>> Joni Rutter: THANK YOU,
EVERYONE FOR JOINING TODAY.
OUR 36th MEETING OF THE
NATIONAL CENTER FOR ADD VAPS GS
TRANSLATIONAL SCIENCE ADVISORY
COUNCIL TODAY.
I WILL GAVEL US IN.
YOU MAY COMMENCE.
I THINK I JUST BROKE THE GAVEL.
[LAUGHTER]
IT'S A GOOD START, YES.
OKAY, WELL, I DON'T HAVE REALLY
ANY OPENING STATEMENTS, JUST TO
INVITE YOU HERE TODAY TO HEAR A
LITTLE BIT MORE ABOUT WHAT
NCATS HAS BEEN DOING OVER THE
COURSE SINCE WE LAST MET.
ANNA WILL GO OVER OUR AGENDA.
AND THEN WE WILL GO AHEAD AND
GET STARTED WITH THE
PROCEEDINGS.
ANNA, WE WILL TURN IT OVER TO
YOU.
>> Anna Ramsey-Ewing: THANK
YOU, JONI.
WE WILL COMMENCE WITH OUR ROLL
CALL.
MEMBERS IN ATTENDANCE PLEASE
INDICATE SO.
SERGIO?
>> HERE.
>> PAUL HARRIS?
>> ANNI KENNEDY.
>> KELLY McVERNY?
>> HERE.
>> KEITH MULLER.
>> THE ONLY NOT IN ATTENDANCE
TODAY IS PAULA SHARMIN.
THE OPEN SESSION WILL TAKE
PLACE OVER ONE DAY.
AS YOU CAN SEE WE WILL BEGIN
WITH OUR DIRECTOR'S REPORT.
AFTER THAT WE HAVE A
PRESENTATION BY PETER MARKS BY
THE FDA.
WE WILL HAVE A SHORT LUNCH
BREAK TODAY, FOLLOW BY TWO
PROGRAM UPDATES.
ONE FROM DIVISION OF RARE
DISEASES RESEARCH INNOVATION.
THEN DIVISION OF PRECLINICAL
INNOVATION.
THEN A VERY SHORT BREAK AND
MOVE INTO OUR CLEARANCE OF
CONCEPTS.
THIN A SHORT DECISION FOR OUR
WRAP-UP AND FINALLY ADJOURN.
NEXT SLIDE, PLEASE.
ALL RIGHT.
SO BECAUSE WE ARE MEETING IN
PERSON AND WE ARE ALSO
PARTICIPATING BY ZOOM, THE
FOLKS ARE IN THE ROOM ARE NOT
REQUIRED TO BE CONNECTED TO
ZOOM.
IT'S ALSO IMPORTANT TO TURN
YOUR TABLE MICROPHONE ON WHEN
YOU ARE SPEAKING TO MAKE SURE
EVERYONE CAN HEAR YOU.
IF YOU ARE PARTICIPATING
VIRTUALLY, PLEASE USE THE RAISE
HAND OR CHAT FEATURE AND WAIT
UNTIL WE CALL UPON YOU TO
PROVIDE YOUR INPUT.
ALL PARTICIPANTS ALLOW SPEAKERS
TO MOVE THROUGH THEIR
PRESENTATIONS NOTING YOUR
COMMENTS OR QUESTIONS AND
NOTING THEM WHEN CALLED ON.
WE MUST ENSURE DURING THE
MEETING, DISCUSSIONS OF
CONFIDENTIAL MATTERS ARE NOT
INADVERTENTLY -- EXCUSE ME,
THAT'S ACTUALLY NOT APPROPRIATE
FOR THE OPEN SESSION.
NEXT SLIDE.
WE ARE USING THE VIDEOCAST
FEATURE TODAY.
AUDIENCE MEMBERS MAY SUBMIT
COMMENTS UNTIL JUNE 13 BLG BY
USING THE EMAIL FUNCTION
AVAILABLE ON THE NIH VIDEOCAST
WEBSITE.
OR SEND THEM TO THE NCATS
COUNCIL INBOX.
FOR FUTURE MEETING DATES IN
2024, WE WILL BE MEETING
SEPTEMBER 19-20.
IN 2025, WE ARE MEETING
VIRTUALLY IN JANUARY 30-31.
WE ARE MEETING IN PERSON MAY
22-23rd.
SEPTEMBER 18-19.
AND 2026 WE ARE MEETING
VIRTUALLY IN JANUARY, 29-30.
IN MAY WE EXPECT TO BE IN
PERSON.
THE 21-22.
AND THEN SEPTEMBER 2026 WE ARE
LOOKING FOR AN INPERSON MEETING
SEPTEMBER 17-19.
WITH THAT WE ARE GOING TO HAVE
OUR DIRECTOR'S REPORT TO THE
COUNCIL PRESENTED BY DR.
RUTTER, DIRECTOR OF NCATS.
>> Joni Rutter: THANK YOU SO
MUCH, ANNA, REALLY APPRECIATE
THAT.
GOOD MORNING, EVERYONE.
WELCOME TO THE DIRECTOR'S
REPORT OF COUNCIL.
THERE'S A LOT TO GO THROUGH SO
I WILL GO AHEAD AND JUMP RIGHT
IN.
I WANTED TO START OFF AS I
USUALLY DO WITH A SUMMARY OF
WHAT WE PREPTED LAST COUNCIL SO
YOU REMEMBER KIND OF WHAT WE
TALKED ABOUT LAST TIME.
WHEN WE MET LAST TIME WE DIDN'T
QUITE HAVE THE BUDGET IN PLACE.
WE DIDN'T QUITE HAVE OUR NCAT
SENIOR LEADERSHIP FINALIZED.
NOW I HAVE LOTS OF VIEWS ON
THIS FRONT AND I'M EXCITED TO
TALK TO YOU ABOUT THAT.
WE HAD TALKED ABOUT A VARIETY
OF OTHER PROGRAMS WE ARE MAKING
ADD VAPSES IN LIKE THE ACTIV-6
CLINICAL TRIAL WHICH IS NOW
COMPLETE.
ENROLLMENT IS COMPLETE.
WE ARE IN THE PROCESS OF DOING
ANALYSIS.
WE ARE HOPING IT DO BE
PUBLISHED IN THE NEW YEAR OR
PERHAPS END OF THE YEAR.
THAT'S EXCITING.
WE HAVE NEW APPROACH
METHODOLOGIES OR NAMS, I WILL
TALK ABOUT THAT TOO.
EDITING IS ANOTHER AREA THAT
MOVED INTO PHASE 2.
ANOTHER PROGRAM WE ARE INVOLVED
IN, WE TALKED A LITTLE ABOUT
DATA SCIENCE AND LARGE LANGUAGE
MODELS AND THE WORK IN THAT
SPACE, INCLUDING THE LITCOIN.
SOME OF THE WORK THAT'S BEEN
GOING ON IN DPI RELATED TO THE
IND'S AND NDA SUBMISSIONS.
AND I THINK WE WILL HEAR A
LITTLE MORE ABOUT THAT LATER
TODAY AS WELL.
WE TALKED ABOUT A VARIETY OF
INTERACTIONS.
THE DISCUSSION AROUND THAT
TOPIC SPECIFICALLY PROMPTED US
TO INVITE THE FDA TODAY.
BY THE FDA, I DO MEAN PETER
MARKS.
I GUESS HE ISN'T "THE" FDA BUT
HE IS A MEMBER OF THE FDA AND
WE ARE EXCITED TO HAVE HIM HERE
GIVING AN UPDATE.
WE ALSO HAD DR. MONICA
BERTAGNOLLI GIVE AN UPDATE LAST
TIME AND UP ZAIT FROM DR. BALKI
BALIKRISHNAN.
AND DAN TAGLE.
AND WE WILL HEAR MORE FROM
DOMINIQUE AS WELL.
A LOT GOING ON SINCE THEN AS
WELL.
IT TURNS OUT WE DID GET A
BUDGET SINCE WE HAVE SPOKEN
LAST TIME.
WHAT IS EXCITING, I THINK,
ABOUT THIS IS THAT NCATS WE
WERE PLANNING FOR A DECREASE IN
SUPPORT JUST BECAUSE OF THE
BUDGET SCENARIOS WE WERE
ANTICIPATING.
HOWEVER, NCATS GOT A $5 MILLION
INCREASE.
THIS INDICATES IT'S POSSIBLE TO
GET AN INCREASE EVEN IN BUDGET
MORE AUSTERE BUDGET
ENVIRONMENTS *. I THINK THAT'S
REALLY ATTRIBUTED TO A LOT OF
THE WORK YOU, AS OUR
STAKEHOLDERS DO AND INVOLVED IN
AND HELPING SUPPORT THE WORK WE
DO AT NCATS.
REALLY THIS IS A THANK YOU TO
YOU FOR ALL YOUR HELP AND
SUPPORT.
OUR SPENDING ENVELOPE INCREASED
BY $5 MILLION.
THIS IS ONE OF THE FIRST TIMES
WE HAVE EVER SEEN THIS INCREASE
IN CAN-SPECIFIC LANGUAGE.
WHERE PRIMARILY THEY HAVE GIVEN
SUPPORT TO NCATS BUT NOT
SPECIFICALLY CAN.
THIS IS A NEW CHANGE.
THE LANGUAGE MENTIONED IS WE
COULD DO, WE COULD SUPPORT CAN
UP TO, THAT'S WHAT THE LANGUAGE
HAD SAID BEFORE IN OUR REPORT
LANGUAGE.
NOW IT SAYS $75 SHALL BE
AVAILABLE FOR CAN SUPPORT.
SO THIS IS SOMETHING THAT,
WON'T BE TOO HARD TO GET USED
TO.
I THINK WE ARE EXCITED TO GET
USED TO THIS IDEA AND EXCITED
HOW TO MOVE FORWARD IN THE
SPACE TOO.
I WILL GIVE YOU A LITTLE
INKLING WHAT WE ARE PLANNING.
AT NIH MORE BROADLY THERE HAD
BEEN A $300 MILLION INCREASE IN
DISCRETIONARY FUNDING.
OVERALL THE BUDGET DECREASED.
THERE ARE CERTAIN AREAS
RECEIVING A FEW MORE DOLLARS.
YOU MIGHT BE WONDERING, WELL
YOU DID GET A $5 MILLION
INCREASE, SO WHAT ARE YOU DOING
WITH THAT?
WE WERE PLANNING MORE FOR THE
OTHER SCENARIO TO HAPPEN.
HOWEVER, WE ARE ALWAYS PLANNING
FOR DIFFERENT BUDGET SCENARIOS,
FROM THE POTENTIAL OF YOU KNOW,
PERHAPS MORE OF AN INCREASE AT
DIFFERENT LEVELS, SORRY,
DECREASE OF PARTICULAR LEVELS
WITHIN THE BUDGET WE MIGHT HAVE
TO BE PLANNING FOR.
ALSO TWO AREAS WHERE WE MIGHT
HAVE AN INCREASE.
WE HAVE BEEN TALKING ABOUT THE
IDEAS OF ALL THESE SCENARIOS
THAT COULD TAKE PLACE.
WE ARE ALWAYS FLEXIBLE IN THAT
REGARD.
WE HAVE A STRONG PLANNING
PROCESS SO WE CAN ADAPT TO ANY
SCENARIO.
WE ARE STILL WORKING OUT THE
DETAILS FOR THE $5 MILLION BUT,
ONE OF THE THINGSES WE ARE
GOING TO DO IS SUPPORT
ADDITIONAL AREAS RELATED TO
RARE DISEASES AND TISSUE CHIPS
AND I WILL TALK ABOUT MORE
PROGRAMS LATER TODAY.
BUT WE ARE ALSO TALKING ABOUT
JOINING THE FIGHT FOR MMA.
YOU MAY NOT THINK WE ARE VERY
GOOD KICK BOXERS OR MARSHAL
ARTISTS BUT THAT'S NOT REALLY
WHAT THIS IS ABOUT.
AS I DID MY RHODE --
RESEARCH AND TYPED IN MMA, IT
CAME UP WITH THIS, WHAT WAS
FASCINATING, ONE OF THE MMA
CHAMPIONS, JOHN HOWARD, HE SAID
HE ZOPT CONSIDER HIMSELF
DISABLED BUT HE HAS AUTISM.
HE SAID THROUGHOUT HIS WHOLE
LIFE HE HAD TO OVERCOME A LOT
OF DIFFERENT THINGS GOING ON IN
HIS LIFE RELATED TO THAT
PARTICULAR CONDITION.
AND SO HE WAS ALWAYS LABELD AS
SOMEONE WHO HAD A DISABILITY.
WHAT HE SAID I THOUGHT WAS VERY
POIGNANT WHAT I WILL TALK ABOUT
LATER TODAY.
HE LABELED HIS DISABILITY NOW
IS REALLY AN ADVANTAGE.
BECAUSE ONCE YOU BEAT THE
ADVANTAGE, WHAT'S GOING TO STOP
YOU NOW?
EVEN THOUGH I WASN'T REALLY
LOOKING FOR MMA, I WAS REALLY
EXCITED TO FIND THIS QUOTE FROM
JOHN HOWARD, ALSO KNOWN AS
"DOOMSDAY", MMA CHAMPION.
BUT REALLY WHAT WE ARE TALKING
ABOUT IS METHYLMALONIC
ACIDEMIA, MMA A BODY CAN'T
BREAK DOWN PROTEINS AND FATS
PROPERLY.
THIS RESULTS IN HIGH LEVELS OF
ACID IN THE BLOOD, ACIDEMIA AND
BODY TISSUES.
CHUCK VENDITTI HAS BEEN WORKING
ON MMA AND WORKING ON A GENE
THERAPY.
HE NEEDED AN EXTRA BOOST IN THE
WORK CONDUCTED TO FINALIZE THIS
GENE THERAPY INTO HUMANS.
BECAUSE OF THE SUPPORT WE HAVE
THROUGH CAN, WE ARE GOING TO BE
ABLE TO HELP SUPPORT MOVE THIS
GENE THERAPY IN HUMAN FOR MMA,
THE CLINICAL CENTER, NICHD,
CHILD HEALTH AND HUMAN
DEVELOPMENT AND NATIONAL
INSTITUTE OF NEUROLOGICAL
DISORDERS AND STROKE.
THIS IS DEVELOPING A CRUX OF
EXPERTISE AT NIH THAT CAN
SUPPORT IDEAS LIKE THIS MOVING
FORWARD.
WE ARE GOING TO LEARN, I THINK,
A GREAT DEAL ABOUT THIS.
AS YOU KNOW WE HAVE A VARIETY
OF PROGRAMS ABOUT GENE THERAPY.
THE VECTOR.
THIS IS GOING TO MOVE MORE
QUICKLY THAN BOTH THOSE THINGS.
THIS IS A CAN SPECIFIC ACTIVITY
WE WILL COLLABORATE WITH WITH
NHGRI.
NONE OF THIS CAN HAPPEN WITH
THAT CAN INCREASE UNTIL WE CAN
DEVELOP THIS PARTNERSHIP AND
GET THIS MOVE TO HELP TREAT
KIDS.
THAT IS ONE OF THE AREAS I
WANTED TO MENTION ABOUT THE
BUDGET.
THE SECOND THING IS, DURING
THIS TIME AS THE BUDGET CAME
OUT, WHICH WAS RELATIVELY LATE
IN THE CYCLE.
AROUND THE SAME TIME, LITERALLY
LIKE A DAY AFTER, THE FY 2025
BUDGET CAME OUT WITH THE
PRESIDENT'S BUMG ET.
-- BUDGET.
WHAT IS INTERESTING ABOUT THIS
AND WHAT TO NOTE ABOUT THIS
SLIDE, BECAUSE THE PRESIDENT'S
BUDGET CAME OUT A DAY AFTER THE
FY '24 BUDGET WAS FINALIZED,
THE PRESIDENT'S BUDGET WASN'T
MEASURING AGAINST FY '24, IT
WAS MEASURING AGAINST FY '23.
BEAR THAT IN MIND, IN A WAY THE
PRESENT BUDGET IS ALWAYS THE
FIRST PLACE TO START IN BUDGET
NEGOTIATIONS.
BUT THIS IS A LITTLE NUANCE
THAT IS ATYPICAL WHEN WE TALK
ABOUT THESE BUDGETS.
AND AS IT TURNS OUT THE
APPROPRIATION HEARINGS FOR THAT
BUDGET ARE HAPPENING TODAY.
SO THE QR CODE IS HERE.
WE OBSCURED IT A LITTLE
APPARENTLY SO YOU CAN'T PORT
OVER TO HEAR THAT.
I KNOW I WILL BE CATCHING UP
WITH THAT LATER ON IN THE WEEK.
SO THIS IS WHERE WE STAND NOW
AND CERTAINLY EXCITED TO HEAR
ABOUT WHAT THE HEARINGS WILL DO
NEXT.
FY '25 IS ALSO THOUGHT TO BE AN
AREA WHERE THERE MAY BE MORE
DIFFICULT BUDGET SCENARIOS BUT
WE WILL SEE.
WE ARE KIND OF USED TO
DIFFICULT SCENARIOS AND THAT'S
OKAY.
WE WILL BE READY FOR WHATEVER
HAPPENS.
SO NOW, IN TERMS OF COMINGS AND
GOINGS AT NCATS AS I MENTIONED
WE HAVE BEEN DOING A LOT IN
TERMS OF PUTTING THE LEADERSHIP
TOGETHER.
AS I HAVE SHOWN YOU THE SLIDE
IN THE PAST THERE'S BEEN LOTS
OF RED INSTEAD OF GREEN IN
THESE CATEGORIES BUT YOU WILL
NOTICE THEY ARE ALL GREEN NOW.
I'M EXCITED TO TELL YOU ABOUT
THE LEADERSHIP WE HAVE.
LATER TODAY YOU WILL BE HEARING
FROM MATT HULL AND DOMINIQUE
BUSHARD.
MATT IS NEW SCIENTIFIC DIRECTOR
FROM THE DIVISION OF
PRECLINICAL INNOVATION, AND
DOMINIQUE FROM DISEASE AND RARE.
SPEAKING OF NEW ACTIVITIES I
WILL START WITH NCATS
LEADERSHIP IN DEPUTY DIRECTOR
POSITION.
IT HAS BEEN FABULOUS TO HAVE
ANTON SIMEONOV TO STEP IN THE
ROLE OF THE ACTING DEPUTY
DIRECTOR.
HE HAS DONE IT FLAWLESSLY,
AMAZINGLY, WITHOUT SKIPPING A
STEP AND BROUGHT THE SCIENTIFIC
KNOWLEDGE TO THE TABLE.
I CAN'T THANK HIM ENOUGH FOR
SERVING IN THIS ROLE.
AND HE IS PROBABLY MOST EXCITED
TOO NOW THAT HE DOESN'T HAVE TO
SERVE IN THIS ROLE ANY LONGER,
BUT WE WON'T LET HIM GO SO
EASILY.
I'M EXCITED TO SAY, IT'S MY
PLEASURE TO SHARE TODAY THAT
DR. ANNICA WAYMAN HAS BEEN
SELECTED AS OUR DEPUTY IT
DIRECTOR.
MANY OF YOU KNOW BECAUSE SHE
WAS SITTING AT THE TABLE LAST
COUNCIL AS WELL.
WE ARE EXCITED TO HAVE HER NOW.
SHE IS REALLY THIS RARE AND
HIGHLY TUNED INDIVIDUAL WITH
HER ROLE AS A RESEARCH
SCIENTIST, A SCIENTIST
ADMINISTRATOR, AN EDUCATOR AND
A CHAMPION FOR ADVANCING
TRANSLATIONAL SCIENCES.
SHE HAS BEEN A GREAT MENTOR AND
DEVELOPED THE FUTURE OF
STUDENTS IN TRANSLATIONAL
SCIENCE IN HER FIELD.
CLINICIANS TRANSLATIONAL
SCIENCE, RESEARCHERS.
SO HER DEPTH AND BREADTH IS
REALLY QUITE MASS.
SO WE ARE EXCITED TO BRING THAT
EXPERIENCE HERE AS WELL.
SHE WILL SHARE A VARIETY OF
EXPERIENCES I HAVE, AND SHE
WILL BE CERTAINLY VERY
IMPORTANT FOR GUIDING OUR
STRATEGIC DIRECTION, OUR
STRATEGIC PLANNING AND SETTING
OUT OUR ACTIVITIES OVER THE
NEXT MANY YEARS AHEAD.
I'M LOOKING FORWARD TO HORE
LEADERSHIP.
HER LEADERSHIP.
SHE WILL BE STARTING JUNE 17
AND I WILL GOING ON VACATION
JUNE 22nd, I THINK IT WILL WORK
OUT OKAY, I APOLOGIZED
PROFUSELY FOR THAT.
ANTON WILL BE HELPING OUT.
EXCITED TO HAVE ANNICA AS OUR
DEPUTY DIRECTOR NOW.
NEXT, ANTON HAS BEEN THE
SCIENTIFIC DIRECTOR FOR MANY
YEARS AT NCATS BEFORE HE BECAME
THE ACTING DEPUTY DIRECTOR.
SO IT'S SORT OF A LENGTHY
PROCESS, BECAUSE HE STEPPED
DOWN TO BE IN A LAB AND
CONTINUING WITH HIS SCIENTIFIC
ACTIVITIES.
WE WENT THROUGH A PROCESS WITH
OUR SCIENTIFIC DIRECTOR AS WELL.
I'M ALSO EXCITED TO ANNOUNCE
THAT MATT HALL IS OUR NEW
SCIENTIFIC DIRECTOR AT NCATS.
WE SELECTED HIM THE DAY AFTER
COUNCIL LAST TIME.
THE TIMING WASN'T QUITE RIGHT.
BUT NOW HE IS AN OLD HAT.
SO NO PROBLEM AT ALL.
WE HAVE SORT OF PUT HIM IN
FRONT OF, AND HE WILL BE ABLE
TO TELL YOU MORE ABOUT HIMSELF,
SO I WON'T DWELL ON HIM HERE,
BUT I'M REALLY EXCITED TO HAVE
HIM AT THE TABLE AS WELL TO
CONSIDER HOW WE CAN SET THE
STAGE FOR NCATS LABS AND MOVE
FORWARD IN STRATEGIC PLANNING
IN THE DECADE AS HEAD.
HE HAS DONE HIS LISTENING
SESSIONS AS WELL AND I'M SURE
HE WILL TALK TO YOU MORE ABOUT
THAT.
IN ADDITION WE HAVE A NEW
POSITION AT NCATS.
CHIEF OF STAFF, THIS IS A
POSITION GROWING I WOULD SAY
ACROSS THE NIH.
THIS IS SOMETHING WE DIDN'T
REALLY HAVE AT NCATS IN TERMS
OF INDIVIDUALS SUPPORTING THIS
SORT OF DEFINED ROLE.
AND WHAT, IF YOU LOOK AT CHIEF
OF STAFF POSITION THEY HAVE
VERY DIFFERENT ACROSS THE NIH.
FOR US, GIVE ESPECIALLY THAT WE
ARE THE DOT CONNECTORS, WE WORK
ACROSS DOT-COM, DOT-EDU,
DOT-GOV AND DOT-ORG.
SHE REPRESENTS OD AND NCATS AT
LARGE FOR THESE VARIOUS
OUTREACH ACTIVITIES AND
COLLABORATIONS WORKING ACROSS
NIH, DIFFERENT GOVERNMENT
AGENCIES, ACADEMIA AND INDUSTRY.
SHE HAS HIT THE GROUND RUNNING.
LEADING THE EFFORTS WITH THE
ARPA H FOLKS AS WELL.
BRINGING IN SOMEONE DEDICATED
TO THOSE EFFORTS IS GREAT FOR
US AND I THINK WILL HELP US
MATURE AS AN ORGANIZATION AS
WELL.
SHE SERVED IN A VARIETY OF
CAPACITIES AT NIH.
SHE STARTED AT NCI, FOCUSING ON
BACTERIA POST RNA
TRANSCRIPTIONAL, AND THEN
STRATEGIC PREPAREDNESS AND
RESPONSE.
AND THAT'S WHERE SHE WAS
INVOLVED IN DEVELOPING A LOT OF
THERAPEUTIC AND VACCINE
STRATEGIES FOR THE RESPONSE TO
MONKEY POX OUTBREAK, EBOLA
PREPAREDNESS AND COVID-19 AS
WELL.
SHE IS TRAINED AS CHEMIST AND
MICRO BIOLOGIST AND RECEIVED
HER BACHELOR AT UNIVERSITY OF
NORTH CAROLINA, CHARLOTTE AND
DOCTORATE AT --
AND THEN NEXT I WOULD LIKE TO
INTRODUCE DR. JOSH FESSEL.
HE IS ALSO NOT A NEW FACE
WITHIN NCATS.
HE HAS BEEN A SENIOR DIVISOR
BUT NOW SERVING IN THE ROLE OF
OFFICE OF TRANSLATIONAL
MEDICINE AND THE DIRECTOR IN
THAT ROLE.
THIS IS SORT OF A NEW-ISH
OFFICE, IT'S BEEN IN PLACE NOW
ABOUT FOUR YEARS.
HE IS THE SECOND DIRECTOR IN
THIS ROLE.
HE BRINGS A WEALTH OF CLINICAL
RESEARCH EXPERIENCE AND REAL
WORLD EXPERIENCE AND WE ARE
EXCITED TO HAVE THAT EXPERTISE
AT THE TABLE, SORRY, I'M
HEARING AN ECHO, HOPEFULLY THAT
WILL -- OKAY.
SHE IS WORKING ON IT.
YOU MIGHT HAVE TO LOG OUT.
SOUNDS LIKE IT RESOLVED?
IT HAS.
GOOD.
OKAY, WITH JOSH FESSEL NOW AT
AS THE HEAD OF THE OTM HE WILL
BE ABLE TO FOSTER HOW WE THINK
OF INTEGRATING PROGRAMS AND
INNOVATING ACROSS NCATS.
SO REALLY BRINGING SOME OF THAT
GLUE TO THE TABLE.
IN ADDITION TO THAT, HE WILL BE
WORKING VERY CLOSELY WITHIN OUR
INTRAMURAL PROGRAM.
STRENGTHENING INTERACTIONS WITH
THE CLINICAL CENTER AND
CLINICAL COUNTERPARTS.
SO THERE'S A VARIETY OF THINGS
ON HIS PLATE ALREADY.
HE ALSO HAS WITHIN HIS PURVIEW
TOPICS OF HUMAN SUBJECTS
RESEARCH, DIVISION EQUITY
INCLUSION SENSIBILITY.
DRUG DEVELOPMENT, AS WELL AS
CLINICAL TRIALS AND
ENVIRONMENTAL ICS.
IC -- ETHICS.
WE HAVE A PROGRAM IN
TRANSLATIONAL SCIENCE, ETHICS
OF TRANSLATIONAL SCIENCE THAT
ELAINE COLLIER LEADS.
THE A SMALL BUT MIGHTY PROGRAM.
UNDER THE PURVIEW OF OTM.
SO JOSH, ALSO IN ADDITION TO
ALL THAT, HE SERVES AS,S HE IS
CO-CHAIRING A SUBGROUP ON
LOOKING AT ELECTRONIC HEALTH
RECORDS FOR NIH AND HOW WE DO
THIS MORE BROADLY.
BEING MORE COORDINATED IN OUR
GOALS OF PURSUING REAL-WORLD
DATA AND ELECTRONIC HEALTH, I
SERVE AS THE CO-CHAIR OF ONE OF
THESE COMMITTEES, CALLED THE
DATA SCIENCE POLICY COUNCIL.
THIS IS AN IMPORTANT ACTIVE IN
THAT SPACE AND JOSH IS
COORDINATING.
SO THAT IS EXCITING, BECAUSE HE
HAS ALSO DONE THIS FOR THE CTSA
PROGRAM.
SO THERE'S A WEALTH OF
EXPERTISE HE BRINGS TO THE
TABLE.
I THINK THIS IS THE LAST CHANGE
I WILL MENTION TODAY.
VEPA IS THE SENIOR LICENSING
PATENT MANAGER IN THE OFFICE OF
STRATEGIC ALLIANCES, WORKING
WITH BALKI.
HE JOINED NIH IN 2005, SERVED
IN A VARIETY OF CAPACITIES IN
THAT TIMEFRAME.
HE KIND OF TOOK A BREAK AND WAS
TEACHING AT THE UNIVERSITY OF
MARYLAND SCHOOL OF LAW AND
SUPERVISED ATTORNEYS ENROLLED
IN THE MARYLAND INTELLECTUAL
PROPERTY AND LEGAL RESEARCH.
AND HE CAME BACK TO NCATS AND
WORKING IN OUR OFFICE OF
STRATEGIC ALLIANCES AND WE ARE
DELIGHTED TO HAVE HIS EXPERTISE
AS WELL AND BE AT THE LEVEL OF
SENIOR LEADERSHIP AT O.S.I.
HE RECEIVED J.V. WITH HONORS
UNIVERSITY OF MARYLAND AND
BIOCHEMISTRY IN BANGALORE.
HE HAS THE CHOPS, AND THAT
SHOWS.
HE IS REALLY GREAT TO HAVE.
ALL RIGHT, ONE OTHER THING I
WANTED TO HIGHLIGHT, OUR
EXECUTIVE OFFICER, KEITH
LAMIRANDE RECEIVED ONE OF THESE
LIFE AWARDS.
THIS IS A VERY PRESTIGIOUS
AWARD, THE PRESIDENTAL RANK
AWARD.
CONFERRED TO A FEW INDIVIDUALS
ACROSS THE GOVERNMENT.
THEY RECOGNIZE OUTSTANDING
LEADERS WHO CONSISTENTLY
DEMONSTRATE, STRENGTH,
INTEGRITY, INDUSTRY AND MADE
SIGNIFICANT CONTRIBUTIONS TO
UNITED STATES U.S. GOVERNMENT.
SO KEITH HAS SPENT ABOUT 35
YEARS WITHIN THE NIH WORKING IN
LABS AND MOVING OVER TO
ADMINISTRATION.
AND I CAN'T THINK OF ANYONE
MORE WORTHY THAN KEITH TO GET
THIS HONOR.
SO I WANTED TO TAKE A MOMENT TO
CONGRATULATE KEITH ON THIS WORK.
I WILL SAY THAT OVER THE PAST
FOUR YEARS SINCE I HAVE BEEN AT
NCATS, KEITH HAS REALLY WORKED
VERY HARD TO HELP GROW NCATS.
WE HAVE HIRED OVER 150 PEOPLE
LAST YEAR.
WE ARE GROWING, LARGELY BECAUSE
OF THE SMOOTH ADMINISTRATION
OPERATIONS WE HAVE WITH KEITH
ADD OFFICE OF ADMINISTRATIVE
SCIENCES.
I WANT TO SAY THANK YOU TO
KEITH AND CONGRATULATE HIM.
WHILE I'M CONGRATULATING HIM,
I'M ALSO KURTSING HIM.
-- CURSING HIM.
HE DECIDED 35 YEARS IS A LONG
TIME, SO HE RECENTLY ANNOUNCED
HIS RETIREMENT AS WELL.
I WANT TO TAKE A MOMENT TO SAY
TO THE COUNCIL HE WILL BE
RETIRING, I BELIEVE AT THE END
OF JUNE.
AND SO THIS IS OUR LAST
OPPORTUNITY TO WISH HIM A
FAREWELL HERE WITHIN COUNCIL.
NOW AT THE SAME TIME, WE HAVE
ANNOUNCED THIS WEEK THAT BEKAH
GEIGER WILL BE ACTING EXECUTIVE
OFFICER, SHE HAS BEEN DEPUTY
EXECUTIVE OFFICER NOW FOR A FEW
MONTHS AND SHE WILL BE STEPPING
INTO THIS ROLE, VERY ABLY, VERY
MUCH LOOKING FORWARD TO WORKING
WITH BEKAH AS WELL.
I KNOW WE ARE IN VERY GOOD
HANDS.
SHE HAS BEEN ALSO A LONG-TERM
CHAMPION IN NIH AND SERVED IN A
VARIETY OF CAPACITIES.
HELPING AT THE DEPUTY EXECUTIVE
LEVEL AND I LOOK FORWARD TO HER
LEADERSHIP AS WE DO AN ACTIVE
SEARCH FOR THIS POSITION.
WE ARE KIND OF DONE WITH
LEADERSHIP, NOT QUITE BUT WE
ARE GOING TO GET THERE.
ALL RIGHT.
JUST A FEW OTHER THINGS I
WANTED TO MENTION TOO, AT THE
NIH LEVEL WE ARE RECRUITING NOW
FOR A DIRECTOR OF THE NATIONAL
LIBRARY OF MEDICINE.
PATTY BRENNAN STEPPED DOWN
ABOUT 6-9 MONTHS AGO NOW.
AND THE APPLICATION PERIOD FOR
THIS CHOICES ON MONDAY JULY 1st.
SO IF YOU KNOW ANYONE WHO MIGHT
BE INTERESTED IN THIS
PARTICULAR POSITION, PLEASE
RELAY THIS ANNOUNCEMENT TO THEM.
THIS POSITION OFFERS A VERY
UNIQUE, I THINK EXCITING
OPPORTUNITY BECAUSE THE NLM, I
THINK IT'S A LIBRARY, BUT IT'S
MORE THAN A LIBRARY.
I THINK THERE'S A LOT OF
ACTIVITY THE NLM IS DOING NOW,
BUT ALSO WITH DR. BERTAGNOLLI
AND HER VISION, I SEE IT AS
REALLY THINKING MORE OF A DATA
HUB AND CONVENING RELEVANT DATA
FOR SCIENCE AND ANALYTICS IN
THE FUTURE TOO.
THERE MAY BE REALLY GREAT
OPPORTUNITIES FOR PEOPLE WHO
MAY BE INTERESTED IN THIS
POSITION.
I SHOULD SAY TOO, WE HAVE BEEN
PRETTY SUCCESSFUL BRINGING
PEOPLE INVOLVED IN NCATS
SUPPORTED ACTIVITIES,
ESPECIALLY THE CTSA *. JOSHUA
LEVY JOINED THE NIDCD RECENTLY,
HE IS THE CLINICAL DIRECTOR
THERE, AND SEAN MOONEY WAS
HIRED AND CAME FROM A CTSA
PROGRAM.
WHO KNOWS MAYBE WE WILL SEE
ANOTHER LEADERSHIP.
DR. DEB TUCEY, SHE WAS FROM THE
CTSA.
WENDY IS THE DIRECTOR OF NIAM,
SHE WAS FROM CTSA.
YOU NEVER KNOW, WE MAY GET MORE
FOLKS FROM CTSA JOINING AT THE
NIH LEVEL TOO.
HE IS MOVING VERY QUICKLY.
I WANTED TO SAY ONE OF THE
THINGS HE HAS DONE SINCE HE HAS
BEEN AT CIT.
NIH HAS GREATLY BENEFITED FROM
THE STRIDES PROGRAM.
THIS IS A CLOUD-BASED PROGRAM
WE HAVE.
IT'S REALLY BEEN FOCUSED ON THE
INTRAMURAL PROGRAMS AT NIH.
NOW HE IS OPENING THIS TO THE
EXTRAMURAL COMMUNITY.
I WANT TO MAKE SURE YOU ARE ALL
AWARE OF THIS.
THIS IS AN OPPORTUNITY TO GET
CLOUD-BASED EXPERIENCE AT NO
COST.
THIS IS KIND OF A TRY BEFORE
YOU BUY AND YOU GET $500 IN
CREDITS.
YOU CAN LEARN THE ROPES AND SEE
IF THE SOMETHING YOU WANT TO DO
MORE OF.
THIS IS ONE OF THE ACTIVITIES
HE HAS BROUGHT TO THE TABLE
SINCE HE JOINED THE NIH AND I
WANTED TO MAKE SURE YOU WERE
AWARE OF THAT.
A FEW MORE PIECES OF ACTIVITIES
WE HAVE BEEN DOING.
THIS YEAR RARE DISEASE DAY FELL
ON FEBRUARY 29th AND THAT IS A
RARE, RARE DAY.
ON FEBRUARY 28th THE WHITE
HOUSE HELD FOR THE FIRST TIME A
RARE DISEASE FORUM, AND I WAS
HONOR TODAY BE A PART AS WELL
AS ANNIE KENNEDY.
SHE WAS ALSO MODERATING A
SESSION THAT DAY.
I HOPE THIS IS SOMETHING THE
WHITE HOUSE DID DO, IT RAISED
AWARENESS OF RARE DISEASES.
I AM GRATEFUL FOR THEIR
RECOGNITION OF DOING THAT.
IN ADDITION ON THE RARE DISEASE
DAY AT NIH WAS THE DAY AFTER.
AGAIN, IT WAS A VERY RARE DAY.
ONE HIGHLIGHT I WANT TO
MENTION, DOMINIQUE WILL TALK
MORE ABOUT RARE DISEASE DAY, SO
I DON'T WANT TO STEAL HER
THUNDER.
ONE THING THAT HAPPENED ON THIS
DAY, IT WAS PRETTY SPECIAL, THE
CURE JM ADVOCATE PRESENTED
PROCLAMATION AND THANKS TO
NCATS.
I WAS PRESENTED THIS PLAQUE AND
HONORED TO RECEIVE IT.
JAMES BEST WAS ONE PRESENTING
IT TO ME, SOMEONE I REALLY LOOK
UP TO, LITERALLY AND
FIGURATIVELY.
[LAUGHTER]
BUT TAKE A LOOK AT THIS.
THEIR PROCLAMATION OF THANKS
WAS OVER 600 SIGNATORIES FROM
THE CURE JM COMMUNITY.
JUST A QUOTE FROM ONE OF THE
FRIENDS OF SOMEONE WITH JM, SHE
SAID I'M SO PROUD OF YOUR
ABILITY TO WAKE UP EACH DAY AND
GIVE IT YOUR BEST NO MATTER
WHAT.
I HOPE NIH WILL EARMARK MORE
FUNDING FOR THE VITAL RESEARCH
AND DEVELOPMENT OF LIFE-SAVING
TREATMENTS FOR RARE DISEASES.
THIS WAS HEART WARMING TO
RECEIVE A DEEP NOTE LIKE THIS
FROM PATIENTS, PARENTS,
GRANDPARENT AND FRIENDS OF JM.
I AM PLEASED TO SHARE THIS SO
YOU CAN SEE IT.
WE SENT IT AROUND THE NCATS
COMMUNITY SO THEY WOULD ALL BE
AWARE OF IT.
THIS WAS A DEEPLY TOUCHING
MOMENT FOR US.
IN ADDITION TO SOME OF THOSE
ACTIVITIES ON RARE DISEASE DAY,
WE HAVE A VARIETY OF CHORES.
SO WE HAVE A VARIETY OF PEOPLE
COMING THROUGH TO DO TOURS.
I WANT TO SHOW THE TOURS.
THIS IS ED MANACHURIAN, THE
FOUNDER OF ACT FOR NIH ALONG
WITH RICHARD TURMAN, THE KRRKTS
EO.
THEY WERE ABLE TO COME IN AND
TAKE A NICE TOUR.
THAT'S ALWAYS A NICE THING TO
GIVE THEM A SENSE HOW WE DO
DRUG DEVELOPMENT AND WE
PIXELATE DRUG DEVELOPMENT IN
THAT WAY.
IN ADDITION TO THE ACT FOR NIH,
WE HAD THE EUROPEAN MEDICINES
AGENCY.
WE HAVE BEEN ENGAGING QUITE A
BIT WITH THE FDA BUT ALSO WITH
THE EUROPEAN MEDICINES AGENCY
AS WELL.
WE HAD THE CHIEF MEDICAL
OFFICER COME TO VISIT US.
WE WERE ABLE TO EXPLAIN SOME OF
THE WORK WE HAVE BEEN DOING AT
THE NCATS LABS, TALKING ABOUT
DRUG REPURPOSING, RARE
DISEASES, NEW APPROACH
METHODOLOGIES, ALL THESE THINGS
WE HAVE BEEN TALKING ABOUT,
THEY ARE ALSO INTERESTED IN.
FORMING INTERACTIONS AND
PARTNERSHIPS WITH THE EMA AND
FDA IS INCREDIBLY IMPORTANT FOR
US.
IN ADDITION WE HAVE BEEN DOING
QUITE A NUMBER OF CONGRESSIONAL
BRIEFINGS AND ACTIVITIES.
THIS IS A VERY BUSY SLIDE AND
SOME OF THE IMAGES OVERLAP WITH
SOME OF THE IMPORTANT THINGS
HERE.
I WILL SEE IF I CAN GET RID OF
THE, MAYBE I CAN'T.
ANYWAY, HOPEFULLY YOU WILL GET
THE IDEA.
WE HAVE DONE QUITE A NUMBER OF
CONGRESSIONAL BRIEFINGS WITH
STAFFERS AND WITH DIFFERENT
MEMBERS AROUND CONGRESS.
FOLKS IN DRUG REPURPOSING, RARE
DISEASES, WE TALK A LOT ABOUT
N3C AND ARTIFICIAL INTELLIGENCE
AND QUANTUM RESEARCH.
AND SO THIS IS, WE WERE
ACTUALLY DOWNTOWN YESTERDAY AT
A DEMONSTRATION FOR THE
NATIONAL AI RESEARCH RESOURCES.
THIS IS A PROGRAM THAT NSF RUNS
BUT WE ARE A PART OF IT, AND
N3C IS A PART OF IT, NIAED.
SUSAN GREGURICK IS WITH ODSS.
SAM HAS A TIE ON, HE IS TURNED
AROUND HERE, TALKING WITH THE
LEGISLATIVE FELLOW.
SENATOR HEINRICH'S OFFICE.
WASEM GAWISH AND TALK WITH
REPRESENTATIVE HALEY STEVENS,
ANNA ESHOO, BILL FOSTER.
THEY ARE INCREDIBLY EXCITED
ABOUT A.I. AND HEARING ABOUT
N3C, THEY ASKED A LOT OF
QUESTION.
THAT'S FANTASTIC TO SEE.
SORT OF LAST IN THIS SESSION, I
WANT TO MENTION WE ARE MOVING.
NCATS WILL NO LONGER BE IN THE
DEMOCRACY BOULEVARD SPACE.
THEY WILL PROBABLY BE IN THE
NATIONAL CANCER INSTITUTE
FACILITIES.
LATER IN JUNE.
WE HAVE ANOTHER MONTH OR SO.
WE LOOK FORWARD TO MOVING.
