENT MODELS OUT THERE THAT

8499
06:06:18,519 --> 06:06:19,920
IT'S WORTH EXPLORING.

8500
06:06:19,920 --> 06:06:21,955
>> SO THERE'S A QUESTION
IN THE

8501
06:06:21,955 --> 06:06:22,890
Q&A FROM CAITLIN

8502
06:06:22,890 --> 06:06:23,257
[INDISCERNIBLE].

8503
06:06:23,257 --> 06:06:26,527
WHAT ARE SIGNS THAT AN

8504
06:06:26,527 --> 06:06:33,734
INSTITUTION'S NOT SUPPORTING

8505
06:06:33,734 --> 06:06:34,068
 SUPPORTING

8506
06:06:34,068 --> 06:06:37,004
INNOVATION OR RED FLAGS FOR

8507
06:06:37,004 --> 06:06:38,172
BUILDING YOUR CAREER AND

8508
06:06:38,172 --> 06:06:38,772
RESEARCH TRAINING?

8509
06:06:38,772 --> 06:06:40,040
>> I THINK IF YOU LOOK AT

8510
06:06:40,040 --> 06:06:41,775
PROMOTION IN MOST PLACES, IT

8511
06:06:41,775 --> 06:06:43,544
USED TO IN SOME PLACES A THING

8512
06:06:43,544 --> 06:06:46,080
THAT YOU GOT KUDOS FOR, BUT

8513
06:06:46,080 --> 06:06:47,681
THESE DAYS IT'S JUST PAPERS AND

8514
06:06:47,681 --> 06:06:48,916
FUNDING, RIGHT IN SO IF YOU

8515
06:06:48,916 --> 06:06:52,786
WANTED TO GET TENURE SOMEWHERE,

8516
06:06:52,786 --> 06:06:53,821
YOU KNOW, INNOVATION USED TO

8517
06:06:53,821 --> 06:06:55,122
COUNT BUT MOST PLACES ARE

8518
06:06:55,122 --> 06:06:56,323
ACTUALLY GETTING RID OF IT,

8519
06:06:56,323 --> 06:06:58,025
BECAUSE IT'S SUCH A HIGH RISK

8520
06:06:58,025 --> 06:07:01,729
AND IT REALLY IS HARD TO DEFINE.

8521
06:07:01,729 --> 06:07:03,731
I THINK IT'S EASIER TO DEFINE IF

8522
06:07:03,731 --> 06:07:05,365
YOU BASE IT ON THE SUCCESS IF

8523
06:07:05,365 --> 06:07:06,900
YOU BRING IT ON THE MARKET LIKE

8524
06:07:06,900 --> 06:07:08,669
SOMEBODY LIKE JAMES FOR

8525
06:07:08,669 --> 06:07:09,737
INSTANCE, SO ACADEMIA, SO THAT'S

8526
06:07:09,737 --> 06:07:12,706
1 PLACE TO LOOK, SEE WHAT YOUR

8527
06:07:12,706 --> 06:07:14,108
PROMOTION AND TENURE COMMITTEE

8528
06:07:14,108 --> 06:07:15,242
VIEWS INNOVATION AS AND THAT

8529
06:07:15,242 --> 06:07:17,578
TELLS WHAT YOU THAT INSTITUTION

8530
06:07:17,578 --> 06:07:20,114
VALUES INNOVATION AT, IS MY

8531
06:07:20,114 --> 06:07:22,149
OPINION ABOUT.

8532
06:07:22,149 --> 06:07:22,950
>> YEAH, GO AHEAD.

8533
06:07:22,950 --> 06:07:24,118
>> ONE ANGLE HERE, THERE'S
A LOT

8534
06:07:24,118 --> 06:07:25,753
OF WAYS TO ANSWER THIS QUESTION,

8535
06:07:25,753 --> 06:07:27,755
1 ANGLE IS TO START WITH THE OTM

8536
06:07:27,755 --> 06:07:31,191
OFFICE, THE TECH TRANSFER

8537
06:07:31,191 --> 06:07:31,425
OFFICE.

8538
06:07:31,425 --> 06:07:33,660
AND YOU FIND SOME OF THE

8539
06:07:33,660 --> 06:07:37,064
PRESTIGIOUS COOLS HAVE EXTREMELY

8540
06:07:37,064 --> 06:07:38,031
OVERWORKED UNDERSTAFFED, UNDER

8541
06:07:38,031 --> 06:07:39,399
REWARDED GROUP THAT'S NOT HAPPY

8542
06:07:39,399 --> 06:07:49,309
TO TAKE ON MUCH OF ANYTHING SO

8543
06:07:49,309 --> 06:07:51,612
IF YOU INTERVIEW THE DIRECTOR OF

8544
06:07:51,612 --> 06:07:53,013
THE TECH TRANSFER OFFICE, YOU

8545
06:07:53,013 --> 06:07:54,481
COULD GET A LOT OF INSIGHT INTO

8546
06:07:54,481 --> 06:07:57,284
THE FLAVOR OF THE INSTITUTIONAL

8547
06:07:57,284 --> 06:07:57,584
PRIORITIES.

8548
06:07:57,584 --> 06:07:59,119
>> MOST OF THOSE WILL NOT
HELP

8549
06:07:59,119 --> 06:08:01,421
YOU WITH YOUR CAREER, AS FAR AS

8550
06:08:01,421 --> 06:08:04,291
GETTING TENURED AND GETTING

8551
06:08:04,291 --> 06:08:05,626
PROMOTED, THE TECH TRANSFER

8552
06:08:05,626 --> 06:08:07,327
FOLKS HAVE VERY LITTLE, AT LEAST

8553
06:08:07,327 --> 06:08:10,264
MOST THE PLACES I'VE BEEN, IT

8554
06:08:10,264 --> 06:08:12,933
MAY BE DIFFERENT DR. HOLTZMAN,

8555
06:08:12,933 --> 06:08:14,635
BUT THE INSTITUTIONS I'M AT THE

8556
06:08:14,635 --> 06:08:16,203
TECH TRANSFER PEOPLE ARE THERE

8557
06:08:16,203 --> 06:08:17,671
AND THEY'RE SUPPORTIVE.

8558
06:08:17,671 --> 06:08:18,639
THEY'RE USUALLY THE SILVER

8559
06:08:18,639 --> 06:08:20,240
LINING AT THESE PLACES, BUT MOST

8560
06:08:20,240 --> 06:08:21,575
OF THEM HAVE IMPACT ON WHETHER

8561
06:08:21,575 --> 06:08:23,177
YOU GET PROMOTED OR TENURE AND

8562
06:08:23,177 --> 06:08:25,813
THAT IS THE INSTITUTIONAL

8563
06:08:25,813 --> 06:08:26,413
SUPPORT OF THIS.

8564
06:08:26,413 --> 06:08:28,615
YOU KNOW AND I'VE SEEN A LOT OF

8565
06:08:28,615 --> 06:08:29,683
VERY SMART INNOVATIVE PEOPLE

8566
06:08:29,683 --> 06:08:31,418
DECIDE NOT TO INNOVATE BECAUSE

8567
06:08:31,418 --> 06:08:33,153
IT DISTRACTED THEM FROM GETTING

8568
06:08:33,153 --> 06:08:33,987
THE INCREMENTAL GRANTS AND

8569
06:08:33,987 --> 06:08:35,522
GETTING THE PAPERS AND MOVING ON

8570
06:08:35,522 --> 06:08:37,191
WITH THEIR CAREER.

8571
06:08:37,191 --> 06:08:37,591
YOU KNOW?

8572
06:08:37,591 --> 06:08:39,126
AND THATIA A PROBLEM, NOBODY

8573
06:08:39,126 --> 06:08:40,294
TALKS ABOUT IT, MAYBE IT'S NOT

8574
06:08:40,294 --> 06:08:41,595
AS BIG A PROBLEM AS I'VE SEEN

8575
06:08:41,595 --> 06:08:43,564
BUT IT IS A PROBLEM.

8576
06:08:43,564 --> 06:08:45,065
NYEAH, I CERTAINLY DON'T

8577
06:08:45,065 --> 06:08:46,066
DISAGREE,Y JUST WAS TRYING TO

8578
06:08:46,066 --> 06:08:48,702
MAKE THE POINT THAT IF IT'S AN

8579
06:08:48,702 --> 06:08:49,636
INSTITUTIONAL PRIORITY TELL BE

8580
06:08:49,636 --> 06:08:51,271
AS WELL QUEPED AS SOME OF WHAT

8581
06:08:51,271 --> 06:08:54,541
THE GUYS ARE CALLING INCREMENTAL

8582
06:08:54,541 --> 06:08:54,908
SUPPORT SYSTEM.

8583
06:08:54,908 --> 06:08:56,577
SO IF YOU WALK IN THERE AND

8584
06:08:56,577 --> 06:08:58,846
THERE'S ONLY 1 PERSON ON A

8585
06:08:58,846 --> 06:08:59,780
KEYBOARD, YOU PROBABLY NOT GOING

8586
06:08:59,780 --> 06:09:01,615
TO MAKE A LOT OF PROGRESS IN

8587
06:09:01,615 --> 06:09:04,551
THAT ENVIRONMENT, AND THE REASON

8588
06:09:04,551 --> 06:09:07,154
THAT'S THE CASE OFTEN IN THE

8589
06:09:07,154 --> 06:09:09,957
MIDWEST, I NOTICE, LIKE FROM

8590
06:09:09,957 --> 06:09:11,925
LUCY'S SLIDES THERE'S A LOT OF

8591
06:09:11,925 --> 06:09:13,293
COASTAL ACTIVITY AND NOT MUCH IN

8592
06:09:13,293 --> 06:09:14,795
THE MIDDLE WHEREAS A LOT OF

8593
06:09:14,795 --> 06:09:15,863
PEOPLE ON THIS CALL ACTUALLY ARE

8594
06:09:15,863 --> 06:09:17,397
IN THE MITSD BUT 1 OF THE

8595
06:09:17,397 --> 06:09:22,970
REASONS FOR THAT IS IT'S A VERY

8596
06:09:22,970 --> 06:09:23,637
UNDERDEVELOPED PATHWAY.

8597
06:09:23,637 --> 06:09:24,872
AND IT STARTS AT THE LEVEL

8598
06:09:24,872 --> 06:09:26,073
YOU'RE REFERRING TO IN THE

8599
06:09:26,073 --> 06:09:27,341
ACADEMIC SETTING AND IT MOVES

8600
06:09:27,341 --> 06:09:31,511
RIGHT ON THROUGH THE BIOCHECK

8601
06:09:31,511 --> 06:09:34,248
AND D. C. SECTOR AS WELL, IF

8602
06:09:34,248 --> 06:09:35,782
IT'S WELL DEVELOPED USUALLY THAT

8603
06:09:35,782 --> 06:09:37,284
OFFICE WILL RECOGNIZE IT AND

8604
06:09:37,284 --> 06:09:39,486
SHOULD BE SUPPORTED FOR IT BUT

8605
06:09:39,486 --> 06:09:44,157
IF IT'S NOT, YOU WILL HAVE AN

8606
06:09:44,157 --> 06:09:44,625
UPHILL BATTLE.

