Original subtitles

WELCOME TO THE 29TH MEETING OF

THE NCATS ADVISORY COUNCIL AND I

AM GOING TO SWITCH MY WEB CAM

FROM THE GAVEL TO ME SO

HOPEFULLY YOU CAN SEE ME NOW.

EARLIER THIS MORNING WE WERE

HAVING POSSIBILITIES OF SNOW

ICE, THAT SEEMS TO HAVE GONE

AWAY SO I THINK WE WILL BE IN

GOOD SHAPE GOING FORWARD.

THE MEETING TO SPAN A COUPLE OF

DAYS TODAY AND TOMORROW AND WE

HAVE A PACKED AGENDA, HOPE YOU

WILL FIND IT INTERESTING.

BEFORE WE START, ANNA I WILL

TURN IT TO YOU FOR THE OFFICIAL

ROLL CALL.

>> NEXT SLIDE PLEASE.

PAUL HARRIS.

>> HERE.

>> KRISTINA HARTMAN.

KRISTINA, ARE YOU THERE?

TED HOLEMAN.

>> GOOD MORNING.

REBECCA JACKSON.

>> HERE

>> ANNIE KENNEDY.

>> HERE.

>> MATTHIAS KRRETZLER.

>>ER HUE.

>> KELLY MCVEARRY.

>>ER HUE.

>> KEITH MUELLER

>> HERE.

>> RAJESH RANGANATHAN

>> HERE.

PAULA SHIREMAN.

>> HERE.

>> AND MARSHALL SUMMAR.

THOSE ARE OUR COUNCIL MEMBERS.

JONI, BACK TO YOU.

>> THANK YOU SO MUCH.

THIS IS THE AGENDA FOR TODAY'S

PORTION OF THE NCATS COUNCIL AND

WE HAVE A VARIETY AND

PROGRAMMATIC UPDATES AND CONCEPT

CLEARANCES TO I LOOK FORWARD TO

GOING THROUGH THOSE EVENTS WITH

YOU.

I THINK THEN WE'LL ADJOURN FOR

DAY ONE AND COME BACK TOMORROW

AT 1 P.M. EASTERN TIME AGAIN.

AND WE'LL HAVE A VARIETY OF

PRESENTATIONS FOR TOMORROW AS

WELL.

BEFORE WE START WITH DIRECTOR'S

REPORT I'LL TURN IT TO ANNA FOR

ANNOUNCEMENTS TO MAKE SURE WE

ARE ON THE SAME PAGE FOR VARIETY

OF KEY THINGS YOU NEED TO KNOW

ABOUT BEFORE WE START THE DAY.

ANNA BACK TO YOU.

>> THANK YOU, JONI.

EVERYONE IN ZOOM MEETING IS A

PANELIST TODAY SO YOU ARE FREE

TO SPEAK.

HOWEVER IN ORDER TO MAINTAIN A

SMOOTH FLOW WE REQUEST YOU USE

THE RAISED HAND FEATURE AND WAIT

UNTIL CALLED UPON TO PROVIDE

INPUT.

WE ASK YOU RESERVE USE OF THE

CHAT FOR ACTIVITIES SUCH AS

POSTING LINKS PERHAPS,

INFORMATION, BUT PLEASE USE THE

CHAT TO A MINIMUM.

PLEASE ALLOW SPEAKERS TO MOVE

THROUGH THEIR PRESENTATIONS,

NOTING YOUR COMMENTS AND THEN

PRESENTING THEM WHEN CALLED

UPON.

WE ARE USING THE VIDEOCAST

FEATURE TODAY, SO FOR THOSE OF

YOU WHO ARE NOT IN THE ZOOM

MEETING, YOU MAY SUBMIT COMMENTS

BY USING THE EMAIL FUNCTION

AVAILABLE ON THE NIH VIDEOCAST

SITE.

THERE IS A BUTTON THERE AND OR

YOU CAN SEND EMAIL DIRECTLY TO

THE ADDRESS LISTED IN THE SLIDE

NCATS COUNCILINPUT@MAIL.NIH.GOV.

SO THE FIRST THING I WOULD LIKE

TO DO IS CONSIDERATION OF OUR

MINUTES AND OUR NCATS GENERAL

COUNCIL OPERATING PROCEDURES.

WE ARE SEEKING APPROVAL OF THE

MINUTES FOR THE SEPTEMBER 21ST

MEETING, THEY ARE AVAILABLE IN

THE ELECTRONIC COUNCIL BOOK.

COULD WE HAVE A MOTION TO

APPROVE THE MINUTES FOR THE

SEPTEMBER 21ST MEETING?

>> SO MOVE.

>> THANK YOU.

SECOND?

>> SECOND.

>> ALL IN FAVOR?

>> A.

AYE.

AYE.

>> ANY OPPOSED?

ANY ABSTENTIONS?

THE MINUTES ARE APPROVED.

I WOULD ALSO NOW LIKE TO MOVE TO

SEEKING YOUR APPROVAL FOR

COUNCIL OPERATING PROCEDURES FOR

2022.

THESE ARE ALSO AVAILABLE IN YOUR

ELECTRONIC COUNCIL BOOK.

COULD WE HAVE A MOTION TO

APPROVE THE 2022 COUNCIL

OPERATING PROCEDURES?

>> SO MOVED.

>> HE COULD IS.

>>> SECOND.

>> ALL IN FAVOR.

>> A.

>> ANY OPPOSED?

ANY ABSTENTION?

WITH THAT OUR OPERATING

PROCEDURES FOR 2022 ARE APPROVED

APPROVED.

NEXT SLIDE PLEASE.

FUTURE DATES FOR OUR ADVISORY

COUNCIL MEETING IN 2022 WE PLAN

TO MEET MAY 19 AND SEPTEMBER

22ND FACE TO FACE AS OF NOW.

IN 2023 WE INTEND TO HAVE A

VIRTUAL MEETING ON JANUARY 26

AND 27 AS WE ARE TODAY.

AND TOMORROW AND HAVE FACE TO

FACE MEETINGS ON MAY 25 AND

SEPTEMBER 28TH.

I WOULD LIKE TO POINT OUT TINA

MORRISON FROM THE FDA ALSO

JOINED US TODAY ON BEHALF OF

FRANK WHITEHOLD.

WITH THAT, I WOULD LIKE TO TURN

IT BACK OVER THE JONI.

-- OVER TO JONI.

>> THANK YOU VERY MUCH.

I WILL GET START WITH THE

COUNCIL REPORT AND I HAVE TO SAY

A LOT HAPPENED SINCE WE LAST

MET, SEEMS FOUR MONTHS IS A LONG

TIME.

AND NOT LEAST OF WHICH OF COURSE

IS THAT DELTA HAS COME ON SCENE,

OFF SCENE AND NOW OMICRON IS NOW

THE WE WE ARE DEALING WITH, I

HOPE YOU ARE HANGING IN AND

DOING WELL DURING THIS TIME.

AS I GO THROUGH THE DIRECTOR'S

REPORT PLEASE FEEL FREE TO PUT

QUESTIONS AND COMMENTS IN THE

CHAT AS ANNA MENTIONED, OUR

DEPUTY DIRECTOR CLAIRE SCHMIDT

AND ANN MIGHT BE TAG TEAM WITH

COMMUNICATIONS EDUCATION, THEY

WILL BE MONITORING THE

DISCUSSION AND BE ABLE TO LOOK

AT THE CHAT AND THE RAISED HANDS

AND THEY WILL CALL ON YOU AT THE

END OF THE REPORT AND HOPEFULLY

HAVE GOOD AMOUNT OF TIME FOR

DISCUSSION.

PLEASE DO SO.

FIRST I WANT TO RELAY SOME OF

YOU MAY KNOW, NOT SURE IF

CHRISTINA WILL BE ABLE TO MAKE

IT TODAY, I HOPE SHE IS ON

TODAY, SOME MAY KNOW KRISTINA

HARTMAN IS NO LONGER AT THE

ASSISTANCE FUND.

THIS IS HER LAST OFFICIAL DAY ON

OUR COUNCIL.

SO WE ARE SAYING GOODBYE TO

KRISTINA AND WISH HER WELL IN

HER NEW POSITION AND HER NEW

POSITION PRECLUDES HER SERVICE

ON COUNCIL SO I KNOW THOUGH THAT

WE WILL CONTINUE TO WORK WITH

KRISTINA IN A VARIETY OF OTHER

CAPACITIES.

I KNOW SHE WILL CONTINUE TO

CONTINUE TO GIVE TO THE

SCIENTIFIC IMMUNITY, WE LOOK

FORWARD TO WORKING WITH YOU AND

YOUR NEW ROLE.

>> THANK YOU, JONI.

>> GLAD YOU MADE IT.

NICE TO SEE YOU.

SEVERAL NIH TRANSITION

LEADERSHIP TOO.

DR. COLLINS STEPPED DOWN AS

DIRECTOR OF THE NIH.

HE WAS THE LONGEST SERVING NIH

DIRECTOR AND SERVED THREE

PRESIDENTS.

AND IN CASE YOU MISSED IT, THERE

WAS A WONDERFUL TRIBUTE TO DR.

COLLINS AND THE LINK IS SHOWN

HERE BELOW.

THERE WERE VARIETY OF FAIR WELL

MESSAGES FROM LUMINARY

SCIENTISTS, LIKE JANE GOODALL TO

PRESIDENTS AND VICE PRESIDENTS

AND POLITICIANS TO CO-MEDIANS

AND ACTORS AND MUSICIANS.

SO IT WAS REALLY A WONDERFUL

EVENT SO IF YOU HAVEN'T HAD A

CHANCE PLEASE TAKE A LOOK.

AND AS DR. COLLINS WAS GOING

THROUGH THIS FAIR WELL TOUR HE

ALSO CONDUCTED AN INTERVIEW

PUBLISHED IN JAMA, HE WAS ASKED

ABOUT HIS TOP THREE SCIENTIFIC

ACCOMPLISHMENTS.

HIS RESPONSE INCLUDED THE

FOUNDING OF NCATS AMONG THE TOP

THREE ACCOMPLISHMENTS. I

CERTAINLY COULDN'T AGREE MORE,

COULDN'T BE MORE PROUD OF THAT.

WITH THE VISION OF HIS VISION

FORMING NCATS AND THEN CHRIS

AUSTIN'S LEADERSHIP THE LAST TEN

YEARS IT IS BUILT NCATS FROM

GROUND UP AND NOW IT HAS COME

INTO ITS OWN.

IT IS I THINK EXCITING FOR US TO

BE AND I HOPE YOU ARE AS EXCITED

AS I AM TO CONSIDER THE NEXT

DECADE OF WHAT NCATS IS DOING

FOR TRANSLATION SCIENCE AND RARE

DISEASE RESEARCH AS WELL.

SO I WANTED TO DEFINITELY CALL

THAT OUT FOR YOU TODAY.

THE POSITION OF DIRECTOR IS

PRESIDENTIALLY APPOINTED AND

ALSO SENATE CONFIRMED.

THIS PROCESS IS WHOLLY OWNED BY

THE WHITE HOUSE.

NIH IS NOT INVOLVED AT ALL SO WE

WON'T HAVE ANY EXTRA RUMORS OR

ANY IDEAS FOR THAT MATTER.

NO CANDIDATES TO DATE HAVE BEEN

PUT FORWARD BUT IN THE MEANTIME,

THE PRESIDENT HAS ASKED DR.

LARRY TABAK TO BE ACTING NIH

DIRECTOR AND HE SERVED AS THE

NIH PRINCIPLE DEPUTY DIRECTOR

WITH DR. COLLINS.

ALONG WITH THAT DR. TARA SCHWETZ

HAS TAKEN HIS PLACE AS ACTING

NIH PRINCIPLE DEPUTY DIRECTOR.

MORE TO COME AS WE HEAR NEWS AND

WE WILL HEAR ABOUT IT AT THE

SAME TIME SO STAY TUNED.

OPEN SEARCHES, THE NCATS

DIRECTOR POSITION HAS BEEN

POSTED SINCE WE LAST MET.

THIS ONE DOES NOT REQUIRE A

PRESIDENTIAL APPOINTMENT OR

SENATE CONFIRMATION.

BUT SIMILAR TO THE NIH DIRECTOR

POSITION THIS ONE IS ALSO NOT

WHOLLY OWNED BY NCATS.

THE SEARCH FOR THIS POSITION IS

CONDUCTED BY THE NIH DIRECTORS

OFFICE.

THIS IS THE ANNOUNCEMENT ON THE

SCREEN, IT APPEARED IN A NUMBER

OF VENUES. IT IS OPEN UNTIL THE

END OF THIS MONTH, JANUARY 31ST

AND SO PLEASE HELP SPREAD THE

WORD AND WITH ANY LUCK WE'LL

HAVE AN UPDATE IN MAY I HOPE ON

THIS PROCESS AS WELCH FEW OTHER

NCATS POSITIONS UNDERGOING

TRANSITION THAT I WANT TO TELL

YOU ABOUT.

THE FIRST IS DR. ANTON HE'S BEEN

IN THE ROLE FOR SEVEN YEARS AND

HE DECIDED TO STEP DOWN TO FOCUS

ON OTHER HATS HE WEARS IN THE N

CATS LABS IN THE DIVISION OF

PRE-CLINICAL INNOVATION.

HE'S LOOKING FORWARD TO DEVOTING

THE BULK OF TIME AT CHEMICAL

GENOMICS BRANCH.

WHERE HE'S BRANCH CHIEF OF THAT

MANY THE N CATS LABS.

BUT IN THE MEANTIME ANTON HAS

GRACIOUSLY AGREED TO STAY ON AS

SCIENTIFIC DIRECTOR UNTIL WE

CONCLUDE A SEARCH.

WITH THE NCATS DIRECTOR POSITION

MOVING FORWARD IN PARALLEL WHICH

HOPE TO BEGIN THE NATIONWIDE

SEARCH FOR THE NEXT SCIENTIFIC

DIRECTOR FOLLOWING THE, IN CATS

SEARCH BY A FEW MONTHS.

HOPEFULLY NOT TOO MUCH TIME.

I I'M GRATEFUL WE WILL CONTINUE

TO WORK WITH ANTON IN HIS ROLE

AND HE'S BEEN REALLY A TERRIFIC

PARTNER IN THINKING ABOUT HOW TO

POSITION NCATS LABS GOING

FORWARD.

WE REMAIN IN GOOD HANDS AND

CONGRATULATE ANTON FOR ALL THE

WORK HE CONDUCTED MAKING THE

NCATS LABS WHAT IT IS NOW,

REALLY SUPERMEN SUPER.

I CAN'T BLAME HIM FOR WANTING TO

BACK TO THE LAB EITHER.

WAY TO GO,ANTON.

THANK YOU.

LASTLY, COURTROOM ANN PARISER IS

RETIRING, I ASKED HER TO PRESENT

TO COUNCIL, YOU WILL HEAR HER

PRESENTATION TOMORROW.

I ASKED HER TO PRESENT BECAUSE

THERE IS INCREDIBLE PROGRESS SHE

LED FOR OFFICE OF RARE DISEASE

RESEARCH OVER LAST FIVE YEARS,

SHE CAME TO NCATS FROM THE FDA

TO LEAD THIS OFFICE.

AND SINCE SHE ARRIVED SHE

EXPANDED THE OFFICE SHE'S LED

WORK TO DESCRIBE AND UNDERSTAND

THE BURDEN OF RARE DISEASES.

AND SHE'S DEVELOPED INCREDIBLE

RELATIONSHIPS WITH W IN THE AS

WELL AS ACROSS THE RARE DISEASE

BIOMEDICAL SPACE TO DRIVE ALL

AREAS AND MOST RECENTLY LED THE

WORK FOR NCATS ORPHAN DRUG

STATUS FOR ONE OF THE PAT

TRIALS.

SO WE ARE GOING TO MISS ANN'S

LEADERSHIP AND PASSION FOR RARE

DISEASE RESEARCH SO WE WISH HER

WELL IN RETIREMENT TOO.

DR. PJ BROOKS IS WITH ANN ALONG

THE WAY AND I HAVE ASKED HIM TO

STEP IN AS ACTING DIRECTOR UNTIL

A SEARCH IS COMPLETE.

SO THEY WILL TAG TEAM TOMORROW

FOR THEIR PRESENTATION AS SORT

OF A WARM HAND OFF.

AS WITH THE SCIENTIFIC DIRECTOR

TRANSITION THAT I MENTIONED

BEFORE, THERE WILL BE A SEARCH

FOR NEW DIRECTOR FOR OFFICE OF

RARE DISEASE RESEARCH AS WELL.

THAT WILL COMMENCE RELATIVELY --

AT SIMILAR TIME AS WE CONDUCT

THESE OTHER SEARCHES.