KIND OF BEING CO-LOCATED IN THE
SAME SPACE.
NOT ONLY WILL WE BE IN THE SAME
SPACE AND BUILDING FOR
EXTRAMURAL, WE WILL BE 15
MINUTES AWAY FROM THE NCATS LAB
BY FOOT, YOU DON'T HAVE TO
DRIVE.
JUST WANT TO BRIEFLY RUN
THROUGH SOME PROGRAMMATIC
UPDATES.
YOU WON'T SPEND TOO MUCH TIME.
WE ARE STILL WORKING THROUGH
THE NOVEL APPROACH IN THE
COMPLIMENTARY.
THIS IS THE COMPLEMENT ANIMAL
RESEARCH IN EXPERIMENTATION.
BRING IN DIGITAL AND LOOKING AT
mRNA AND OTHER ASSAYS.
WE HOPE THIS WILL BE A VERY
EFFECTIVE WAY OF DEVELOPING
BETTER TOOLS THAT ARE
PREDICTIVE TOOLS FOR MORE
EFFICIENT EFFECTIVE RESEARCH IN
BIO MEDICINE.
STAY TUNED FOR MORE WORK IN
THIS SPACE.
HOPEFULLY AT OUR NEXT COUNCIL
WE WILL BE ABLE TO GIVE YOU A
LITTLE MORE ABOUT THAT.
I WANT TO HIGHLIGHT ONE OF THE
PROGRAMS CHRISTINED TOUCHED ON
EARLIER WHEN WE WERE IN OUR
CLOSED SESSION TODAY.
THIS IS AN INITIATIVE ON THE
PRE-CLINICAL PROOF OF CONCEPT
STUDIES FOR RARE DISEASES.
THANKS, STEVE.
THIS HAS CREATED A MASSIVE
RESPONSE FROM THE COMMUNITY.
SO MUCH WE AREN'T ABLE TO FUND
THE REALLY GOOD SCIENCE.
OUR GOAL IS TO PROVIDE FUNDING
TO TEST EFFICACY OF NOVEL
AGENTS IN ESTABLISHED RARE
DISEASES, ESPECIALLY
PRECLINICAL MODELS.
SUPPORT EFFICACY TESTING FOR
REPURPOSING OF APPROVED
THYROPUET ICS TO TREAT RARE
DISEASES.
THIS REQUIRES PARTNERSHIP PLAN
WITH RARE DISEASE STEERING AND
OVERSIGHT COMMIT EYES
CONSISTING OF EXPERTS,
PATIENTS, PATIENT ADVOCATE.
TR-24-023.
HERE IS YOUR QR CODE IF YOU ARE
INTERESTED IN THIS, WE HOPE YOU
APPLY.
YOU CAN SEE THE BREAK DOWN OF
THE MODALITIES WE ARE LOOKING
AT HERE.
QUITE A NUMBER OF MODALITIES,
INDICATION AREAS.
AS WELL AS THE REPURPOSING OR
NOT APPROACHES HERE TOO.
QUITE A SMATTERING OF
ACTIVITIES IN RESEARCH.
A GREAT PROGRAM.
EARLIER THIS YEAR WE HAD THE
ASSOCIATION FOR CLINICAL AND
TRANSLATIONAL SCIENCE AWARD
MEETING.
THIS IS THE TRANSLATIONAL
SCIENCE 2024 IS WHAT IT IS
KNOWN AS.
THIS WAS HELD IN LAS VEGAS.
WE WERE STANDING IN FRONT OF
THE FAMOUS FOUNTAINS BUT IT
WASN'T GOING OFF BECAUSE IT WAS
WINDY SO WE HAVE OUR SAD FACES
ON.
THIS IS AN INCREDIBLE INSPIRING
AND ENGAGING MEETING.
I WANTED TO START THIS OFF AS
HIGHLIGHTING KEY AREA OF
SCIENCE MENTIONED.
THIS IS ONE AREA OF SCIENCE
THAT RECEIVED A CLINICAL
RESEARCH ACHIEVEMENT AWARD.
TOP TEN.
S THIS IS A PROGRAM KNOWN AS
GEMINI OUT OF TUFTS UNIVERSITY.
THEY TOOK TARGET SEQUENCING
SPECIFIC GENES THAT MAY BE
RELATED TO SPECIFIC DISEASES
AND HOLDING.
AND THEY COMPARED THE TWO
APPROACHES.
WHAT THEY FOUND WAS ESSENTIALLY
WHEN YOU COMPARE WHOLE GENOME
SEQUENCING, WHEN YOU DID IT, IT
DIDN'T REPORT 19 OF THE
VARIANTS FOUND BY TARGET
SEQUENCING.
ON THE OTHER HAND TARGET
SEQUENCING DIDN'T REPORT 164
VARIANTS THAT WHOLE GENOME
SEQUENCING IDENTIFIED.
SO WHOLE GENOME SEQUENCING WITH
THE WIN HERE IDENTIFYING MORE
IMPORTANT VARIANTS.
NOW THE OTHER INTERESTING THING
THEY LOOKED AT WAS THE MEDIAN
TIME TO RETURN THE TEST RESULTS.
IT TOOK ABOUT FOUR DAYS TO GET
THE TARGETED GENE SEQUENCING
RESULTS AND A LITTLE OVER SIX
DAYS TO GET THE WHOLE GENOME
SEQUENCING RESULTS.
THE NUMBER OF VARIANTS
IDENTIFIED, PERHAPS A LITTLE
BIT LONGER ISN'T A BHATT BAD
THING.
-- BAD THING.
I WOULD ONLY IMAGINE THAT WOULD
GET BETTER OVER TIME.
THIS IS PROVIDING EVIDENCE HOW
IMPORTANT WHOLE GENOME
SEQUENCING IS ESPECIALLY IN THE
RARE DISEASES SPACE.
THIS IS A PROGRAM CALLED EARLY
CHECK.
ALSO OUT OF THE CPSA PROGRAM TO
ASSIST THE IA, COLLABORATIVE
INNOVATION AWARD.
THIS IS A LARGE SCALE PROGRAM
IN NORTH CAROLINA.
THEY DO THREE SCREENING
RESEARCH PROGRAMS HERE.
THE DESIGNED TO IDENTIFY
CHILDREN WITH RARE HEALTH
CONDITIONS BEFORE SYMPTOMS
APPEAR.
THEY STARTED BY SPINAL
HUFF-THEY STARTED THIS IN 2018.
AS OF SEPTEMBER EARLY CHECK
SCREENED MORE THAN 10,000 BABY
AS CROSS NORTH CAROLINA AND THE
FAMILIES WERE INVOLVED IN THIS
VIRTUAL RECRUITMENT.
SO THIS IS ALL DONE RIGHT
BEFORE THE PANDEMIC HIT.
BUT I THINK REALLY SET THE
STAGE WELL FOR HAVING THIS MOVE
FORWARD.
AS A RESULT OF THE PROJECT,
BECAUSE THEY HAD BEEN LOOKING
FOR FRAGILE X AND SMA, THEY
WERE ABLE TO ADD THOSE TO THE
STANDARD NEWBORN SCREENINGS IN
NORTH CAROLINA WHICH IT WASN'T
ON BEFORE.
THIS IS MAKING A DIFFERENCE
ADDING THE RARE DISEASES TO THE
TABLE BUT ALSO GARNERING A LOT
OF EVIDENCE NEEDED FOR WHOLE
GENOME SEQUENCES AND HOW
IMPORTANT IT IS FOR NEWBORN
SCREENING.
SOMETHING TO KEEP YOUR EYE ON.
I WOULD SAY EARLY CHECK
SPECIFICALLY ALSO LOOKS FOR
TYPE I DIABETES AND PROVIDES
COUNSELING FOR THE CASES WHERE
THEY IDENTIFY THOSE.
I WANT TO BRIEFLY MENTION THIS,
THE KIND OF A DIFFERENT
TECHNOLOGY.
THIS IS OUT OF WASH-U.
ST. LOUIS TEAM, USED A
STREAMLINE WHOLE GENOME
SEQUENCING TO OBTAIN GENETIC
PROFILES FOR PATIENTS WITH
MYELOID CANCERS.
WHEN THEY DID THIS, THEY DID
WHOLE GENOME SEQUENCING.
ALL 40 AND 91 COPY NUMBER
ALTERATIONS THAT HAD BEEN
IDENTIFIED THROUGH CYTOGENETIC
PROCESSES AS WELL SORT OF THE
STANDARD PRACTICE.
BECAUSE THEY WERE ABLE TO GET
AT ALL OF THOSE, IT GAVE US
HIGH CONFIDENCE THIS MAY BE
ANOTHER TOOL TO USE IN
IDENTIFYING THOSE SPECIFIC
GENETIC ABNORMALITIES
ASSOCIATED WITH THESE DISEASES.
IT COULD HELP US IDENTIFY MORE
ACCURATE TREATMENTS FOR THESE
VERY SPECIFIC DISEASES.
SO IT LOOKS VERY PROMISING.
ONE MORE PROGRAM, ACTUALLY TWO
MORE PROGRAMS I WANT TO MENTION.
THESE ARE TWO COLLABORATIVE
CTSA GENOMES PROGRAMS.
ON THE LEFT SIDE THIS IS
LOOKING AT SORT OF A REAL-WORLD
RANDOMIZED CLINICAL TRIAL STUDY
ON IMPACT OF WHOLE GENOME
SEQUENCING IN ETHNICALLY AND
RACIALLY DIVERSE POPULATION OF
HEALTHY INFANTS.
THIS IS ROBERT GREEN AND HIS
COLLEAGUES.
THEY DEVELOPED THIS IDEA OF
DOING GENOME SEQUENCING IN
THESE KIDS AS A WAY TO SCREEN
IN UNDERSERVED COMMUNITIES WITH
THE GOAL IN MIND TO DETERMINE
HOW IT COULD BECOME MORE
ROUTINE IN PEDIATRIC CARE.
LEARNING ESSENTIALLY THIS IS A
PRETTY STRAIGHT-FORWARD
APPROACH AND HIGHLY REGARDED AS
A WAY OF MOVING FORWARD OR
IDENTIFYING DISEASES IN THESE
KIDS.
ONE MORE OF THESE COLLABORATIVE
PROGRAMS I WANT TO MENTION IS
ON THE RIGHT-HAND SIDE WITH KEN
MENDEL.
HE IS OUT OF BOSTON.
THIS IS THE GENOME INFORMATION
COMMON.
FIND DATA WITH BIO SPECIMEN
DATA AT THIS SCALE.
I WANT TO HIGHLIGHT THAT AS A
RESOURCE THAT'S PROVEN HIGHLY
VALUABLE IN THIS SPACE.
AND WITH JUST SORT OF RIFFING
OFF WHAT DR. BERTAGNOLLI
PRESENTED, THIS IS A VISION SHE
HAS BEEN BRINGING TO THE NIH
ESTABLISHING A RESEARCH NETWORK
IN PRIMARY CARE SETTINGS.
I'M EXCITED ABOUT PLAYING IN
THIS ROLE.
I'M ACTUALLY CO-CHAIRING SOME
ACTIVITIES RELATED TO THIS
PARTICULAR PROGRAM AND PROJECT.
SO I WILL RUN THROUGH IT VERY
QUICKLY HERE.
AS YOU PROBABLY ARE ALL AWARE,
OUR LIFE SPAN IN THE U.S. IS
ACTUALLY GETTING SHORTER OVER
TIME.
AND AS WE RANK IN FIRST WORLD
COUNTRIES WE ARE AROUND 35.
THE ACTUALLY GOING DOWN EVEN
MORE.
SO THIS IS ONE OF THOSE THINGS
WE ARE TRYING TO UNDERSTAND
WHAT THE REAL ISSUES ARE.
BUT WE KNOW WHAT SOME OF THEM
ARE FOR SURE.
THAT IS DESPITE HAVING HIGHEST
HEALTH EXPENDITURES IN THE
WORLD, WE ARE SEEING MORE AND
MORE GAPS IN HEALTH DISPARITIES
AND THOSE IN UNDERSERVED
COMMUNITIES.
THE GOAL OF THIS PROGRAM IS
STARTING TO ADDRESS THOSE
DISPARITIES, SPECIFICALLY
MOVING MORE INTO THE PRIMARY
CARE SPACE.
THAT'S WHAT THESE ACTIVITIS ARE
MEANT TO DO.
THE APPROACH IS SHOWN HERE TO
EXPAND ENROLLMENT EFFORTS IN
EXISTING NIH STUDIES AND PILOT
WAYS TO GET INTO THE PRIMARY
CARE COMMUNITIES.
WE WANT TO PARTNER WITH
EXISTING CLINICAL RESEARCH
NETWORKS AND RESOURCES TO
EXPAND THAT GROWTH.
ENGAGE WITH MORE SITES IN THIS
WORK.
AS YOU GO TO THE DOCTOR YOU
PROBABLY SPEND 15 MINUTES WHEN
YOUR PRIMARY CARE DOCTOR, THIS
WON'T BE AN EASY OR STRAIGHT
FORWARD THING TO DO, WE DON'T
WANT TO ADD BURDEN TO THE
PRIMARY CARE ENVIRONMENT OR THE
PHYSICIANS WHO HAVE LITTLE TIME.
THE MORE WE CAN DO TO
STREAMLINE ACTIVITIES, YET
AGAIN MAKE ADVANCES TO MOVE
INTO THAT PRIMARY CARE SPACE,
THIS IS WHERE WE ARE TRYING TO
MAKE THE CHANGES.
WE ARE TRYING TO IMPLEMENT THE
STUDY DESIGN, WORKING WITH
THESE NETWORK PARTNERS TO
FIGURE OUT HOW TO DO SUSTAINED
COMMUNITY ENGAGEMENT AND
IMPROVE ON CLINICAL INNOVATIONS
WITHIN THE PRIMARY CARE SETTING.
WE RECEPTLY CAME OUT WITH A
ROA, ANOTHER TRANSACTION AWARD
ANNOUNCEMENT.
WE WANT TO MAKE SURE IT'S AT
LEAST OUT OF THE GATE.
MORE OF A PILOT STAGE ACTIVITY.
WITH THE ROA WE HOPE TO LAUNCH
THIS BY END OF THE YEAR.
WE ARE ON A QUICK TIMELINE TO
DO THIS.
WE ARE STARTING OFF WITH $5
MILLION IN FY '24.
WE WILL EXPAND THIS, WE HOPE,
IN YEARS TO COME.
I HOPE YOU HAVE HAD A CHANCE TO
LOOK AT THIS RESEARCH
OPPORTUNITY.
HERE IS THE LINK TO THAT
OPPORTUNITY.
AND THE NUMBER SO YOU CAN
GOOGLE IT AND FIND OUT A LITTLE
BIT MORE.
I WANT TO TAKE A SPECIFIC NOTE
ON THE ELIGIBILITY.
THIS IS LOCATED IN STATE
JURISDICTIONS AT LEAST 25% OF
CENSUS TRACT DEFINED AS RURAL.
THIS IS DEFINED BY OUR RUCA
CODES.
THIS IS WHERE WE START,
PROBABLY NOT WHERE WE END, BUT
WHERE IT WILL START IN TERMS OF
ELIGIBILITY.
THERE ALSO HAS TO BE FUNDING TO
THE NIH INSTITUTIONAL
DEVELOPMENT ALWARD, CLINICAL
AND TRANSLATIONAL LAB.
AND THE NHL CLINICAL AND
TRANSLATIONAL SCIENCE PROGRAM
AS WELL.
OUR CTSA PLEASE STAY TUNED FOR
THAT.
AND RESEARCH INSTITUTE NETWORK
AS WELL.
THESE ARE THE INITIAL ELIGIBLE
INSTITUTIONS THAT WILL BE
CONSIDERED FOR THIS.
AND THEN IF WE HOPE TO EXPAND
IT GOING FORWARD.
ALL RIGHT, SO I WILL END WITH A
LITTLE BIT OF A TEASER.
WE WILL HAVE KIND A GAP BETWEEN
NOW AND A LATER COUNCIL
DISCUSSION WE WILL HAVE THAT I
HOPE YOU HAVE ALL RECEIVED OUR
STRATEGIC PLAN AND HAVE YOU HAD
A LOOK AT THE STRATEGIC PLAN
AND WILL BE READY TO ENGAGE IN
THIS CONVERSATION.
BUT WE ARE NOW AT THE POINT IN
TIME THIS IS YOUR LAST CHANCE
TO PROVIDE THAT INPUT.
AND I LOOK FORWARD TO
DISCUSSING THIS WITH YOU.
AS YOU KNOW, WE HAVE FIVE GOALS
WE ARE OUTLINING.
AND WE WILL HAVE A LITTLE BIT
OF A DISCUSSION TODAY.
AND THEN THAT WILL FINALIZE
SORT OF OUR INPUT.
WE ARE ASKING FOR PEOPLE TO
INPUT UNTIL JUNE 14th, I
BELIEVE.
SO YOU STILL HAVE A LITTLE BIT
OF TIME.
BUT WE HOPE TO CAPTURE A LOT OF
YOUR THINKING AT LEAST TODAY TO
REALLY GET THAT KICKED OFF.
ACTUALLY, THAT'S MY LAST SLIDE
I THINK, THE SUMMARY DISCUSSION
IS HERE.
I WON'T GO THROUGH ALL OF THIS,
I LOOK FORWARD TO HAVING PETER
MARKS HERE AND UPDATES FROM
DOMINIQUE AND MATT AS WE GO
FORWARD.
I THINK I WILL STOP HERE.
AND SEE IF THERE ARE ANY
QUESTIONS?
NO QUESTIONS, EXCELLENT.
EASY.
IF THERE ARE NO QUESTIONS, HOW
ARE WE DOING ON TIME FOR PETER?
AS YOU ARE THINKING ABOUT
QUESTIONS.
>> WE ARE OKAY ON TIME BUT WE
HAVEN'T CONFIRMED -- HE IS ON.
>> Joni Rutter: HI, PETER, IF
YOU ARE ON.
>> Peter Marks: I'M ON.
>> Joni Rutter: I KNOW WHAT
HAPPENS WITH THIS COUNCIL.
WE ALSO GET WOUND UP IN
QUESTIONS.
WE CAN PERHAPS ALSO SAVE IT FOR
LATER TODAY.
IF YOU HAVE A QUESTION, ANNIE?
>> I JUST WANTED TO SAY THANK
YOU.
THIS IS JUST AN ENORMOUS BODY
OF WORK YOU JUST PRESENTED TO
US, I JUST WANT TO MAKE SURE WE
ACKNOWLEDGE THAT AND SAY THANK
YOU.
ACKNOWLEDGE THE MEMBERS AND
TEAM MEMBERS WHO ARE LEAVING.
AND WELCOME THOSE WHO ARE
JOINING, SO JUST A THANK YOU ON
BEHALF OF THE COUNCIL FOR THAT.
>> Joni Rutter: THANK YOU.
WE APPRECIATE THAT.
ANYTHING ELSE?
OKAY, WELL, IF NOT, I'M GOING
TO DO ONE MORE SLIDE HERE.
IT IS A GREAT OPPORTUNITY,
PETER.
I'M SORRY, SERGIO?
>> SERGIO: I'M SORRY I JUST
RAISED MY HAND AT THE LAST
MINUTE.
JUST TO ASK YOU IF WE ARE GOING
TO HAVE SOME TIME TO DISCUSS
THE NATIONAL STRATEGIC PLAN?
>> Joni Rutter: YES, THERE WILL
BE A DEDICATED SESSION AT THE
END OF THE DAY TODAY AFTER
PRESENTATIONS SO THAT WE CAN
HAVE THAT DISCUSSION,
ABSOLUTELY.
>> SERGIO: THANK YOU.
>> Joni Rutter: WONDERFUL,
THANK YOU, SERGIO.
NOW, PETER, REALLY GLAD TO HAVE
DR. PETER MARKS HERE TODAY.
AND PETER, I'VE BEEN TALKING A
LOT ABOUT OUR INTERACTIONS WITH
THE FDA OVER MANY COUNCIL
ROUNDS.
AND AT THE LAST COUNCIL ROUND
WE REALLY TALKED SPECIFICALLY
ABOUT THE IMPORTANCE OF THOSE
INTERACTIONS WITH NCATS AND THE
FDA BECAUSE OF THE CLOSE
RELATIONSHIP WE HAVE IN THE
SCIENCE WE SUPPORT AND HOW
CRITICAL IT IS TO FIND A WAY TO
STREAMLINE THOSE ACTIVITIES,
UNDERSTANDING WHERE THE HOLES
ARE, ET CETERA, HOW TO FILL THE
HOLES BETTER AND ALSO HELP THE
COMMUNITY WITH THEIR
APPLICATIONS AND THE SCIENCE
THEY DO AS IT MOVES FORWARD
INTO MORE REGULATORY SPACE.
WE THOUGHT IT WOULD BE
FANTASTIC TO HAVE DR. PETER
MARKS HERE TODAY TO TALK MORE
ABOUT WHAT HE IS THINKING ABOUT.
AS MANY OF YOU KNOW, PETER IS
THE HEAD OF THE CENTER ON
BIOLOGICS WHICH IS RESPONSIBLE
FOR ASSURING THE SAFETY AND
EFFECTIVENESS OF BIOLOGICAL
PRODUCTS, INCLUDING VACCINES,
ALLERGENIC PRODUCTS, BLOOD
PRODUCTS, CELLULAR TISSUE AND
GENE THERAPIES.
DR. MARKS RECEIVED HIS GRADUATE
DEGREE IN CELLULAR MOLECULAR.
COMPLETED INTERNAL MEDICINE
RESIDENCY AT BRIGHAM HOSPITAL
IN BOSTON.
WHERE HE THEN JOINED THEIR
ATTENDING STAFF, CLINICIAN
SCIENTIST, EVENTUALLY SERVED AS
THE CLINICAL DIRECTOR OF
HEMATOLOGY.
HE THEN WENT ONTO WORK SEVERAL
YEARS IN THE PHARMACEUTICAL
INDUSTRY.
PRIOR TO RETURNING TO ACADEMIC
MEDICINE AT YALE UNIVERSITY
WHERE HE LED THE ADULT
LEUKEMIA, THE CHIEF CLINICAL
OFFICER OF -- CANCER HOSPITAL.
HE JOINED THE FDA IN 2012 AS
DEPUTY DIRECTOR OF CBER.
DR. MARKS IS BOARD CERTIFIED IN
INTERNAL MEDICINE, FELLOW OF
AMERICAN COLLEGE OF PHYSICIANS
AS WELL.
AND IN 2022 HE BECAME A MEMBER
OF THE NATIONAL ACADEMY OF
MEDICINE.
AND THAT'S ONE OF THE HIGHEST
HONORS IN THE FIELD OF HEALTH
SCIENCE AND MEDICINE.
SO, SUPER HONORED TO HAVE PETER
HERE TODAY.
I WILL SAY TOO, BECAUSE I
COULDN'T RESIST SHOWING THIS
SLIDE.
THREE VERY DIFFERENT PHOTOS,
TOO BAD THE PICTURES ON THE
SCREEN ARE COVERING THIS UP.
PETER IS VERY STUDIOUSLY
THINKING ABOUT WHAT HE IS GOING
TO SAY.
JANET IS NO LONGER AT THE FDA
AND ABLE TO SAY WHATEVER SHE
WANTS, I DON'T KNOW IF THAT
STOPPED HER BEFORE.
THE ALWAYS GOOD TO HEAR WHAT
PETER IS THINKING, HE IS AN
INCREDIBLY INSPIRING PERSON.
I'M OFTEN INSPIRED BY WHAT
PETER IS DOING AND WHAT HE SAYS.
IT'S THRILLING TO HAVE HIM HERE
TODAY TO GIVE A LITTLE BIT A
PRESENTATION.
PETER, I WILL TURN IT OVER TO
YOU.
>> Peter Marks: THANK YOU SO
MUCH, I WILL SHARE MY SLIDES.
IT'S WONDERFUL TO HAVE
COLLEAGUES LIKE YOU AND TO BE
ABLE TO PARTICIPATE HERE.
LET'S SEE, SOMEONE NEEDS TO
STOP SHARING.
SO STOP YOUR SHARING.
OKAY, THAT'S OKAY, I THINK.
BECAUSE YOU DO WANT ME TO SHARE.
YOU SHOULD HOPEFULLY BE ABLE TO
SEE MY SLIDES FULL SCREEN?
>> Joni Rutter: YES.
>> Peter Marks: MUCH OF THIS
WILL BE THINGS YOU ARE VERY
FAMILIAR WITH.
THIS PUTS IT TOGETHER KIND OF
FROM OUR REGULATORY PERSPECTIVE.
THIS WON'T BE A TYPICAL
REGULATORY TALK.
BECAUSE I'M NOT GOING TO CITE
FDA REGULATIONS FROM THE 21CFR.
I'M GOING TO TRY TO TALK ABOUT
WHAT WE ARE ACTUALLY TRYING TO
DO TO ACTUALLY HELP MOVE THINGS
THROUGH THE PROCESS TO GET THEM
TO PATIENTS.
TALK ABOUT CURRENT PROGRESS IN
THE FIELD OF GENE THERAPY.
TALK ABOUT EXISTING CHALLENGES
IN PRODUCT DEVELOPMENT, WHAT WE
ARE DOING TO ADDRESS THE
CHALLENGES AND WHAT WE ARE
TRYING TO PUT IN PLACE IN TERMS
OF POLICIES.
THE GENE THERAPY HAS SOME
REALLY EXCITING PROSPECTS HERE
OF HAVING ONE DOSE OF SOMETHING
THAT COULD LEAD TO LONG TERM
CLINICAL BENEFIT, PERHAPS SOME
CIRCUMSTANCES EVEN CURE.
THIS CONCEPT OF ONE DOSE HAVING
AN EFFECT IS VERY EXCITING.
NOT JUST IN HIGH INCOME
COUNTRIES LIKE THE UNITED
STATES BUT AROUND THE WORLD
WHERE DELIVERING MEDICINES ON A
REGULAR BASIS WOULD BE
CHALLENGING, WHERE AS GENE
THERAPY COULD BE GAME-CHANGING.
I THINK ONE OF THE EXCITING
THINGS FOR ME, YOU HAVE YOUR
WORK DIRECTLY, THERE'S A HIGH
PROBABILITY OF SUCCESS WITH
YOUR CONSTRUCT THAN YOU WOULD
HAVE WITH SOME OTHER PRODUCTS,
SUCH AS SMALL MOLECULES, THAT'S
NOT TO SAY SMALL MOLECULES
AREN'T WONDERFUL, BUT USING THE
POWER OF GENETICS AND
THOUGHTFULNESS YOU CAN
HOPEFULLY STACK THE DECK IN
FAVOR OF ANY CANDIDATE BEING
SUCCESSFUL.
IF YOU USE A PLATFORM APPROACH
YOU CAN ADDRESS A LOT OF
DIFFERENT DISEASES USING THE
SAME BACKBONE OF A PLATFORM.
NOW THERE ARE SOME
DISADVANTAGES RIGHT NOW, ONE IS
THE COMPLEXITY AND COST OF
MANUFACTURER OF THESE PRODUCTS.
WE WILL TALK ABOUT HOW WE MAY
SEE THINGS TO ADDRESS SOME OF
THAT.
POTENTIAL FOR IRREVERSIBLE SIDE
EFFECTS.
THAT OBVIOUSLY, GENOTOXICITY IS
SOMETHING WE HAVE TO KEEP IN
MIND.
GENE THERAPIES IN THE CURRENT
EMBODIMENT THINGS REASONABLY
COMPLICATED TO GIVE.
GIVING LARGE DOSES OF AN
ASSOCIATED VIRUS, ASSOCIATED
WITH AN INFLAMMATORY RESPONSE
THAT HAS TO BE MANAGED.
RIGHT NOW YOU WON'T SEE THEM
GIVEN AT THE LOCAL HEALTH STOP.
THEY WILL BE IN THE HANDS OF
SPECIALIZED PHYSICIAN.
THIS IS A NEW BUSINESS MODEL
AND EVERYONE IS KIND OF
STRUGGLING WITH IT.
BOTH THE COMPANIES MAKING THESE
PRODUCTS AS WELL AS
PARTICULARLY PAYERS AND
GOVERNMENTS WHO ARE TRYING TO
UNDERSTAND HOW TO DEAL WITH
THESE VERY EXPENSIVE PRODUCTS
THAT CAN SOMETIMES BRING A LOT
OF ISSUES.
OKAY.
SO IN TERMS OF DELIVERING GENE
THERAPIES, THERE ARE TWO
GENERAL WAYS WE SEE THEM
DELIVERED, EITHER EX VIVO OR
INVIVO.
THE CHIMERIC, SOMETIMES WITH
LENTIVIRAL VECTORS.
OR USING SOME OTHER MEANS OF
INTRODUCING, OR CELL DISRUPTION
TO GET THE CONSTRUCT IN.
THE OTHER WAY IS DIRECTLY
ADMINISTERED GENE THERAPIES,
THROUGH ADENO -- WE ARE
STARTING TO SEE THE DEVELOPMENT
OF LIPID NANO PARTICLES AS A
WAY OF GETTING THESE IN AND I
WILL SAY MORE ABOUT THAT LATER.
ONE THING I WOULD WE REMISS IS
NOT INTRODUCING THE FACT GENE
THERAPY IS NOW UNDERGOING A
TRANSITION WHERE WE ARE
STARTING TO SEE GENOME EDITING,
IN OTHER WORDS FIXING THE BASE
PAIRS THAT ARE NOT CORRECT IN
THE GENOME.
BECOMING INCREASING MODALITY.
NOW, THIS IS NOT TO SAY GENOME
EDITING TOOLS HAVE BEEN AROUND
FOR YEARS AND INCLUDED THINGS
LIKE ZINC FINGER NUCLEASES.
TRANSCRIPTION ACTIVATOR-LIKE
EFFECTOR NUCLEASES.
WE NOW HAVE CRISPR BASED
EDITORS THAT GETS OVER PROBLEMS
WITH THE FIRST GENERATION
CRISPR THEY DON'T REQUIRE
DOUBLE STRANDED BREAKS.
AND NOT ABLE TO JUST CLIP BUT
POTENTIALLY REPLACE.
WE ARE STARTING TO SEE THIS
EVOLUTION IN GENE THERAPY.
WE HAVE SEEN NOW PRODUCTS THAT
ARE COMING ALONG.
IN 2023 WE HAD SIX PRODUCTS
APPROVED AND THIS IS, I THINK,
SHOWING THE PACE OF PROGRESS.
ONE WAS CASGEVY.
QUITE EXCITING.
I THINK THIS ABILITY TO HAVE
PRODUCTS NOW, WE NOW HAVE TWO
PRODUCTS FOR SICKLE-CELL
DISEASE AND BETA THALASSEMIA IS
QUITE EXCITING.
BUT TO SEE EDITED CELL-BASED
THERAPY COME ALONG A LITTLE
MORE THAN A DECADE THAN THE
IDENTIFICATION OF CRISPR I
THINK IS QUITE EXCITING.
WITH ALL THAT SAID, GENE
THERAPY IS AT A CRITICAL
JUNCTURE DUE TO COMBINATION OF
FACTORS, MANUFACTURING KHAL
LEPGS, CLINICAL DEVELOPMENT
TIMELINES, AND DIFFERENT GLOBAL
REGULATORY REQUIREMENTS WHICH
IS SLOWING DOWN PROGRESS OF
PRODUCTS GETTING TO THE BENEFIT
OF PATIENTS.
SO WE ARE TRYING TO DO WHAT WE
CAN AT CENTER FOR BIOLOGICS,
SOME IN COLLABORATION WITH
NCATS.
I THINK WE REALLY VALUE THAT
COLLABORATION.
IN TERMS OF ADVANCING
MANUFACTURING OF GENE THERAPIES
AND GETTING THEM THROUGH THE
PROCESS, THAT'S IN PART THROUGH
THE SPOKE GENE THERAPY
CONSORTIUM WORK BUT ALSO OTHER
COLLABORATIONS, SUCH AS PAY GT
WITH NCATS.
SO ADVANCING MANUFACTURING, WE
ARE TRYING TO APPLY THE
PLATFORM TECHNOLOGY PROVISION
IN THE 2023 OMNIBUS.
WE ARE WORKING TO MORE CLEARLY
DEFINE THE USE OF ACCELERATOR
APPROVAL FOR GENE THERAPY,
EXPLORING AND REVIEW WITH OTHER
REGULATORY AUTHORITIES AND
MOVING FORWARD COMMUNICATION
PILOT FOR RARE DISEASES.
I WILL TELL YOU ABOUT ONE
MINUTE OF EACH OF THESE AND TRY
TO GET YOU, GIVE YOU BACK A
LITTLE TIME.
IN TERMS OF MANUFACTURING
SOLUTIONS, IT'S BEEN RECOGNIZED
THAT A LOT OF INNOVATION COMES
OUT OF ACADEMIC LABORATORIS.
THE PROBLEM IS, HAVING BEEN AN
ACADEMIC MYSELF, WE TEND TO DO
IT THE WAY OUR COLLEAGUE DID IT
IN OUR LOCAL INSTITUTIONMENT
WHEN YOU WANT TO TRANSFER A
PROCESS TO A CONTRACT
MANUFACTURER TO A COMPANY,
OFTEN TIMES THOSE PROCESSES
HAVE TO BE REWORKED.
AND THAT REWORK COSTS A LOT OF
MONEY.
WE ARE TALKING MILLIONS OF
DOLLARS SOMETIMES, AS WELL AS
TIME.
AND SO, WE ARE THINKING THAT
STANDARDIZING THESE PROTOCOLS
USED BY ACADEMICS COULD
POTENTIALLY BE HELPFUL.
THIS IS ONE OF THE THINGS THE
SPOKANE GENE THERAPY CONSORTIUM
IS DOING BY TRYING TO CREATE A
PLAY BOOK.
ADENO ASSOCIATED VIRUSES ARE
THINGS THAT COULD BE AUTOMATED.
THEY SIMPLY HAVEN'T BEEN
BECAUSE NOBODY HAS HAD THE
IMPETUS TO, THERE HASN'T BEEN A
LARGE ENOUGH MARKET.
THIS CONCEPT OF AUTOMATION
COULD BE VERY HELPFUL.
IF NOT FOR THE ENTIRE PROCESS
AT LEAST THE DOWNSTREAM PROCESS.
THIS IS A SCHEMATIC TO SAY, WE
ACTUALLY HAVE MACHINES THAT DO
THIS IN MANUFACTURING ALREADY,
IT JUST HASN'T BEEN APPLIED TO
THIS PROCESS.
IT COULD HELP IMPROVE
REPRODUCABILITY AND DECREASE
COST.
GOOD THINGS.
IDEALLY YOU WOULD WANT
SOMETHING TO REPLACE THIS SODA
DISPENSER AS A GENE THERAPY
DISPENSER, THE A NICE MODEL FOR
SOMEONE IN THE MANUFACTURE
WORLD, IT'S A FABRICATION
DEVICE WITH DISPOSABLES AND
CONSUMABLES.
MOVING ON,SPEAKING OF PLATFORMS
WHERE WE THINK WE CAN MAKE SOME
REAL PROGRESS HERE.
GENE THERAPIES HAVE SOMETHING
ABOUT THEM THAT MANY OTHER
PRODUCTS DO NOT HAVE.
WHICH IS, OFTEN TIMES THE
VECTOR BACKBONE IS THE SAME
BETWEEN DIFFERENT CONSTRUCTS.
NOW IT'S NOT THE SAME FOR EVERY
CONSTRUCT.
BUT THAT SAID, FOR A GIVEN
GROUP OF AV8'S WITH INSERTS OF
A CERTAIN SIZE RANGE WE WOULD
LIKE TO SEE IF WE COULD MAKE
MORE PROGRESS QUICKLY BY
ALLOWING SWAPPING OUT OF THE
INSERT AGAIN WITHIN A GIVEN
SIZE RANGE WHILE MAINTAINING
THE SAME MANUFACTURING PROCESS,
THE SAME CONTROLS AND
LEVERAGING THE TOXICOLOGY.
THAT MAY ALLOW MORE
STREAMLINING AND MOVE FROM ONE
PRODUCT TO THE NEXT WITH LESS
BURDEN.
CONGRESS PASSED THE PLATFORM
TECHNOLOGIES ACT AS PART OF
OMNIBUS APPROPRIATIONS ACT OF
2023.
ONE PRODUCT, HAVE THEIR
PLATFORM DESIGNATED SO THEY CAN
USE CROSS-REFERENCE AND NOT
HAVE TO GO BACK AND REDO ALL OF
THE TOXICOLOGY, MANUFACTURING
INFORMATION FOR SUBMISSION WHEN
THEY COME IN WITH THESE
PLATFORM PRODUCTS.
WE ARE IN THE PROCESS OF
ISSUING GUIDANCE BUT WE THINK
IT WILL BE PARTICULARLY
IMPORTANT, PARTICULARLY BECAUSE
WHEN WE COME TO CRISPR, IT'S
THE POSTER CHILD OF A PLATFORM
THERAPY.
WHEN YOU THINK ABOUT IT, WHEN
YOU LOOK AT THE RIGHT AT THE
PRIME EDITOR, WHICH ARE
REMARKABLY VERSATILE.
IF YOU THINK OF A PRIME EDITOR
THAT WANTS TO TARGET THINGS IN
THE LIVER FOR INSTANCE.
BY A LIPID NANOPARTICLE YOU CAN
PROBABLY DO WITH A NON-STUDDED
LIPID NANO PARTICLE, WHETHER
YOU ARE TARGETING A METABOLIC
DISORDER OF ONE SORT OR ANOTHER
YOU ARE GOING TO HAVE A VERY
SIMILAR CONSTRUCT WITH THE
EXCEPTION OF A SMALL SEGMENT OF
RNA.
SO THE ABILITY TO USE THIS
PLATFORM APPROACH MAY ALLOW US
TO REALLY MOVE THROUGH
DIFFERENT PRODUCTS BY HAVING A
WHOLE HOST OF PRODUCTS
REFERENCE ONE SERIES OF
MANUFACTURING.
YOU CAN JUST FOCUS IN ON
OFF-TARGET EFFECTS AND SPECIFIC
CLINICAL EFFECTIVENESS FOR EACH
OF THE PRODUCTS.
FINALLY, THE LAST COUPLE THINGS
WE WANT TO TRY TO LEVERAGE
ACCELERATED APPROVAL AS MUCH AS
WE CAN.
IN THE GENETIC MEDICINE SPACE,
WE HOPE AT LEAST SOME OF THE
TIME WE WILL BE ABLE TO
LEVERAGE ENZYME ACTIVITY
LEVELS, STRUCTURAL PROTEIN
LEVELS, THINGS WE CAN MEASURE
THAT WILL REASONABLY LIKELY
PREDICT CLINICAL BENEFIT SO WE
CAN MOVE THESE PRODUCTS FORWARD
MORE PERFECTLY.
WHEN YOU THINK ABOUT IT NOT ALL
ANIMAL MODELS ARE PERFECT, BUT
WHERE WE HAVE GOOD ANIMAL
MODELS WE CAN USE THEM TO HELP
US PREDICT BENEFIT BASED ON
LEVELS SEEN IN HUMANS OF A GENE
THERAPY.
OR WE CAN SOMETIMES DERIVE THAT
INFORMATION FROM HUMAN
OBSERVATIONS WHERE IN SOME
CASES HUMANS ARE PARTIALLY
DEFICIENT IN PROTEINS YET SHOW
NO ADVERSE EFFECTS.
IF WE CAN REACH THOSE LEVELS
WITH A GENE THERAPY WE HAVE
SOMETHING THAT IS LESS LIKELY
TO PREDICT CLINICAL BENEFIT, WE
CAN GET THINGS OVER TO THE
APPROVED PRODUCTS, COMPANIES
GET A REVENUE STREAM, BUT MORE
IMPORTANTLY, PATIENTS GET
ACCESS SOONER AND THEN WE GET
THE CONFIRMATORY EVIDENCE IN
THE COURSE OF TIME AFTERWARDS.
IT MEANS OCCASIONALLY THINGS
WON'T CONFIRM BUT WE CAN LIVE
WITH THAT IF WE ARE HELPING
MORE PEOPLE OVERALL.
AND THEN THE LAST TWO AREAS I
WOULD LIKE TO SPEAK ABOUT, ONE
IS THE FACT WE DO RECOGNIZE
THAT GLOBAL, OR LACK OF GLOBAL
HARMONIZATION AROUND THIS GENE
THERAPY SPACE LEADS TO
PROBLEMS, PARTICULARLY FOR RARE
DISEASES, WHERE IN SOME CASES
THE BARRIERS TO ENTRY AND NEW
REGULATORY ENVIRONMENT PREVENT
PEOPLE FROM BRINGING A
WELL-DOCUMENTED SAFE AND
EFFECTIVE THERAPY TO A NEW
REGULATORY ENVIRONMENT.
SO WE ARE TRYING TO WORK WITH
OUR COLLEAGUES, PARTICULARLY AT
EMA RIGHT NOW BUT WE WILL
EXPAND THIS PILOTING WAYS TO
BOTH GET TO CONVERGENT
REGULATORY APPROACHES BUT ALSO
TO DO COLLABORATIVE REVIEW AND
HOPEFULLY THAT WILL AGAIN MOVE
PRODUCTS THROUGH THE PROCESS.
IMPORTANTLY HERE JUST THE
BOTTOM LINE HERE IS IF WE HAVE
A POPULATION OF 30-50 PEOPLE IN
THE UNITED STATES WITH A RARE
DISEASE TO BE TREATED IN A
GIVEN YEAR, THAT'S NOT
COMMERCIALLY VIABLE.
BUT IF YOU MELD THAT WITH A
POPULATION OF 70-80 PATIENTS IN
EUROPE, AND ANOTHER 70-80
PATIENTS IN ASIA, SUDDENLY
THERE'S A VERY COMMERCIALLY
VIABLE PRODUCT.
IF WE CAN GET THERE, THAT MIGHT
BE VERY GOOD FOR ALL OF OUR
DIFFERENT PATIENT POPULATIONS.
THE LAST INITIATIVE OUR START
PILOT.