8607
06:09:44,625 --> 06:09:47,561
>> I WOULD ADD HERE THAT
IN THE

8608
06:09:47,561 --> 06:09:50,530
CAREER, AS SOMEBODY WHO

8609
06:09:50,530 --> 06:09:51,131
[INDISCERNIBLE] ACADEMIA AND

8610
06:09:51,131 --> 06:09:54,868
TART THE SUMP AND THAT --

8611
06:09:54,868 --> 06:09:56,103
COMPANIES AND WITH THAT IN MIND,

8612
06:09:56,103 --> 06:09:59,039
TALKING TO THE TECH TRANSFER

8613
06:09:59,039 --> 06:09:59,773
OFFICE HELPFUL AND THEN

8614
06:09:59,773 --> 06:10:01,074
UNDERSTAND THE PROCESS OF TAKING

8615
06:10:01,074 --> 06:10:03,010
AN IP OUT OF THE UNIVERSITY AND

8616
06:10:03,010 --> 06:10:05,112
GO TO A COMPANY, WHAT ARE THE

8617
06:10:05,112 --> 06:10:06,480
REQUIREMENTS FROM THE UNIVERSITY

8618
06:10:06,480 --> 06:10:07,714
SIDE WHERE SOME UNIVERSITIES

8619
06:10:07,714 --> 06:10:09,683
MIGHT BE ASKING FOR A LOT, AND

8620
06:10:09,683 --> 06:10:13,587
THAT MIGHT BE SCARY AND OTHERS

8621
06:10:13,587 --> 06:10:14,988
AWAY OR INVESTORS SO REALLY

8622
06:10:14,988 --> 06:10:17,424
TRYING TO UNDERSTAND WHAT THE

8623
06:10:17,424 --> 06:10:19,326
UNIVERSITY REQUIRES, IS IT BOARD

8624
06:10:19,326 --> 06:10:20,694
SEATS, IS IT SPECIFIC EQUITY AND

8625
06:10:20,694 --> 06:10:24,498
THEN TRY TO UNDERSTAND, OKAY,

8626
06:10:24,498 --> 06:10:27,501
ASK THAT MODEL THERE TO

8627
06:10:27,501 --> 06:10:28,635
SUCCESSFUL COMPANIES AS BEING

8628
06:10:28,635 --> 06:10:30,070
ABLE TO ATTRACT OTHER PARTNERS

8629
06:10:30,070 --> 06:10:32,239
AND INVESTORS AND TRYING TO

8630
06:10:32,239 --> 06:10:33,173
UNDERSTAND IF IT FITS WITH WHAT

8631
06:10:33,173 --> 06:10:37,277
YOU WANT TO DO WITH YOUR CAREER 

8632
06:10:37,277 --> 06:10:37,544
NGREAT.

8633
06:10:37,544 --> 06:10:38,912
AND THE OTHER THING TO THAT

8634
06:10:38,912 --> 06:10:40,647
COMMENT OR WHAT EVERY WE CALL

8635
06:10:40,647 --> 06:10:43,083
THEM THESE DAYS, LOOK FOR ROLE

8636
06:10:43,083 --> 06:10:45,285
MODELS, WHOSE DONE THIS AT THIS

8637
06:10:45,285 --> 06:10:47,854
PLACE AND WHAT'S THERE

8638
06:10:47,854 --> 06:10:49,022
PERSPECTIVE ON TRYING TO GET IT

8639
06:10:49,022 --> 06:10:50,524
DONE IN THAT ENVIRONMENT AND IF

8640
06:10:50,524 --> 06:10:52,492
YOU'RE NOT FINDING ANY, OR EVEN

8641
06:10:52,492 --> 06:10:54,895
IF THEY DO AND THEY EXPRESS

8642
06:10:54,895 --> 06:10:55,829
FRUSTRATION, ONCE AGAIN, YOU'RE

8643
06:10:55,829 --> 06:11:02,169
PROBABLY LOOKING AT A LOT OF

8644
06:11:02,169 --> 06:11:02,436
OBSTACLES.

8645
06:11:02,436 --> 06:11:04,571
>> I'VE ONLY 11 THING TO
ADD ON

8646
06:11:04,571 --> 06:11:06,840
THIS IS THAT EVEN THOUGH OUTSIDE

8647
06:11:06,840 --> 06:11:09,810
OF UNIVERSITY, I'M AT VANNEDDER

8648
06:11:09,810 --> 06:11:12,980
BILL, VANDERBILT MEDICAL COOL IS

8649
06:11:12,980 --> 06:11:15,415
#1 IN NIH FUNDING, SO WE ATTRACT

8650
06:11:15,415 --> 06:11:19,019
A LOT OF FUNDING, THE STUDENTS

8651
06:11:19,019 --> 06:11:21,621
ARE EXCITED ABOUT TECH TRANSFERS

8652
06:11:21,621 --> 06:11:23,924
BUT THERE'S NO INFRASTRUCTURE,

8653
06:11:23,924 --> 06:11:25,559
NO INCUBATORS, NO OUTSIDE MOUSE

8654
06:11:25,559 --> 06:11:26,860
FACILITIES, 1 OF THE LAST

8655
06:11:26,860 --> 06:11:28,028
SPIN-OFF COMPANY I DID I

8656
06:11:28,028 --> 06:11:30,564
LITERALLY HAD TO SET UP A LAB

8657
06:11:30,564 --> 06:11:32,332
UNDERNEATH A REALLY NICE BAR

8658
06:11:32,332 --> 06:11:36,536
DOWN TOWN, BUT YOU KNOW IT'S --

8659
06:11:36,536 --> 06:11:38,305
SOME PLACES IT'S HARD TO

8660
06:11:38,305 --> 06:11:39,339
SPIN-OFF COMPANIES BECAUSE

8661
06:11:39,339 --> 06:11:41,742
THERE'S NO PLACE TO DO IT TO.

8662
06:11:41,742 --> 06:11:42,642
>> RIGHT.

8663
06:11:42,642 --> 06:11:47,714
CREATING THAT KIND OF

8664
06:11:47,714 --> 06:11:48,949
ENVIRONMENT'S CHALLENGING.

8665
06:11:48,949 --> 06:11:50,650
WE HAVE THE SAME ISSUE IN

8666
06:11:50,650 --> 06:11:52,986
ST. LOUIS AND THEN WE HAVE LOTS

8667
06:11:52,986 --> 06:11:55,055
OF OTHER ISSUES THAT GO TO THE

8668
06:11:55,055 --> 06:11:56,823
MAYOR BUT HAVING THAT IS A

8669
06:11:56,823 --> 06:11:57,958
CHALLENGE FOR THE MIDWEST IN

8670
06:11:57,958 --> 06:11:58,258
GENERAL.

8671
06:11:58,258 --> 06:12:00,193
A LITTLE BIT IN CHICAGO, SIN

8672
06:12:00,193 --> 06:12:02,029
SUDDEN ATY CHILDRENS HAS DONE A

8673
06:12:02,029 --> 06:12:04,097
BETTER JOB I THINK THAN MOST

8674
06:12:04,097 --> 06:12:07,734
PLACES BUT THERE'S A LOT OF RISK

8675
06:12:07,734 --> 06:12:09,970
ADVERSITY IN WHAT WE'RE CALLING

8676
06:12:09,970 --> 06:12:10,871
TRANSLATIONAL RESEARCH AND

8677
06:12:10,871 --> 06:12:13,440
ST. LOUIS, 1 OF THE REASONS

8678
06:12:13,440 --> 06:12:15,042
WE'RE NOT GROWING AS A

8679
06:12:15,042 --> 06:12:16,510
METROPOLITAN AREA IS THAT

8680
06:12:16,510 --> 06:12:20,013
THERE'S A VERY RISK ADVERSE

8681
06:12:20,013 --> 06:12:21,548
APPROACH TO ALL KINDS OF

8682
06:12:21,548 --> 06:12:23,383
ACTIVITY ANDS THAT INCLUDES

8683
06:12:23,383 --> 06:12:23,717
MEDICINE.

8684
06:12:23,717 --> 06:12:25,152
>> IN GENERAL, YEAH, YEAH.

8685
06:12:25,152 --> 06:12:25,986
>> I WANTED TO --

8686
06:12:25,986 --> 06:12:28,455
>> I HAVE A QUESTION FOR

8687
06:12:28,455 --> 06:12:29,956
ENRIQUE, YOU MENTIONED THE

8688
06:12:29,956 --> 06:12:31,091
FOUNDATION HAS GUIDELINES OF

8689
06:12:31,091 --> 06:12:36,963
AISK  -- BASICALLY A PATH TO

8690
06:12:36,963 --> 06:12:39,399
COMMERCIALIZATION OR A PATH TO A

8691
06:12:39,399 --> 06:12:39,599
CURE.

8692
06:12:39,599 --> 06:12:40,333
THESE GUIDELINES ARE PUT

8693
06:12:40,333 --> 06:12:42,702
TOGETHER BY THE LAB, BY PFIZERS

8694
06:12:42,702 --> 06:12:44,871
ACADEMIC AND THE ACADEMIC

8695
06:12:44,871 --> 06:12:45,806
COMMUNITY, IT'S BASICALLY SORT

8696
06:12:45,806 --> 06:12:49,876
OF A WHITE PAPER ON WHAT THE

8697
06:12:49,876 --> 06:12:51,511
FOUNDATION THINKS, HOW MUCH

8698
06:12:51,511 --> 06:12:53,447
FRICTION DO YOU SEE BETWEEN

8699
06:12:53,447 --> 06:12:56,283
THESE GUIDELINES AND SORT OF

8700
06:12:56,283 --> 06:12:57,818
INDIVIDUAL VISIONS OF THERAPIES

8701
06:12:57,818 --> 06:12:59,886
FOR CF THAT YOU ENCOUNTERED.

8702
06:12:59,886 --> 06:13:02,656
YOU SEE A LOT OF FRICTION OR DO

8703
06:13:02,656 --> 06:13:05,025
PEOPLE USUALLY JUST GO ALONG

8704
06:13:05,025 --> 06:13:05,759
WITH ALL THE LITTLE CHECK

8705
06:13:05,759 --> 06:13:07,427
CONTROLLER OF BANKS THAT YOU

8706
06:13:07,427 --> 06:13:07,761
HAVE?

8707
06:13:07,761 --> 06:13:09,296
>> YEAH, I WOULD SAY WE
HAVEN'T

8708
06:13:09,296 --> 06:13:10,664
SEEN TOO MUCH FRICTION.

8709
06:13:10,664 --> 06:13:12,766
THE GUIDANCE THAT WE PRESENTED

8710
06:13:12,766 --> 06:13:19,539
TODAY ARE REALLY FOCUSED ON

8711
06:13:19,539 --> 06:13:20,440
PRECLINICAL GENETIC MEDICINES

8712
06:13:20,440 --> 06:13:21,541
AND VERY EARLY STAGE COMPANIES

8713
06:13:21,541 --> 06:13:23,243
AND WE TRY TO ENGAGE COMPANIES

8714
06:13:23,243 --> 06:13:25,245
AT EARLY STAGE SO WE IMPLEMENT

8715
06:13:25,245 --> 06:13:27,280
THOSE GUIDANCE EARLY ON AND HELP

8716
06:13:27,280 --> 06:13:29,182
COMPANIES GET ON THAT CHECK LIST

8717
06:13:29,182 --> 06:13:29,716
FROM THE BEGINNING.

8718
06:13:29,716 --> 06:13:33,153
SO WHEN THEY COME TO US, WE HAVE

8719
06:13:33,153 --> 06:13:36,022
SPECIFIC CF DATA WE ARE ALIGNED

8720
06:13:36,022 --> 06:13:38,158
IN TERMS OF LOOK FOR EXAMPLE THE

8721
06:13:38,158 --> 06:13:39,025
SAME THINGS.