IN THE MEANTIME, WE WELCOME PJ

IN THIS ACTING ROLE AND WE WON'T

SKIP A BEAT WITH HIS LEADERSHIP

THERE.

SO THAT WAS A LOT OF PERSONNEL

CHANGES AND THERE'S A LOT GOING

ON WITH OTHER ANNOUNCEMENTS AND

EVENTS I WILL RELAY.

ALSO VERY EXCITING BECAUSE ONE

IS TO REALLY RECOGNIZE AND

CONGRATULATE OUR LEADERS IN THE

NCATS AND CTSA COMMUNITY.

SHERRY BURKIN ANDREA GARCIA, KEN

MANDL AND LIZ MCNALLY AND KEITH

NORRIS, AND MARCELLA SMITH, IT

UNDERSCORES THE IMPORTANT WORK

WE DO SO CONGRATULATIONS TO ALL

THESE FOLKS.

IN THE SAME VEIN, COLLEEN KELLY,

MEGAN SRINIVAS AND CH ARKANSAS

YANG ARE ALSO RECOGNIZED FOR

THEIR WORK WHAT THEIR ARE DOING

NOW, SOME SCHOLARS AND TRAININGS

AND DR. CHAO YANG IS IN

COMMUNITY ENGAGEMENT, SELECTED

AS PEOPLE, 40 PEOPLE WITHIN

THEIR CATEGORIES WHO ARE UNDER

40.

SO CONGRATULATIONS TO THEM, I

THINK WE ARE DEFINITELY IN GOOD

HANDS GOING FORWARD.

RARE DISEASE DAY IS FEBRUARY 28,

THE LINK AT THE BOTTOM TO

REGISTER IF YOU HAVEN'T DONE SO

ALREADY.

IT IS REALLY A PACKED AGENDA,

ADDRESSING DIVERSITY IN RARE

DISEASE, RESEARCH INEQUITY OF

CARE, IT IS ABOUT PERSONALIZED

MEDICINE IMPORTANCE OF ADVOCACY

AND COLLABORATIONS.

NATURAL HISTORY DATA COLLECTION.

FOR GENE THERAPY TRIALS.

THE USE OF TELEHEALTH AND

EFFORTS ON ADDRESSING THE

DIAGNOSTIC ODYSSEY, THIS IS

REALLY A I THINK A VERY EXCITING

AGENDA WITH MEATY TOPICS REALLY

PRIMING RELEVANT FOR WHAT WE DO

SO HOPE YOU CAN JOIN US I

GUARANTEE YOU THAT THIS WOULD BE

ONE OF THE BEST MEETINGS YOU

ATTEND ALL YEAR SO MAKE IT IF

YOU CAN OR ALERT CUR COLLEAGUES

AS WELL.

SO FINALLY WE HAVE TALKED ABOUT

ARPAH IN PREVIOUS MEETINGS.

AND SO THIS ROUND I DECIDED DR.

SCHWETZ IS NOW BACK TO NIH SO I

ASKED TARA TO JOIN US TO GIVE

UPDATE ON TOPIC OF ARPA H.

SHE'S ACTING PRINCIPAL DEPUTY

DIRECTOR BUT THE LAST SIX MONTHS

SHE'S BEEN DETAILED TO THE

OFFICE OF SCIENCE TECHNOLOGY AND

POLICY AT OSTP.

SHE'S WORKING VERY CLOSELY WITH

DERRICK LANDER ON PLANNING ARPA

H SO I WILL LET HER UPDATE.

I DON'T WANT TO STEAL HER

THUNDER.

SHE WILL FOLLOW ME DIRECTLY.

WAR PA THAT WILL BE THE NEXT TALK.

TO ROUND OUT I HAVE A FEW

UPDATES ON DIVERSITY AND EQUITY

AND INCLUSION OR DEI EFFORT AT

NCATS AND THE NIH.

THE NIH IS HOLDING A SERIES OF

LISTENING SESSIONS AND I HAPPEN

TO HOLD ONE LAST WEEK FOR THE

HEALTH CENTERS AN SYSTEMS, AND

THERE'S ALSO A FEW COMING UP SO

I PUT THOSE DATES HERE SO YOU

CAN SEE THEM BUT THERE IS A LINK

AT THE BOTTOM THAT YOU CAN CHECK

INTO TO SEE OTHER ACTIVITIES

THAT ARE GOING ON IN THIS SPACE

AND REGISTER FOR ONE OF THESE

ACTIVITIES IF YOU ARE ABLE TO.

ENCOURAGE YOU TO JOIN OR SEND TO

COLLEAGUES IF THEY ARE

INTERESTED AS WELL.

IN ADDITION TO THE UNITE EFFORTS

WE ARE DOING AT NCATS WE

ESTABLISHED AN INTERNAL DEIA

HEALTH DISPARITIES GROUP TO

BUILD AND SUSTAIN A WELCOMING

AND INCLUSIVE ENVIRONMENT TO

MAKE SURE THAT OUR PROCESSES AND

OPERATIONS ARE ALL INCLUSIVE OF

THAT HAVE THINKING AND JUST -- I

GUESS IT WAS OVER THE SUMMER

PROBABLY NOW WE ARE ALSO MAKING

SURE THESE ARE IDEAS AND TOPICS

THAT ARE DISCUSSED AT MEETINGS

AND CONFERENCES THAT WE SUPPORT.

ANY TOPICS THAT CAN COVER THE

SCIENCE NEEDS TO ALSO ADDRESS

HEALTH EQUITY IF THERE IS AN

OPPORTUNITY TO DO SO.

WE ENCOURAGE TO HAPPEN IN THEK

SCIENTIFIC OFFICE AS WELL.

ANOTHER CTSA INVESTIGATOR

PUBLISHED A PIECE ABOUT DEI IN

CLINICAL TRANSITIONOR RATIONAL

RESEARCH AND THIS PAPER I DON'T

HAVE A LINK BUT I DID PUT THE

TITLE ON THE SLIDE HERE.

THIS PAPER LAYS OUT A PATH

FORWARD AND IT ALSO REPRESENTS I

THINK A DEEPER EFFORT WITHIN THE

STEERING COMMITTEE AS WELL AS

CTSA COMMUNITY AS LARGE.

THERE IS NOW A DEI TASK FORCE

WITHIN THE STEERING COMMITTEE OF

THE CTSA PROGRAM.

A LOVE OF THE EFFORTS WITH THAT

TASK FORCE BUT ALSO PRIOR TO THE

DEVELOPMENT OF THAT TASK FORCE

THERE'S BEING DISCUSSIONS WITH

CTSA PROGRAM ABOUT THIS.

THIS PARTICULAR PAPER I THINK IS

A CULMINATION OF LOT OF THOSE

DISCUSSIONS THAT TALKS ABOUT

LEADERSHIP AND TRAINING AND

RESEARCH AND CLINICAL TRIALS AND

PROVIDES CONCRETE GOALS THAT

TRANSFORM AND BUILD CAPACITY AS

WELL AS BUILD TRUST.

SO I'M COMMITTED TO TRANSLATING

THESE SORTS OF EFFORTS INTO

MEASURABLE ACTION AS WE GO

FORWARD.

I WANT TO SPEND A FEW SECONDS ON

THE BUDGETS NOT MUCH TO RELAY

SINCE THE LAST MEETING

UNFORTUNATELY.

THE CONTINUING RESOLUTION THAT

WAS TO DECEMBER WAS REUPPED IF

YOU WILL, WE HAVE A CONTINUING

RESOLUTION THROUGH FEBRUARY 18.

JUST REMINDER THAT THE FULL

HOUSE VOTED AND APPROVED THEIR

APPROPRIATIONS BILLS BACK IN

JULY AND THE SENATE -- THEY

COULDN'T AGREE ON THE TOP LINE

APPROPRIATION NUMBERS BUT

MAJORITY RELEASED DRAFT BILLS IN

REPORTS FOR PUBLIC AWARENESS ON

I THINK THAT WAS -- YEAH,

OCTOBER 18.

THESE BILLS HAVEN'T BEEN

FORMALLY INTRODUCED.

WE ARE HOPEFUL OVER THE NEXT

COMING WEEKS WE WILL START TO

SEE THAT MOVEMENT AND FEBRUARY

IS GOING TO BE A CONFUSING

MONTH, NOT ONLY IS THAT

HAPPENING BUT ALSO THE

PRESIDENT'S BUDGET COMES OUT

EVERY FEBRUARY SO THE FY 23

BUDGET IS ANTICIPATED TO BE

RELEASED IN EARLY FEBRUARY AS

WELL.

SO JUST STAY TUNED TO WHAT'S

HAPPENING IN TERMS OF THE

CONTINUING RESOLUTION AS WELL AS

THE FY 22 BUDGET.

I'M ALSO TAKING A MOMENT TO

HIGHLIGHT SOME OF THE RESEARCH

FUNDING OPPORTUNITIES THAT ARE

CURRENTLY ON THE STREET.

I WON'T WALK THROUGH ALL OF

THESE BUT I WANTED YOU TO HAVE

THIS LIST SO YOU WOULD BE ABLE

TO LOOK AT THE DIFFERENT FUNDING

ANNOUNCEMENT OPPORTUNITIES AND

THE TITLES, IT REALLY HIGHLIGHTS

THE BREADTH OF ACTIVITIES THAT

WE ARE INVOLVED IN AND HOPE YOU

AND COLLEAGUES CAN SPREAD THE

WORD OR APPLY TO THESE

ACTIVITIES.

SO I WANT TO MOVE ON TO THE MORE

SCIENCE PORTION OF MY REPORT AND

WILL IS A LOT TO RELAY HERE BUT

WANT TO GIVE A HEADS UP ON OTHER

THINGS THAT WILL BE GIVING YOU

UPDATES ON AS WE GO FORWARD.

VALERIE GORDON WILL TALK ABOUT

INCLUSION MONITORING REPORTS.

THIS IS A REQUIREMENT FOR US TO

RELAY TO YOU ON A, IT IS NOT A

ROUTINE BASIS BUT IT IS A

ROUTINE BASIS BUT NOT FREQUENT

BASIS.

SO SHE WILL TELL YOU ABOUT THAT

REPORT AND WHY IT IS IMPORTANT

THAT WE GET YOUR INPUT ON THAT.

THEN AFTER VALERIE GOES LILY

PORTILLA FROM OFFICE OF

STRATEGIC ALLIANCE WHO WILL

UPDATE ON ACTIVITIES IN HER

OFFICE AND THEN AS I MENTIONED

ANN AND PJ WILL BE TALKING ABOUT

ACTIVITIES AROUND RARE DISEASE

SPACE TOO.

FINALLY KEITH WILL BE TALKING

ABOUT A NEW APPROACH WE ARE TAKE

FOGGER OUR ORGANIZATION OF

NCATS, THAT IS ALSO SOMETHING WE

NEED TO RELAY SO HE WILL TELL

YOU ABOUT THAT MORE TOWARDS THE

END.

NOW I WANT TO DIVE INTO THE

RESEARCH EFFORTS WE HAVE BEEN

DOING.

BEFORE I I SHOULD SAY BECAUSE OF

THE NATURE OF NCATS OUR TEAM

BASED APPROACH, OUR FOCUS ON

PLATFORM TECHNOLOGIES, APPROACH

ON RARE DISEASE AUTOMATION AND

ROBOTICS ACTIVITIES EMPHASIS ON

DATA SCIENCE AND OUR -- ALL HE

IS LEAD TO OUR ABILITY TO

RESPOND TO PUBLIC HEALTH

EMERGENCIES, AND GET INVOLVED IN

VARIETY OF DISEASE RELATED

ACTIVES BUT CERTAINLY OVER THE

LAST COUPLE OF YEARS IT HAS BEEN

FOCUSED ON THIS -- THE COVID

PUBLIC HEALTH EMERGENCY.

ALL OF WHAT WE CAN BRING TO THE

TABLE ARE QUINTESSENTIALLY NCATS

SO I WILL GIVE YOU A SLIVER OF

THE LARGER PIE OF ACTIVITIES WE

DO NOW BECAUSE OF THE COVID

RELATIONSHIP TO THOSE ACTIVITIES

BUT IT DOES JUST REPRESENT A

PIECE SLIVER OF WHAT WE ARE

DOING.

I WILL TELL YOU PROGRESS ON THE

COVID WORK -- COVERING A VARIETY

OF AREAS BUT WILL START WITH THE

CLINICAL TRIALS AND THEN MOVE

INTO THE ANTIVIRAL PROGRAM ON

PANDEMICS AND DIVE INTO OUR

ELECTRONIC HEALTH RECORD

NATIONAL COVID COHORT

COLLABORATIVE ACTIVITIES.

THEN AT THE END OF THE TALK JUST

GIVE YOU A FAIR WARNING, AT THE

END OF THE TALK I WANT TO SWITCH

GEARS THE LAST TEN OR SO MINUTES

TO WALK THROUGH SOME HIGH LEVEL

GOALS AND I WANT TO TACKLE OVER

THE NEXT DECADE AND WITH WHAT I

PRESENT TODAY I HOPE IT SERVE AS

BROAD RANGE OF SCIENTIFIC

PURSUIT AND THE GOALS OUTLINED

EMBRACE THAT APPROACH AND WILL

SERVE TO HELP DRIVE THAT SUCCESS

AS WE GO FORWARD.

SO FIRST UP OF COURSE IS THE --

I MENTION CLINICAL TRIALS.

THE CTSA PROGRAM AGAIN HAS BEEN

DOING THE LION'S SHARE OF THE

COVID CLINICAL TRIAL WORK

THROUGH THE ACTIVE PROGRAM THAT

WE HAVE THROUGH THE FNIH AS WELL

AS THE NIH AND VARIETY OF

PRIVATE PARTNERS.

WITH THIS PUBLIC PRIVATE PARTNER

FRAMEWORK WE HAVE -- WE HAVE

DONE A VARIETY OF CLINICAL

TRIALS AND SOME OF THEM HAVE

BEEN NCATS WHOLLY SUPPORTED LIKE

THE CONVALESCENT PLASMA TRIALS

BUT SOME THROUGH THE PUBLIC

PRIVATE PARTNERSHIP THAT NCATS

HAS SPONSORED.

MOST MONEY COMING ARE NOT NCATS

DOLLARS, THESE ARE DOLLARS

(INAUDIBLE) RESPONSE.

I'M GOING TO TALK ABOUT THE

CONVALESCENT PLASMA TRIALS,

THERE'S EARLY WORK COMING OUT, A

BIGGER STORY TO TELL THAT WE

WILL HEAR MORE OVER THE NEXT

COMING MONTHS BUT THERE'S ONE

REPORT OUT THAT I CAN TALK ABOUT

RELATED TO THE CONTAIN TRIAL.

I'M TALKING ABOUT SOME OF THE

OTHER ACTIVITIES TOO.

TESTIFY CONTAIN STUDY IS LOOKING

AT EFFICACY OF COVID-19

CONVALESCENT PLASMA IN

HOSPITALIZED PATIENTS.

THIS IS OUT OF THE NEW YORK

UNIVERSITY AND CTSA HUBS AND

AFFILIATES WITH THEM.

AND THE TOP LINE IS THAT

CONVALESCENT PLASMA DID NOT MEET

THE PRE-SPECIFIED CRITERIA FOR

EFFICACY IN THIS STUDY.

THEY WERE USING THE HUE SCORE

FOR SEVERITY, 11 POINT SCALE

WHERE 0 IS ASYMPTOMATIC AND 10

IS DEATH.

AND THIS GRAPH HERE SHOWS

THERE'S ODDS RATIOS POST TIER

YEAR PROBABILITY ANALYSIS SO

THAT RED LINE THERE IN THE

MIDDLE IF YOU ARE TO THE LEFT OF

THAT LINE THAT SHOWS A LITTLE

BIT MORE BENEFIT, BUT FOR THIS

THERE'S NO SIGNIFICANT BENEFIT

THAT IS INDICATED IN GENERAL

WITH CONVALESCENT PLASMA.

BUT WHAT WAS INTERESTING IS WHAT

THEY FOUND WHEN THEY DID

SUBGROUP ANALYSIS IS THAT EARLY

IN THE PANDEMIC WHEN THERE WERE

NO TREATMENTS, IT SHOWED SOME

EFFECTIVENESS IN THAT SUBGROUP

ANALYSIS.

HOWEVER, AS REMDESIVIR AND

CORTICAL STEROIDS AND OTHER

TYPES OF TREATMENT VACCINES ARE

ON THE SCENE, IT HAS -- IT HAS

WANED IN THAT BENEFIT SO THERE'S

NO LONGER REALLY A BENEFIT FOR

CONVALESCENT PLASMA THAT WE ARE

SEEING NOW.

THE CONVALESCENT PLASMA STORY

ISN'T DONE YET SO KEEP YOUR EYE

OUT FOR OTHER REPORTS COMING

SOON FROM THE WORK WE SUPPORTED

AS WELL.

