>> SO, WELCOME BACK, EVERYBODY.
IT IS MY ABSOLUTE PLEASURE TO
INTRODUCE OUR NEXT SPEAKER
MONICA BER TIG NOELI. AS MANY
KNOW SHE PREVIOUSLY SERVED AS
DIRECTOR OF NATIONAL CANCER
INSTITUTE BEFORE STARTING ROLE
AS NIH DIRECTOR THIS PAST
NOVEMBER. SHE HAS BEEN A CANCER
SURGEON FOR MORE THAN 35 YEARS
BRAND JOINING NCI SHE JOINED IN
SURGERY AND ONCOLOGY AT HARVARD
MEDICAL SCHOOL.
AND SAR COMA CENTERS AT DANA
FARM INSTITUTE THROUGHOUT CAREER
SHE HAS LED TRANSLATIONAL
SCIENCE INITIATIVES WITHIN
NCI-FUNDED COOPERATIVE GROUPS
PROGRAMS AND HAS WAY TOO MANY
ACCOMPLISHMENTS AND CREDENTIALS
TOE GO THROUGH THIS SHORT TIME.
I COULD SPEND NEXT HOUR
DESCRIBING THESE. YOU WOULD
LOVE TO HEAR FROM HER. I WILL
TURN IT OVER TO LOOK FOR DR.
BERTAGNOLLI. I WILL UPLOAD YOUR
SLIDES.
>> HOW ARE WE DOING? YES,
PLEASE, THE SLIDES.
THANK YOU SO MUCH FOR THE KIND
INTRODUCTION. THIS IS AN
INVITATION I ACCEPTED AFTER
BECOMING NIH DIRECTOR IN
NOVEMBER. I'M EXCITED ABOUT
POTENTIAL PARTNERSHIP BETWEEN
NIH AND INDUSTRY AND HAVE TO SAY
THROUGHOUT MY CAREER I HAVE BEEN
ABLE TO PARTICIPATE IN
PARTNERSHIPS OF THIS TYPE THAT
HAVE BEEN INCREDIBLY VALUABLE.
NEXT SLIDE.
I WILL HAVE SOMEBODY RUN --
THANK YOU.
BEFORE BECOMING NIH DIRECTOR I
WAS A CANCER SURGEON AND
RESEARCHER AND YOU SEE FROM THE
PHOTOS I GREW UP FROM
INTERMOUNTAIN WEST AND STILL
RETURN THERE.
THIS HAS GIVEN ME A PASSION FOR
ENSURING THAT INNOVATION IN
HEALTH REACHES EVERYONE WHO CAN
BENEFIT INCLUDING THOSE WHO LIVE
IN RURAL COMMUNITIES FAR FROM
MAJOR HUBS OF ACADEMIC LIFE.
THESE ARE TOPICS I WILL DISCUSS
TODAY.
NEXT SLIDE.
LY HELP INTRODUCE YOU TO NIH.
YOU KNOW NIH.
THIS IS A BIG AND VERY COMPLEX
PLACE AND WITH THIS CONFERENCE
HOPE TO MAKE IT EASIER FOR YOU
TO NAVIGATE.
AT NIH WE SEEK FUNDAMENTAL
KNOWLEDGE ABOUT NATURE AND
BEHAVIOR OF LIVING SYSTEMS AND
TO APPLY THAT KNOWLEDGE IN WAYS
THAT IMPROVE HEALTH FOR ALL.
TODAY'S DISCUSSIONS WILL REVOLVE
AROUND TRANSLATIONAL PART OF OUR
MISSION AND ACTUAL APPLICATION
OF KNOWLEDGE TO IMPROVE HEALTH.
NEXT SLIDE.
NIH TODAY SUPPORTS RESEARCH
AROUND THE COUNTRY AND INDEED
THROUGHOUT THE WORLD AND TRAINS
INVESTIGATORS CONDUCTING
RESEARCH IN ITS OWN
LABORATORIES.
NEXT SLIDE.
NIH BUDGET IN FISCAL YEAR 2023
WAS $47.3 BILLION AND LARGE
MAJORITY OF NIH SPENDING IS MADE
UP OF GRANTS TO RESEARCHERS AT
UNIVERSITIES, MEDICAL SCHOOLS
AND SCIENTIFIC INSTITUTIONS
OUTSIDE OF THE NIH WALLS.
IN FUTURE YEARS WE HOPE TO
INCORPORATE MORE INFORMATION ON
THIS VERY LARGE EXTRAMURAL
RESEARCH PROGRAM INTO THIS
CONFERENCE.
TODAY, WE ARE FOCUSING ON THE
NIH INTRAMURAL RESEARCH PROGRAM
TO WHICH WE DEVOTE ABOUT 11% OF
NIH'S TOTAL BUDGET.
NEXT SLIDE.
INTRAMURAL RESEARCH PROGRAM
LARGELY LOCATED IN MARYLAND
INCLUDES MORE THAN 3,000 ACTIVE
PROJECTS AND MORE THAN 6,000
SCIENTISTS ENGAGED IN
COLLABORATIVE RESEARCH AND
ACTIVE IN PATENTING AND
LICENSING OUR WORK.
NIH IS MADE UP OF 27 INSTITUTES
AND CENTERS AND EACH HAS ITS OWN
SPECIFIC RESEARCH AGENDA.
EACH ADMINISTERS ITS OWN BUDGET.
23 OF INSTITUTES AND CENTERS
ARE INVOLVED IN FUNDING AND
DIRECTING INTRAMURAL RESEARCH.
NEXT SLIDE.
AT NIH, WE TRY TO KEEP OUR
ULTIMATE GOALS IN THE FOREGROUND
ALWAYS WHEN WE THINK ABOUT
DELIVERING ON NIH'S MISSION, WE
KNOW THAT INCLUDES MAKING SURE
INSIGHTS AND DISCOVERIES
EMERGING INTO RESEARCH GET OUT
INTO THE WORLD AND IMPROVE NIH'S
LIVE AND IS NOT DONE WHEN
INVESTIGATORS PUBLISH A PAPER.
PEOPLE'S LIVES CAN ONLY GET
BETTER IF THEY CAN ACCESS
PRODUCTS AND SERVICES THAT ARE
BUILT UPON OUR WORK.
NIH DOES NOT BRING NEW PRODUCTS
AND SERVICES TO MARKET.
AGENCY AND NATION RELY ON
PRODUCTIVE INDUSTRY PARTNERSHIPS
TO ACHIEVE THIS NEXT SLIDE.
THERE ARE UNIQUE BENEFITS FROM
NIH INDUSTRY PARTNERSHIPS.
CHANCE TO WORK TOGETHER TO
IMPROVE HUMAN HEALTH IS AT THE
TOP OF THE LIST.
TO THAT END AGENCY'S INTRAMURAL
PROGRAM DELIVERS TOP-NOTCH
SCIENCE AND INDUSTRY PARTNERS
HAVE OPPORTUNITY TO TURN THOSE
DISCOVERIES INTO NEW PRODUCTS
AND SERVICES AND INTRAMURAL
PROGRAM IS SPECIAL IN THAT IT
FOCUSES PRIMARILY ON WORK THAT
IS NOT OR WILL NOT BE DONE
ELSEWHERE.
ADVANCING TECHNOLOGIES CRYO-EM
AND DATA INITIATIVES AND
SUPERCOMPUTING FACILITIES
DEPARTMENT OF DEFENSE RARE
DISEASE CHARACTERIZATION AND
THERAPY DEVELOPMENT WORK AGO
CROSS THE WORLD AND ACROSS ALL
OF GOVERNMENT TO IDENTIFY
EMERGING INFECTIOUS DISEASE
THREATS TARGETING FOR PREVENTION
AND TO IDENTIFY A VERY FEW
ASPECTS OF NIH INTRAMURAL
RESEARCH THAT WE RELY ON.
THESE FRUITFUL INDEAFORS SPIN
OFF FUNDAMENTAL KNOWLEDGE THAT
APPLIES TO EVERYTHING IN HEALTH
AND BROAD PARTNERSHIP IS
ENCOURAGED AND PRODUCED MANY
SUCCESSES.
PUBLIC HEALTH CHALLENGE WE FACE
IS [INDISCERNIBLE] AND 10,000
DISEASES WE KNOW, 5% HAVE
TREATMENTS OR CURES AND
TYPICALLY TAKES MORE THAN 10
YEARS TO GO FROM AN IDEA FOR
DRUG FROM TESTING PROCESS TO
BRING IT TO MARKET AND LARGE
MAJORITY CANDIDATE THERAPIES
FAIL AND FOSTERING PUBLIC
PRIVATE COLLABORATION REMAINS
CRITICAL.
WE HAVE LOTS OF WORK TO DO.
NEXT SLIDE.
THE OPPORTUNITIES FOR NIH
INDUSTRY COLLABORATION TAKE A
VARIETY OF FORMS.
FOR EXAMPLE, NIH LICENSES ARE
PATENTS AND TECHNOLOGY AND
SUPPORTS COLLABORATIVE RESEARCH
THROUGH CREDICE AND AGENCY
SUPPORTS PUBLIC AND PRIVATE
PARTNERSHIPS AND EXAMPLE I LIKE
TO HIGHLIGHT ACCELERATING
MEDICINE PARTNERSHIPS IF YOU
DON'T KNOW AMP IS A PARTNERSHIP
AMONGST NIHFDA BIOPHARMA
COMPANIES AND NON-PROFIT
INSTITUTIONS AND GOAL IS TO
TRANSFORM CURRENT MODEL
DEVELOPING NEW DIAGNOSTICS AND
CURES AND PARTNER EXPERTISE AND
RESOURCES AIMING TO IMPROVE
UNDERSTANDING OF BIOLOGICAL
PATHWAYS AND TO VALIDATE
INFORMATION THAT COULD BE
RELEVANT TO DEVELOPING MULTIPLE
THERAPEUTICS.
WE ARE VERY APPRECIATIVE OF OUR
PRODUCTIVE RELATIONSHIP WITH THE
-- THE INSTITUTE FOR THE -- THE
FOUNDATION FOR THE NIH IN
ACHIEVING THE AMP ACTIVITIES.
NCAPS, NATIONAL CENTER FOR
ADVANCING TRANSLATIONAL SCIENCE
IS NIH INSTITUTION THAT IS
DEVOTE ENTIRELY TO TRANSLATIONAL
ACTIVITIES THAT IS ANOTHER HUB
OF THE PUBLIC AND PRIVATE
PARTNERSHIPS AND SUPPORT SMALL
BUSINESSES DEVELOPING TECHNOLOGY
WITH COMMERCIAL POTENTIAL
THROUGH SBIR PROGRAM.
WE HAVE INNOVATION
ENTREPRENEURSHIP TRAINING
PROGRAMS LIKE ICORE AND
INVESTIGATORS CAN ACCESS TOOLS
TO BRING TREATMENTS TO PATIENTS.
NIH DOESN'T LOOK FOR
COLLABORATIONS TO BE ARM'S
LENGTH TRANSACTIONS AND LOOK FOR
TRUE COLLABORATIONS AND
OPPORTUNITIES TO SHARE
SCIENTIFIC AND REGULATORY
EXPERTISE, SHARE MATERIALS AND
OTHER RESOURCES THAT WE CAN
CONTRIBUTE FROM THE PUBLIC END.
NIH HAS MANY TOOLS TO ENABLE
INDUSTRY PARTNERSHIPS MEETING
NEEDS OF ALL PARTIES ADVANCING
PUBLIC HEALTH.
AS A GOVERNMENT AGENCY, WE HAVE
TO OPERATE WITHIN CERTAIN
STATUTORY FRAMEWORKS AND HAVE A
DIVERSITY OF OPTIONS HELPING US
BE CREATIVE AND FLEXIBLE AND
ULTIMATELY DELIVER WIN, WIN, WIN
APPROACHES FOR THE AGENCY, OUR
PARTNERS, AND THE US TAXPAYER.
NEXT SLIDE.
I WOULD LIKE TO TOUCH ON
EXAMPLES OF SUCCESS STORIES AND
THIS SLIDE SHOWS JUST A FEW OF
THE VACCINES AND THERAPEUTIC
PRODUCTS ON THE MARKET THAT USE
SOME FORM OF TECHNOLOGY LICENSED
FROM NIH'S INTRAMURAL PROGRAM
INCLUDING VACCINES FOR COVID-19
AND RSV AND TREATMENT FOR
PROGERIA AND ABILITY TO CLIFR
COVID-19 VACCINES IN RECORD TIME
REFLECTS YEARS OF COLLABORATIVE
RESEARCH STARTING WELL BEFORE
THE PANDEMIC NIH INDUSTRY
ANDACDEMIA WORK TOGETHER ON
DEVELOPMENT OF MRNA VACCINES FOR
CORONAVIRUSES HPV VACCINE GUARDO
SIL AND [INDISCERNIBLE] NCI
SCIENTISTS ARE THOUGHT TO HAVE
AVERTED OVER 80,000 CASES AND
NEARLY 27,000 DEATHS FROM
CERVICAL CANCER IN THE UNITED
STATES ALONE PREVENTING NEARLY
600,000 FUTURE YEARS OF LIFE
LOST FROM 2008 TO 2019 A DECADE
ONLY.
-- INCLUDING LOXTERNA TO TREAT
RETINAL DISEASE AND
[INDISCERNIBLE] AND ROCTAVIAN TO
TREAT HEMOPHILIA RNA MORE TO
COME.
EXPERTISE THAT ARE 27 INSTITUTES
AND CENTERS THAT BRING TO
PARTNERSHIPS AND SPANS
DIAGNOSTICS DEVICES AND
APPLICATIONS FOR ARTIFICIAL
INTELLIGENCE AND POTENTIAL
APPLICATIONS INCLUDE MED TECH
WEARABLES AND TECHNOLOGY FOR
HEALTH AND INTRAMURAL PROGRAM
COLLABORATOR DEVELOPED A SLEEP
MONITORING DEVICE CONNECTING
COMPREHENSIVE SLEEP DATA WORKING
WITH NIH ON CLINICAL VALIDATION
OF THE DEVICE THIS COMPANY
SONOLOGY CAN SERVE AS A CASE
STUDY FOR THIS.
COLLABORATED WITH NIH IN 2017 TO
STUDY THE EFFECT OF DIET AND
ENVIRONMENT ON SLEEP.
IN 2019, THE COMPANY PARTNERED
ALSO WITH THE VA TO EVALUATE
VETERANS ACROSS THE COMPANY
HELPING TO REDUCE VA COST FOR
SLEEP TESTING OF ITS PATIENTS.
IN 2021 SONOLOGY WON A MED TECH
BREAKTHROUGH AWARD FOR ITS
DEVICE.
I ENCOURAGE YOU TO VISIT NIH'S
OFFICE FOR TECH TRANSFER
WEBSITE.
YOU CAN SEE THE URL HERE.
SITE LISTS NIH TECHNOLOGIES
AVAILABLE FOR LICENSING.
THESE NUMBERS ARE UPDATED DAILY.