8722
06:13:39,025 --> 06:13:40,193
ARE YOU DEMONSTRATING IN THE

8723
06:13:40,193 --> 06:13:41,161
GOLD STANDARD EXPERIMENTS THAT

8724
06:13:41,161 --> 06:13:43,363
YOUR APPROACH IS WORKING IN

8725
06:13:43,363 --> 06:13:45,132
CYSTIC FIBROSIS, SO I WOULD SAY

8726
06:13:45,132 --> 06:13:47,501
WE HAVE REALLY ENCOUNTERED TOO

8727
06:13:47,501 --> 06:13:52,539
MUCH PUSH BACK AROUND THOSE

8728
06:13:52,539 --> 06:13:53,573
GUIDE LINES OR PARALLEL

8729
06:13:53,573 --> 06:13:55,442
APPROACHES TO GETTING TO THE

8730
06:13:55,442 --> 06:13:55,809
SAME RESULT.

8731
06:13:55,809 --> 06:13:57,577
I THINK THE CF COMMITTEE QUITE

8732
06:13:57,577 --> 06:14:02,415
UNIQUE IN TERMS OF HOW THEIR

8733
06:14:02,415 --> 06:14:04,017
RESOURCES AND HOW, THE

8734
06:14:04,017 --> 06:14:05,986
COMMITTEE'S QUITE CLOSE AND THEN

8735
06:14:05,986 --> 06:14:07,154
HOW THESE RESOURCES ARE OPEN AND

8736
06:14:07,154 --> 06:14:09,823
PEOPLE ARE ABLE TO ACCESS AND

8737
06:14:09,823 --> 06:14:16,963
UNDERSTAND WHAT'S NEEDED TO MOVE

8738
06:14:16,963 --> 06:14:20,934
THERAPY FORWARD. 

8739
06:14:20,934 --> 06:14:22,135
>> WAS THAT QUESTION
DIRECTED AT

8740
06:14:22,135 --> 06:14:25,071
ALL TO THE COMMON STREET NOTICES

8741
06:14:25,071 --> 06:14:26,139
THAT THE CFF LIKES TO TAKE A

8742
06:14:26,139 --> 06:14:30,610
PIECE OF THE PROFITS IN THE

8743
06:14:30,610 --> 06:14:30,844
PROCESS?

8744
06:14:30,844 --> 06:14:32,145
>> I MEAN I THINK IF THEY

8745
06:14:32,145 --> 06:14:33,747
INVEST, THEY HAVE A RIGHT TO ASK

8746
06:14:33,747 --> 06:14:34,481
FOR THAT.

8747
06:14:34,481 --> 06:14:35,882
NTHAT'S WHAT I'M ASKING.

8748
06:14:35,882 --> 06:14:37,517
>> I THINK HA THEY GET
DINGED

8749
06:14:37,517 --> 06:14:42,455
UNFAIRLY I THINK FOR WHAT HAPPEN

8750
06:14:42,455 --> 06:14:43,290
WIDE VERTEX, WHEN YOU THINK

8751
06:14:43,290 --> 06:14:44,925
ABOUT IT THEY WOULD HAVE NEVER

8752
06:14:44,925 --> 06:14:46,660
GONE DOWN THE TRACK WITHOUT THE

8753
06:14:46,660 --> 06:14:47,294
FOUNDATION A INVESTMENT SO

8754
06:14:47,294 --> 06:14:48,895
WHETHER YOU WANT TO TALK ABOUT

8755
06:14:48,895 --> 06:14:51,498
GREED OR WHATEVER, YOU KNOW IT

8756
06:14:51,498 --> 06:14:53,099
WOULDN'T HAVE HAPPENED WITHOUT

8757
06:14:53,099 --> 06:14:56,002
BOB BELL INVESTING IN VERTEX,

8758
06:14:56,002 --> 06:14:57,504
AND NOW, CF PATIENTS HAVE THIS

8759
06:14:57,504 --> 06:14:57,704
DRUG.

8760
06:14:57,704 --> 06:14:59,606
SO IT'S 1 OF THESE KINDS OF

8761
06:14:59,606 --> 06:15:01,441
THINGS WHERE REALLY CAN YOU PUT

8762
06:15:01,441 --> 06:15:03,777
1 SIDE TO BALANCE, YOU KNOW?

8763
06:15:03,777 --> 06:15:05,312
MAYBE THERE SHOULD BE MORE

8764
06:15:05,312 --> 06:15:07,948
ADVOCACY ON MAKING THE DRUGS

8765
06:15:07,948 --> 06:15:09,349
CHEAPER, MAYBE I'LL --

8766
06:15:09,349 --> 06:15:09,716
[LAUGHTER]

8767
06:15:09,716 --> 06:15:12,786
>> I DO SAY WE -- NDON'T
GET MAD

8768
06:15:12,786 --> 06:15:15,522
AT ME NWE DO HAVE VERY

8769
06:15:15,522 --> 06:15:16,456
[INDISCERNIBLE] AS WELL, WE DO

8770
06:15:16,456 --> 06:15:20,327
NOT HAVE THE ABILITY TO CONVINCE

8771
06:15:20,327 --> 06:15:22,128
A PHARMACEUTICAL COMPANY OR TO

8772
06:15:22,128 --> 06:15:24,164
DISCUSS PRICING WITH

8773
06:15:24,164 --> 06:15:24,664
PHARMACEUTICAL COMPANY.

8774
06:15:24,664 --> 06:15:26,833
I WOULD SAY VENTURE PHILANTHROPY

8775
06:15:26,833 --> 06:15:28,034
WHICH YOU HAVE MENTIONED IS

8776
06:15:28,034 --> 06:15:29,269
REALLY KIND OF SOMETHING THAT

8777
06:15:29,269 --> 06:15:31,838
WAS PIE OR NEARED BY THE

8778
06:15:31,838 --> 06:15:34,708
FOUNDATION -- PIONEERED BY THE

8779
06:15:34,708 --> 06:15:36,576
FOUND FOUNDATION AND IT'S

8780
06:15:36,576 --> 06:15:38,678
ALLOWED US TO TACKLE A LOT OF

8781
06:15:38,678 --> 06:15:40,780
THE DIFFERENT CHALLENGES WHEN IT

8782
06:15:40,780 --> 06:15:42,515
COMES TO CYSTIC FIBROSIS AND

8783
06:15:42,515 --> 06:15:46,286
THEN WE'RE REALLY TRYING TO --

8784
06:15:46,286 --> 06:15:48,722
OR I'VE SEEN A LOT OF

8785
06:15:48,722 --> 06:15:49,789
FOUNDATIONS TAKE THIS APPROACH

8786
06:15:49,789 --> 06:15:51,091
AS WELL, BECAUSE ALL FOUNDATIONS

8787
06:15:51,091 --> 06:15:52,626
ARE LOOKING FOR SUSTAINABLE WAYS

8788
06:15:52,626 --> 06:15:55,428
TO CONTINUE MOVING TOWARDS THEIR

8789
06:15:55,428 --> 06:15:55,662
MISSION.

8790
06:15:55,662 --> 06:15:57,964
AS YOU KNOW GETTING TO CURE

8791
06:15:57,964 --> 06:15:59,132
CYSTIC FIBROSIS OR ANY GENETIC

8792
06:15:59,132 --> 06:16:00,867
DEC IS NOT CHEAP OR EASY AND

8793
06:16:00,867 --> 06:16:02,702
IT'S GOING TO TAKE TIME, SO

8794
06:16:02,702 --> 06:16:08,541
THAT'S 1 WAY WE'RE TRYING TO

8795
06:16:08,541 --> 06:16:10,110
ACHIEVE HERE.

8796
06:16:10,110 --> 06:16:12,078
>> OKAY, DO YOU FEEL -- I
WILL

8797
06:16:12,078 --> 06:16:13,480
PUSH THE BUTTONS HERE SINCE

8798
06:16:13,480 --> 06:16:14,281
NOBODY ELSE WANTS TO.

8799
06:16:14,281 --> 06:16:17,884
SO IT'S THE ISSUE OF THE MISSION

8800
06:16:17,884 --> 06:16:18,118
ENRIQUE.

8801
06:16:18,118 --> 06:16:23,456
SO WHEN I WAS A CHILD, YOU KNOW,

8802
06:16:23,456 --> 06:16:24,491
CHARITABLE FOUNDATIONS SUPPORTED

8803
06:16:24,491 --> 06:16:25,392
RESEARCH FOR RESEARCH SAKE AND

8804
06:16:25,392 --> 06:16:26,826
THEY DIDN'T CAN A LOT OF

8805
06:16:26,826 --> 06:16:31,698
QUESTIONS ABOUT THE IP ON A

8806
06:16:31,698 --> 06:16:32,932
MONTHLY BASIS EVEN.

8807
06:16:32,932 --> 06:16:34,768
SO IT DOES CHANGE THE

8808
06:16:34,768 --> 06:16:36,503
TRAJECTORY, THERE'S NOTHING

8809
06:16:36,503 --> 06:16:39,105
WRONG WITH BEHAVING LIKE A VC IN

8810
06:16:39,105 --> 06:16:41,308
SOME SENSE BUT IT DOES -- I

8811
06:16:41,308 --> 06:16:45,178
THINK TO SAY, IT'S ALL FOR 1 AND

8812
06:16:45,178 --> 06:16:47,881
1 FOR ALL KIND OF THING IF THE

8813
06:16:47,881 --> 06:16:50,817
PATIENT BENEFITS IT'S NOT QUITE

8814
06:16:50,817 --> 06:16:53,787
THE FULL STORY ON HOW THINGS

8815
06:16:53,787 --> 06:16:56,122
HAVE SHIFTED AND I -- SOME OF

8816
06:16:56,122 --> 06:16:57,390
THE DPRAITEST DISCOVERIES, OF

8817
06:16:57,390 --> 06:16:58,658
MEDICINES AND THERE AREN'T MANY,

8818
06:16:58,658 --> 06:17:00,160
RIGHT, CAME OUT OF VERY BASIC

8819
06:17:00,160 --> 06:17:03,563
RESEARCH, THAT NOBODY WOULD HAVE

8820
06:17:03,563 --> 06:17:06,266
INVESTED FOR COMMERCIAL ELEMENT,

8821
06:17:06,266 --> 06:17:09,602
SO SOMETIMES THOSE ENTERPRISES

8822
06:17:09,602 --> 06:17:11,204
WILL SUFFER BECAUSE THEY DON'T

8823
06:17:11,204 --> 06:17:14,374
PORTRAY A DIRECT ROUTE TO THE

8824
06:17:14,374 --> 06:17:15,508
MARKETPLACE AND THAT'S GOING TO

8825
06:17:15,508 --> 06:17:17,977
HAPPEN MORE OFTEN THAN NOT.

8826
06:17:17,977 --> 06:17:20,113
>> I AGREE AND I THINK
WE'RE ON

8827
06:17:20,113 --> 06:17:22,816
THE SAME PAGE, THE CF FOUNDATION

8828
06:17:22,816 --> 06:17:27,587
ALSO FUNDS ACADEMICS AND THAT IS

8829
06:17:27,587 --> 06:17:31,524
COMPLETELY NO STRINGS  ATTACHED

8830
06:17:31,524 --> 06:17:33,460
AND WE FUND ALMOST THE SAME

8831
06:17:33,460 --> 06:17:35,362
AMOUNT IN FOUNDATIONS AND

8832
06:17:35,362 --> 06:17:37,297
RESEARCH IN THE CF COMMUNITY

8833
06:17:37,297 --> 06:17:40,200
THAT ARE VERY BASIC WITHOUT

8834
06:17:40,200 --> 06:17:40,767
EXPECTING ANY RETURNS.