AS WELL AS OTHERS.

ACTIVE ONE ENDED ENROLLMENT WITH

THE LAST PATIENT FOLLOW-UPS THAT

WILL HAPPEN IN FEBRUARY AND

MARCH.

THIS ACTIVE ONE STUDY LED BY

BILL WASHINGTON UNIVERSITY ST.

LOUIS CTSA ALONG WITH 30 OTHER

CTSA SITES INVOLVED WITH OTHER

SITES TOO.

THIS IS A DOUBLE BLIND PLACEBO

CONTROL TRIAL AND HOSPITALIZED

PATIENTS WITH RANDOMIZED ACROSS

THREE DIFFERENT

IMMUNOMODULATORS.

SENATE P -- SENEPIVEROC ENDED

EARLY BUT TOP LINE RESULTS DID

COMPLETE ENROLLMENT AND WE WILL

BE HEARING MORE ABOUT THESE

AROUND MAY OR SO WHEN WE GOT TO

GET THAT DATA AND ANALYSIS

COMPLETED.

STAY TUNED IN MAY, HOPEFULLY

WE'LL UPDATE THEN.

ACTIVE 6 IS LED BY DUKE AND DCRI

AS WELL AS VANDERBILT AS

COORDINATING CENTER.

IT IS AN OUTPATIENT TRIAL TO

TEST EXISTING DRUGS APPROVED FOR

OTHER INDICATIONS TO SEE IF THEY

WERE BE REPURPOSED FOR COVID-19

SYMPTOM RELIEF.

THIS PARTICULAR CLINICAL TRIAL

IS LOOKING AT THREE MAIN DRUGS,

IVERMECTIN AND FLUVOXAMINE.

WE ARE SENDING THESE MEDICATIONS

TO PATIENT HOME AND DO FOLLOW-UP

ONLINE OR THROUGH TELEPHONE

SURVEYS SO IT IS A UNIQUE TRIAL

WITHIN THE ACTIVE PARTNERSHIP.

BUT IT IS GOING VERY WELL AND

TARGETED ENROLLMENT SHOULD BE

MET BY END OF MAY, SORRY BY END

OF I GUESS I'LL SAY SPRING,

PROBABLY MARCH, APRIL MORE

LIKELY.

AND HOPEFULLY WE'LL BE ABLE TO

GIVE MORE DETAILS ON THIS

ACTIVITY IN OUR MAY COUNCIL.

WE ARE ALSO RAMPING UP OUR

ANTIVIRAL PROGRAM FOR PANDEMICS.

THIS IS A COLLABORATION WITH M

CATS, NIAID, AND BARTA AND THIS

IS TO CREATE PHASE 2 TRIAL READY

ANTIVIRALS FOR RAPID PANDEMIC

RESPONSE SO THEY WILL GET

THROUGH CERTAINLY AT LEAST PHASE

2 AND BE READY OR GET THROUGH

PHASE 1 AND BE READY FOR PHASE 2

IN CASE THERE IS ANOTHER NEED

FOR A PANDEMIC RESPONSE.

WE HAVE DONE SURVEY OF VIRAL

FAMILIES AND ESSENTIALLY SEVEN

VIRAL FAMILIES THOUGHT TO BE OF

THE HIGHEST FOR PANDEMIC

POTENTIAL.

SO THOSE ARE SHOWN HERE ON THE

LEFT.

GIVEN WE ARE CURRENTLY IN THE

PANDEMIC OF THE CORONA VIRUS

FAMILY, MOST OF THE WORK WE DO

CURRENTLY IS RELATED TO CORONA

VIRUS.

SOME OF THE PROJECTS DOING TO

DATE, ARE LISTED HERE WITH CLEAR

CREEK BIO COLUMBIA UNIVERSITY

AND NCATS SUPPORTING THESE

ACTIVITIES AS WELL.

WE ARE LOOKING AT THINGS LIKE

PROTEASE INHIBITORS TO STOP

VIRAL REPLICATION WAY TO STOP

VIRAL REPLICATION AND

INFECTIVITY.

WE ARE ALSO LOOKING AT COUPLE OF

ANALOGS RNA DEPENDENT RNA

POLYMERASES, AND THESE SORTS OF

INHIBITORS TO ALSO HELP PREVENT

VIRAL REPLICATION.

SO THESE ARE SOME OF THE

PROJECTS IN THE WAY IN OUR NCATS

LABS IS LEADING THIS EFFORT FOR

NCATS.

WE HAVE A GREAT TEAM PUSHING

REALLY A LOT OF HE IS SORTS OF

ACTIVITIES -- THESE SORTS OF

ACTIVITIES AND WE ARE OPEN FOR

BUSINESS SO ANY IDEAS ALONG THE

WAY, WE ARE HAPPY TO HEAR ABOUT

THEM.

EXCITING STUFF AS THIS IS

GETTING OFF THE GROUND.

SO I WANT TO ALSO JUST SHOW YOU

SOME OTHER ACTIVITIES THAT WE

ARE DOING IN TERMS OF MAKING

SURE DATA ARE READILY ACCESSIBLE

AND AVAILABLE FOR SCIENTISTS AND

RESEARCHERS AND OTHERS.

WE ARE LEADING THIS EFFORT WITH

THE ACTIVE GROUPS TO FOLLOW ALL

THE ACTIVE RELATED ACTIVITIES

AND VACCINES AND THERAPEUTICS.

AND THIS IS SORT OF A ONE PLACE

WE CAN GO TO EASY TO READ DATA

IN THIS OPEN DATA PORTAL.

IT IS ON OUR NCATS WEBSITE.

IT IS -- SO JUST TO ORIENT YOU

AT THE TOP CORNER WITH ALL THOSE

LITTLE DOTS THAT ARE DIFFERENT

COLORS THOSE REPRESENT THE

DIFFERENT WAVES OF THE DIFFERENT

VARIANTS OF THE VIRUS.

ON THE LEFT-HAND SIDE, ALL THAT

IS REALLY REPRESENTING JUST THE

OMICRON WAVE DATA.

SO ALL THE DATA WE COLLECTED AS

OMICRON HAS COME ON TO SCENE AS

RELATES TO THE VACCINES AND THE

DIFFERENT MOAN:AL ANTIBODIES AS

WELL AS OTHER THERAPEUTICS.

SO ALL THOSE DOTS THERE ON THE

RIGHT HAND SIDE INDICATE THAT

ACTIVITY IS WANED BUT IF THE DOT

IS COLORED ON THE LEFT-HAND

SIDE, THAT MEANS NO REDUCTION IN

BENEFIT AND THOSE ARE ACTUALLY

VIABLE CANDIDATES FOR

THERAPEUTICS TO HELP THE OMICRON

PIECE OF THE PANDEMIC.

ON THE RIGHT HAND SIDE SHOWS THE

DIFFERENT KINDS OF ASSAYS THAT

WE CONDUCT TO SHOW THIS

VIABILITY EFFICACY, THESE TYPES

OF MEDICATIONS.

SO IT IS REALLY A GREAT RESOURCE

TO LOOK, NOT JUST AT CURRENT

RATE BUT ALSO THE OTHER WAVES TO

SEE THE DIFFERENCES AND WHY IT

IS VERY IMPORTANT WE HAVE THIS

LARGE THERAPEUTICS PROGRAM, THAT

IS HELPFUL AS WE MOVE FORWARD IN

THIS PANDEMIC WHICH IT IS -- I

DON'T KNOW THAT ANY OF US COULD

HAVE PREDICTED IT WOULD BE HERE

TODAY WHERE WE ARE SO I HOPE WE

CAN CONTINUE TO MAKE THESE

THINGS AVAILABLE AND CONTINUE

THE ACTIVITIES GOING FORWARD.

THE OTHER MAJOR AREA IS AS I

TALKED BEFORE IS MAKING

ELECTRONIC HEALTH RECORD

INFORMATION AVAILABLE FOR

STUDYING COVID-19.

THIS IS A PARTNERSHIP WITH THE

SAVE PROGRAM 2H ALONG WITH

NATIONAL INSTITUTE ON GENERAL

MEDICAL SCIENCES AND THE IDEA

CLINICAL RESEARCH PROGRAM AS

WELL AS NON-PROFIT HEALTH IT

ORGANIZATION WORKING TO IMPROVE

HEALTH EQUITY.

NCATS IS JOINED UP WITH OTHER

FEDERAL AGENCIES AS WELL FOR

EXAMPLE THE FDA, OFFICE OF

NATIONAL COORDINATOR FOR HEALTH

INFORMATION AND TECHNOLOGY AS

WELL AS BRTA AND A FEW OTHERS.

TO DATE, THERE ARE NOW OVER 10

MILLION ELECTRONIC HEALTH

RECORDS WITHIN THE DATA SET WITH

3.7 MILLION REPRESENTED COVID

POSITIVE CASES.

OVER 12 BILLION ROWS OF DATA

WITH ENTIRE COMMUNITY WHO IS

MAKING HEADWAY ON ORGANIZING AND

ANALYZING THIS DATA.

AND THERE IS A DASHBOARD HERE AT

THE BOTTOM, A LINK THERE YOU CAN

GO TO NCATS DASHBOARD AND YOU

CAN SEE THIS DATA COLLECTED.

BUT AS I MENTION 12 BILLION ROWS

OF DATA SO THINK ABOUT THAT ROWS

OF DATA, AND IF YOU RUN THE

SPREADSHEET AND PRINT OUT THAT

SPREADSHEET AND YOU THINK ABOUT

EACH CELL IN A ROW IS PROBABLY

AN INCH, THE AMOUNT OF DATA THAT

IS BEING COLLECTED, IF YOU WERE

TO TAKE THE PREED SHEET AND MAKE

LINEAR ACROSS EACH ROW, THAT ROW

WOULD GO AROUND THE EARTH MORE

THAN THREE TIMES.

SO I AM GLAD WE ARE DIGITAL, IT

IS VERY HELPFUL BUT IT IS A LOT

OF DATA BEING CHECKED AND REALLY

IMPRESSIVE WORK THAT THIS GROUP

HAS DONE.

WE HAVE DONE BIT MORE TO AUGMENT

DATA AS YOU MIGHT IMAGINE,

ELECTRONIC HEALTH RECORDS CAN BE

MESSY AND INCOMPLETE SO WE WANT

TO MAKE SURE WE ARE DOING OUR

DUE DILIGENCE TO GET AS MUCH

INFORMATION AS POSSIBLE.

SO WE HAVE THE DATA HERE IS NOT

REALLY THAT CRITICAL BUT IT IS

NOW INCLUDING MORTALITY DATA, IT

IS INCLUDING VIRAL VARIANT

SEQUENCE DATA SO WE KNOW WHICH

VARIANTS PARTICULAR PEOPLE HAVE

BEEN EXPOSED TO AND HOW THAT

MIGHT DRIVE OUTCOMES.

WE ARE GETTING CMS DATA WHICH

INCLUDES CLAIMS DATA,

PRESCRIPTIONS AND OTHER

INDICATORS OF HEALTHCARE

UTILIZATION.

SO THESE ADDED ON DATA SETS WILL

BE MADE AVAILABLE LIKELY

STARTING IN BEGINNING OF

FEBRUARY.

AND WE ARE NOT DONE THERE, WE

WANT TO GO AND THINK ABOUT WHAT

IS NEXT SO WORKING TOWARD

FIGURING WAYS OF OBTAINING ICU

WAGE INFORMATION FORM AS WELL AS

IMAGING DATA.

SO WE HAVE REALLY MOVED THE BAR

IN ADOPTING PRIVACY TECHNIQUESES

TO ENABLE THIS TYPE OF ACTIVITY

AND MAXIMIZE POTENTIAL OF THESE

EXTERNAL DATA SETS AS WELL AS

OTHER FUNDED REPOSITORIES LIKE

THOSE BEING DEALT FOR RECOVER

PROGRAM THAT POST ACUTE SEQUELAE

OF COVID CONDUCTED OUT OF NHLBI

WE ARE PARTNERING WITH THEM SO A

VARIETY OF THINGS PULLED INTO

AND I THINK THE IS MAKING THIS

DATA SYSTEM VERY ROBUST.

I WILL GIVE YOU A SENSE OF

ACTIVITIES ONGOING IN A MOMENT

BUT I WANT TO RELAY TWO MORE

POINTS THAT ARE NEW RELATED TO

THAT.

ONE IS WORKING TOWARDS A TRIBAL

CONSULTATION, WE HAVE BEEN

WORKING THIS OVER A YEAR BUT THE

REASON THAT IT TOOK A BIT TO GET

THERE, THERE'S OTHER ACTIVITIES

AROUND COVID THAT ARE ALSO

TRIBAL CONSULTATION SO WE HAVE

BEEN IN THE QUEUE AND IT IS NOW

OUR TURN.

THE REASON THIS IS IMPORTANT,

WHEN WE GET TO DEMOGRAPHIC FROM

DATA CONTRIBUTORS IT CONTAINS

THE OMB RACE TABLES WHICH

INCLUDES AMERICAN INDIAN ALASKA

NATIVE OR AIAN DATA.

THESE ARE SELF-IDENTIFIED OR

PROVIDER IDENTIFIED

CLASSIFICATION OF RACE FROM

EXISTING DATA.

WE DON'T RECEIVE TRIBAL

AFFILIATION DATA, SIMPLY THE

RACE TABLES WE GET.

KEEPING INTEGRITY OF THE TRIBAL

SOVEREIGNTY AND PRIVACY WHILE WE

ARE WAITING TRIBAL CONSULTATION

WE HAVE BEEN MEETING WITH OUR

TRIBAL HEALTH RESEARCH OFFICE

AND CENTERS ACROSS THE COUNTRY

AND OTHER EXPERIENCED NIH POO TO

DISCUSS OPTIONS WHAT WE CAN DO

UNTIL WE HAVE THAT CONSULTATION.

BASED ON DIVISION CUSHIONS WITH

THE TRIBAL RESEARCH OFFICE,

NCATS DECIDED AT THAT TIME

OUTSET WHEN WE FIRST MADE THE

DATA PUBLICLY AVAILABLE WE

OBSCURED THE AIAN RACE DATA AS

WELL AS OBSCURED THE 2,659 ZIP

CODE DATA THAT OVERLAPS WITH

TRIBAL LANDS TO PREVENT

INFERENCE OF TRIBAL AFFILIATION.

THESE ARE TWO APPROACHES WE CAN

DO UNTIL WE HAVE TRIBAL

CONSULTATION.

WE LOOK FORWARD TO MEETING WITH

TRIBAL LEADERS AND HAVE AN

OPPORTUNITY TO PROVIDE WHETHER

AND HOW TO UTILIZE THESE SORTS

OF DATA AND (INAUDIBLE) WHICH

MIGHT BENEFIT THE COMMUNITY AS

WELL.

IN FEBRUARY WE WILL BE HAVING

THIS TRIBAL CONSULTATION AND

VARIETY OF ACTIVITIES WILL

FOLLOW SUIT.

THE SECOND AND LAST UPDATE IS

REALLY GIVE YOU A I WANT TO SHOW

AN OPPORTUNITY THAT CAME ACROSS

WITH US, THIS IS A REAL WORLD

EVIDENCE STUDY OUT OF UNIVERSITY

OF COLORADO BY THE INDIANS --

THEY ARE LOOKING AT

EFFECTIVENESS OF TREATMENT WITH

MONOCLONAL ANTIBODIES.

THERE IS AN OPPORTUNITY HERE TO

LOOK AT THE IMPACT OF VIRAL

VARIANTS SPECIFICALLY ON

MONOCLONAL ANTIBODY

INTERVENTION.

SO THIS IDEA CAME ACROSS BECAUSE

OF DISCUSSION WE HAD WITH JANET

WOOD COKE WHCOKC, ABOUTING DEPUTY

COMMISSIONER OF THE FDA BUT ALSO

WHITE HOUSE LEADING A LOT OF

ACTIVITIES RELATED TO THE COVID

RESPONSE.

SO THIS CAME OUT OF JANET'S HEAD

AND WE WANTED TO SEE THIS KIND

OF STUDY HAPPEN.

AND BECAUSE OF THE OPPORTUNITIES

WE HAVE WITHIN NCATS THERE WAS A

WAY TO GET GET COVID SPECIFIC

FUNDS TO SUPPORT THIS KIND OF

ACTIVITY.

NOW BEHOLD THIS PROJECT IS

ESSENTIALLY TO ENROLL 4,000

INDIVIDUALS SOME WHO RECEIVED

MONOCLONAL ANTIBODIES AND SOME

WHO DIDN'T AND FOLLOW THE

INDIVIDUALS WITH TELEPHONE

SURVEYS AND LIKE, WE ALSO

RECEIVE ELECTRONIC HEALTH RECORD

INFORMATION TOO.