YOU CAN SEE AREAS THAT THESE
CURRENTLY AVAILABLE TECHNOLOGIES
ENCOMPASS.
NEXT SLIDE.
INDUSTRY COLLABORATION OFFERS
UNIQUE BENEFITS AND AGENCY HAS
ROBUST IN-HOUSE EXPERTISE
HELPING COMPANIES COMMERCIALIZE
PRODUCTS EXPERTISE ON TECHNOLOGY
TRANSFER AND BUSINESS
DEVELOPMENT WITHIN INDUSTRY
EXPERIENCE AND ENTREPRENEURS IN
RESIDENCE AND INNOVATION-FOCUSED
TRAINING AND NETWORKING
OPPORTUNITIES.
IN-HOUSE EXPERTS CAN BE
PARTICULARLY VALUABLE FOR
START-UPS AND EMERGING COMPANIES
WE WORK WITH.
INDEED, 1/3 OF NIH'S MOST
SUCCESSFUL TECHNOLOGIES WERE
LICENSED TO EARLY-STAGE
COMPANIES.
NEXT SLIDE.
I SAID EARLIER THAT NIH IS
ALMOST 2S DO XEN INSTITUTE AND
CENTERS CONDUCTING INTRAMURAL
RESEARCH AND HUNDREDS OF LABS
CONDUCTING WIDE VARIETY OF
RESEARCH AND TRUE HEART OF
PROGRAM IS NIH CLINICAL CENTER
THAT IS A WORLD CLASS
INSTITUTION WHERE WE ROUTINELY
CONDUCT SMALL FIRST IN HUMAN
HIGH RISK CLINICAL TRIALS THAT
IS THE NATION'S ONLY
FULL-SERVICE HOSPITAL THAT IS
DEVOTED ENTIRELY TO BIOMEDICAL
RESEARCH.
NEXT SLIDE.
COLLABORATION HAS BENEFITS TO
INDUSTRY AND NIH.
AND NIH OFFERS ACCESS TO CUTTING
EDGE BASIC TRANSLATIONAL AND
CLINICAL RESEARCH AND ACCESS TO
OUR UNIQUE RESOURCES AND
EXPERTISE.
WE OFFER THE OPTION TO LICENSE
NIH TECHNOLOGIES FOR RESEARCH,
DEVELOPMENT AND
COMMERCIALIZATION AND PARTNERING
WITH INDUSTRY CAN GIVE US AT NIH
ACCESS TO ADDITIONAL INNOVATIONS
AND COMMERCIAL DEVELOPMENT
EXPERTISE.
MOST OF ALL CAN SERVE OUR
MANDATE TO GET MEDICAL SOLUTIONS
TO PATIENTS THAT NEED THEM.
WE VIEW INDUSTRY AS OUR PARTNERS
RATHER THAN REVENUE RESOURCES.
AND SEE COLLABORATION AS A TRUE
EXCHANGE OF IDEAS AND EFFORTS.
THIS HAS -- IT IS WHAT HAS
ALWAYS LED TO SUCCESS AND WE
DON'T LOOK FOR SPONSORED FOR
FEE-FOR-SERVICE RESEARCH.
ROYALTIES ARE USED TO
REINVESTORS AND REINVEST NOT
TOWARDS A PROFIT MARGIN FOR NIH.
WE ALSO HAVE A STATUTORY
PREFERENCE FOR WORKING WITH
SMALLER BUSINESSES.
ULTIMATELY, NIH SUPPORT CAN FREE
UP OTHER CAPITAL FOR BUSINESSES
AND EXTEND THE RUNWAY FOR
DEVELOPING PRODUCTS.
WHEN SUCCESSFUL PARTNERSHIPS
DELIVER PRODUCTS TO BENEFIT OF
PATIENTS, WE AT NIH WIN.
NEXT SLIDE.
I WOULD BE REMISED NOT TO
MENTION NATIONAL CENTER
ADVANCING TRANSLATIONAL
SCIENCES.
NCATS FOCUSES IMPROVING PROCESS
OF TURNING OBSERVATIONS INTO
INTERVENTIONS THAT IMPROVE
HEALTH.
CENTER CAN PROVIDE
COLLABORATORS WITH ACCESS TO
TRANSLATIONAL SCIENCE WORKFORCE
TO STATE OF THE ART FACILITIES
AND INFORMATICS CAPABILITIES.
STRONG COMMITMENT TO TEAM
SCIENCE AND ACCESS TO INNOVATIVE
PARTNERSHIPS.
N CATS INTRAMURAL PROGRAM AND
INDUSTRY PARTNERS LED TO
APPROVED TREATMENTS FOR MUSCULAR
DISTROPHY -- POMPEI DISEASE,
FRAGILE X SYNDROME, BETA
THAT'LLCEMIA AND MANY OTHER
CONDITIONS.
NEXT SLIDE.
FINALLY NIH AIMS FOR A BALANCED
RESEARCH PORTFOLIO AND POLICIES
PROMOTING HIGH RETURN ON
INVESTMENT FOR US TAXPAYERS.
RETURN ON INVESTMENT INCLUDES
THE LAUNCH OF NEW PRODUCTS BASED
ON NIH-FUNDED RESEARCH AND, OF
COURSE, IMPROVEMENTS IN HUMAN
HEALTH.
WE LOOK ALSO AT RETURN ON
INVESTMENT AND PUBLIC BENEFITS
THROUGH OTHER LENS THROUGH
ECONOMIC GROWTH, JOB CREATION
AND BOOSTING FOLLOW-ON
INNOVATION AND IN PAST DECADES
TECHNOLOGY LICENSED FROM NIH HAS
BEEN INTEGRATED IN OVER 1,000
PRODUCTS BROUGHT TO MARKET THAT
PRODUCE REPORTED SALES EXCEEDING
$130 BILLION AND THOSE SALES IN
TURN SUPPORTED AVERAGE OF 74,500
EMPLOYMENT POSITIONS IN THE PAST
20 YEARS ALONE AND NIH WORKS
TOWARDS CREATION SCORES OF NEW
START-UP COMPANIES THAT ARE
OUTSIDE OF CONTRIBUTIONS TO THE
ECONOMY.
A LOT OF THE AGENCY'S WORK IS
FOCUSED ALSO ON SPURRING FURTHER
RESEARCH AND DEVELOPMENT.
NIH-FUNDED RESEARCH CAN AND DOES
LEAD TO NEW RESEARCH TOOLS FOR
IDENTIFICATION OF NEW APPROACHES
TO APPROVING WAYS THAT WE DO
SCIENCE.
FINALLY, RESEARCH TOOLS LIKE
REAGENTS AND CELL LINES, FOR
EXAMPLE, THEY MAKE UP LARGEST
PORTION OF INTRAMURAL PATENT
LICENSING PORTFOLIO AND
INVESTIGATORS ARE CONTINUALLY
CONTRIBUTING TOOLS AND RESEARCH
THAT SCIENCE HAS DONE AND ARE
LICENSING TECHNOLOGY OUT SO
OTHERS CAN USE IT.
NEXT SLIDE.
YOU HAVE HEARD AND WILL CONTINUE
TO HEAR ME TALK ABOUT THE
CRITICAL IMPORTANCE OF EQUITY
AND ACCESS.
PROMOTING EQUITABLE ACCESS TO
THE PRODUCTS OF NIH RESEARCH IS
COMPLEX.
WITHOUT A DOUBT, AGENCY WANTS TO
SEE MORE INNOVATION AND WORKING
PARTNERSHIP TO DELIVER MORE
PRODUCTS AND SERVICES THAT PATI
WANT AND NEED AND DOING THAT
HAVING A MEANINGFUL POSITIVE
IMPACT ON HUMAN HEALTH.
WE NEED PROMISING INDUSTRY
PARTNERS WORKING HAND IN HAND
WITH NIH AND I HOPE THAT TODAY'S
MEETING IS VERY HELPFUL TO YOU
AND HOPE IT SEEKS YOU TO INQUIRE
MORE ABOUT THE POSSIBILITIES FOR
YOUR PARTICULAR COMPANIES ON
WHAT NIH MAY HAVE TO OFFER YOU.
I HOPE THAT IT ENGENDERS MANY
FOLLOWUP CONVERSATIONS LEADING
TO SUCCESSFUL PARTNERSHIPS.
THANK YOU SO MUCH.
>> OKAY. I'M DOING MY BEST TO
MANAGE TECHNOLOGY HERE.
SO, THERE ARE -- ONE QUESTION I
CAN SEE IN THE CHAT, FIRST OF
ALL, THANKS SO MUCH FOR THAT
SPECTACULAR TALK HIGHLIGHTING
ALL THINGS I LOVE ABOUT NIH.
>> YOU HAVE TO STOP RIGHT THERE
THOUGH.
YOU HAVE TO TELL EVERYONE WHAT
OUR ASSOCIATION IS.
>> MONICA AND I WERE COLLEGE
ROOMMATES.
>> WE WERE COLLEGE ROOMMATES AND
IT IS A WONDERFUL CHANCE FOR ME
TO BE ABLE TO WORK WITH AMY ON
THIS REALLY IMPORTANT EFFORT
ACROSS NIH AND REALLY FIRST
INTERACTION SINCE I CAME ON AS
NIH DIRECTOR AND PLEASED THAT
THIS IS WHERE BOTH CAREERS HAVE
BROUGHT US.
>> ME AS WELL.
I WAS TRYING IT KEEP IT
PROFESSIONAL BUT ON A PERSONAL
NOTE IT IS A PLEASURE TO
INTRODUCE MONICA TO ALL OF YOU
THROUGH THIS VENUE.
THERE IS A QUESTION IN THE CHAT.
CAN YOU COMMENT -- YOU PROBABLY
CAN'T.
CAN YOU COMMENT ON PROPOSED
CHANGES TO THE BAIL DOLE ACT AND
INFLUENCING FUTURE NIH INDUSTRY
COLLABORATIONS?
>> HAPPY TO TAKE THAT ON AND
HAPPY FOR THAT QUESTION.
THERE HAS BEEN INTEREST ON PART
OF THE LEGISLATORS IN MAKING
SURE THAT AMERICAN PEOPLE
RECEIVE A FAIR RETURN ON
INVESTMENT FOR THE PUBLIC DOLLAR
AND CONTRIBUTION TO BIOMEDICAL
RESEARCH.
WE KNOW AND YOU HEARD ME SAY TO
REALLY WANT TO SEE THE FRUITS OF
NIH-FUNDING TO TURN INTO
MARKETABLE PRODUCTS AND
SERVICES.
WE ARE BEING ASKED ACROSS ALL OF
THE FEDERAL GOVERNMENT AND NOT
JUST NIH BUT ALL OF GOVERNMENT
TO TAKE A LOOK AT
GOVERNMENT-INVESTMENT IN HEALTH
AND EVALUATE HOW WE CAN MAKE
SURE THAT THE -- THE PRODUCTS OF
PUBLIC FUNDING REALLY ARE
ACCESSIBLE AND AVAILABLE TO THE
AMERICAN PEOPLE.
THAT IS WHAT THE DISCUSSION IS
ABOUT.
IT WILL TAKE PLACE ON ALL OF
GOVERNMENT LEVEL.
FINALLY, ONE THING THAT I CAN'T
THINK OF A BETTER WAY TO MAKE
SURE NIH-FUNDING ACTUALLY
PRODUCES GOODS AND SERVICES
BENEFITING PEOPLE THAT ENGAGE
WITH INDUSTRY PARTNERS LIKE WE
CHAMPION WITH THIS MEETING
TODAY.
>> IF -- I ONLY SEE THAT ONE
QUESTION IN THE CHAT.
IF OTHER PEOPLE HAVE QUESTIONS,
PLEASE PUT THEM IN -- IT IS NOT
CALLED THE CHAT BUT QUESTIONS IN
THE -- IN THE FORMAT THAT YOU
HAVE.
I'M HAPPY TO POSE THEM TO
MONICA.
ONE QUESTION THAT IS: WILL
THERE BE SLIDES AVAILABLE?
THIS ENTIRE PROGRAM IS BEING
RECORDED AND WILL BE MADE
AVAILABLE.
IT IS AVAILABLE FOR THE NEXT
FOUR WEEKS FOR THE FORMAT YOU
HAVE.
YOU CAN GO INTO SWAP CARD AND BE
ABLE TO ACCESS ALL TALKS AND IN
TERMS WHETHER SPECIFIC SLIDES
FROM THE SESSION WILL BE
AVAILABLE, I WILL LEAVE TO
MONICA TO ANSWER THAT.
>> OH, YEAH. OF COURSE YOU CAN
SHARE THEM WIDELY.
>> THAT IS THE ANSWER TO THE
QUESTION.
I GUESS SINCE THERE ARE NO OTHER
QUESTIONS IN THE CHAT, MY
QUESTION TO YOU WOULD BE DO YOU
HAVE SUGGESTIONS?
I KNOW YOU HAVE LOTS OF
EXPERIENCE WITH WORKING WITH
INDUSTRY ON THE OUTSIDE.
NOW YOU ARE ON THE INSIDE.
HOW -- DO YOU HAVE SUGGESTIONS
FOR WAYS TO INCREASE OUR -- OUR
INTERACTION WITH INDUSTRY OR
SIMPLIFY THE -- THE INTERACTIONS
WE HAVE TO MAKE THEM -- MAKE
THEM HAPPEN MORE QUICKLY?
THAT IS ALWAYS AN ISSUE IN
GOVERNMENT.
>> YEAH. YOU KNOW, THE FIRST
REALLY KEY PRINCIPLE THAT I WILL
SAY THAT THE THING THAT MAKES
THINGS GOT QUICKEST IS WHEN
THERE IS VERY CLEAR THERE IS
MUTUAL BENEFIT AND FINDING SWEET
SPOTS WE ARE VERY CLEAR THIS SAY
COLLABORATION THAT THIS NEEDS TO
HAPPEN.
I THINK WE DON'T DO AS MANY OF
THOSE AS WE COULD.
I THINK THAT WE NEED TO REALLY
LOOK FOR MANY MORE OF THOSE.
THE -- WHEN THOSE HAPPEN, YOU
GET A REAL WILLINGNESS ON THE
PART OF BOTH OF THE INDUSTRY
PARTNER AND THE NIH PARTNER TO
REALLY, YOU KNOW, GET AGREEMENTS
SET AND READY TO GO.
I HAVE ALWAYS FOUND THAT WHEN
THERE IS VERY STRONG MUTUAL
SELF-INTEREST, THAT MAKES THINGS
FLOW VERY QUICKLY.
THAT DOESN'T MEAN WE SHOULDN'T
DIVE INTO MORE DIFFICULT ISSUES.
YOU KNOW, FOR EXAMPLE, THE -- I
HAVE HAD LOTS OF EXPERIENCE
WORKING WITH THE PHARMACEUTICAL
INDUSTRY LOOKING AT NEW USES OF
CURRENT MARKET -- CURRENTLY
MARKETED DRUGS.
YOU KNOW, THAT COULD BE A REALLY
CHALLENGING COLLABORATION SPACE.