8835
06:17:40,767 --> 06:17:43,870
>> WE GOT A BIT OFF TOPIC
ON

8836
06:17:43,870 --> 06:17:45,405
WHAT THE PANEL IS SUPPOSED TO BE

8837
06:17:45,405 --> 06:17:47,507
ON, NOT THAT I DON'T THINK

8838
06:17:47,507 --> 06:17:51,344
VENTURE FILL AN THOPY IS NOT

8839
06:17:51,344 --> 06:17:52,579
WONDERFUL BUT -- [LAUGHTER]

8840
06:17:52,579 --> 06:17:54,247
>> LET ME COME FROM THE
FEDERAL

8841
06:17:54,247 --> 06:17:55,515
GOVERNMENT PERSPECTIVE AND THE

8842
06:17:55,515 --> 06:17:56,349
PARTNERSHIP WE'VE BEEN ABLE TO

8843
06:17:56,349 --> 06:18:00,120
HAVE WITH THE FOUNDATION, YOU

8844
06:18:00,120 --> 06:18:01,388
KNOW THE INVESTMENT DOES HAVE

8845
06:18:01,388 --> 06:18:03,256
RETURN BUT THEN IT CONTINUES TO

8846
06:18:03,256 --> 06:18:06,359
BE REINVESTED INTO THE COMMUNITY

8847
06:18:06,359 --> 06:18:08,228
AS ENRIQUE HAS SAID, SO WE

8848
06:18:08,228 --> 06:18:10,764
WOULDN'T HAVE NEW PROGRAMMING

8849
06:18:10,764 --> 06:18:13,800
LIKE PATH TO THE CURE WHICH IS

8850
06:18:13,800 --> 06:18:15,435
HEAVILY INVESTING IN THERAPIES

8851
06:18:15,435 --> 06:18:17,637
OR GENE THERAPIES THAT ARE LEFT

8852
06:18:17,637 --> 06:18:18,938
BEHIND FROM VERTEX, AND THEY

8853
06:18:18,938 --> 06:18:20,673
HELPED TACKLE THE SITUATION OF

8854
06:18:20,673 --> 06:18:22,542
GETTING A DISEASE MODIFYING

8855
06:18:22,542 --> 06:18:24,978
THERAPY TO MARKET, BY SUPPORTING

8856
06:18:24,978 --> 06:18:27,347
VERTEX, CONTINUE TO INVEST INTO

8857
06:18:27,347 --> 06:18:28,748
BASIC SCIENCE AND THEN IN TURN,

8858
06:18:28,748 --> 06:18:33,186
CREATE HIGH RISK, HIGH REWARD

8859
06:18:33,186 --> 06:18:35,388
PROGRAMS THAT THE NIH CERTAINLY

8860
06:18:35,388 --> 06:18:38,525
CAN'T NECESSARILY DO AT THE SAME

8861
06:18:38,525 --> 06:18:41,694
SCALE BECAUSE WE DON'T HAVE THAT

8862
06:18:41,694 --> 06:18:42,061
CAPITAL.

8863
06:18:42,061 --> 06:18:43,563
WE DO HAVE PROGRAMS THAT WE DO

8864
06:18:43,563 --> 06:18:45,799
TRY TO DO IN PARALLEL AND IN

8865
06:18:45,799 --> 06:18:48,234
PARTNERSHIP WITH THE FOUNDATION,

8866
06:18:48,234 --> 06:18:50,503
AND YOU KNOW TRY TO HAVE THOSE

8867
06:18:50,503 --> 06:18:53,740
COLLABORATIVE EFFORTS TO MAKE

8868
06:18:53,740 --> 06:18:55,475
SURE THAT YOU KNOW THE SPACE IS

8869
06:18:55,475 --> 06:18:59,679
WELL FUNDED AND SO I THINK THAT

8870
06:18:59,679 --> 06:19:01,314
FROM A GOVERNMENT PERSPECTIVE,

8871
06:19:01,314 --> 06:19:03,116
LIKE WE COULDN'T REALLY HAVE THE

8872
06:19:03,116 --> 06:19:04,217
TYPE OF SCIENTIFIC PORTFOLIO

8873
06:19:04,217 --> 06:19:08,588
THAT WE DO THAT IS SO SUCCESSFUL

8874
06:19:08,588 --> 06:19:11,891
IN CF, WITHOUT THAT TYPE OF

8875
06:19:11,891 --> 06:19:13,860
STRATEGY, AND WHAT WOULD BE

8876
06:19:13,860 --> 06:19:14,227
BEAUTIFUL --

8877
06:19:14,227 --> 06:19:15,929
>> [INDISCERNIBLE]
MENTIONED

8878
06:19:15,929 --> 06:19:16,863
THAT AS WELL.

8879
06:19:16,863 --> 06:19:18,131
>> JOHN, YEAH, YEAH PUT
THAT IN

8880
06:19:18,131 --> 06:19:19,432
THE Q&A AS WELL.

8881
06:19:19,432 --> 06:19:21,000
WHAT'S BEAUTIFUL ABOUT THAT IS

8882
06:19:21,000 --> 06:19:23,102
IT'S A MODEL I THINK WE SHOULD

8883
06:19:23,102 --> 06:19:23,470
LEARN FROM.

8884
06:19:23,470 --> 06:19:27,607
I THINK THAT OTHER ORGANIZATIONS

8885
06:19:27,607 --> 06:19:28,942
ARE ALREADY COMING UP ON THAT

8886
06:19:28,942 --> 06:19:31,411
AND WE WILL HEAR FROM SOME OF

8887
06:19:31,411 --> 06:19:32,278
THEM TOMORROW ABOUT SHARED

8888
06:19:32,278 --> 06:19:33,680
LEARNING FROM THE FOUNDATION AND

8889
06:19:33,680 --> 06:19:35,181
HOW THEY'RE ABLE TO CATALYZE

8890
06:19:35,181 --> 06:19:37,016
RESEARCH AND INDUSTRY

8891
06:19:37,016 --> 06:19:39,052
PARTNERSHIPS, IN SIMILAR WAYS,

8892
06:19:39,052 --> 06:19:40,920
YOU KNOW BUILDING UP THEIR

8893
06:19:40,920 --> 06:19:43,523
PROFILE, SO, YOU KNOW, THE KEY,

8894
06:19:43,523 --> 06:19:45,859
I THINK IS THE VENTURE

8895
06:19:45,859 --> 06:19:47,794
PHILANTHROPY PIECE OF IT IS THAT

8896
06:19:47,794 --> 06:19:49,929
WHATEVER COMES IN, ALSO GOES

8897
06:19:49,929 --> 06:19:51,097
BACK OUT.

8898
06:19:51,097 --> 06:19:55,201
BUT, YEAH, I JUST WANTED TO ADD

8899
06:19:55,201 --> 06:19:56,569
THAT, AS VIEWING IT FROM THE

8900
06:19:56,569 --> 06:20:00,707
LENS OF WHERE WE ARE ON THE SIDE

8901
06:20:00,707 --> 06:20:01,875
OF HAVING PARTNERSHIPS WITH THE

8902
06:20:01,875 --> 06:20:03,042
FOUNDATION BUT I CAME INTO THE

8903
06:20:03,042 --> 06:20:05,111
PANEL BECAUSE AS A CO-HOST I

8904
06:20:05,111 --> 06:20:06,312
CAN'T ASK A QUESTION AND WANTED

8905
06:20:06,312 --> 06:20:08,181
TO ASK THE QUESTION, --I REALLY

8906
06:20:08,181 --> 06:20:14,220
WANTED TO FOLLOW UP ON THE PEER

8907
06:20:14,220 --> 06:20:21,261
TO PEER M-TEBURKEULOSEIS

8908
06:20:21,261 --> 06:20:23,596
 -- MENTORING AND CONTACTS, WE

8909
06:20:23,596 --> 06:20:25,331
TRY TO GET THE PEER TO PEER

8910
06:20:25,331 --> 06:20:27,634
PROGRAM, AND THEY ARE AT THE

8911
06:20:27,634 --> 06:20:28,568
COMMITTEE MEETINGS AND THEY THEY

8912
06:20:28,568 --> 06:20:31,070
MEET AND THEY WORK TOGETHER WHEN

8913
06:20:31,070 --> 06:20:33,640
THEY CAN AND THEY SORT OF CROSS

8914
06:20:33,640 --> 06:20:35,308
TALK WHEN THEY MAKE SENSE, BUT I

8915
06:20:35,308 --> 06:20:38,344
WONDERED IF YOU ALL HAD OTHER

8916
06:20:38,344 --> 06:20:39,546
IDEAS OF HOW PEOPLE CAN BETTER

8917
06:20:39,546 --> 06:20:40,847
LEARN FROM EACH OTHER, THAT'S

8918
06:20:40,847 --> 06:20:42,348
BASICALLY PART OF THE PLATFORM,

8919
06:20:42,348 --> 06:20:43,983
WE'RE TRYING WITH THIS WORKSHOP,

8920
06:20:43,983 --> 06:20:47,854
IS TO BRING IN EXPERTS LIKE YOU

8921
06:20:47,854 --> 06:20:50,356
ALL, BUT YOU KNOW IT'S SO

8922
06:20:50,356 --> 06:20:53,293
EXPENSIVE AND SO HARD TO DEVELOP

8923
06:20:53,293 --> 06:20:55,028
THESE NOVEL TECHNOLOGIES AND

8924
06:20:55,028 --> 06:20:57,230
INTERVENTIONS AND DRUGS OR GENE

8925
06:20:57,230 --> 06:21:00,600
THERAPY, WHATEVER THEY MAY BE,

8926
06:21:00,600 --> 06:21:04,003
HOW CAN WE FIND BETTER WAYS TO

8927
06:21:04,003 --> 06:21:05,505
GET PEOPLE TO PEER MENTOR EACH

8928
06:21:05,505 --> 06:21:08,641
OTHER THROUGH THE PROCESS, AND I

8929
06:21:08,641 --> 06:21:10,476
ASK THAT BECAUSE WE'RE ACTUALLY

8930
06:21:10,476 --> 06:21:12,645
EVEN TRYING THAT HERE AT THE

8931
06:21:12,645 --> 06:21:16,182
NIH, SO THE SOMATIC CELL GENOME

8932
06:21:16,182 --> 06:21:17,951
EDITING PROGRAM IS IN PHASE 2,

8933
06:21:17,951 --> 06:21:19,352
IT'S FUNDED BY THE COMMON FUND

8934
06:21:19,352 --> 06:21:22,355
AND WE ARE HAVING A GROUP OF

8935
06:21:22,355 --> 06:21:23,723
INVESTIGATORS WORKING TOGETHER

8936
06:21:23,723 --> 06:21:26,693
ACROSS DISEASES, TO LEARN FROM

8937
06:21:26,693 --> 06:21:28,761
INTERACTIONS WITH THE FDA IN

8938
06:21:28,761 --> 06:21:32,532
ORDER TO STREAMLINE GENE THERAPY

8939
06:21:32,532 --> 06:21:34,801
APPROVALS THROUGH THE FDA.