BASICALLY TRYING TO APPLY WHAT
WE LEARNED DURING THE COVID-19
PANDEMIC TO THE BENEFIT OF RARE
DISEASE THERAPY PRODUCTS *. THE
IDEA HERE IS WE FOUND THAT
BEING ABLE TO HAVE VERY
CONSTANT COMMUNICATION, ONGOING
COMMUNICATION WITH SPONSORS WAS
VERY HELPFUL FOR THEM, AS THEY
DEVELOP PRODUCTS IN THE
COVID-19 SPACE, IT PROBABLY
SHAVED AT LEAST 25% OF THE
TOTAL TIME OF DEVELOPMENT OF
THE VACCINES, PROBABLY MORE
THAN THAT, BECAUSE PEOPLE
DIDN'T HAVE TO WAIT AROUND FOR
MEETINGS TO OCCUR, WE JUST
ANSWERED QUESTIONS WHEN THEY
CAME UP.
WE HAVE A PILOT WE INTEND TO DO
THE SAME THING FOR SEVERAL RARE
DISEASE PRODUCTS.
WE WILL PICK PROBABLY THREE
ELIGIBLE PRODUCTS.
KEEP YOUR EARS OPEN BECAUSE THE
ANNOUNCEMENTS WILL BE SOON.
AND HOPEFULLY, WE WILL BE ABLE
TO SHOW USING METRICS BETWEEN
VARIOUS STAGES OF DEVELOPMENT
THIS ACTUALLY MAKES A
DIFFERENCE, IF IT DOES, WE WILL
EXPAND THE PILOT.
ULTIMATELY CENTER FOR BIOLOGICS
WE ARE TRYING TO ADVANCE THE
REGULATORY FRAMEWORK FOR GENE
THERAPY AND UP AND COMING FIELD
OF GENOME EDITING BECAUSE WE
WOULD LIKE TO SEE THE BENEFITS
BROUGHT TO THE GREATEST NUMBER
OF PEOPLE.
SO THANKS VERY MUCH.
>> Joni Rutter: THANK YOU,
PETER, THAT WAS TERRIFIC.
THANK YOU FOR THE PRESENTATION.
WE DO HAVE SOME TIME FOR
QUESTIONS.
AND I HAVE A LOT.
WELL, MAYBE NOT A LOT.
BUT I HAVE SOME.
SO I'M HAPPY TO GET US STARTED,
WHILE I'M STARTING I HOPE OWNS
THINK OF QUESTIONS.
PETER, YOU MENTIONED COGENT AND
HOW IMPORTANT IT IS FOR
THINKING ABOUT MORE GLOBALLY
ADDRESSING THE ISSUE OF
COMMERCIALIZATION.
I COULDN'T HELP BUT THINK ABOUT
TOO IN THE OTHER APPROACH YOU
TALKED ABOUT IN TERMS OF THE
PLATFORM TECHNOLOGY IN HOW,
JUST THE IDEA OF THE PLATFORM
BASED APPROVALS COULD ALSO
POTENTIALLY HELP
COMMERCIALIZATION AS WELL,
BECAUSE THERE MIGHT BE, IT
WOULD SEEM TO CUT NOT ONLY THE
TIME IT TAKES TO DEVELOP THE
THERAPIES BUT ALSO POTENTIALLY
THE COSTS ASSOCIATED WITH THE
DEVELOPMENT OF THE THERAPIES
TOO.
SO THERE MIGHT BE MORE
COMMERCIAL INTEREST FROM JUST
THAT PLATFORM-BASED APPROACHES.
IS IT, I'M ASSUMING THAT, YOU
PROBABLY CAN'T REMARK ON THIS,
BUT ARE THERE GROUPS YOU KNOW
ABOUT WHO ARE BEGINNING TO
GENERATE THOSE KINDS OF
PLATFORM TECHNOLOGIES THAT
MIGHT BE VERY HELPFUL FOR THAT,
REALLY GETTING AT THAT
COMMERCIALIZATION FOR RARE
DISEASES IN THE GENE THERAPY
SPACE?
>> Peter Marks: I'M AWARE OF
VARIOUS GROUPS TALKING ABOUT
HOW TO GO ABOUT THIS.
BECAUSE I THINK THE ECONOMY OF
SCALE OF BEING ABLE TO MOVE
HERE, AND BEING ABLE TO PUT
TOGETHER WHAT MIGHT BE A
PORTFOLIO OF PRODUCTS, THAT
EACH OF WHICH MIGHT NOT BE
ROBUSTLY COMMERCIALLY VIABLE
BUT TOGETHER FORM SOMETHING
THAT IS COMMERCIALLY VERY
VIABLE, I THINK IS SOMETHING
THAT IS ATTRACTIVE.
IN SOME WAYS IT'S A LITTLE BIT,
IT'S WEIRD TO SAY THIS, IT'S A
LITTLE LIKE THE GENERIC MODEL.
YOU AREN'T GOING TO MAKE AS
INCREDIBLE AMOUNT OF MONEY FROM
EACH OF THESE PRODUCTS BUT FROM
HAVING THEM TOGETHER THEY CAN
CREATE A ROBUST COMMERCIAL
VENTURE.
>> Joni Rutter: GRIT.
-- GREAT.
THANK YOU FOR THAT.
I SEE PAUL, GO AHEAD.
>> PAUL: PETER, THANK YOU, THAT
WAS A GREAT PRESENTATION.
ON THE LAST SLIDE YOU PRESENTED
YOU WERE TALKING ABOUT
INNOVATION IN COMMUNICATION
EFFICIENCY.
AND PILOTS YOU ARE WANTING TO
SORT OF ROLL OUT.
CAN YOU TALK A LITTLE BIT MORE
ABOUT THE MECHANISMS FOR THAT
EFFICIENT COMMUNICATION?
>> Peter Marks: YEAH.
SO THE WAY WE PLAN TO DO THIS,
I THINK IF THAT'S WHAT YOU ARE
ASK, HOW ARE WE ACTUALLY GOING
TO MAKE IT WORK.
THE IDEA HERE, THE REASON WHY
WE CAN'T JUST SAY WE WILL DO
THIS FOR EVERYONE, WE WILL
ASSIGN A PROJECT MANAGER AND
THE COMPANY WILL HAVE THE
PROJECT MANAGER'S PHONE NUMBER
AND EMAIL AND WE WILL SAY OKAY,
WHEN AN ISSUE COMES UP, LET THE
PROJECT MANAGER KNOW, IF IT'S A
CMC MINOR ISSUE WE WILL GET ONE
OF OUR CMC REVIEWERS ON THE
PHONE.
IF IT'S A MAJOR ISSUE WE WILL
ARRANGE A MEETING IN A WEEK'S
TIME BUT WE WON'T ASK THEM TO
SUBMIT A BRIEFING, USUALLY IT'S
SUBMIT A BRIEFING BOOK.
HERE SUMMARIZE THE ISSUE IN
EMAIL SO OUR FOLKS CAN
UNDERSTAND IT AND LET'S GET ON
PHONE AND WORK IT OUT.
THE IDEA HERE IS NOT TO GET
STUCK AT EACH PLACE WHERE WE
OFTEN HAVE THINGS GET STUCK.
MY HYPOTHESIS, ESPECIALLY WITH
SMALLER COMPANIES, SOMETIMES
WHEN WE HAVE FORMAL
CORRESPONDENCE WITH THEM, THEY
DON'T UNDERSTAND WHAT WE ARE
SAYING.
BECAUSE THEY DON'T, THEY DO
SOMETHING DIFFERENT THAN WHAT
WE THOUGHT WE WERE TELLING THEM.
THEY COME BACK TO US.
WE SAID NO, NO, WE DIDN'T MEAN
FOR YOU TO DO THAT, DO THIS.
AND THIS WAY, THE NICE THING
ABOUT THIS, I CAN SAY TO YOU,
YOU KNOW, I'M GOING TO GET OFF
THIS CALL IN FIVE MINUTES, DO
YOU UNDERSTAND THAT.
AND YOU CAN SAY OH YES, I
UNDERSTAND YOU ARE GOING TO GET
OFF IN FIVE MINUTES AND I CAN
NOD MY HEAD, WE HAVE GONE
THROUGH THE CYCLE OF
COMMUNICATION.
THAT'S PART OF THE IDEA HERE,
WE CAN MAKE SURE THAT WHEN WE
CONVEY ADVICE, THAT IT'S REALLY
UNDERSTOOD.
IT SEEMS SO SIMPLE.
YOU KNOW, THIS IS LIKE, IT
SHOULD BE ELEMENTARY SCHOOL.
BUT I CAN'T TELL YOU.
THERE ARE PROGRAMS THAT HAVE
NEARLY BEEN TERMINATED BECAUSE
OF ISSUES WITH COMMUNICATION
LIKE THIS WITH FDA.
>> SOUNDS LIKE A GOOD LIGHT
WEIGHT INITIATIVE TO MOVE
FORWARD.
THANK.
>> Peter Marks: I APPRECIATE IT.
>> Joni Rutter: ANNIE KENNEDY.
>> HI DR. MARKS, THANK YOU FOR
BEING HERE AND WONDERFUL TO SIT
AT AN NCATS ADVISORY COUNCIL
MEETING AND INTERACTING WITH
YOU, SO THANK YOU FOR BEING
HERE.
I STARTED MY CAREER IN INTRA
MUSCULAR CLINIC, I NEVER
THOUGHT I WOULD SEE A DAY WHEN
GENE THERAPY ARE BENEFITING THE
PATIENTS AND FAMILIES I'M
SERVING, MUCH OF THAT IS DUE TO
THE LEADERSHIP OF YOU AND YOUR
TEAM BUT ALSO THE LEADERSHIP OF
THOSE ON THIS CAMPUS WHERE WE
ARE TODAY, WHO MOVED SO MANY OF
THOSE PROGRAMS FORWARD AND
PIPELINES MOVED INTO REGULATORY
REVIEW.
SO MY FACEBOOK FEEDS ARE RIGHT
NOW FLOODED WITH PHOTOS OF
INDIVIDUALS WHO ARE DIAGNOSED
WITH SMA TYPE I GRADUATING FROM
PRE-SCHOOL WITH THEIR LITTLE
CAPS AND GOWNS.
I THINK OF THE FAMILIES I SPENT
TIME WITH THEM IN CLINIC YEARS
AGO AS WE DEVELOPED THE
DIAGNOSES AND THEIR OUTCOMES
WERE QUITE DIFFERENT AT THAT
TIME.
THAT'S WHAT THE PROMISE OF
SCIENCE IS.
BUT I ALSO THANK THE TEAMS WHO
UNDERSTAND THAT ENDPOINTS ARE
DIFFERENT IN DIFFERENT DISEASES
AND FAMILIES TO WHOM A
MEANINGFUL BENEFIT IS GOING UP
A STAIR, SO PATIENTS WHO NOW
BENEFIT FROM THE DUCHEN GENE
THERAPY AND CAN NOW RIDE BIKES
OR GO TO A FRIEND'S HOUSE FOR A
SLEEP OVER, THEY CAN CROSS THE
THRESHOLD OF THAT FRIEND'S HOME
BECAUSE OF THAT THERAPY.
EVEN THOUGH WE BENEFITED FROM
THESE ROBUST GENE THERAPY
PROGRAMS THERE ARE STILL THOSE
WHO ARE BEING LEFT BEHIND.
FOR MANY REASONS.
AND YOU ARTICULATED SOME THAT
ARE BEING DEALT WITH.
THERE ARE FAMILIES WHO, NOT
BECAUSE OF REDOSING BUT DON'T
MEET INCLUSION CRITERIA BECAUSE
OF PREEXISTING ANTIBODIES AND
THEY HAVE THE AB TO BEGIN WITH.
THIS COULD BE A QUESTION FOR
NCATS OR YOU.
I APPRECIATE UNDERSTANDING
WHAT'S BEING DONE AT THIS POINT
TO ENABLE THOSE WHO CURRENTLY
HAVE THE ANTIBODIES TO AB AND
ARE EXCLUDED FROM CLINICAL
TRIAL OR APPROVED THERAPIES, IS
THERE WORK BEING DONE TO HELP
OVERCOME THAT ENTRY BARRIER
INTO THE GENE THERAPY PROGRAMS
BEING WORKED ON?
>> Peter Marks: I WILL TELL YOU
THREE QUICK AREAS OF RESEARCH
GOING ON.
SIMPLEST THERE ARE INDIVIDUALS
LOOKING AT WAYS TO CLEAR THE
ANTIBODIES AND MAKE GENE
THERAPY POSSIBLE, OR AT LEAST
REDUCE TO POSSIBILITIES YOU CAN
ESSENTIALLY OVERWHELM AND HAVE
THE GENE THERAPY OCCUR.
THAT'S SOMETHING WE HAVE KNOWN
HOW TO DO.
WE HAVE DONE FOR OTHER THINGSES.
THAT'S ONE WAY.
THE OTHER IS PEOPLE ARE
OBVIOUSLY DEVELOPING NOVEL
CAPSIDS THAT MAY EVADE.
BUT THE OTHER DECIDING TO MOVE
ON FROM AEV TO CONSTRUCTS TO
USING NANO PARTICLES FOR
CRISPR, FOR THOSE WITH
ANTIBODIES, IT IS SIDE STEPPING
THE ISSUE.
THERE ARE A VARIETY OF
DIFFERENT THINGS GOING ON.
I AGREE, I WISH THERE WOULD BE
MORE RAPID PROGRESS IN SOME OF
THESE AREAS BUT THERE ARE
OBVIOUSLY SERIOUS EFFORTS GOING
ON IN ALL THREE OF THOSE
EXAMPLES.
THERE ARE MORE BUT AT LEAST
THREE EXAMPLES.
>> ANNIE: THANK YOU.
>> Joni Rutter: PETER, I HAVE
ANOTHER QUESTION BECAUSE I
DON'T SEE OTHERS JUST YET.
ANOTHER QUESTION ALONG THE SAME
LINES AROUND SORT OF INITIATING
THE RESEARCH NEEDED TO SUPPORT
SORT OF THE NEXT PHASE WE ARE
MOVING INTO, I AM WONDERING IF
THIS IS A GOOD OPPORTUNITY TO
THINK HARD HOW WE CAN USE THE
SMALL BUSINESS PROGRAMS TO
SUPPORT RESEARCH TO GET IN THE
MANUFACTURING SPACE AND PERHAPS
OTHER ISSUES YOU WERE TALKING
ABOUT.
AND YOUR THOUGHTS ON PERHAPS
THAT VERY SPECIFIC APPROACH OF
WORKING WITH COMPANIES, BIOTECH
COMPANIES.
>> Peter Marks: JONI, I THINK
THAT IS PROBABLY ONE OF THE
GREATEST FOCUSES WE NEED TO GET
INTO.
HOW ARE WE GOING TO
MANUFACTURER THESE THINGS MORE
EFFICIENT AND EFFECTIVELY.
I THINK THAT IS AN IMPORTANT
PLACE.
I THINK IT'S NOT JUST WITH THE
TRADITIONAL GENE THERAPY
COMPANIES, IT'S WITH COMPANIES
THAT MAKE MANUFACTURING
EQUIPMENT TO ACTUALLY HAVE THEM
DEVELOP THE TOOLS THAT COULD BE
APPLIED HERE.
IT'S UNFORTUNATE, ACADEMICS
DON'T GET PAPERS OUT OF, I MEAN
THERE ARE SOME THAT DO.
MANUFACTURING EQUIPMENT IS NOT
REALLY EXCITING TO DESIGN FROM
AN ACADEMIC STANDPOINT BUT IT
IS ABSOLUTELY CRUCIAL IF WE ARE
GOING TO HAVE GENE THERAPIES
FOR MANY DIFFERENT RARE
DISEASES AND WE HAVE TO GET
MORE EFFICIENT.
THE KIND OF BENCH TOP APPROACH
WILL NOT BE SUSTAINABLE.
SO I THINK THIS APPLICATION OF
KIND OF GETTING SMALL
BUSINESSES INTERESTED IN THIS,
IT'S TRUE, THERE ARE A LOT OF
ENGINEERS IN SMALL BUSINESSES
WHO MIGHT BE VERY WILLING TO
TAKE THIS CHALLENGE UP.
>> Joni Rutter: YEAH.
IT SEEMS LIKE WE NEED TO DO A
LOT IN THE MANUFACTURING SPACE
TO GET US TO WHERE WE NEED TO
BE.
SO I THINK FIRING ON ALL
CYLINDERS WE HAVE THE ABILITY
TO DO SO WOULD BE GREAT.
ANY OTHER QUESTIONS FOR PETER?
ANNIE, GO AHEAD.
>> ANNIE KENNEDY: IT'S ME AGAIN.
THERE'S ANOTHER IMPORTANT
INTERSECTION MAYBE NOT ALL MY
COUNCILMEMBERS ARE AWARE, THE
WORK THAT NCATS AND FDA AND
PERHAPS CBER IS WORKING ON.
THE INTERSECTION OF PATIENTS
NOT AVAILABLE FOR CLINICAL
TRIALS THE POINT OF WHICH THEY
ARE IDENTIFIED AND DIAGNOSED
AND ELIGIBILITY CRITERIA ON A
LABEL FOR PRODUCTS THAT'S
APPROVED.
THAT'S BECAUSE OF THE CRITERIA
NECESSARY TO HAVE THE CONDITION
IN THE NEWBORN SCREENING NOW.
WE JUST, A LITTLE WHILE AGO
HEAR DR. RUTTER GIVE A
PRESENTATION ABOUT SOME WORK IN
DIAGNOSTICS NCATS IS DOING AND
OTHERS ARE DOING TO REALLY HELP
INNOVATE IN OUR DIAGNOSTICS
THROUGHOUT THE U.S., SO WE CAN
BETTER DETECT PATIENTS,
ESPECIALLY FOR DISEASES EARLIER.
I KNOW THIS IS AN AREA, DR.
MARKS, YOU ARE ALSO CONCERNED
ABOUT.
WE HAVE PRODUCTS NOW APPROVED
THAT ARE LIFE-ALTERING, NOT
LIFE-SAVING BUT NOT GETTING TO
PATIENTS SOON ENOUGH, FAST
ENOUGH BECAUSE THE PATIENTS
ELIGIBLE AREN'T IDENTIFIED AT
THE OPTIMUM THERAPEUTIC WINDOW.
>> Peter Marks: THERE ARE
MULTIPLE ISSUES HERE, RIGHT?
THERE'S THE ISSUE OF PROPER,
EARLY DIAGNOSIS.
THAT HAS TO DO WITH THE FACT WE
NEED TO DO A BETTER JOB AT
ESSENTIALLY NEWBORN SCREENING
IN THE COUNTRY.
BUT THEN ALSO THERE'S THE
ABILITY TO GET OUT THE TESTS
THAT ARE NECESSARY SO THAT, AND
GET OUT THE WORD OF THESE
THERAPIES AS THEY COME ALONG.
AND SO I THINK WE WILL NEED TO
CONTINUE TO WORK ALONG THESE,
AS WE HAVE, I THINK WE ARE
GOING TO HAVE INCREASING NUMBER
OF THERAPEUTIC OPTIONS, BUT AN
INCREASING NUMBER OF OPTIONS
FOR PEOPLE WHO PREVIOUSLY
DIDN'T HAVE ANY OPTIONS WHERE
IDENTIFYING THOSE WHO MIGHT
BENEFIT WILL BE CRITICAL.
WE WILL ALSO HAVE TO IDENTIFY
WAYS POTENTIALLY OF HAVING
ACCESS FOR THOSE WHO MIGHT BE
DIAGNOSED LATE.
WHERE THEY MAY NOT HAVE FALLEN
INTO THE CLINICAL TRIAL
POPULATIONS.
I THINK THAT IS WHAT YOU ARE
TRYING TO GET AT, ANNIE AS WELL.
THAT'S SOMETHING WE WILL HAVE
TO THINK ABOUT DOING
EFFECTIVELY AND EFFICIENTLY.
>> Joni Rutter: YES, GO AHEAD,
MIKE.
>> MIKE: PETER, YOU MENTIONED
YOU KNOW, THE POTENTIAL FOR A
GLOBAL DEMAND WOULD REALLY MAKE
IT MORE COMMERCIALLY ATTRACTIVE.
BUT I'M JUST WONDERING IF YOU
CAN SPEAK TO THE EXTENT OF THE
GLOBAL REGULATORY HARMONIZATION
WE HAVE SEEN IN VACCINES, THIS
IS REALLY AN OBSTACLES FOR
MANUFACTURERS TO GO THROUGH THE
REGULATORY ENVIRONMENTS.
>> Peter Marks: WE HAVE BEEN
WORKING WITH WHO ON CELL
THERAPY REGULATORY CONVERGENCE,
THERE WAS A MEETING HELD LAST
WEEK IN MUSCOT, OMAN ON THIS.
FOR VACCINES, YOU ARE FAMILIAR
WITH WHO LISTING WHEN A MAJOR
REGULATORY AUTHORITY APPROVES A
VACCINE, THE MANUFACTURER CAN
APPLY TO BE LISTED WITH WHO AND
OTHER REGULATORS WITH OTHER
COMPANIES DON'T HAVE THE
CAPACITY JUST KEY IN ON THAT
WHO-LISTED VACCINE.
I THINK WE WILL NEED SOMETHING
VERY SIMILAR IN THE GENE
THERAPY SPACE, PARTICULARLY THE
GENE THERAPY SPACE.
WE HAVE COUNTRIES THAT ARE
AFFLUENT ENOUGH TO BE ABLE TO
AFFORD THESE PRODUCTS.
IF YOU LOOK AT THE MIDDLE EAST,
THAT'S A GREAT EXAMPLE.
YET, THEY DON'T HAVE THE
REGULATORY CAPACITY TO REVIEW
THEM.
THEY ARE PERFECTLY WILLING TO
KEY OFF AN FDA OR EMA APPROVAL.
WE NEED TO HELP THEM FIND THE
PATHWAY TO DO SO.
THAT'S WHERE WE MAY NEED TO
HEAD HERE, THE FASTER WE CAN
GET THERE, THE BETTER OFF WE
MAY BE.
>> Joni Rutter: THANK YOU VERY
MUCH.
ANY OTHER QUESTIONS?
ALL RIGHT.
SEEING NONE.
PETER, IT'S BEEN A REAL
PLEASURE TO HAVE YOU HERE AT
THE COUNCIL TODAY.
PERHAPS THIS SHOULDN'T BE THE
ONLY OCCURRENCE FOR THAT.
WE MIGHT HAVE TO INVITE YOU
BACK AGAIN.
BUT WE WILL GIVE YOU A LITTLE
BIT A BREAK BETWEEN NOW AND
THEN.
THANK YOU FOR BEING HERE, I
APPRECIATE YOU TAKING TIME OUT
OF YOUR BUSY SCHEDULE TODAY.
>> Peter Marks: THANK YOU, I
THINK WE HAVE REALLY ENJOYED
COLLABORATING.
IT'S JUST SUCH AN IMPORTANT
PART OF WHAT WE DO, SO THANK
YOU, NCATS.
THANK YOU.
>> Joni Rutter: ALL RIGHT.
THANK YOU VERY MUCH.
OKAY, WITH THAT, WE ARE
ACTUALLY AHEAD OF SCHEDULE,
WHICH RARELY HAPPENS, SO I
THINK WE NEED TO SEIZE THE
OPPORTUNITY HERE AND TAKE
ADVANTAGE OF THE FACT THAT WE
HAVE A LITTLE BIT MORE TIME.
SO WHAT I WOULD LIKE TO DO IS
ACTUALLY GIVE YOU A LITTLE BIT
MORE TIME TO HAVE SOME LUNCH.
AND THEN WE WILL GO AHEAD AND
CONVENE AT 12:45.
12:40?
WE NEED TO CONVENE AT 12:40 SO
THAT WE CAN GET STARTED
PROMPTLY AT 12:45, I KNOW WE
ARE GOING TO HAVE A GOOD
DISCUSSION AT THE END WHEN WE
TALK ABOUT THE STRATEGIC
PLANNING.
SO HAPPY TO TALK ABOUT ANY
QUESTIONS ALONG THE WAY, I HOPE
YOU ARE SAVING THEM FOR THE END
OF THE DAY WHEN WE CAN HAVE A
LITTLE MORE OPEN DISCUSSION AS
WELL.
WITH THAT, LET US CONVENE FOR
LUNCH.
THANK YOU.
IN THAT CASE, WE WILL GET
STARTED.
I WILL TELL YOU, WE HAVE OUR
CLOCK ON THE SCREEN IS ONE TIME
OUR CLOCKS IN THE ROOM.
THERE ARE A COUPLE, THEY ARE ON
DIFFERENT TIMES.
FOLLOW THE ONE ON YOUR SCREEN
IF ALL ELSE FAILS.
THANK YOU FOR A GOOD LUNCH.
I HOPE EVERYBODY HAD A CHANCE
TO CATCH UP WITH ONE ANOTHER.
I WANT TO INTRODUCE DR.
DOMINIQUE BUSHARD, SHE WILL
GIVE AN UPDATE.
>> I'M DOMINIQUE BUSHARD.
I'M THE DIRECTOR OF THE
DIVISION OF RARE DISEASES
RESEARCH INNOVATION.
WE WILL GO THROUGH SOME OF THE
PROGRAMMATIC UPDATES.
I WISH WE COULD TALK ABOUT ALL
OF THE WONDERFUL THINGS THE
DIVISION HAS BEEN UP TO, BUT
THERE'S JUST NOT ENOUGH TIME
TODAY.
THIS WILL BE SOME HIGHLIGHTS.
BEFORE I BEGIN, I WOULD BE
REMISS IF I DIDN'T ACTUALLY
SHOW YOU WHO IT IS THAT MAKES
UP THIS EXCELLENT TEAM RUNNING
ALL THE WORK I WILL BE TALKING
ABOUT TODAY.
THIS IS THE DRDRI STAFF.
AND IN THE RARE DISEASE
COMMUNITY WE OFTEN SAY RARE
DISEASES ARE SMALL BUT MIGHTY.
I SAY THE SAME THING ABOUT
DRDRI.
WE ARE A SMALL BUT MIGHTY TEAM.
IT'S THESE INDIVIDUALS WHO WORK
HARD EVERY DAY TO ACCOMPLISH
ALL OF THE PROGRAMS WE RUN AND
ADMINISTER AS WELL AS THE ONES
WE WILL JUST BE HIGHLIGHTING
TODAY.
BEFORE WE BEGIN, I THINK IT'S
IMPORTANT TO JUST TALK ABOUT
WHY RARE DISEASE RESEARCH IS SO
IMPORTANT.
THIS IS A SLIDE THAT MANY OF
YOU MAY KNOW SOME OF THE
EFFECTS ON HERE.
THERE ARE NOW MORE THAN 10,000
RARE KNOWN DISEASES.
THIS IS A STAGGERING NUMBER.
WE DON'T KNOW HOW MANY
AMERICANS ARE AFFECTED.
WE KNOW IT'S MILLIONS.
MILLIONS OF AMERICANS ARE
AFFECTED BY RARE DISEASES EVERY
DAY.
ONLY 5% OF THOSE DISEASES HAVE
TREATMENT.
IT'S A HUGE UNMET NEED.
IT'S REALLY AN UNFATHOMABLE
UNMET NEED.
YOU PARTNER THAT WITH THE RIGHT
SIDE OF THE SLIDE, THE TIME
FROM EARLY DEVELOPMENT TO THE
MEDICINE CABINET TAKES 10-15ER
>>S.
-- YEARS.
AND NINE OUT OF TOUCHDOWN OF
THOSE PROMISING THERAPEUTIC
CANDIDATES THAT ENTER CLINICAL
TRIALS WILL FAIL.
WITH A TREMENDOUS NUMBER OF
RARE DISEASES WITH NO
THERAPEUTICS, A 10-15 TIMELINE
TO GET A CANDIDATE IN THE
MEDICINE CABINET AND 90% OF
THOSE WILL FAIL.
THIS SHOWS THE COMPOUNDING
PROBLEM WE HAVE IN RARE DISEASE
THERAPEUTIC DEVELOPMENT AND WHY
MORE RESEARCH IS NEEDED.
THEN WE ADD TO THAT, WHAT IS
THE BURDEN OF RARE DISEASES,
THIS IS REALLY JUST LOOKING AT
ECONOMIC BURDEN.
THIS SLIDE SHOWS WORK DONE LED
BY ANNIE KENNEDY.
THE EVERY LIFE FOUNDATION.
I LIKE THIS BECAUSE IT BREAKS
DOWN WHAT WAS LOOKED AT.
THEY LOOKED AT NOT ONLY DIRECT
MEDICAL COSTS, WHICH IS WHAT WE
MIGHT THINK OF WHEN WE THINK OF
A BURDEN OF A HEALTH CONDITION.
BUT ALSO THE INDIRECT COSTS.
SO THERE'S QUITE A BIT OF
PRODUCTIVITY LOST FOR
INDIVIDUAL WITH THE RARE
DISEASE.
BUT POTENTIALLY ALSO CARE
TAKERS OR THOSE AROUND THEM
HELPING THEM DURING THEIR RARE
DISEASE JOURNEY.
AS WELL AS NON-MEDICAL AND
UNCOVERED HEALTHCARE COSTS.
SO WHAT THEY FOUND WAS THAT THE
TOTAL ECONOMIC BURDEN OF RARE
DISEASE IN THE U.S. IN 2019 WAS
ALMOST A TRILLION DOLLARS.
SO WITH ALL THAT SAID, WE WANT
TO BRING THIS BACK TO NCATS AND
OUR ROLE.
THIS HAS A HOME AT NCATS, WILL
HEAR FROM MY COLLEAGUE, MATT
HALL WILL TALK ABOUT RARE
DISEASE WORK IN THEIR DIVISION.
IT REALLY SPANS ACROSS THE
NCATS DIVISION AND OFFICES.
WE CREATE OPPORTUNITIES TO BE
ABLE TO FUND INNOVATIVE
RESEARCH TO ACCELERATE MEDICAL
RESEARCH ACROSS RARE DISEASES.
I THINK WE WILL CONTINUE TO
HAVE INCREASINGLY HAVE ABILITY
TO DATA WE CAN USE.
BUT WE CREATE RESOURCES TO
EDUCATE, ENGAGE AND EMPOWER THE
RARE DISEASE COMMUNITY.
THE RARE DISEASE COMMUNITY IS,
I THINK UNIQUE IN THE DRUG
DEVELOPMENT ECOSYSTEM BECAUSE
THEY ARE OFTEN PART OF THE
SOLUTION, IF NOT THE DRIVERS OF
SOLUTIONS FOR RARE DISEASES.
IF WITH DON'T HAVE AN EDUCATED
AND EMPOWERED GROUP, IT'S GOING
TO BE HARD FOR THEM TO BRING
ABOUT THE SOLUTIONS IN THE MOST
EFFICIENT AND EFFECTIVE WAY.
WE REALLY PARTNER WITH OUR
COMMUNITY.
THIS SLIDE, I GIVE YOU OVERVIEW
OF THE RARE DISEASES THE DRDRI
INVEST IN.
WE BREAK IT DOWN INTO THREE
CATEGORIES.
THE PATIENT SUPPORT AND
INFORMATION.
FUNDING RESEARCH AND
DEVELOPMENT PROGRAMS.
AS WELL AS CONVENING AND
PARTNERING.
THIS NEXT SLIDE HIGHLIGHTS
WHICH ONES WE ARE GOING TO TALK
ABOUT TODAY.
SOME OF THEM BRIEFLY, SOME OF
THEM IN A LITTLE MORE DEPTH.
THERE ARE SIGNIFICANT
BOTTLENECKS.
SOME OF WHICH WE HAVE
IDENTIFIED AND SOME WE HAVE YET
TO IDENTIFY.
AT NCATS WE WORK TO ADDRESS
THOSE LONG STANDING BOTTLENECKS
TO GET TO THOSE NEW TREATMENTS
FOR ALL PEOPLE MORE QUICKLY.
OVER THESE NEXT FEW SLIDES I
WILL TALK ABOUT SOME OF THE
PROGRAMS THE DRDRI IS INVOLVED
IN TO TRY TO TACKLE THESE
BOTTLENECKS.
THIS IS THE IMPACT OF RARE
DISEASES ON PATIENTS IN
HEALTHCARE SYSTEMS.
THIS IS QUANTIFYING FINANCIAL
AND MEDICAL UTILIZATION AND
COSTS WITHIN FOUR DIFFERENT
HEALTHCARE SYSTEM DATABASES.
SO PRELIMINARY ANALYSIS FOR THE
PATIENTS.
THIS WAS A COLLABORATIVE EFFORT
THAT WAS BETWEEN NCATS INDUSTRY
AS WELL AS ACADEMIC
INSTITUTIONS AND INTERNATIONAL
HEALTHCARE COMPANIES.
THIS WAS REALLY NEAT BECAUSE IT
WAS CONDUCTED IN PARALLEL WITH,
BUT NOT HAVING KNOWLEDGE OF THE
WORK BEING DONE AT EVERY LIFE.
REALLY WE CAME UP WITH THE SAME
CONCLUSION.
THE METHODS WERE QUITE
DIFFERENT.
WE SELECTED 14 RARE DISEASES.
THEY SENT DISEASES THAT HAVE
IC9S AND 10 CODES WE CAN LOOK
AT THE ELECTRONIC HEALTH
RECORDS.
WE CAME TO THE SAME CONCLUSION.
WHICH REALLY LET'S US KNOW THAT
WE ARE IN THE RIGHT BALLPARK
FOR THE BURDEN OF DISEASE.
THE OTHER ASPECT THIS STUDY
LOOKED AT WAS THE RARE DISEASE
JOURNEY OF THE PATIENT.
AND THIS FIGURE HERE HIGHLIGHTS
WHAT YOU SEE ON THE LEFT IN THE
GRAY AREA IS THE COST.
YOU HAVE DOLLARS ON THEIR
MEDICAL JOURNEY BEFORE
DIAGNOSIS.
AND ON THE RIGHT IS THE COSTS
THAT WERE AFTER DIAGNOSIS.
THIS PARTICULAR PATIENT THAT WE
FOLLOWED IN THIS PATIENT
JOURNEY HAS A NEURODEGENERATIVE
DISEASE, CALLED BATTEN DISEASE.
HAVING THAT DIAGNOSIS HELPS
THEM TO BETTER UNDERSTAND AND
BETTER TREAT.
THIS HIGHLIGHTS HOW IMPORTANT
IT IS TO GET TO DIAGNOSIS.
THIS IS SOMETHING WE MENTIONED
EARLIER.
WE REALLY NEED TO BE ABLE TO
GET TO DIAGNOSIS EARLIER SO WE
CAN THEN APPLY TREATMENT
FASTER, MORE APPROPRIATE
TREATMENT FASTER.
AND THIS LEADS ME TO ANOTHER
INITIATIVE, ONE CALLED THE
SHORTENING DIAGNOSTIC ODYSSEY.
THIS IS A GRANT THAT ALICE
CHEN, I'M SORRY, I FORGOT TO
MENTION AINLAY TINSDALE THE
INITIATIVE AND ALICE CHEN IS
LEADING THE DIAGNOSTIC ODYSSEY
GRANT MECHANISM.
WE ASKED APPLICANTS TO USE
ANALYSIS IN CLINICAL APPROACHS
TO COME UP WITH SOLUTIONS TO
SHORTEN THE DIAGNOSTIC ODYSSEY.
HERE ARE THE THREE AWARDS MADE.
THEY ALL USE TECHNOLOGY
DIFFERENTLY TO CREATE SOLUTIONS.
AT RARE DISEASE DAY THIS YEAR,
THERE WAS A HIGHLIGHT BY DR.
LALANI ABOUT WHAT THEY ARE
USING, AS WELL AS DR. MULANI.
I WILL GO OVER WHAT DR. LALANI
SHOWED ME.
I ENCOURAGE YOU TO WATCH THE
RARE DISEASE WEBCAST TO HEAR
FROM BOTH OF THEM.
DR. LALANI'S PROJECT WAS CALLED
PROJECT GIVE, GENETIC INCLUSION
BY VIRTUAL EVALUATION FOR THE
RIO GRANDE VALLEY.
THE PROJECT GIVE TEAM WAS MADE
UP OF INDIVIDUALS ON THE BOTTOM
ROW COULD SEE INDIVIDUALS FROM
BAYLOR COLLEGE OF MEDICINE.
ON THE TOP YOU SEE THE
INDIVIDUALS THAT UNIVERSITY OF
TEXAS RIO GRANDE VALLEY.
THESE ARE PARTNERS THEY FOUND
IN THE LOCAL COMMUNITY WHO WERE
REALLY A CRITICAL PART OF THE
SUCCESS OF THIS PROJECT.
BECAUSE WHAT THEY RECOGNIZED IS
THAT THEY REALLY NEEDED LOCAL
INDIVIDUALS WITH WHOM THEY
COULD HELP TO BUILD COMPETENCE
AND EXPERTISE TO THEN ALLOW FOR
BETTER INTERACTION BETWEEN
POPULATION IN THE RIO GRANDE
VALLEY AND EXPERTISE THEY ARE
ABLE FOUGHT FORWARD.
THE RIO GRANDE VALLEY IN TEXAS,
AS YOU CAN SEE ON THE BOTTOM OF
THE MAP IS KIND OF AN AREA ON
THE SOUTHERN TIP OF TEXAS.
THIS IS REALLY AN AREA THAT HAS
A VERY HIGH HISPANIC POPULATION.
94% OF THE POPULATION IS
HISPANIC WITH A VERY HIGH
UNINSURED RATE AS WELL AS A
VERY HIGH POVERTY RATE.
THIS IS A MEDICALLY UNDERSERVED
AND SOCIOECONOMIC DISADVANTAGED
AREA WHERE THERE'S A LACK OF
EXPERTISE IN THE PROVIDERS AS
WELL AS LACK OF ACCESS.
THIS WAS AN AREA THEY WANTED TO
TARGET BECAUSE THE DISEASE RATE
234 RARE DISEASES WAS
NEGLIGIBLE BECAUSE THEY DIDN'T
HAVE ACCESS.
SO THE AIMS WERE TO REDUCE THE
TIME OF DIAGNOSIS.
TO USE A MACHINE-ASSISTED
VIRTUAL WEB-BASED SERVICE.
IT ALLOWED THEM TO USE
TECHNOLOGY TO REACH A
POPULATION THEY WOULDN'T HAVE
OTHERWISE BEEN ABLE TO REACH,
BUT USING LOCAL PROVIDERS.
THEY WANTED TO PROVIDE GENETIC
DIAGNOSIS THROUGH WHOLE GENOME
SEQUENCING.
BUILDING GENOMIC COMPETENCE TO
EXPEDITE THE REFERRALS.
AND THEY COULD GET ACCESS TO
THE PATIENTS TO PROVIDE SERVICE
TO THEM.
THIS SHOWS THEY HAVE TARGET
ENROLLMENT OF 100.
THEY WERE ABLE TO ROLL 115.
71 OF WHOM RECEIVED WHOLE
GENOME SEQUENCING RESULT.
THIS STUDY SHOWS DIAGNOSTICALLY
HAS BEEN 35%.
WHICH IS A SIGNIFICANT
DIAGNOSTIC RATE FOR INDIVIDUALS
WHO HAVE BEEN WITHOUT A
DIAGNOSIS THEIR ENTIRE LIVES.
AND THIS NEXT SLIDE SHOWS YOU
SOME OF THE FACES.
YOU SEE THESE ARE NOT JUST
NEWBORNS WHO ARE JUST, IT'S
BEEN DAYS SINCE THEY HAD THEIR
RARE DISEASES, IT'S INDIVIDUALS
LIVING UNDIAGNOSED, THEREFORE
NOT HAVING COMMUNITY OF
INDIVIDUALS THEY SHARE THEIR
DIAGNOSIS, NOT HAVING
TREATMENTS THAT ARE GEARED
TOWARDS THEIR DIAGNOSIS.
THIS PROGRAM REALLY HELPS TO
DEMONSTRATE HOW YOU CAN SOLVE
THIS ISSUE.
EVEN IN A MEDICALLY UNDERSERVED
AREA.
I HIGHLIGHT THIS ONE EXAMPLE
OF, YOU KNOW, WE HAVE MANY
EXCELLENT AWARDS.
BUT THIS IS JUST ONE EXAMPLE
THAT SHOWS A PROGRAM THAT USES
TECHNOLOGY, BUILDS TRUST WITH
THE COMMUNITY AND SHOWS HOW TO
MODEL THIS AND CREATE, REACH
OTHER MEDICALLY UNDERSERVED
AREAS IF THIS WERE TO BE
REPLICATED IN OTHER REGIONS.
SO MOVING ON TO OUR NEXT HURDLE.
WITH RARE DISEASES WE KNOW
THERE ARE SMALL NUMBERS OF
INDIVIDUALS.
WE KNOW THEY MAY BE -- IT'S
CHALLENGING TO COLLATE THE DATA
AND REALLY LEARN ENOUGH ABOUT
THESE DISEASES TO HAVE
EFFECTIVE CLINICAL TRIALS.
ONE OF THE PROGRAMS THAT'S OUR
FLAGSHIP IS THE RARE DISEASE
CLINICAL RESEARCH NETWORK.
THIS WAS ESTABLISHED BY THE
RARE DISEASES ACT OF 2002 AND
BEEN IN PLACE SINCE 2003.
IT'S BEEN JUST OVER 20 YEARS
SINCE THIS HAS BEEN IN PROCESS.
THIS WAS THE HIGHLIGHT OF OUR
UPDATE LAST YEAR.
THERE WAS A SIGNIFICANT AMOUNT
OF TIME SPENT ON THE RARE
DISEASES CLINICAL RESEARCH
CENTER OF EXCELLENCE LAST YEAR.
I WON'T GROW THROUGH THE
PROGRAM AND HOW IT RUNS BUT
REALLY JUST SOME HIGHLIGHTS.
THIS CURRENT CYCLE, THE RDCRN
CYCLE 4, 2019-2024, BECAUSE OF
THE COVID NO-COST EXTENSION
WOULD GO INTO 2025.
WHAT THIS HIGHLIGHTS IS THE
NUMBER OF RESEARCH GROUPS AND
MANY OF THE METRICS ON THE
DISEASES AND THEIR SUCCESSES.
SO THERE ARE 20 RESEARCH GROUPS.
EACH ONE OF THESE HAS TO STUDY
AT LEAST THREE DISEASES.
HERE WE TACKLE ONE OF THE
PROBLEMS.
ASKING THE RESEARCHERS TO STUDY
AT LEAST THREE DISEASES AT ONCE.
WE ARE ENCOURAGING THEM TO LOOK
AT WHAT'S COMMON AMONG THE
DISEASES SO THAT WE CAN GET TO
SOLUTIONS THAT WILL BE
APPLICABLE BEYOND JUST ONE
DISEASE AT A TIME.