AND THIS IS A WAY FOR US TO

DETERMINE THE EFFECTIVENESS OF

THE TREATMENT AND PREVENTING

HOSPITALIZATION AND ATTRACT --

TO TRACK THOSE LONGER TERM

SYMPTOMS AS WELL.

SO THE UNIVERSITY OF COLORADO

TEAM RECOGNIZED THAT WE REALLY

NEEDED TO UNDERSTAND WHO IS MORE

AT RISK TO GET THE DISEASE AND

MORE AT RISK TO BE HOSPITALIZED

SO THAT THE LIMITED SUPPLY

PERHAPS SOMETIMES OF THESE

MONOCLONAL ANTIBODIES COULD BE

USED AND DIRECTED EFFICIENTLY

AND MOST EQUITABLY.

SO THESE ARE THE DATA THAT I'M

SHOWING HERE ON THE SCREEN, IT

IS THIS IS OUT OF NET ARCHIVE,

NOT PUBLISHED YET BUT IT IS

PRE-PRINT SERVER ON MED

ARCHIVES.

SO STILL ONGOING BUT EARLIER

RESULTS THESE ARE DATA THROUGH

JULY SHOW IMPRESSIVE RESULTS BY

DAY 28 FOR EXAMPLE, THIS FIRST

RED LINE HERE, THE MONOCLONAL

ANTIBODY SHOW 50% EFFECTIVENESS

PREVENTING HOSPITALIZATION AND

THEN DOWN AT BOTTOM YOU SEE IT

IS QUITE EFFECTIVE IN TERMS OF

THE LENGTH OF STAY AND

MINIMIZING LENGTH OF STAY OF

THOSE WHO ARE HOSPITALIZED.

THEN THERE'S ALSO BY DAY 90 IN

TERMS OF ALL CAUSE MORTALITY,

THERE'S 83% EFFECTIVENESS IN

PREVENTING DEATH.

SO THESE ARE REALLY IMPORTANT

DATA FOR US TO UNDERSTAND AND

KNOW AND FIGURE HOW TO BEST USE

THESE KINDS OF TREATMENTS IF

THEY ARE IN LIMITED SUPPLY.

SO WE LOOK FORWARD TO I HOPE

BEING ABLE TO EXPAND SOME OF

THIS WORK, AND ESPECIALLY AS THE

PANDEMIC CONTINUES, THIS KIND OF

INFORMATION IS VERY VALUABLE.

THIS IS A LOT MORE WORK COMING

OUT OF N 3C, HIGHLIGHT A FEW

PAPERS HERE IN TERMS OF

PUBLICATION AS WELL AS PRE-PRINT

AND SOME OF THE NEWS OUTLETS

CAPTURING A LOT OF THE WORK

GOING ON IN NTC SO THAT'S

EXCITING TO SEE THIS UPTAKE OF

THIS PARTICULAR DATA SET, IT IS

QUITE RICH AND IT IS GETTING

RICHER WITH THAT INFORMATION I

KNOW IT WILL BE VALUABLE.

WITH THAT ADDED INFORMATION.

IT IS HARD SOMETIMES GET

MAGNITUDE ASIDE FROM THE FACT

THE DATA GO AROUND EARTH THREE

TIMES.

LILY PORTILLA SENT ME NUMBERS OF

AGREEMENTS IN SUPPORT OF N 3C

THAT WE OBTAIN TO DATE, IT IS

AMAZING, THIS IS TRULY AN

ENTERPRISE LEVEL COLLABORATION

WITH OVER 400 PAYMENTS IN PLACE

THAT SUPPORT THIS WORK.

SO I JUST GIVE YOU A SENSE OF

THE MAGNITUDE OF THAT.

SO I'M GOING TO SWITCH GEARS A

LITTLE BIT NOW, I WANT TO CLOSE

OUT BY TALKING ABOUT WHAT IS

NEXT FOR NCATS.

ON DECEMBER 7 AS YOU MAY HAVE

REMEMBERED IT IS OUR BIRTHDAY,

AND WE HAD OUR BIRTHDAY PARTY

AND IT WAS JUST REALLY A

WONDERFUL EVENT.

WE HAD OUR 10TH ANNIVERSARY

CELEBRATION WHICH WHERE WE HAD

771 ATTENDEES AND AVAILABLE ON

DEMAND, YOU ARE WELCOME TO VIEW

ON DEMAND AS WELL.

BUT IT GAVE US AN OPPORTUNITY TO

TAKE STOCK AND TALK ABOUT MANY

OF THE PROGRAMS AND TOPICS THAT

ARE LISTED HERE FROM PATIENT

IMPACT TO DATA DRIVEN SOLUTIONS.

IN ADDITION O THE COVID WORK WE

HAVE BEEN DOING.

WE TALKED TO SUMMARIZE IF YOU

WILL, WE TALK ABOUT HOW

TRANSITIONAL SCIENCE IS REALLY

PROVEN ITSELF OVER AND OVER TO

DRIVE INNOVATION AND LEAD

TRANSFORMATIONAL APPROACHES THAT

BRING US TO OUR ABILITY TO DO

MORE PREDICTIVE PRE-CLINICAL

PIPELINE DEVELOPMENT, TO EXPAND

OUR REPERTOIRE IN TECHNOLOGIES,

TO REVOLUTIONIZE DIAGNOSTIC

TREATMENTS TO PRIORITIZE DEI AND

ALL THE ACTIVITIES THAT WE

SUPPORT.

IN WAYS THAT WE TRAIN THE NEXT

GENERATION OF SCIENTISTS AND

ENGAGE OLD STAKEHOLDERS,

SHOULDN'T SAY OLD BUT PERHAPS

WHO HAVE BEEN OUR STAKEHOLDERS A

FILE AND FINDING NEW ONES WE

WANT TO BUILD THAT TRUST IN AND

NOSHTURE RELATIONSHIPS GOING

FORWARD.

THIS IS THE WAY TO BRING YOUR

TREATMENTS TO ALL PEOPLE MORE

QUICKLY.

THIS IS NOT JUST OUR MANTRA.

THIS IS OUR VISION.

WITH THAT VISION IT PROVIDED AN

OPPORTUNITY TO LOOK AHEAD O THE

NEXT DECADE, IN THE KEY NODE

ADDRESS DURING THAT EVENT I SET

FORTH THREE AUDACIOUS GOALS THAT

ARE FORWARD-LOOKING AND I WANT

TO SET A PATH NEXT DECADE HOW TO

OBTAIN THEM.

NCATS COUNCIL HEAR YOUR THOUGHTS

AND REACTS AND AS WE GET A

PERMANENT DIRECTOR, FOR NCATS

THERE WILL BE AN OPPORTUNITY TO

HONE AND DEVELOP AND METRACISE

THESE MORE.

BUT I WANT TO RUN LIEU THEM

BECAUSE ED I WOULD LIKE YOUR

REACTIONS SO GOAL NUMBER ONE,

MORE TREATMENT.

PERIOD.

RIGHT NOW WE HAVE 5% DISEASE

THAT HAVE A TREATMENT SHOWN HERE

IN THAT -- THIS BOTTOM BAR HERE

ON THE GRAPHIC WITH -- WE HAVE

ABOUT -- WE KNOW THERE'S OVER

7,000 RARE DISEASE TO DATE BUT

MORE ARE ON THE WAY.

WE ONLY HAVE 500 THERAPIES SO 5%

OF DISEASE HAVE A TREATMENT.

LET'S WORK TO GET THAT NUMBER UP

SO THAT A QUARTER OF KNOWN

DISEASE HAVE A TREATMENT IN THE

PIPELINE SO WE WENT 5% TO 25%.

WE ALSO KNOW 90% OF DRUGS IN

CLINICAL TRIALS PAIL BECAUSE OF

THE LACK OF EFFICACY AND SAFETY

CONCERNS.

IN VITRO ASSAYS THAT

RECAPITULATE HUMAN PHYSIOLOGY

AND PATHOLOGY SHOW INCREDIBLE

PROMISE BEING MORE PREDICTIVE OF

TOXICITY AND EFFICACY IN HUMANS

SO WE NEED TO REALLY ENABLE THAT

KIND OF DEVELOPMENT AND THOSE

TYPES OF TECHNOLOGIES TO HAVE A

WIDE ARRAY OF RESOURCES THAT

DON'T JUST MODEL TISSUES AND

ORGANS BUT MODEL DIVERSITY OF

INDIVIDUAL, MODEL GENETIC MAKE

UP, ENVIRONMENTAL EXPOSURES.

SO THERE'S MORE WORK TORR DO

HERE.

-- WORK TO DO HERE.

THERE'S OTHER INITIATIVES AS

WELL THAT WILL DEMOCRATIZE AND

DISSEMINATE NEW TREATMENT

APPROACHES, WE TALK ABOUT THE

PAGT PROGRAM AND THERAPY

CONSORTIUM.

THE SOMATIC CELL GENE EDITING

INITIATIVE; THESE ARE POISED TO

TRANSPERFORM THE PIPELINE FOR

GENE TARGETED THERAPIES BY

BREAKING DOWN THE BARRIERS OF

THE PRE-CLINICAL TO CLINICAL

APPROACHES BUT ALSO ADDRESS THE

MANUFACTURING AND THE REGULATORY

HURDLES IN THE PIPELINE AND SO

ANY RARE DISEASE CAUSE BY SINGLE

GENETIC MUTATION WE HOPE WILL BE

ABLE TO HAVE THE OPPORTUNITY TO

GET FIXED BY SOME OF THESE.

AND WHEN YOU THINK ABOUT 80% OF

THE RARE DISEASE ARE CAUSED BY

SINGLE GENE DISORDERS, THAT

MAKES IT SO THAT IF THIS ONLY

WORKS FOR HALF, THAT'S HUNDREDS

OF DISEASES.

THESE ARE WHY THE TYPES OF

PROGRAMS WHAT WE DO AT N CATS

WITH SRI AND ACADEMIA AND

PATIENTS, PATIENT ADVOCACY

GROUPS AND REGULATORS AND

FEDERAL PARTNERS WHO COME TO

IDENTIFY AND DIAGNOSE WHAT IS

CAUSING THIS SLOW PACE OF

TREATMENT AND DEVELOPMENT OF THE

TREATMENT AND WE ARE WORKING

HARD AND COLLABORATIVELY TO

BUILD PROGRAMS THAT WILL BRING

ABOUT THIS REVOLUTIONARY

TRANSFORMATION SO THIS IS A

MODEL THAT WE LOOK FORWARD TO.

GOAL TWO ALL PEOPLE.

WE WANT TO DRAMATICALLY INCREASE

INCLUSIVITY IN ALL AREAS, WORK

FORCE TRAINING RESEARCH CLINICAL

TRIALS AND HEALTH OUTCOMES.

AND JUST WHEN YOU THINK ABOUT

COVID, VERY RECENT AND STARK

EXAMPLES, ARE COMING OUT FROM

COVID FOR SURE, BUT THE COVID-19

OCCURRENCE OF DEATH AMONG RACIAL

ETHNIC POPULATIONS IN RURAL

COMMUNITIES IN OUR COUNTRY HAVE

BEEN ONE OF THE MOST PAINFUL

ACTS TO SEE DURING THIS

PANDEMIC.

BLACK AND AFRICAN AMERICANS

HISPANIC AND LATINO, AMERICAN

INDIAN AND ALASKA NATIVE ARE

CONTRACTING AND DYING FROM

COVID-19 THAN WHITE ASIAN

COUNTER PARTS IN THOSE

POPULATIONS.

THERE WAS A RECENT PRE-PRINT OUT

OF THE NIGMS CTR PROGRAM USING M

3C SHOWING RATES IN RURAL

COMMUNITIES ARE 40% HIGHER THAN

IN OTHER COMMUNITIES.

THIS IS A BIG DEAL.

WE NEED TO ADDRESS IT, WE NEED

TO DO BETTER.

CTSA ACROSS THE COUNTRY

PRIORITIZED ENGAGEMENT

PARTNERSHIPS AND INNOVATING

AROUND STRATEGIES THAT MEET

PEOPLE WHERE THEY ARE, MOBILE

VANS, COMMUNITY, CENTERS PLACES

OF WORSHIP, GROCERY STORES

LIBRARIES, ALL THOSE SORTS OF

THINGS.

THESE EFFORTS ARE MADE CTSA

CORNERSTONE OF THE TRUSTED

COMMUNITY PARTNERS THAT HAVE AND

WE NEED TO ENABLE THEM MORE TO

PIVOT RAPIDLY ON A VARIETY OF

HEALTH OUTCOMES WE ARE

INTERESTED IN PURSUING AS WELL.

TALK ABOUT DEIA EFFORTS A BIT

AGO AND THE POINT IS THAT

STUDIES OF TOMORROW, WHAT WE DO

IN THE NEXT DECADE ENSURE NO ONE

IS LEFT BEHIND IN OUR PLANNING

DESIGNING OUR IMPLEMENTING.

RESEARCH IMPROVES LIVES FOR ALL.

INCREASING DIVERSITY IN ALL WAYS

IS GOAL NUMBER TWO.

GOAL 3, MORE QUICKLY, WE WANT TO

REDUCE THE TIME IT TAKES FOR A

TREATMENT TO REACH THE MARKET BY

HALF.

IT TAKES 10 TO 15 YEARS AND

$2.6 BILLION FOR DRUG TO MAKE IT

TO MARKET.

WHICH NEED REDUCE THAT TIME.

TO DO THAT WE NEED TO FAIL FAST,

EARLIER AND IMPORTANTLY WE NEED

TO FAIL FORWARD AND THAT IS A

KEY FACTOR BECAUSE WHEN YOU FAIL

FORWARD YOU ARE TRIPPING OVER

THE VULNERABILITIES THAT WE HAVE

FOUND AND YOU CAN FIX THEM BUT

YOU -- WHEN YOU FAIL FORWARD,

YOU NOT HOLDING YOURSELF BACK

YOU ARE FAILING FORWARD TO MAKE

SURE YOU IDENTIFY THOSE

VULNERABILITIES AND MITIGATE

THAT RISK.

IT IS IMPORTANT TO DO THAT.

WE WANT TO PROMOTE EMPLOY MORE

STRATEGIES ADDRESSING MANY

DISEASES AT A TIME AND FINDING

WHAT IS COMMON ACROSS DISEASE

AND TARGETING THOSE UNDERLYING

MECHANISMS.

ON TOP OF THAT HAVE TRAINING THE

NEXT GENERATION OF SCIENTISTS IS

HUGELY IMPORTANT TO HELP RETHINK

HOW WE TRAIN OUR SCIENTISTS AND

ENCOURAGING TO THINK MORE

REVOLUTIONARY AND

TRANSFORMATIONAL TERMS AS

OPPOSED TO MORE EVOLUTIONARY

TERMS.

FINDING COLLABORATION AND PART

SHIPS ARE CRITICAL FOR NCATS AND

WHAT WE DO AND WHEN WE THINK

ABOUT EXECUTING AGREEMENTS THOSE

CAN TAKE TIME SO WHAT WE HAVE

BEEN ABLE TO DO IS QUICKLY

LAUNCH COLLABORATION BECAUSE WE

USE COLLABORATIVE THEM PLACE OR

AGREEMENT THEM PLACE THAT ALLOW

COLLABORATIONS TO HAPPEN ON

ORDER OF HOURS, DAYS SOMETIMES

BUT THERE'S BEEN A COUPLE OF

THEM DONE ON HOURS AND NOT

MONTHS.

SO THIS TIME SAVING IS HUGELY

IMPORTANT FOR US.

THE COVID SCENARIO FOR THE

CLINICAL TRIALS HAVE REALLY

IDENTIFIED HOW MASTERED UMBRELLA

PROTOCOLS ARE REALLY HELPING US

TO BE CLINICAL TRIAL READY.

THINKING SINGLE IRB REVIEW

BOARDS AND ALLOWING THEM TO

ACTIVATE MORE EFFICIENTLY AND

ENROLL PARTICIPANTS SOONER,

THESE ARE THE KEY ACTIVITIES.

AT THE LAST COUNCIL I TALK DATA

SCIENCE EFFORTS AND NEW TOOLS TO

SEE PATTERNS OF BIG DATA THAT WE

HAVEN'T SEEN BEFORE.

OF COURSE N 3C SHOWS US WE CAN

CATALYZE THE HEALTH DATA AS A

PRINCIPLE THAT IS SOMETHING TO

PUSH FOR ALL DISEASES.

THINKING OTHER APPROACHES LIKE

DRUG REPURPOSING, OF COURSE IF

YOU CAN DO DRUG REPURPOSING CUT

THAT TIME LINE TO COUPLE OF

YEARS AS OPPOSED TO TEN YEARS

THAT IT TAKES TO DEVELOP DRUGS

BECAUSE WE KNOW A LOT ABOUT

THOSE DRUGS BEING REPURPOSED,

BEEN FDA APPROVED FOR EXAMPLE SO

OTHER WAYS TO GAIN E FORB SHIES

SO A LOT OF -- EFFICIENCIES.