8940
06:21:34,801 --> 06:21:37,303
AND SO, WE'RE PILOTING THAT BUT

8941
06:21:37,303 --> 06:21:40,473
THAT'S ONLY 1 PROGRAM AT THE

8942
06:21:40,473 --> 06:21:43,610
NIH, AND I'M JUST THINKING IF

8943
06:21:43,610 --> 06:21:44,844
THERE ARE STRATEGIES YOU CAN

8944
06:21:44,844 --> 06:21:46,045
THINK OF HOW THE COMMUNITY CAN

8945
06:21:46,045 --> 06:21:47,880
BE BETTER AT PEER TO PEER

8946
06:21:47,880 --> 06:21:52,385
MENTORING TO SORT OF HELP

8947
06:21:52,385 --> 06:21:53,987
CATALYZE INNOVATION AND EVEN

8948
06:21:53,987 --> 06:21:55,388
PREVENT KIND OF COMPETITION AND

8949
06:21:55,388 --> 06:21:57,256
REALLY HAVE THIS SHARED GOAL FOR

8950
06:21:57,256 --> 06:21:59,325
ESPECIALLY FOR RARE DISEASES.

8951
06:21:59,325 --> 06:22:03,262
>> YOU CAN HAVE -- YOU CAN
HAVE

8952
06:22:03,262 --> 06:22:05,431
THE GRANTS MAYBE REQUIRE OR YOU

8953
06:22:05,431 --> 06:22:07,367
KNOW SUGGIEST OR ADVISE FOR

8954
06:22:07,367 --> 06:22:08,701
CONSULTANT ON THERE FOR INSTANCE

8955
06:22:08,701 --> 06:22:10,970
IS SOMEBODY THAT'S SEASONED SO

8956
06:22:10,970 --> 06:22:12,939
THAT IF SOMEBODY'S TRYING THIS

8957
06:22:12,939 --> 06:22:17,744
FOR THE FIRST TIME THEY MAY FIND

8958
06:22:17,744 --> 06:22:20,346
DR. HOLTZMAN AND DR. WEST TO

8959
06:22:20,346 --> 06:22:21,748
PARTNER WITH THEM EVEN IF IT'S

8960
06:22:21,748 --> 06:22:24,150
NOT THE SAME TECHNOLOGY.

8961
06:22:24,150 --> 06:22:25,652
SPEAKING OF THE FOUNDATION BEING

8962
06:22:25,652 --> 06:22:26,819
AN EXAMPLE, THAT'S SOMETHING

8963
06:22:26,819 --> 06:22:29,656
THIS WHEN THEY COME IN TO DO

8964
06:22:29,656 --> 06:22:31,924
RESEARCH, IF YOU HAVENER DONE

8965
06:22:31,924 --> 06:22:33,192
RESEARCH BEFORE, THEY LIKE TO

8966
06:22:33,192 --> 06:22:35,261
HAVE SOMEBODY WITH RESEARCH AS A

8967
06:22:35,261 --> 06:22:36,229
COLLABORATOR BUT THEY'RE OPEN TO

8968
06:22:36,229 --> 06:22:38,097
YOU COMING INTO THE CF FIELD AS

8969
06:22:38,097 --> 06:22:40,700
LONG AS DO YOU THAT SO IT'S THE

8970
06:22:40,700 --> 06:22:42,235
WAY THEY PROMOTE PEOPLE LEARNING

8971
06:22:42,235 --> 06:22:45,705
ABOUT CF.

8972
06:22:45,705 --> 06:22:48,007
>> CAN I COMMENT ON
SOMETHING,

8973
06:22:48,007 --> 06:22:51,978
THIS IS JOHN ENG LEHEART, I WILL

8974
06:22:51,978 --> 06:22:59,652
MAKE 1 COMMENT ABOUT THE STORY

8975
06:22:59,652 --> 06:23:01,521
LUCY TOLD ABOUT COLLABORATION,

8976
06:23:01,521 --> 06:23:02,822
THEY WERE TALKING ABOUT THE GENE

8977
06:23:02,822 --> 06:23:04,524
THERAPY SPACE AND I TRIED TO

8978
06:23:04,524 --> 06:23:06,726
CONVINCE BIG PHARMA TO GET

8979
06:23:06,726 --> 06:23:07,927
INVOLVED IN THAT, AND WHEN

8980
06:23:07,927 --> 06:23:09,429
PFIZER DID TAKE IT UP FOR A

8981
06:23:09,429 --> 06:23:10,596
WHILE BUT DIDN'T WANT TO

8982
06:23:10,596 --> 06:23:13,132
CONTINUE WITH IT AND YOU KNOW I

8983
06:23:13,132 --> 06:23:15,535
WOULD SAY SO FROM MY

8984
06:23:15,535 --> 06:23:16,335
PERSPECTIVE, I COULDN'T QUIT MY

8985
06:23:16,335 --> 06:23:19,639
DAY JOB TO MAKE THE COMPANY, AND

8986
06:23:19,639 --> 06:23:23,142
THOSE SORT OF INFORMA GET

8987
06:23:23,142 --> 06:23:24,877
TOGETHERS ARE GATHERING BETWEEN

8988
06:23:24,877 --> 06:23:26,913
VC OR INVESTORS IN THAT SPACE

8989
06:23:26,913 --> 06:23:28,081
THAT ARE SCIENTISTS AND WANT TO

8990
06:23:28,081 --> 06:23:29,582
HEAR ABOUT THE LATEST

8991
06:23:29,582 --> 06:23:31,984
TECHNOLOGY, AT ANY SORT OF

8992
06:23:31,984 --> 06:23:33,286
CONFERENCE, WOULD BE -- MIGHT

8993
06:23:33,286 --> 06:23:35,121
REPLICATE THAT TYPE OF THING

8994
06:23:35,121 --> 06:23:36,289
BECAUSE I NEVER WOULD HAVE

8995
06:23:36,289 --> 06:23:43,830
PREDICTED THAT THAT WOULD HAVE

8996
06:23:43,830 --> 06:23:46,466
BEEN THE OUTCOME.

8997
06:23:46,466 --> 06:23:47,467
>> NIH INNOVATORS
CONFERENCE.

8998
06:23:47,467 --> 06:23:48,234
>> YEAH, I WAS GOING TO
SAY, I

8999
06:23:48,234 --> 06:23:49,769
HAPPEN TO BE ON THE KEY STONE

9000
06:23:49,769 --> 06:23:53,873
BOARD SO I PASSED ALL OF THIS

9001
06:23:53,873 --> 06:23:56,242
INFORMATION ON TO THEM AS AN

9002
06:23:56,242 --> 06:23:58,344
IDEA FOR A KEY STONE SYMPOSIA

9003
06:23:58,344 --> 06:24:00,513
FOR NO OTHER BASIS BECAUSE WE

9004
06:24:00,513 --> 06:24:02,582
DON'T -- WE REALLY HAVE

9005
06:24:02,582 --> 06:24:03,483
SPECIALIZED HIGH SCIENCE, KIND

9006
06:24:03,483 --> 06:24:06,385
OF DRUG ORYEBTED MEETINGS BUT WE

9007
06:24:06,385 --> 06:24:08,588
DON'T HAVE THIS WHERE YOU TAKE A

9008
06:24:08,588 --> 06:24:09,822
MORE GENERALIZED APPROACH TO

9009
06:24:09,822 --> 06:24:11,124
BRING PEOPLE TOGETHER IN THIS

9010
06:24:11,124 --> 06:24:13,359
SPACE TO DO WHAT WE'RE DOING

9011
06:24:13,359 --> 06:24:14,527
TODAY AND TOMORROW AND SO WE'LL

9012
06:24:14,527 --> 06:24:16,496
SEE WHERE THAT GOES, BUT I THINK

9013
06:24:16,496 --> 06:24:19,398
THAT KIND OF FORUM, YOU KNOW,

9014
06:24:19,398 --> 06:24:21,901
EVEN GORDON, YOU KNOW THERE ARE

9015
06:24:21,901 --> 06:24:26,439
A LOT OF THESE, YOU KNOW PLACES

9016
06:24:26,439 --> 06:24:27,073
--

9017
06:24:27,073 --> 06:24:28,040
>> COLD SPRING HARBOR.

9018
06:24:28,040 --> 06:24:29,208
>> YEAH, I WAS JUST
BLOCKING ON

9019
06:24:29,208 --> 06:24:30,042
THAT NAME.

9020
06:24:30,042 --> 06:24:32,712
BUT YEAH, THERE ARE

9021
06:24:32,712 --> 06:24:33,146
OPPORTUNITIES TO --

9022
06:24:33,146 --> 06:24:34,947
>> SO NONE OF US ARE GOING
TO

9023
06:24:34,947 --> 06:24:36,649
LIKELY GO TO J. P. MORGAN SO

9024
06:24:36,649 --> 06:24:38,484
HAVING THOSE KIND OF SETTINGS,

9025
06:24:38,484 --> 06:24:40,319
WHERE YOU CAN BRING HIGH SCIENCE

9026
06:24:40,319 --> 06:24:43,723
TOGETHER WITH PEOPLE IN THE VC,

9027
06:24:43,723 --> 06:24:45,758
INVESTOR SPACE, I THINK WOULD BE

9028
06:24:45,758 --> 06:24:46,826
VERY INTERESTING AND YOU KNOW

9029
06:24:46,826 --> 06:24:48,461
WHAT YOU SPEND A FEW DAYS

9030
06:24:48,461 --> 06:24:50,897
TOGETHER LIKE THAT, GOOD IDEAS

9031
06:24:50,897 --> 06:24:53,733
COME FORWARD AND GET SUPPORTED.

9032
06:24:53,733 --> 06:24:54,801
SO I'LL GET BACK TO YOU ON HOW

9033
06:24:54,801 --> 06:24:56,669
IT GOES WITH THE KEY STONE

9034
06:24:56,669 --> 06:24:58,171
LEADERSHIP BUT I THINK THAT

9035
06:24:58,171 --> 06:25:02,341
WE'RE NOT REALLY DOING THAT AT

9036
06:25:02,341 --> 06:25:02,708
PRESENT.

9037
06:25:02,708 --> 06:25:05,111
USUALLY IT'S THE HOT PAPER THAT

9038
06:25:05,111 --> 06:25:06,145
GETS INVITED.

9039
06:25:06,145 --> 06:25:07,113
IT'S NOT SOMEBODY WHO'S TRYING

9040
06:25:07,113 --> 06:25:09,015
TO START A COMPANY.

9041
06:25:09,015 --> 06:25:15,087
>> AND I KNOW THAT ARE
THERE

9042
06:25:15,087 --> 06:25:16,222
THESE INVESTOR MEETINGS AND

9043
06:25:16,222 --> 06:25:20,059
THEY'RE VERY GIG  -- BIG AND ITS

9044
06:25:20,059 --> 06:25:22,028
HARD TO GO THERE AND FEEL

9045
06:25:22,028 --> 06:25:22,295
CONNECTED.

9046
06:25:22,295 --> 06:25:23,996
I TALK TO PEOPLE AND THEY'RE

9047
06:25:23,996 --> 06:25:25,398
LIKE, I DON'T KNOW WHO TO TALK

9048
06:25:25,398 --> 06:25:26,933
TO, WHERE TO GO, THERE'S SO MUCH

9049
06:25:26,933 --> 06:25:29,836
GOING ON HERE THAT MAYBE A

9050
06:25:29,836 --> 06:25:31,637
SMALLER VENUE STRUCTURA AS

9051
06:25:31,637 --> 06:25:32,471
YOU'RE SAYING MIGHT WORK BETTER

9052
06:25:32,471 --> 06:25:34,440
BECAUSE IT WILL HAVE A FOCUS, IT

9053
06:25:34,440 --> 06:25:36,309
WILL BE LONG, IT'LL BE SLEEP,

9054
06:25:36,309 --> 06:25:38,377
IT'LL BE RARE LUNG DECS, IT'LL

9055
06:25:38,377 --> 06:25:39,712
BE CHRONIC LUNG DECS, WHATEVER

9056
06:25:39,712 --> 06:25:43,216
IT IS, IT MIGHT BE A LITTLE MORE

9057
06:25:43,216 --> 06:25:44,517
FOCUSED AND EASIER FOR PEOPLE TO

9058
06:25:44,517 --> 06:25:46,485
CONNECT WITH EACH OTHER.