ONE THING I WILL SAY IS WE ALL
KNOW THAT THOSE DRUGS AND
SERVICES BOTH ON THE PHARMAEND
AND ON NIH-END, WE HAVE A REAL
MANDATE TO MAKE SURE THAT
TESTING AND DATA THAT UNDERLIES
THEIR USE IS THE BROADEST
POSSIBLE AVAILABLE.
I CAN'T THINK OF A BETTER
PARTNER THAN NIH, WHICH IS VERY
BROAD AND CLINICAL TRIAL REACH
THAT GETS INTO DIVERSE
COMMUNITIES.
I CAN'T THINK OF A BETTER
PARTNER FOR INDUSTRY COMMUNITIES
WHEN THERE IS A NEED TO MAKE
SURE THAT DATA UNDERLYING USE OF
AGENTS IS, YOU KNOW, REPRESENTS
ALL PEOPLE.
THAT IS A GREAT CONTRIBUTION.
I THINK THAT IS ONE THAT SHOULD
BE CONSIDERED MORE.
FINALLY, WHEN WE SAY THAT WE
WANT PARTNERSHIPS, WE ARE ALSO
VERY WILLING TO LISTEN TO NEW
STRATEGIES THAT WILL WORK BEST
FOR INDUSTRY PARTNERS.
YOU KNOW, IT DOESN'T -- JUST
EVEN ENGAGING DOESN'T -- AND
PROCESS DOING THIS DOES NOT NEED
TO BE A ONE-WAY STREET.
WE WOULD WELCOME ANY
INTERACTIONS LEADING TO BETTER
RESULTS FOR BOTH OF US.
OOPS.
YOU ARE ON MUTE, AMY.
>> I KNOW. LIKE I SAID, I'M
HAVING TECHNOLOGICAL
DIFFICULTIES.
THERE IS ANOTHER QUESTION IN THE
CHAT.
DOES NIH HAVE A DATA BASE WITH
PATENTS TECHNOLOGIES AND
THERAPEUTIC AREAS?
COMPLICATED QUESTION.
>> CAN I JUMP IN?
>> YEAH. YOU GOT IT.
>> IN THE SWAP CARD IS A LINK
SAYING NIH TECHNOLOGIES LEADING
TO DATA BASE WITH COLLABORATION
AND OPPORTUNITIES.
YOU CAN CLICK ON THAT AND GET
ALL OF THE INFORMATION. SORRY
TO INTERRUPT.
>> NOT AN INTERRUPTION. THANK
YOU VERY MUCH, MICHAEL.
THERE IS ONE OTHER QUESTION IN
THE CHAT THAT IS RELATED TO
SPECIFIC TOPICS IN THE NIH
STRATEGIC PLAN FOR THE NEXT FIVE
YEARS THAT I'M NOT SURE THAT IS
IN THE -- REALLY IN THE SCOPE OF
-- OF THIS MEETING.
SO.
>> I WOULD BE HAPPY TO SPEAK TO
IT, IF YOU WANT.
I WILL TELL YOU WE HAVE TWO --
TWO AREAS OF FOCUS THAT WE ARE
ACTIVELY WORKING ON FROM THE
DIRECTOR'S OFFICE AND ACTIVELY
WORKING ACROSS ALL INSTITUTES
AND CENTERS AND ONE OF THEM IS
AN APPROACH TO REALLY
ORGANIZING, COORDINATING AND
USING OUR REALLY INCREDIBLE DATA
RESOURCES IN A MUCH BETTER WAY
TO PRODUCE MUCH BETTER RESULTS.
THIS IS A CENTRAL ACTIVITY
WHERE YOU KNOW YOU KNOW THERE IS
AN NIH-DATA SHARING POLICY
ASKING EVERY ONE OF THE GRANTEES
TO DESCRIBE HOW DATA THEY
GENERATED COULD BE REUSED BY
BROADER RESEARCH COMMUNITY AND
WE ARE FOCUSED ON FINDING WAYS
TO IMPLEMENT THAT IN WAYS THAT
DON'T JUST ALLOW DATA TO BE
SHARED BUT TO BE USED.
SO THE REASON THAT I ALSO BRING
THIS TO THIS PARTICULAR
COMMUNITY IS ACCESS TO DATA THAT
COULD BE SHARED THROUGHOUT THE
ENTIRE BIOMEDICAL RESEARCH
ENVIRONMENT THAT IS ONE OF THE
KEY GOALS.
THAT MEANS THAT OUR PARTNERS --
RELATIONSHIPS WITH INDUSTRY
PARTNERS WILL ALSO LOOK TO THAT
AS A -- AS A STANDARD FOR HOW WE
WOULD APPROACH OUR WORK.
NO THE THAT WE WOULD WANT TO,
YOU KNOW, OF COURSE WE CAN PUT
ADEQUATE PROTECTIONS ON THINGS
LIKE INTELLECTUAL PROPERTY BUT
ALSO WANT AN ENVIRONMENT WHERE
DATA FROM CLINICAL CARE
ENVIRONMENT COULD BE ACTIVELY
SHARED.
THAT IS ONE INITIATIVE.
ANOTHER REALLY SEEKS AT A LEVEL
TO HAVE MORE IMPACT AT DELIVERY
OF CARE INDIVIDUAL PATIENT
TREATMENT LEVEL.
WE ARE LOOKING TO FOCUS
ACTIVITIES ON ACHIEVING THE LAST
MILE OF BEING ABLE TO GET
PROMISING NEW TREATMENT TESTED
IN PATIENT POPULATIONS THROUGH
MORE AND MORE AGILE AND NIMBLE
AND EFFECTIVE CLINICAL TRIAL
APPROACHES AND GET MORE
PROMISING TREATMENT OPTIONS AND
TECHNOLOGIES ACTUALLY DELIVERED
TO PEOPLE THAT NEED THEM.
I THINK THESE BROAD INITIATIVES
WOULD HAVE GREAT IMPACT TO DO
THEM IN COLLABORATION WITH
INDUSTRY PARTNERS.
I THINK YOU ARE BACK ON MUTED
AGA
AGAIN.
>> I'M TRYING TO AVOID EXTRA
NOISE WHEN YOU ARE SPEAKING AND
ACCELERATED PROGRAM COORDINATED
WITH FDA AND THEY FILED PATENTS
FOR THERAPEUTICS AND HAVE
COLLABORATIVE SCIENTISTS ON
OUTSIDE AND FDA-GUIDED LAB.
>> I DON'T KNOW WHETHER THE
QUESTION IS SPEAKING OF
ACCELERATED MEDICINES
PARTNERSHIP AND POINTING TO BEST
INFORMATION THAT IS FOUND ON
FOUNDATION OF NIH WEBSITE AND
VARIOUS ACTIVITIES CONDUCTED
THERE OR WHETHER THAT SOUNDS
LIKE THIS IS WHAT THIS IS FOR.
THERE IS LABORATORY WORK
INVOLVED AND YOU KNOW THAT NIH
CONTRIBUTED SIGNIFICANTLY TO
ACCELERATED APPROVAL OF
THERAPEUTICS THAT ARE OUT THERE
AND ONE OF THE YOU KNOW SWEET
SPOTS FOR US HAS BEEN TO
REEMPHASIZE RARE DISEASES WHERE
IT IS REALLY HARD AND CAN BE
REALLY DIFFICULT TO GATHER PASH
YENLTS TOGETHER TO PRODUCE DATA
REQUIRED FOR ACCELERATED
APPROVAL FOR A DRUG AND CLINICAL
CENTER REALLY SHINES AND NETWORK
THAT IS RUN BY THE NCI.
ALSO FOR -- FOR, AGAIN,
POPULATIONS, SPECIAL INDICATIONS
FOR POPULATIONS THAT HAVE NOT
TRADITIONALLY BEEN STUDIED ALONE
BY INDUSTRY AND ELDERLY PATIENTS
WITH COMORBID CONDITIONS OR
PATIENTS LOCATED IN VERY DIVERSE
COMMUNITIES.
>> I THINK MAYBE OUR LAST
QUESTION OR NOT.
I WILL STATE THE LAST QUESTION
OR ONE I WOULD POSE FOR THE LAST
QUESTION AND QUESTION CAME IN
WHERE DOES ONE GO IN NIH FOR
COLLABORATIONS AND PROJECTS
RELATED TO TELEHEALTH AND
BIOCOMPUTING?
>> BIOCOMPUTING IS ONE I DON'T
UNDERSTAND.
I'LL TELL YOU A PLACE TO START,
PERHAPS, CERTAINLY CALLING THE
MAIN OFFICE AND I THINK MUCOLA
WILL HAVE A LINK FOR YOU TO ASK
QUESTIONS LIKE THIS.
>> RIGHT.
>> WHERE ARE QUESTIONS.
>> I WILL SAY SOUNDS LIKE
INSTITUTE YOU MIGHT WANT TO LOOK
AT IS BOB INSTITUTE OF
BIOTECHNOLOGY THAT HAS AN
INCREDIBLE PROGRAM FOLK YUTSED
ON AND GREAT KNOWLEDGE AND
PARTNERSHIP IN INDUSTRY AND THIS
MIGHT BE FOCUS FOR THE
QUESTIONER AND IF THAT IS THE
LAST QUESTION I WILL END BY
THANKING YOU FOR BEING HERE.
WE ARE EAGER TO WORK WITH YOU
AND THANK YOU TO DR. CLION FOR
INVITING ME TO THIS TALK AND
LOOK FORWARD TO A PRODUCTIVE
MEETING FOR YOU ALL.
THANKS SO MUCH.
>> THANK YOU. LAST MESSAGE IN
THE CHAT IS THANK YOU. WE ARE
GRATEFUL FOR EVERYTHING THAT THE
NIH DOES.
>> THANK YOU. TAKE CARE. HAVE
A GREAT MEETING.
>> THANK YOU. BYE, BYE.
>> HELLO.
>> HELLO. I WILL START SHARING
MY SCREEN.
WELCOME, EVERYBODY. I'M JOHN
TIS DALE AT THE NATIONAL HEART,
LUNG, AND BLOOD INSTITUTE AND
JOINED BY MY COLLEAGUE WHO IS A
CHIEF SCIENTIFIC OFFICER WHO WAS
PREVIOUSLY AT BLUEBERG. WE
WORKED TOGETHER FOR A DECADE AND
PASSING IT TO HER TO SET THE
STAGE. WE WILL GO BACK AND
FORTH AS WE DID FOR THAT DECADE
TO SOLVE PROBLEMS AS THEY CAME
ALONG TO THIS JOURNEY DEVELOPING
FIRST GENE THERAPY OF THE FIRST
MOLECULAR DISEASE.
>> THANK YOU. WHAT A JOURNEY IT
W
WAS.
>> WHAT WE WILL TRY TO
ACCOMPLISH FOR YOU IN THE NEXT
30 MINUTES IS SICKLE CELL
DISEASE AND SPECIFICALLY
DEVELOPMENT OF GENE THERAPY FOR
SICKLE CELL DISEASE THAT IS
TESTED IN PATIENTS IN STUDY
HTB206 THAT YOU WILL COME TO
FIND OUT DIVIDED INTO THREE
GROUPS.
WE WILL FOCUS ORIGINALLY AT
FIRST ON GROUP A THAT IS
ORIGINAL PATIENTS TREATED AS
PART OF THE STUDY.
WE WILL DIVE INTO SOME OF THE
NUANCES IN THE GROUP A PATIENTS
RELATED TO EFFICACY OUTCOMES AN
TALK BEING PROTOCOL REFINEMENTS
INSTITUTED TO IMPROVE EFFICACY
AND WHAT LED TO FDA APPROVAL FOR
GENE THERAPY FOR SICKLE CELL
DISEASE.
TAKE IT AWAY WITH SICKLE CELL
DISEASE AND TREATMENTS.
>> THANK YOU, MELISSA.
I HOPE TO CONVINCE YOU IT IS
TIME TO ENVISIONED BEFORELY
AVAILABLE CURES AND UNDERLYING
OF THE S ON PURPOSE.
WE DIDN'T INVENT ONLY WAY TO DO
THIS.
WE ARE HAPPY THERE ARE NOW
MULTIPLE WAYS TO THINK ABOUT
CURING THIS DISEASE.
IT IS ABOUT TIME.
WE HAVE KNOWN ABOUT THE DISEASE
FOR MORE THAN A CENTURY THAT WAS
ORIGINALLY DESCRIBED BY A
MEDICAL STUDENT IN WEST INDIES
AND PAIN AND PROLIVERAL BLOOD
FILM AND RED CELLS HAD A SICKLE
SHAPE AND FIRST MOLECULAR
DISEASE DESCRIBED BY LINUS
PAULING AND COLLEAGUES IN 1949
BASED ON DIFFERENCE BETWEEN
HEMOGLOBIN MOLECULE IN PATIENTS
AND NORMALS.
YOU KNOW, LINUS WENT ON TO GET A
NOBLE PRICE IN PROTEIN
BIOCHEMISTRY WORK BASED IN PART
ON FINDINGS THAT RESULTS FROM
SINGLE SUBSTITUTION OF POSITION
6 OF BETA GLOBIN CHAIN PRODUCING
ABNORMAL HEMOGLOBIN IN RED CELLS
PRONE TO DEOXYGENATION
PROPENSITY TO HAVE A ROCK HARD
HEMOGLOBIN INSIDE THAT OBSTRUCTS
CIRCULATION MAKING RED CELLS
FRAGILE CAUSING SEVERE ANEMIA
AND FREQUENT PAIN PARTS OF BODY
ARE DEPRIVED OF CIRCULATION AND
ORGAN AND STROKE IN KIDS AND
EARLY MORTALITY AND RARE IN
US100,000 INDIVIDUALS IN THE US
WITH THIS DISEASE THAT IS NOT
RARE IN THE WORLD AND
CONCENTRATED IN AREAS THERE IS
MALARIA.
THIS EVOLVED AS AN EVOLUTIONARY
PROTECTION AGAINST MALARIA WITH
SINGLE COMPUTATION OF THE GENE
AND MANY MILLIONS ACROSS SUBIS A
HAIRANT AFRICA AND INDIA AND
LIMITED AND MOSTLY SUPPORTED AND
WE NEED CLINICAL TRIALS TO TEST
CURATIVE APPROACHES.
I MADE THIS SLIDE A FEW YEARS
AGO WITH COLLEAGUES HERE AT NIH
DESCRIBING CURRENT THERAPIES
WITH DRUGS GETTING RED CELL
THROUGH CIRCULATION BEFORE IT
SICKLES AND HYDROXY RHEA.