SO THESE ACTIVITIES ARE AROUND

THAT APPROACH AND GOALS ARE

ASPIRATIONAL BUT THE WHOLE IDEA

IS TO BRING MORE TREATMENT TO

ALL PEOPLE.

AS I TALK TO OUR INTERNAL TEAM

MORE ON THESE IDEAS IT IS CLEAR

TO ME WE CAN MAKE HEADWAY ON

THEM.

IMPORTANTLY THE GOALS WE

FINALIZE WILL GUIDE AND -- US TO

THE NEXT DECADE AND KEEP US

FOCUSED ON MAKING THESE GOALS A

REALITY.

SO I PLAN TO USE THESE TO ALSO

HELP FRAME SOME OF THE SUCCESS

THAT WE HAVE MADE, AND WITH THAT

I'M INTERESTED IN YOUR THOUGHTS

ON ALL THINGS WE DO TODAY AN

ESPECIALLY AROUND THESE THREE

AMBITIOUS AND AUDACIOUS GOALS.

I WILL STOP HERE AND I'M GOING

TO TURN IT OVER TO CLAIRE AND

PENNY TO MANAGE THE DISCUSSION.

THANK YOU VERY MUCH.

I APPRECIATE YOUR ATTENTION.

>> GREAT, JONI.

THANKS SO MUCH.

MS. KENNEDY, WOULD YOU LIKE TO

VERBALIZE THE COMMENT YOU PUT IN

THE CHAT BOX?

>> SURE, I ALSO HAVE A QUESTION

IF I CAN JUMP IN WITH THAT.

I WANT TO THANK THOSE WHO ARE

STEP DOWN FROM THEIR ROLES AND

NCATS AND ESPECIALLY DR. PARISER

ONER HER RETIREMENT FROM PUBLIC

SERVICE AT NCATS AND PREVIOUSLY

AT FDA WITHOUT A DOUBT HER

SERVICE CHANGED OUR RARE DISEASE

LANDSCAPE FOREVER SO I THINK WE

NEED TO RECOGNIZE THAT AND

ACKNOWLEDGE THAT WE HAVE A DEPTH

OF GRATITUDE TO YOU.

WE ARE REALLY EAGER TO CONTINUE

TO WORK WITH DR. BROOKS WE KNOW

A LOT OF MOMENTUM IN ORDR IS

BECAUSE OF SHARED LEADERSHIP SO

LOOKING FORWARD TO WORKING WITH

BOTH OF YOU THROUGHOUT THIS

TRANSITION AND DR. BROOKS IN THE

MONTHS AND HOPEFULLY YEARS

AHEAD.

THANK YOU.

I DID HAVE A QUESTION, IF I CAN

DO THAT AS WELL.

SO FIRST, THANK YOU FOR THIS

WONDERFUL PRESENTATION, I FEEL

LIKE I GET TO MAYBE HEAR YOU

MONTHLY AND EVERY TIME THERE'S A

LOT OF NEW INFORMATION THAT YOU

BRING FORWARD AND A LOT OF GREAT

PROGRESS.

A COUPLE OF THINGS YOU PRESENTED

SPEAKS OUT TO ME AS AGILITY OF

NCATS AND THE ABILITY TO NCATS

TO PIVOT.

ONE EXAMPLE YOU GAVE WAS IN A

CONVERSATION WITH DR. WOODCOCK,

A CONCEPT AND IDEA SHE HAD HAS

NOW COME TO FRUITION AND YOU ARE

SOON GOING TO HAVE DATA AROUND

REAL WORLD EVIDENCE.

UTILIZING MONOCLONAL ANTIBODIES.

THAT IS A QUICK PIVOT.

ONE THOUGHT WE DON'T HAVE TO

ANSWER HERE BUT ONE QUESTION IS

HOW DO WE BETTER COMMUNICATE NOT

JURY OUR IMMEDIATE STAKE HOLDERS

BUT THE GENERAL PUCKLY THE

IMPACT NCATS HAS ON ANSWERING

SOME OF OUR URGENT PUBLIC HEALTH

ISSUES.

AND ADDRESSING SOME OF THOSE.

SO THAT SERVES SORT OF STRIKESES

ME AS NCATS IS ONE OF OUR BEST

KEPT SECRETS AND ONE OF THE MOST

POWERFUL RESOURCES AND TOOLS.

AS YOU WERE PRESENTING THAT

JUMPED OUT AT ME TIME AND AGAIN.

THANK YOU FOR THE WORK YOU ARE

LEADING.

>> THANK YOU SO MUCH, ANNIE.

ECHOING YOUR THANKS TO ANN

PARISER, WHICH I COULDN'T AGREE

MORE, YOUR QUESTIONS, I DON'T

KNOW HOW TO ANSWER YOUR

QUESTION.

EXCEPT TO SAY THAT AGREE WE NEED

TO THINK ABOUT WAYS TO

COMMUNICATE THIS KIND OF

INFORMATION, I THINK AT THIS

STAGE HOW WE ARE GATHERING THIS

INFORMATION AGAIN THE ONE I

PRESENTED IN THERE WILL BE

OPPORTUNITIES FOR US TO DO

CERTAINLY PRESS RELEASES TO GET

THAT INFORMATION OUT MORE AND

BUT I THINK THERE IS MORE TO

LEARN TOO IN TERMS OF HOW WE

THINK ABOUT WHO IS GOING TO BE

AT RISK OR HOSPITALIZATION, WHO

WE NEED TO TARGET FOR THESE

KINDS OF THERAPIES.

AND NOT ONLY THAT, YOU MENTIONED

THE PUBLIC COMMUNICATIONS BUT

THERE'S ALSO THE PHYSICIAN

COMMUNICATION, MONOCLONAL

ANTIBODIES ARE NOT REALLY A

WIDESPREAD THERAPEUTIC IN MANY

SPACES SO THERE'S PHYSICIANS WHO

DON'T KNOW A LOT ABOUT

MONOCLONAL ANTIBODIES AND HOW

THEY ARE USED AND WHY USEFUL FOR

COVID-19.

SO A LOT OF THE WORK THAT I

THINK WE ARE FINDING IS HOW DO

WE DO WE COMMUNICATE TO

STAKEHOLDERS AND PHYSICIAN

COMMUNITY SO THEY UNDERSTAND HOW

TO USE THESE TYPES OF THERAPIES

AND WHEN TO USE THEM AS WELL,

RECOGNIZING THAT WITH DIFFERENT

VIRAL VARIANTS YOU MIGHT HAVE

DIFFERENT RESPOND -- HAVE

DIFFERENT RESPONSES TO THE

MONOCLONAL ANTIBODIES.

SO THAT IS ONE THING.

THE OTHER IDEA WE HAVE, I THINK

WE DO A REALLY GOOD JOB

COMMUNICATING TO SCIENTIFIC

COMMUNITY, THROUGH THE OPEN DATA

PORTAL BECAUSE PEOPLE CAN

UNDERSTAND HOW TO READ THAT

INFORMATION BUT WE TRY TO DO A

GOOD JOB IN HOW WE DISPLAY THAT

DATA TO MAKE IT AS EASILY

DIGESTIBLE AS POSSIBLE.

THE OPEN DATA PORTAL IS A

WONDERFUL EXAMPLE OF THAT, IT IS

EASY TO COMMUNICATE THAT.

THEN THE QUESTION IS HOW DO WE

THINK ABOUT COMMUNITIES AND I

WOULD LOVE TO HEAR IDEAS THIS

GROUP MIGHT HAVE TO BE ABLE TO

DO MORE OUTREACH ON EDUCATING

FOLKS AND PERHAPS THAT IS MORE

WEB PAGES BUT THERE ARE OTHER

MORE ACTIVE KINDS OF THINGS THAT

WE CAN DO.

SO I LIKE THAT IDEA, AS WE

TALKED ABOUT MORE RECENT DATA

FROM MONOCLONAL ANTIBODY DATA,

WE ARE SEEING THAT SAME IDEA OF

HOW DO WE THINK ABOUT THIS

INFORMATION OUT THERE,

DISSEMINATION IS A KEY ASPECTS

OF WHAT WE DO.

SO WE ARE TRYING TO FIGURE OUT

THAT AS WELL.

THAT IS A VERY GOOD POINT.

>> DR. CRESLER WOULD YOU LIKE

THE GO?

>> THANK YOU FOR SHOWING HOW

NCATS IS RESPONDING TO THE ACUTE

NEEDS AT HAND AND STILL KEEP

LONG TERM STRATEGY IN HAND AND

REALLY WANT TO 5 TO 25% PUSH FOR

DEVELOPING TREATMENTS.

IT SOUNDS AUDACIOUS TO ACTUALLY

IMPOSSIBLE TO DO AND I WILL

COUNT ON THAT.

BECAUSE WHAT WE ARE SEEING RIGHT

NOW IS THAT WE ARE DEFINING AND

REDEFINING HOW WE WORK WITH

HUMANS AS OUR HEAT MAP AND CELL

BASED -- WE ARE ALIGNING CIVIL

AND MACHINERY AND MOST

IMPORTANTLY WHY THEY FAIL.

WHY IT MIGHT NOT BE POSSIBLE TO

DEVELOP SPECIFIC TARGETED

THERAPIES FOR TWO AND A HALF

THOUSAND DISEASES AND I THINK WE

HAVE ALL THE INSTRUMENT TIME AT

HAND AND NCATS HAS THE TOOLS TO

DEPLOY THEM, TO IDENTIFY SHARE

COMMON PATHWAYS OF DISEASE

MANIFESTATIONS WHICH CAN PREVENT

A LESION WHICH WOULD CAUSE A

PHENOTYPE IN THE PATIENTS TO

REMAIN SUB CLINICAL.

I KNOW THAT THAT HAS BEEN

DISCUSSED INSIDE NCATS FOR QUITE

A WHILE IN SEVERAL KNOWLEDGE

ACTIVITIES, I HEADED THAT WAY

BUT IT IS RARELY -- REALLY

IMPRESSIVE TO SEE HOW QUICKLY

DISEASE SPECIFIC ACTIVITIES IN

THE CELL MAPPING WE ARE MAKING

FOCUS TO IDENTIFY THESE PATIENT

SPECIFIC CHANNELS AND HOW TO

ENTERPEER WITH THEM IN A TRANS--

INTERFERE WITH A TRANSLATIONAL

SENSE.

THIS IS THE INSTITUTE WHO

LEVERAGE ALL THEIR KNOWLEDGE AND

EXOSOME DISCUSSIO DISCUSSIONS WITH VERY

SPECIFIC APPLICATION ALREADY BUT

I THINK WHATEVER YOU CAN PUSH

BACK, THIS IS JUST ARBITRARY

NUMBER, NO IT ACTUALLY ISN'T.

>>

>> SORRY ABOUT THAT MA,THIAS.

THANK YOU SO MUCH FOR THOSE

COMMENTS.

I WAS WORRIED TOO THAT IT MIGHT

BE -- THAT 25% NUMBER MIGHT BE

TOO BIG BUT AT THE SAME TIME I

AGREE WITH YOU.

I THINK WE HAVE TO GO FOR IT.

YOUR POINT ABOUT THE SUB

CLINICAL UNDERSTANDING OF THOSE

END POINTS IS ALSO IMPORTANT AND

I THINK WITH THE IN VITRO ASSAYS

THAT WE CAN PUT TOGETHER

PRINTING AND TISSUE CHIPS WE ARE

USING, IT IS ALSO TO START

IDENTIFYING WHAT THE SUB

CLINICAL PHENOTYPES ARE AS

BIOMARKERS THAT CAN BECOME

SOMETHING USED CHUNKILY AND AN

AREA WE WILL TRY TO DRIVE

FORWARD AS WELL.

APPRECIATE YOUR COMMENTS.

>> THAT WAS TERRIFIC.

THANK YOU SO MUCH.

WHAT I WOULD LIKE TO EMPHASIZE,

WE ARE ALMOST SEEING EMERGENCE

OF NEW TYPE OF FIELD OF

MEDICINE.

WHICH IS AROUND SMALL PATIENT

GROUP POPULATIONS WHO HAVE YOU

BEEN SUBDIVIDED BY MOLECULAR AND

OTHER TYPES AND THE RARE DISEASE

FIELD THAT I LIVE IN, WE ARE

LITERALLY SEEING 11 NEW

CONDITIONS A WEEK.

WHEN YOU LOOK AT THE LINKING OF

GENETIC VARIATION TO THAT.

THAT REALLY DOESN'T FIT THE

CLASSIC MODEL, THE STATISTICS

ARE DIFFERENT, THE EVIDENTIARY

PROOF IS DIFFERENT.

THE CLINICAL TRIALS MODELS AND

THERE'S REALLY M CATS IS BETTER

POSITIONED TO DEAL WITH THIS AND

TO LEAD THE WAY THAN ANY OTHER

GROUP.

BECAUSE IT IS DISEASE AGNOSTIC

TO A CERTAIN EXTENT.

THE METHODOLOGIES THAT APPLY TO

THESE SMALL GROUPS CAN GO ACROSS

MULTIPLES BUT WE NEED A HOMING

TOOL -- WE ARE BUILDING A

TOOLBOX, WE HAVE TO CONTINUE TO

BUILD THAT.

SO THE ONLY THING I WOULD ADD ON

TOP OF THAT VERY GOOD

PRESENTATION IS BUCKLE UP BUT

ALSO IT IS VERY IMPORTANT TO

ENGAGE THE FDA CONSISTENTLY AND

ACTIVELY.

BECAUSE IF WE DON'T A LOT OF THE

WORK THAT WE DO IS GOING TO BE

WAISTED WHEN PEOPLE HAVE TO GO

BACK AND USE A DIFFERENT CLASSIC

MODEL WHEN TAKING TRIALS THROUGH

FOR APPROVAL.

>> THAT IS A GREAT POINT,

MARSHALL.

THANK YOU FOR BRINGING UP THAT

SPECIFIC ISSUE AROUND THE FDA

AND I'M GRATEFUL FOR THE WORK

THAT THE FDA DOES AND THEY --

THEY WORK HAND IN HAND WITH US

ALONG THE WAY.

CASE IN POINT THAT I WOULD SAY

IS WITH THE SPOKE GENE THERAPY

CONSORTIUM, THIS IS ANOTHER ONE

OF THESE PUBLIC PRIVATE

PARTNERSHIPS THAT WE DO WITHIN

THE NIH DUE TO THE ACCELERATED

MEDICINE PROGRAM.

FOR THAT PARTICULAR PROGRAM FDA

IS IN IT FROM THE BEGINNING.

AND THEY ARE HELPING US BUILD IT

AND DEVELOP THE FRAMEWORK FOR IT

BECAUSE IT IS NOT JUST ABOUT THE

SCIENCE THAT WE NEED TO SUPPORT

IN TERMS OF UNDERSTANDING THE

ADENOASSOCIATED VIRAL THERAPIES

BUT IT IS ALSO THAT WE NEED TO

FIGURE OUT THAT MODEL OF HOW TO

WORK THROUGH REGULATORY SPACE SO

THAT IT IS NOT JUST A ONE OFF,

IT IS A PLATFORM TECHNOLOGY.

I THINK THE DISCUSSION WE HAVE

HAD WITH PETER MARKS AT THE FDA

HAVE BEEN REALLY EXCITING THAT

THEY ARE VERY MUCH INTO THAT

KIND OF IDEA AND WE LOOK FORWARD

TO WORKING THROUGH THAT THAT

PLATFORM IN AND OF ITSELF FOR

SPOKE GENE THERAPY PROGRAM BUT

IF IT WORKS IT BECOMES A BIGGER

PLATFORM WHICH YOU CAN APPLY

OLGONUCLEOTIDE TYPES OR OTHER

THINGS SO IT WILL BECOME A

PLATFORM OF PLATFORMS AND I

THINK THAT'S REALLY EXCITING.

>> ONE MORE THING TOO, I SAT ON

COUPLE OF FDA COMMITTEES WHERE

WE TALK ABOUT HOW WE IN THE

MODERN AGE HOW WE DO CLINICAL

TRIALS AROUND THESE.

THERE'S ANOTHER SIDE TOO, NOT

JUST WHAT WE ARE GIVING TO

PATIENTS IS HOW WE ARE TESTING

THE OUTCOMES.