9059
06:25:46,485 --> 06:25:46,786
>> RIGHT.

9060
06:25:46,786 --> 06:25:48,855
EARLIER STAGE WITH SMALLER FOLKS

9061
06:25:48,855 --> 06:25:51,224
THAT MIGHT NOT GET INVITED TO

9062
06:25:51,224 --> 06:25:53,059
THE BIG SAN FRANCISCO MEETING OR

9063
06:25:53,059 --> 06:25:53,326
SOMETHING.

9064
06:25:53,326 --> 06:25:56,229
>> WE WILL SAY THAT WE
HAVE

9065
06:25:56,229 --> 06:25:58,331
WORKED WITH THE CF FOUNDATION ON

9066
06:25:58,331 --> 06:25:59,732
CONCEPTS EXACTLY LIKE THAT, AND

9067
06:25:59,732 --> 06:26:01,434
I THINK THE FOUNDATION HAS DONE

9068
06:26:01,434 --> 06:26:04,170
THIS A FEW TIMES, MAYBE, WITH

9069
06:26:04,170 --> 06:26:06,439
LONG WOOD AND ALSO SEPARATELY

9070
06:26:06,439 --> 06:26:08,975
THEMSELVES ON JUST NO 1 HAS

9071
06:26:08,975 --> 06:26:10,910
ANYTHING SPECIFIC QUITE YET TO

9072
06:26:10,910 --> 06:26:12,612
START A COMPANY AROUND BUT THERE

9073
06:26:12,612 --> 06:26:14,146
IS A THESIS THAT WE'RE

9074
06:26:14,146 --> 06:26:17,049
INTERESTED IN AND WHETHER IT'S

9075
06:26:17,049 --> 06:26:18,651
DELIVERY OR IT'S OPPORTUNITIES

9076
06:26:18,651 --> 06:26:22,788
TO FIGURE OUT CARGOS, VERY

9077
06:26:22,788 --> 06:26:24,891
SPECIFIC, AND 1 THING THAT KIND

9078
06:26:24,891 --> 06:26:26,058
OF CATALYZES THIS IS THAT PEOPLE

9079
06:26:26,058 --> 06:26:28,261
NEED TO BE ABLE TO GET

9080
06:26:28,261 --> 06:26:29,695
COMFORTABLE WITH SHARING

9081
06:26:29,695 --> 06:26:30,930
UNPUBLISHED DATA AND THEN BEING

9082
06:26:30,930 --> 06:26:34,667
A LITTLE BIT MORE OPEN WITH THE

9083
06:26:34,667 --> 06:26:35,501
THINGS THAT THEY'RE WORKING ON

9084
06:26:35,501 --> 06:26:36,869
WHICH I KNOW IS DIFFICULT

9085
06:26:36,869 --> 06:26:40,139
BECAUSE, YOU KNOW SCOOPING AND

9086
06:26:40,139 --> 06:26:43,175
EVERYTHING ELSE BUT I KNOW WHEN

9087
06:26:43,175 --> 06:26:46,379
WE'VE STARTED THESE KIND OF

9088
06:26:46,379 --> 06:26:48,281
CONVERSATIONS IT'S ALWAYS A --

9089
06:26:48,281 --> 06:26:52,184
LET'S PLAN OUT AND WE TALKED

9090
06:26:52,184 --> 06:26:53,019
WITH ENRIQUE ABOUT THIS, WHO

9091
06:26:53,019 --> 06:26:56,222
WOULD BE IN THE SAME ROOM WITH

9092
06:26:56,222 --> 06:26:58,991
WHO WITHOUT A FEAR OF POTENTIAL

9093
06:26:58,991 --> 06:27:00,760
COMPETITION AND THEN GETTING

9094
06:27:00,760 --> 06:27:01,794
THAT TOGETHER TOCCATAALIZE THE

9095
06:27:01,794 --> 06:27:02,128
CONVERSATION.

9096
06:27:02,128 --> 06:27:05,331
SO IT'S ALWAYS NICE TO BE ABLE

9097
06:27:05,331 --> 06:27:06,832
TO BRING FOLKS TOGETHER AROUND

9098
06:27:06,832 --> 06:27:08,701
SPECIFIC TOPICS BUT I GUESS

9099
06:27:08,701 --> 06:27:09,969
THERE'S DIFFERENT COMPLICATIONS

9100
06:27:09,969 --> 06:27:13,239
THAT ARISE THAT REALLY, MAYBE

9101
06:27:13,239 --> 06:27:14,507
LIMIT THE CONVERSATION IN A

9102
06:27:14,507 --> 06:27:16,042
PARTICULAR WAY, BUT THENOT OTHER

9103
06:27:16,042 --> 06:27:18,778
HAND, I THINK THERE'S ALWAYS --

9104
06:27:18,778 --> 06:27:20,746
I THINK PEOPLE ARE OPEN TO

9105
06:27:20,746 --> 06:27:22,949
TALKING, IF YOU FIND VENTURE

9106
06:27:22,949 --> 06:27:25,017
CREATION GROUPS WHICH I THINK

9107
06:27:25,017 --> 06:27:26,285
MOST GROUPS ADVERTISE QUITE

9108
06:27:26,285 --> 06:27:28,120
LOUDLY THAT THEY ARE AND YOU

9109
06:27:28,120 --> 06:27:29,722
WANT TO THROW OUT SOME IDEAS

9110
06:27:29,722 --> 06:27:30,656
ABOUT A PARTICULAR THING, NOT

9111
06:27:30,656 --> 06:27:33,159
THAT YOU HAVE A PARTICULAR

9112
06:27:33,159 --> 06:27:35,461
TECHNOLOGY, BUT JUST HEY, WHAT

9113
06:27:35,461 --> 06:27:37,196
ABOUT THIS GENERAL TOPIC.

9114
06:27:37,196 --> 06:27:38,331
THAT'S ALWAYS A CONVERSATION

9115
06:27:38,331 --> 06:27:39,365
THAT COULD BE OPEN FROM THE

9116
06:27:39,365 --> 06:27:41,467
OTHER DIRECTION AS WELL, INSTEAD

9117
06:27:41,467 --> 06:27:43,903
OF WAITING FOR OUTREACH OR

9118
06:27:43,903 --> 06:27:45,805
SOMETHING LIKE THAT.

9119
06:27:45,805 --> 06:27:47,640
>> YOU KNOW, IN SOME WAYS

9120
06:27:47,640 --> 06:27:48,708
CATALYZE HAS ALREADY ACHIEVED

9121
06:27:48,708 --> 06:27:50,409
SOME OF THAT AND HOW THEY BRING

9122
06:27:50,409 --> 06:27:52,745
ALL THE INVESTIGATORS TOGETHER,

9123
06:27:52,745 --> 06:27:54,880
AND YOU KNOW MAYBE YOU CAN

9124
06:27:54,880 --> 06:28:00,052
EXPAND ON THAT, MA RRAH AND GROW

9125
06:28:00,052 --> 06:28:00,419
THAT MODEL.

9126
06:28:00,419 --> 06:28:02,488
IN THERE YOU HAVE A BUNCH OF

9127
06:28:02,488 --> 06:28:03,255
INNOVATORS THAT COME TOGETHER

9128
06:28:03,255 --> 06:28:04,390
BECAUSE THEY'RE IN THE PROGRAM

9129
06:28:04,390 --> 06:28:06,225
AND IT SORT OF GRANDFATHERS IN

9130
06:28:06,225 --> 06:28:08,194
THE PEOPLE THAT GOT FUNDING

9131
06:28:08,194 --> 06:28:09,829
BEFORE, TOO, I STILL GET ALL THE

9132
06:28:09,829 --> 06:28:10,763
INVITES AND EVERYTHING, SO HAVE

9133
06:28:10,763 --> 06:28:12,264
YOU A STRUCTURE THERE THAT MAY

9134
06:28:12,264 --> 06:28:13,566
ACTUALLY BE SOMETHING THAT CAN

9135
06:28:13,566 --> 06:28:16,302
TURN INTO A COLLABORATION

9136
06:28:16,302 --> 06:28:18,804
BETWEEN GOVERNMENT, VENTURE

9137
06:28:18,804 --> 06:28:20,072
FOUNDATION AND ACADEMIA, THAT

9138
06:28:20,072 --> 06:28:21,707
COULD GROW, MAYBE AND REALLY

9139
06:28:21,707 --> 06:28:24,677
CAPTURE THIS INNOVATION SPACE

9140
06:28:24,677 --> 06:28:26,679
THAT A LOT OF FUNDING MECHANISMS

9141
06:28:26,679 --> 06:28:30,383
RIGHT NOW DON'T NECESSARILY DO.

9142
06:28:30,383 --> 06:28:30,950
>> YEAH, RIEWR RIGHT.

9143
06:28:30,950 --> 06:28:33,219
WE TRY TO KEEP PEOPLE QUOTE IN

9144
06:28:33,219 --> 06:28:35,054
THE PROGRAM EVEN AFTER THEY SUN

9145
06:28:35,054 --> 06:28:36,722
SET FROM THEIR GRANT BECAUSE YOU

9146
06:28:36,722 --> 06:28:38,090
NEVER KNOW WHAT YOU'RE GOING TO

9147
06:28:38,090 --> 06:28:39,258
BENEFIT FROM THROUGH THE WEBINAR

9148
06:28:39,258 --> 06:28:42,461
SERIES AND ALL THE PROGRAMMING

9149
06:28:42,461 --> 06:28:46,198
THAT RTI HELP SUPPORT, YOU KNOW,

9150
06:28:46,198 --> 06:28:48,000
THE NETWORK WILL GROW BIGGER AS

9151
06:28:48,000 --> 06:28:49,635
MORE PEOPLE GET FUNDED AND THEN,

9152
06:28:49,635 --> 06:28:51,070
YOU KNOW MAYBE THAT PEER TO PEER

9153
06:28:51,070 --> 06:28:53,706
MODEL WILL CONTINUE TO BE MORE

9154
06:28:53,706 --> 06:28:55,875
SUCCESSFUL, I THINK WE'RE ON OUR

9155
06:28:55,875 --> 06:29:00,012
THIRD RENEWAL OF THAT PROGRAM

9156
06:29:00,012 --> 06:29:01,313
WITH THE RECEIPT BASE I JUST PUT

9157
06:29:01,313 --> 06:29:03,616
IN THE CHAT EARLIER TODAY, SO

9158
06:29:03,616 --> 06:29:06,252
IT'S STILL KIND OF A NEWISH AND

9159
06:29:06,252 --> 06:29:08,954
I THINK WE'RE LEARNING AND MIKE

9160
06:29:08,954 --> 06:29:10,723
HAS BEEN A STELLAR LEADER IN

9161
06:29:10,723 --> 06:29:12,091
THAT PROGRAM AND CERTAINLY WE

9162
06:29:12,091 --> 06:29:15,828
CAN TAKE THAT FEEDBACK TO HIM AS

9163
06:29:15,828 --> 06:29:16,762
WELL.