IT PREVENTS SICKLING AND
ALLOGENEIC TRANSPLANTATION
TAKING BONE MARROW STEM CELLS
FROM SOMEONE ELSE THAT IS FROM A
BROTHER OR SISTER INTO
TRANSPLANT AND GENE EDITING
THERAPY AND VIRAL VECTORS WE
WILL TALK ABOUT AND CRISPR WE
WON'T HAVE TIME TO TALK ABOUT
AND HAVING TO MOVE THIS DOTTED
LINE OVER NOW THEY NOW RECENTLY
IN DECEMBER HAVE BEEN FDA
APPROVED AND WE AND OTHERS ARE
WORKING ON WAYS TO DEVELOP IN
VIVO GENE THERAPY AND MELISSA
DIDN'T TELL US WHAT SHE IS UP TO
PROBABLY PART OF THIS IN HER
PIPELINE -- YOU CAN CURE KIDS
WITH SICKLE CELL DISEASE WITH
STANDARD BONE MARROW TRANSPLANT
USING IMMUNOOBLATED -- AND IT
WORKS AND WORKS MOEFT OF THE
TIME AND WE HAVE BETTER OVER THE
YEARS TO MODERN DAY KID WITH SIB
BLING CAN EXPECT 95% CHANCE OF
BEING CURED AND DEAL MOSTLY WITH
ADULT PATIENTS WE COULDN'T DO
WITH ADULT PATIENTS. ORGAN
[INDISCERNIBLE] THAT IS
ACCUMULATED OVER THE YEARS -- WE
ASKED A QUESTION WHETHER WE
COULD DO IT WITHOUT OBLATING AND
NO CHEMOTHERAPY AND ANTIBODY FOR
LYMPHOCYTES AND ORAL DRUG TO
PROMOTE TOLERANCE THAT WORKED IN
9 OF FIRST 10 AND RESOLUTION OF
DISEASE AND NO ANEMIA OR
[INDISCERNIBLE] DISEASE THAT IS
A COMMON AND CAN BE DEADLY
COMPLICATION OF A TRANSPLANT
GOING ON EXTENDING IT TO 30
PATIENTS WITH RULES FOR WEENING
IMMUNOSUPPRESSION AND WEENED
MORE THAN 80% OF PATIENTS AND
IMMUNOSUPPRESSION IN FIRST YEAR
AND SET STAGE FOR US LAUNCHING
STUDIES INTO TRANCE PLANTS WITH
COURT ANY IN THE INTRAMURAL
PROGRAM RUNNING EFFORTS -- THIS
IS MORE OR LESS MYELOID SERIES
COMING FROM DONOR AROUND 50%
FROM LYMPHOID CELLS AND 80% FROM
MYELOID CELLS RESOLUTION OF
ANEMIA AND HEMOLYTIC PARAMETERS.
WE NEED TO KNOW WHEN WE REACHED
THAT THRESHOLD AND BY TIME
TAUGHT ABOUT ANSWERING QUESTION
WE HAD 100 PATIENTS TRANCE
PLANTED IN INTRAMURAL PROGRAM
AND 67 WITH LONG-TIME FOLLOW UP
MONITORING RED CELLS COMING FROM
DONOR AND PERCENTAGE THEY WERE
AND PERCENTAGE OF MYELOID CELLS
AND NEUTROPHILS SHORT HALF LIFE
AND REFLECT STEM CELL
COMPARTMENT CAME FROM DONOR AND
ALL DATA AND AT SAME TIME THREE
PATIENTS DESPITE HAVING
RESOLUTION OF DISEASE EARLY HAD
DECLINING LEVELS OF DONOR
MYELOID CHIMERISM AND
NEUTROPHILS COMING FROM DONOR
AND ALL PATIENTS HAD SICKLE
TRAIT DONORS AND THEY WERE LIKE
CARRIER STATE WHEN MYELOID
TIMERS FELL BELOW 20% WE SAW
UPTICK IN HEMOGLOBIN S SHOWING
RETURN OF DISEASE.
THIS TAUGHT US A VERY IMPORTANT
LESSON.
WE WONDERED WHY IT IS 1 IN 5
HEMOPOETIC STEM CELLS MAKE ALL
RED CELLS FROM DONOR IN
CIRCULATION AND THOUGHT IT WAS
FROM DIFFERENCES IN LIFESPAN OF
THE RED CELL COMING FROM DONOR
VERSUS RED CELLS FROM THE
RECIPIENT KNOWING SICKLE CELLS
RED CELLS HAVE A SHORT HALF LIFE
AND DEVELOPING MATHEMATICAL
MODEL TO PREDICT ONLY BASED ON
LIFESPAN WHAT WOULD HAPPEN WITH
FALLING DONOR MYELOID CHIMERISM
LEVELS?
IN RED ON THE RIGHT HAND PANEL
YOU SEE OBSERVED DATA PRACH
FALLING DONOR MYELOID CHIMERISM
COMING FROM 60% DOWN TO 10% OVER
THE YEARS POST TRANSPLANT THAT
IS TIME AND YEARS.
YOU CAN SEE WHEN IT REACHES 20%
DONOR MYELOID CHIMERISM LEVEL IN
RED MEASURED HEMOGLOBIN S RISES
IF OF THE% AND WE SAW RETURN OF
SYMPTOMS TAKING INTO ACCOUNT
LIFESPAN AND PREDICTED RESULTS
IN BLUE HERE ARE SUPERIMPOSABLE
ON OBSERVED CELLS AND THAT IS
BECAUSE THEY HAD A LONGER
LIFESPAN AND THEY TAKE OVER IN
PERIPHERY AND WE SAID CAN WE DO
GENE EDITION AND DO SAME THING
GETTING 1 IN 5 HEMOPOETIC STEM
CELLS MAKING DISEASE GO AWAY.
WE USE VIRAL VECTORS PACKAGING
BETA DMROBIN GENE AND REGULATORY
ELEMENTS ACTING LIKE TROEJEN
HORSE MAKING GENETIC
MODIFICATION EFFICIENT ENOUGH TO
PRODUCE NORMAL HEMOGLOBIN AND
CARTOON MAKES IT SIMPLE.
IT HAS BEEN A COMPLICATED
PROCESS AND STRINGENT
REQUIREMENTS FROM HEMOGLOBIN
SICKLE CELL AND EXPRESSION OF
THE THERAPEUTIC GENE AND LEAVING
HEMOGLOBIN S IN PLACE IN GENOME
HAS TO BE ENOUGH TO OVERCOME
THAT. THAT WAS REALLY DIFFICULT
TO DO OVER THE YEARS AND SOME
EARLIER VECTORS I TALKED ABOUT
IN ONE OF THE SESSIONS THIS
MORNING COULD NOT PACKAGE
COMPLEX PAY LOADS AND BECAME
FEASIBLE SWITCHING TO HIV-1 AS
BACKBONE TO VECTORS SHOWN BY
MICHELE'S GROUP AND LATER FEL
EEP'S GROUP WHO TESTED THIS IN
TWO TRANSGENIC MODELS OF SICKLE
CELL DISEASE AND VECTOR SERVED
AS BACKBONE FOR VECTOR USED IN
CLINICAL TRIAL.
THIS IS HTP26 STUDY.
WE COULDN'T MOBILIZE WITH THEN
ONLY REAGENT FOR MOBILIZATION
GCSF BECAUSE OF COMPLICATIONS
WITH PATIENTS WITH SICKLE CELL
DISEASE AND USED ORIGINAL
MANUFACTURING PROCESS PRODUCING
TRANSDUCTION EFFICIENCIES WE
THOUGHT WERE SUFFICIENT AND
LEARNED LATER WERE NOT. THESE
CELLS GO TO CENTRALIZED
MANUFACTURING FACILITY TRANCE
DEUCED WITH VECTOR FROZEN IN ALL
LOT RELEASE CRITERIA ARE MET
PATIENTS COME BACK AND
CONDITIONING AND GET RID OF ALL
MARROW --
THESE RESULTS HAVE BEEN
PUBLISHED AND WILL TAKE YOU TRU
SOME OF THE OBSERVATIONS THAT
HAD US BACK AND FORTH ON MANY
ZOOM MEETINGS OVER THE YEARS AND
VISITS BACK AND FORTH THAT IS.
VECTOR COPY NUMBER WAS 1 AND
WHEN WE MEASURED AFTER
RECONSTITUTION IN PATIENT VECTOR
COPY NUMBER DROPPED 10 FOLD TO
.1.
10% IS NOT ENOUGH WE HAD LEVELS
OF HEMOGLOBIN COMING FROM VECTOR
SHOWN IN PINK AND TOTAL
HEMOGLOBIN DEPICTED IN CRUMBS
BEING AS HIGH AS 20% IN ONE
PATIENT AND MORE LIKE 5% AS
OVERALL MEAN AND WE RECEIVED
[INDISCERNIBLE] IN TWO PATIENTS
IN GROUP A AND PASSING TO
MELISSA TO TELL YOU HOW WE WENT
ABOUT SORTING THIS OUT SKWL
THANK YOU, JOHN.
HOWEVER, IN 2018 WE HAD A
PATIENT DIAGNOSED WITH
[INDISCERNIBLE] SYNDROME THREE
YEARS FOLLOWING TREATMENT WITH
WHAT AT TIME WAS CALLED WENTY
GLOBIN INVESTIGATIONAL PRODUCT
THAT BECAME PART OF FOUNDATIONAL
DATA USED FOR APPROVAL.
NOW THIS PATIENT JOHN IS SUPER
FAMILIAR WITH AND ONE OF HIS
PATIENTS ABLE TO RELATIVELY
QUICKLY DETERMINE THAT BLAST
CELLS IN MARROW AND BLOOD OF
THIS PATIENT DIDN'T INCLUDE
ANTIVIRAL INSERTION INSIGHTS
MEANING THERE WAS NO MECHANISTIC
RATIONALE TO SUPPORT PRESENCE OF
VECTOR COULD HAVE CAUSED THE
CAN
CANCER.
WE WERE, YOU KNOW, HAPPY WITH
THE OUTCOME THAT IT DIDN'T
APPEAR TO BE VECTOR RELATED.
AND WE WERE HOPEFUL THIS WOULD
BE A ONE-OFF EVENT. I MENTIONED
EARLIER JOHN AND I HAVE SEEN
ABOUT EVERY POTENTIALLY
THEORETICAL THING THAT COULD GO
WRONG IN A CELL AND GENE THERAPY
SETTING.
SECOND CASE OCCURRING IN 2021
WITH A COMPLICATED CASE.
2021 PATIENT DIAGNOSED WITH
ACUTE MYELOID LEUKEMIA 5.5 YEARS
AND WERE ABLE TO ENRICH CELLS IN
PATIENT.
IN THIS PARTICULAR CASE, THERE
WAS AN INSERTION IN THE BLAST
CELLS OF THE PATIENT THAT LED TO
THE QUESTION WAS THE INSERTION
OF THE LENTIVIRAL VECTOR CAUSING
DEVELOPMENT OF AML OF THIS
PATIENT OR IT IS HAPPENSTANCE
AND THIS PATIENT DEVELOPED AML
FOR OTHER REASONS THAT HAPPENS
THIS IS CELL THAT IS INITIATING
CELL THAT CONTAINED VECTOR
INSERTION AND THERE FOR COULD BE
CONSIDERED PASSENGER MUTATION
AND TONS OF WORK WENT INTO THIS
WALKING THROUGH IT BRAEFLY AND
GOING TO NEXT SLIDE YOU CAN LOOK
AT DETAILS HOW WE APPROACH THIS
CASE.
SO WHAT WE DETERMINED CRITICAL
COMING UP WITH LIST OF RATIONALE
WHAT WE WOULD NEED TO SAY
WHETHER OR NOT WITH A HIGH
PROBABILITY WHETHER OR NOT THE
VECTOR COULD BE POTENTIALLY A
DRIVER OR PASSENGER MUTATION WAS
A CHECKLIST ESSENTIALLY OF
ATTRIBUTES.
WE KNEW THERE WAS A VECTOR
PRESENT IN TUMOR CELLS AND THERE
WERE DRIVER MUTATIONS THAT
EXISTED IN TUMOR CELLS.
WASN'T ONLY THING THAT WAS
UNUSUAL ABOUT CELLS WAS PRESENCE
OF VECTOR THAT CONTAINED
CLASSICAL DRIVER MUTATIONS OF
AML AS WELL.
THE OTHER QUESTION WE HAD WAS IF
THE VECTOR IS CAUSATIVE OF
LEUKEMIA WE SHOULD BE ABLE TO
DETECT MOLECULAR OR BIOLOGICAL
CHANGE DRIVING EMERGENCE OF
DISEASE AND QUESTIONS WE ASKED
INCLUDE AND I WON'T READ THROUGH
THIS LIST FOR SAKE OF TIME.
INCLUDING IS VECTOR
TRANSCRIPTION ALLEY ACTIVE IN
CELLS AND TURNING ON OR OFF THE
GENE THAT IS KNOWN PROTOONCO
GENE SUPPRESSOR RESPECTIVELY TO
BIOLOGICALLY LINK PRESENCE OF
VECTOR TO POTENTIAL EMERGENCE OF
DISEASE?
LONG STORY SHORT AND GOING
THROUGH DETAILS HERE BUT ANSWER
IS NO IT DIDN'T APPEAR THAT
VECTOR WAS ACTUALLY INFLUENCING
ANY IN THESE CELLS THAT APPEARS
TO BE A PASSENGER MUTATION.
SO FIRST AND FOREMOST
INTEGRATION WAS MAPPED AND WE
WERE ABLE TO DETERMINE THAT
THERE WAS A SINGLE INSERTION IN
THE BLAST CELLS IN A GENE CALLED
VAM P 4.
AND BEFORE THIS CASE I DON'T
THINK I HEARD OF THE GENE VAM P
4.
IT DOESN'T SEEM TO BE A VERY
INTERESTING GENE THAT IS
CERTAINLY NOT A PROTOON CO GENE
NOR A TUMOR SUPPRESSOR AND IN
ADDITION ASSERTION IS IN REGION
4 THAT IS NOT UNUSUAL.
LENTIVIRAL VECTORS HAVE
INTEGRATION WITH EVENTS AND
REGIONS WITH DNA THAT IS ALIGNED
WITH PATTERN OF THIS ASSERTION
AND PRESUMED UNLIKELY TO HAVE
ANY SORT OF POTENTIAL IMPACT ON
TRANSCRIPTION ON GENE ITSELF AND
WE DID THAT WORK TO SHOW THAT.
IF WE GO TO NEXT SLIDE, PLEASE.
WE DID RNA-SEQUENCING TO
DETERMINE IF THERE WAS
EXPRESSION CHANGES SPECIFICALLY
WITHIN THE VAM P 4 GENE AND
LOCUST AND BROADLY FROM A
CHROMOSOME PERSPECTIVE AND
SEEING ANYTHING ATTRIBUTED TO
PRESENCE OF VECTOR ASSERTION.