WE NEED A NEW SET OF TOOLS THAT

ARE MORE DESIGNED AROUND

PATIENTS WHO ARE GEOGRAPHICALLY

SEPARATED IN OTHER WORDS ONLINE

DIGITAL TOOLS AND COVID TAUGHT

US NOTHING ELSE CONCERNING MOST

EVERY NIH PROTOCOL WAS FULL OF

PROTOCOL VIOLATIONS BECAUSE

PATIENTS COULDN'T COME IN ANY

MORE FOR THINGS BUT NONE OF THE

TOOLS ARE VALIDATED SO THERE IS

A LOT OF ROOM FOR PARTNERSHIP ON

DEVELOPING TOOLS, VALIDATING THE

TOOLS.

SO THAT NOT ONLY CAN WE PROVIDE

PLATFORMS LIKE OUR GENE THERAPY

BUT ALSO WE HAVE WAYS TO SAY

OKAY IS IT EFFICACIOUS, AND CAN

WE DO THAT IN A WAY THAT ISN'T

GOING TO CRUSH THE FAMILIES AND

PATIENTS.

THEY ALREADY GOT ENOUGH ON THEIR

PLATE ALREADY.

>> ABSOLUTELY.

AND AS YOU KNOW, THEY ARE THE

ONES DOING ALL THE MONEY RAISING

AND FOR HERMING THOSE PARTICULAR

DISEASE, IF THIS WORKS IT WOULD

BE A HUGE BURDEN OFF OF THEIR

SHOULDERS.

>> VERY EXCITING.

THANK YOU.

>> DR. HARRIS, YOU ARE NEXT.

>> THANK YOU.

ECHO OTHER COMMENTS THAT GREAT

PRESENTATION, LOVE THE GOALS.

I SORT OF FOCUS ON THE MORE

PEOPLE AND THINKING ABOUT THE

FACT THAT GIVEN WORK YOU GUYS

HAVE DONE WITH N 3C NHLBI HAS

DONE WITH BIODATA CATALYST AND

THIS IS A NEW ERA WE ARE IN AND

IT DEMOCK TIDESES ACCESS TO SOME

RESOURCES THAT WERE UNHEARD OF A

DECADE AGO.

BUT I THINK TO MAKE IT REALLY

WORK, WE NEED TO THINK ABOUT

INVESTING IN THE WORK FORCE AND

MAKING SURE THAT PEOPLE ARE DATA

PROFICIENT AND INVEST IN WHAT

YOU CALL TRANSLATORS OR MATCH

MAKERS, BEING ABLE TO PAIR DATA

SCIENTISTS WITH CLINICAL EXPERTS

WITH COMMUNITY ENGAGEMENT

EXPERTS, ET CETERA.

THAT IS GOING TO BE NEEDED TO

TAKE US FROM WHERE WE ARE TO

WHERE WE COULD BE BUT SO JUST

ENCOURAGE US ALL TO BE THINKING

STRATEGICALLY ABOUT THAT SORT OF

WORK.

>> GREAT IDEA.

WITHIN THE N 3C WHAT I DON'T

TALK ABOUT TOO MUCH IS THERE ARE

THINGS CALLED DOMAIN TEAMS.

THE IDEA BEHIND IT, IT'S GETTING

TO THAT, IT IS ABOUT PAIRING

STATISTICIAN WITH CLINICIAN ON A

PARTICULAR OUTCOME THEY MIGHT BE

LOOKING AT BUT A STATISTICIAN IS

NOT GOING TO KNOW WHETHER IS

IMPORTANT IN TERMS OF

UNDERSTANDING END POINTS SO

DOING THAT KIND OF PAIRING IS

GOOD BUT WHAT YOU ARE SAYING IS

THAT -- THAT MIGHT BE NECESSARY

BUT IT IS NOT SUFFICIENT.

I THINK YOU ARE RIGHT, WE HAVE

BEEN THINKING ABOUT THIS, WE

HAVE BEEN PARTNERING WITH NIH

AND THE OFFICE OF DATA SCIENCE

AND STRATEGY, THEY ARE DOING A

VARIETY OF PROGRAMS ON SOMETHING

CALLED IN THE HEAD TRYING TO

MAKE SURE THEY ARE TOOLS AND

RESOURCES AVAILABLE FOR ALL

COMMUNITIES TO BE MORE DATA

PROFICIENT BUT EVEN THERE I FEAR

IT IS NECESSARY BUT NOT

SUFFICIENT SO THAT IS SOMETHING

TO KEEP UPDATED ON, IT IS A VERY

ASTUTE POINT AND I WROTE IT

DOWN.

THANK YOU.

>> DR. JACKSON.

>> HI, JONI.

THANK YOU FOR THAT OVERVIEW AND

TOUCHING SOME OF THE HIGHLIGHTS

OVER THE LAST SEVERAL MONTHS.

I GUESS I WANT TO FOLLOW-UP ON

PAUL.

SO I HAVE TO MAKE SURE I RAISE

MY HAND BEFORE PAUL SO HE

DOESN'T FEEL WHAT I WAS GOING SO

SAY BUT IT IS ALSO AROUND SAME

CONCEPT OF ALL PEOPLE AND DATA

DEMOCRATIZATION.

I WONDER WHAT ARE KIND OF THE

EFFORTS THAT ARE GOING TO BE

GOING FORWARD WITH N CATS

SPECIFICALLY AROUND DATA EQUITY

ACCESSIBILITY PARTICULARLY FOR

COMMUNITY STAKEHOLDERS, AND

OTHERS REDUCING BIAS WITH DATA

FROM INCEPTION TO FINAL

ANALYSIS.

THEN ADDRESSING SOME OF THE

ISSUES AROUND DIGITAL EQUITY AS

WELL.

ARE THESE AREAS THAT NCATS IN

FACT TAKE SOME LEADERSHIP BOTH

AROUND THE RARE DISEASE BUT

BROADLY AROUND TRANSLATIONAL

SCIENCE, BECAUSE IT IS THE

QUALITY OF OUR DATA, DON'T

INCLUDE KIND OF THE BREADTH OF

OTHER THINGS THAT IMPACT HUMAN

HEALTH, THEN WE WILL CONTINUE TO

MAKE ERRORS AS WE MOVE FORWARD

NOT UNDERSTANDING EVERYTHING

FROM EXOSOME ALL THE WAY THROUGH

SOCIAL DETERMINANTS OF HEALTH OR

EVEN ISSUES ASSOCIATED WITH

STRUCTURAL RACISM.

>> THANK YOU FOR BRINGING THAT

UP.

THE SHORT ANSWER IS THAT AT

NCATS WE HAVE JUST STARTED TO --

MAYBE THIS IS THE LONG ANSWER.

SHORT ANSWER YES.

THE LONG ANSWER THOUGH IS MORE

INTERESTING.

WE JUST STARTED TO BUILD A

REALLY DEEP AND WIDE FOCUS ON

DATA SCIENCE.

AND WE HAVE A DATA SCIENCE LEAD

THAT IS MANAGING ACROSS THE

NCATS AND A LITTLE BIT ABOUT

WHAT WE HAVE COME UP WITH, WE

HAD A RETREAT TO TALK ABOUT DATA

SCIENCE AND WE ARE NOW HAVING A

FOCUSED EFFORT IN THE TYPES OF

DATA SETS AND RESOURCES THAT WE

HAVE.

ABLE TO MAKE SURE THAT --

AVAILABLE.

TO MAKE SURE WE ARE TALKING

ABOUT THE IDEA OF TRANSPARENCY

AND WHAT IS IN THE DATA HOW TO

UNDERSTAND WHAT IS IN THE DATA.

ALSO TO MAKE SURE THAT WE CAN

IDENTIFY BIASES IF THEY ARE IN

THE DATA.

IF YOU KNOW AND IDENTIFY YOU CAN

INTERPRET THINGS BETTER.

THE WHOLE IDEA OF COURSE THEN

TOO IS UNDERSTAND WHAT BIASES

ARE SO YOU CAN ADDRESS THEM AND

BRING MORE DATA INTO EVALUATE

THAT.

I WILL SAY ONE OF THE THINGS FOR

THE N 3C WE HAVE BEEN WORKING

ON, IS A MORE CONCRETE EXAMPLE

WE REALLY WANTED TO WORK WITH --

BECAUSE THE DATA TYPES THEY ARE

BRINGING IN ARE AREAS MORE

UNDERSERVED SO WE WANTED TO MAKE

SURE WE -- THAT TYPE OF DATA WAS

REPRESENTED IN THE N 3C COHORT.

EVEN THEN I THINK WE STILL NEED

TO DO A BETTER JOB AND ENSURE

THAT WE ARE GETTING THAT

DISTRIBUTION AND REPRESENTATION

OF THE UNITED STATES SO THAT WE

HAVE THAT DATA TRANSPARENCY SO

THAT THINGS WE INTERPRET FROM

THE STUDIES THAT WE DO ARE

GENERALIZABLE IN A WAY.

AND I THINK THAT IS SO

IMPORTANT.

AND THIS GOES BACK TO WHY I

THINK I'M EXCITED ABOUT THE

TRIBAL CONSULTATION WE ARE ABOUT

TO HAVE THAWS BAY IS ANOTHER

AREA WE NEED TO BOLSTER AND

SUPPORT BUT ALSO RECOGNIZE THAT

THE -- THEY HAVE TRIBAL

SOVEREIGNTY OVER DATA THEY HAVE.

AND WE WANT TO MAKE SURE WE ARE

HONORING THAT AND RECOGNIZING

THAT.

SO WE WANT TO USE THAT VERY

WELL, AND SO THAT THAT COMMUNITY

THEN CAN TAKE A LOOK AT THE DATA

AND APPLY IT NECESSARILY TO HELP

THEM THINK ABOUT WHAT KINDS OF

RESOURCES THEY MIGHT NEED GIVEN

SPECIFIC SCENARIOS IN THAT

COMMUNITY AND THINK ABOUT DOING

THAT AS WELL.

SO THIS IDEA OF BIAS IS

SOMETHING WE THINK ABOUT AND

TALK ABOUT A LOT AND I AM --

THIS IS -- I THINK OVER THE NEXT

YEAR AT N CATS DATA SCIENCE AND

THIS PARTICULAR IDEA IS ONE OF

THE BIGGER THINGS THAT WE WILL

BE FOCUSING ON.

>> MS. HARTMAN YOU ARE NEXT.

>> THANK YOU.

>> FEW FOR THE FAIR WELL, I'M

SAD THIS IS MY LAST MEETING BUT

LOOK FORWARD TO WORKING WITH ALL

OF YOU MANY LEADING GOVERNMENT

AFFAIRS FOR ALLIANCE FOR

REGENERATIVE MEDICINE SO STILL

IN THIS SPACE BUT I WANT TO

COMMENT MORE WEARING MY HAT AS A

RARE MOM, ONE OF THE MOST

IMPORTANT THINGS I HAVE SEEN

WORKING WITH NCATS HAS BEEN

CHAMPIONSHIP RARE DISEASE.

ANNIE SAID QUITE A BIT ABOUT

THIS AND ECHOED COMMENTS AND

SAME WITH MARSHALL SUMMERS RARE

IS A PUBLIC HEALTH PRIORITY.

THIS HAS BEEN AN ISSUE THAT WE

HAVE BEEN TRYING TO PUSH WITH

THE ASSISTANCE FUND WORKING

CLOSELY WITH DR. SUMMER AND

CHILDREN'S NATIONAL WORKING

CLOSELY WITH ANNIE KENNEDY AND

HER COLLEAGUES AT THE EVERY LIFE

FOUNDATION AND THE REST OF THE

RARE DISEASE COMMUNITY.

NCATS HAS ALWAYS BEEN THE

BIGGEST CHAMPION FOR RARE

DISEASE.

JONI YOUR LEADERSHIP IN THIS

SPACE IS CRITICAL AND WE HOPE

THIS WILL CONTINUE.

PERSONALLY AND PROFESSIONALLY

VERY IMPORTANT TO ME THAT THIS

CONTINUES.

I KNOW WE WILL HEAR FROM TARA ON

ARPA H AND THIS IS AN ISSUE THE

RARE COMMUNITY IS FOCUSED ON HOW

TO ENSURE THAT RARE DISEASES ARE

ALSO PRIORITIZED WITHIN ARPA H

THE RARE DISEASE COMMUNITY THE

ASSISTANCE FUND EVERY LIFE

GLOBAL GENES RARE -- THE WHOLE

SLEW OF ORGANIZATIONS PUT A

WHITE PAPER TOGETHER, HAPPY TO

SHARE IF YOU HAVEN'T SEEN IT, IT

IS VERY SHORT BUT OUTLINES WHAT

RARE DISEASE COMMUNITY WOULD

LIKE TO SEE, ARPA H FOCUS ON AND

WORK WITH NCATS GOING FORWARD.

SOME OF THE RARE PARENTS AND I

HAVE HAD A THE CUSHION ABOUT

WHAT WAS RAISED IN -- DISCUSSION

ABOUT WHAT WAS RAISED IN TERMS

OF NIH AND FDA WORK CLOSE

TOGETHER AND MAKING SURE THEY

ARE WORKING WITH CMS SO THAT

ONCE THESE DRUGS HIT THE MARKET

PATIENTS CAN ACCESS THEM AS WELL

AND THAT IS SOMETHING WE SPENT

TIME ON AT THE ASSISTANCE FUND.

WHEN IT COMES TO EVEN GETTING

THESE DRUGS INTO THE PIPELINE

AND THROUGH THE FDA, ONE

CONVERSATION WE HAVE BEEN HAVING

AS CAREGIVERS OF RARE DISEASE

QUITE A BIT IS THE NEED FOR A

NEW LOOK AT THE RISK CALCULATION

THAT FDA IS USING.

THIS IS SOMETHING A LOT OF US

HAVE SPOKEN PUBLICLY ABOUT BUT

THERE NEEDS TO BE A LARGER

CONVERSATION.

WHEN CHILDREN ARE DYING, THAT IS

SOMETHING THAT THE FDA DOES

UNDERSTAND.

MANY OF YOU MAY REALIZE WITH MY

SITUATION MY DAUGHTER HAS A

SEVERE DEVELOPMENTAL DISABILITY.

SO SHE'S ESSENTIALLY A PERPETUAL

TODDLER, SHE CAN BARELY WALK,

SHE CAN'T TALK, SHE'S GOING TO

BE SIX SOON, SHE CANNOT COMPLETE

ANY OF THE ACTIVITIES OF DAILY

LIVING WITHOUT ASSISTANCE.

IT IS VERY MUCH LIFE ALTERING.

THE RISK CALCULATION FOR US IS

ALSO VERY DIFFERENT.

WE -- THERE WAS JUST A

CONVERSATION THAT LUKE ROSEN

RELEASED ABOUT HOW CAREGIVERS

WOULD LIKE TO TIME OUR LEAVING

THIS PLANET WITH OUR CHILDREN

BECAUSE THE THOUGHT OF LEAVING

THEM HERE ALONE IS TERRIFYING

BECAUSE WE ARE NOT SET UP IN

THIS SOCIETY TO CARE FOR THEM.

IT IS IMPORTANT TO CONSIDER THIS

RISK CALCULATION.

FOR THESE CHILDREN AS WELL.

BECAUSE RIGHT NOW, WE DON'T HAVE

A GOOD SOLUTION.

A LOT OF US ARE AWAKE A LOT OF

NIGHTS THINKING ABOUT THIS.

THE HOPE THAT THE SCIENCE WILL

MOVE FORWARD IS CRITICAL.

I'M ALSO HOPEFUL THAT WE'LL DO

MORE WORK AND THIS IS NOT NCATS

JOB BUT LOOKING HOW WE DO TAKE

CARE OF THESE CHILDREN.

THERE IS NOT ENOUGH OF THE -- IN

THE WORLD TO CARE FOR THEM.

WE DON'T WANT TO HAVE ANOTHER

WILLOW BUTCH, THE INSTITUTION

WHERE THEY ARE NOW DOING MY

DAUGHTERS UNNATURAL HISTORY

STUDY TRANSFORMED SINCE THE

'80s.

THESE ARE THINGS THAT NEED TO BE

BROUGHT INTO THE OPEN.

WE NEED DISCUSSION WITH THE

PARENTS AND WITH THE CAREGIVERS.

S ESPECIALLY FOR CHILDREN BRAIN

ARE NOT WORKING CORRECTLY AND

CANNOT SPEAK OR THINK FOR

THEMSELVES.

IT IS A SOCIETAL QUESTION.

THAT I THINK WE REALLY NEED TO

ADDRESS.

ONE LAST THING, MY RESEARCH

EMAILED US WORKING ON THE ARPA H

GETTING PRY YOUR PHASE FOR ARPA

H, HE SAID COLLEAGUES WERE

ASKING HIM WHY WOULD WE FOCUS ON

RARE DISEASES?

WHY WOULDN'T WE FOCUS ON SOME

MORE COMMON DISEASES?