9164
06:29:16,762 --> 06:29:18,931
>> AND THEN YEAH, I WONDER
IF

9165
06:29:18,931 --> 06:29:21,967
IT'S WORTH THINKING ABOUT MAYBE

9166
06:29:21,967 --> 06:29:23,836
A SMALL SCALE PARTNER ASK

9167
06:29:23,836 --> 06:29:24,670
CONFERENCE TYPE OF MEETING WHERE

9168
06:29:24,670 --> 06:29:26,972
YOU CAN STILL HAVE THOSE 30

9169
06:29:26,972 --> 06:29:30,810
MINUTE MEETINGS, BUT AT A MORE

9170
06:29:30,810 --> 06:29:33,079
SPECIFIC TOPIC BASE, STILL

9171
06:29:33,079 --> 06:29:34,713
INVITE VCs AND INVESTORS BUT

9172
06:29:34,713 --> 06:29:36,749
THEN NOT HAVE THE GIANT J. P.

9173
06:29:36,749 --> 06:29:38,417
MORGAN WHICH IN MANY WAYS IT'S

9174
06:29:38,417 --> 06:29:40,953
SO BIG, IT'S OVERWHELMING EVEN

9175
06:29:40,953 --> 06:29:45,191
FOR PEOPLE LIKE LARGE INVESTORS.

9176
06:29:45,191 --> 06:29:48,127
>> CAN I ASK LUCY, ALL YOU
CF

9177
06:29:48,127 --> 06:29:49,728
GUYS, DOES THIS HAPPEN AT THE

9178
06:29:49,728 --> 06:29:51,931
CF, IS THERE SORT OF A SATELLITE

9179
06:29:51,931 --> 06:29:53,432
MEETING WITHIN THE MEETING AT

9180
06:29:53,432 --> 06:29:55,401
THE CF MEETING THAT ATTACKS THIS

9181
06:29:55,401 --> 06:29:55,935
KIND OF PROBLEM?

9182
06:29:55,935 --> 06:29:58,871
, 

9183
06:29:58,871 --> 06:30:00,606
YOU KNOW BRINGING WHAT WE TALK

9184
06:30:00,606 --> 06:30:02,141
ABOUT IN A SENSE?

9185
06:30:02,141 --> 06:30:04,543
>> SO SOME OF THAT HAPPENS
AT

9186
06:30:04,543 --> 06:30:07,012
CFAC, AND THEY COME TO CFAC, THE

9187
06:30:07,012 --> 06:30:07,746
SUMMER RESEARCH CONFERENCE AS

9188
06:30:07,746 --> 06:30:11,684
WELL AS SOMETIMES BUT WE DON'T

9189
06:30:11,684 --> 06:30:15,254
NECESSARILY HAVE THE FORUM OR

9190
06:30:15,254 --> 06:30:16,288
THE VENUE JUST YET.

9191
06:30:16,288 --> 06:30:18,457
WE DO GO TO A LOT OF CONFERENCES

9192
06:30:18,457 --> 06:30:21,560
AND DO A LOT OF OUTBOUND

9193
06:30:21,560 --> 06:30:22,728
OUTSOURCING BECAUSE THERE IS --

9194
06:30:22,728 --> 06:30:23,929
THIS IS LIKE SOMETHING I STARTED

9195
06:30:23,929 --> 06:30:28,267
WHEN I WAS A KID, HAS NOW GROWN

9196
06:30:28,267 --> 06:30:30,236
INTO RAIS, THE RESPIRATORY

9197
06:30:30,236 --> 06:30:31,403
INNOVATION SUMMIT.

9198
06:30:31,403 --> 06:30:33,606
I DON'T KNOW IF ANYBODY'S

9199
06:30:33,606 --> 06:30:34,607
FAMILIAR WITH THAT SO THAT

9200
06:30:34,607 --> 06:30:36,942
HAPPENS ON THE FRIDAY AND

9201
06:30:36,942 --> 06:30:39,478
SATURDAY BEFORE ATS BEGINS AND

9202
06:30:39,478 --> 06:30:40,412
THAT'S ESSENTIALLY FORUM FOR

9203
06:30:40,412 --> 06:30:42,014
SMALL COMPANIES TO COME AND

9204
06:30:42,014 --> 06:30:43,182
PRESENT EARLY STAGE WORK AND

9205
06:30:43,182 --> 06:30:45,451
PITCH, YOU KNOW FOR FURTHER

9206
06:30:45,451 --> 06:30:45,684
FUNDING.

9207
06:30:45,684 --> 06:30:47,186
IT'S NOW UP TO SEVERAL HUNDRED

9208
06:30:47,186 --> 06:30:50,055
PEOPLE THAT TBET TOGETHER, WITH

9209
06:30:50,055 --> 06:30:53,125
POSTERS AND TALKS, IN AN EFFORT

9210
06:30:53,125 --> 06:30:55,761
TO BRING INVESTORS AND FOUNDERS

9211
06:30:55,761 --> 06:31:00,666
TOGETHER, YOU KNOW AT AN EARLY

9212
06:31:00,666 --> 06:31:00,900
STAGE.

9213
06:31:00,900 --> 06:31:04,637
SO AM I THE ONLY 1 THAT KNOWS

9214
06:31:04,637 --> 06:31:05,237
ABOUT IT?

9215
06:31:05,237 --> 06:31:06,539
IS THAT BAD IN.

9216
06:31:06,539 --> 06:31:07,473
>> I DEFINITELY KNOW ABOUT
IT,

9217
06:31:07,473 --> 06:31:09,875
IT'S A GOOD FORUM, PART OF THE

9218
06:31:09,875 --> 06:31:12,044
SITUATION WITH AT LEAST IN MY

9219
06:31:12,044 --> 06:31:14,713
EXPERIENCE IS THAT, IT'S NOT

9220
06:31:14,713 --> 06:31:16,782
QUITE ADDRESSING RARE NEEDS.

9221
06:31:16,782 --> 06:31:20,286
I THINK THAT, YOU KNOW THEY HAVE

9222
06:31:20,286 --> 06:31:21,487
THE PARWHICH IS ANOTHER MEETING

9223
06:31:21,487 --> 06:31:27,893
THAT HAPPENS ON SATURDAY WITH

9224
06:31:27,893 --> 06:31:29,228
THE ADVOCACY EFFICACY SUBGROUP,

9225
06:31:29,228 --> 06:31:31,263
THAT THERE NEEDS TO BE A GROUP

9226
06:31:31,263 --> 06:31:33,432
FOCUSED ON LUNG DISORDERS WITHIN

9227
06:31:33,432 --> 06:31:34,867
THE INNOVATION SUMMIT OR

9228
06:31:34,867 --> 06:31:36,769
ENCOURAGING THAT PEOPLE TO COME

9229
06:31:36,769 --> 06:31:39,538
THERE, WITH THAT MISSION AND SO

9230
06:31:39,538 --> 06:31:40,673
THEIR PARTICIPATING WITH THE

9231
06:31:40,673 --> 06:31:42,508
SMALLER AND LARGER GROUPS THAT

9232
06:31:42,508 --> 06:31:45,177
ARE AT THE SUMMIT.

9233
06:31:45,177 --> 06:31:45,444
>> YEAH.

9234
06:31:45,444 --> 06:31:46,946
>> I WAS JUST GETTING AT
THE

9235
06:31:46,946 --> 06:31:50,449
GENERAL NOTION, NOT THE RARE

9236
06:31:50,449 --> 06:31:52,284
DISEASE BUT YOU'RE RIGHT, IT'S

9237
06:31:52,284 --> 06:31:54,153
MORE ALL COMERS AND RESPIRATORY

9238
06:31:54,153 --> 06:31:54,386
DISEASE.

9239
06:31:54,386 --> 06:31:56,055
>> I THINK WE'RE OVER
TIME, DO

9240
06:31:56,055 --> 06:31:56,889
WE STILL --

9241
06:31:56,889 --> 06:31:58,490
>> YEAH, I JUST WANTED TO

9242
06:31:58,490 --> 06:32:01,794
TRANSITION INTO CLOSING OF THE

9243
06:32:01,794 --> 06:32:03,629
WORKSHOP, SO WE STAY ON SCHEDULE

9244
06:32:03,629 --> 06:32:05,497
FOR TODAY AND I INVITED CAITLIN

9245
06:32:05,497 --> 06:32:07,566
AND JEFF TO GIVE A FEW WORDS

9246
06:32:07,566 --> 06:32:08,734
ABOUT, YOU KNOW GENERAL TAKE

9247
06:32:08,734 --> 06:32:11,103
AWAYS FROM TODAY AND THEN ALSO A

9248
06:32:11,103 --> 06:32:12,004
LITTLE BIT ABOUT WHAT WE'RE

9249
06:32:12,004 --> 06:32:14,974
GOING TO BE DOING TOMORROW.

9250
06:32:14,974 --> 06:32:16,909
SO YOU KNOW FOLKS THAT ARE STILL

9251
06:32:16,909 --> 06:32:18,510
ON THE CALL CAN YOU KNOW THINK

9252
06:32:18,510 --> 06:32:19,445
ABOUT THE STRATEGIES WE'RE GOING

9253
06:32:19,445 --> 06:32:22,147
TO TAKE FROM TODAY'S DISCUSSION

9254
06:32:22,147 --> 06:32:24,683
AND SORT OF BRIDGE IT INTO

9255
06:32:24,683 --> 06:32:28,287
TOMORROW'S SESSIONS AS WELL.

9256
06:32:28,287 --> 06:32:29,922
>> WELL, I WOULD BE HAPPY
TO

9257
06:32:29,922 --> 06:32:30,589
KICK THAT OFF.

9258
06:32:30,589 --> 06:32:33,125
I JUST WANT TO START OUT BY

9259
06:32:33,125 --> 06:32:36,395
THANKING ALL THE SPEAKERS TODAY

9260
06:32:36,395 --> 06:32:37,496
IN THE PANELISTS AND EVERYONE

9261
06:32:37,496 --> 06:32:38,430
DID A FANTASTIC YOB CORPS AND

9262
06:32:38,430 --> 06:32:39,398
WALKING THROUGH THE YOWRNY IT

9263
06:32:39,398 --> 06:32:41,166
TAKES TO GO FROM AN IDEA OR

9264
06:32:41,166 --> 06:32:43,102
CONCEPT AROUND A THERAPEUTIC AND

9265
06:32:43,102 --> 06:32:45,204
JUST HOW MUCH WORK IS INVOLVED

9266
06:32:45,204 --> 06:32:48,474
TO MATURE THAT IDEA INTO A DRUG

9267
06:32:48,474 --> 06:32:50,109
ASSET, AND IN PARTNERING AS

9268
06:32:50,109 --> 06:32:51,610
WE'VE HEARD IN THIS GREAT

9269
06:32:51,610 --> 06:32:53,145
DISCUSSION TODAY AROUND WORKING

9270
06:32:53,145 --> 06:32:54,780
WITH REGULATORS AND ULTIMATELY

9271
06:32:54,780 --> 06:32:56,382
VENTURE CAPITAL AND OTHER

9272
06:32:56,382 --> 06:32:57,850
MECHANISMS THROUGH THE

9273
06:32:57,850 --> 06:32:59,485
GOVERNMENT OR OTHERWISE TO TRY

9274
06:32:59,485 --> 06:33:01,654
AND GENERATE FUNDS TO DEVELOP

9275
06:33:01,654 --> 06:33:02,688
THESE THERAPIES BECAUSE IT IS

9276
06:33:02,688 --> 06:33:04,323
VERY MUCH A TEAM SPORT AND THERE

9277
06:33:04,323 --> 06:33:06,091
ARE A LOT OF EXPERTS THAT YOU

9278
06:33:06,091 --> 06:33:09,028
NEED TO TALK TO THAT -- AND

9279
06:33:09,028 --> 06:33:11,063
THANKFULLY, THERE'S GROUPS LIKE

9280
06:33:11,063 --> 06:33:12,564
THE CYSTIC FIBROSIS FOUNDATION

9281
06:33:12,564 --> 06:33:13,932
AND THE CHILDHOOD INTERSTITIAL

9282
06:33:13,932 --> 06:33:15,000
LUNG DEC GROUP THAT ARE OUT

9283
06:33:15,000 --> 06:33:16,869
THERE THAT YOU CAN PARTNER WITH

9284
06:33:16,869 --> 06:33:18,804
TO GET EDUCATED ON THESE

9285
06:33:18,804 --> 06:33:19,872
DIFFERENT DISEASES, AS YOU'RE

9286
06:33:19,872 --> 06:33:21,507
TRYING TO MATURE YOUR ASSETS TO

9287
06:33:21,507 --> 06:33:22,007
THE CLINIC.