AND YOU ARE SEEING HERE YOU KNOW
ESSENTIALLY WE ARE LOOKING AT
DISTRIBUTION OF TRANSCRIPTION OF
GENES KIND OF BROADLY GOING LEFT
TO RIGHT AND ACROSS ALL OF
CHROMOSOME 1 IN THE CENTER PANEL
AND LOOKING AT GENES CENTERED IN
10 MEGA BASINS OF THE SITE AND
AS FRANKLY AS A POSITIVE CONTROL
EXAMPLE ON RIGHT-HAND SIDE HERE
WE HAVE CHROMOSOME 7 AND KNOW
BLAST CELLS HERE HAVE MONOSOMY 7
THERE IS LEFTWARD SHIFT OF
EXPRESSION THAT IS CONSISTENT
WITH LOSS OF ENTIRE CHROMOSOME 7
AND SEE FOR THE OTHERS THAT
EVERYTHING IS CENTERED AROUND
ONE AND THIS IS NORMALIZED TO
BLAST DEPLETED CELL POPULATIONS
IN THIS PATIENT THAT YOU CAN SEE
THERE IS NO CHANGES HERE IN
TRANSCRIPTIONAL PROFILE
ATTRIBUTED TO PRESENCE OF VECTOR
AND IN FACT THIS PARTICULAR GENE
IS ACTUALLY PRETTY WELL INSERTED
WE HAVE TONS OF PATIENTS WHO
HAVE INSERTION SITES IN VAM P 4
ACROSS OUR [INDISCERNIBLE]
PROGRAM AND THAT'LLCEMIA
PROGRAM. YOU ARE LOOKING AT IN
GRAPH TRIANGLE INSERTION SITE
FOUND IN PATIENT AND THERE IS --
AGAIN, VERY CONSISTENT WITH
INTEGRATION PATTERN OF ANTIVIRAL
VECTOR. YOU SEE OTHER PATIENTS
WITH INSERTION CITES IN SIMILAR
REGION OF INSERTION CITES AND
PATIENTS DON'T HAVE EVIDENCE OF
HEMATOLOGIOGICAL MALIGNANCY B
NOR DO THEY HAVE EVIDENCE OF
EXPANDED CLONE THAT IS
NONMALIGNANT BUT COULD SUGGEST
THERE IS SOME SORT OF ADVANTAGE
TO HAVING THE INSERTION IN THIS
VAM P 4 GENE THAT IS LEADING TO
EXPANSION OF A CELL CONTAINING
THAT INSERTION.
WE SAW NONE OF THAT.
THAT IS GREAT AND CONSIST YENLT,
AGAIN, WITH A PASSENGER MUTATION
AND SO JUST TO KIND OF
SUMMARIZE, AGAIN, FOR THE SAKE
OF TIME, WE WILL REALLY
HIGH-LEVEL THIS AND SHOW THAT
ABUNDANCE OF EVIDENCE SUPPORTED
THIS IS UNLIKELY TO BE A DRIVER
INSERTION EVENT.
YOU KNOW?
IT IS ACTUALLY SUBSTANTIALLY
MORE LIKELY THIS HAPPENS TO BE
THE CASE THAT THIS PATIENT
DEVELOPS LEUKEMIA AND HAPPENED
THAT THE CELL THAT WAS
INITIATING CELL CONTAINED A
VECTOR INSERTION AND QUESTION
WAS, WELL, HOW DOES THAT HAPPEN?
WHAT -- IF -- IF THE PRESENCE OF
THE VECTOR IS NOT CAUSATIVE OF
LEUKEMIA, WHAT IS DRIVING IT?
WE HAVE TWO PATIENTS NOW BOTH
TREATED IN GROUP A WITH ORIGINAL
MANUFACTURING PROCESS WHERE WE
HAD RELATIVELY LOW TRANSDUCTION
EFFICIENCIES OF DRUG PRODUCTS
WHO HAVE NOW DEVELOPED
HEMOTOLOGIC MALIGNANCY AND BOTH
HAD SIMILAR DRIVER MUTATIONS AND
MUTE ARGSS IN [INDISCERNIBLE]
AND MONOSOMY 7 VERY CLEAR
MUTATIONS.
AND WE WERE CURIOUS TO
UNDERSTAND WHAT COULD
BEINISHATING EVENT HERE?
YOU KNOW, EVERYBODY ON OUR
TRIALS ARE GETTING OT OL GAUS
GENE THERAPY AND THERE ARE LOTS
OF PIECES HERE THAT SEEM TO
SUGGEST IN EVENT WHERE YOU HAVE
OT OL GAUS TRANSPLANT RECEIVING
A DRUG PRODUCT WITH LOW
TRANSDUCTION EFFICIENCY LEADING
TO LACK OF CLINICAL BENEFIT
WHICH IS WHAT PATIENTS IN GROUP
A SAW THAT YOU ARE LIKELY
EXACERBATING SOME HEMOTOLOGIC
IMPACTS OF THE DISEASE.
YOU HAVE STRESSORS THAT ARE
OCCURRING BECAUSE OF CHRONIC
ANEMIA ASSOCIATED WITH SICKLE
CELL DISEASE THAT IS LIKELY
EXACERBATED IN CONTEXT OF
RECEIVING A DRUG PRODUCT WITH A
RELATIVELY LOW CELL DOSE IN
TRANSACTION EFFICIENCY AND DOG
THIS IN REVERSE ORDER AND
REFINEMENTS IN ORDER TO
MALIGNANCIES THAT I DESCRIBED
THAT WE BELIEVE STRONG WILL I
THAT PROTOCOL REFINEMENTS ARE
LIKELY GOING TO MITIGATE DISEASE
IN OUR KIND OF FURTHER COHORTS
OF PATIENTS.
GOING TO THE NEXT ONE.
I CAN'T REMEMBER IF WE DECIDED I
WAS OR YOU WERE GOING TO DO
THIS.
>> YOU GO AHEAD, YUP.
>> OKAY. SORRY.
[LAUGHING].
SO, ONE INTERESTING THING WE
NOTICED ABOUT THESE CELLS COMING
FROM BONE MARROW OF SICKLE CELL
DISEASE PATIENTS THEY DON'T LOOK
LIKE IT COMING FROM BONE MARROW
OF HEALTHY INDIVIDUALS THAT YOU
SEE DEPICTED IN GRAPH AND
HISTOGRAM THERE IS POPULATIONS
OF CD4 POSITIVE CELLS WITHIN
BONE MARROW SICKLE CELL DISEASE
IN THAT LOWER HISTOGRAM AND
POPULATION TOWARDS LEFT AND
[INDISCERNIBLE] POPULATION
TOWARDS RIGHT AND WE CALL CD4
HIGH AND IN HEALTHY INDIVIDUALS
DOMINANT POPULATION IS AT CD4
HIGH POPULATION AND IN SICKLE
CELL INDIVIDUALS, THIS COULD BE
UP TO 60% OF BONE MARROW IS
ACTUALLY CD34 DIM AND IN
COLLABORATION WITH JOHN AND
ALEXIS WHO IS DEPICTED AT BOTTOM
OF THE SLIDE WE DID WORK LOOKING
INTO CD34 DIM CELLS TO TRY TO
FIGURE OUT WHAT THEY ARE ARE
THEY STEM CELLS CONTRIBUTING TO
LONG-TERM HEMOPOETICESIS AND
CONTAMINATING IN STARTING
MATERIAL FOR MANUFACTURER OF
HEMOPOETIC STEM CELLS IN GENE
THERAPY PRODUCT AND IN ORDER TO
OVERCOME THIS AND GET, YOU KNOW,
A BETTER QUALITY OF CD34
POSITIVE PRODUCT AND B MORE STEM
CELLS GENERALLY AND CAVEAT ABOUT
BONE MARROW HARVEST FOR SICKLE
CELL DISEASE PATIENTS IT IS
CHALLENGING TO GET CD34 -- MOST
PATIENTS UNDERGO MULTIPLE ROUNDS
OF BONE MARROW HARVEST THAT IS
AN INCREDIBLY INTENSE AND, YOU
KNOW, PAINFUL PROCEDURE TO HAVE
OCCURRING MULTIPLE TIMES.
SO, ONE THING THAT JOHN
MENTIONED PREVIOUSLY IS THAT,
YOU KNOW, THIS WAS A NECESSITY.
YOU CANNOT USE GCSF TO MOBILIZE
SICKLE CELL DISEASE PATIENT THAT
IS CONTRAINDICATED AND AROUND
THIS TIME PEOPLE STARTED TO
THINK ABOUT ARE THERE OTHER
DRUGS YOU COULD USE TO
POTENTIALLY BE APPLIED TO SICKLE
CELL DISEASE PATIENTS?
ONE DRUG IS [INDISCERNIBLE] AND
WORKING TOGETHER WE WERE ABLE TO
DETERMINE A REGIMENT THAT WORKED
WELL AND ALLOWING FOR COLLECTION
OF EMOBILIZED [INDISCERNIBLE]
ADMINISTRATION OF
[INDISCERNIBLE] WITHOUT GCSF AND
THIS WORK ALSO WAS PUBLISHED AND
HERE WE ARE DEMONSTRATING THAT
WE CAN GET A LOT MORE CELLS.
THEY LOOK MORE LIKE HEALTHY
INDIVIDUAL CD34 CELLS THAN THEY
LOOK LIKE BONE MARROW CD34 CELLS
COMING FROM SICKLE CELL DISEASE
PATIENTS.
HEALTHIER CELLS AND MORE CELLS
AND IMPLEMENTED MANY
MANUFACTURING CHANGES TO
INCREASE TRANSDUCTION EFFICIENCY
OF DRUG PRODUCTS ABLE TO GET
HIGHER EFFICIENCY TRANSDUCTION
OF PRODUCTS AND WERE ULTIMATELY
ABLE TO MAKE WHAT WE ASSUMED
WOULD BE SUB S STAN SHALLY MORE
EFFICACIOUS FOR MANUFACTURING
CONDITIONS.
THIS WAS TESTED.
AS JOHN REFERENCED BEFORE HB206
HAS MULTIPLE GROUPS SKIPPING
GROUP B AND GOING TO GROUP C.
GROUP C WE REQUIRED REGIMENTS
THAT WERE THOUGHT TO ESSENTIALLY
TAKE SOME OF THE PRESSURE OFF
THE BONE MARROW AND ARIDGE ROW
POETIC STRESS OFF MARROW OF
SICKLE CELL PATIENTS AND
CHANGING SOURCE OF STEM CELLS
AND REFINED MANUFACTURING
PROTOCOL TO INCREASE
TRANSDUCTION EFFICIENCY
INCREASING CELL DOSE WE WERE
ADMINISTERING THAT LED TO
DIFFERENT AND BETTER RESULTS FOR
PATIENTS TREATED AND WILL LET
JOHN WALK THROUGH CLINICAL DATA
HERE.
>> I CAN UPDATE YOU NOW.
THANKS, MELISSA, ON GROUP C
RESULTS.
WE HAD NOW 47 PATIENTS FOR
EFFICACY AND SAFETY ANALYSIS.
WITH 35.5 MONTHS OF FOLLOW UP
AND OVERALL EXPOSURE 106.2
PATIENT YEARS AND LONGEST FOLLOW
UP OF 61 MONTHS AND HAD A GOOD
SET OF PATIENTS ON DATA SETS TO
LOOK AT.
THIS IS A.
THIS IS TYPICAL OF SICKLE CELL
TRANSPLANT PROTOCOL AND AGE OF
ENROLLMENT MEETING OF 23 MALE
GREATER THAN FEMALE AND SEE IN
ALOE TRANSPLANTS TOO AND MOSTLY
BETA S BETA S PAIN CRISIS YOU
SEE HERE THAT HAVE VASOOCCLUSIVE
PATIENTS AND FEW PATIENTS WITH
HISTORY OF STROKE.
PATIENTS GETTING TRANSFUSED OR
HYDROXY URIA AND BASELINE
HEMOGLOBIN OF 8.7 TWO
MOBILIZATION CYCLES PER PATIENT
AND DOSE 6.5 AND GOT GOOD DOSE
OF [INDISCERNIBLE] AND END OF
THE PATIENTS RECOVERY AS
EXPECTED PATIENTS STAYED IN
HOSPITAL FOR A MONTH AND VECTOR
COPY IN PRODUCT WAS 4 AND MOST
CELLS HAD VEKOR AND PERIPHERAL
BLOOD VECTOR AT THIS TIME
REMAINED MEDIAN OF 1 AND
COMPARISON OF GROUP A AND GROUP
A AND .1 NOW 60 MONTHS OF FOLLOW
UP IT IS STABLE BUT LOW TO
EXPECT CLINICAL BENEFIT AND IT
IS MEDIAN OF 1.45 THAT WAS
ASSOCIATED WITH COMPLETE
RESOLUTION OF ALL DOES AND 88%
OF PATIENTS THESE ARE ACUTE PAIN
EVENTS DEFINED AS SHOWN HERE ON
THE RIGHT.
IMPORTANTLY 100% OF ADOLESCENT
PATIENTS THAT WE THINK THIS IS
BECAUSE PAIN HASN'T HAD
OPPORTUNITY TO BECOME CHRONIC
YET.
WE HAD 94% OF PATIENTS
EXPERIENCING COMPLETE RESOLUTION
OF ALL SEVERE VOES THAT ARE
ENDPOINTS FOR FDA TO CONSIDER
THIS.
AMONG THOSE PATIENTS WITH PAIN
EVENT POST INFUSION THEY WERE
MOSTLY BRIEF NOT LIKE ONES
BEFORE THAT COULD LASS FOR WEEKS
AND IMPROVEMENTS IN PAIN
INTENSITY AND PAIN INTERFERENCE
AND FATIGUE AND ALL THESE ARE
GOING IN THE RIGHT DIRECTION
THAT ARE CLINICAL WILL I
MEANINGFUL IMPROVEMENTS IN
MEASURES AS REPORTED NOT BY US
BUT BY PATIENTS THAT YOU CAN SEE
HEMOFWLOBIN ATA32 HEMOGLOBIN
ENCODED IN VECTOR IN BLUE
PERCENTAGE OF TOTAL HEMOGLOBIN
AND IMPORTANT TO NOTE THAT IT
GOES TO THE NORMAL RANGE AND
NEARLY HALF OF IT IS COMING FROM
VECTOR.
86.8% PATIENTS ACHIEVED ARE
GLOBIN RESPONSE TOO THAT WAS
INCREASING GREATER THAN 3 GRAMS
OR WEIGHTED AVERAGE OF 30% OF
HEMOGLOBIN T87Q COMING FROM
VECTOR OR ARISE GREATER THAN 10.
100% OF THOSE REACHING THIS GOAL
HAD IT BE DURABLE.
THESE ARE HOM OL SOUS MARKERS
LIKE I SHOWED YOU FOR ALLOGENEIC
TRANSPLANT PROTOCOL THEY
APPROACH LEVELS AND
[INDISCERNIBLE] RECEPTIC YOU'LLO
SITES BEING ELEVATED IN
ALOSETTING AND CONTRAST TO GROUP
A WE SAW ONLY THOSE B SUL FAN
RELATED TREATMENT OR ADVERSE
EVENTS AND NO CASES OF VEENO OK
COLLUSIVE DISEASE AND NO VECTOR
COMPLICATIONS INCLUDING
REPLICATION-COMPETENT LENTI
VIRUS AND ONE DEATH
UNFORTUNATELY IN INDIVIDUAL WITH
SIGNIFICANT CARDIO PULMONARY
DISEASE WITH A SUDDENEN DEATH
RELATED TO PRE-EXISTING CARDIO
PULMONARY DISEASE AND RESULTS
COMPARE FAVE RABBLY TO
ALOTRANSPLANT SETTING ASKED TO
COMPARE THEM TO WHAT IS SEEN
FROM ALORECIPIENTS THAT NEVER
HAD THESE THINGS DESCRIBED AND
ASSUME THEY GO AWAY AND LOOKED
AT PATIENTS ALEXIS AGAIN AND
FOUND THAT AFTER ALOTRANSPLANT
WE HAVE LOTS OF PAIN EVENTS IN
SECOND YEAR ALMOST NONE SHOWN IN
RED WITH EXCEPTION OF PATIENTS
AT TOP EXPERIENCING GRAPH
REJECTION AND WILL PASS IT BACK
TO MELISSA FOR HER TO SAY A
COUPLE WORDS ABOUT APPROVAL.