ANOTHER KEY PART OF THIS IS THE
PATIENT ADVOCACY PARTNER.
LISTED HERE.
THERE ARE 170 PATIENT ADVOCACY
PARTNERS IN THIS CURRENT CYCLE.
ONE OF THE REQUIREMENT FOR THE
APPLICATION THEY HAVE TO ENGAGE
A PATIENT ADVOCACY GROUP IN
THEIR CONSORTIUM.
REALLY THE PATIENT ADVOCATE ARE
AN IMPORTANT PART OF THIS.
AND SIGNIFICANTLY CONTRIBUTE TO
THE RESEARCH THAT IS CONDUCTED
THROUGH THE RDCRN.
I WANT TO HIGHLIGHT THIS IS A
VERY CROSS-NIH COLLABORATIVE
PROJECT.
THERE ARE TEN INSTITUTES AND
CENTERS INVOLVED IN CURRENT
CYCLE OF THE RDCRN.
IN GETTING TO SOME OF THE
IMPACTS OF THIS, THIS SLIDE
HERE REALLY JUST HIGHLIGHTS ONE
OF THE TRANSLATIONAL IMPACTS.
I THINK JONI HAD MENTIONED THIS
EARLIER IN HER TALK.
IT IS EXCELLENT TO STUDY THESE
RARE DISEASES.
ALL OF THESE CONSORTIUM HAVE TO
CONDUCT A NATURAL HISTORY STUDY
WHICH ARE GETTING THEM READY
FOR CLINICAL TRIALS.
ULTIMATELY WHAT WE WANT ARE
TREATMENTS FOR INDIVIDUALS WITH
RARE DISEASE.
WHAT THE TABLE SHOWS IS THE 12
DIFFERENT FDA INDICATIONS THAT
HAVE BEEN FDA APPROVED FOR 11
DIFFERENT RARE DISEASES.
THESE WERE NOT SPONSORED BY
NCATS.
AS YOU CAN SEE THERE ARE
COMPANIES LISTED FOR THE
SPONSORS OF THESE DRUG
APPLICATIONS.
THE PIVOTAL PHASE 3 STUDIES ARE
ALSO NOT SPONSORED BY NCATS BUT
IT'S REALLY ALL THE WORK THAT
THE RDCRN DOES TO MAKE THESE
DISEASES TRIAL-READY, SO THEN
PHARMACEUTICAL COMPANIES CAN
COME AND SELECT THE DISEASES
THAT ALREADY HAVE THE STUDY,
HAVE THE OUTCOME MEASURES AND
MAY HAVE CONDUCTED EARLY
CLINICAL BASED TRIALS AND TAKE
IT ACROSS THE FINISH LINE.
THE OTHER THING I WANT TO
HIGHLIGHT ON THIS SLIDE, IF YOU
LOOK AT THE TIME LINE ON THE
APPROVED DATES ON THE RIGHT,
FIVE THESE HAVE BEEN IN THE
LAST YEAR PLUS.
IT BUILDS THE UNDERLYING
INFRASTRUCTURE FOR THESE
DISEASES THAT TAKE SIGNIFICANT
AMOUNT OF TIME TO DEVELOP SO
THEN THEY ARE READY TO HAVE
THESE PIVOTAL STUDIES.
I HOPE THIS IS REALLY JUST THE
TIP OF THE ICEBERG THAT AS EACH
ONE OF THESE RESEARCH GROUPS
CONTINUES TO ADVANCE THEIR
RESEARCH, BECAUSE THOUGH EACH
GET FIVE YEARS TO STUDY THESE
DISEASES, THIS NUMBER WILL
CONTINUE TO ACCELERATE AND WE
WILL CONTINUE TO SEE MORE
TREATMENTS.
YOU WANT TO HIGHLIGHT AND THIS
WAS TALKED ABOUT LAST YEAR
DURING THE BIG HIGHLIGHT BUT WE
ARE A LITTLE FURTHER ALONG IN
THIS PROCESS.
WE ARE WORKING CLOSELY WITH OUR
COLLEAGUES IN ITRB TO SHARE THE
DATA.
WHAT THIS WILL DO, IT TAKES THE
CONSENTED DATA FROM THE RDCRN
STUDY, IDENTIFIES T THE
COORDINATING CENTER PROVIDES
THE ASSURANCE OF DATA QUALITY,
DATA USE LIMITATIONS, MAKING
SURE DATA IS AVAILABLE AND THEN
DATA WILL BE PASSED ONTO THE
NIH IN THE SHARED DATA
ENVIRONMENT.
IT WILL BE GOVERNED THROUGH AN
NCAT STACK.
AND THEN AVAILABLE TO THE
PUBLIC.
THIS WILL BE REALLY A
TREMENDOUS RESOURCE FOR THE
PUBLIC TO HAVE ACCESS TO THE
DATA THAT'S BEEN COLLECTED
THROUGH THE RDCRN.
I WANT TO BRING YOUR ATTENTION
TO THE RDCRN WEBSITE.
YOU COULD GO TO
RAREDISEASESNETWORK.ORG.
DISEASES THAT ARE STUDIED AND
WORK THAT'S BEEN DONE.
THE A GREAT RESOURCE FOR
RESEARCHERS TO LOOK TO SEE WHAT
DISEASES ARE IN THERE AND
WHAT'S BEEN DONE, AND EVEN
PATIENTS AND PATIENT ADVOCATE
TO LEARN ABOUT IT.
FINALLY THE LAST THING I WILL
MENTION ABOUT THE RDCRN WE HAVE
A NOFO AVAILABLE.
THIS FOR THE NEXT CYCLE.
WE RECOMPETE EVERY FIVE YEARS.
SO THE DUE DATES ARE IN AUGUST
2024.
WE ENCOURAGE GROUPS TO THINK
ABOUT YOUR CONSORTIUM AND HOW
TO FIND DISEASES THAT HAVE
COMMONALITIES AND PUT FORTH AN
APPLICATION.
THROUGH THE QR CODE GET MORE
INFORMATION ABOUT THE
APPLICATION, AND THERE'S A
TECHNICAL ASSISTANCE WEBINAR
THAT WILL BE JUNE 3rd.
ANOTHER WAY WE WORK TO CREATE
SOLUTIONS FOCUSING ON MORE THAN
ONE DISEASE AT A TIME IS FOCUS
ON GROUND BREAKING GENETIC
APPROACHES, AS MARK MENTIONED
EARLIER THAT ARE REALLY
AMENABLE TO BEING MORE
STREAMLINED AND MORE BROADLY
APPLICABLE.
RARE DISEASES, THE DATA SHOW
ABOUT 70-80% OF RARE DISEASES
ARE CAUSED BY GENE MUTATIONS
WHICH ARE VARIANT.
SO THEREFORE USING GENETIC
APPROACHES MAY BE RELEVANT TO A
SIGNIFICANT NUMBER OF RARE
DISEASES.
I WILL SHOW THREE DIFFERENT
PROGRAMS.
ALTHOUGH THE FIRST ONE I WILL
ONLY SHOW YOU THIS SLIDE.
THE PLATFORM VECTOR GENE
THERAPY.
DR. MATT HALL WILL TALK ABOUT
THIS IN MORE DETAIL DURING HIS
PRESENTATION.
I WANTED TO HIGHLIGHT HOW THIS
IS AN EXAMPLE WHERE WE WORK SO
CLOSELY WITHIN THE NCATS
ECOSYSTEM.
MANY DIVISIONS AND OFFICES ARE
INVOLVED.
YOU COULD SEE THE DIFFERENT
FACES, COMING FROM DIFFERENT
DIVISIONS AND OFFICES.
NOT JUST NCATS WE ALSO WORK
WITH OTHER NIH INSTITUTES.
I THINK THIS REALLY
DEMONSTRATES IT, IT TAKES BROAD
EXPERTISE.
IT HAS TO BE INDIVIDUALS THAT
BRING DIFFERENT EXPERTISE TO
THE TABLE TO BE ABLE TO CREATE
SOLUTIONS THAT WILL BE
TRANSFORMATIVE.
SO I WILL LET DR. HALL TELL YOU
MORE ABOUT PAYGT.
I WILL TALK MORE ABOUT TALKING
ABOUT THE BESPOKE GENE THERAPY
CONSORTIUM.
THIS IS A PUBLIC PRIVATE
PARTNERSHIP THAT IS LED BY
NCATS AND COORDINATED THROUGH
THE FOUNDATION OF NATIONAL
INSTITUTES OF HEALTH OR FNIH.
THIS IS FROM THE PUBLIC SECTOR
AS WELL AS NUMERATOR PRIVATE
SECTORS AND NONPROFITS AS WELL.
IT'S TO STREAMLINE THE
FRAMEWORK TO ACCELERATE GENE
THERAPIES FOR RARE DISEASES.
ULTIMATELY WE WANT THIS TO BE A
PLATFORM WHERE LEARNINGS ARE
SHARED ACROSS COMMUNITY WHERE
WE ARE IMPROVING THE PROCESS,
SHARING THEM WITH THE COMMUNITY
SO THAT FUTURE SPONSORS, IT'S
DE-RISKED AND POTENTIALLY,
KNOW, AS PETER MARKS WAS
SAYING, YOU KNOW, A LOT OF
EFFICIENCIES HAVE BEEN WORKED
OUT AND SHARED SO THEY CAN
BENEFIT FROM THE LEARNING OF
THIS GENE THERAPY CONSORTIUM.
AND P.J. BROOKS AND JAMES FROM
DRDRI LEAD THIS PROGRAM.
ONE OF THE WAYS WE ARE SHARING
LEARNINGS IS BY CREATING A
PLAYBOOK.
THE VERSION 1.0 WAS JUST
RELEASED THIS YEAR AT THE
ACCELERATING PARTNERSHIP
MEETING.
BY LEVERAGING THE UP TO EIGHT
CLINICAL TRIALS THAT WILL BE
PERFORMED, CONDUCTED IN THE
BGTC, THE COMBINED EXPERTISE OF
ALL THE CONSORTIUM MEMBERS, WE
DEVELOPED THIS PLAYBOOK FOR
STREAMLINING THIS PRODUCT
DEVELOPMENT AND REGULATION OF
THE REGULATORY PATHWAY FOR
THESE AAV GENE THERAPY.
THE REGULATORY PLAYBOOK IS TO
ACT AS AN OPERATIONAL GUIDE
WITH TOOLS TO EMBARK ON THE
REGULATORY PROCESS.
PART OF WHY I MENTION THE PAYGT
IS THE LEARNINGS WE HAVE FROM
THESE PROGRAMS ACTUALLY HELP
EACH OTHER.
THE ORGANIZATION SECTION OF THE
PLAYBOOK SECTION 1.0 CAME FROM
OUR LEARNINGS.
WE LEARN FROM THE DIFFERENT
PROGRAMS AND APPLY THEM TO
CREATE EFFICIENCIES.
BGTC WILL CONTINUE TO ITERATE
AND CREATE NEW VERSIONS OF THE
PLAYBOOK AS WE GET FURTHER
ALONG THE PROCESS AND HAVE MORE
LEARNINGS TO SHARE.
I ENCOURAGE INDIVIDUALS TO
FOLLOW ALONG IN THE PLAYBOOK BY
LEARNING HOW TO IMPROVE THE
PROCESS.
THE FINAL GENETIC TECHNOLOGY I
WILL TALK ABOUT IS IN OUR
PROGRAM THAT SOMATOIC CELL
GENOME EDITING PROGRAM.
NOW THIS IS A PROGRAM THAT IS
CO-LED BY NIH COMMON FUND AND
NCATS AND NINDS.
THIS AIMS TO REDUCE THE BURDEN
OF CHANGE CAUSED BY GENETIC
CHANGES.
FY18-FY23 THE GOAL WAS TO
IMPROVE EFFICACY AND
SPECIFICITY OF GENOME EDITING
APPROACHES.
PERFORM AND ASSESS EFFECTIVE
GENOME EDITING TOOLS IN
SOMATOIC CELLS.
AND THEN THIS IS WHERE WE ALL
REALLY WANT TO BE, PHASE 2.
P.J. BROOKS AND LINDA HO FROM
MY DIVISION LEADS THIS PROGRAM.
DID I LOSE MY SLIDES?
WHAT I WAS GOING TO SHOW YOU,
THERE'S A TARGETED GENOME
EDITOR PRIZE, CALLED TARGETED
THAT IS CURRENTLY OUT.
THIS PRIZE CHALLENGE DELIVERING
GENOME EDITORS TO THESE SOMATIC.
ONE IS PROGRAMABLE DELIVERY
SYSTEMS FOR GENOME EDITING IN
DIFFERENT ORGANS AND CELLS.
TWO, HIGHLY EFFICIENT NON-VIRAL
DELIVERY.
LET ME SEE IF I CAN FIND.
HERE WE GO.
HIGHLY EFFICIENT NON VIRAL
DELIVERY ACROSS THE BLOOD BRAIN
BARRIER.
THIS SPEAKS TO WHAT YOU BROUGHT
UP EARLIER, ANNIE KENNEDY,
THERE ARE INDIVIDUALS WHO MAY
NOT BE ABLE TO USE AAV.
AV MAY NOT BE THE BEST DELIVERY
SYSTEM BECAUSE OF PREEXISTING
IMMUNITY OR OTHER REASONS.
SO TARGET AREA II OF THIS PRIZE
CHALLENGE WAS TO IDENTIFY NON
VIRAL DELIVERY THAT COULD CROSS
THE BLOOD-BRAIN BARRIER.
SO PHASE ONE, THIS IS A
MULTI-PHASE AWARD.
PHASE ONE HAS COMPLETED, THERE
WERE 30 WINNERS ACROSS THE TWO
DIFFERENT TARGET AREAS.
SUBMISSION DEADLINE IN JANUARY
OF 2025.
THIS WILL WORK TO CREATE
SOLUTIONS.
I'M GOING TO CLOSE TALKING
ABOUT OUR CONVENING AND
PARTNERING.
WHERE WE KNOW IT TAKES MULTIPLE
DIFFERENT STAKEHOLDERS.
AND WE NEED TO ENGAGE THEM ALL
AND BRING THEM TOGETHER IN
ORDER TO CREATE NEW SOLUTIONS.
YOU WILL HIGHLIGHT TWO RECENT
CONFERENCES AND WORKSHOPS WE
HAVE HAD.
SO ONE WAS ABOUT TEN DAYS AGO
NOW.
WE WORKED WITH OUR COLLEAGUES
AT THE FDA.
THE RARE DISEASE TEAM FOR
CENTER FOR DRUG EVALUATION AND
RESEARCH TO PUT TOGETHER THIS
NATURAL HISTORY STUDIES AND
REGISTRIS IN THE DEVELOPMENT OF
RARE DISEASE TREATMENTS.
THIS WAS A HYBRID WORKSHOP THAT
THE FOUNDATION HELPED TO CO-RUN
WITH US.
THIS WAS AN EXAMPLE HOW WE CAN
WORK TO EDUCATE THE COMMUNITY.
NOT ONLY THE PATIENTS AND
ADVOCATE WHO ARE OFTEN THE ONES
TRYING TO LEAD THE DEVELOPMENT.
SOMETIMES THEY ARE THE ONES
THAT ARE FUNDING AND RUNNING
THE REGISTRIES AS WELL AS
RESEARCHERS OR SPONSORS WHO
REALLY NEED TO UNDERSTAND WHAT
IS THE BEST WAY TO COLLECT DATA
TO USE IT FOR REGULATORY
PURPOSES.
IT'S ONE THING TO COLLECT
NATURAL HISTORY STUDIES DATA TO
LEARN ABOUT RARE DISEASES FROM
A CLINICAL CARE PERSPECTIVE.
AND DIFFERENT IF YOU WANT TO
USE IT FOR REGULATORY PURPOSES,
THERE ARE CONSIDERATIONS YOU
HAVE TO TAKE IN ORDER TO MAKE
SURE IT COULD BE USED IN THE
WAY YOU ARE INTENDING.
SO ONE OF THE THEMES THAT CAME
OUT THERE WAS START WITH THE
END IN MIND.
SOMETIMES YOU DON'T KNOW THE
END.
BUT CERTAINLY IF YOU ARE
THINKING ABOUT THERAPEUTIC
DEVELOPMENT, WHAT WAS DISCUSSED
IN THIS WORKSHOP, I THINK,
REALLY HELPED TO HIGHLIGHT WHAT
THE KEY FACTORS ARE THAT NEED
TO GO INTO DEVELOPING THE
NATURAL HISTORY STUDY REGISTRY.
AT THE CONFERENCE THERE WERE
ABOUT 230 INDIVIDUALS
REGISTERED IN PERSON AND ABOUT
3,000 VIRTUAL REGISTRANTS.
THIS IS QUITE A BIT OF NEED AND
INTEREST.
I WILL CLOSE OUT WITH TALKING
ABOUT OUR RARE DISEASE DAY.
BECAUSE THIS IS ONE OF MY
FAVORITE DAYS.
BEFORE I WAS IN MY ROLE BEFORE
I STARTED IN SEPTEMBER.
I USED TO ATTEND ON THE OTHER
SIDE, PATIENT ADVOCATE SIDE,
I'M A MOTHER OF AN INDIVIDUAL
WHO LIVES WITH A RARE DISEASE.
WHAT I LOVE ABOUT OUR RARE
DISEASE DAY IS COMMUNITY IT
BRINGSES TOGETHER.
IT IS PALPABLE, YOU ARE IN THE
ROOM AND SURROUNDED BY PEOPLE
THAT WANT TO BE THERE AND WANT
TO BE PART OF A COMMUNITY AND
WANT TO BE PART OF THE SOLUTION.
THESE PHOTOS, WHILE JUST STILL
SHOTS CAN'T CAPTURE THE
ENTHUSIASM AND EXCITEMENT IN
THERE.
IT HIGHLIGHTS HOW ENGAGED THIS
COMMUNITY IS.
WE HAVE ABOUT 600 INDIVIDUALS,
IN-PERSON ATTENDANTS AND I
THINK ABOUT 1300 VIRTUALLY.
WE KNOW NOT EVERYONE CAN TRAVEL
IN, ESPECIALLY WITH MEDICAL
DISEASES THEY MAY NOT BE ABLE
TO, OR A CAREGIVER OF A CHILD
WHO CAN'T LEAVE.
THROUGH OUR HYBRID VIRTUAL
PLATFORM IT ALLOWS INTERACTION.
COMMUNITY MEMBERS CAN INTERACT
WITH EACH OTHER, AND CONNECT,
SO THEY DON'T MISS OUT ENTIRELY
IF THEY AREN'T THERE IN PERSON,
THOUGH WE LOVE IT WHEN THEY ARE
IN PERSON.
WE HAD OVER 100 EXHIBITORS
WHICH HIGHLIGHT THERE'S IS SO
MUCH GOING ON IN THE RARE
DISEASE SPACE AND PEOPLE ARE
EAGER TO SHARE THEIR LEARNING,
TO CONNECT WITH OTHERS.
AND REALLY WHAT THEY WANT IS TO
NOT HAVE THAT FIRST SLIDE OF
LESS THAN 5% OF RARE DISEASES
HAVE TREATMENT.
SO THESE PEOPLE ARE THE BRAINS
AND HEART BEHIND THIS, BUT IT'S
A PROGRAM THAT WE ENGAGED OTHER
INDIVIDUALS FROM THE NIH, AS
WELL AS OUTSIDE TO HELP TO PLAN
IT.
THESE ARE JUST, YOU KNOW, A FEW
MORE SLIDES HIGHLIGHTING SOME
OF THE UNIQUE ASPECTS OF THIS
CONFERENCE.
YOU SEE THERE ON THE TOP LEFT,
WE HAD INDIVIDUALS REACH OUT TO
US AND SAY WE HAVE RARE DISEASE
ART WE WANT TO SHARE WITH THIS
COMMUNITY.
THERE WERE DIFFERENT ART
EXHIBITS ABOUT RARE DISEASE,
PEOPLE THAT ARE THRIVING OR
LIVING WITH RARE DISEASES THAT
WANT TO SHARE THEIR STORY.
DOCTOR EARLIER TALKED ABOUT THE
COUNCIL WHICH IS REALLY AN
INCREDIBLE MOMENT TO SEE THEM
SHARE THAT PROCLAMATION.
DR. MONICA BERTAGNOLLI.
WE WERE HONORED TO HAVE HER
COME AND PRESENT.
AND I HAD THE OPPORTUNITY TO
SHARE A LITTLE BIT ABOUT MY
JOURNEY, AS I MENTIONED, I'M A
MOTHER TO A -- SHE IS NO
LONGER THAT LITTLE, SHE IS 17
YEARS OLD.
A YOUNG LADY WITH A RARE
DISEASE AND HOW IT REALLY
LAUNCHED ME INTO A DIFFERENT
CAREER PATH THAN I THOUGHT I
WAS GO.
SCHOOL GRADLATION.
-- GRADUATION.
THIS SOMETHING WE HAVE THE
SUPPORT OF THE BROADER NIH
COMMUNITY.
THE BUILDS LIT UP FOR RARE
DISEASE DAY ARE SYMBOLIC FOR
THE CROSS NIH SUPPORT WE HAVE
FOR IT RARE DISEASE DAY.
I'M GOING TO LAND ON THE FINAL
SLIDE THAT HIGHLIGHTS SOME
PROGRAMS AND INITIATIVES THAT
WE HAVE AT DRDRI.
I WAS ONLY ABLE TO TOUCH ON A
FEW OF THEM.
BUT I'M HAPPY TO OPEN IT UP FOR
QUESTIONS.
>> Joni Rutter: FANTASTIC.
THANK YOU, DOMINIQUE.
WE DO HAVE TIME FOR QUESTIONS.
SO ANY QUESTIONS FROM
COUNCILMEMBERS?
YOU SAID IT ALL.
I SEE ANNIE LOOKING AT ME.
>> I TRY NOT TO MONOPOLIZE.
FIRST, JUST REALLY THANK YOU.
MAYBE NOT A QUESTION, AS MUCH
AS YOU HAVE ONE SLIDE THAT HAD
A LOT DIFFERENT INFOGRAPHICS OF
IT, I SENT A PICTURE OF IT AND
SENT IT TO MY TEAM.
THERE ARE SOME IMAGES THAT
SHOULD BE WRAPPED AROUND
METROBUSES AROUND TOWN AND
BILLBOARDS.
BUT THAT IMAGE ITSELF THAT HAD
ALL THE INFOGRAPHICS STARTED
WITH THE NUMBER OF GCRN SLIDE
THAT TALKED ABOUT THE NUMBER OF
DISEASES, PROJECTS, PARTNERS
AND CLINICAL SITES AND 11 AND
12 PATIENT COMMUNITIES.
I THINK THE MORE THOSE MESSAGES
CAN BE AMPLIFIED AND SHARED,
THE BETTER.
BUT THERE IS NO MORE PROOF IN
THE PUDDING OF THE WORK THAT IS
BEING DONE HERE.
NOT JUST HERE BUT THROUGHOUT
THE NATION OF FUNDING.
IT WAS ARTICULATED BEAUTIFULLY
IN THAT SLIDE.
I WANT TO APPLAUD THE MANY
PEOPLE WHO HELPED TO PUT THAT
TOGETHER AND TO SAY MORE, MORE
OF THAT.
I REALLY LIKE THAT SLIDE TOO.
IF WE CAN GET IT ON THE
METROBUS WE WILL TRY TO FIGURE
IT OUT.
>> Joni Rutter: EMILY?
EMILY IS OUR COMMUNICATIONS
DIRECTOR AND IS AMAZING AT WHAT
SHE DOES.
THANK YOU FOR THAT COMMENT.
ANY OTHER COMMENTS FROM THE
COUNCIL?
>> GREAT QUESTION, GREAT TALK.
GOING BACK TO THE SLIDE ABOUT
THE DRUG APPROVAL.
IS THERE SOME WAY TO TRACE IS
BACK TO THE RDCRN AND SEE IF
THERE WAS SOMETHING SPECIAL OR
IF IT WAS LUCK?
>> GREAT QUESTION.
IS THERE SOMETHING ABOUT THOSE
THAT ALLOWED THEM TO BE MORE
DRUG-READY I WILL SAY?
THAT HASN'T BEEN LOOKED AT, TO
MY KNOWLEDGE.
CERTAINLY SOMETHING WE CAN LOOK
AT.
I WON'T EVEN TELL YOU MY GUT.
THERE ARE SOME CONSORTIA THAT
MAY NOT HAVE APPROVAL.
THERE ARE PROBABLY OTHER WAYS
TO MEASURE SUCCESS BEYOND THAT.
IT WOULD PROBABLY BE
INTERESTING IF THEY ARE ALREADY
DOING IT TO GO BACK AND LOOK AT
THE FIRST EARLY CONSORTIA STILL
BEING FUNDED AND LOOK TO SEE IF
THERE ARE OTHER MEASURES OF
SUCCESS THAT WE COULD ALSO BE
AMPLIFYING.
AND THEN WHAT IS THAT SECRET
SAUZ.
-SAUCE.
AND IF THERE ARE UNDER
PERFORMERS AND WHAT COULD BE
DONE TO BRING THEM ALONG AS
WELL.
>> THOSE ARE GREAT SUGGESTIONS,
THANK, ANNIE.
AND BALKI, THANK YOU FOR THE
QUESTION AS WELL.
>> Joni Rutter: ANY OTHER
QUESTIONS?
I'M LOOKING ONLINE.
I DON'T SEE ANY HANDS.
THANK YOU SO MUCH, DOMINIQUE,
REALLY APPRECIATE YOUR
PRESENTATION.
NEXT UP I'M PLEASED TO PRESENT
MATT HALL.
SO TAKE IT AWAY.
MATT.
>> MATT HALL: THANK YOU, JONI.
I WAS LUCKY ENOUGH TO PRESENT
TO COUNCIL ONCE, BUT IT WAS
REMOTE.
IT'S NICE TO SEE PEOPLE HERE IN
PERSON AND SHARE A LITTLE BIT
ABOUT MYSELF SO THE
INTRODUCTION I'M PLANNING TO
USE IS AN INTRODUCTION TO ME.
THIS WAS A REQUEST, NOT THAT I
WANT THAT.
I WILL TALK ABOUT THE OFFICE OF
PRECLINICAL INNOVATION AND OUR
ROLE WITH PARTNERS OUTSIDE NIH
AND NCATS AS WELL.
TALK ABOUT OUR APPROACH AND
SOME THINGS I'M LOOKING FORWARD
TO WORKING WITH MY COLLEAGUES
ON DPI IN THE FUTURE, SUCH AS
BUILDING RESEARCH PLATFORMS FOR
RARE DISEASES AND FILLING IN
SOME OF THOSE GAPS.
MYSELF, YOU MAY HAVE DETECTED A
SLIGHT ACCENT, AUSTRALIAN BY
BIRTH AND STUDIED AT THE
UNIVERSITY OF SYDNEY, I HAVE
THE OBLIGATORY KANGAROO PHOTO
HERE.
DURING MY Ph.D. I WAS LUCKY
ENOUGH TO LIVE IN THIS REALLY
HAPPY INTERSECTION BETWEEN
MOLECULAR PHARMACOLOGY,
UNDERSTANDING HOW DRUGS WORK
AND INORGANIC CHEMISTRY,
CISPLATIN, WHICH IS ONE OF THE
MOST COMMONLY USED DRUG IN
CHEMOTHERAPY.
I DECIDED THIS IS WHERE I
WANTED TO BE.
SO I CAME HERE.
I CAME FROM THE NATIONAL
INSTITUTES OF HEALTH WHERE I
WORKED WITH MIKE GOTTESMAN.
A PROTEIN, IT TAKES DRUGS AND
PUMPS THEM OUT OF CELLS SO THEY
CAN'T GET IN.
ONE CHALLENGE WITH
CHEMOTHERAPY, CELLS MAY
INITIALLY BE KILLED BY
CHEMOTHERAPY BUT THEY BECOME
UNRESPONSIVE OVER TIME.
I SPENT A LOT OF TIME WORKING
ON DEVELOPING THERAPEUTICS.
THESE PUMPS PLAY A CRITICAL
ROLE IN THE BLOOD-BRAIN
BARRIER, KEEPING DRUGS OUT OF
THE BRAIN.
A COLLEAGUE WITH ME AT NCATS.
KYLE BRIMACOMBE.
A FEW OTHER ENTHUSIASTIC AND
BRIGHT-EYED YOUNG PEOPLE
BUILDING THIS DRUG DISCOVERY
CENTER.
WE DESIGNED AN ASSAY.
I WAS A COLLABORATOR.
BEING A DIFFERENT KIND, ON
DIFFERENT SIDE OF THE FENCE.
WE WERE MIXING CELLS, GREEN
ONES THAT WERE VERY RESISTANT
AND RED ONES THAT WERE
SENSITIVE.
IT WAS A VERY SUCCESSFUL
RESEARCH PROJECT AND RESULTED
IN A NICE DESCRIPTION AND
BETTER UNDERSTANDING.
BUT IT MADE ME WANT TO BE AT
NCATS AND CONTINUE WORKING, BE
PART OF WHAT THEY WERE DOING
AND I COULDN'T BE HAPPIER.
THAT'S AMAZING.
I'M REALLY HAPPY.
SO I WORK WITH THIS TEAM HERE
AT DPI.
THIS IS MY DAY JOB AS A
RESEARCH LEAD AS PART OF A TEAM.
THIS LOOKS LIKE ONE GROUP,
THAT'S HOW IT IS SUPPOSED TO
LOOK BECAUSE THAT'S HOW WE
OPERATE.
THERE'S THREE GROUP, MEDICINAL
CHEMISTRY, MIN CHEN WHO LEADS
THE DATA SCIENCE.
AND MYSELF AS WELL.
SOME OF MY BIOLOGY GROUP TEAM
LEADS IN THE PHOTO, ON THE
RIGHT.
WE WORK TOGETHER PARTNERING
WITH OTHERS OUTSIDE NCATS TO
DEVELOP ASSAYS, SCREENINGS AND
DEVELOP MOLECULES FOR A RANGE
OF TARGETS AND RANGE OF
DISORDERS, MANY OF WHICH ARE
RARE DISEASES.
BY THE TIME COVID CAME, WE WERE
REALLY LUCKY TO TAKE EVERYTHING
WE WERE DOING AT DPI, KIND OF A
PLATFORM PLAYBOOK AND DIRECT IT
TOWARDS COVID.
REALLY GRATIFYING IN THIS PAPER
PUBLISHED LAST YEAR WITH
FRANCIS COLINS AND OTHERS.
I WAS INCLUDED IN.
TWO OTHER KEY HIGHLIGHTS DR.
COLINS AND OTHERS POINTED OUT
FROM THE EARLY DAYS OF OUR
RESPONSE, THE OPEN DATA PORTAL
I SPOKE ABOUT 3R50E6 YUSLY,
WHICH WAS REALLY INTENTIONALLY
GENERATING TRANSLATIONAL
SCIENCE DATA AND SHARING WITH
THE COMMUNITY OPENLY.
OPEN DATA SHARING.
AND ACTIV TRACE PROGRAM, OPEN
PUBLIC PRIVATE PARTNERSHIP.
IT MADE A LOT OF DIFFERENCE
FROM A COVID RESPONSE.
SO AS COVID WAS WINDING UP, I
FIND MYSELF HERE AS THE
SCIENTIFIC DIRECTOR AND
OPPORTUNITY TO ACKNOWLEDGE
ANTON, NOT ONLY MY PREDECESSOR
BUT LUCKY ENOUGH TO HAVE AS A
COLLEAGUE GOING FORWARD.
I WILL HAVE THE WISDOM AND
EXPERTISE TO RELY ON, PROBABLY
DIFFERENT TO MANY.
THANK YOU, ANTON FOR EVERYTHING
YOU HAVE DONE AND WILL DO, EVEN
THOUGH YOU DON'T KNOW WILL BE
DOING IT.
[LAUGHTER]
A LITTLE PIVOT TALKING ABOUT
RARE DISEASES AND MARRYING UP
WITH DOMINIQUE'S PRESENTATION.
I TOOK MY DAUGHTERS TO A MUSEUM.
HAD ALL THESE PILLS AND
MEDICINE.
I KNOW MY I.T. FRIENDS ARE KEY
TO PRESSING PLAY ON THIS.
WHAT YOU WILL SEE, I'M WALKING
ALONG AND JUST TAKING A VIDEO
OF THIS INCREDIBLY LONG 60 FOOT
DISPLAY OF PILLS AND MEDICINES
SOMEONE TAKES IN A LIFETIME.
THE A REPRESENTATIVE DISPLAY OF
RECORDS OF INDIVIDUAL PEOPLE.
IT'S THE AVERAGE PERSON,
THERE'S NO SOUND JUST MY
DAUGHTER TELLING ME SHE IS
HUNGRY IN THE BACKGROUND.
IT'S AN INCREDIBLY COMPELLING
DISPLAY, SEE THE VACCINES.
WORKING WITH RARE DISEASE
PATIENTS ALL THE TIME.
IT CERTAINLY DOESN'T LOOK LIKE
THIS, BUT IT'S WHAT WE WANT
RARE DISEASE PATIENTS TO HAVE.
WI WANT THEM TO HAVE THOSE
MEDICINES.
I THINK IT STRUCK ME IN MY OWN
LIFE I HAVE TWO FAMILY MEMBERS
WITH, TWO NEPHEWS, LIVING VERY
DIFFERENT RARE DISEASE LIVES.
COHEN WHO HAS CYSTIC FIBROSIS
AND BENEFITS ALREADY FROM A
YOUNG AGE.
A TRANSPORTER LIKE -- PROTEIN.
IT DOESN'T GET TO THE SURFACE
TO PUMP THINGS THE WAY IT IS
SUPPOSED TO.
ON THE OTHER HAND I HAVE A
YOUNG NEPHEW ARCHER.
AFTER A LITTLE BIT OF
DIAGNOSTIC ODYSSEY, HE WAS
DIAGNOSED WITH DIGEORGE
SYNDROME, CAUSED BY LOSSES OF
30-40 GENES.
A COMPLEX RARE DISEASE.
THERE AREN'T TREATMENTS, IT'S
THE 95% DOMINIQUE WAS TALKING
ABOUT.
THINKING BACK TO THAT DISPLAY,
IT'S EASY TO UNDERSTAND HOW WE
ARE ALSO DEDICATED TO THE
MISSION OF TRYING TO BRING
TREATMENTS AND UNDERSTANDING TO
RARE DISEASES.
DIVISION OF PRECLINICAL
INNOVATION, I'M NOW THE
CLINICAL DIRECTOR UNDERSTANDING
TARGETS, DEVELOPING ASSAYS,
EXECUTING STREENINGS AND THE
CHEMISTRY NEEDED TO CREATE DRUG
LEADS AND EXPERTISE.
IT'S REALLY INCREDIBLE COMPLEX
SET OF STUDIES REQUIRED TO GO
TO THE FDA AND ASK APPROVAL FOR
CLINICAL TRIALS TO BE INITIATED.
THESE SCIENTISTS, THERE IS
ORGANIZATION BUT THEY WORK
TOGETHER ACROSS THIS
ORGANIZATION.
IN DEDICATED TEAMS AND AN ON AD
HOC WAY TO SOLVE PROBLEMS.
GENUINELY DIFFERENT ENVIRONMENT.
MY PERSPECTIVE I'M BRINGING IN
IS TO EMBRACE THIS, VERY
INTENTIONALLY BUILT.
WHEN I THINK OF THE DIVISION OF
PRECLINICAL INNOVATION, MY JOB
IS TO RECOGNIZE THE UNIQUE
NATURE OF DIVISION OF
PRECLINICAL INNOVATION, MURAL
ENVIRONMENT, NCATS INTRA MURAL
ENVIRONMENT, IT'S UNIQUE.
WE CONTINUE TO BUILD OUR
REPUTATION AS WORLD CLASS
TRANSLATIONAL SCIENCE INSTITUTE.
MY GOAL OVER THE COURSE OF MY
WORK TOO, WHEN PEOPLE THINK
ABOUT MEDICINE, THEY THINK
ABOUT NCATS.
THAT'S WHAT I WANT PEOPLE TO
THINK OF, AND WHEN PEOPLE THINK
OF RARE DISEASES, I WANT THEM
TO THINK OF NCATS.
SO THAT'S KIND OF THE LENS I
COME TO THIS THROUGH.
IT'S QUITE A UNIQUE ENVIRONMENT
BUT ALSO GIVES US A GREAT DEAL
OF RESPONSEABILITID.
A RELATIVELY STABLE ENVIRONMENT.
BUT IT ALSO MEANS WE HAVE THE
CONFIDENCE TO CHANGE WHAT WE
ARE DOING.
WE NEED TO EMBRACE THAT, AND WE
ALSO HAVE THE OBLIGATION TO
TAKE ON VERY HIGH RISK SCIENCE
AND LONG-TERM PROJECTS.
THERE'S NO REASON WE SHOULDN'T,
WE HAVE THE STABILITY TO
UNDERTAKE THOSE WITHOUT FEAR OF
THE CONSEQUENCES OF FAILURE.
THOSE ARE THE KIND OF PROJECTS
THAT MAY NOT FIT INTO A
TRADITIONAL ACADEMIC FUNDING
SPACE OR MECHANISM.
COURSE WE CAN WORK WITH OUR
LEADERSHIP TO DEFINE WI CAN
WORK TOGETHER, NOT JUST WITHIN
DRDRI.
THE OPERATIONAL MODEL WHICH IS
UNIQUE FROM OTHER PROGRAMS AT
NIH.
THE LABS AND DEDICATION TO
BUILDING TECHNOLOGIES ANLD
TRYING TO UNDERSTAND THOSE.
BUT ALSO I WISH HI MORE TIME TO
TALK ABOUT IT TODAY, I HOPE WE
HAVE A TALK SOMETIME ABOUT
INTRAMURAL AND EXTRAMURAL
PARTNERSHIPS.
THE WAY WE WORK WITH COLLEAGUES
AROUND THE TABLE HERE,
COUNCILMEMBERS, BUILDING
PROGRAMS WITH THE DIVISION OF
RARE DISEASE RESEARCH AND
INNOVATION.
EXTRAMURAL, INTRAMURAL, OFFICE
OF STRATEGIC INITIATIVES AND
OTHERS.
SO BEING A RESEARCH IN A
RESEARCH LAB ORGANIZATION, ONE
THING I'M THINKING ABOUT IS
MAKING SURE WE HAVE BALANCE.
WHEN WE ARE CONDUCTING SCIENCE,
MUCH IS COLLABORATIVE, OUR
PARTNERS ARE COLLABORATORS WHO
COME TO US, BE IT ACADEMIC OR
PARENT.
THEY COME TO US WITH A
THERAPEUTIC IDEA, CANDIDATE
PRODUCT OR JUST LOOKING FOR
ADVICE IN THIS TRANSLATIONAL
SPACE.
WHAT WE BRING IS OUR EXPERTISE
IN DRUG FORMING DEVELOPMENT TO
PARTNER WITH THEM.
AND WE HAVE AN INTEREST FOR
IMPROVING THE DRUG DISCOVERY
PROCESS TO REDUCE THE FAILURE
RATE OF THE PROCESS.
REALLY OUR COLLABORATORS COME
TO US, SPEAKING EXPERTISE.
WE USE THAT EXPERTISE THAT WE
MAINTAIN TO INFORM THE RESEARCH
DECISIONS WE MAKE.
ADVANCING TRANSLATIONAL SCIENCE
COMPONENT OF WHAT WE DO.
MY GOAL IS MAKE SURE WE HAVE
THE SPACE FOR SCIENTISTS TO
THRIVE, COLLABORATE AND
INNOVATE TO MAKE SURE WE ARE
BRINGING THERAPEUTICS TO
PATIENT.
AND THEY ARE MANAGED AS A
PORTFOLIO AS A WHOLE.
I CAN SHARE IN THE FUTURE WITH
YOU.
THERE WILL BE INTERNATIONAL
REPUTATION OF OUR PROGRAM.
WE ALREADY HAVE INTERNATIONAL
REPUTATION.
I WON'T GO TOO DEEPLY BUT GIVEN
WHAT I TOLD YOU ABOUT THE
UNIQUE NATURE OF OUR RESEARCH
PROGRAM AND HIGHLY
COLLABORATIVE NATURE, WE ARE
WELL PLACED AND I WANT TO WORK
WITH MY DPI COLLEAGUES, EMBRACE
THE ENVIRONMENT BEING BUILT AND
EMBRACE THE CHALLENGES THAT THE
RARE DISEASE COMMUNITY FACES TO
WORK TOGETHER TO WORK ON NOT
JUST THERAPEUTICS BUT THE
RESOURCES WE CAN MAKE AVAILABLE
TO THE RARE DISEASE COMMUNITY.
AND PLATFORMS WE CAN BUILD FROM
A RESEARCH POINT OF VIEW TO
ENABLE RESEARCH COMMUNITY TO
BRING MORE UNDERSTANDING AND
THERAPEUTICS FOR MORE RARE
DISEASES.
I THINK IT'S IMPORTANT TO PAUSE
AND SAY OF COURSE, DPI HAS A
WONDERFUL RECORD OF CLINICAL
SUCCESS AND ONE THAT IS GROWING.
WE FILED NOW ACROSS ALL THE
SCIENTIFIC GROUPS, OVER 50 IND
APPLICATIONS.
I SAY OVER 50, WE ARE DOING
CENSUS TAKING TO CAPTURE.
SOME ARE MOLECULES, AND SOME
THERAPEUTIC CANDIDATES WE
ENABLED.
I WILL GIVE YOU SOME EXAMPLES,
WE ARE ALREADY UP TO THREE DRUG
APPROVALS.
WHICH IS AMAZING.
NCATS HASN'T EXISTED OVER THE
LIFETIME OF A SINGLE DRUG
DEVELOPMENT LIFETIME.
ONE CHRONIC YEAST INFECTIONS,
ONE AADC DEFICIENCY, AND
DUCHENNE MUSCULAR DYSTROPHY.
I KNOW THAT WILL BE THE CASE
FROM THE LISTENING SESSIONS.
ONE OF MY EARLY PRIORITIES IS
REALLY MAKING SURE WE CAPTURE
AND COMMUNICATE THE SUCCESSES
WITH THE SCIENTIFIC COMMUNITY
WITH PEOPLE WHO CARE ABOUT
NCATS WHO WANT TO TELL THE
STORY INCLUDING OUR OWN
SCIENTISTS.