AND WHAT ALL OF US DISCUSSED

AGAIN OFFLINE QUITE A BIT IS WE

WOULD KILL FOR CORTICOSTEROIDS

IF OUR KIDS HAD ASTHMA, KILL FOR

INSULIN PUMPS LIKE IF YOU ARE

DEALING WITH DIABETES, NOT TO

SAY WE CAN'T DO BETTER IN THOSE

SPACES BUT IF YOU CAN PERFORM

BASIC ACTIVITIES OF DAILY

LIVING, YOU HAVE GOT A HUGE STEP

UP.

SO I THINK WE CAN DO MORE WORK

TO TRY TO GET PEOPLE TO THAT

PLACE WHERE THEY CAN FUNCTION AT

EVEN BASIC LEVEL IN SOCIETY AND

THAT'S THE HOPE THE SCIENCE AND

NCATS IS BRINGING TO US BUT I

REALLY DO THINK BENEED TO HAVE

NCATS PULL IN, YOU HAVE BEEN

GOOD AT THIS, THE PATIENT

CAREGIVER COMMUNITIES, MAKE SURE

FDA IS REALLY LISTENING, MAKE

SURE CMS IS REALLY LISTENING THE

WHITE HOUSE IS LISTENING, AND

THAT WE ARE MOVING THIS FIELD

FORWARD AND WE ARE TAKING INTO

ACCOUNT THAT PERSPECTIVE WHEN

THINKING ABOUT RISK.

ONE LAST THING, IN TERMS OF THE

DATA DEMOCRATIZATION, I AGREE

THAT IS SO CRITICAL.

AND I'M THRILLED MY DAUGHTERS

DISEASE DAY IS PARTICIPATING IN

RARE X.

THE DATA IS OWN AND AVAILABLE

AND PATIENTS AND THEIR FAMILIES

MAKE THE DECISION TO SHARE IT

AND EVENTUALLY, I DON'T WANT TO

PUT KELLY ON THE SPOT BUT SHE'S

BEEN LEADING A LOT IN MAKING

SURE,IOTONLY THAT PATIENTS OWN

THEIR DATA BUT THAT THEY GET

COMPENSATED FOR THEIR DATA.

THAT IS IMPORTANT GOING TO THE

FUTURE.

WE KNOW THE STORY OF HENRY

LACKS, IF THE PATIENTS

THEMSELVES ARE THE ONES

GENERATING THESE MILLIONS AND

BILLIONS OF DOLLARS THAT SOME OF

THOSE RESOURCES ARE COMING BACK

FOR WHAT THEY PUT IN.

THAT IT SEEMS TO ME TWO CENTS,

HAPPY TO SHARE THE ARPA H WHITE

PAPER BUT I THINK NCATS IS

WELL-POSITIONED TO KEEP LEADING

THIS DISCUSSION AND MOVING IT

INTO THE 21ST CENTURY AND

HOPEFULLY WILL HAVE 21ST SENT

ARE I TREATMENTS AND CURES

HITTING THE MARKET AND AVAILABLE

FOR KIDS LIKE MY DAUGHTER AND

MORE.

THANK YOU.

>> THANK YOU SO MUCH, REINFORCES

WHY WE ARE GOING TO MISS YOU SO

MUCH ON COUNCIL.

HUE FOR JOINING US TODAY.

VERY WELL SAID.

ALL OF IT WAS WELL SAID AND WE

DO HAVE TINA MORRISON HERE, I

DON'T WANT IF YOU WANT TO

RESPOND QUICKLY, YOU HAVE A

COMMENT IN THE BOX.

DO YOU WANT TO SAY SOMETHING AND

UNMUTE YOURSELF?

>> THANKS FOR THE CHANCE, I

APPRECIATE IT.

CAN YOU HEAR ME OKAY?

>> WE CAN.

YES.

>> I AM GOING TO SHARE A LINK

FROM THE RECENT SUMMIT SPONSORED

BY THE CENTERS OF EXCELLENCE AND

REGULATORY SCIENCE AND

INNOVATION.

THERE WAS A REALLY IMPORTANT

PANEL DISCUSSION WITH FIVE FDA

COMMISSIONERS THE CURRENT ACTING

COMMISSIONER MODERATED THE

SESSION JANET WOODCOCK AND ONE

FASCINATING PIECE OF THE

DISCUSSION WAS FOCUSING ON RARE

DISEASE, TALKING ABOUT OUR

RELATIONSHIP WITH CMS, AND

PAYERS TO REALLY THINK ABOUT

WHAT STRUCK ME JONI IN YOUR

COMMENTS ABOUT GETTING MORE

TREATMENT TO ALL PATIENTS, OR

ALL PEOPLE, I FORGET YOUR EXACT

VISION BUT THE THING THAT CAME

TO MY MIND IS WE CAN DEVELOP THE

TREATMENTS BUT WHAT IS THE ROLE

OF NCATS AND HOW TO GET TO THE

PATIENT AND A LOT OF THAT HAS TO

DO WITH PAYERS AND CONVERSATION

WITH START WITH CMS AND JUST

THINKING THE ROLE FDA CAN PLAY

IN HELPING THAT AND I KNOW THAT

FDA HAS WORKED MY PAST

EXPERIENCES COME FROM

(INAUDIBLE) I WAS A REGULATOR

THERE FOR SEVERAL YEARS.

WE CONSTANTLY TALKED ABOUT THIS

CONVERSATION THAT NEEDS TO

HAPPEN BETWEEN FDA AND CMS AS A

START TO SAY WHAT IS THE DATA

FDA NEEDS?

WHAT IS THE DATA CMS NEEDS?

IS THERE WAY TO WORK TOGETHER SO

THAT ONCE SOMETHING LEAVES FDA

AND LANDS ON CMS DESK IS THERE A

WAY FOR US TO ANSWER THOSE

QUESTIONS FOR THEM IN THAT

PASSING OF THE BATON WHERE WE

WORK TOGETHER WHEN BATON IS

PASSED AND WE SUPPORT CMS

MISSION TO MAKE DECISIONS FASTER

BECAUSE SOMETIMES THAT IS A HUGE

HURDLE EVEN THE TREATMENTS ARE

THERE PATIENTS STILL CAN'T GET

THEM.

>> THANK YOU SO MUCH FOR THOSE

COMMENTS.

I COULDN'T AGREE MORE, THE GOAL

OR THE THREE GOALS I OUTLINE IN

MANTRA OF MORE TREATMENT TO ALL

PEOPLE MORE QUICKLY, THAT IS

NCATS CAN'T DO THAT ALONE.

IT IS ABOUT THE ENTIRE

BIOMEDICAL SYSTEM COMING

TOGETHER AND THE PATIENT AND

PATIENT ADVOCACY GROUPS COMING

TOGETHER.

KRISTINA, I APPRECIATE YOUR THE

WAY YOU CAN ARTICULATE THESE

THINGS BECAUSE YOU ARE A RARE

MOM AND YOU SEE THESE FIRSTHAND

AND I THINK THAT IS VALUABLE TO

BRING INTO THESE DISCUSSIONS.

SO THAT WE CAN FIGHT THOSE --

THE OPERATIONAL PIECE OF THIS

ARE SO IMPORTANT AND CRITICAL

FOR US TO ADDRESS THE AT THE

SAME TIME TIME WE ADDRESS

SCIENCE.

I.

A A RARE KID I GUESS.

I HAVEN'T THOUGHT ABOUT THAT HOW

TO SAY THAT BUT HOW YOU PHRASE

IT AS BEING A RARE MOM, IS SO

COMPELLING.

MY MOM HAD A RARE DISEASE, IT

WASN'T A GENETIC DISEASE, IT WAS

MORE DIFFERENT KINDS OF CANCER

RELATED CONDITIONS BUT IT WAS A

RARE DISEASE NONETHELESS SO

THESE KINDS OF ISSUES ARE JUST

REALLY IMPORTANT TO ME AND THE

MORE WE BRING THESE PEOPLE AND

COMMUNITIES TOGETHER OUR VOICES

ARE GOING TO BE LOUDER AND

LOUDER.

AND WILL CREATE THAT SWELL OF

OPPORTUNITIES COMMUNITY SO THAT

WE CAN BE SUCCESSFUL FOR THESE

GOALS.

I THINK UNFORTUNATELY I HAVE TO

CUT OFF OUR DISCUSSION BECAUSE

WE DO HAVE OUR SPECIAL GUEST WHO

HAS ARRIVED.

TARA SCHWETZ, I HOPE YOU ARE

ABLE TO BE ON SCREEN HERE.

I WANT TO QUICKLY INTRODUCE TARA

TARA.

FOR MUCH OF 2021 LAST YEAR TARA

SCHWETZ HAS BEEN ON DETAIL TO

WHITE HOUSE OFFICE OF SCIENCE

TECHNOLOGY AND POLICY AS THE

ASSISTANT DIRECTOR FOR

BIOMEDICAL SCIENCE INITIATIVES.

IN THIS ROLE SHE'S LED THE

EFFORTS TO STAND UP THE ADVANCE

RESEARCH PROJECTS AGENCY HEALTH

FOR ARPA H AND SHE'S BEEN

WORKING WITHIN THE BIDEN

ADMINISTRATION TO PROPOSE ASH PA

H TO TACKLE SOME OF THESE ISSUES

THAT WE HAVE BEEN TALKING ABOUT

AS WELL.

NOW SHE IS IN HER NEW ROLE AS

ACTING PRINCIPAL DEPUTY DIRECTOR

AT THE NIH SO WE ARE HAPPY TO

HAVE TARA BACK AT THE NIH, BUT

HER CONNECTIONS WITH THIS

PARTICULAR ARPA H OSTP LIAISON

AND DETAILING HAS -- I THINK

WILL PROVE VERY BENEFICIAL AS WE

GO FORWARD NO MATTER WHAT THAT

MIGHT LOOK LIKE.

SO TARA, I WILL TURN IT TO YOU

AND HAVE YOU GIVE US A STATUS

UPDATE ON WHAT YOU KNOW ABOUT

ARPA H.

THANK YOU SO MUCH FOR JOINING

US.

>> THAW FOR HAVING ME.

I HOPE YOU CAN HEAR ME.

I'M GOING TO PREFACE THIS I HAVE

BEEN HAVING INTERNET TROUBLE ALL

DAY ON AND OFF SO I HAVE BACK

UPON BACK UP HERE IF I NEED TO

SWITCH NETWORKS AND COMPUTERS

AND EVERYTHING ELSE.

I'M GLAD I WAS ABLE TO GET ON

COUPLE OF MINUTES EARLY TO HEAR

THE END OF THAT DISCUSSION

BECAUSE I THINK THIS TIES SO

NICELY WITH WHAT SOME OF THE

ROLES THAT ARPA H CAN PLAY

PARTICULARLY TO THE LAST

COMMENTS BECAUSE I HOPE ARPA H

IS A FACILITATOR BETWEEN THE

RESEARCH EFFORTS COMING OUT

WORKING WITH FDA AND CMS MORE

CLOSELY, THAT WILL BE A CRITICAL

ROLE: SO WE CAN HELP ADVANCE

THAT AS ARPA H HOPEFULLY GETS

PULLED TOGETHER AND IS ABLE GET

LAUNCHED.

I KNOW Y'ALL HEARD ABOUT ARPA H

BEFORE AND CLEARLY BY DISCUSSION

THIS IS NOTHING COMPLETELY NEW

FOR ALL OF YOU BUT JUST TO SORT

OF TAKE A STEP BACK AND ENSURE

EVERYONE IS ON THE SAME PAGE, I

WANT TO REMIND FOLKS ARPA H IS

ADVANCE RESEARCH PRODUCT AGENCY

FOR HEALTH.

THIS IS BEING PROPOSED AS A NEW

ENTITY THAT WILL EQUITABLY

BENEFIT HEALTH OF ALL AMERICANS

BY HELPING CATALYZE FULL BREAK

THROUGH, THE SHORT AND SIMPLE OF

IT.

IF YOU CAN GO TO THE NEXT SLIDE.

THINKING ABOUT WHAT COULD BE

DONE AND WHAT HAS BEEN DONE OVER

THE LAST SEVERAL DECADES.

WE KNOW SCIENCE HAS DONE AMAZING

THINGS.

WE ARE AT THIS REALLY

UNPRECEDENTED MOMENT OF PROGRESS

PARTICULARLY AS WE ARE STILL

MISPANDEMIC BUT WE HAVE BEEN

ABLE THE SEE SOME AMAZING

SCIENTIFIC WORK COME OUT OF WORK

DONE AND SUPPORTED BY NCATS BUT

IT TAKES CAUSES TAKE A STEP BACK

AND SAY WHAT MORE CAN WE DO TO

SPEED REVOLUTIONIZE AND

TRANSFORM MEDICINE AND IMPROVE

THE HEALTH OF ALL AMERICANS.

SO IF WE IMAGINE FOR -- FOR

INSTANCE, IF WE CAN PROGRAM

MRNA TO PRE-VACCINATE US

AGAINST 50 MOST COMMON MUTATIONS

TA DRIVE CANCER, THIS GIVES OUR

BODIES A CHANCE TO TARGET AND

ELIMINATE BEFORE THEY CAN CREATE

A TUMOR, WHAT IF WE COULD

DEVELOP DRUGS THAT HAVE PRECISE

MOLECULAR ZIP CODES THAT WOULD

ALLOW FOR UNPRECEDENTED

TARGETING ACROSS THE BLOOD BRAIN

BARRIER TO SPECIFIC CELLS IN OUR

BODY, WITHOUT ANY UNTOWARD SIDE

EFFECTS AND THESE ARE JUST

COUPLE OF EXAMPLES AND THERE ARE

OTHERS ON THE SLIDE AS WELL OF

THE POTENTIAL CAPABILITIES AND

PLATFORMS THAT COULD BE POSSIBLE

IF WE COULD HARNESS THE BEST

IDEAS AT THE RIGHT TIME WITH THE

RIGHT AMOUNT OF RESOURCES.

I'M SURE Y'ALL HAVE BETTER IDEAS

YOU ARE HIKING AS YOU LOOK AT

THIS SLIDE.

I THINK THAT FOR ME WHAT THAT

SIGNAL IS POTENTIAL FOR

OPPORTUNITIES HERE THAT WE HAVE

WITH ARPA H IS EVEN MORE

EXCITING AND REALLY TREMENDOUS.

SO WE MOVE TO THE NEXT SLIDE.

I JUST WANT TO QUICKLY GO OVER

WHAT POTENTIAL VERSION OF THE

MISSION COULD READ BECAUSE OF

COURSE AT MOMENT ARPA H IS STILL

JUST A PROPOSAL.

BUT WHAT WE HAVE HERE IS THAT

AID COULD BENEFIT THE HEALTH OF

ALL AMERICANS BY UTILIZING

HEALTH BREAK THROUGHS NOT

READILY ACCOMPLISHED THROUGH

TRADITIONAL RESEARCH OR

COMMERCIAL ACTIVITY.

TO ME THE LAST POINT IS REALLY

KEY BECAUSE WHAT WE ARE TALKING

ABOUT IS DOING THINGS THAT CAN'T

BE DONE CURRENTLY, THAT ARE

REALLY INCREDIBLY DIFFICULT TO

DO.

THAT IS THE SPACE THAT ARPA H

WANTS TO LIVE IN.

ADVANCING THIS MISSION AND THE

IDEAS MENTIONED PREVIOUSLY, IS

GOING TO REQUIRE A NOVEL

APPROACH TO MORE SUPPORTING

BROAD BIOMEDICAL RESEARCH.

AND THE IDEA WITH ARPA H IS TO

LEVERAGE --

>> WE HAVE LOST YOUR AUDIO.

HOPEFULLY YOU CAN STILL HEAR US?

>> I CAN.

>> I HEAR TARA JUST FINE.

HAVE OTHERS?

>> SAME HERE.

>> I'M HEARING HER FINE AS WELL.

>> WE CAN HEAR HER.

>> OKAY.

GOOD.

I WAS WORRIED FOR A SECOND.

I HAVE OTHER DEVICES TO SWITCH

TO IF NEEDED.

HOPEFULLY AUDIO GETS CORRECTED

ON YOUR END AND OTHERS CAN STILL

HEAR ME.

TO GO BACK TO THIS APPROACH,

THAT ARPA H IS GOING TO TAKE,

LEVERAGING WHAT WAS PIONEERED AT

DARPA, IT'S UTILIZED THERE FOR

60 PLUS YEARS, AND HAS ALSO BEEN

APPLIED IN OTHER AREAS OF R&D

MORE RECENTLY.

IF YOU THINK ABOUT ARPA E, AND

EVEN BRTA, AND WE REALLY WANT TO

EMBRACE THIS APPROACH FOR

BIOMEDICAL AND HEALTH RESEARCH.

WE PROPOSE TO CREATE THIS

DISTINCT ENTITY WITHIN NIH

THAT'S HAVE AUTONOMY AND

INDEPENDENCE RESOURCES AND

AUTHORITIES TO TACKLE SOME OF

THESE BIG CHALLENGES FACING

HUMAN HEALTH.