WE WILL TAKE QUESTIONS.
>> YES. YOU KNOW, FOR THE SAKE
OF TIME I WANT TO QUICKLY SAY
[INDISCERNIBLE] FOR THIS.
YOU KNOW, LIKE I SAID
PREVIOUSLY, WE ENCOUNTERED A LOT
OF CHALLENGES WITH THE
DEVELOPMENT OF THIS PROGRAM AND
YOU KNOW PARTLY I THINK BECAUSE
OF UNDERLYING DISEASE NUANCES
THAT REALLY ONLY BECAME CLEARER
OVER TIME AND PARTLY BECAUSE
THIS IS HARD AND GENE AND CELL
THERAPY IS REALLY HARD.
YOU KNOW, WE WERE -- IT WAS
INCREDIBLY FORTUNATE TO WORK
WITH JOHN AND HIS TEAM AND THAT
WE HAD A GREAT RELATIONSHIP WITH
NHLBI.
I DON'T. I CAN'T IMAGINE WE
WOULD BE ABLE TO OVERCOME
OBSTACLES IN A SHORT TIME PERIOD
OTHERWISE AND WE HAD AGAIN I
MENTIONED EARLIER INCREDIBLY
ALIGNED MOTIVATIONS AND
INTERESTS AND WANTED TO REALLY
DO WHAT WAS RIGHT FOR PATIENTS
WITH SICKLE CELL DISEASE.
WE ARE FORTUNATE WITH RECENT
APPROVALS AND PRODUCT THAT WAS
HERE TODAY COMING FROM BLUEBERG
BIOWAS APPROVED AND NOW MARKETED
I'M NO LONGER WITH THEM AND
CAN'T SPEAK TO THEM CURRENTLY
THIS IS NOT BLUEBERG MARKETING
NOW.
FOR SICKLE CELL PATIENTS ANOTHER
PRODUCT APPROVED IN DECEMBER AS
WELL.
THAT IS A GENE-EDITED PRODUCT
THAT IS A HEMOPOETIC STEM-CELL
BASED PRODUCT TONS OF WORK AND
DEDICATION AND TIME AND
FORTUNATE TO HAVE COLLABORATION
WE HAD AND RELATIONSHIPS WE WERE
ABLE TO BUILD.
>> I WANTED TO SAY ONE WORD
ABOUT THE PERSON WHOSE PICTURE I
HAD UP.
ACTUALLY FIRST PATIENT ON
PROTOCOL AND WHO WAS DESCRIBED
WITH MDS A FEW YEARS OUT DIDN'T
SURVIVE UNFORTUNATELY.
HE WAS A REAL PIONEER AND TOLD
US WHEN CONSENTED THAT HE WANTED
TO DO THIS FOR OTHER PATIENTS
WITH THE DISEASE REALIZING IT
MIGHT TAKE TIME FOR US TO GET
THERE AND REMINDING THANKING
PARTICIPANTS IN CLINICAL TRIALS.
WITHOUT THEM, WE COULDN'T DO ANY
OF THIS.
THEY ARE REALLY PARTNERS
BRINGING TRANSFORMATIVE
THERAPIES TO BEAR.
THANK YOU.
>> THANK YOU FOR THAT FANTASTIC
AND INSPIRING COLLABORATION
STORY.
WE ARE RUNNING A LITTLE SHORT OF
TIME.
SO, MAYBE WE HAVE TIME FOR ONE
QUESTION AND CAN GO ON TO THE
NEXT SESSION.
>> SO, MELISSA COULD YOU TALK
ABOUT YOUR EXPERIENCE ACTUAL
GOING THROUGH THE PAPERWORK AND
DEBT TRANSFER AND ACTUALLY
MAKING ALL THIS HAPPEN?
>> YEAH.
I THINK THAT THERE IS LOTS OF
ADVANTAGES TO THAT.
THERE IS LOTS OF FLEXIBILITY IN
TERMS OF UNDERLYING WORK AND
MOTIVATIONS WERE CLEARLY ALIGNED
AS WELL.
WE ENCOUNTERED ISSUES AND
TACKLED THEM TOGETHER AND AT
TIMES GOOD BENEFIT IS A PRETTY
BROAD DEFINITION WHAT YOU ARE
WORKING ON AND IT SEEMED THAT WE
COULD KEEP EXPANDING GOING OFF
IN TANGENTS AND DETAILS TO WORK
PROBLEMS AS THEY CAME UP YOU
KNOW INSTEAD OF SOMETIMES WHEN
YOU HAVE ACADEMIC RELATIONSHIP
IT IS VERY MUCH DEFINED IN SCOPE
OF ORIGINAL AGREEMENT AND NOT
LOTS OF FLEXIBILITY AND WIGGLE
ROOM TO GO BEYOND THAT.
IT IS NOT CASE HERE.
I SAID EARLIER I HOLD RECORD FOR
LONGEST IT HAS TAKEN TO GET FOR
A SPECIFIC ASPECT OF ONE THING
WE DID.
>> WHOLEHEARTEDLY I WILL
CONTINUE TO SAY IT WAS WORTH IT.
RIGHT?
IN THAT CASE JUICE WAS WORTH THE
SQUEEZE.
WE WANTED TO WORK WITH JOHN.
YOU HAVE A PLETHORA OF EXPERTS
WHO ARE NOT ONLY INCREDIBLY
SMART PEOPLE BUT DEDICATED AND
PATIENT FOCUSED AND DRIVEN.
THERE IS HUGE ADVANTAGES TO
WORKING WITH INDIVIDUALS THAT
EMBODY SAME VALUES THAT ARE
EMBODIED.
I HOPE A LOT ON THE CALL DO OR
STRIVE TO DO.
>> THANK YOU SO MUCH.
I WILL NOW TURN THIS OVER TO
MICHAEL WHO WILL INTRODUCE THE
NEXT SPEAK ER.
>> THANK YOU.
>> THANK YOU VERY MUCH.
WELCOME, EVERYBODY.
NIH IS LARGELY KNOWN AS BEING
THE LARGEST BASIC BIOMEDICAL
RESEARCH CENTER THAT IS THOUGHT
OF AS A LARGE FUNDER OF ACADEMIC
RESEARCH PUBLISHING ACADEMIC
PAPERS.
WHAT IS LESS KNOWN AND THIS
EVENT IS CERTAINLY HELPING TO
ADDRESS IS ALL OF THE WORK WE DO
WITH INDUSTRY PARTNERS TO GET
IMPORTANT UNMET MEDICAL NEEDS TO
PATIENTS THAT NEED THEM AND NIH
TRANSFER OFFICERS AND GREAT
REPRESENTATIVE OF GO-TO PLACE
FOR HEALTHCARE INDUSTRY THOUGHT
LEADER OVERCOMING TECHNOLOGY AND
KNOWLEDGE GAP CONDUCTING FIRST
TRIAL AND GETTING PRODUCT TO
MARKET.
I HAD TECHNOLOGY ANALYSIS AND
MARKETING UNIT WITHIN NCI'S TECH
TRANSFER CENTER AND BUSINESS
DEVELOPMENT GROUP AND IN
ADDITION TO PEOPLE SEEING ON
SCREEN HELPING PUT TOGETHER THIS
EVENT MY MISSION IS TO ACT AS
CONNECTORS TO OUTSIDE COMMUNITY
AND WOULD BE GLAD TO HEAR FROM
ATTENDEES IF WE CAN BE OF
SERV
SERVICE.
I'LL TURN IT TO ANDY BURKE TO
TALK ABOUT TECH TRANSFER AT
NATIONAL INSTITUTES OF HEALTH.
>> THANK YOU, MICHAEL. I
APPRECIATE THE INTRODUCTION. I
WILL SHARE MY SCREEN.
I HOPE THAT IS UP FOR EVERYBODY.
ALL RIGHT. THERE WE GO. THANK
YOU FOR THE KIND INTRODUCTION
AND OPPORTUNITY TO SPEAK TODAY.
WE HELP TO ENABLE SUCCESSFUL
SCIENTIFIC PARTNERSHIPS.
BEFORE I DO THAT I WANT TO TAKE
AN OPPORTUNITY TO TALK ABOUT
MYSELF. THIS IS PURPOSEFUL.
I WANT YOU TO APPRECIATE
PERSPECTIVE YOU ARE RECEIVING
TODAY TO UNDERSTAND THE SOURCE
OF THE INFORMATION AND MAYBE I
WILL INFORM SOME DECISIONS YOU
MIGHT DRAW FROM IT.
I SHOW THIS SORT OF ABBREVIATED
CHART AT THE TOP.
IF YOU LOOK YOUR WAY DOWN, YOU
NOTICE I WILL COME TO YOU TODAY
FROM DEEP IN THE ROSTER.
I VERY MUCH ENGAGE IN THE
DAY-TO-DAY TRANSACTIONAL WORK OF
TECH TRANSFER AND BIOMEDICAL
RESEARCH SCIENTIST IN TRAINING
AND I HAVE HAD OPPORTUNITY TO DO
HUNDREDS OF LICENSES AND
AGREEMENTS.
I WILL CELEBRATE THE FACT THAT
WE HAVE BEEN ABLE TO GET TWO FDA
TREATMENTS APPROVED OVER THE
FINISH LINE AND FINGERS CROSSED
WE HAVE ANOTHER PARTNER THAT
WILL BE SUCCESSFUL AS WELL.
I SHARE WITH YOU IN HOPE THAT MY
PERSPECTIVE WILL BE A USEFUL
COMPLEMENT TO THE INFORMATION
YOU RECEIVE FROM SENIOR STAFF IN
EARLIER TALKS.
I LIKE THE OPPORTUNITY TO TALK
ABOUT THE EXCITING AREA OF TECH
TRANSFER OR INTERFACE BETWEEN
ACADEMIC INNOVATION ON ONE SIDE
AND COMMERCIAL DEVELOPMENT THAT
IS NECESSARY TO GET A PRODUCT TO
PATIENTS AND ADDRESS UNMET
MEDICAL NEED THAT MICHAEL WAS
REFERENCING THAT IS ENOUGH ABOUT
ME AND I WILL TALK ABOUT
SOMETHING OF SUBSTANCE HERE.
WHEN I WAS FRAMING TODAY'S TALK
OR THINKING ABOUT IT I THOUGHT
IT WOULD BE USEFUL TO TALK ABOUT
THREE MAIN CATEGORIES OF
INFORMATION.
WHY WOULD YOU WANT TO WORK WITH
NIH?
THIS IS PERSPECTIVE OUTSIDE
PARTY LOOKING IN.
WHY WOULD YOU WANT TO WORK WITH
NIH?
WHAT CAN WE OFFER YOU IF YOU
WERE TO WORK WITH US? SEVERAL
THINGS OTHERS MENTIONED THAT I
WILL TOUCH ON BRIEFLY HERE.
YOU MADE LEAP AND DECIDED WE
COULD BE A PARTNER FOR YOU AND
INFORMATION ABOUT NIH TECH
TRANSFER STRUCTURE AND
ORGANIZATION AS LARGE AS OURS
HOW DO YOU FIND TECHNOLOGY
TRANSFER CONTACT? THAT COULD BE
A CHALLENGE WALKING THROUGH IN
DETAIL AND WHAT CAN WE DO FOR
YOU AND ENABLE SCIENCE AND WHAT
WE CAN DO TO ENABLE LICENSE THAT
WILL HELP YOU GET ACCESS TO
CRITICAL TECHNOLOGIES.
FINALLY IN SOMETHING THAT I WILL
HOPEFULLY TEE UP AN INFORMATIVE
Q & A ENGAGE IN MYTH BUSTING AND
WE WILL ADDRESS WORD ON THE
STREET INFORMATION THAT PEOPLE
KNOW ABOUT NIH TRANSFER ABOUT
THINGS WE CAN AND CAN'T DO AND
PARSING FICTION THERE.
STARTING I WANTEDED TO CELEBRATE
OR REMARK ON THE INCREDIBLE
BREADTH OF RESEARCH THAT GOES ON
AT NIH.
I THINK THAT PEOPLE ON OUTSIDE
LOOKING IN THINK OF NIH AS A
MONOLITH THAT IS ONE NIH.
IF YOU LOOK CLOSELY YOU REALIZE
THAT NIH IS 27 DISCRETE
INSTITUTES AND CENTERS THAT IS
27 DIFFERENT ORGANIZATIONS EACH
WITH THEIR OWN SPECIFIC FOCUSES
SUPPORTING BROADER NIH MISSION.
YOU HAVE WITHIN INSTITUTES YOU
HAVE A WHOLE RANGE OF PARTICULAR
RESEARCH PROGRAMS THAT FOCUS ON
SPECIFIC DISEASES OR PROCESSES
NATIONAL CANCER PROCESS THAT
EFFECT IHEALTH NATIONAL
INSTITUTE AND NATIONAL EYE
INSTITUTE NIAD FOR INFECTIOUS
DISEASES OR ALLERGIES AND NORMAL
RESEARCH BIOLOGY TO DISEASE
TREATMENT PREVENTION
DISSEMINATION OF KNOWLEDGE ELTS
AND UPSHOT FOR POTENTIAL PARTNER
IS WHATEVER YOUR FIELD OF
INTEREST I HAVE EXTREMELY HIGH
CONFIDENCE THAT SOMEWHERE IN 27
INSTITUTES AND CENTERS IS A
RESEARCHER THAT KNOWS AND CARES
ABOUT YOUR AREA AS MUCH AS YOU
DO.
RIGHT?
SO JUST FOR EXAMPLE SAY YOU ARE
A -- SAY YOU ARE A CELL THERAPY
START-UP INTERESTED IN
DEVELOPING NEXT GENERATION
T-CELL THERAPIES FOR COMMON
EPITHELIAL CANCERS TOPIC NEAR
AND DEAR TO PROFESSIONAL HEART.
IF THAT IS YOU YOU WILL BE PROUD
THAT NIH IS HOME TO SURGERY
BRANCH AND UNDERSTATEMENT OF.
PERHAPS HIS RESEARCH WOULD BE
INTERESTED TO YOU.
MAYBE NOT INTERESTED IN CELL
THERAPY BUT ALIGNED IN
INFECTIOUS DISEASE SPACE AND
HUMANITY AND MALARIA AND IF THAT
IS THE CASELY INTRODUCE YOU TO
KAIRO LEANA WHO IS DISTINGUISHED
INVESTIGATOR AT ANYAD AND WORLD
RECOGNIZED EXPERT IN PARASITE
INTERACTIONS IN MALARIA.