THIS IS A MOCK UP OF A WEB PAGE
WE ARE IN THE PROCESS OF
PUTTING TOGETHER WITH WONDERFUL
NCATS COMMUNICATION PAGE WHERE
PEOPLE WILL SEE THOSE THINGS
LISTED OUT CONTRIBUTED TO THE
FILING OR APPROVAL FOR.
WILL BE ABLE TO CLICK ON THAT
AND GET INFORMATION ABOUT THE
PRODUCT, WHAT PATIENTS ARE
APPROVED THROUGH TREATMENT OF
THIS MEDICATION.
WE ARE GOING TO HAVE A STRONG
FOCUS EARLY ON, COMMUNICATING
THE AMAZING CONTRIBUTION, NOT
JUST ALONG SIZE BUT
THERAPEUTICS.
ONE PROGRAM THAT EXISTED IN DPI
I KNOW YOU WOULD HAVE HAD
PRESENTATIONS ON PREVIOUSLY.
ESTABLISHED FORMALLY IN 2009
AND RUNNING BY 2010.
IT'S REALLY HAD AN AMAZING
PIPELINE OF THERAPEUTICS FOR
RARE DISEASES.
I MENTIONED THE THREE APPROVALS
AND A SIGNIFICANT NUMBER OF
IND'S, SORT OF IND FILING LINE.
YOU CAN SEE WHERE SOME OF THE
PRODUCTS ARE, CONTINUING
THROUGH CLINICAL TRIALS.
IT'S AN INCREDIBLY EXCITING
PROGRAM, PROFESSIONAL PROGRAM.
AROUND THE PROJECT MANAGERS
MANY OF WHOM HAVE PRIVATE
SECTOR EXPERIENCE.
STEER THE SCIENCE, PARTNERSHIP
WITH THE COLLABORATOR AND
SCIENCES WITHIN.
I CAN'T GIVE YOU DEEP DETAILS
ABOUT THEM, THE NEW PROJECTS
THAT HAVE BEEN ACCEPTED FOR
2024 HAVE BEEN SENT THEIR
ACCEPTANCE LETTERS.
THE AGREEMENTS AREN'T SIGNED
YET, SO I CAN'T LIST THEM OUT
FOR YOU.
I WOULD SAY THERE ARE TEN
ACCEPTED, AND NINE ARE FOR RARE
DISEASES.
YOU WANT TO SHARE WITH YOU, AND
REALLY CALL THAT OUT.
PARTNERS COME FROM ACROSS THE
SECTOR, ACADEMIA, BIOTECH,
CONSORTIUM, EIGHT ARE NEW
MOLECULAR ENTITY, ONE IS
REPOSITIONING PROJECT.
YOU CAN SEE THERE ARE A NUMBER
OF INDICATIONS THAT THESE NEW
PROJECTS COULD BE BUCKETED
INTO, THEY ARE ARRANGED BY
SUCCESS.
YOU CAN SEE REALLY DIVERSE IN
TERMS OF THE DISEASE AREA.
SOME PROJECTS MAY TAKE A YEAR
OR TWO, SOME MAY TAKE FIVE OR
MORE YEARS.
BUT THE COMMITMENT IS THERE IN
EACH CASE.
TO MOVE THESE THROUGH THE
PIPELINE AS APPROPRIATE AND
DEVELOP THEM AND GET THEM TO
THE FDA FOR IND EVALUATION AND
PATIENTS IN CLINICAL TRIALS.
DOMINIQUE TOUCHED BRIEFLY ON
PAVE-GT.
INCREDIBLY VALUABLE FROM A
SCIENTIFIC POINT OF VIEW, THE
HYPOTHESIS THAT CAME FROM DRI.
THIS IS A COLLABORATION FROM
DRDRI AND DPI.
TO TAKE A PLATFORM VECTOR
PROECH.
-- APPROACH.
TO INCREASE EFFICIENCY IN
CLINICAL TRIALS FOR GENE
THERAPIES.
WITH DPI'S EXPERTISES IN
PRECLINICAL DEVELOPMENT.
AND OF COURSE OUR LABORATORY
CAPABILITIES.
THE WAY THIS IS BEING PUT
TOGETHER UNDER THE CONCEPT
THESE FOUR, THERE ARE TWO
DIFFERENT DISEASES.
GENES IN TWO DIFFERENT RARE
DISEASE FAMILIES.
THE FOUR GENE THERAPY PROJECTS
ARE DEVELOPED IN PARALLEL,
NCATS PRE-CLINICAL SCIENTISTS.
THIS IS A TRANSNIH PARTNERSHIP
THAT BRINGS TOGETHER THE
CLINICAL CENTER TO CONDUCT
THOSE CLINICAL TRIALS.
THE COMMONALITIES WILL BE
LEVERAGED RIGHT AWAY THROUGH
THE PRE-CLINICAL PHASE AND
CLINICAL PROTOCOL AS WELL.
BEYOND THAT THOUGH, I SHOULD
SAY THIS PROGRAM, ALL THE GENE
THERAPY PRODUCTS ARE ADVANCING
THROUGH THE PIPELINES.
YOU COULD SEE THE CHEVRONS
HIGHLIGHTED HERE.
FDA INTERACTIONS HAVE ALREADY
DONE.
ALL WORKING THROUGH THE
DEVELOPMENT TO MAKE SURE IT'S
KNOWN THEY COULD BE
MANUFACTURED WELL AND DEFINED
DISCRETELY.
I WILL RETURN TO PAVE-GT
SHORTLY.
BUT ONE THING TO HIGHLIGHT,
IT'S NOT JUST ABOUT DEVELOPING
THE GENE THERAPIES FOR THOSE
PATIENTS BUT ACROSS INTYRE
COMMUNITY.
ENTIRE COMMUNITY.
THE COMMUNICATIONS WITH THE FDA
ALL THE DOCUMENTS BEING
CREATED, THE INTENTION IS TO
MAKE ALL OF THIS AS OPEN AS
POSSIBLE.
NOT JUST THE CLINICAL TRIAL
DATA BUT ASSOCIATED WITH
PREPARING THAT CLINICAL TRIAL.
ALL OF THAT IS MADE AVAILABLE
ON THE WEB.
YOU REQUEST CLICK ON THIS QR
CODE, YOU SHOULD DO LATER, NOT
NOW, BECAUSE I'M HERE AND YOU
ARE LISTENING TO ME.
BUT LATER YOU CAN SEE YOU CAN
SUBSCRIBE FOR UPDATES.
ALL OF THE DOCUMENTS AND
REQUESTS ARE THERE, CAN THINK
ABOUT THIS IN A WAY AS
REGULATORY REPURPOSING.
THE HOPE IS THIS DOCUMENTATION
AND SUCCESS IT IS LEADING TO
COULD BE LEARNED FROM BY OTHERS
TRYING TO WORK IN RARE DISEASE
FOUNDATIONS, BIOTECH.
SEE THE DOCUMENTATION AND WHAT
HAS WORKED AND WHAT COULD BE
REPURPOSED TO ACCELERATE AS
MANY AS POSSIBLE.
WONDERFUL EXAMPLE OF DATA
SHARING.
I WANT TO PUT A LITTLE VIGNETTE
ON OTHER I'M PARTICULARLY
EXCITED BY CURE ID.
DR. -- LEADS, PARTNERSHIP
WITH THE US FDA, CREATING A
DATA WAREHOUSE.
FOR INFECTIOUS DISEASES WITH
THE ORIGINAL GOAL BUT ALSO NOW
RARE DISEASES AS PART OF THAT
CONVERSATION.
THERE ARE NO APPROVED
TREATMENTS BUT THERE ARE PEOPLE
TREATED AND TREATED
SUCCESSFULLY.
WE WANT TO CAPTURE EACH CASE
STUDY.
CAPTURE TREATMENT EXPERIENCE
FROM CLINICIANS, EXPERIENCE.
THINKING ABOUT HEALTH RECORDS
AS WELL AND IDENTIFY SUCCESSES.
AND SHARE IT BACK WITH THE
SCIENTIFIC COMMUNITY SO OTHERS
CAN LEARN DPR IT.
IT'S AVAILABLE FOR iPHONE APP,
I DON'T WANT TO SAY A
PARTICULAR MANIER.
MANUFACTURER.
YOU CAN ACCESS THAT WEB PAGE,
BOTH HAVE EQUIVALENT
FUNCTIONALITY.
IT CAME OUT AT A PERFECT TIME,
THERE WAS AN UPDATE TO COUNCIL
AROUND 2019 AND THEN COVID CAME.
WE HAD A SIGNIFICANT AMOUNT OF
USES.
WE HAVE OVER 2,000 REGISTERED
USERS AND THE CLINICIAN REPORTS
ARE COMING IN.
WE HAVE BEEN ABLE TO TURN IN
ELECTRONIC HEALTH RECORDS.
EVEN THOUGH WE ARE THE U.S. AND
CARE ABOUT THE U.S. TAXPAYER,
NOT JUST U.S. ACTIVITY.
ALL OF THIS IS AVAILABLE TO THE
SCIENTIFIC COMMUNITY.
IT COULD BE KICK STARTING NEW
DRUG REPURPOSING PROGRAMS AS
WELL.
A WOND FULL EXAMPLE OF CROWD
SOURCING AND PROVEN TO BE VERY
SUCCESSFUL.
I THINK THE LAST THING I WANTED
TO SHARE WITH YOU TODAY, WAS AN
EXAMPLE OF THE KIND OF SCIENCE
AT DPI THAT REALLY EXCITES ME
BECAUSE I DIDN'T HAVE ANYTHING
TO DO WITH IT GETTING STARTED
AT ALL.
IT DIDN'T COME FROM LEADERSHIP,
IT CAME FROM THE SCIENTISTS
WITHIN DPI.
I SHOWED YOU OUR ORG OR
STRUCTURAL KHAIRT LATER ON.
I HAVE HIGHLIGHTED SOME OF THEM
HERE, WE ALL FOUND A FUNCTIONAL
LAB, STEM CELL, PHARMACOLOGICS
PROGRAM.
SMALL MOLECULE DISCOVERY,
INFORMATICS AND AUTOMATION, ALL
OF THE UNDERLYING TECHNOLOGIES
NEEDED TO MAKE NCATS SPECIAL.
THEY HAVE FOUND EACH OTHER AND
THEY ARE WORKING TOGETHER TO
TRY TO BUILD THIS PROGRAM
CALLED I3D-RARE.
WHAT IS IT?
WE KNOW THE PROBLEM.
DOMINIQUE DESCRIBED THE PROBLEM.
THE PROBLEM IS NOT ONLY THEY
DON'T HAVE TREATMENT, BUT THEY
DON'T HAVE A PIPELINE AVAILABLE.
WE DON'T HAVE THE UNDERSTANDING
AND MODELS NEEDED FROM A
PRE-CLINICAL DEVELOPMENT POINT
OF VIEW.
SO TACKLE THIS LACK OF
DISEASE-RELEVANT MODELS.
RECOGNIZE THAT NON ANIMAL MODS
NOT ONLY DON'T EXIST, SORRY,
ANIMAL MODELS DON'T EXIST FOR
RARE DISEASES BUT THE FDA
MODERNIZATION ACT 2.0, I DON'T
THINK CAME UP THIS MORNING,
WHEN DR. MARKS WAS SPEAKING.
CELL-BASED ASSAYS TO
INVESTIGATE SAFETY AND
EFFECTIVENESS OF A DRUG.
IF WE CAN CREATE COMPLEX
CELLULAR MODELS RARE DISEASE,
DO IT WITH A PLATFORM APPROACH
WE HAVE THE ABILITY TO
DEMONSTRATE AS PART OF THE
REGULATORY WORK.
BRINGING TOGETHER THE RESEARCH
ENVIRONMENT AND DRUG DISCOVERY
ENVIRONMENT WE HAVE MAXIMIZING
THE EFFICIENCY OF THOSE.
THESE PERSONALIZED OR
PREDICTIVE CLINICAL ASSAYS IS
PART OF THE GOAL.
STEM CELL DIFFERENTIATION IN
THE CELL PLEX.
WE HAVE A PROGRAM DESCRIBED TO
COUNCIL BEFORE.
THESE CELLS COULD BE BOUGHT TO
CREATE 3D CELLULAR MODEL.
BIO PRINTED.
RIGHT TO TISSUES ON A CHIP, WE
HAVE A WONDERFUL PROGRAM AT
NCATS THROUGH EXTRAMURAL.
USE THESE MOD HES TO DEVELOP
ASSAYS.
ASSESS SAFETY.
ARTIFICIAL INTELLIGENCE AND
OTHER OMICS MODELS AND OTHER
THINGS TO BEAR.
SO THEY ARE AVAILABLE FOR THE
SCIENTIFIC COMMUNITY TO
REPRODUCE.
ONE NICE ADVANTAGE OF AN
APPROACH LIKE THIS, A, IT'S NOT
MICE.
B, IT'S NOT JUST WUNL MOUSE
STRAIN.
YOU COULD BRING STRAINS FROM
DIFFERENT DONORS TO STUDY
REPRESENTATIVE CELL LINES
ACROSS A SINGLE DISEASE AND
REALLY EMPOWER OUR
UNDERSTANDING OF THE IMPACT OF
DIVERSITY IN THE RESEARCH WE
ARE DOING.
MOST OF US KNOW WE ARE ONLY
USING ONE CELL LIERN, IT'S
POSSIBLE THE SAME CELL LINE
PILOT STUDIES.
PAVE-GT, MODELS TO DEMONSTRATE
THE WORK.
ALSO ALAGILLE SYNDROME.
DISEASE MODELING, BUILDING
MODELS FOR THEM, DR. CATHERINE
CHEN HERE WHO WILL SPEAK LATER
ABOUT PROPOSAL IN THIS WORK.
AND ATAXIA AS WELL.
BRINGING ALL THESE SCIENTISTS
TOGETHER, WE DIDN'T BRING THEM
TOGETHER, THEY BROUGHT EACH
OTHER TOGETHER, THEY EMBRACED
THAT TEAM SCIENCE APPROACH WE
LOVE AT NCATS AND HOW TO BUILD
ON WHAT THEY STARTED AND
MAXIMIZE THE BENEFITS TO
PATIENTS.
THEY ORGANIZED WORKSHOP TO
BRING BACK FROM COMMUNITY, THIS
LINKS TO A VIDEO, DON'T GO TO
YET, YOU ARE WATCHING THIS
VIDEO, DO IT LATER.
YOU SEE A LOT OF INTERACTION OF
PEOPLE FROM INDUSTRY, NONPROFIT
SECTORS, GOVERNMENT AND
ACADEMIA, PROVIDING ADVICE AND
IMPACT ON THE I3D.
THERE ARE A SIGNIFICANT NUMBER
OF ATTENDEES, VERY EXCITING,
INCLUDING AN INTERNATIONAL
AUDIENCE AND HAVING A ZOOM
INSTANCE OR VIDEOCAST ALWAYS
MAXIMIZES THE NUMBER OF PEOPLE
WHO CAN LEARN FROM WHAT WE ARE
DOING AND PROVIDE.
WITH THAT, 28 MINUTES, I TRIED
TO GET THERE.
I JUST WANT TO REINFORCE, WE
HAVE A DEEP COMMITMENT TO THE
RARE DISEASE COMMUNITY AND TRY
TO GET.
WE WANT TO KEEP PATIENTS AT THE
CENTER OF EVERYTHING WE DO, DPI
SCIENCE IS AT THE ENTER AS WELL.
I WANT TO THANK THEM AND YOUR
ATTENTION AND LEAVE SOME IMAGES
OF THE PEOPLE DOING THE WORK
I'VE BEEN DESCRIBING TODAY.
THANK YOU VERY MUCH.
[ APPLAUSE ]
>> Joni Rutter: THANK YOU, MATT
THAT WAS TERRIFIC.
WE HAVE TIME FOR QUESTIONS.
I WANT TO MAKE A CLARIFICATION,
WHEN I SPOKE THIS MORNING ABOUT
THE IMAGING THERAPY, IT'S A
METHYLMALONIC ACIDEMIA, MATT IS
WORKING ON ACIDEMIAS AS WELL.
SO NOW WE ARE ACTUALLY TALKING
ABOUT THREE DIFFERENT ORGANIC
ACIDEMIA GENES WE ARE TRYING TO
PUSH THROUGH.
BUT THE MMA WON'T GO THROUGH,
IT'S ACTUALLY AHEAD OF THE GAME.
WE ARE GOING TO CAPITALIZE ON
THE FACT WE HAVE EVERYTHING IN
PLACE BECAUSE OF THE WORK THAT
WE HAVE BEEN DOING IT THE
INTRAMURAL PROGRAM AND BECAUSE
OF DOMINIQUE'S DIVISION AS WELL.
I THINK THAT'S JUST ONE OF THE
THINGS I WANTED TO MAKE SURE IT
WAS QUITE CLEAR.
>> YES, THANK YOU.
WE REALLY DEVELOPED AS THE
LEADER OF GENE THERAPIES AT NIH
OR FOR NIH, BUT FROM AN
INTELLECTUAL POINT OF VIEW,
UNDERSTANDING THE CLINICAL
CHALLENGES BUT ALSO
UNDERSTANDING THE COMPLEX
PRE-CLINICAL DEVELOPMENT
PIPELINE.
IT'S VERY EXPENSIVE SCIENCE IS.
BUT WORKING WITH OTHERS WE ARE
EXCITED FOR NEW GENE THERAPIES
AND GETTING THEM TO PATIENTS.
>> Joni Rutter: I COULDN'T
AGREE MORE.
ONE THING IT BRINGS IS THE
POWER OF THE PROGRAM MANAGERS
HELPING US TO DO THAT SO WELL.
COORDINATING ACTIVITIES NOT
JUST THROUGH NCATS BUT THE
GROUPS ACROSS NIH AND THEY ARE
INCREDIBLY EFFECTIVE.
I SEE YOUR HAND UP, GO AHEAD.
>> PAUL: ALL OF THE PROGRAMS
YOU INHERITED AS WELL AS ONES
YOU ARE PUSHING FORWARD.
YOU DID A GREAT JOB TELLING US
HOW THE SCIENCE, YOU HAVE GREAT
MECHANISMS TO LISTEN TO
SCIENTISTS AND THEY ARE
EMPOWERED TO CHIRP THEIR VOICE
AND THAT'S ONE OF THE REASONS
THIS IS COMING TOGETHER.
YOU ALSO HAVE NCATS GREAT
RESOURCE IN YOUR CONNECTION TO
ADVOCACY, AND COMMUNITY
ENGAGEMENT.
CAN YOU TALK A LITTLE HOW YOU
ARE USING THAT INPUT TO SUPPORT
THE DECISION IN THE PROGRAM
DIRECTION?
>> YEAH, CERTAINLY,
COLLABORATION PROPOSALS HAVE
COME TO US FROM ACADEMICS.
SOME, TAKE THE PRIVATE SECTOR,
SOME COME DIRECTLY FROM RARE
DISEASE FOUNDATION PARENTS.
OFTEN THROUGH CONNECTIONS
THROUGH DRDRI.
SOME WE PROVIDE ADVICE AND
GUIDANCE, OTHERS WE FORM
COLLABORATION TO HELP THEM WITH
THAT PROCESS AND MATCH MAKE.
I WOULD SAY THAT FROM A
COLLABORATION POINT OF VIEW
THAT OVER CONCENTRATED WE DO
THE BEST WE CAN, BUT LIMITED BY
THE BUDGET WE HAVE.
BUT WE DO A LOT OF MATCHMAKING
TO TRY TO BE AS HELPFUL AS
POSSIBLE IN PARTNERSHIP WITH
DRDRI.
IN TERMS OF COMMUNITY INPUT IN
THE PROJECTS WE TAKE ON, SOME
OF THE LATER STAGE PROJECTS I
WAS DESCRIBING, RARE DISEASES
PROGRAM THEY NEED TO SEE THE
PATHWAY AND HAS TO UNDERSTAND
THE LANDSCAPE OF OTHER
THERAPEUTICS AND WHAT IS THE
ENGAGEMENT IS WITH RARE DISEASE
COMMUNITY AND RARE DISEASE
FOUNDATIONS.
ONE OF THE THINGS JONI AND I
HAVEN'T HAD A LONG CONVERSATION
ABOUT YET, BUT ON MY LIST WHEN
BEING INTERVIEWED, IS HOW TO
BUILD A SCIENTIFIC ADVISORY, I
WAS GOING TO SAY BOARD, BUT
SOME TYPE OF GROUP WITH THE
RARE DISEASE COMMUNITY, RATHER
THAN FROM SCIENTISTS THEMSELVES.
AND TRY TO GET FEEDBACK FROM
THEM AND ALSO LET THEM HEAR
FROM US ABOUT WHAT WE ARE DOING.
COUNCIL IS A GREAT FORMAL WAY
TO BUILD ENGAGEMENT THERE.
RARE DISEASE DAY, MY FAVORITE
DAY OF THE YEAR, LIKE
EVERYBODY, WE GET TO GO,
DOMINIQUE YOU SAID BEFORE, BUT
THE IDEA THAT EVERYBODY
ACTUALLY WANTS TO BE THERE,
UNLIKE MOST THINGS I GO TO.
IT GENUINELY FEELS THAT WAY.
IT'S THE MOST MAGICAL DAY OF
THE YEAR.
TRYING TO NOT MAKE THAT A
ONE-DAY A YEAR THING IS VERY
IMPORTANT TO ME.
>> Joni Rutter: ALL RIGHT.
ANY OTHER QUESTIONS FOR DR.
HALL?
OKAY, SO MAYBE IN ANOTHER YEAR
WE WILL HAVE ANOTHER TOUR.
>> I WAS ABOUT TO SAY, PLEASE
COME AND VISIT ANY TIME.
AT LEAST TWO PEOPLE WHO WILL
NOT LIKE ME SAYING THAT.
PLEASE COME AND VISIT ANY TIME.
I'M SORRY I WASN'T HERE WHEN WE
DID THE LUNCH TOUR BUT ALWAYS
WELCOME.
THANK YOU VERY MUCH.
>> Joni Rutter: THANK YOU VERY
MUCH.
OKAY, WE ARE ABOUT, I WOULD SAY
ABOUT MAYBE FIVE MINUTES AHEAD
OF SCHEDULE.
SO LET'S TAKE A 20-MINUTE BREAK.
AND WE WILL RECONVENE AT 2:20,
WHERE WE WILL GO THROUGH OUR
CONCEPT CLEARANCES.
AND RIGHT AFTER THAT, WE WILL
GO INTO OUR STRATEGIC PLANNING
DISCUSSION.
OH.
AND THERE ARE SNACKS BACK HERE.
SO PLEASE, HELP YOURSELVES.
THANK YOU.
CLEARANCE OF CONCEPTS OF
SPECIAL INITIATIVES.
TAN TAGLE, ARE YOU THERE?
>> DAN: I AM.
>> I THINK HE IS IN JAPAN AND
IT'S LIKE 3:00 IN THE MORNING.
>> DAN: THANK YOU, JONI.
I'M DAN TAGLE, DIRECTOR FOR THE
OFFICE OF SPECIAL INITIATIVES
HERE AT NCATS.
IF YOU GO TO THE NEXT SLIDE,
PLEASE, OUR MISSION IN THE
OFFICE IS TO DEVELOP INNOVATIVE
SOLUTIONS THROUGH DISRUPTIVE
TECHNOLOGIES IN ORDER TO
ADDRESS MAJOR TRANSLATIONAL
PROBLEMS.
WITH THE END IN MIND OF
CREATING A PARADIGM SHIFT IN
THE FIELD.
SO THE NEXT SLIDE SHOULD GIVE
YOU, SORT OF LIKE A BIRD'S EYE
VIEW OF WHAT'S GOING ON RIGHT
NOW IN THE OFFICE.
WE CERTAINLY HAVE A NUMBER OF
DIFFERENT PROGRAMS THAT SPANS
FROM FUNDAMENTAL RESEARCH TO
REGULATORY ACCEPTANCE AND ALL
ADHERE TO PRINCIPLE OF
DEVELOPING, DEMONSTRATING AND
DISSEMINATING THE TECHNOLOGY.
FOR THIS CONCEPT WE HAVE A
PARTICULAR CONCEPT ON QUANTUM
TECHNOLOGIES THAT WILL BE
INTRODUCED BY DR. GEETHA
SENTHIL.
SHE HOLDS A DOCTORATE IN
MOLECULAR GENETICS FROM
UNIVERSITY OF GANDHI AND
SCOTLAND.
BECOMING ONE OF THE LEADING
EXPERTS NOT ONLY WITHIN NCATS
BUT WITH NIH IN QUANTUM
TECHNOLOGIES AND HELP
COORDINATE EFFORTS ACROSS
AGENCIES AND AS WELL AS
GLOBALLY.
WITH THAT I WILL TURN IT OVER
TO GEETHA.
>> GEETHA: THANK YOU, DAN FOR
THE INTRODUCTION.
I'M DELIGHTED TO PRESENT THIS
QUANTUM ENABLED TECHNOLOGIES
FOR BIOMEDICAL APPLICATIONS FOR
YOUR CONSIDERATION.
ONE OF THE CHALLENGES WE
DISCUSSED TODAY EARLIER IS ALSO
WHAT WE ARE GOOING TO TALK
ABOUT AGAIN, THE DETECTION OF
DISEASES EARLY ON.
WE ARE UNABLE TO DETECT MANY
DISEASES EARLY ON AND CAN'T
ACCURATELY DIAGNOSE THEM.
BECAUSE OF THE HIGH BACKGROUND
NOISE.
WE ARE UNABLE TO MONITOR THE
DISEASE OVER TIME BECAUSE THE
SIGNALS FROM THESE METHODS ARE
NOT STABLE, SO WE CAN'T JUST
TRACK THE CHANGES IN DISEASE
MARKERS.
THE OTHER ISSUE WE HAVE
COMPUTATIONAL LIMITATIONS IN
ANALYZING COMPLEX DATA.
THE CURRENT MACHINE LEARNING
METHODS DON'T REALLY WORK WELL
WITH SMALL DATASETS THAT ARE
NOISY, SUCH AS IN THE CASE OF
RARE DISEASES, WE HEARD FROM
DOMINIQUE WE HAVE SPARSE
INFORMATION ON THESE PATIENTS.
AND DIAGNOSING IS A CHALLENGE,
ACCURATE DIAGNOSIS IN RARE
DISEASES.
THE OTHER PROBLEM IS THE
METRICS WE HAVE CAN'T DETECT
SUCH DIFFERENCES TO DETECT
EARLY CHANGES RELATED TO
DISEASE.
SO THESE STRUGGLES WE HAVE IN
EARLY DETECTION AND ACTUAL
DIAGNOSIS.
AND THERE ARE NOW NEXT
GENERATION QUANTUM TECHNOLOGIES
THAT CAN HELP US SOLVE THIS
PROBLEM.
AND WHAT WE ARE PROPOSING IS TO
CATALYZE THE APPLICATIONAL
QUANTUM TECHNOLOGY TO PROVIDE
INNOVATIVE SOLUTIONS AND
BROADEN OUR CAPABILITIES IN
EARLY DETECTION, DIAGNOSIS AS
WELL AS THERAPEUTIC DEVELOPMENT.
WHY ARE WE EXCITED ABOUT THE
NEXT GENERATION QUANTUM
TECHNOLOGIES AND WHY DO WE
THINK THEY CAN SOLVE THIS PROB
HEM?
-PROBLEM.
[ NO AUDIO ]
>> JONI, I'M SORRY TO
INTERRUPT, BUT WE CANNOT HEAR.
WE LOST AUDIO.
>> Joni Rutter: OKAY, I SEE THUMB'S UP.
THANK YOU, SOMEHOW WE GOT
DISCONNECTED UP THERE.
>> GEETHA: I CAN GO BACK TO ONE
MINUTE BACK TO TALK ABOUT THE
QUANTUM SENSING TECHNOLOGY.
WE USE ENGINEERED ATOMS WE CAN
DETECT SINGLE MARKER WITH GREAT
SENSITIVITY.
LIKE THE CURRENT METHOD, THEY
ARE NOT SENSITIVE, THEY NEED
HUNDREDS AND THOUSANDS OF
MOLECULES TO BE PRESENT BEFORE
THEY WERE ABLE TO PICK UP THE
DISEASE.
IN THIS CASE THE CURRENT
METHODS DEPEND ON AVERAGE OF
SIGNALS FROM THOUSANDS OF
MOLECULES SO THEY ARE NOT
PRECISE OR SENSITIVE.
IN THE QUANTUM SENSORS SINCE WE
ARE MEASURING IT IN AN ATOMIC
SCALE WE ARE ABLE TO LOOK AT
INDIVIDUAL MARKERS SUGGESTED
CELLS, WE CAN MONITOR THEM OVER
TIME INSTEAD OF AVERAGING THE
SIGNALS.
WE CAN BE MORE PRECISE,
ACCURATE AND BETTER RESOLUTION.
IN THE CASE OF QUANTUM
COMPUTING, LET'S SEE, THE SLIDE
IS NOT ADVANCING.
OKAY.
THE CASE OF QUANTUM COMPUTING,
THE ENGINEERED ATOMS COULD BE
USED TO PERFORM COMPUTATIONAL
TASKS WITH ATOMS CALLED KUBITS.
-- QUBIT.
WE CAN COMPUTE ON COMPLEX
BIOMEDICAL DATA AT MUCH HIGHER
SPEED.
AND THEY CAN PROVIDE IMPROVED
PREDICTION.
SMALLER TRAINING DATASETS WHICH
IS PARTICULARLY INTERESTING IN
THE CASE OF RARE DISEASES.
WHERE ARE WE WITH THESE TWO
TECHNOLOGIES?
I WILL GIVE YOU TWO EXAMPLES TO
TALK ABOUT THE STAGE OF THESE
TECHNOLOGIES.
THE FIRST ONE IS QUANTUM
SPECIFIC TECHNOLOGIES, MORE
MATURE IT'S READY FOR YOUR OWN
APPLICATION AND EXAMPLE OF
QUANTUM ATOMIC MAGNETOMETER.
STUDIES ARE BEING DONE THROUGH
CLINICAL DEVICES CALLED SQUID
-- WHICH ARE BULKY AND THEY
NEED CRYOGENICS, THEY ARE
EXPENSIVE,.
THE MODERN ARE WEARABLE,
PORTABLE AND DON'T NEED
CRYOGENICKS.
WE CAN GET MUCH BETTER SIGNAL
QUALITY IN DETECTING REALLY
SUBTLE DIFFERENCES IN CARDIAC
FUNCTION DEFECTS AS WELL AS IN
THE BRAIN ACTIVITY.
THEY ARE EASY TO USE ON
CHILDREN TO DETECT FOR EXAMPLE,
IN EPILEPSY AND RARE DISEASES.
IF YOU HAVE HANDHELD DEVICE OF
THIS KIND TO USE ON A PREGNANT
WOMAN TO DETECT RARE GENETIC
CONDITION, FETAL CARDIAC
ARRHYTHMIA.
THEY ARE COST EFFECTIVE, THEY
CAN BE SCALABLE AND DEPLOYED IN
MORE SITUATIONS.
THEY ARE THE ADVANTAGES.
THE TYPE OF QUANTUM COMPUTING,
UNLIKE QUANTUM SENSING THIS IS
EARLY STAGE, IT'S EMERGING
THOUGH RAPIDLY.
THIS IS ONE EXAMPLE WHERE
QUANTUM COMPUTING COULD BE
USEFUL IN ACCELERATING DRUG
DISCOVERY.
THE CURRENT INDUSTRY STANDARD
COMPUTATIONAL METHODS ARE
TEDIOUS, LENGTHY, INVOLVES
MULTIPLE STEPS AND TAKES YEARS
AND MILLIONS OF DOLLARS AND
THEY ARE NOT ALL THAT ACCURATE.
WITH QUANTUM COMPUTING, THE
HOPE IS THEY CAN DO THIS MUCH
FASTER AND DO DRUG TARGET
INTERACTIONS SIMULATIONS MORE
ACCURATELY AND TO DESIGN MORE
SAFER EFFECTIVE DRUGS.
SO THE OPPORTUNITY WE HAVE,
WHAT IS THE OPPORTUNITY?
THERE ARE CURRENTLY SEVERAL
FUNDAMENTAL TECHNOLOGY
DEVELOPMENTS OCCURRING ACROSS
TRANSFEDERAL ACTIVITIES, THIS
IS A NATIONAL PRIORITY AT THE
WHITE HOUSE TO DOWNLOAD THE
TECHNOLOGY AS WELL AS ACCESS A
WAY OF ADOPTION OF THIS
TECHNOLOGY IN GLOBAL
APPLICATION.
SO WE DID TWO WORKSHOPS.
PROVIDING QUANTUM EXPERTS AND
BY MEDICAL EXPERTS, HAD THEIR
SESSIONS TO IDENTIFY SOME
OPPORTUNITIES.
WE HAVE BEEN COLLABORATING WITH
DOE.
WHAT WE LEARNED IS THERE ARE
SEVERAL AVAILABLE CORE QUANTUM
CORE TECHNOLOGIES, THEY HAVE
GREAT POTENTIAL BUT NOT BEING
DIRECTED TO BUY YO MEDICAL
APPLICATIONS.
THERE'S NO FOCUS INVESTMENT.
BIOMEDICAL EXPERTS ARE NOT
FAMILIAR WITH QUANTUM
TECHNOLOGY OPPORTUNITIES.
THE OTHER IS COST OF GENERATION.
THE OPPORTUNITY IS TO DERISK
THIS TECHNOLOGY TOWARD SOLVING
TRANSLATIONAL PROBLEMS IN EARLY
DETECTION SHUN AND DIAGNOSE I
DISCUSSED EARLIER AND ADVANCING
THE PROTOTYPE TECHNOLOGIES TO A
REAL WORLD APPLICATION.
WE ARE PROPOSING TO LAUNCH A
PRIZE CHALLENGE COMPETITION,
TWO OF THEM, TWO TARGET DATA
SENSING AND COMPETING TO
CATALYZE THE APPLICATION OF
THIS TECHNOLOGY TO
MULTI-DISCIPLINARY EFFORTS.
AND SPECIFICALLY AND QUANTUM
SENSING AREA CHALLENGE TO FOCUS
ON DEVELOPING NOVEL SCALABLE
ENHANCING PRE-CLINICAL DRUG
DISCOVERY.
DEVELOPING NON INVASIVE
SENSITIVE APPROACHES FOR
ENABLING EARLY DETECTION AND
IMPROVING ACTIVITY OF DIAGNOSE.
BUT PORTABLE AVAILABLE DEVICES
FOR INCLUDING PATIENT CARE AND
HEALTH MONITORING IN CASE OF
QUANTUM COMPUTING WHILE WE ARE
EXPECTING TO DEVELOP
ALGORITHMS, QUANTUM ALGORITHMS
FOR ACCELERATING DISCOVERY AND
IMPROVING MOLECULAR SIMULATION
AND DIAGNOSE RECEIVE DIAGNOSIS,
PARTICULARLY IN THE CASE OF
RARE DISEASES.
THE PRIZE CHALLENGE IS A
CRITICAL MECHANISM TO DERISK
THIS TECHNOLOGY TO US.
WE CAN RAPIDLY IDENTIFY BEST
SOLUTIONS AND MOVE THEM FORWARD
MORE QUICKLY.
THE REWARD THE TEAM OF QUANTUM
EXPERTS AND ENVIRONMENTAL
EXPERTS TO COME UP WITH THE
BEST SOLUTIONS.
WE ARE PARTNERING WITH NIH
AGENCIES WHO SHARE INTEREST IN
THE PART I SEE WHO ARE JOINING
ARE LISTED BELOW, NATIONAL
INSTITUTE OF BIOMEDICAL
ENGINEERING AND BIO
ENVIRONMENTAL ENGINEERING,
IBID, DATA SCIENCE STRATEGY,
CENTER FOR INFORMATION
TECHNOLOGY.
HOW WE PLAN TO LAUNCH THIS
INITIATIVE IS IN TWO STAGES,
STAGE ONE WE DO IDEATION.
INVITE INNOVATIVE SOLUTIONS
FROM A DIVERSE RANGE OF
AUDIENCE INCLUDING INDUSTRIES,
ACADEMIA AND QUANTUM EXPERTS
WHO ARE NOT NEUTRAL, SO IT'S A
GREAT WAY TO HAVE THEM COME
FORWARD TO BRING THEIR
SOLUTIONS.
IN STAGE TWO WE WILL IMPLEMENT
THE BEST SOLUTION TO REACTION
TO FACTOR CHALLENGE THROUGH
OTHER FUNDING MECHANISM TO
ADVANCES IN TECHNOLOGY TO
USEFUL AND VISIBLE APPLICATION.
THE BILATERAL AND TRANSLATIONAL
FIELD, THE SUCCESS OF THIS
PROGRAM, THEY ARE MORE
SENSITIVE AND ACCURATE THAN
WHAT WE HAVE CURRENTLY, THIS
INCLUDES QUANTUM ENHANCEMENT
MICROSCOPY,.
IT COULD ENHANCE THE CURRENT
COMPUTATIONAL LANDSCAPE WE WORK
WITH FOR DRUG DEVELOPMENT AS
WELL AS DIAGNOSIS.
THIS TECHNOLOGICAL, HAVE PUBLIC
HEALTH IMPACT, TO LEAD TO
BETTER TOOLS FOR THE DETECTION,
DIAGNOSIS AND THERAPEUTIC
DEVELOPMENT.
THE TEAM WE WILL BUILD WILL
HAVE A POSITIVE IMPACT ON
NATIONAL QUANTUM ECONOMY
ECOSYSTEM.
WE WILL CATALYZE THE NOVEL AREA
OF QUANTUM TECHNOLOGY
APPROACHES TOWARDS APPLICATIONS
IN DEVELOPING HUMAN HEALTH
CONDITIONS AND WE ARE EXCITED
TO LEVERAGE THIS UNPRECEDENTED
AND TIMELY OPPORTUNITY THAT
QUANTUM TECHNOLOGIES OFFER IN
ADVANCING DETECTION AND
DIAGNOSIS.
WITH THESE WE WILL BE ABLE TO
DETECT DISEASES EARLY ON AND
MORE ACCURATELY AND PROVIDE
BETTER PATIENT CARE.
AS WELL AS CONTRIBUTE TO THE
WORLD MISSION OF DEVELOPING
MORE TREATMENTS MORE QUICKLY
AND MAKE THEM MORE ACCESSIBLE
TO ALL.
THANK YOU, AND WITH THAT I
WOULD LOVE TO HEAR FROM YOU,
WHAT YOU ARE THINKING.
YOUR TALKS ON THIS INITIATIVE
AS WELL AS ANY AREAS WE SHOULD
CONSIDER AS PART OF THIS
INITIATIVE.
>> Joni Rutter: OKAY, THANK YOU
SO MUCH, GEETHA FOR THAT
OVERVIEW FOR THIS CONCEPT.
WE HAVE TWO DISCUSSANTS.
I WILL TURN IT OVER TO THEM
FIRST, STARTING WITH SERGIO
AGUILAR-GAXIOLA.
SERGIO?
>> SERGIO: THANK YOU.
THANK YOU FOR VERY, I MEAN AN
EXCELLENT OVERVIEW OF THIS
CONCEPT.
IT IS A LITTLE BIT OUTSIDE OF
THE KIND OF THINGS THAT I DO,
BUT IT IS, I THINK THERE IS A
NEED FOR THIS KIND OF
TECHNOLOGY AND YOU IDENTIFIED
WHAT IS THE PROBLEM THAT NEEDS
TO BE SOLVED IN THE PROPOSED
SOLUTION AS WELL.
I'M MORE ON THE SIDE, HOW DO
YOU ACTIVELY LISTEN TO THOSE
WHO STAND TO BENEFIT FROM THIS
TECHNOLOGY FOR EXAMPLE.
HOW DO YOU ENGAGE WITH THEM IN
A MEANINGFUL MANNER?
I WOULD LOVE TO HAVE YOUR
THOUGHTS ON, YOU KNOW, HOW DO
YOU GO ABOUT ENGAGING,
ESPECIALLY THOSE WHO STAND TO
BENEFIT FROM THIS TECHNOLOGY.
>> GEETHA: THANK YOU, DR.
AGUILAR-GAXIOLA.
WE HAVE THOUGHT OF RAISING
AWARENESS OF BIOMEDICAL EXPERTS
OR QUANTUM EXPERTS BRINGING THE
COMMUNITIES TOGETHER TO BUILD A
MULTI-DISCIPLINARY
TEAMS,ACTUALLY.
WE HAVE BEEN DOING THIS THE
PAST COUPLE YEARS.
THE WORKSHOPS WERE A WAY TO
BUILD THESE COMMUNITIES AND NOW
WE ARE IN TOUCH WITH BOTH.
WE ARE PLANNING FOR THE NEXT
WORKSHOP WITH INDUSTRY
PARTNERS, AND WE HAVE BEEN IN
REGULAR COMMUNICATION WITH THE
FEDERAL AGENCIES WHO ARE
SUPPORTING THIS TECHNOLOGY AND
TRANSLATIONAL TECHNOLOGIES.
AND WE HAVE SCIENTIFIC INTEREST
GROUP AT NIH, THE A
PUBLIC-FACING GROUP, WHERE WE
CONDUCT MONTHLY SEMINARS
INVITING TECHNOLOGY DEVELOPERS,
AS WELL AS BIOMEDICAL
SCIENTISTS TO CONTINUE THIS
DIALOGUE AND EDUCATE BOTH
FIELDS TO EXCHANGE KNOWLEDGE
AND IDEAS, SO THERE IS THIS
ONGOING EFFORT WHERE WE SEE
CONTINUING FURTHER.
>> Joni Rutter: AND JUST TO ADD
ONTO THAT, YOU KNOW, I THINK TO
SERGIO'S POINT AS WELL IS, YOU
KNOW, THINKING ABOUT WHO IS
GOING TO BENEFIT FROM THE
TECHNOLOGY THAT GETS GENERATED.
YOU SHOWED THAT REALLY NICE
PHOTO OF THE MEG WITH BIG
MAGNET AND PERHAPS GETTING THAT
TO A POINT WHERE IT'S ACTUALLY
SOMETHING VERY SMALL THAT CAN
FIT ON SOMEONE'S HEAD AND TAKE
IMAGES OF THE BRAIN FOR
EXAMPLE, OR DETECT WAVES IN THE
BRAIN.