9288
06:33:22,007 --> 06:33:25,311
SO I THINK, WE LEARNED A LOT OF

9289
06:33:25,311 --> 06:33:26,011
GREAT LESSONS TODAY AROUND

9290
06:33:26,011 --> 06:33:27,680
TRYING TO GET US TO THE CLINIC

9291
06:33:27,680 --> 06:33:29,782
AND THAT'S A PERFECT SET UP FOR

9292
06:33:29,782 --> 06:33:31,083
CAITLIN AROUND WHERE WE'RE

9293
06:33:31,083 --> 06:33:33,719
HEADED TO TOMORROW.

9294
06:33:33,719 --> 06:33:35,554
>> WAY TO PASS THE BATON.

9295
06:33:35,554 --> 06:33:37,856
YEAH, REALLY GREAT SESSION,

9296
06:33:37,856 --> 06:33:38,891
TODAY, MAKES ME APPRECIATE ALL

9297
06:33:38,891 --> 06:33:39,958
THE BASIC SCIENCE RESEARCHERS

9298
06:33:39,958 --> 06:33:42,561
AND THE PEOPLE THAT ARE IN THE

9299
06:33:42,561 --> 06:33:43,962
TRENCHES, FAILING MORE OFTEN

9300
06:33:43,962 --> 06:33:46,031
THAT YOU SUCCEED SO THAT WE CAN

9301
06:33:46,031 --> 06:33:46,965
ACTUALLY MAKE POSITIVE CHANGE SO

9302
06:33:46,965 --> 06:33:48,767
THANKS TO ALL OF YOU WHO ARE

9303
06:33:48,767 --> 06:33:49,034
INVOLVED.

9304
06:33:49,034 --> 06:33:50,402
TOMORROW'S GOING TO BE A BIT OF

9305
06:33:50,402 --> 06:33:52,471
A DIFFERENT FLAVOR, WE'RE GOING

9306
06:33:52,471 --> 06:33:54,206
TO TALK ABOUT MORE OF WHAT

9307
06:33:54,206 --> 06:33:58,444
HAPPENS IN THE CLINICAL SPACE SO

9308
06:33:58,444 --> 06:33:59,745
NOT ONLY THE CLINICAL TRIAL

9309
06:33:59,745 --> 06:34:01,613
ASPECTS OF THINGS BUT ALSO HOW

9310
06:34:01,613 --> 06:34:03,682
WE ENGAGE WITH FOUNDATIONS AND

9311
06:34:03,682 --> 06:34:06,051
PATIENTS AND WHERE THEY MAY

9312
06:34:06,051 --> 06:34:07,386
BE-FUL ALONG THE PATH AND IT WAS

9313
06:34:07,386 --> 06:34:11,757
REALLY NICE TO HAVE THAT INITIAL

9314
06:34:11,757 --> 06:34:13,926
TALK FROM THE CF FOUNDATION

9315
06:34:13,926 --> 06:34:15,627
BECAUSE THEY ARE THE GOLD

9316
06:34:15,627 --> 06:34:17,730
STANDARD FOR ALL OF US

9317
06:34:17,730 --> 06:34:18,997
FOUNDATIONS FOR WHICH WE'RE ABLE

9318
06:34:18,997 --> 06:34:20,165
TO FUNCTION EMPLOY SO I WANT TO

9319
06:34:20,165 --> 06:34:23,736
GIVE YOU A BIT OF A FLAVOR FOR

9320
06:34:23,736 --> 06:34:24,737
THE TALKS TOMORROW.

9321
06:34:24,737 --> 06:34:25,938
WE WILL SPEND SOMETIME IN THE

9322
06:34:25,938 --> 06:34:27,773
MORNING LEARNING ABOUT THE

9323
06:34:27,773 --> 06:34:30,275
CHALLENGES TO TRIAL DEVELOPMENT

9324
06:34:30,275 --> 06:34:31,643
AND INFANTS AND CHILDREN WITH

9325
06:34:31,643 --> 06:34:32,878
RARE LUNG DEC.

9326
06:34:32,878 --> 06:34:34,513
WE WILL THINK A LITTLE BIT ABOUT

9327
06:34:34,513 --> 06:34:37,049
WHAT GOES INTO DESIGNING AND

9328
06:34:37,049 --> 06:34:37,783
EXECUTING CLINICAL TRIALS, PIT

9329
06:34:37,783 --> 06:34:39,885
FALLS TO,A VOID, THINGS TO MAKE

9330
06:34:39,885 --> 06:34:41,286
SURE WE CONSIDER, WE WILL LEARN

9331
06:34:41,286 --> 06:34:44,690
A LITTLE BIT FROM THE FIRST

9332
06:34:44,690 --> 06:34:47,192
CLINICAL TRIAL IN PEDIATRIC AND

9333
06:34:47,192 --> 06:34:49,027
INTERSTITIAL LUNG DEC, WHAT WE

9334
06:34:49,027 --> 06:34:50,629
LEARN PRACTICES THAT PROCESS

9335
06:34:50,629 --> 06:34:53,098
TALK ABOUT CONTRACTING FOR

9336
06:34:53,098 --> 06:34:53,732
INVESTIGATOR INITIATED CLINICAL

9337
06:34:53,732 --> 06:34:53,966
TRIALS.

9338
06:34:53,966 --> 06:34:55,834
THEN WE WILL HAVE ANOTHER LUNCH

9339
06:34:55,834 --> 06:34:57,202
BREAK AS WE DID TODAY AND THEN

9340
06:34:57,202 --> 06:34:59,004
IN THE AFTERNOON WE WILL FOCUS

9341
06:34:59,004 --> 06:35:00,272
ON SUCCESS STORIES WE HAVE FROM

9342
06:35:00,272 --> 06:35:02,007
THIS CLINICAL SIDE OF THINGS,

9343
06:35:02,007 --> 06:35:03,742
WE'RE LUCKY TO HAVE AN ADULT

9344
06:35:03,742 --> 06:35:05,577
PERSON WITH CF SPEAKS MORE ABOUT

9345
06:35:05,577 --> 06:35:07,045
HIS EXPERIENCE WITH CLINICAL

9346
06:35:07,045 --> 06:35:08,947
RESEARCH, SO FROM A PATIENT

9347
06:35:08,947 --> 06:35:10,682
PERSPECTIVE AND WE WILL LEARN

9348
06:35:10,682 --> 06:35:13,619
FROM THE PC D FOUNDATION THEIR

9349
06:35:13,619 --> 06:35:16,355
EXPERIENCE WITH CLINICAL TRIALS,

9350
06:35:16,355 --> 06:35:18,891
AND WE'LL HEAR ABOUT THE

9351
06:35:18,891 --> 06:35:21,126
MEDICATION PIPELINE FOR

9352
06:35:21,126 --> 06:35:22,528
PULMONARY FIBROSIS HAS HAD GREAT

9353
06:35:22,528 --> 06:35:24,029
STRIDES OVER THE PAST SEVERAL

9354
06:35:24,029 --> 06:35:25,297
YEARS AND THEN FINALLY AS A

9355
06:35:25,297 --> 06:35:27,466
GREAT EXAMPLE OF HOW THE LAMB

9356
06:35:27,466 --> 06:35:29,334
FOUNDATION HAS REALLY HELPED

9357
06:35:29,334 --> 06:35:31,870
BRING ABOUT TARGETED THERAPY, IN

9358
06:35:31,870 --> 06:35:34,139
THAT PARTNERSHIP, AND THEN

9359
06:35:34,139 --> 06:35:35,240
PERHAPS, THE MOST REALLY

9360
06:35:35,240 --> 06:35:36,942
EXCITING PART OF THE DAY IS,

9361
06:35:36,942 --> 06:35:38,110
BREAK OUT SESSIONS IN THE

9362
06:35:38,110 --> 06:35:39,912
AFTERNOON, SO WE'LL HAVE 3

9363
06:35:39,912 --> 06:35:42,014
SEPARATE BREAK OUT SESSIONS THAT

9364
06:35:42,014 --> 06:35:44,183
-- WITH DIFFERENT MODERATORS,

9365
06:35:44,183 --> 06:35:45,451
THAT WILL HOPEFULLY HELP

9366
06:35:45,451 --> 06:35:46,819
CONTINUE THE SAME KIND OF

9367
06:35:46,819 --> 06:35:47,553
CONVERSATION WE'VE BEEN HAVING

9368
06:35:47,553 --> 06:35:50,289
TODAY, SO THAT WE CAN REALLY TRY

9369
06:35:50,289 --> 06:35:51,690
TO GET ACTION ITEMS AND IDEAS

9370
06:35:51,690 --> 06:35:53,125
ABOUT HOW TO MAKE THIS PROCESS

9371
06:35:53,125 --> 06:35:53,559
BETTER.

9372
06:35:53,559 --> 06:35:55,294
SO WITH THAT WE JUST HOPE THAT

9373
06:35:55,294 --> 06:35:58,464
WE WILL SEE YOUR FACES TOMORROW,

9374
06:35:58,464 --> 06:35:59,865
I PROMISE FOR THE BASIC SCIENCE

9375
06:35:59,865 --> 06:36:01,366
PEOPLE AND THE RESEARCH PEOPLE,

9376
06:36:01,366 --> 06:36:03,435
WE WILL -- IT WILL BE AN

9377
06:36:03,435 --> 06:36:06,572
INTERESTING DAY AND THERE'S SOME

9378
06:36:06,572 --> 06:36:08,173
PATIENT FACES TO JUST HELP

9379
06:36:08,173 --> 06:36:09,441
CONTINUE TO INSPIRE PEOPLE TO DO

9380
06:36:09,441 --> 06:36:13,312
THE GOOD WORK YOU GUYS ARE

9381
06:36:13,312 --> 06:36:13,512
DOING.

9382
06:36:13,512 --> 06:36:16,682
>> ANY OTHER FINAL WORDS,
MARRAH

9383
06:36:16,682 --> 06:36:18,283
NNO THAT COVER ITS NICELY AND

9384
06:36:18,283 --> 06:36:19,785
LOOK FORWARD TO SEEING EVERYBODY

9385
06:36:19,785 --> 06:36:21,220
TOMORROW AT THE MEETING.

9386
06:36:21,220 --> 06:36:23,689
>> SEE IN THE MORNING
GUYS,

9387
06:36:23,689 --> 06:36:33,689
THANK YOU.