MAYBE NEITHER OF THOSE TICKLE
YOUR FANCY AND YOU ARE MORE
INTERESTED IN RARE OR FANNED OR
GENETIC DISEASES IN AREAS OF
METABOLISM AND YOU WOULD PERHAPS
BE DECIDED TO LEARN ABOUT WORK
OF BILL WHO IS A SENIOR
INVESTIGATOR AND ANOTHER MEMBER
OF NATIONAL ACADEMY OF MEDICINE;
RIGHT?
LONG-TIME LEADER OF UNDIAGNOSED
DISEASES PROGRAM AND EXPERT IN
RARE GENETIC DISORDERS AND
FANTASTIC AND NICE GUY TO BOOT.
YOU KNOW, BEYOND FANTASTIC HUMAN
CAPITAL AVAILABLE AT NIH, THERE
IS SOME PARTICULAR RESEARCH
RESOURCES THAT REALLY SEPARATE
US FROM THE CROWD.
I'M SURE THAT OTHERS HAVE
REMARKED ON THIS EARLIER AND
WILL HIGHLIGHT TWO THAT ARE
PARTICULARLY RELEVANT I THINK.
FIRST, NANOTECHNOLOGY
CHARACTERIZATION LAB THAT IS A
FACILITY BASED OUT OF NATIONAL
LAB IN FRED ERIC MARYLAND
PARTNERSHIP FOOD AND DRUG
ADMINISTRATION AND NATIONAL
INSTITUTES OF STANDARDS AND
TECHNOLOGY AND MISSION TO
SUPPORT DEVELOPMENT OF
NANOTECHNOLOGIES AND HOW YOU
LEVERAGE INCREDIBLE CAPABILITIES
OF NANOSCALE SCIENCE TO ADDRESS
PROBLEMS IN MEDICINE?
STAFF AT NANOTECHNOLOGY
CHARACTERIZATION LAB DEVELOPED
AN INCREDIBLY ROBUST ASSAY
CASCADE FOR REVIEW OF NEW
NANOMATERIALS AND PURPOSE OF
THAT IS TO SUPPORT REGULATORY
REVIEW OF NANOMEDICINES AND IF
YOU ARE A COMPANY OPERATING THIS
SPACE AND HAVE A NEW MATERIAL
YOU CAN WORK WITH NANOTECHNOLOGY
CHASHGIZATION LAB SUBMITTING
MATERIAL FOR REVIEW THAT WILL
RETURN YOU TO A TREASURE TROVE
OF VALUABLE DATA A RESOURCE THAT
NEEDS NO INTRODUCTION AND IF YOU
HAVE BEEN TO NIH CAMPUS IN BETH
ES DAH YOU HAVE SEEN THIS
BUILDING WORLD'S LARGEST
HOSPITAL DEVELOPED IN CLINICAL
RESEARCH.
1300 PHYSICIANS AND RESEARCHERS
AT ANY ONE TIME 1600 ACTIVE
ONGOING CLINICAL STUDIES AND
ENGAGED IN CLINICAL RESEARCH OR
WANT TO BE ENGAGED IN CLINICAL
RESEARCH, THIS IS THE PROMISE
LAND.
NOW, I GUESS AT NO SURPRISE;
RIGHT?
COMBINING MISSION-DRIVEN
ENVIRONMENT WITH NIH FANTASTIC
HUMAN CAPITAL AND RESOURCES YOU
DESCRIBED CREATING ENVIRONMENT
THAT IS RIGHT FOR INNOVATION AND
HIGHLIGHT THAT HERE LOOKING AT
SUMMARY STATISTICS THAT NIH TECH
TRANSFER PUT OUT IN RECENT
REVIEW PERIOD DECADE BETWEEN
2012 AND 2022 AND THOSE
INTRAMURAL RESEARCHERS DISCLOSED
TO OFFICES OVER 3,000 NEW
INVENTIONS AND TECH TRANSFER
OFFICE REVIEWING THESE AND
MAKING DECISIONS TO FILE PATENT
PROTECTION OVER 1800 TIMES
UNITED STATES AND PATENT
PORTFOLIO IN THE UNITED STATES
IS GLOBAL AND GOES BEYOND US
FILINGS WE ARE REFERENCING OVER
HERE.
WE DON'T SECURE THESE FOR SAKE
OF HOLDING PATENT RIGHTS THAT
ARE A TOOL TO INCENTIVIZE
COMMERCIAL DEVELOPMENT OF
INVENTIONS AND TECH TRANSFER
OFFICES WORK WITH OUTSIDE
PARTIES TO TRANSFER INTELLECTUAL
PROPERTY AND THAT SCIENTISTS
HAVE GENERATED FOR BENEFIT OF
PUBLIC THROUGH COMMERCIALIZATION
AND RESULT OF PRODUCTS
ADDRESSING THAT NEED AND SAME
TIME PERIOD TECH TRANSFER
PRACTICES EXECUTE OVER 3100
LICENSES OVER 1,000
[INDISCERNIBLE] AND LISTENING TO
LAST TALK IT IS AMAZING THING TO
SUPPORT COLLABORATIVE RESEARCH
STUDIES WITH SCIENTISTS AND
ORGANIZATIONS AND BEYOND THAT
OVER 52,000 TRANSACTIONAL
AGREEMENTS IN SUPPORT OF THAT
MISSION.
AND SO I -- OBVIOUSLY THE NIH
HAS A WEALTH OF VALUABLE
BIOMEDICAL INVENTIONS THROUGH
PARTNERSHIPS WITH OUTSIDE
INDUSTRY THAT PRODUCTS REACH THE
MARKET.
WE ARE FORTUNATE THAT OVER 39FDA
APPROVED THERAPIES OR VACCINES
HAVE -- HAVE MADE IT ACROSS THAT
FINISH LINE.
I HYLIEKT A FEW OF THEM HERE;
RIGHT? FROM CELL AND GENE
THERAPY SPACE AND THAT IS 50% OF
ALL FDA APPROVED CAR T PRODUCTS
HAVE INVENTIONS COMING OUT OF
NIH TO GUARDO SIL AND IT IS AN
AMAZING BODY OF WORK.
ALL RIGHT. I KNOW THAT IS AN
INCREDIBLY WHIRLWIND BENEFIT TO
WORKING AT NIH AND CONVINCED YOU
SHOULD BE WORKING WITH US AND
QUESTION IS HOW SHOULD YOU DO
IT?
QUESTION IS TIMELY.
WE ARE HERE TO ADDRESS JUST
THAT.
NIH TECH TRANSFER OFFICES ARE
GATEWAYS TO COLLABORATIONS AND
LICENSES.
IF YOU START AT THE TOP, RIGHT?
THERE IS NIH OFFICE OF
TECHNOLOGY TRANSFER THAT IS AT
AN NIH LEVEL ABOVE INDIVIDUAL
INSTITUTES THAT IS A CENTRAL
OFFICE NOT SO MUCH ENGAGED IN
DAY-TO-DAY TRANSACTIONAL WORK OF
TECH TRANSFER PROVIDING CENTRAL
RESOURCES TO LOWER INSTITUTE
LEVEL TECH TRANSFER OPS MANAGING
DATA BASES AND SOME ABSOLUTELY
CRITICAL FUNCTIONS ALLOWING
PROCESSES TO WORK EFFICIENTLY.
REAL MEAT OF IT IS THE FOCUS OF
INTEREST FOR AN OUTSIDE PARTY
THERE ARE 8 INSTITUTE LEVEL TECH
TRANSFER OFFICES PROVIDING
OFFICES FOR CENTERS FOR DISEASE
CONTROL AND PREVENTION THAT IS
IN 8 OFFICES WORKING WITH IC
INVESTIGATORS PATENT RIGHTS NEW
INVENTIONS REPORTED TO US
LICENSES AND TRANSACTIONAL
AGREEMENTS FOR INVESTIGATORS ON
THEIR DOCKET.
WE DO MANY THINGS AND WANT TO
POINT OUT TWO POPULAR AREAS WE
DON'T HAVE MUCH ENGAGEMENT.
IF INTERESTED IN COMPETITIVE
GRANT FUNDING FOR SCIENTIFIC
WORK OR IF INTERESTED IN SBR
FUNDING, YOU WILL ENGAGE WITH
TECH WILL NOT BE PRODUCTIVE.
YOU HAVE TO GO TO ANOTHER SPACE.
OTHER SPACES FOR LICENSING
COLLABORATION AND TRANSACTIONAL
[INDISCERNIBLE] WE ARE HOME FOR
THAT; RIGHT?
I MENTIONED 27 INSTITUTES AND
CENTERS IN NIH AND CITING 8 TECH
TRANSFER OFFICES HERE.
DON'T WORRY BALANCE OF
INSTITUTES SERVICES ARE STILL
MET AND STILL PROVIDED FOR THEM
AND SMALLER INSTITUTES CONTRACT
WITH SOME OF ONE OR MORE LARGER
OFFICES TO HAVE SERVICES
FULFILLED THAT I HIGHLIGHT
INSTITUTES THAT DON'T HAVE
OFFICE OF THEIR OWN BUT TECH
TRANSFER SERVICES MET THROUGH A
LARGER TECH TRANSFER OFFICE.
ALL RIGHT. IF WE GENERALLY ARE
ORIENTED WITH TECH TRANSFER
STRUCTURE AT NIH AND TALKING
ABOUT COMMON SCENARIOS HOW TO
ENGAGE WITH OFFICER AND FIND
REALLY THE PERSON YOU NEED TO
GET TO TO HAVE THE CONVERSATION
ABOUT LICENSING OR COLLABORATIVE
SCIENCE?
IN MY MIND TWO MOST COMMON
SCENARIOS; RIGHT?
FIRST YOU MADE CONTACT WITH NIH
PRINCIPAL INVESTIGATOR AND YOU
HAD A GOOD DIALOGUE ABOUT WHAT
TO DO TOGETHER AND WHAT IS
POSSIBLE AND IS ALIGNMENT OF
INTEREST AND IN THAT CASE NIH
PRINCIPLE INVESTIGATOR IF THEY
BELIEVE BENEFIT FROM ENGAGING
TECH TRANSFER OFFICER TOOLS WE
HAVE THAT WOULD FACILITATE THAT
INTERACTION THEY WILL MAKE
DEFERRAL TO US. YOU DON'T HAVE
TO DO NAVIGATING THAT WILL REFER
COMPANY TO US AND WE WILL REACH
OUT TO YOU DIRECTLY.
THAT IS FAIRLY STRAIGHTFORWARD.
SECOND IS IF YOU START COLD AND
PERHAPS DON'T HAVE EXISTING
RELATIONSHIP WITH NIH PRINCIPLE
INVESTIGATOR THEY LEFT NIH AND
ARE AT OTHER INSTITUTE AND
LOOKING AT PAPER PUBLISHED SOME
YEARS AGO AND PATENT FILING OF
INTEREST TO YOU AND EXPLORING
OPPORTUNITIES TO COLLABORATE AND
IMPORTANT TO FIND RIGHT TECH
TRANSFER PERSON TO TALK TO
DEPENDS ON INSTITUTE OR CENTER
PI WORKED FOR GOOGLE OR ONLINE
SEARCH TOOL AND NAME + NIH WILL
POP UP INSTITUTE THEY WORKED FOR
AND WHAT I LISTED HERE
CONVENIENTLY LISTS TECH
TRANSPORT FOR INSTITUTE WITHIN
NIH GO THERE E-MAIL US MAKE
CONNECTION AND LET'S TALK.
I THINK TWO SCENARIOS PROBABLY
COVER 99.9% OF HOW YOU WOULD GET
IN TOUCH WITH TECH TRANSFER
OFFICE.
I WANT TO BE DEFINITIVE HERE AND
FOR WHATEVER REASON THOSE TWO
THINGS DON'T APPLY YOU TO, I
HAVE LISTED TWO CONTACTS.
IF YOU NEED TO PICK UP THE RED
PHONE AND MAKE CONTACT WITH A
LIVE INDIVIDUAL WHO CAN HELP TO
GET YOU TO THE RIGHT PLACE,
THESE ARE PEOPLE TO DO IT.
ALL RIGHT. HOAR A, YOU
NAVIGATED ROUGH WATERS MAKING
CONTACT WITH TECH TRANSFER AND
QUESTION IS WHAT CAN WE DO FOR
YOU NOW?
WE UNDERSTAND YOUR TIME IS
VALUABLE AND WILL START
ENGAGEMENT WITH A NEEDS
ASSESSMENT AND WILL UNDERSTAND
WHAT YOU ARE SEEKING AND WHAT
REQUIREMENTS ARE AND WE WILL
CRAFT A WORK PRODUCT AROUND THAT
THAT MEETS NEEDS AND INTERESTED
IN SCIENTIFIC COLLABORATION
ENGAGING WITH NIH PRINCIPLE
INVESTIGATOR TO MAKE SURE THEY
ARE INTERESTED AND ALIGNMENT ON
RESEARCH OBJECTIVES AND PUTTING
AGREEMENT STRUCTURES IN PLACE TO
SUPPORT SCIENCE INTENDED AND IF
YOU DON'T KNOW OR HAVE NOT MADE
CONTACT WITH NIHPI AND
COLLABORATION IDENTIFYING
INTERNAL CHAMPION TO HAVE
CONVERSATION AND SEE IF A
COLLABORATIVE PROJECT MAKES
SENSE AND INTERESTED IN
ACCESSING MATERIAL AND
INTELLECTUAL PROPERTY WE HAVE
VARIETY OF AMECHANISM GREEMENT
MECHANISMS.
WE WILL FIND AGREEMENT
APPROPRIATE FOR CIRCUMSTANCES
AND MAKE SURE THAT WE CAN PUT
APPROPRIATE TERMS IN PLACE FOR
YOU PRACH YOU ARE THINKING BACK
TO CONVERSATION WITH FRIENDS
BROTHER'S COUSIN RAISED CONCERN
ABOUT NIH CAN'T DO X OR WON'T BE
ABLE TO DO Y; RIGHT? I THINK
THIS IS A GREAT OPPORTUNITY TO
CLEAR THE AIR AND TALKING ABOUT
PERVASIVE MYTHS OUT THERE.
WHAT IS FACT AND REALLY FICTION;
RIGHT?
COMMON MISCONCEPTIONS ABOUT TECH
TRANSFER, LET'S START WITH
LOW-HANGING FRUIT.
FIRST NIH ONLY DOES BASIC
RESEARCH; RIGHT? YOU SEE WHERE
THIS MIGHT HAVE COME FROM AND
INCREDIBLY LARGE FUNDER OF BASIC
RESEARCHERS ACADEMIC PARTNERS
UNDER GRANTS AND HAS BECOME
CLEAR THROUGH TODAY'S
PRESENTATIONS WE DO MORE THAN
THAT AND 6,000 SCIENTISTS AND
CLINICIANS ENGAGED IN SPECTRUM
OF BIOLOGICAL RESEARCH INCLUDING
TRANSLATIONAL WORK NIH CENTER IS
DOING CLINICAL WORK AND WE ARE
DOING MORE THAN JUST BASIC
RESEARCH.