SO I THINK RIGHT NOW IT'S ABOUT
UNDERSTANDING WHERE OUR
OPPORTUNITIES ARE WITH
TECHNOLOGY AND WHERE THEY MAY
BENEFIT MOST FOR THE
COMMUNITIES WE SERVE.
I DON'T KNOW THAT WE CAN
PINPOINT THAT YET, EXACTLY.
I WILL SAY ONE OF THE
OPPORTUNITIES I DO SEE IS
CERTAINLY IN THE COMPUTING
SPACE IS THINKING ABOUT THE
DIAGNOSTIC ODYSSEY AND PERHAPS
USING ALGORITHM DEVELOPMENT TO
IDENTIFY WAYS TO APPROACH THOSE
MORE.
YOU THINK WE HAVE A LOT TO
LEARN BEFORE WE START TO ENGAGE
THE COMMUNITY SO WE CAN GO AND
HAVE A DEEPER UNDERSTANDING OF
HOW TO SPEAK ABOUT THE BENEFITS
THAT QUANTUM SENSING AND
QUANTUM COMPUTING CAN HAVE.
BUT I THINK YOUR POINT IS WELL
TAKEN AS WELL, WE NEED TO BE
THINKING ABOUT THOSE
COMMUNITIES TOO AND BEING
ENGAGED EARLIER ON.
I THINK THAT WILL ALSO HELP US
BE AT THE FOREFRONT OF DRIVE
ING HOW IMPORTANT IT IS TO
BRING QUANTUM SENSING AND
QUANTUM COMPUTING INTO THE
BIOMEDICAL SPACE.
ONE OF THE THINGS WE ARE TRYING
TO DO NOW IS ENSURE NIH AND THE
FOLKS AT THE NIH WORKING ON
THIS, LIKE GEETHA WHO IS
LEADING A LOT OF THESE EFFORTS
IS ENSURING THAT WE HAVE THE
ABILITY TO START LEADING THE
USE CASES FOR WHAT THESE TECH
NOLS ARE FOR AND THEN DRIVING
THAT TO HELP STRENGTHEN THE USE
OF THEM IN MORE WORLD KINDS OF
APPLICATION AND BRING IN THOSE
COMMUNITIES THAT WILL BENEFIT
THE MOST.
WE AREN'T QUITE SURE YET HOW TO
ANSWER YOUR QUESTION.
BUT I THINK IT'S AN IMPORTANT
ONE AND WE ARE CONTINUING TO
MONITOR IT AS WELL.
>> SERGIO: THAT'S A WONDERFUL
COMMENTS, JONI.
YOU REALLY APPRECIATE THAT.
BECAUSE AS I HAVE SEEN SINCE
THE TIME I HAVE BEEN IN THE
COUNCIL, YOU ARE LEADING, ALONG
WITH THE TEAM, EFFORTS TO
REALLY GROUND WHATEVER IS BEING
DONE WITH THE INPUT OF
LISTENING TO OTHERS.
AND ONE SUGGESTION I MIGHT
HAVE, DR. SENTHIL IS AS THIS
IS, YOU KNOW, AS YOU GET THE,
AS THIS GETS CLEARANCE, THAT
YOU ENDEAVOR TO, YOU KNOW,
THERE ARE SEVERAL METHODS TO
REALLY GATHER COMMENTS DIRECTLY
FROM COMMUNITIES, ESPECIALLY
THOSE THAT STAND TO BENEFIT.
THERE IS SUCH NOW CALLED
COMMUNITY ENGAGEMENT STUDENTS.
IT USE TO BE COMMUNITY REVIEW
BOARDS THAT REALLY GROUND THE
RESEARCHERS BY LISTENING TO
COMMUNITIES ON WHAT MATTERS TO
THEM AND THE INPUT.
AND WHAT I HAVE SEEN,
PERSONALLY, IS THAT IT HAS BEEN
OF TREMENDOUS HELP FOR THE
RESEARCHERS THEMSELVES.
BECAUSE THEY BRING ANGLES THAT
THEY WERE NOT CONTEMPLATING.
>> Joni Rutter: I THINK THAT'S
A BRILLIANT SUGGESTION.
AND WE WILL CERTAINLY FIGURE
OUT THE BEST TIMING FOR OUR
APPROACHES TO DOING THAT.
MAYBE THAT'S STARTING NOW, IT'S
NOT TOO EARLY, THINKING OF THE
PRIZE COMPETITION AS A MEANS TO
ENGAGE HOW WE CAN GET STARTED
IN THE SPACE WOULD BE GREAT.
I SEE DAN HAS HIS HAND UP.
DAN, DO YOU HAVE AN EXTRA
COMMENT TOO?
>> DAN: YOU ACTUALLY JUST
STATED IT, JONI.
I WAS JUST GOING TO STATE TO
SERGIO, THE USE OF THE PRIZE
COMPETITION IS TO CROWD SOURCE
POTENTIAL SOLUTIONS AND IN SOME
WAYS BROADEN THE INPUT WE ARE
TRYING TO GET, NOT ONLY FROM
THE TECHNOLOGY EXPERT, BUT ALSO
TO GET THE IDEAS FROM THE
PEOPLE WHO WILL BENEFIT FROM
THIS TECHNOLOGY.
SO THANK YOU FOR THOSE
THOUGHTFUL SUGGESTIONS AS WELL.
>> Joni Rutter: YEAH, AND MAYBE
THAT'S SOMETHING WE NEED TO
STATE IN THE COMPETITION
ITSELF, WE WOULD LIKE THE
SUBMITTERS TO INCLUDE
APPROACHES FOR ENGAGEMENT OF
THE COMMUNITY, LAY INDIVIDUALS,
FOR EXAMPLE, WHO MAY BENEFIT
FROM THIS TYPE OF WORK.
THAT'S SOMETHING WE CAN ASK
THEM TO WEIGH IN ON AS WELL.
>> SERGIO: WE WOULD LOVE THAT,
JONI, ONE MORE QUESTION, SORRY
TO BE TAKING TOO MUCH TIME.
THIS IS ALSO WHAT ALL OF YOU
STARTED.
THE LISTENING SESSIONS.
AND REALLY CONNECT WITH, IN A
MUCH MORE PURPOSEFULLY MANNER
WITH THOSE THAT STAND TO
BENEFIT THE MOST.
>> Joni Rutter: WONDERFUL,
THANK YOU SO MUCH.
WE HAVE ANOTHER DISCUSSANT
BEFORE WE OPEN IT UP TO
COMMENTS OR QUESTIONS.
ANNIE KENNEDY?
>> ANNIE KENNEDY: I THINK MY
COMMENTS ARE ALIGNED WITH
SERGIO.
I'M NOT THE EXPERT WHOSE GOING
TO COMMENT ON QUANTUM ENABLED
TECHNOLOGY FOR BIOMEDICAL
APPLICATION BUT I DO COMMENT
NCATS ON SEEING THE OPPORTUNITY
FOR THIS INNOVATION TO SOLVE
FOR SOME OF THE REAL BARRIERS.
AND I COMMEND FOR THE PRIZE
CHALLENGE TO BRING IN MAYBE NEW
STAKEHOLDERS WHO AREN'T ALREADY
ENGAGING WITH THE SOON TO BE
INSTITUTE.
BUT THE ONE THING I WILL MAYBE
UNDERSCORE THAT SERGIO ALREADY
RAISED IS THAT, YOU KNOW, AND I
THINK IT'S SOMETHING MATT SAID
IN HIS PRESENTATION EARLIER, I
DO ENCOURAGE WE THINK WITH THE
END-USER IN MIND AT THE
BEGINNING AS MUCH AS POSSIBLE.
WE CAN'T ALWAYS DO THAT.
BUT AS MUCH AS POSSIBLE WE
SHOULD.
WE SHOULD START HIGHER AND THEN
GO MORE GRANULAR, BUT MAYBE A
WAY OF GOING ABOUT THAT, HAVE
THE LIST OF THE TRANSNIH
INSTITUTE YOU ARE WORKING WITH.
AND MAYBE THERE'S AN
OPPORTUNITY AS PART OF THE
PRIZE SELECTION COMMITTEE,
WHETHER OR NOT THERE ARE
PATIENT REPRESENTATIVES ON THE
COUNCIL OF THOSE NIH
INSTITUTES, AND INCLUDING
SOMEBODY HERE FROM THIS
COUNCIL, MAYBE HAVE
REPRESENTATION FROM THOSE
PATIENT GROUPS AS PART OF THE
SELECTION.
SO AT LEAST YOU START WITH SOME
SEMBLANCE OF PATIENT
REPRESENTATION, AND THINK ABOUT
WHO IS THE PART OF VETTING SOME
OF THE APPLICANTS AND THEN
MAYBE GET MORE SPECIFIC WHEN
YOU THINK ABOUT THE TECHNOLOGY,
HAVING PEOPLE WHO ARE MAYBE A
LITTLE MORE SPECIFIC TO THOSE
POPULATIONS THAT YOU ARE
WORKING ON TECHNOLOGY FOR.
AND THEN THE ONE THING I WILL
SAY, THE IMAGES IN THE
PRESENTATION YOU PROVIDED
REALLY CALLED TO MIND IS JUST
TO KEEP IN MIND THAT ALL
PATIENT -- AREN'T CREATED
EQUAL.
I REALLY APPRECIATE IMAGE OF
TECHNOLOGY BEING REFINED FOR
SOMETHING MORE DEFINED.
THINK WHO WE ARE DESIGNING THE
TECHNOLOGY FOR.
GETTING TO BE PART OF THE
PROJECTS IN WEARABLE AND
ROBOTICS AND WE SPENT MILLIONS
OF DOLLARS DEVELOPING
TECHNOLOGY AND HAVE WONDERFUL
PATIENT REPRESENTATIVES BE PART
OF THIS PROJECT.
OFTEN TIMES THEY WERE
CAREGIVERS WHO WERE PART OF
THIS PROJECT.
AND DEVELOPED WEARABLE
TECHNOLOGY.
AND AT THE END OF THE DAY YOU
HAD A TEENAGER WHO WAS SUPPOSED
TO WEAR IT, AND LOOK AT IT AND
SAY THAT LOOKS LIKE AN ANKLET
FOR A PRISONER AND I'M NOT
GOING TO WEAR THAT IN HIGH
SCHOOL.
OR DEVELOP A ROBOTIC WEARABLE
SUIT THAT WAS MODIFIED FROM
SOMETHING THAT HAD BEEN
DEVELOPED IN THE MILITARY AND
THE SENSORS WERE JUST TOO HEAVY
FOR THE INDIVIDUAL WITH THE
MUSCLE DISEASE.
SO REALLY THINKING ABOUT WHO IS
THE SUB POPULATION WE ARE
SPECIFICALLY LOOKING FOR AND OF
COURSE FURTHER DOWN, MAYBE
THINKING ABOUT YOUR PATIENT
REPRESENTATION MORE BROAD AND
THEN GETTING MORE SPECIFIC.
>> Joni Rutter: THESE ARE
FANTASTIC COMMENTS.
I THINK WE CAN CERTAINLY WORK
TOGETHER TO MAKE SURE WE
INCORPORATE THEM, IF THIS DOES
PROCEED FORWARD.
I THINK IT'S BY DESIGN WE HAVE
BOTH SERGIO AND ANNIE PROVIDING
THESE COMMENTS TO US ON
SOMETHING THAT IS SO DEEPLY,
HIGHLY TECHNICAL.
I THINK THAT'S REALLY THE ANGLE
WE NEED TO MAKE SURE WE ARE
DESIGNING FOR, FROM THE VERY
BEGINNING WHEN WE START THIS.
I THINK WE ARE PROBABLY NOT
GOING TO SEE THE FRUITS OF MUCH
OF THIS TECHNOLOGY FOR ANOTHER
5-10 YEARS BUT THINKING ABOUT
THAT NOW IS CRITICAL.
I'M TAKING NOTES ON YOUR
COMMENTS AND WE WILL BE SURE TO
INCORPORATE THOSE.
I WOULD LIKE TO OPEN IT UP FOR
ANY OTHER DISCUSSION FROM OUR
COUNCILMEMBERS.
I DON'T KNOW IF ANYBODY HAS
ANYTHING.
PAUL, THANK YOU.
>> PAUL: GREAT PRESENTATION,
THANK YOU SO MUCH.
CAN YOU TALK A LITTLE MORE
ABOUT YOUR EXPECTATIONS ON THE
MECHANICS OF THE CHALLENGE.
IS THERE GOING TO BE ONE, TWO.
>> THERE WILL BE TWO
CHALLENGES, ONE FOR COMPUTING
AND ONE FOR SENSING.
THEY WILL HAVE TWO STAGES.
IN THE IDEATION PHASE WE WILL
GRADUATE THE PROPOSAL.
ASKING THE TEAM THAT ALREADY
HAS THE PROTOTYPE TECHNOLOGY IN
HAND TO COME FORWARD.
IT SHOULD HAVE ALREADY SOME
TECHNICAL FEASIBILITY TO BE
APPLIED IN A BIOMEDICAL CONTEXT.
TO HOW IT WORKS IN A NOVEL
BIOMEDICAL TRANSLATIONAL
CONTEXT.
SO THE QUANTUM TECHNOLOGY
EXPERTS ARE EXPECTED TO
COLLABORATE WITH SOMEONE FROM
BIOMEDICAL OR CLINICAL SCIENCES.
AND TO ANNIE'S POINT, HAVING
THE END GOAL IN MIND WHEN ARE
OPTIMIZING ENGINEERING
TECHNOLOGY TOOL WHO WANT TO
HAVE THE AUDIENCE.
>> PAUL: THAT'S THE IDEATION.
>> GEETHA: WE WILL HAVE THESE
REVIEWERS AND THE SELECTED ONES
WILL BE ALLOWED TO GO FORWARD
TO IMPLEMENTATION.
MILESTONE DRIVEN, THEY HAVE TO
DEMONSTRATE IF THEY ARE MAKING
PROGRESS ON WHAT THEY PROPOSE
AND WILL GET A SMALL REWARD IF
THEY SHOW PROGRESS.
INITIAL DEMONSTRATION OF THE
APPROPRIATE TYPE TECHNOLOGY.
IF THEY COULD BE THESE
PROTOTYPE DEVICES COULD BE FROM
DISCIPLINES LIKE NASA FOR
EXAMPLE, THEY HAVE A PROTOTYPE
TECHNOLOGY, COULD BE OPTIMIZED
TO MAKE THEM BIO MECHANICAL.
>> SORRY, DO YOU HAVE TO WIN
IDEATION?
>> GEETHA: YES.
THE PUBLIC TOO WILL BE ALLOWED
TO GO FORWARD TO THE
IMPLEMENTATION PHASE AND AS
THEY ACHIEVE MORE MILESTONES
THEY GET REWARD.
ONCE THEY ACCOMPLISH THE
MILESTONE, AT THE END, WHOEVER
DELIVERS WORKING TECHNOLOGY
THEY WILL BE THE WINNER.
AND THERE WILL BE A RUNNER-UP.
>> I MAY HAVE MISSED IT BUT
WHAT IS THE FUNDING LEVEL, OR
COULD YOU SPEAK TO THE FUNDING
LEVEL?
>> GEETHA: WE NEED TO APPROVE
THE CONCEPT FIRST AND THEN WE
WILL FIGURE THAT OUT.
>> THE REASON I'M WONDERING
THAT, I WONDER IF THAT
IMPLEMENTATION IS A CHALLENGE
OR IT'S OVER TO THE SPI, SCPR.
>> GEETHA: WE ARE THINKING OF
REDUCTION TO PRACTICE AND OTHER
FUNDING OPPORTUNITY.
OTHER FUNDING MECHANISM.
THE CONCEPT IS FOR PRIZE
CHALLENGES AND DEPENDING ON HOW
THE SOLUTION IS COMING AND
WHAT'S A SUITABLE WAY OF
IMPLEMENTING FORWARD AND WITH
PERMISSION FROM OUR DEA, WE
WILL EXPLORE THOSE.
>> Joni Rutter: THANK YOU VERY
MUCH.
ANY OTHER QUESTIONS?
AND KELLY, I SEE YOUR COMMENT
ONLINE.
THAT WOULD BE GREAT TO MAKE
CONNECTIONS FOR SURE.
IF HAVE ANY OTHER COMMENTS I'M
HAPPY TO TAKE THOSE TOO.
>> KELLY: THIS IS GREAT ARE
TACKLING THIS.
I DON'T REALLY HAVE ANY
QUESTIONS, I DON'T HAVE ENOUGH
DEPTH WITH QUANTUM COMPUTING.
I GUESS I HAD QUESTIONS ABOUT,
IT SOUNDS LIKE YOU ARE NOT, ONE
QUESTION IS WERE YOU BUYING A
QUANTUM COMPUTER?
BUT IT SOUNDS LIKE YOU ARE
COLLABORATING WITH OTHER
INSTITUTES.
>> Joni Rutter: THAT'S RIGHT.
>> KELLY: THERE'S ONE COMMENT I
DON'T KNOW IF THIS WOULD SHAPE
IN ANY WAY THE COMPETITION.
AGAIN, HAVING NOT DONE THIS
RESEARCH OR USE THESE
TECHNOLOGIES PERSONALLY, BUT
HAVING KIND OF COLLABORATED
WITH THOSE WHO HAVE BEEN VERY
EXCITED ABOUT IT.
ONE THING THAT WAS REALLY
STRIKING TO ME, IS THAT THE
QUANTUM COMPUTERS ARE JUST NOT
CREATED EQUALLY.
THERE WAS A GROUP PART OF THIS
NATIONAL SCIENCE FOUNDATION
CONSORTIUM FOR ACCELERATED
COMPUTING HAD THESE TEAMS OF
PEOPLE WORKING ON ALGORITHMS AN
STARTED DOING BASIC TESTING ON
DIFFERENT QUANTUM PARADIGMS.
SO I THINK LIKE THE NAMES I
REMEMBER ARE D-WAVE OR REGETTI
QUANTUM COMPUTING.
BUT IT WAS INTERESTING, THERE
WASN'T A LOT OF -- THEY WERE
HAVING PROBLEMS WITH THE
ALGORITHMS TRANSFERRING FROM
ONE QUANTUM COMPUTING
ENVIRONMENT AND TECH NOOLG
STACK TOGETHER, I DON'T KNOW IF
THAT IS IMPORTANT OR RELEVANT
TO YOUR CHALLENGE.
BUT IT WAS MAKING THE
SCIENTISTS LEADING REALLY
SCRATCHING THEIR HEADS.
>> GEETHA: I'M HAPPY TO TAKE
THAT QUESTION.
AS ITS QUANTUM COMPUTING
HARDWARE IS EVOLVE, AND THERE
ARE MULTIPLE HARDWARES OUT
THERE.
AND SEVERAL DIFFERENT
ALGORITHMIC COMPANIES.
WHAT WE ARE ASKING IN THE PRIZE
CHALLENGE IS THINKING OF A
PROBLEM.
CHOOSING THE RIGHT PROBLEM IS
AMUSING.
THAT IS QUANTUM AMENABLE.
THE RIGHT HARDWARE THAT WOULD
BEAL GROW RHYTHM DESIGN TO
SOLVE THAT PROBLEM.
AND SURE, HOW YOU CAN SIMULATE
IF THAT WORKS ON ITS SIMULATOR
AND TAKE IT TO HARDWARE.
THEY CAN TEST IT ON DIFFERENT
HARDWARE.
MOST ALGORITHM DEVELOPERS THEY
ARE FAMILIAR WITH DIFFERENT
HARDWARE, WHAT I LEARNED FROM
COMMUNITIES THEY CAN ADAPT THE
ALGORITHMS TO DIFFERENT
HARDWARE.
BUT THE MORE MATURE ONES ARE
SUPER CONDUCTING QUBITS.
THE DEVELOPERS ARE VERY
FAMILIAR WITH THE PROBLEM.
I MUST SAY I HAVE TO REITERATE
THIS EARLY STAGE FOR QUANTUM
COMPUTING, WE WANT BIOMEDICAL
SCIENTISTS TO GET ENGAGED WITH
THINKING ABOUT HOW TO SOLVE A
PROBLEM IN QUANTUM ALGORITHM
LANGUAGE RIGHT, CODE, SOLVE AND
APPLY THAT YOU CAN ENHANCE THE
CURRENT LANDSCAPE.
THERE ARE CERTAIN ADVANTAGES
WITHIN NOISY INTERMEDIATE
SCALE, QUANTUM COMPUTERS.
CERTAIN PROBLEMS BEING TESTED.
>> Joni Rutter: THANK YOU VERY
MUCH.
IF THERE AREN'T ANY OTHER
COMMENTS, ANNE?
>> OKAY, COULD I HAVE A MOTION
TO APPROVE THE CONCEPT?
SECOND?
ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
THANK YOU.
THE CONCEPT IS APPROVED.
>> Joni Rutter: NEXT WE HAVE
ANOTHER CONCEPT FROM OFFICE OF
POLICY, COMMUNICATIONS AND
EDUCATION.
PENNY BURGOON WILL KICK US OFF
INTRODUCING.
>> PENNY: GOOD AFTERNOON.
I'M HERE TO GIVE A BACKGROUND
AND BRIEF INTRODUCTION ON THE
OFFICE OF THIS CONCEPT FROM THE
OFFICE OF POLICY COMMUNICATIONS
AND EDUCATION.
NEXT SLIDE, PLEASE.
THIS IS A CONCEPT FROM THE
EDUCATION BRANCH.
THIS BRANCH IS REALLY WORKING
TO DEVELOP A VISION FOR A
HIGHLY-SKILLED LARGE DIVERSE
TRANSLATIONAL SCIENCE WORKFORCE.
ALONG WITH THE COMPASS NEEDED
TO ACCELERATE PROGRESS ALONG
THE TRANSLATIONAL PIPELINE WITH
MORE HEALTH SOLUTIONS FOR ALL
PEOPLE.
BY DR. AMANDA VOGEL.
DR. BELEN HURLE, DR. HODGES AND
DR. JUAREZ.
THE WORK IS EXTENSIVE FOR
PEOPLE INCLUDING COLLABORATING
WITH THOSE ACROSS OUR
INTERMURAL ACTIVITIES TO
ADVANCE UNDERSTANDING.
THEY ALSO PRODUCE ORIGINAL
SCHOLARSHIP.
SO WITH THAT, I'M GOING TO --
NEXT SLIDE, PLEASE.
THESE ARE JUST SOME OF THE
EDUCATIONAL BRANCH GOALS, BUT
IN THE INTEREST OF TIME, I'M
GOING TO LET AMANDA VOGEL SO
SHE CAN TELL YOU ABOUT THE
CONCEPT TODAY FOR
TRANSFORMATIONAL EDUCATION AND
TRAINING.
>> AMANDA: THANK YOU, I'M
AMANDA VOGEL, ACTING DIRECTOR
FOR THE EDUCATION BRANCH.
ONE OF THE CORE GOALS IS TO
COLLABORATE, DISSEMINATE
KNOWLEDGE AND AMPLIFY THAT
KNOWLEDGE THAT COMES THROUGH
THE SCIENTIFIC COMMUNITY.
THAT CONCEPT FOR A CHALLENGE
PRIZE IS ALIGNED WITH THAT GOAL.
NEXT SLIDE, PLEASE.
SO IT SEEMS LIKE CHALLENGE
PRIZES ARE THE THEME OF THE DAY.
AND THIS PRIZE COMPETITION THAT
I'M SHARING WITH YOU TODAY, AS
A CONCEPT IS FOR TRANSLATIONAL
SCIENCE EDUCATION AND TRAINING.
THE GOAL OF THIS CONCEPT IS TO
USE THIS MECHANISM AS AN AVENUE
TO IDENTIFY, AMPLIFY AND
DISSEMINATE TRAINING THAT HAVE
BEEN GENERATED BY THE COMMUNITY.
NEXT SLIDE, PLEASE.
I WANT TO START BY GIVING YOU A
LITTLE BIT BACKGROUND TO THIS
CONCEPT.
SO NCATS HAS ARTICULATED A
VISION FOR THE FIELD OF TS
EDUCATION WAND TRAINING TO
EQUIP THE WORKFORCE WITH
KNOWLEDGE AND SKILLS THAT ARE
SPECIFIC TO IMPROVING AND
ACCELERATING TRANSLATIONAL
RESEARCH AND TRANSLATIONAL
PROGRESS.
AND THIS IS IN ORDER TO ENHANCE
BOTH THE SPEED AND IMPACT OF
OUR WORK.
THIS VISION IS REFLECTED IN THE
TRANSLATIONAL SCIENCE
PRINCIPLES SHOWN ON THE RIGHT
SIDE OF THESE SLIDE, THESE
PRINCIPLES EXEMPLIFY EFFECTIVE
SCIENTIFIC AND OPERATIONAL
APPROACHES TO ADVANCE
TRANSLATIONAL PROGRESS.
SOME EXAMPLES OF PRINCIPLES AS
SHOWN ON THE RIGHT ARE
EMPHASIZING CREATIVITY AND
INNOVATION.
FOCUSING ON UNMET NEEDS,
ENHANCING EFFICIENCY AND SPEED
AND LEVERAGING CROSS
DISCIPLINARY TEAM-BASED SCIENCE
AND BOUNDARY CROSSING
PARTNERSHIPS INCLUDING
PARTNERSHIPS ACROSS GOVERNMENT
UNIVERSITIES AND INDUSTRY AND
WITH COMMUNITIES AND PATIENT
PARTNERS AND RESEARCH.
ANOTHER KEY PART OF OUR VISION
FOR TRANSLATIONAL SCIENCE
EDUCATION AND TRAINING IS TO
EXPAND ACCESS TO TRANSLATIONAL
SCIENCE KNOWLEDGE FOR LEARNING
ACROSS TRAINING AND CAREER
STAGES.
IN A VARIETY OF ROLES, IT
INCLUDES COMMUNITY AND PATIENT
PARTNERS AS I MENTIONED.
WE SHARED SOME RELATED
MATERIALS TODAY IN A FOLDER
THAT WAS DISTRIBUTED TO
COUNCILMEMBERS WHO ARE HERE IN
PERSON.
AND THESE INCLUDE AN ARTICLE
THAT SHARES OUR VISION FOR
TRANSLATIONAL SCIENCE,
EDUCATION AND TRAINING AND SOME
FUN STICKERS THAT FEATURE THE
TRANSLATIONAL SCIENCE
PRINCIPLES.
FOR THOSE OF US JOINING ONLINE,
YOU CAN FIND THE SAME MATERIALS
AS YOU CAN GO TO THE NCATS WEB
PAGE AND SEARCH FOR
TRANSLATIONAL SCIENCE
PRINCIPLES.
AT THE BOTTOM OF THE PAGE YOU
WILL FIND A LINK TO OUR ARTICLE
THAT IS INCLUDED IN THE FOLDER
FOR THOSE HERE IN PERSON.
SO THE SECOND POINT THAT I
WOULD LIKE TO MAKE ON THIS
SLIDE IS AS A FIELD
TRANSLATIONAL SCIENCE,
EDUCATION AND TRAINING IS STILL
VERY MUCH IN DEVELOPMENT.
AND THERE ARE TRAIL BLAZERS IN
THE COMMUNITY GENERATING NOVEL
EDUCATION AND TRAINING
OPPORTUNITIES WHERE THEY ARE
LAYING OUT CORE CONTENT IN
TRANSLATIONAL SCIENCE AND
EDUCATION AND TRAINING AND
DEVELOPING METHODS TO CONVEY
THAT CONTENT TO VARIED
AUDIENCES.
TIES ARE TRAIL BLAZERS OF
COURSE IN ACADEMIA, FOR
EXAMPLE, OUR CTSA SUPPORTED
INSTITUTIONS AND ALSO TRAIL
BLAZERS IN OTHER SETTINGS, FOR
EX ESPECIALLY, INDUSTRY AND
PROFESSIONAL SOCIETIES.
WHAT THEY ARE DEVELOPING IN
THESE SETTINGS MAY BE RELEVANT
TO OTHERS IN THE SAME SETTINGS
BUT ALSO BROADLY RELEVANT
ACROSS SETTINGS.
BUT THOSE APPROACHES TO
EDUCATION AND TRAINING MAY NOT
HAVE BEEN SHARED BROADLY.
SO AS EVIDENCE OF THIS THE
EDUCATION BRANCH CONDUCTED A
SCHOLARSHIPING REVIEW OF THE
PEER REVIEW LITERATURE ON
TRANSLATIONAL SCIENCE EDUCATION
AND TRAINING.
WHILE LOOKING AT A 15-YEAR
PERIOD WE FOUND ONLY 29
ARTICLES THAT DESCRIBE
EDUCATIONAL AND TRAINING
OPPORTUNITIES THAT REFLECT THE
NCATS UNDERSTANDING OF
TRANSLATIONAL SCIENCE.
WE KNOW THERE THERE IS MORE OUT
THERE.
TO SUM UP THIS SLIDE WE FEEL
THERE'S A NEED TO IDENTIFY,
AMPLIFY AND DISSEMINATE
EXEMPLARY MODELS FROM THE
SCIENTIFIC COMMUNITY AND PLAN
TO USE OUR ROLE AT NCATS TO DO
THAT WITH THIS CHALLENGE.
NEXT SLIDE, PLEASE.
YOU SEE IN A VERSION THIS SLIDE
AND DOMINIQUE'S PRESENTATION
THIS MORNING, I THINK THAT
UNDERSCORES ACCENT RATE AREAS.
ACCELERATE AREAS.
THEY OVERCOME LONG STANDING
CHALLENGES ALONG THE
STRANSLATIONAL RESEARCH
PIPELINE AND THOSE INNOVATIONS
AND ADVANCED PROGRESS BY BOTH
ACCELERATING THE SPEED AND OUR
RESEARCH AND ENHANCING ITS
IMPACT AND THIS SLIDE HERE ARE
SOME EXAMPLES OF TRANSLATIONAL
RESEARCH KHAL LINGS AND RELATED
TRANSLATIONAL SCIENCE
SOLUTIONS, THESE ARE EXAMPLES
THAT WE HOPE TO EQUIP OUR
WORKFORCE WITH AND THE FIELD
WILL BIN FIT FROM.
SO THE FIRST BULLET POINT UNDER
BOTTLENECKS TO ADDRESS THE
CHALLENGE OF INCOMPATIBLE
DATABASES, WE INVESTED IN DATA
INTEROPERABILITY AND
INTEGRATION AND EXAMPLE
PROVIDED HERE TO ADDRESS THE
CHALLENGE OF ADMINISTRATIVE
BURDEN FOR STUDY START UP.
WE FOCUS ON STREAMLINING
BUSINESS PROCESSES SUCH AS THE
SMART IRP AND.
WE FOCUS ON DEVELOPMENT OF
MODEL THAT'S MIMIC THE
STRUCTURE AND FUNCTION OF HUMAN
TISSUE AS THROUGH THE TISSUE
CHIP INITIATIVE.
SO TRANSLATIONAL SCIENCE,
EDUCATION AND TRAINING AIMS TO
EQUIP OUR WORKFORCE FOR THESE
INITIATIVES.
NEXT SLIDE, PLEASE.
WE ARE PROPOSING THIS
TRANSLATIONAL SLIDE TO RAISE
AWARENESS WHAT IT CAN DO AND
THE VALUE IT COULD ADD TO OUR
WORKFORCE PREPARATION AND
ACCELERATE DISSEMINATION OF
EXEMPLARY MODELS INCLUDING CORE
CONTENT FOR EDUCATION TRAINING.
INNOVATIVE TEACHING APPROACHES
AND MODELS OF WAYS OF REACHING
DIVERSE LEARNER DOLLARS.
NEXT SLIDE, PLEASE.
-- LEARNERS.
THIS SLIDE WILL RISE, REWARD
AND DISSEMINATE TRANSLATIONAL
SCIENCE EDUCATION AND TRAINING
PROGRAMS THAT PROVIDE CONTENT
THAT ALIGNS WITH THE NCATS
UNDERSTANDING OF THE FIELD OF
TRANSLATIONAL SCIENCE.
EQUIP LEARNERS, KNOWLEDGE AND
SKILLS TO OVERCOME BOTTLENECKS
AND ROADBLOCK THAT'S ARE
COMMONLY FOUND IN RESEARCH
ACROSS DISEASES AND CONDITIONS.
AND LEVERAGE INNOVATIVE
EDUCATION AND TRAINING MODALITY
TEACHING APPROACHES AND
FORMATS, THAT HELP REACH BROAD
AND DIVERSE AUDIENCES.
NOW I WANT TO TALK ABOUT WHY WE
ARE PROPOSING A CHALLENGE PRIZE
VERSUS A GRANT.
THERE ARE TWO ADVANTAGES OF A
CHALLENGE PRIZE.
FIRST OF ALL, IT RECOGNIZES
WORK THAT HAS ALREADY BEEN DONE.
THIS CHALLENGE WILL REWARD AND
AMPLIFY ADDITIONAL
OPPORTUNITIES.
IT ALLOWS US TO BEAT THE BUSHES
FOR WHAT IS ELEVATED AND
DISSEMINATED.
THE PRIZE WILL BE BROADLY
INCLUSIVE A WIDE RANGE OF
MODALITIES, IN DICTION TO
COURSES, CERTIFICATES DEGREE
PROGRAMS WE ARE THINKING MORE
BROADLY TO CAPTURE MODALITIES
THAT CAN GET TO PEOPLE ACROSS
TRAINING AND CAREER STAGES, SO
FOR EXAMPLE, WORKSHOPS,
CONSULTING SEMINAR SERIES ET
CETERA.
IT EXPANDS THE APPLICANT POOL
TO CAPTURE DIVERSE APPROACHES.
THE IDEA BEHIND THE CHALLENGES
IS THAT THEY ARE AN OPEN
INNOVATION INITIATIVE.
THE IDEA IS THAT YOU LEVERAGE
THE PUBLIC TO SIMULATE
INNOVATION.
THAT YOU INCLUDES ACADEMIA,
INDUSTRY, PROFESSIONAL
SOCIETIES, ET CETERA.
WE THINK DOING THIS WE WILL GET
SOME CROSS FERTILIZATION OF
IDEAS AND WE WILL GENERATE,
IDENTIFY IDEAS THAT HAVEN'T
BEEN PUBLISHED FOR EXAMPLE, IN
THE PEER REVIEW LITERATURE.
INCLUDING UNDERGRAD AND
GRADUATE EDUCATION AND EARLY,
MID AND SENIOR CAREER.
OUR INTENTION IS WE WILL BE --
I WANT TO TALK ABOUT HOW THIS
CHALLENGE CONCEPT FITS INTO OUR
OTHER EFFORTS AND AS WE EXPECT
TO SEE AS THE IMPACT.
WE BELIEVE THIS WILL CONTRIBUTE
TO OUR WORK IN DEVELOPING
TRANSLATIONAL SCIENCES OF FIELD
OF STUDY.
IT WILL DO THIS BY
COMPLEMENTING EXISTING
ACTIVITIES AND RESOURCES AT
NCATS.
INCLUDES EXTRAMURAL FUNDING FOR
TRANSLATIONAL SCIENCE EDUCATION
AND TRAINING.
AS WELL AS ONLINE COURSES THAT
WE HAVE DEVELOPED THROUGH THE
EDUCATION BRANCH.
THAT WE ARE MAKING AVAILABLE TO
THE PUBLIC IN THE FUTURE.
AND OUR ORIGINAL SCHOLARSHIP
FROM THE GRANT THAT'S
CONTRIBUTING TO THE NATIONAL
CONVERSATION ON DIRECTIONS FOR
ADVANCING TRANSLATIONAL SCIENCE
EDUCATION AND TRAINING.
ENHANCING ACCESSIBILITY.
FINALLY OUR INTEND IN THIS
PRIZE WILL IDENTIFY, AMPLIFY
EXEMPLARS OF TRANSLATIONAL
SCIENCE EDUCATION THAT MAY NOT
HAVE BEEN SHARED BROADLY.
NEXT SLIDE, PLEASE.
THE CONCEPT AIMS TO RAISE
AWARENESS OF THE VALUE ADD OF
THE TRANSLATIONAL SCIENCE
EDUCATION AND TRAINING THAT
ALIGNS WITH THE NCATS DIVISION.
AND ULTIMATELY WE HOPE IT WILL
CONTRIBUTE TO ENHANCING THE
QUALITY OF TRANSLATIONAL
SCIENCE AND TO INCREASING
ACCESS TO THOSE OPPORTUNITIES.
THIS WILL HELP EQUIP MORE
MEMBERS OF THE COMMUNITY WITH
THE SKILLS AND KNOWLEDGE NEEDED
TO HELP ACCELERATE PROGRESS
ALONG THE TRANSLATIONAL
PIPELINE.
NEXT SLIDE, PLEASE.
WITH THAT I WOULD LIKE TO OPEN
IT UP TO YOUR COMMENTS.
WE ARE VERY INTERESTED TO HEAR
YOUR GENERAL COMMENTS ON
POTENTIAL WAYS TO IMPROVE THE
CONCEPT AND OTHER KEY POINTS TO
CONSIDER.
AND WE'RE SPECIFICALLY
INTERESTED IN HOW YOUR IDEAS
FOR HOW WE CAN EFFECTIVELY
OUTREACH COLLEAGUES ACROSS
SECTORS TO SOLICIT APPLICATION.
>> Joni Rutter: THANK YOU VERY
MUCH.
WE HAVE TWO DISCUSSANTS, SERGIO
AGUILAR-GAXIOLA, PLEASE GO
AHEAD, YOU ARE FIRST AGAIN.
>> SERGIO: THANK YOU, JONI AND
THANK YOU, MS. VOGEL FOR THIS
EXCELLENT PRESENTATION.
YOU KNOW, YOU HAVE SO MUCH
INFORMATION THAT RESONATES WITH
ME, IT'S ATTRACTIVE.
THAT I WISH TO HAVE TIME TO
REALLY LOOK AT YOUR SLIDES MORE
CAREFULLY.
BUT I WILL COMMENT ON SOMETHING
THAT, THERE ARE THREE SLIDES I
WAS LOOK AT VERY CAREFULLY OF
WHAT YOU PRESENTED THAT TRIGGER
MY COMMENT, MY MOST IMPORTANT
COMMENT.
AND THAT IS THAT IN ONE OF YOUR
SLIDES, THE ONE OF THE GOALS OF
THE EDUCATION BRANCH, YOU HAVE
THE LAST OF SEVERAL STAGES IN A
PROCESS, CONVENE, COLLABORATE,
DISSEMINATE AND AMPLIFY
COMMUNITY EXPERTISE.
AND THEN, YOU ALSO HAVE ON THE
OBJECTIVES AND AREAS OF
EMPHASIS ON THE CONCEPT THAT
YOU ARE SHARING WITH US, WITH
SEVERAL OBJECTIVES FOR RELATED
TO RECOGNIZE REWARD AND
DISSEMINATE.
AND THEN FINALLY, YOU HAVE IN
THE IMPLEMENTATION AND EXPECTED
IMPACT.
LET ME TELL YOU THAT, WHAT I'M
RESPONDING TO IS THAT THERE IS
A STRONG SENSE ON WHAT YOU ARE
PRESENTED THAT EDUCATION IS
UNIDIRECTIONAL.
THAT YOU GO AND ALLOCATE
COMMUNITIES.
WHEN IN FACT, COMMUNITIES CAN
EDUCATE US AS WELL.
AND MAYBE THAT IS NOT WHAT YOU
WERE INTENDING TO PRESENT.
BUT I GOT THAT IMPRESSION.
MAYBE I'M MISTAKEN.
AND I WOULD LOVE FOR YOU TO
POINT, IF I'M IN ERROR.
BUT YOU KNOW, JUST TO GIVE YOU
A SENSE.
THE AAMC THREE YEARS AGO HAD
BEEN DEVELOPING AND PERFECTING
THED TEN PRINCIPLES OF
COMMUNITY ENGAGEMENT, EXCUSE
ME, TEN PRINCIPLES OF
TRUSTWORTHINESS.
THE FIRST ONE IS THE
COMMUNITIES ARE ALREADY
EDUCATE, THAT'S WHY THEY DON'T
TRUST YOU, OKAY.
AND WE STILL, EVEN WITH THIS
INCREDIBLE PROGRESS THAT WE
HAVE DONE ON TRANSLATIONAL
SCIENCE, AND TRANSLATIONAL
RESEARCH, WE ARE STILL, FROM MY
PERSPECTIVE, NOT MADE ENOUGH
PROGRESS TO REALLY CONSIDER,
THAT WHEN WE GO AND ENGAGE
COMMUNITIES, EMPHASIZING THE
POINT THAT I MADE AT THE
BEGINNING IN ONE OF YOUR
SLIDES, AND THAT IS THE SLIDE
ON CONVENE, COLLABORATE,
DISSEMINATE AND AMPLIFY
COMMUNITY EXPERTISE.
IS THAT WE NEED TO BE, FROM MY
OPINION, MORE HUMBLE.
AND REALLY, AND MAKE IT
BIDIRECTIONAL OR
MULTI-DIRECTIONAL COMMUNICATION.
I'M GOING TO STOP THERE.
YOU HAVE DONE IT ALREADY, NCATS.
YOU LEAD THE FOR THE ABOUT
LISTENING SESSIONS AND I WAS
VERY IMPRESSED WITH THE
INFORMATION THAT WAS COLLECTED
THROUGH THOSE LISTENING
SESSIONS.
I THINK THAT IN THIS CONCEPT
LISTENING ACTIVELY AND
ATTENTIVELY IS IMPORTANT TO BE
INCLUDED.
I'M GOING TO STOP HERE.
>> THANK YOU, FIRST IT WASN'T
MY INTENTION TO SUGGEST A
LINEAR PROCESS WITH THAT SLIDE
WITH THE ARROWS.
AND ALSO I REALIZED THAT IN USE
OF THE WORD COMMUNITY I WASN'T
CLEAR.
WE ARE THINKING OF COMMUNITY
BROADLY TO INCLUDE ANYONE
OUTSIDE OF GOVERNMENT WHOSE
DEVELOPED AN EDUCATION OR
TRAINING OPPORTUNITY IN
TRANSLATIONAL SCIENCE.
AND IN OPENING UP COLLEGE,
CREATING A CHALLENGE RATHER
THAN A GRANT, WE OPEN UP THE
OPPORTUNITY FOR THOSE OUTSIDE
ACADEMIA TO SUBMIT AN
APPLICATION TO BE RECOGNIZED
FOR THEIR TRAINING OR EDUCATION
OPPORTUNITY.
SO IN FACT, ONE OF THE IDEAS WE
HAD IN MIND IN DOING THAT, IT
WOULD OPEN IT UP TO EXPERTISE
OUTSIDE OF ACADEMIA.
I WOULD IMAGINE FOR EXAMPLE,
ALSO GOING ALONG WITH OUR THEME
OF EXPANDING ACCESS, I COULD
IMAGINE, FOR EXAMPLE, SOMEONE
APPLYING WITH AN OPPORTUNITY TO
ENGAGES COMMUNITY MEMBERS
PARTNERS IN RESEARCH.
PERHAPS YOU ARE POINTING TO A
LACK CLARITY IN COMMUNICATION,
WHICH WILL BE REALLY VALUABLE
TO US AS WE DEVELOP THIS
FURTHER.
I WOULD ALSO BE VERY INTERESTED
IN YOUR FEEDBACK AND
RECOMMENDATIONS AROUND HOW TO
ENGAGE WITH A BROADER RANGE OF
STAKEHOLDERS SO WE ARE MAKING
THEM AWARE OF THIS OPPORTUNITY.
BECAUSE IN FACT, YOUR
PRIORITIES THAT YOU HAVE
REFLECTED IN YOUR COMMENTS, I
THINK ARE 100% ALIGNED WITH
OURS, IN TERMS OF BROADENING
ENGAGEMENT AND USING THIS
MECHANISM TO BROADEN ENGAGEMENT
WITH PARTNERS IN RESEARCH WHO
HAVE UNIQUE EXPERTISE, THAT IS
NOT REFLECTED FOR EXAMPLE IN
THAT LITERATURE REVIEW, RIGHT?
SO THE POINT HERE IS TO
COMPLIMENT THAT SORT OF REVIEW
THAT SUMMARIZES WHAT IS IN THE
LITERATURE WITH OTHER
MECHANISMS THAT IDENTIFY
COMMUNITY EXPERTISE AND
DISSEMINATE IT.
>> SERGIO: THANK YOU.
CAN I CALL YOU AMANDA?
>> AMANDA VOGEL: YES.
>> SERGIO: THANK YOU.
I APPRECIATE YOUR COMMENTS
TRULY, AND I WOULD BE MORE THAN
HAPPY TO SPEAK WITH YOU,
BECAUSE I HAVE SEVERAL IDEAS ON
HOW YOU CAN GO ABOUT DOING WHAT
YOU JUST SHARED WITH US THE
THAT ARE MAJOR DEVELOPMENTS ON
MEANINGFUL COMMUNITY ENGAGEMENT.
LANDMARK WORK THAT HAVE, YOU
KNOW, THE METHODOLOGY HOW WE
CAN GO ABOUT DOING THAT
PRECISELY.
AND NOT ONLY THAT, TO MEASURE
THE PROCESS OF ENGAGING
COMMUNITIES IN MEANINGFUL WAYS.
AMANDA, PLEASE, REACH OUT, OR I
CAN REACH OUT.
JONI, I WOULD LOVE TO HAVE THAT
OPPORTUNITY WITH AMANDA AS WELL.
>> Joni Rutter: THANK YOU, SO
MUCH.
THAT SOUNDS TERRIFIC AND A
GREAT OPPORTUNITY TO ALSO HAVE
YOU KIND OF HELP BE AN
AMBASSADOR AND GET THE WORD OUT
AS WELL IF THIS PROJECT
INITIATIVE MOVES FORWARD.
WE HAVE ONE MORE DISCUSSANT,
PAUL HARRIS, GO AHEAD.
>> GREAT PRESENTATION AND A
THOUGHTFUL APPROACH.
AS I WAS READ THIS, AND
LISTENING TO YOU, I THINK IT
WOULD BE WISE TO FOCUS ON THE
DISSEMINATION PIECE OF THIS.
YOU HAVE IT THERE IN YOUR LAST
BULLET.
I WOULD FRIP THAT QUESTION
AROUND, MAYBE THE CHALLENGE,
SINCE YOU ARE LOOKING FOR
THINGS ALREADY DEVELOPED, MAYBE
YOU TELL THE REVIEWERS WHERE
THIS FILLS A GAP THAT IS NOT,
WHERE THIS FILLS A GAP AT THIS
POINT.
AND THE CHALLENGE IS HOW DO WE
TAKE WHAT, HOW DO YOU TAKE WHAT
YOU HAVE, AND DISSEMINATE IT
WIDELY?
WAND YOU KNOW, IF IT'S A
TRADITIONAL-TYPE WORKSHOP OR
COURSE, MAYBE THAT'S A COURSE
ERROR OF COURSE.
IF IT'S AN APPLICATION THAT'S
SOMETHING ELTS.
I WOULD PUT THE ONUS ON THE
GROUP TO USE THE MONEY TO HELP
WITH DISSEMINATION RATHER THAN
NCATS INVESTING IN THE MIDDLE
GROUND AND THEN THINKING THEY
ARE GOING TO DO THE
DISSEMINATION PIECE.
DOES THAT MAKE SENSE?
>> ANDREA VOGEL: YEAH, THANK,
YOU ARE RIGHT.
IDENTIFYING EXEMPLARS IS JUST
THE BEGINNING AND SEN
DISSEMINATION IS THE ULTIMATE
GOAL.
>> IF I GO OUT AND WIN A
CONCEPT CHALLENGE CONTEST AND I
SPEND MONEY ON INSTITUTIONAL
THINGS, IS IT EASY TO DO THAT,
VERSUS TAKING THE PRIZE MONEY
AS AN INDIVIDUAL OR OUTSIDE
GROUP.
I JUST DON'T KNOW THE ANSWER.
BUT IF IT'S THE LATTER, IF IT'S
HARD TO BRING IT IN OUTSIDE OF
BEING, AND SPEND IT LIKE A
GRANT, IT MAY BE PROHIBITIVE TO
ME TO ACTUALLY DO THE WORK.
>> I WILL TAKE THAT.
SO THE FIRST THING YOU HAVE TO
DO IS CHECK WITH YOUR ACTUAL
INSTITUTIONAL OFFICIALS.
ANYBODY CAN DONATE, IT WOULD BE
SPENT AS YOU PRESCRIBE.
I WOULD IMAGINE.
>> THIS IS GOING TO GET VERY
COMPETITIVE ALREADY, I DON'T
KNOW.
[LAUGHTER]
>> Joni Rutter: ARE THERE ANY
OTHER COMMENTS FROM OUR
COUNCILMEMBERS?
YES, MERMELSTEIN?
>> THANK YOU, GREAT
PRESENTATION AND I LOVE THE
CONCEPT AND FOCUS ON EDUCATION.
WITHIN THE PTSA ORGANIZATION
THERE ARE MANY INNOVATIONS AND
ACCOMPLISHMENTS AS YOU SAID
WITH EDUCATION.
MAYBE IN YOUR COMPETITION THINK
ABOUT BUILDING DEMAND, SO THE
CTSA PROGRAM CERTAINLY BUILT
PROGRAMS AND THEY ARE
STRUGGLING WITH THE FACT THAT
THINK BUILD THEM AND NOBODY
COMES.
SO MAYBE WHAT YOU NEED TO BUILD
INTO THIS COMPETITION IS HOW DO
YOU INCREASE THE DEMAND.
FOR THIS KIND OF EDUCATION.
THERE'S BEEN VERY MANY
INNOVATIONS BUT THERE'S VERY
LITTLE MOTIVATION AND DEMAND
WITHIN ACADEMIA RIGHT NOW FOR
THEM.
THAT SHOULD BE FACTORED IN
SOMEHOW SO IT TRULY HAS AN
AUDIENCE TO DISSEMINATE TO
THOSE WHO ARE ALREADY PART.
>> ANDREA VOGEL: THANK YOU SO
MUCH.
I WOULD SAY ONE OF THE MESSAGES
WE HEARD REPEATEDLY IS WHERE IS
THE SPACE IN THE CURRICULUM TO
INTEGRATE NEW CONTENT.
SO NOW YOU ARE MAKING ME THINK
ABOUT WHETHER THAT'S A THEME WE
CAN HIGHLIGHT IS, YOU KNOW,
APPROACHES THAT ARE EASY TO
UPTAKE, EASY TO IMPLEMENT,,
INTEGRATE WITH CURRICULUM OR
FOR SELF STUDY OR PROFESSIONAL
DEVELOPMENT.
I WOULD BE VERY INTERESTED IN
ADDITIONAL THOUGHTS RELATED TO
THINKING ABOUT HOW WE QUO
SIMULATE SHARING OF MODELS THAT
OVERCOME THAT CHALLENGE.
>> I THINK THE OTHER PART I
REALLY LIKES WITH YOUR
PRESENTATION IS FOCUS ON NOT
ACADEMIA, AND I THINK PERHAPS
THINK THROUGH ANOTHER AUDIENCE
FOR THIS, WHO ARE THE TRANSLATE
YOORS, AMONG THE USE MEDIA AND
REPORTERS AND HOW THESE
TRANSLATIONAL FINDS GET
COMMUNICATED TO THE PUBLIC TO
POLICY MAKERS TO BUILD THAT.
CAN YOU EDUCATE THEM?
>> Joni Rutter: THANK YOU.
GREAT IDEA.
THAT'S IMPORTANT.
ANY OTHER COMMENTS FROM OUR
COUNCIL?
ALL RIGHT.
ANNA, I THINK OVER TO YOU, IF
THERE'S NO FURTHER DISCUSSION.
>> ANNA: INDEED, CAN I HAVE A
MOTION TO APPROVE THE CONCEPT?
>> SERGIO: SO MOVED.
>> ANNA: SECOND?
ALL IN FAVOR?
OPPOSED?
ANY ABSTENTIONS?
WITH THAT, THE CONCEPT IS
APPROVED.
THANK YOU.
>> Joni Rutter: MAKING NOTE.
WE WILL MOVE FORWARD WITH OUR
LAST CONCEPT, IT'S COMING OUT
OF OUR SMALL STEPS NCATS.
WE HOPE TO MOVE THROUGH
RELATIVELY BRIEFLY.
GO AHEAD, MATT.
>> MATT: I ONLY HAVE THREE
SLIDES.
YOU ALREADY HAD HEARD ME SPEAK
TODAY.
YOU KNOW ABOUT THE API AND YOU
ARE GOING TO HEAR FROM TWO
PEOPLE, DR. CATHERINE CHEN IS
GOING TO SPEAK ABOUT AND DR.
SAM MICHAEL WILL JOIN REMOTELY
TO DESCRIBE A CONCEPT OUT OF
THE ASPIRE PROGRAM TO REMIND
YOU, YOU HAVE SEEN
PRESENTATIONS ON THIS, THE
AUTOMATION OF REALLY CLOSE
LABORATORY.
THESE COME OUT OF CLOSE
CONTRACTS PROJECT.
IT'S A LITTLE BIT DIFFERENT, IT
COMES FROM MY COLLEAGUE FROM
DPI.
BOTH OF MY COLLEAGUES WILL
PRESENT THE CONCEPTS TO YOU ONE
AT A TIME, AS AN OPPORTUNITY
FOR FEEDBACK AND THEN MOVE TO
THE NEXT, I'M COGNIZANT OF
TIME, BUT WE PRACTICED AND THEY
HAVE VERY EFFICIENT
PRESENTATIONS FOR YOU.
CATHERINE, DO YOU WANT TO COME
OVER?
YEP.
>> CATHERINE: THANK YOU FOR
HAVING ME HERE, THIS IS A
PRESENTATION ON BEHALF OF
MYSELF AND MY COLLEAGUE DR.
WESHAM.
IT'S SCALABLE GENERATION OF
LIVER AND BRAIN ORGANOIDS
DERIVED FROM HUMAN IPS CELLS.
SO CURRENTLY THERE'S INCREASING
USAGE OF HUMAN IPS CELLS
DERIVED ORGANOIDS AS A DISEASE
MODEL SYSTEM FOR DRUG
DEVELOPMENT AND RESEARCH.
IT COULD BE OBTAINED FROM SKIN
FIBROBLASTS AND BLOOD CELLS.
IPS CELLS HAVE UNLIMITED
EXPANSION CAPABILITIES AND THEY
CAN BE DIFFERENTIATED INTO
VARIOUS DISEASE RELEVANT 2D
CELLS AND ORGAN NOID TO STUDY
DISEASE IN VITRO AND TEST
EFFICACY AND TOXICITY OF DRUG
CANDIDATES.
SO CURRENTLY THERE'S INCREASED
DEMAND FOR THESE ORGANOIDS,
HOWEVER ONE OF THE BOTTLENECKS
IS GENERATION OF THE ORGANOID
IS VERY LABOR INTENSIVE.
ALSO THEY LACK SCALABILITY, AS
WELL AS BATCH TO BATCH
REPRODUCABILITY.
THAT'S WHY DPI IS PROPOSING
THIS CONCEPT TO IMPROVE THE
EFFICIENCY OF THE METHODS AND
REDUCE COSTS FOR GENERATION OF
IPS SCALES DERIVED ORGANOIDS
NOR DISEASE MODELING.
THE SO THE OBJECTIVES OF THIS
CONTRACT SOLICITATION IS FOR
SCALABLE PRODUCTION FOR LIVER
AND BRAIN ORGANOID WITH SOME
LEVEL OF AUTOMATION.
BASED ON NEEDS OF CPI
SCIENTISTS FOR RESEARCH, WE ARE
LIMITING THIS TO BRAIN AND
LIVER ORGANOIDS.
EVEN PARTIALLY AUTOMATED WOULD
REDUCE THE COST OF THE
ORGANOIDS AND ALSO IMPROVE THE
BATCH TO BATCH.
SOME OF THE OTHER KEY CRITERIA
IS THAT ORGANOIDS SHOULD SHOW
THE PROPER IDENTITY MARKERS FOR
EACH TYPE OF ORGANOID AND
SHOULD BE OPTIONS TO INTEGRATE
AS THEY READ OUT SUCH AS
FLUORESCENT BIO SENSORS TO BE
ABLE TO DETECT CELSIUS CHANGES
IN THE IMPACT LIVE ORGANOID.
AND IN TERMS OF IMPLEMENTATION
AND EXPECTED IMPACT AS I
MENTIONED DISEASE MODELING
USING IPSL DERIVED ORGAN NOID
ORIGINALLY THE FAST GROWING
RESOIX AREA, AND BOTTLENECK
THAT WE'RE HOPING TO ALLEVIATE
BY SCALABLE PRODUCTION OF HIGH
QUALITY ORGANOIDS.
AND COMMERCIAL AVAILABILITY OF
THESE ORGANOIDS.
ALSO THIS EFFORT WILL BENEFIT
THE PILOT PROJECT OF THE I3D
PROGRAM.
MATT ALREADY TOLD YOU ABOUT,
THE STATUS IS SEVERAL GROUPS
WITHIN THE I3D WORKING ON PILOT
PROJECT THAT'S FALL UNDER THE
I3D WARE UMBRELLA.
THE CELL LINE FOR ORGANOID
GENERATION CAN EITHER BE MADE
AVAILABLE FROM NCATS.
IN TERMS OF THE START IPS CELL
LINE WE CAN SELECT CELL LINES
WITH DIVERSITY IN AGE, GENDER
AND RACE FOR THE ORGANOID
GENERATION.
AND IN SUMMARY THIS CONCEPT
AIMS TO IMPROVE THE EFFICIENCY
OF THE METHODS AND REDUCE COSTS
IN GENERATION OF HUMAN IPS CELL
DERIVED LIVER AND BRAIN
ORGANOIDS FOR DISEASE MODELING.
OUR HOPE IS THAT BY PROVIDING
HIGH QUALITY ORGANOIDS AT A
MORE AFFORDABLE PRICE, WE WOULD
ENABLE BROAD USE OF THIS
PLATFORM FOR DISEASE MODELING
AND DRUG DISCOVERIES.
AND THE PRIOR PRESERVATION OF
THE ORGANOIDS WILL FACILITATE
COMMERCIALIZATION OF THE
ORGANOIDS AS AN OFF THE SHELF
CATALOG PRODUCT.
SO CURRENTLY IF WE WANT TO OUZ
ORGANOIDS FOR RESEARCHING OUR
LAB WE EITHER HAVE TO GENERATE
THEM OURSELVES OR PUT TOGETHER
A STATEMENT OF WORK IN A
CONTRACT TO HAVE A BATCH
GENERATED AT A CONTRACT
RESEARCH ORGANIZATION.
SO WITH THAT IT'S COSTLY AND WE
SEE A LOT OF BATCH TO BATCH
VARIABILITY.
WITH THAT WE SEE IT COULD BE
OFFERED AS AN OFF THE SHELF
PRODUCT.
IN ADDITION INTEGRATION OF BIO
SENSE SORES WILL FACILITATE
DOWNSTREAM ASSETS.
AND WITH THAT I THANK YOU FOR
YOUR TIME AND WELCOME FEEDBACK.
>> Joni Rutter: THANK YOU SO
MUCH.
WE HAVE TWO DISCUSSANTS, ONE IS
ROBIN.
>> THANK YOU.
IT DOESN'T JUST GET
CONCENTRATED AT ONE POINT.
SO A BROAD REACH THAT WAY.
>> OKAY, THANK YOU.
>> Joni Rutter: FANTASTIC.
AGREE COMPLETELY.
WE NEED TO FIGURE OUT AND MAKE
SURE THESE GET ADOPTED IF THEY
ARE VERY USEFUL.
THANK YOU FOR THOSE COMMENTS.
KELLY McVEARRY?
>> DR. CHEN, THIS IS SO EXCITE,
I'M REALLY THRILLED THIS IS
GOING TO BE PUT OUT THERE TO
COMMERCIALIZE.
YOU ASKED FOR FEEDBACK ON WHO
WOULD BE EARLY ADOPTERS.
IT SEEMS TO ME THAT THERE'S AN
INTERESTING ROLE THAT THE
PATIENT ADVOCACY GROUPS COULD
PLAY IN MAKING SURE THAT SOME
OF THE BIOTECH COMPANIES AND ET
CETERA SORT OF PRIORITIZE THIS.
BUT I'M NOT SURE, I MEAN I
CAN'T IMAGINE THAT YOU DON'T
HAVE THE RIGHT ECOSYSTEM OF
EARLY ADOPTERS.
IF THERE'S A SPECIFIC PROBLEM I
WOULD LOVE TO KNOW WHY.
MY QUESTION, I WAS SORT OF
SURPRISE TODAY SEE THIS
QUESTION, WHO WOULD BE THE
EARLY ADOPTERS?
BECAUSE IT SEEMS LIKE THIS WILL
BE EMBRACED.
SO I'M CURIOUS ABOUT THAT AS A
BUSINESS PROBLEM TO SOLVE AND
HOW WE CAN HELP.
FOR THE WHICH DISEASES WOULD
BENEFIT MOST FROM THIS, I THINK
IT'S SO SENSIBLE TO GO AFTER
BRAIN AND LIVER.
I WAS KIND OF HOPING TO SEE
KIDNEY THERE.
I WAS THINKING BECAUSE WHEN YOU
ASKED WHICH DISEASES I WAS
THINKING WOULDN'T THIS BE GREAT
TO BRING INTO THE LUPUS
POPULATION.
SO I COULD ELABORATE MORE.
I WOULD LOVE FOR YOU TO ADDRESS
THOSE QUESTIONS AND GET SOME
CLARITY ABOUT WHY YOU ASKED THE
QUESTION WHO WOULD BE THE EARLY
ADOPTERS.
AND THEN I THINK I HAVE A
PRACTICAL QUESTION, BY
RELEASING THIS ONTO AN SBR AND
STTR MECHANISM, MY
UNDERSTANDING OF THAT PROCESS
THERE ARE REAL CONSTRAINTS OF A
STAGE OR PHASE ONE IS A PRETTY
SMALL BUCKET OF MONEY, AND
PHASE TWO IS A MUCH LARGER
BUCKET OF MONEY WHEN THERE'S
BEEN SOME EARLY PROOF.
BUT THAT STILL SEEMS TO BE
SMALL FOR SOME OF THE GOALS
HAVE TO PO TO* ACHIEVE BATCH TO
BATCH REPRODUCABILITY AND
ULTIMATELY FIELD OFF THE SHELF
PRODUCT.
SO I'M JUST WONDERING IF THE
SAME DOLLAR LIMITS APPLY FOR
THE PARTICULAR OPPORTUNITY YOU
ARE GOING TO RELEASE.
THOSE ARE MY THREE THOUGHTS TO
DISCUSS.
>> YEAH, THANK YOU.
SO THIS IS A PHASE ONE.
SO THEN THE CHOICE FOR LIVER
AND BRAIN IS MOSTLY BASED ON
THE RESEARCH THAT WE HAVE
CURRENTLY GOING ON.
AND THEN ALSO IT'S LOOKING AT
WHICH ORGANOID TECHNOLOGY IS
MORE MATURE TO BE ADOPTED IN
THIS FASHION.
YEAH, I DEFINITELY THINK, MAYBE
IF THIS WAS SUCCESSFUL THAN IN
LATER PHASES IT COULD BE
EXPANDED TO OTHER ORGANOIDS AS
WELL.
AND I THINK, I GUESS THE OTHER
QUESTION IS THE MONEY LIMIT ON
THIS?
>> YEAH, ARE YOU GOING TO BE
ABLE TO ACHIEVE YOUR GOALS WITH
THIS PROGRAM IF THE NORMAL
DOLLAR LIMITS ARE IN PLACE,
THAT'S MY UNDERSTAND, TYPICALLY
GOVERN THESE PROGRAMS?
>> YEAH, THAT'S A GREAT
QUESTION, DR. McVEARRY.
$225,000 ISN'T A LOT OF MONEY,
SO THE STATEMENT HAS BEEN
CAREFULLY CRAFTED SO THE AIMS
ARE ACHIEVABLE WITH THAT MONEY.
WE MIGHT FOCUS ON CERTAIN PLATT
FOORM BUILD.
CAPABILITIES SUCH AS
REPRODUCABILITY.
WE WILL BE ABLE TO WORK OUT
THOSE THINGS, BUT IF WE DO
SUCCESSFULLY DEVELOP PHASE TWO,
IT WILL BE A LARGER PART OF
MONEY, OR SOME OF THE MORE
COMPLICATED ASPECTS SUCH AS THE
BIO SENSOR, THE DIVERSITY OF
THE HIGH PSV POOL, BECAUSE ONCE
YOU START WIDENING THE LENGTH,
TERMS OF REQUIREMENTS, THE
AMOUNT OF MONEY YOU NEED GETS
EXPONENTIALLY START TO GREEN.
WE HAVE TO BE CAREFUL.
JUST 2-3 VERY IMPORTANT
QUESTIONS TO ASK.
>> RIGHT.
THIS WILL HAVE A HUGE TOTAL
ADDRESSABLE MARKET IN THE
UNFUNDED MAZE 3.
-- PHASE 3.
GETTING IT TO THAT POINT SEEMS
HARD WITH THE DOLLAR
CONTRAINTS, THAT'S ALL.
SO THANK YOU FOR YOUR VERY
THOUGHTFUL ANSWER.
>> Joni Rutter: AND JUST ONE
MORE SORT OF TANGENTIALL YE
RELATED ASPECT TO THIS AS YOU
RECALL, MANY MODELS WE HOPE
WILL BE INFUSED IN THAT SPACE
AS WELL.
SO THERE MAY BE SOME PARALLEL
EFFORTS.
THEY WON'T BE THE SPR COMMUNITY
BUT THIS, I THINK WILL WORK IN
PARALLEL WITH SOME OF THE
CONFUSION OF THE EXCITEMENT AND
TECHNOLOGIES AND GENERATED
THROUGH THERE AND PERHAPS THERE
WILL BE SOME CONVERGENCE TO
ULTIMATELY GET MORE TISSUES AND
ORGANS REPRESENTED AS WELL.
BUT STARTING HERE WILL
HOPEFULLY GIVE US A FOCAL POINT
TO MOVE THIS FORWARD.
>> ONE MORE POINT, MS.
McVEARRY, ONCE SOMEONE UNLOCKS
PROOF OF CONCEPT OR FEASIBILITY
PROBLEM THEN OTHER PRIVATE
FUNDING MIGHT EVEN FLOW IN.
BECAUSE WHAT HAPPENS IS SMALL
BUSINESS IS ABLE TO SHOW THIS
IS A SUSTAINABLE BUSINESS, FOR
WHICH THEY HAVE SOLVED ONE OF
THE REALLY IMPORTANT VARIABLES
AND PROBLEMS,
>> RIGHT.
THANK YOU.
>> Joni Rutter: ARE THERE ANY
OTHER COMMENTS FROM OUR COUNCIL?
OKAY, HEARING NONE.
ANNA, WE WILL TURN IT OVER TO
YOU.
>> ANNA: ALL RIGHT, MAY HAVE I
A MOTION TO APPROVE THE CONCEPT?
SECOND.
ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT, IT'S APPROVED.
>> Joni Rutter: THANK YOU, WITH
THAT WE HAVE ONE MORE TO GO.
I WILL KICK IT OVER TO MICHAEL
TO GET US STARTED.
THIS IS DEVELOPMENT OF
VERSATILE SMALL FOOTPRINT
BEFRNL TOP DEVICE TO PERFORM
BATCH EVAPORATION.
[READING RAPIDLY]
AS DAN TAGLE SAID AND MATT HALL
SAID AS PART OF THE ASPIRE
PROCESS WE ARE TRYING TO FOCUS
ON THE AUTOMATION OF CHEMISTRY.
AND FOR THE PERSPECTIVE, MOST
FOLKS ARE AWARE OF THE STRONG
CLOSE TO TWO DECADE PROCESS OF
AUTOMATION OF BIOLOGY AND THAT
COUPLES WITH 20 PLUS YEARS OF
BILLIONS OF INVESTMENT IN
INDUSTRY TO OVERCOMING SOME OF
THESE BOTTLENECKS.
THIS ISN'T THE MOST GLAMOROUS
COMPONENT.
BUT THAT SIMILAR INVESTMENT HAS
NOT BEEN MADE IN THE AUTOMATION
OF CHEMISTRY.
REALLY TOWARD THAT GOAL OF
AUTOMATE UGH CHEMISTRY.
NEXT SLIDE, PLEASE.
THEY SHOULD BE ABLE TO REACH
90% TOTAL EVAPORATION IN UNDER
TEN MINUTE DOLLARS.
THIS SAW KEY POINT, THEY WANT
TO GET DOWN TO 100%.
IN THIS CASE WE WANT TO DO IT
IN A BATCH FASHION TO GET US TO
90% COMPLETELY.
SO SOME KEY AREAS THAT WE WILL
HAVE TO BE ABLE TO USE, WE WILL
HAVE TO USE HIGH RECOVERY VIALS
SPECIFIED BY NCATS.
AGAIN, THIS ISN'T A GLAMOROUS
TOPIC, BUT PART OF THE REASON
BIOLOGICAL SCREENING HAS BEEN
SUCCESSFUL, THE SBS FOOTPRINT
WHICH SAY MICRO TITER PLATE.
WE ARE TRYING TO UTILIZE
EXISTING VIALS THAT ARE IN THE
CHEMICAL WORKFLOW, BUT STANDARD
VIAL SO WE CAN AUTOMATE EVERY
STEP THE PROCESS.
RELATED TO THAT HAS TO BE
SUITABLE FOR USE WITHIN
EXISTING AUTOMATED CHEMICAL
SENSE WORTH FLOW DOLLARS AND
FINALLY A DEVICE MUST BE
AUTOMATION FRIENDLY OR HAVE AN
API FOR THIS DEVICE.
WE HAVE THE DESIGN OF A
MOLECULE, COMPLETE WORKFLOW
FROM START TO FINISH, FROM
EVERYTHING FROM THE VIRTUAL
DESIGN TO ULTIMATELY HAVE THE
SYNTHESIS OF WHATEVER COMPOUND
WE ARE LOOKING TO MAKE IN AN
AUTOMATED FASHION.
NEXT SLIDE, PLEASE.
SO FROM THE IMPLEMENTATION
EXPECTED IMPACT, WE ALREADY --
IF WE ARE DOING AN SBIR WE
HAVE A GOOD IDEA GET SOMETHING
TO WORK, BUT IN THIS CASE, WE
WOULD LIKE TO HAVE I COMMERCIAL
DEVICE WE CAN JUST BUY OFF THE
SHELF.
NOT JUST FOR US, BUT FOR OTHERS
IN THE RESEARCH COMMUNITY.
OTHER LABORATORIES WOULD LIKELY
USE THIS AS WELL SINCE MORE AND
MORE FOLKS ARE TRYING TO GET
INVOLVED WITH AUTOMATIC
CHEMISTRY.
SO FOR THE PROGRAMMATIC
SIGNIFICANCE OF THIS, HAVING
THE AUTOMATED BATCH EVAPORATED
WOULD BE A KEY COMPONENT.
THERE ARE OTHER MEANS OF
WORKFLOWS BUT THEY ARE SLOW AND
NOT SUITABLE FOR AUTOMATION OR
ONLY PERFORM FULL EVAPORATION
ON ONE VIAL AT A TIME.
IMAGINE YOU ARE TRYING TO CLOSE
AFTER ONE ARTICLE OF COLLEGING.
IT'S NOTE REALLY AN EXPEDITED
WAY.
GET OVER THIS GOAL TO CREATE
MORE COME PRINCE
GEORGE-VALEMOUNTS.
THIS IS A KEY DEVICE THAT NCATS
IS ALREADY USING, PROTOTYPE IN
OUR LABORATORY, THERE'S BEEN
STRONG INTEREST EXPRESSED BY
OTHER LABS, THIS WAS PRESENTED
AT SLAS CONFERENCE THIS PAST
WINTER.
AS PART OF A BROADER PORTFOLIO
NCATS WORKED ON.
THIS WAS THE DEVICE MANY
CHEMISTS FOLLOWED UP AND TALKED
TO ENGINEER ESSENTIALLY HOW DO
I GET ONE OF THESE.
NEXT SLIDE, PLEASE.
SOME EYE VAP RATION, THAT'S A
ROTOAPP.
THE VIALS IN THE LAB WARE IT'S
REALLY NOT AUTOMATION FRIENDLY
AT ALL.
THIS IS REALLY NOT SUITABLE FOR
US.
ON THE RIGHT THAT'S A BIOTAGE,
V 10.
THE SEMI AUTOMATED AND SERIAL
PROCESS, MEANING ONE VIAL AT A
TIME.
NEXT SLIDE, PLEASE.
SO THE BATCH, THIS IS A
RENDERING OF WHAT THIS LOOKS
LIKE, ON THE BOTTOM A HEATING
BLOCK WITH FOUR DIFFERENT
MODELS.
THEN WE PUT THIS UNDER PRESSURE
WITH NITROGEN AND HEAT UP TO
50% CELSIUS.
WE WILL IMPLEMENT THAT WITH A
BIOTAGE.
WHAT WE WANT TO SHOW IS FOR
COMMON SOLVENTS USED WE WANT A
WIDE VARIETY OF SOLVENTS,WE
HAVE THIS UNDER 2 PSI OF
NITROGEN PRESSURE.
WE WERE UNDER TEN MINUTES.
WE USE WATER AS A CONTROL JUST
TO GIVE AN EXAMPLE WHAT IT
TAKES FROM AN EVAPORATION
PERSPECTIVE.
WE CAN DO FOUR AT A TIME.
FINALLY FOR THE OPT MYCATION,
IF WE ONLY DID SERIES, IT TAKES
ABOUT 8 MILS.
IF WE IMPLEMENT, AND IF WE
LIFRNL THOSE TOGETHER THE
PROSAYS GOES FROM 60 MINUTES,
WE SAVE CLOSE TO 60% OF THE
TIME MAKING IT AN EFFICIENT
PROCESS BY LINKING THESE TWO
TOGETHER.
NEXT SLIDE, PLEASE.
IN SUM ARE YOU WE PROPOSE THIS
CONCEPT DEVELOP A BATCH DEVICE
MULTIPLE OF RECOVERING
MULTI-MENT.
THIS IS JUST A KEY BOTTLENECK
RIGHT NOW MANY GROUPS ARE
STRUGGLING WITH REGARDS TO
AUTOMATING CHEMICAL SYNTHESIS.
NEXT SLIDE, PLEASE.
AND SO IN THIS CASE, THIS IS TO
THE COUNCIL.
WHAT MIGHT IMPROVE THIS, WHAT
ARE KEY POINTS TO CONSIDER,
WHAT DOES SUCCESS LIKE.
ONE OF THE QUESTIONS WE HAVE
HAD IS WITH REGARD TO NCATS
PATENT.
WE WOULD DO A NON-EXCLUSIVE
LICENSE TO ANYONE WHO COMES UP
WITH A MODEL HERE.
WE HAVE THE IP BECAUSE WE DID
COME UP WITH THIS IDEA.
BUT WE IN NO MEANS WANT TO MAKE
THIS RESTRICTED, THE WHOLE
INTENTION OF ASPIRE IS TO HAVE
RESOURCES AVAILABLE TO THE
BROADER COMMUNITY.
>> Joni Rutter: GREAT, THANK
YOU SO MUCH, SAM.
WE WILL MOVE INTO THE
DISCUSSION.
KELLY McVEARRY, PLEASE GO AHEAD.
>> THAT WAS REALLY COOL, SAM,
THANK YOU SO MUCH.
IT STRIKES ME THIS HAS
POTENTIAL FOR BEING FUNDAMENTAL
LABORATORY TECHNOLOGY THAT'S
JUST IMPROVES PROCESSES BEYOND
BIOMEDICAL RESEARCH, CONSIDER
QUESTIONS TO SIMULATE
CONVERSATION.
I WAS GOING TO MARKET SIZING.
WOULD YOU BE ABLE TO EXPLAIN TO
US, OUR YOU SEE THE USE CASES
BEYOND BIOMEDICAL RESEARCH AS
THAT HELPS FRAME WHAT KIND UP
TAKE, OR DO YOU SEE IT AS A
MORE LIMITED MARKET OF USERS.
>> WE STARTED THINKING OF THIS
SPECIFIC TO THE NEEDS OF THE
LABORATORY, TAKING A STEP BACK
TO YOUR POINT.
THE GOAL, THERE'S A VARIETY OF
REASONS PEOPLE WANT TO
EVAPORATE SOLVENTS.
YOU COULD CONSIDER COOKING
APPLICATIONS AS A REDUCTION.
THEY REALLY AREN'T THAT
EXPENSIVE.
WE HAD A CUSTOM MADE HEATING
BLOCK, IT MIGHT BE A CHALLENGE
IN KITCHENS TO GET THE PRESSURE.
BUT TRUTH BE TOLD THAT JUST
MAKES IT GO A LITTLE BIT FASTER.
SO THERE'S POSSIBILITY THERE
COULD BE UTILITY BEYOND JUST
THE BENCH TOP DEVICE IN A
CHEMICAL HOOD.
BECAUSE THERE'S MANY
APPLICATIONS WHERE QUICK LE
EVAPORATING SOMETHING, ONE IS
FOOD PROCESSING, FOOD
TECHNOLOGIES.
IT'S NOT A SPACE WE ULTIMATELY
GET INVOLVED WITH BUT
POTENTIALLY COULD BE AN
ADDITIONAL MARKET THAT WOULD
HAVE INTEREST.
WE HAVE OUR NEED BUT I LIKE
PUTTING SOMETHING OUT THERE.
THE BROADER BUSINESS COMMUNITY.
MANY FOLKS READ THESE, THEY MAY
HAVE UTILIZATION FOR THIS
TECHNOLOGY WE HAVEN'T QUITE
CONSIDERED.
>> THAT'S A GREAT NOTE TO END
ON.
THAT WAS A TERRIFIC ANSWER.
SO MY FOLLOW-UP QUESTION IS YOU
MENTIONED THAT YOU HAVE THE IP,
IT'S GOING TO BE A
NON-EXCLUSIVE LICENSE THAT'S
AVAILABLE TO SBIR RECIPIENTS
THAT ARE SUCCESSFUL.
DO YOU SEE ANY RISK BY NOT
OFFERING AN EXCLUSIVE LICENSE,
IF SOMEBODY REALLY WANTS TO
JUMP IN.
BECAUSE IT POSES A LITTLE BIT A
RISK TO THE SMALL BUSINESS TO
THINK THEY CAN'T COMMERCIALIZE
AFTER MAKING A MULTI-YEAR
INVESTMENT TO KIND OF
PROTOTYPE, AND SCALE TO
PRODUCTION.
>> IT'S A GOOD QUESTION AND ONE
WE TALKED ABOUT.
ONE OF THE CHALLENGES WE SAW
WITH POTENTIAL FOR AN EXCUSIVE.
IS THE TIME IT TAKES TO EXECUTE
THEM.
I THINK PHASE ONE SOME OF THE
TECHNICAL SIDES GO UP TO 375
AND PHASE TWO WAS 2 MILLION
OVER TWO YEARS.
WE HAVE SEEN THIS IN THE PAST,
THE CHALLENGE IS THE DOLLAR
AMOUNTS ARE GOOD BUT REALITY TO
GET A COMMERCIAL PRODUCT NOT
ENOUGH.
I THINK THE POINT ABOUT THAT
INVESTMENT REQUIRED, IF SOMEONE
HAS A SUCCESSFUL PRODUCT I
THINK WE WOULD BE OPEN TO A
LICENSE.
PART OF THE REASON FOR PHASE
ONE IN PARTICULAR IS THE TIME
IT TAKES TO EXECUTE AN
EXCLUSIVE LICENSE MIGHT TAKE
TOO LONG BEFORE WE HAVE TIME TO
GET TO THE NEXT STEP.
IF THAT ANSWERS YOUR QUESTION.
IT'S A CONSIDERATION WE HAVE,
WE UNDERSTAND THE CONCERN OF
THE SMALL BUSINESS.
THE SBR'S THE DOLLARS GO QUICK,
THE TIME TOO, IT GOES VERY FAST.
>> THANK YOU, IT'S EXCITING.
>> IN TERMS OF EXCLUSIVE
LICENSE DEBATE, WHAT HAPPENS IN
THESE PRODUCT DEVELOPMENT
CYCLES THERE WILL BE A LOT OF
FOLLOW-ON PATENTS THAT
ENGINEERING COMPANIES WILL FILE
ON.
THEY WILL AUTOMATICALLY AGREE
ADDITIONAL FENCES, I DON'T
THINK HAS HAVING AN-LIKE SAM
SAID IT MAY BE TOO EXPENSIVE
AND OUT OF REACH AND TOO LONG
TO EXECUTE TO GET THIS PROJECT
STARTED.
SO I THINK THE NON-EXCLUSIVE
LICENSE WOULD GET THINGS
STARTED.
ONE THING START THERE WILL BE
FURTHER AUTOMATION PROGRESSION
OF TECHNOLOGY DEVELOPMENT AND
FURTHER PATENTING, I THINK IT
IS HARD TO PREDICT RIGHT NOW.
>> YEAH, THAT'S GREAT, THANK
YOU.
>> Joni Rutter: AND THEN WE
HAVE KEITH MUELLER.
>> KEITH MUELLER: GREAT
PRESENTATION, YOU ANSWERED ALL
THE QUESTIONS I WAS WRITING
DOWN, YOU ANSWERED DURING THE
PRESENTATION.
SO THAT'S GREAT.
MY FOCUS IS AROUND WHAT DOES
SUCCESS LOOK LIKE.
I'M HEARING TWO THINGS.
WITH UTILIZATION A PRODUCT
BEING MORE WIDESPREAD THAN JUST
IN THE LAB AT NCATS AND I THINK
THAT'S A LEGITIMATE QUESTION TO
ASK AND ANSWER IF THIS ROLLS
OUT.
THE OTHER ONE WE HAD, AND THIS
IS SORT OF NAIVETE IF YOU WILL
SOMEBODY WHOSE NOT A CHEMIST
FROM DOING CHEMICAL RESEARCH.
AND UNDERSTANDING THE 60
MINUTE, 24 MINUTE GAIN THAT
CAME THROUGH REALLY CLEAR IN
THE PRESENTATION.
I APPRECIATE THAT.
I WOULD LIKE TO HEAR THE FINE
AIL I GUESS, CONNECTION HOW
DOES THAT IN TURN INFLUENCE THE
FINAL PRODUCT OF THE RESEARCH,
WHAT DOES IT MEAN TO SPEED UP
THE PROCESS TO THE ULTIMATE
OUTCOME OF THE RESEARCH
ACTIVITY.
>> IT'S A GREAT QUESTION.
TO THE POINT FIRST POINT, YEAH,
THIS IS REALLY ON OUR SIDE, THE
MARKET RESEARCH IS JUST BEING
INVOLVED IN THE ASPIRE PROGRAM
AND OVERCOMING THESE
BOTTLENECKS WE HAVE BEEN IN
TOUCH WITH MANY LABS IN THE
PROGRAM FROM AROUND THE WORLD,
LITERALLY.
THIS IS A KEY BOTTLENECK, THE
OTHER IS PURIFICATION.
THERE'S REALLY TWO PROBLEMS WE
SEE AS BOTTLENECKS.
THIS LEADS INTO YOUR SECOND
QUESTION, REALLY THE MORE
IMPORTANT QUESTION, SAY WE CAN
OVERCOME THOSE BOTTLENECKS,
THIS COMES DOWN TO THE
PRINCIPLE BEHIND THE ASPIRE
PROGRAM IS THIS WHOLE DESIGN
MAKE, TEST, ANALYZE PARADIGM.
THE GOAL IS WE WANT TO REDUCE
THAT ITERATION TIME TO GO
FASTER AND FASTER AND HOPEFULLY
GET TO MORE MOLECULES, THAT'S
IF WE CAN PRODUCE MOLECULES
FASTER, THAT COULD LEAD TO
TESTING AND TESTING COULD LEAD
TO TREATMENTS.
THIS IS PART OF THE REDUCTION
OF THE OVERALL RATE LIMITING
AND WE HAVE SEEN FOLKS OUT
THERE THAT HAVE THESE AUTOMATED
SYSTEMS THEY CAN GO REALLY FAST.
FOR ANY PROCESS ULTIMATELY YOUR
RATE LIMITING STEP IS WHAT
DRIVES YOUR TOTAL TIME FOR THAT
ITERATION, WHICH IS JUST A RATE
LIMITING STEP WE HAVE TO
OVERCOME.
BROADER PROCESS YOU WERE
MENTIONING WHEN WE LOOK AT THE
INTYRE WORKFLOW.
TAKE THESE RATE LIMTING STEPS
ONE AT A TIME.