SECOND ONE COMING UP OFTEN IS
THAT NIH WORKS WITH ONLY OR
PRIMARILY WORKS WITH ACADEMIC
ORGANIZATIONS AND COMES PROBABLY
FROM HISTORY OF GRANT FUNDING TO
UNIVERSITIES.
I MENTIONED IN THE FAST FEW
YEARS WE HAVE DONE THOUSANDS OF
KREDAS AND LICENSES AND
COLLABORATIONS WITHIN INDUSTRY
AND 39FDA APPROVED TREATMENTS
THAT ARE IN TREATMENTS AND
VACCINES THAT ARE PRODUCT OF
COLLABORATIONS AND LICENSES
WITHIN INDUSTRY AND WE WORK MORE
WITH ACADEMIC ORGANIZATIONS AND
NIH ONLY WORKS WITH DOMESTIC AND
US-BASED ORGANIZATIONS AND
POTENTIALLY YOU CAN SEE WHERE
THIS MIGHT COME FROM AND US
FEDERAL AGENCY AND SEE MAYBE WE
HAVE LIMITATIONS WHO WE CAN WORK
WITH.
FACT IS THAT NIH IS INTERESTED
IN DOING GOOD SCIENCE.
GOOD SCIENCE CAN BE DONE
GLOBALLY.
WE PARTNER INTERNATIONALLY AND
ON THE LICENSING SIDE OR
COMMERCIAL SIDE THERE IS NO
STATUTORY PREFERENCE FOR
US-BASED COMPANIES IN LICENSING
AND STATUTORY PREFERENCE FOR
SMALL BUSINESS BUT PREFERENCE IS
NOT BASED ON WHAT COUNTRY ARE
YOU HEADQUARTERED IN?
WHERE RESOURCES ARE AVAILABLE TO
COMPANIES US-BASED OR
NON-US-BASED AND NIH IS
INTERESTED IN DEVELOPING
INTERNAL IDEAS.
NO. IF WE OR SCIENTISTS ARE
APPROACHED BY COMPANY OR OUTSIDE
COLLABORATOR WITH COMPELLING
RESEARCH PLAN, THAT COULD BE
BASIS OF COLLABORATION OR BASIS
OF JOINT SCIENCE PROGRAM.
ALL RIGHT. LET'S TALK ABOUT
SOMETHING A LITTLE MORE WEIGHTY.
RIGHT?
NIH'S TECH TRANSFER PRACTICES
ARE CUMBERSOME AND TIME
CONSUMING CAN'T ALIGN WITH NEEDS
OF COMMUNITY BECAUSE TIMELINE IS
SIMPLY TOO LONG; RIGHT?
LET'S BREAK IT DOWN.
AS I SAID, WE UNDERSTAND TIME IS
VALUABLE AND WHY WE START WITH
NEEDS ASSESSMENT TO UNDERSTAND
WHAT YOU NEED AND WHAT ARE
REQUIREMENTS OF ENGAGEMENT AND
HOW CAN WE BEST MEET THEM?
WE INVOLVE THE NIHPI AS
APPROPRIATE PARTICULARLY FOR
COLLABORATIVE SCIENCE AND
ENSURING WE ARE ALIGNED ON
INTEREST AND EXPECTATIONS AND AS
I SAID 50,000 + AGREEMENTS
DIDN'T HAPPEN BECAUSE WE DON'T
KNOW WHAT WE ARE DOING AND WE
ARE TRANSACTION EXPERTS AND
MISSION-DRIVEN PROFESSIONALS WE
CAN ABSOLUTELY KNOW WHAT IT
TAKES TO GET ACROSS THE FINISH
LINE AND GET IT DONE IN TIMELY
FASHION AND THIS IS AN
OPPORTUNITY TO TALK ABOUT WHAT
IS CHARACTERIZED AS BEST
PRACTICES FOR SUCCESSFUL
OUTCOMES AND NO. 1 ENGAGING WITH
TECH TRANSFER OFFICE UP FRONT
AND STARTING ON RIGHT TRACK AND
NOT COMMIT TO AGREEMENT FORM
WITHOUT THINKING THROUGH FULL
SCOPE OF ENGAGEMENT; RIGHT?
MAKE SURE WE HAVE AGREEMENT IN
PLACE AND NOT WASTING TIME ON
USING A MODEL NOT APPROPRIATE
FOR THE CIRCUMSTANCES AND OTHER
THING ENGAGING WITH IT UP FRONT
IT ALLOWS US TO RUN PROCESSES IN
PARALLEL NOT SERIES.
THERE ARE CERTAIN REQUIREMENTS
THAT SIMPLY MUST BE MET THAT WE
CAN RUN ON THE SIDE WHILE YOU
ARE WORKING WITH A PRINCIPLE
INVESTIGATOR ON FINALIZING
RESEARCH PLAN.
WE CAN ARRIVE AT THE END WITH
THE NECESSARY TASKS ACCOMPLISHED
AND WE HAVE TO POSITION TO
EXECUTE IN TIME AND MANNER.
NEXT THING IS REALLY THE MINDSET
THAT THE NIH IS NOT A CONTRACT
RESEARCH ORGANIZATION.
RIGHT?
I THINK THAT WE WILL HAVE A MUCH
MORE PRODUCTIVE DISCUSSION IF
PEOPLE WHEN WE ARE ENGAGING WITH
COMPANIES AND THERE IS AN
UNDERSTANDING THIS WILL BE A
PARTNERSHIP AND WE ARE NOT A SET
OF HANDS ON LAB BENCH AND
AWAITING INSTRUCTIONS.
IT GOES WITHOUT SAYING PER ALL
AGREEMENTS THERE ARE TWO PARTIES
THAT NEED TO COME TO A CONSENSUS
WE ARE IN POSITION TO EXECUTE.
IT IS RARELY THAT ONE SIDE IS
ONE THAT IS RESPONSIBLE FOR
DELAY AND OTHER SIDE HAD NO
INVOLVEMENT MERELY SITTING THERE
WITH A PEN WAITING TO SIGN;
RIGHT?
THAT'S -- ALL RIGHT. NEXT ONE
THAT IS OFTEN RAISED THAT NIH IS
NOT ABLE TO GRANT EXCLUSIVE
RIGHTS TO PATENT RIGHTS.
THIS IS FALSE.
LAW RECOGNIZES CERTAIN
SITUATIONS INTELLECTUAL PROPERTY
OR EXCLUSIVE RIGHTS PROPERTY
COULD BE INCENTIVE TO TIME AND
CAPITAL AND LABOR NECESSARY FOR
PRACTICAL -- THAT ALLOW US TO
GRANT EXCLUSIVE LICENSES IN
CERTAIN CIRCUMSTANCES.
FIRST IS IF YOU ARE NOT WORKING
WITH [INDISCERNIBLE] AND SIMPLY
SEEKING A LICENSE TO
GOVERNMENT-OWNED INTELLECTUAL
PROPERTY.
WE HAVE ABILITY TO GRANT
EXCLUSIVE LICENSE PROVIDED WE
MEET AND ARE ABLE TO DETERMINE
CERTAIN THINGS PUBLIC BENEFIT
RESULTING FROM LICENSE AND
EXCLUSIVITY AND REASONABLE
INCENTIVE AND SCOPE GRANTING NOT
MORE REASONABLY NECESSARY AND
CREATE ANTITRUST LAW AND ABILITY
TO DETERMINE THIS WILL COME FROM
INFORMATION PROVIDED IN LICENSE
APPLICATION AND WOULD SAY IN
GENERAL THAT THESE CRITERIA ARE
OFTEN THAT WHEN INTERESTED OR
COMPANY IS DEVELOPING A PRODUCT
THAT REQUIRES EXTENSIVE
REGULATORY REVIEW OF TREATMENT
OR THERAPY OR REGULATED DEVICE
AND TIMELINES ARE EXTENDED AND
COSTS OF DEVELOPING IT ARE HIGH
AND ODDS OF SUCCESS ARE LOW,
THERE IS REASONABLE BASIS FOR
GRANT OF EXCLUSIVITY IN CASES
AND CAN BE.
SECOND WORKING IN KREDA AND
ALLOWING OPTION LICENSE
INVENTIONED DEVELOPED IN KREDA
AND TWO STRAIGHTFORWARD
MECHANISMS TO OBTAINING
EXCLUSIVE LICENSE.
>> IF YOU DON'T HAVE OPPORTUNITY
TO GO OVER THIS IN DETAIL I WANT
TO PROVIDE A LIST THAT IS
SOMETHING THAT YOU COULD
CONSIDER IN SLIDES AT A LATER
DATE.
I LIST THESE PARTNERS HERE.
WOULD BE FANTASTIC TO GO INTO IN
MORE DETAIL AND IF YOU HAVE
QUESTIONS WE CAN ADDRESS IT IN
MORE Q & A PORTION AND FANTASTIC
ORGANIZATION AND LOVED IT PAST
10 YEARS WE HAVE BEEN HERE AND
WE ARE A MISSION DRIVEN
ORGANIZATION AND SUPPORT BROADER
MISSION OF NIH BY ALLOWING
RESEARCH TO PARTNER WITH
EXTERNAL INDUSTRY TO
COMMERCIALIZE CUTTING-EDGE
MEDICAL RESEARCH THAT WE ASK YOU
TO REALLY JOIN US ON THIS
MISSION.
RIGHT?
WE WOULD LOVE TO WORK WITH YOU
AND I EMPHASIZE TO GET IN TOUCH
WITH US. WE WOULD LOVE TO HEAR
FROM YOU AND WEBSITES I LIST
HERE SHOW NIH TECHNOLOGIES
AVAILABLE FOR LICENSE AND
WEBSITE SHOWS AND BRINGS YOU TO
NIH INTRAMURAL RESEARCH PAGE
LOOKING AT DESCRIPTION OF
DIFFERENT SCIENTIFIC RESEARCH
PROGRAMS ONGOING IN NIH RESEARCH
COMMUNITY THAT COULD BE OF
INTEREST TO YOU FOR POTENTIAL
PARTNERING.
WITH THAT, I KNOW WE ARE TIGHT
ON TIME AND I'M HAPPY TO STOP
AND PROVIDE AN OPPORTUNITY FOR
QUESTIONS.
>> THIS IS NOT A LOT OF
QUESTIONS THAT I PUT A COUPLE IN
THE CHAT AND NOT SURE IF YOU CAN
SEE THAT.
>> OKAY. LET'S SEE. NIH ACCEPT
SPONSORED RESEARCH PROGRAMS IF
INDUSTRY?
WE HAVE A -- IT DEPENDS HOW YOU
ARE DEFINING A SPONSORED
RESEARCH PROGRAM.
WE HAVE A CREATIVE PROGRAM AND
HAVE MECHANISMS BY WHICH WE CAN
COLLABORATE WITH INDUSTRY AND
KREDA ALLOWS US TO DO A
COLLABORATIVE PROGRAM AND
COLLABORATIVE SCIENTIFIC PROGRAM
AND ALLOWS US TO RECEIVE FUNDING
FROM OUTSIDE PARTY IF NECESSARY
TO CONDUCT SCIENCE AND OFFER US
TO EXCLUSIVE LICENSE THAT IS
DEVELOPED UNDER THAT RESEARCH
PROGRAM.
POTENTIALLY THAT ADDRESSES THE
QUESTION THAT IS THRUST THERE.
WE HAVE OTHER MECHANISMS
ENGAGING WITH INDUSTRY THAT
DON'T OFFER SAME BENEFITS YOU
WILL SAY; RIGHT?
WE CAN SET UP COLLABORATION
AGREEMENT ALLOWING TWO-WAY
EXCHANGE OF INFORMATION AND
MATERIALS ALLOWING PARTIES TO
ENGAGE IN COLLABORATIVE SCIENCE.
LAW DOESN'T ALLOW US TO OFFER
EXCLUSIVE LICENSE OPTION AND
CAN'T ACCEPT FUNDING UNDER THAT
MECHANISM AND PARTIES TO
DETERMINE WHICH IS MOST
APPROPRIATE FOR CIRCUMSTANCES.
>> IF I CAN STEP IN FOR A
SECOND.
>> PLEASE.
>> SPONSORED RESEARCH AGREEMENTS
EXIST IN ACADEMIA.
NIH DOESN'T DO THAT.
WE HAVE TO BE DRIVEN BY OWN
MISSION AND DESIRE TO DO
RESEARCH PROJECTS.
>> YEAH.
>> WE WON'T DO FEE-FOR-SERVICE
STUFF.
IT IS WHAT OFTEN, YOU KNOW,
SPONSORED RESEARCH REFERS TO IN
ACADEMIA.
>> RIGHT.
>> MAKING DISTINCTION.
>> THAT IS GOOD. I MENTIONED
EARLIER WE ARE NOT C ARE. O.
WE ARE LOOKING FOR A PARTNERSHIP
THIS IS NOT PAY TO PLAY
SITUATION YOU ARE PAYING FOR
SPECIFIC RESEARCH TO BE DONE AND
WE SEND YOU RESULTS FROM THE
OTHER SIDE.
DOESN'T WORK LIKE THAT.
>> QUICK COMMENT ON ANDY'S
REMARK IT IS A MECHANISM THAT
SOME RELATIONSHIPS WE CAN'T TAKE
IN FUNDING WHEN NEEDED.
IT IS IMPORTANT TO MENTION THAT
WE HAVE AN OVERHEAD RATE THAT IS
CAPPING 15%.
>> UH-HUH.
>> 26 TO THE 27 INSTITUTES HAVE
0% OVERHEAD RATE AND INSTANCES
REQUESTING FUNDS FROM PARTNERS
TO MENTION ALL OR A SIGNIFICANT
PORTION OF THE FUNDS WILL GO TO
MOVING YOUR PRODUCT FORWARD
RATHER THAN OTHER USES.
>> SURE.
>> IN INTEREST OF TIME WE ARE A
LITTLE OVER.
>> OKAY.
>> I THINK WE CAN CLOSE YOUR --
I WANT TO REMIND EVERYBODY THAT
WE WILL TAKE A BREAK FOR 30 -- A
LITTLE LESS THAN 30 MINUTES.
WHEN YOU COME BACK YOU WILL
ENTER ONE OF THE THREE PANELS
THAT ARE CONCURRENT AND START AT
3:15.
PLEASE PICK ONE OF THEM.
SOMETIMES YOU MIGHT WANT TO
LISTEN TO MORE THAN ONE.
YOU CAN DO IT AFTER THE FACT
THAT WILL BE AVAILABLE AS VIDEO
AND ON DEMAND SAME PLATFORM UP TO FOUR WEEKS AFTER THIS
CONFERENCE COMING BACK PICK A PANEL AND THEY WILL BE ONE OF
THE LAST SESSIONS FOR THE DAY. THANK YOU, ANDY AND MICHAEL.
>> THANK YOU.