THAT'S PART OF THE REASON WE
STARTED WORKING ON THIS.
WE HAVE TO BE AMENABLE TO
AUTOMATION.
THE GOAL IS REDUCE THAT TOITAL
TIME, HERE IS THE DESIGN OF THE
MOLECULE, ALL THE WAY TO WE
MADE IT, WE PURIFIED IT, WE
VALIDATED IT, AND THIS IS A KEY
STEP IN THE WORKFLOW.
THE DREAM IS ULTIMATELY WE WILL
BE TO THE POINT WE HAVE THE
WORKFLOW MEASURED IN HOURS.
THE REALITY RIGHT NOW THAT WILL
BE WEEKS, DAYS.
WE ARE TALKING ABOUT HOURS FOR
THAT WITH A BATTERY OF TESTS.
SO JUST GREAT QUESTION, BUT
OVERALL TO REALLY OVERCOME A
RATE-LIMITING STEP.
>> Joni Rutter: GREAT, THANK
YOU VERY MUCH.
ARE THERE ANY OTHER QUESTIONS
FROM OUR COUNCILMEMBERS?
SEEING NONE, I WILL TURN IT
OVER TO ANNA FOR A VOTE.
>> MAY I HAVE A MOTION TO
APPROVE THE CONCEPT.
>> SO MOVED.
>> SECOND.
>> ALL IN FAVOR?
OPPOSED?
ANY ABSTENTIONS?
WITH THAT OUR FOURTH CONCEPT
FOR THE DAY IS APPROVED.
THANK YOU.
>> Joni Rutter: ALL RIGHT.
AND WE WILL NOW WITH OUR LAST
TOPIC OF THE DAY, SUPER EXCITED
ABOUT THIS DISCUSSION, I ARE
TURN IT OVER TO MERIDETH
TEMPLE-O'CONNOR WHO HAS BEEN
LEADING THE EFFORTS ON OUR
STRATEGIC PLANNING PROCESSES
WITH HER VERY CAPABLE TEAM AND
I WILL TURN IT OVER TO HER FOR
THE PRESENTATION AND DISCUSSION
AFTERWARDS.
>> THANK YOU VERY MUCH.
LET ME SURE I CAN ADVANCE THIS.
THANK YOU.
WE WANT TO FOCUS REALLY ON THE
DISCUSSION TODAY.
AND THE COUNCILMEMBERS HAVE HAD
THE OPPORTUNITY TO SEE THE
DRAFT PLAN IN ADVANCE.
BUT I DID WANT, FOR THE BENEFIT
OF COUNCIL AND FOR FOLKS
LISTENING ON THE VIDEOCAST TO
HEAR HOW WE GOT WHERE WE ARE.
WE ARE EXCITED TO BE HERE.
AS CAN YOU SEE WE ARE ALMOST AT
THE END OF OUR TIMELINE.
WHICH IS FANTASTIC TO SEE.
WE STARTED THIS NOVEMBER 2022
WHEN JONI WAS NAMED THE
PERMANENT DIRECTOR FOR NCATS.
SO A KEY THEME THROUGHOUT ALL
OF IT HAS BEEN OUR ENGAGEMENT.
ENGAGEMENT WITH ALL OF OUR
CONSTITUENTS, INTERNALLY AND
EXTERNALLY.
AS YOU SAW ON THE PRIOR SLIDE,
IT'S GOING CRAZY ON ME.
IT'S REALLY BEEN A TREMENDOUS
ASSET SO OUR STRATEGIC PLANNING
PROCESS OVERALL.
THIS IS REALLY MEANT TO JUST
REMIND FOLKS OF THE BREADTH AND
DEPTH OF CONVERSATIONS WE HAVE
HAD ACROSS OUR CONSTITUENT
COMMUNITIES.
AND IT REALLY DID, WE COULDN'T
CONTINUE ON OR ELSE WOULDN'T BE
ABLE TO SEE THINGS BUT IT
REALLY HAS CONTINUED PAST
AUGUST.
WE HAD INBOX GOING, THE RFI
THAT WAS IN THE FALL FOR THE
PUBLIC TO COMMENT ON OUR
FRAMEWORK.
AND WE HAVE CONTINUED TO ENGAGE
STAFF AT DIFFERENT LEVELS.
WE HAVE HAD TOWN HALLS.
WE MET WITH INDIVIDUAL GROUPS.
ACROSS THE ORGANIZATIONAL
STRUCTURE TO INFORM THE
FRAMEWORK AND THE DRAFT PLAN.
SOMETHING ELSE I WANT TO TALK
ABOUT BRIEFLY, NOT ONLY WE GOT
TRY MEND US AMOUNT FEEDBACK AS
YOU SAW OP THE FIRST SLIDE,
OVER 1700 COMMENTS.
WE GOT 50 PLUS COMMENTS TO THE
RFI.
BUT FROM ALL OF THOSE MEETINGS
ABOUT 1700 COMMENTS THAT WE
DIGESTED, TEAM REALLY DID AN
EXCELLENT JOB USING TOOLS THAT
NIH HAS FOR THIS PURPOSE TO
ANALYZE THE INPUT THAT WE
RECEIVED.
THESE WERE KEY TOPICS THAT
EMERGED.
THESE WERE SOME OF THE BIGGER
ONES.
WE WANTED TO SHOW AND ENDED UP
DOING AN ANALYSIS TO SHOW HOW
DIFFERENT GROUPS WE ENGAGE WITH
ON THAT PRIOR SLIDE HAD A LOT
COMMON GROUND.
SO YOU COULD SEE FROM LEFT SIDE
TO THE RIGHT HOW THE DIFFERENT
GROUPS MAPPED TO THE DIFFERENT
KEY TOPICS.
AND THERE WAS A LOT OF
CRISSCROSS, THAT WAS ACTUALLY
GREAT FOR US TO SEE, THAT SHOWS
THERE'S A LOT OF COMMON GROUND
ACROSS OUR DIFFERENT
CONSTITUENTS IN TERMS OF THE
INPUT THEY PROVIDED AND HOW
THAT HAS INFORMED PLAN 6789
THIS IS THE FRAMEWORK LAST FALL
AS A RESULT OF THE INPUT.
SO THAT WAS SORT OF BY DESIGN.
THE ENGAGEMENT WAS REALLY MEANT
TO INFORM HOW WE STRUCTURED
THIS.
BECAUSE OF THE WAY THE FEEDBACK
CAME IN AND BECAUSE JONI
ALREADY HAD A VERY CLEAR VISION
FOR HOW SHE WANTED NCATS TO
MOVE FORWARD, WE WERE ABLE TO
LEVERAGE THAT, BOTH IN OUR
ENGAGEMENT PROCESS AS WELL AS
IN BUILDING THE FRAMEWORK TO
REFLECT THAT FEEDBACK.
SO WE RESULTED IN FIVE GOALS,
AS YOU SEE HERE.
WE HAD A CROSS CUTTING ACTIVITY
AND EFFORTS UNDER GOAL FOUR AND
THEN WE DECIDED BASED ON THE
INPUT AND FEEDBACK FROM OUR
COMMUNITY AND THE IMPORTANT
INTERNALLY OF STEWARDSHIP TO
ELEVATE IT TO BEING A GOAL AS
WELL.
I WOULD BE REMISS TO NOT
ACKNOWLEDGE THIS HAS BEEN A
TEAM EFFORT.
I PARTICULARLY WANT TO CALL OUT
DRS.JESSICA WALRATH.
AND JEN.
MANY OTHER PEOPLE PLAYED A ROLE.
WE CALLED OUT THOSE WHO PLAYED
A SPECIFIC ROLE IN THE PROCESS.
ALL OUR CONSTITUENTS INTERNALLY
AND EXTERNALLY HAVE BEEN VERY
ENGAGED.
OUR STAFF AT EVERY LEVEL HAVE
BEEN SUPER ENGAGED THROUGHOUT
THIS TIME WHICH HAS BEEN
EXTREMELY REWARDING TO SEE.
THE LAST THING BEFORE WE SHIFT
TO DISCUSSION, I WANTED TO MAKE
A PLUG, THE DRAFT PLAN IS OUT
ON OUR WEBSITE.
AND YOU CAN EITHER GET TO IT
THROUGH THE QR CODE, THROUGH
OUR WEBSITE IF YOU GOOGLE NCATS
STRATEGIC PLAN, IT'S THE FIRST
PAGE THAT COMES UP.
AND IT WILL BE OUT UNTIL JUNE
14th FOR COMMENTS FROM THE
PUBLIC.
AND YOU CAN EMAIL YOUR COMMENTS
TO THE EMAIL ADDRESS HERE.
IT'S ALSO ON OUR WEBSITE AS
WELL.
WITH THAT, WHAT I WOULD LIKE TO
DO WITH DR. RUTTER IS HAVE A
COUPLE OF QUESTIONS, REALLY
JUST TO KIND OF BREAK THE ICE,
IF YOU HAVE A FAVORITE GOAL OR
OBJECTIVE, WE WOULD LOVE TO
HERE THAT FROM THE
COUNCILMEMBERS.
ALSO DO YOU FEEL THE GOALS AND
OBJECTIVES ALIGN WITH OUR
VISION AND MISSION?
IS ANYTHING MISSING THE MARK?
THOSE ARE SORT OF OUR FRAMING
QUESTIONS FOR THE DISCUSSION.
OTHERWISE WE WOULD JUST LIKE TO
HEAR ANY THOUGHTS THAT YOU
WOULD LIKE TO OFFER.
I DO HAVE EACH GOAL A SLIDE FOR
EACH GOAL WITH ITS OBJECTIVES
WE CAN JUMP AROUND IF THAT'S
HELPFUL.
BUT WE WANTED TO FOCUS MORE ON
CONVERSATION THAN JUST WALKING
THROUGH THAT TOGETHER.
SO WITH THAT --
>> Joni Rutter: WITH THAT,
THANK YOU SO MUCH, MERIDETH,
THAT WAS TERRIFIC.
AND I THINK I WOULD LIKE TO
JUST START US OFF WITH THAT ICE
BREAKER.
I ASKED THIS QUESTION TO THE
I.T. DIRECTORS WHEN WE HAD THE
ENGAGEMENT WITH THE I.T.
DIRECTORS.
I WAS REALLY DELIGHTED TO HEAR
THEIR FAVORITE GOAL
COLLECTIVELY.
THEY ALL HAD DIFFERENT ONES BUT
ONES JUMPED OUT.
SO I'M KIND OF CURIOUS WITH YOU
WHAT YOUR FAVORITE GOAL IS.
AND THEN WOULD LOVE TO HEAR
YOUR THOUGHTS ON HOW THESE
ALIGN WITH THE VISION MISSION
AS YOU SEE IT AND ARE WE
MISSING THE MARK.
SO PLEASE WOULD LOVE TO HERE
YOUR COMMENTS ON THESE THINGS.
AND SERGIO, I SEE YOUR HAND UP,
SO YOU GET THE HONOR OF GOING
FIRST.
THANK YOU.
>> SERGIO: A LITTLE PRAISE OF
THE PROCESS YOU HAVE GONE
THROUGH.
FOR ALMOST AT LEAST A YEAR AND
A HALF.
AND ALL THE LISTENING YOU DID
FOR THIS DRAFT THAT YOU ARE
PROVIDING US WITH, IT'S A MAJOR
PIECE OF WORK.
VERY PROUD YOU HAVE DONE THIS
AND THAT NCATS HAS TAKEN THE
LEAD ON REALLY CONSULTING WITH
SO MANY PEOPLE FROM SO MANY
DIFFERENT DISCIPLINES.
THAT'S PRAISE.
I HAVE TWO FAVORITE GOALS.
NUMBER TWO AND IT'S NUMBER FIVE.
BUT NUMBER TWO, MY GOODNESS, I
THINK THAT IS CAPTURED IN SOME
OF THE INITIAL TAKE AWAYS YOU
WERE SHARING WITH US IN THE
FIRST MEETING THAT WE ATTACHED
ON THE THE STRATEGIC PLAN.
NUMBER TWO IS ENABLE ALL PEOPLE
TO CONTRIBUTE TO AND BENEFIT
FROM TRANSLATIONAL SCIENCE.
I HAVE A FAVORITE OBJECTIVE
WITHIN THAT.
WHICH IS RELATED TO
ESTABLISHING TRUST, OR BUILDING
TRUST.
I THINK THAT IS SO CRITICALLY
IMPORTANT THAT WE FOCUS ON THAT.
I ENDEAVOR TO LOOK FOR HOW MANY
TIMES TRUST IS MENTIONED IN
DOCUMENT, IT'S FIVE TIMES.
BUT SOME THEM ARE IN THE TITLES.
BUT I ALSO ENDEAVOR TO SEARCH
FOR TRUSTWORTHINESS.
AND IT'S MENTIONED NONE.
AND I THINK THAT IS A MAJOR,
MAJOR AREA.
BECAUSE IT'S NOT ONLY BUILDING
TRUST.
IT'S HOW DO WE AS
ORGANIZATIONS, INCLUDING NCATS,
OUR ACADEMIC HEALTH CENTERS,
HOW WE CAN BECOME A TRUSTWORTHY
ORGANIZATIONS.
THERE IS A LOT OF WORK BEING
DONE IN THAT AREA.
AND I THINK THAT IS MISSING.
AND I'M GOING TO STOP HERE.
>> Joni Rutter: FANTASTIC,
THANK YOU SO MUCH, SERGIO,
APPRECIATE YOUR THOROUGH
READING OF IT.
I HAVE TAKEN DOWN THOSE NOTES.
WE HAVE #2 AND #5 IS YOUR
FAVORITE SO FAR.
KEITH MUELLER, GO AHEAD.
>> MY FAVORITE IS #2.
SPEAKING ON BEHALF OF RURAL
POPULATIONS, THAT SPEAKS TO ME
VERY LOUDLY, THE A POPULATION
THAT IS NOT ALWAYS CONSIDERED.
AND WITHIN THAT, REALLY KIND OF
HONED IN ON OBJECTIVE 2.1 TO
WORK TOWARD BROADER INCLUSION
OF PATIENTS, FAMILIES AND
CAREGIVERS AS PARTICIPANTS IN
TRANSLATIONAL SCIENCE.
I THINK OF THAT, AS ONE OF THE
SISTER ORGANIZATIONS, SCIENCE
BEGINS WITH EVEN ASKING THE
QUESTION EVEN BEFORE THE STUDY
DESIGNS AND GOALS ARE WRITTEN.
SO THAT'S A CHALLENGE, I THINK
THAT IS INHERENT IN OBJECTIVE
ONE THAT DIDN'T COME RIGHT OUT
FROM THE LANGUAGE ON THE PAPER.
>> Joni Rutter: GREAT, THANK
YOU FOR THAT FEEDBACK.
THAT'S FANTASTIC.
OKAY, SO WE HAVE TWO FOR #2.
ANYONE ELSE?
>> I WILL DO A LITTLE LIKE
SERGIO AND PICK TWO.
I WILL SAY MY FAVORITE IS
PROBABLY #3.
I WOULD LIKE SPECIFICALLY
OBJECTIVES 3.1 AND 3.2.
I THINK THEY ARE VERY NCATS-IAN.
IF YOU THINK OF GENERALIZABLE
PROBLEMS.
#2.
WHICH IS MY SECOND FAVORITE
GOAL.
>> Joni Rutter: THAT'S A GREAT
COMMENT.
IN MANY WAYS MANY OF THESE
FEEDBACK ON ONE ANOTHER, WHICH
IS KIND OF THE BEAUTY OF IT AS
WELL.
ADVANCES IN ONE AREA WE HOPE
WILL PROVIDE ADVANCES IN
OTHERS, THAT'S WHAT WE EXPECT
TO SEE.
THANK YOU FOR THAT.
ANYONE ELTS?
-- ELSE?
KELLY McVEARRY.
S
>> KELLY: I LIKE THE WAY ONE,
TWO, AND THREE MIRROR WHAT YOU
HAVE CONSISTENTLY SAID, JONI,
WHEN YOU HAVE BEEN OUT THERE
ENGAGING COMMUNITIES.
I JUST LIKE THE WAY YOUR GOALS
LINEUP WITH YOUR CORE MESSAGE
AND HOW IMPORTANT, CONSISTENT
AND INCLUSIVE IT IS.
THIS IS REALLY TAKING THE
FUTURE MISSION OF NCATS, ARE
JUST AMPLIFYING A MESSAGE OF
INCLUSIVITY IN TRANSLATIONAL
SCIENCE AND I'M GRATEFUL TO SEE
THAT.
>> Joni Rutter: FANTASTIC.
THANK YOU.
OKAY.
SO NOW THAT WE HAVE YOUR
FAVORITES, I'M HEARING #2, I
THINK, IS WINNING.
AND IT SOUNDS LIKE THEY ARE ALL
REPRESENTED IN WHAT ARE TALKING
ABOUT.
PERHAPS GOAL #4 IS A LITTLE
LESS TANGIBLE BECAUSE IT'S SO
CROSS-CUTTING MAYBE THAT'S WHY
IT WASN'T NECESSARILY CALLED
OUT SPECIFICKLY.
BUT I THINK THAT MAKES A LOT OF
SENSE IN MANY WAYS.
IT'S IMPORTANT TO THAT COST TO
NATURE, I WILL TELL YOU WHEN I
WAS REALLY EXCITED WHEN I HAD
THE ENGAGEMENT WITH THE IC
DIRECTORS THEY WERE EXCITED
ABOUT GOAL #5 ADVANCING
TRANSLATIONING SCIENCES.
I HOPE WE CAN REALLY LIVE UP TO
THE PROMISES WE EXPECT
OURSELVES TO MAKE ON THESE
ACTIVITIES.
I LIKE TO SAY, THESE ARE BIG
GOALS.
AND WE ARE PROBABLY GOING TO
FAIL ON SOME ASPECTS OF THEM.
WE MAY NOT HIT THE MARK ON ALL
OF THEM.
BUT WE ARE GOING TO BE TRYING
FOR THEM.
AS LONG AS WE ARE TRYING FOR
THEM, THEY WILL BE MOVING
FORWARD, THAT MEANS WE WILL
FAIL FORWARD AND LEARN FROM
THAT AND REDIRECT AND HOPEFULLY
CONTINUE MAKING PROGRESS.
AND I THINK MATT HALL SAID THIS
IN HIS PRESENTATION TOO, WANT
TO TAKE THE MIND SET THAT WE
CAN FAIL AND WE CAN FAIL
FORWARD AND THAT'S THE WAY WE
WILL MAKE THOSE ADVANCES IN A
WAY THAT WILL BE VERY VALUABLE
TO THE COMMUNITY.
SO THANK YOU FOR THAT INPUT.
ARE THERE ANY OTHER COMMENTS
THAT YOU HAVE?
ANYTHING MISSING THE MARK?
OR ANY SPECIFIC GOALS YOU WANT
TO CALL OUT WHILE WE HAVE YOU
HERE FOR THIS DISCUSSION?
>> JONI, MAYBE I CAN ADD A
COUPLE THINGS WHILE FOLKS ARE
THINKING ABOUT THAT.
LIKE YOU, I'M NOT SURPRISED #4
WAS THE HARDEST FOR US TO TRY
TO FIGURE OUT HOW TO DO.
SO ONE OF THE THINGS I LOVE
BEING AT NCATS AND I WILL
DISCLOSE THIS IS MY SIXTH OR
SEVENTH IC AT NIH, I HAVE BEEN
IN SOME OTHER PLACES.
BUT I THINK IS HOW WE DON'T
SILO, RIGHT?
THAT'S SOMETHING WE ACTIVELY
WORK AGAINST.
SOMETHING WE HEARD FROM OUR
CONSTITUENTS.
SO IT'S REALLY NICE WHEN YOU
CAN HAVE A STRATEGIC PLAN THAT
MAPS PERFECTLY TO YOUR
ORGANIZATION, RIGHT?
AND WE WERE TRYING TO DO BOTH
THINGS, RIGHT IS THIS SEE HOW
WE DO THINGS AS A TEAM.
MATT SORT OF CHIMED IN ON THIS
IN HIS PRESENTATION ABOUT, HE
HAS GOT A STRUCTURE BUT THEN
PEOPLE CROSS THAT ALL THE TIME
TO SOLVE THE PROBLEMS THAT WE
ARE WORKING ON.
SO WE ARE TRYING TO REFLECT
THAT IN OUR PLAN.
WHICH MAKES IT HARD BECAUSE
THERE'S A DESIRE TO WANT TO PUT
THEM IN ONE PLACE AND THEY ONLY
LIVE IN THAT ONE PLACE OR THAT
ONE GOAL.
BUT WE DO SEE THIS AS A LIVING
BREATHING DOCUMENT WHERE THE
GOALS, ESPECIALLY THE
OBJECTIVES UNDER #4 WILL CROSS
CUT.
AND OTHER THINGS WILL TOO, WE
PICKED SOME PLACES, ESPECIALLY
IN GOALS 1-3 TO PUT THEM THERE
BECAUSE WE FELT THE MAJORITY OF
THE WORK WE ARE DOING OR
ENVISION DOING OVER THE NEXT
5-10 YEARS WOULD BE IN THAT
SPACE BUT NOT WITH THE THOUGHT
WE WOULD BE LIMIT TO OURSELVES
AND TO OUR COMMUNITIES IN THAT
WAY.
SO THAT WAS ONE POINT.
THE OTHER THING I WANTED TO
REITERATE I KNOW WE TALKED
ABOUT BEFORE WITH THE PLAN IS,
YOUR VIEW, JONI, AND MINE AS
WELL IS NOT SUPPOSED TO BE
SOMETHING THAT IS JUST PUT ON
THE SHELF, WE'RE DONE NOW, WE
ARE GOING TO RELEASE IT
PERMANENTLY AND WE ARE
TARGETING JULY, OKAY, YOU KNOW.
NO.
WE VERY MUCH ARE GOING TO PLAN
AS PART OF THE IMPLEMENTATION
TO BE EC WHICHING IN WITH
OURSELVES AND WITH YOU ALL HOW
WE ARE MAKING PROCESS.
THAT'S HOW TO JONI'S POINT WE
WILL KNOW IF WE ARE FAILING, IF
WE ARE MAKING THE MARK, IF WE
ARE NOT, AND WHAT WE ARE
LEARNING AT THE SAME TIME.
YOU WANT US TO KNOW THAT'S PART
OF THE GAME PLAN AS WELL.
>> Joni Rutter: OKAY, THANK YOU.
I SEE KELLY McVEARRY'S HAND IS
UP.
NOT SURE IF THAT WAS LEFT OVER.
>> NO.
>> Joni Rutter: SHE WAS VERY
CLEAR ABOUT HER NO.
AND SERGIO, PLEASE GO AHEAD.
>> SERGIO: KELLY, ARE YOU GOING
TO SHARE?
>> Joni Rutter: SHE SAID HER
COMMENT WAS FROM BEFORE.
>> SERGIO: OH.
WELL, THERE ARE SO MANY
POSITIVE THINGS IN STRATEGIC
PLAN.
I'M GOING TO ENDEAVOR TO READ
IT EVEN MORE CAREFULLY.
BUT ONE IMPRESSION THAT I GOT
BY READ IT THE WAY I DID, IN
THE QUESTION ARE ANY MISSING
THE MARK IS THAT I FELT THAT
THERE WAS NOT ENOUGH EMPHASIS
IN LOCAL COMMUNITIES, FOR
EXAMPLE, IN NEIGHBORHOODS.
AND YOU KNOW, THE FACTORS, FOR
EXAMPLE, THAT, YOU KNOW, ARE
CONTRIBUTE TO DISEASE.
KIND OF AN OPPORTUNITY TO
ASSESS AND INTERVENE IN THE
MANY FACTORS, ONCE AGAIN THAT
IMPACT HEALTH AND LEAD TO
DISPARITIES.
AND I THINK THIS IS IMPORTANT
IN TWO LIGHTS.
AND THAT'S THE OTHER PIECE THAT
I WANT TO BRING TO YOUR
ATTENTION.
I WAS POSITIVELY IMPRESSED WHEN
DR. BERTAGNOLLI, YOU KNOW,
JOINED US IN THE PREVIOUS.
I WAS IMPRESSED BECAUSE OF
SEVERAL REASONS.
ONE OF THEM IS HER BACKGROUND
AND EXPERIENCE.
LIVED EXPERIENCE ON COMMUNITY
ENGAGEMENT.
YOU KNOW.
IT WAS REALLY INSPIRING.
ONE OF THE THINGS SHE STARTED
WITH, THAT REALLY HAS RESONATED
WITH ME IS ABOUT THE BIG, BIG
PICTURE, WHAT IS GOING ON IN
THE U.S., DESPITE THE U.S.
INVESTING, WHAT 19% OF THE
GROSS DOMESTIC PRODUCT IN
HEALTH, THAT WE ARE NOT DOING
THAT WELL, COMPARED TO ANOTHER
NATIONS.
YOU KNOW, MORTALITY RATES,
MORBIDITY RATES.
SHE DECIDED TO SHARE THAT
INFORMATION WITH US.
BECAUSE OBVIOUSLY IT WEIGHS
IMPORTANTLY ON HER.
AND I WAS REMINDED ALSO OF WHAT
ROB GAELIS, FDA DIRECTOR NOW,
USE TO BE P.I. OF DUKE
UNIVERSITY, CTSA, THAT WHEN HE
PRESENTED TO US IN ONE OF THE
NATIONAL MEETINGS, IT WAS
EXACTLY THE SAME THING.
THE SAME TOPIC.
YOU KNOW.
OF ALL THE THINGS HE COULD HAVE
PRESENTED.
THOSE ARE BIG PICTURES THAT I
WOULD LOVE TO SEE THEM INCLUDED
IN THE STRATEGIC PLANNING, IN
SOME WAYS.
BIG PICTURES, BUT YOU KNOW, WE
ARE CONTRIBUTING TOWARDS THAT
AND WE COULD BE PART OF THE
SOLUTION FOR MEGATRENDS THAT
ARE HAPPENING IN THE U.S.
RELATED TO HEALTH, MORTALITY,
TRUNCATED LIFE EXPECTANCY, ET
CETERA.
>> Joni Rutter: THANK YOU VERY
MUCH, SERGIO, I COMPLETELY
AGREE, AND APPRECIATE YOUR
COMMENTS.
IT'S AN OPPORTUNITY FOR US.
WHEN WE DEVELOPED THE STRATEGIC
PLAN WE DIDN'T HAVE THE SORT OF
LUXURY OF HAVING DR.
BERTAGNOLLI'S VISION AT THE
TIME WE STARTED.
BUT WHAT WE NOW HAVE IS THE
OPPORTUNITY TO ENSURE WE ARE
INTEGRATING THAT IN OUR
THINKING.
I THINK THERE'S OPPORTUNITIES
FOR US TO DO THAT TO GET TO
YOUR POINT.
AND RECOGNIZING IT'S A BIG
PICTURE BUT CERTAINLY AS PART
OF WHAT WE ARE DOING IN
ADDRESSING HER VISION, YOU
KNOW, WORKING WITH HER CLOSELY
ON THAT, I THINK WE HAVE A VERY
GOOD OPPORTUNITY TO THINK ABOUT
HOW WE WORK MORE CLOSELY WITH
PRIMARY CARE NETWORKS IN
FEDERALLY QUALIFIED HEALTH
SYSTEMS AND HEALTH CENTERS.
AND HERE I SEE A REAL VALUE IN
THE COMMUNITY ADVISORY BOARD
MEMBER SHIP.
AND PERHAPS IN SOME WAY IS
EXTENDING SOME OF THE WORK THAT
THEY DO IN HELPING TO RAISE
VOICES AND HOW THEY WORK WITH
THEIR OWN COMMUNITIES WITHIN
THE CTSA PROGRAM.
I WOULD LOVE TO SEE WAYS IN
WHICH WE COULD REALLY ENHANCE
THAT.
I THINK THERE ARE A VARIETY OF
OPPORTUNITIES OF HOW WE CAN GET
THERE.
DID YOU SAY PERHAPS WHERE THAT
MIGHT, YOU THINK THAT IS
PROBABLY MOSTLY IN GOAL #2?
I THINK THAT MAKES A LOT OF
SENSE.
>> YES.
I'M THINKING ON GOAL #2.
ALONG WITH, ONCE AGAIN THE
EMPHASIS ON BUILDING TRUST AS,
YOU KNOW, WE ENGAGE WITH ALL
THAT CAN POTENTIALLY BENEFIT.
AND THAT ALSO, I THINK THAT IN
#5.
>> Joni Rutter: #5, YEAH.
>> SERGIO: WHICH IS AT THE
HEART OF WHAT WE ARE DOING.
THE TRANSLATIONAL WORK WE HAVE
BEEN ENGAGED WITH.
THOSE ARE THE PLACES.
AND I WILL CONTINUE TO SEND
SOME COMMENTS.
>> THAT WOULD BE GREAT.
>> Joni Rutter: THAT WOULD BE
GREAT.
ACTUALLY, IF YOU WOULD LIKE,
PLEASE ALSO, WE HAVE CERTAINLY
TAKEN A LOT OF NOTES.
I'VE WRITTEN DOWN A VARIETY OF
THINGS BUT IF YOU WOULD LIKE TO
ALSO SEND US A NOTE AS WELL TO
FOLLOW-UP, THAT WOULD BE
FANTASTIC.
PLUS YOU HAVE THE BENEFIT OF
HEARING THE CONVERSATION AND
HAVE UNTIL THE MIDDLE OF JUNE
UNTIL THEY ARE FINALIZED BEFORE
WE WRAP IT UP AND HOPEFULLY GET
THIS POLISHED AND THEN OUT TO
THE PUBLIC FOR THE FINAL
STRATEGIC PLAN.
>> AND THEN GETTING TO THE
STRATEGIC DOING.
>> Joni Rutter: THAT'S RIGHT.
STRATEGIC PLANNING IS IMPORTANT.
BUT I REALLY WANT TO GET TO THE
STRATEGIC DOING.
OKAY.
ANY OTHER COMMENTS BEFORE WE
END HERE?
YEAH, DR. MUELLER?
>> A COUPLE MORE COMMENTS ON
GOAL #2, OBJECTIVES #2 AND #3,
I GUESS IT IS.
THING THAT'S ARE BOTH EXCITING
AND INCREDIBLY CHALLENGING.
ENGAGING CONSTITUENTS THAT ARE
HARD TO REACH IN HARD TO REACH
COMMUNITIES, THAT'S IN
OBJECTIVE 2.2.
AND AN IMPORTANT APPROACH OF
USING DIGITAL HEALTH
TECHNOLOGIES TO SUPPORT
DECENTRALIZED TRIALS.
THE THIRD OBJECTIVE.
AND I I'M THINKING OF WORK WE
ARE DOING RIGHT NOW WITH OUR
NATIONAL ADVISORY COMMITTEE ON
RURAL HEALTH AND HUMAN SERVICES
OF THE USE OF TECHNOLOGY TO
OVERCOME SOME OF THE ACCESS
BARRIERS WE HAVE IN RURAL AND
UNDERSERVED COMMUNITIES.
AND I THINK ABOUT THE
DISCUSSIONS WE HAD HERE ABOUT A
PRIZE OF DEVELOPING THE RIGHT
TECHNOLOGY IN THE CONTEXT OF
EVERYTHING THAT NCATS DOES.
WE TALKED ABOUT THAT MODEL A
LITTLE BIT TO TRY TO GET
TECHNOLOGY THAT GOT RURAL
PATIENTS AND RURAL PERSONS MORE
ENGAGED WITH THE HEALTHCARE
DELIVERY SYSTEM.
BOTH APPLAUDING PUTTING THESE
THINGS IN THE OBJECTIVES AND
THROWING OUT THE BIG YELLOW
FLAG OF WHAT WILL BE DARNED
DIFFICULT.
A LOT OF US WILL BE RIGHT THERE
WITH YOU TO TRY TO GET THAT
ACCOMPLISHED.
>> Joni Rutter: THANK YOU FOR
THAT.
AND THANK YOU FOR RECOGNIZING
THE DIFFICULTIES OF THAT TOO,
THIS IS ALSO WHY WE WANTED TO
CO-DESIGN THIS.
WE NEED ALL OF YOUR HELP TO
MAKE IT AS SUCCESSFUL AS IT
POSSIBLY COULD BE.
SO IF YOU SEE YOURSELF IN THE
PLAN ITSELF, THAT WILL HELP US
ACCOMPLISH MORE TOGETHER.
AND YOU SHARE YOUR PASSION WITH
THE RURAL HEALTH AND THE RURAL
DELIVERY SYSTEMS TOO.
SINCE I SHARE MY ROOTS IN THE
RURAL COMMUNITY, SO I THINK
THAT'S INCREDIBLY IMPORTANT.
SO THANK YOU FOR THAT.
ANY OTHER COMMENTS FROM COUNCIL?
SERGIO?
>> SERGIO: JUST A VERY BRIEF
COMMENT TO THE EXCELLENT
COMMENTS THAT DR. MUELLER JUST
SHARED WITH US.
AND THAT IS, I USED TO USE
ALSO, FOR YEARS, I USED THE
HARD-TO-REACH POPULATIONS.
BUT I'M NOW CONVINCED THAT, AND
PREFER TO USE HARDLY REACHED
POPULATIONS.
IF WE USE HARD-TO-REACH, WE PUT
THE ONUS ON THOSE POPULATIONS,
WHEN THE ONUS IS ON US WHAT
EFFORTS CAN WE MAKE TO REALLY
GET TO THEM.
BECAUSE I'M THOROUGHLY
CONVINCED, ESPECIALLY DURING
THE PANDEMIC THAT WE CAN REACH
THEM.
IF WE JUST ENDEAVOR OURSELVES
AND PUTTING OUR RESOURCES TO
IT, I'M STOPPING NOW.
>> Joni Rutter: THANK YOU, I
WAS THINKING THE SAME THING.
IF WE HAVEN'T REACHED IT'S
BECAUSE PERHAPS WE HAVEN'T
TRIED TO REACH HARD ENOUGH.
I THINK THIS OFFERS US AN
OPPORTUNITY FOR BEING A LITTLE
MORE CREATIVE HOW WE THINK
ABOUT THOSE COMMUNITIES WE
THINK ARE HARD TO REACH.
THANK YOU FOR THAT.
I THINK WE ARE DEFINITELY
HAVING A MIND MELD THERE, I
APPRECIATE THE COMMENTS
BRINGING THAT UP TOO.
I TOOK JUST HIGH LEVEL THINKING
ABOUT TRUST BEING SORT OF, IT'S
CERTAINLY IN THIS DOCUMENT AND
INFUSED PERHAPS A LITTLE MORE
RECOGNITION AROUND BEING
TRUSTWORTHY TO THE COMMUNITY.
AND, THINKING ABOUT A LITTLE
MORE INTEREST ABOUT THE
IMPORTANCE OF THE LOCAL
COMMUNITY AND FOCUSING ON THE
SOLUTIONS WITH THOSE MORE LOCAL
COMMUNITIES.
INFUSING SOME OF THE IDEAS THAT
WE ARE HEARING A LITTLE BIT
MORE FROM THE NIH STRATEGIC
DIRECTION IN THINKING ABOUT THE
PRIMARY CARE DIRECTION AND
REALLY ENGAGING THAT PIECE OF
THE COMMUNITY, SO THAT WE DON'T
CONTINUE TO, EXPAND INEQUITIES
BUT REALLY ADDRESS THE
INEQUITIES SO WE ARE GEARING
MORE TOWARDS HEALTH EQUITY.
AND THEN THINKING ABOUT HOW THE
MORE QUICKLY SIDE OF THINGS IN
3.1 AND 3.2.
PAUL MENTIONED THOSE ARE VERY
NCATSIAN.
LINING UP WITH THE CORE MISSION
OF INCLUSIVITY AROUND ALL OF
THE DIFFERENT MESSAGES AND I
THINK THAT WAS KELLY'S COMMENT
ABOUT THIS SORT OF MORE
TREATMENTS FOR MORE PEOPLE MORE
QUICKLY, I THINK TO SAY THESE
GOALS QUICKLY.
BUT ALSO MESSAGING THE
INCLUSIVENESS THAT IT TAKES TO
DO IT, IT'S TIME THAT GENERATES
THE MOST PRODUCTIVE SCIENCE.
THE HARDLY REACHED POPULATIONS
WE SHOULD PROBABLY ADDRESS THAT.
THE USE OF TECHNOLOGY AND
ACCESS BARRIERS AND PERHAPS
LOOKING TO RURAL COMMUNITIES TO
HELP US ADDRESS AND ANSWER
THOSE CONCERNS FROM BOTH THE
HEALTH SIDE AND THE DELIVERY
SIDES.
SO THOSE ARE SOME HIGH LEVEL
COMMENTS BUT I KNOW WE TOUCHED
ON MORE.
I WANT TO REITERATE, WE ARE
OPEN STILL FOR INPUT ON THE
STRATEGIC PLAN FOR JUNE 14th.
PLEASE TELL ALL YOUR FRIENDS
AND FAMILY, IF YOU REALLY WANT
TO TELL YOUR FAMILY TO WEIGH
IN, THAT WOULD BE GREAT, THEY
ARE PART OF THE COMMUNITY.
WOULD APPRECIATE YOUR
ENGAGEMENT WITH YOUR COLLEAGUES
AND FRIENDS TOO.
SO WITH THAT, I THINK, IF THERE
ARE NO OTHER POINTS OF
DISCUSSION, WE CAN GO AHEAD AND
MOVE ON.
I BELIEVE THAT IS REALLY
WRAPPING UP TODAY.
AND WE ARE JUST A LITTLE OVER
FIVE MINUTES OVER.
SO WE DO HAVE ONE THING THAT'S
NOT ON OUR AGENDA TODAY THAT I
WANTED TO TURN IT OVER TO ANNA
TO INTRODUCE.
>> THANK YOU, JONI.
SO AS IS CASE FOR OPEN SESSIONS
THE FEDERAL ADVISORY COMMITTEE
MEETINGS WE DO HAVE AN
OPPORTUNITY TO HEAR FROM THE
PUBLIC, I WOULD LIKE TO EXTEND
AN OFFER TO A MEMBER OF THE
PUBLIC WHO HAS JOINED US TODAY,
SHE IS HEADING TO THE MIC, DR.
CATHERINE KREBS, WHO IS THE
PHYSICIANS COMMITTEE FOR
RESPONSIBLE MEDICINE.
>> HELLO.
HELLO, OKAY, HI, I'M CATHERINE
KREBS.
THANK YOU FOR OPPORTUNITY TO
PROVIDE INPUT.
THE COMMITTEE COMMENDS THE
SPECIFIC NON-ANIMAL RESEARCH AT
THE NIH, INCLUDING ONGOING
NCATS PROGRAM, NEWLY APPROVED
COMPLIMENTARY COMMON FUND
PROGRAM.
GREAT PROGRESS TO ADVANCE
WE WANT TO HIGHLIGHT THE
IMPORTANT ROLE THAT SCIENTIFIC
REVIEW PLAYS IN THE SUCCESSFUL
USE AND DEPLOYMENT OF NAMS AND
FEW MEASURES THAT CAN HELP
ENSURE NAMS ARE EVALUATED AND
SKRUKTIVE AND EQUITABLE MANNER.
LIKE SPECIFIC REVIEW CRITERIA
IN FUNDING OPPORTUNITY
ANNOUNCEMENTS.
BROADENING THE POOL OF NAM'S
EXPERTISE AVAILABLE FOR
SCIENTIFIC GROUPS.
TSR IS ALREADY WORK ON THIS BY
EXPANDING BIAS AWARE PS AND
MITIGATION TRAINING TO INCLUDE
INFORMATION AND ABOUT
SCIENTIFIC BIAS.
BROADER EFFORTS TO COMMUNICATE
THE VALUE OF TRANSLATIONAL
SCIENCE ARE LAID OUT IN THE
NCATS DRAFT AND STRATEGIC PLAN,
WE SUPPORT THIS GOAL AND
ENCOURAGE NCATS TO CLARIFY THE
RORNT ROLE OF SCIENTIFIC REVIEW
IN ADVANCING NAMS AND
TRANSLATIONAL SCIENCE IN THE
STRATEGIC PLAN.
LASTLY WE ENTHUSIASTICALLY
SUPPORT AND CAST EXEMPLARY
STRATEGIC PLANNING ACTIVITIES
WHICH INCLUDE OPEN AND
COMPREHENSIVE GATHERING OF
STAKEHOLDER INPUTS AND GROUND
PEOPLE DISCUSSIONS ON RFI ON
THE DRAFT STRATEGIC PLAN
FRAMEWORK AND RFI ON THE
STRATEGIC PLAN.
WE ENCOURAGE NCATS TO SHARE THE
SUCCESS OF THESE APPROACHES
WITH THE ICO'S AND THE NIH WIDE
STRATEGIC PLAN TEAM TO SIN
SPIRE SOME MORE EFFORTS.
>> THANK YOU.
>> Joni Rutter: WONDERFUL.
THANK YOU SO MUCH FOR THOSE
COMMENTS AND JOINING US HERE
TODAY.
SO CATHERINE, OOPS I DID THE
WRONG THING HERE.
I'M NOT SURE WHAT I DID.
SHE ENDED IT, SO I THINK WE ARE
OKAY.
I WANTED TO RECOGNIZE THE
PHYSICIANS COMMITTEE FOR
RESPONSIBLE MEDICINE.
THEY SENT US A LETTER PENNED BY
CATHERINE KREBS.
THEY SENT US A LETTER FOR
PUBLIC COMMENT.
AND BECAUSE IT IS FOR PUBLIC
COMMENT AND FOR OUR COUNCIL, I
WANT TO FIRST ALL THANK THEM
FOR THE COMMENT AND THE LETTER.
AND MAKE SURE THAT FOR THE
COUNCILMEMBERS FOR YOU ALL TO
SEE THE LETTER WE WILL BE
MAKING SURE YOU HAVE IT IN YOUR
INBOX, RELATIVELY SOON.
AS SOON AS COUNCIL IS OVER SO
YOU HAVE THAT AND THAT WILL
REFLECT THE LETTER IN THE
COMMENTS AND IN OUR MINUTES
FROM THE COUNCIL TODAY.
THANK YOU AGAIN.
AND WITH THAT, I BELIEVE WE CAN
ADJOURN.
AND SO I WILL GAVEL US OUT.
THANK YOU, AGAIN FOR A
SUCCESSFUL MEETING.
[ END 4:38 P.M. ET ]