IN THE NEXT SLIDE WE TALK ABOUT

THIS ARPA MODEL.

FOR ANYONE WHO MAY BE LESS

FAMILIAR JUST AGAIN TO LEVEL A

LITTLE, THE MODEL EMBRACES A

PROGRAM MANAGER CENTRIC

MENTALITY, IT PROMOTES ACTIVE

PROJECT MANAGEMENT AND

ACCOUNTABILITY THROUGH

QUANTIFIABLE METRICS AN STAGE

GATING.

IT APPROACH THE ORGANIZATION

THROUGH A LENS OF BEING LEAN AND

MINIMUMMABLE BUT BUILDS IN A

FAIR AMOUNT OF AUTONOMY AND

ACCOUNTABILITY.

IT FOCUSES ON MISSION DRIVEN

RATHER THAN REQUIREMENT OR CAST

DRIVEN -- TASK DRIVEN RESEARCH.

MENT THAT WHICH IS BOLD AND

RISKY BUT COULD OFFER REALLY

HIGH RETURN.

T THAT ADHERES TO URGENT TIME

BOUND PRINCIPLES THAT ARE

APPLIED NOT JUST TO THE PROGRAM

-- BUT ALSO TO THE STAFF TENURE.

IN REFERENCE TO SORT OF THIS

HIGH RISK HIGH REWARD APPROACH I

LIKE TO GIVE THE ANALOGY THAT

WHAT WE WANT TO DO WITH ARPA H

IS ON SOME LEVEL, HITTING -- IT

IS LIKE THROWING A DART IN A

DARK ROOM.

IF YOU HIT A DARK DART BOARD, ON

THE BULLS EYE IT IS AMAZING.

AND IF YOU DON'T YOU LEARN THAT

IT IS NOT IN THAT LOCATION AND

NOW YOU CAN AIM SOMEWHERE ELSE.

BUT OBVIOUSLY BUILDING OFF AND

LEARNING FROM WHAT YOU ARE DOING

BUT HAVING THE POTENTIAL TO HAVE

A REALLY HIGH IMPACT SO IF WE

MOVE TO THE NEXT SLIDE.

TO ENUMERATE THE GOALS OF ARPA

H, POTENTIAL GOALS OF ARPA H.

THEY ARE HERE SUPPORTING

TRANSFORMATIVE HIGH RISK HIGH

REWARD RESEARCH OF COURSE BUT

DOING SO BY EMBRACING A SENSE OF

URGENCY AND SPEEDING THE

APPLICATION IMPLEMENTATION OF

BREAK THROUGHS AT VARIETY OF

DIFFERENT LEVELS.

SO WE SAY FROM THE MOLECULAR TO

THE SOCIETAL.

AND RECOGNIZING THAT IS PRETTY

BROAD BUT DOING SO IN A WAY THEY

SERVE ALL PEOPLE.

TO BUILD CAPABILITIES AND

PLATFORMS THAT ARE BROADLY

APPLICABLE ACROSS A WIDE RANGE

OF DISEASE AND CONDITIONS.

TO REVOLUTIONIZE HOW WE PREVENT

DETECT TREAT CURE THEM.

WE WANT TO FOCUS ON CONVERTING

USE DRIVEN IDEAS SO THOSE IDEAS

WITH DIRECT APPLICATION INTO THE

SOLUTION FOR PATIENTS REALLY

QUICKLY.

AND THEN HELP OVERCOME MARKET

FAILURES THROUGH CREATIVE

SOLUTIONS AND DERISKING.

THERE ARE AREAS RIPE FOR

TRANSFORMATION IF STIMULATED

THEM THROUGH THE RIGHT SUPPORT

AND COLLABORATION.

IF WE MOVE TO THE NEXT SLIDE.

THE GUIDING PRINCIPLES THAT

DRIVE ARPA H ECHO ELEMENTS OF

THE ARPA MODEL.

ARPA H IS CENTERED AROUND AND

SHOWING THIS RISK TOLERANCE OR

RISK ACCEPTANCE, AND INSTILLING

KEY FOUNDATIONAL VALUES THAT ARE

INHERENT IN THE ARPA MODEL.

BUT IN ORDER TO DO THAT WE

RECOGNIZE CULTURE IS KEY TO ARPA

H SUCCESS.

THAT IS SOMETHING NCATS WORKED

HARD TO BUILD AND DEVELOP OVER

ITS TEN PLUS YEARS NOW HISTORY.

AND THINKING ABOUT WHERE ARPA H

SHOULD LIVE, THE ADMINISTRATION

SUPPORTS ARPA H, HE PROPOSES A

COME PENT OF NIH.

AND LINKED TO NIH REALLY DRAW ON

THE VAST KNOWLEDGE EXPERTISE AND

INFRASTRUCTURE THAT CURRENTLY

EXISTS HERE AT NIH.

INCLUDING INSTITUTES LIKE NCATS

AND THINKING CONNECTIONS AND

COLLABORATIONS AND SYNERGIES

THAT WE CAN BUILD THERE.

BUT RECOGNIZING THAT IT DOES

NEED TO BE DISTINCT AND HAVE ITS

OWN CULTURE AND ORGANIZATION

AUTONOMY.

IN DOING SO ARPA H IS GOING TO

SEEK THE BRING ON THE MOST

INNOVATIVE PEOPLE WITH THE MOST

NOVEL IDEAS THAT WILL FORM

ROBUSTLY DIVERSE AND

COLLABORATIVE TEAM.

AND EQUITY IS GOING TO BE

ESSENTIAL AND GOING TO BE

CONSIDERED ESSENTIAL ELEMENT OF

ARPA H AND KEY IN ITS INTERNAL

PRACTICES LIKE HIRING AND IN THE

PROJECTS AND PROGRAMS IT

SUPPORTS, I LOOK AT THIS IN SORT

OF THREE WAYS.

RIGHT?

THINKING ABOUT ROBUST EQUITY IN

THE PEOPLE THAT HIRE, THE

PROGRAMS THAT SUPPORT, AND THE

PERFORMERS IT MAKES AWARDS TO.

CONTINUING WHICH IS DOES AROUND

PEOPLE, THE DIRECTOR OF ARPA H

AS WELL AS PROGRAM MANAGERS ARE

INTENDED TO BE TERM LIMITED AND

NOT A 20 YEAR TERM, MUCH SHORTER

TERMS.

THINKING IN THE LIKE THREE TO

FIVE YEAR RANGE.

THIS FIRST DIRECTOR, THEY ARE

GOING TO SET THE STAGE FOR SO

MANY YEARS TO COME AND IT IS

GOING TO BE REALLY CRITICAL FOR

US TO FIND THE RIGHT LEADER,

THAT'S GOING TO EXCEL

TECHNICALLY AND AS A LEADER.

IT WILL ALSO BE A REALLY FLAT

DYNAMIC ORGANIZATION BUILT

NIMBLE AND OPERATIONALIZING

SCIENTIFIC PORTFOLIO.

AND BUSINESS PROCESSES.

AGAIN WE TALK ABOUT THAT SENSE

OF URGENCY AND OPERATING UNDER

TIME BOUND PRINCIPLES.

WHAT WE MEAN BY THAT IN ADDITION

TO TERM LIMITS WHAT I ALWAYS

HEAR WHAT I HAVE HEARD MANY,

MANY TIMES FROM THE DRPA FOLKS I

HAVE SPOKEN WITH IS WHEN THEY

COME ON BOARD, THEY GET THEIR

BADGE AND THEIR BADGE HAS AN

EXPIRATION DATE ON IT AND THAT'S

THEIR TICKING TIME CLOCK.

THEY LOOK AT THAT EVERY DAY WHEN

THEY PLUG TO COMPUTER OR WHEN WE

WERE GOING BACK TO THE OFFICE

AND THEY SCANNED IT TO GET IN

THE BUILDING OR THROUGH THE GATE

OR WHATEVER.

IT WAS A CONSTANT REMINDER OF

WHAT THEY WANTED TO ACHIEVE OVER

THAT TIME PERIOD.

I THINK THAT THAT IS JUST A

REALLY NICE MANIFESTATION OF

LIKE THAT CULTURAL ASPECT.

I WILL ALSO MENTION IN TERMS OF

THINKING THROUGH TIME BOUND

PRINCIPLES.

IT IS GOING TO BE GOING TO

OPERATE WITH UTILIZING DIFFERENT

PROCESSES.

FROM THE REST OF NIH, IT LIKELY

WILL NOT BE ISSUING GRANTS BUT

MORE TAKE AGO APPROPRIATE OF

CONTRACTS AILINGOR AND

TRANSACTION AND CHALLENGES,

THINGS YOU ARE FAMILIAR WITH BUT

WANTED TO MENTION THAT.

WITHOUT DWELL ON THIS SLIDE TOO

MUCH LONGER, I JUST WANT TO

REITERATE THE IMPORTANCE OF OPEN

AND TRANSPARENT.

ABOUT PROsS AND APPROACHES AS

IT DEVELOPS THEM.

AND IMPORTANTLY, ENGAGING

STAKEHOLDERS ACROSS A WIDE RANGE

OF SECTORS AND DISCIPLINES EARLY

AND OFTEN.

IF WE ARE DEVELOPING THIS AS WE

TALK ABOUT IT WITH A FOCUS ON

USE DRIVEN RESEARCH, WE WANT TO

BE COGNIZANT ABOUT THE FACT THAT

THOSE END USERS SHOULD BE

INVOLVED AND AT THE TABLE FROM

THE BEGINNING STAGES OF

DEVELOPING A PROGRAM.

AND CONSULTING THROUGH THE

PROCESS.

AND TO MISQUOTE I GUESS, THE --

I GUESS OFTEN USED FIELD OF

DREAMS ANALOGY, IF YOU BUILD IT

THEY WILL COME, WHAT WE DON'T

WANT TO DO IS BUILD IT, HAVE

THEM COME AND THEN WHY ON EARTH

DID YOU BUILD A BASEBALL PEELED

IN THE MIDDLE OF A CORN FIELD?

AND SOMETIMES THAT WORKS AND IT

IS AMAZING BUT OTHER TIMES I ASK

WHY DIDN'T YOU BUILD A SOCCER

FIELD, THAT'S WHAT WE WOULD HAVE

WANTED.

SOMETHING COMPLETELY DIFFERENT.

NOT EVEN A FIELD TO PLAY A

SUPPORT ON SO JUST RECOGNIZING

THE IMPORTANCE OF THAT REALLY

EARLY AND FREQUENT ENGAGEMENT.

AND PROMOTING INDEPENDENCE AND

ACCOUNTABILITY THAT REINFORCED

THROUGH AMBITIOUS MILESTONES AND

METRICS.

THIS WILL BE DRIVEN THROUGH THE

PROGRAM MANAGERS PRIMARILY.

WHO WILL HAVE BROAD AUTONOMY AND

AUTHORITY TO BE CREATIVE AND

DRIVE INVASION AND TRACK METRICS

AND MAKE DECISIONS ABOUT FUTURES

OF THE PROGRAM BASED ON THOSE

METRICS.

THEN OF COURSE FOLLOWING A FAIL

FORWARD APPROACH AND EMBRACING

THE ETHOS OF IF YOU ARE GOING TO

FAIL, FAIL EARLY AND ACCEPTING

FAILURE AS A NORMAL EXPECTED

COMPONENT OF ITS OPERATIONS SO

ESSENTIALLY WHAT THIS MEANS IS

YOU ARE FAILING IF YOU ARE NOT

AT LEAST FAILING OCCASIONALLY.

IF WE CAN MOVE TO NEXT SLIDE.

I WILL CALL OUT OUT AUTHORITIES

NEEDED TO DO THIS BECAUSE THIS

IS REALLY KEY AND CRITICAL TO

SUCCESS.

MOSTLY A LOT OF THEM ANYWAY ARE

AROUND ABILITY TO HIRE AND

RECRUIT THE BEST PEOPLE.

AND HAVING FLEXIBLE FUNDING

AUTHORITIES THAT ALLOW FOR USE

OF MULTIPLE APPROACH TO

DISTRIBUTE RESOURCES.

AND TO DO SO OVER MULTIPLE

YEARS.

SO AGAIN THIS WOULD BE THINGS

LIKE OTHER TRANSACTION AND PRIZE

AUTHORITIES AND MULTI-YEAR

APPROPRIATIONS.

THE PEER REVIEW PROCESS ARE

ROBUST AND EXPERT DRIVEN BUT

DIFFERENT THAN TRADITIONAL NIH

PEER REVIEW PROCESS AND

AUTHORITIES WOULD BE IMMEDIATED

TO ENSURE THIS IS POSSIBLE FOR

ALL MECHANISMS ESPECIALLY IF

PLACED WITHIN NIH

INFRASTRUCTURE.

THERE ARE OBVIOUSLY A FEW OTHER

THINGS LIKE EXEMPTING CERTAIN

INFORMATION FROM FOIA, RELATED

TO COMPANIES BUSINESS AND

COMMERCIALIZATION INVESTMENT

PLANS AND COLLABORATING WITH AND

POTENTIALLY REIMBURSING FDA FOR

CERTAIN ACTIVITIES.

AND THIS WOULD BE A WAY TO

FACILITATE THAT INTERACTION IN A

MUCH MORE ROBUST AND DEFINED

WAY.

IF WE MOVE TO THE NEXT SLIDE.

WE ARE ASKED QUITE A BIT ABOUT

WHERE IDEAS WILL COME FROM.

SO HERE JUST TRY TO ILLUSTRATE

THAT IDEAS ARE GOING TO COME

FROM AND FORMED BY MANY

DIFFERENT SOURCES.

PATIENTS ADVOCATES, ACADEMIA,

INDUSTRY AS WELL AS OTHER

SCIENTIFIC SAKE HOLDERS LIKE

FROM ACROSS THE FEDERAL

GOVERNMENT AND IDEAS GENERATED

AND REFINED BY CONSIDERING THE

DIVERSE ARRAY OF PERSPECTIVES,

BRINGING IN PEOPLE WITH

DIFFERENT BACKGROUNDS AND

EXPERTISE.

DIFFERENT STAKEHOLDERS,

DIFFERENT SCIENTIFIC

DISCIPLINES, DIFFERENT

PARTNERSHIPS AND MORE.

THE ARPA H DIRECTOR HAS AUTONOMY

TO STEP PRIORITIES AND PROVIDE

PROGRAM MANAGER WHOSE WILL COME

BEARING IDEAS WITH THE

INDEPENDENCE TO ENACT THAT

VISION.

WITH ROBUST SUPPORT BUT OFTEN

MINIMAL INVOLVEMENT FROM THE

DIRECTOR.

ESSENTIALLY WHAT THAT MEANS IS

HIRE REALLY GOOD SMART PEOPLE

AND GIVE THEM THE FREEDOM TO DO

WHAT THEY DO BEST.

THAT'S WHAT ARPA H PROGRAM

MANAGERS WILL BE DOING, THEY

WILL BE RESPONSIBLE FOR TAKING

IN FEEDBACK FROM ALL THESE

DIFFERENT SOURCES, THAT THEY ARE

RECEIVING.

AND DRAFTING OLD PROGRAMS NEXT

SLIDE WE DON'T HAVE THE GOOD

IDEAS AND KNOW CHALLENGES AND

ISSUES FACING THE COMMUNITY WE

HAVE BEEN TRYING TO FIRST

FOREMOST SHARE INFORMATION ABOUT

ARPA H AS AVAILABLE ALSO MORE

IMPORTANTLY TO HEAR FROM THE

COMMUNITY.

TO DATE NIH AND OSTP HELD AN

INFORMATION SESSION AVAILABLE

ONLINE, PUBLISHED COMMENTARY IN

SCIENCE AND HAS HELD NUMEROUS

MEETINGS WITH DIFFERENT

ORGANIZATIONS, VARIETY OF

DIFFERENT PRESENTATIONS, SUCH AS

THIS.

WE ALSO UNDERTOOK A SERIES OF

LISTENING SESSIONS.

TO HEAR FROM DIFFERENT VOICES

ACROSS THE COMMUNITY.

GO TO NEXT SLIDE SUMMARIZE THOSE

LISTENING SESSIONS WHICH WERE

LED BY LEADERSHIP FROM ACROSS

THE OSPTP AND NIH INCLUDE ALL 27

INSTITUTE AND CENTER DIRECTORS

AND JONI PARTICIPATED IN ONE OF

THOSE.

OVER THE COURSE OF WE HAD 15

SESSIONS AND THEN ONE FEEDBACK

SESSION, 16.

WE HEARD FROM OVER 5100

STAKEHOLDERS INCLUDING NEARLY

250 ORGANIZATIONS FROM ALL OVER

THE COUNTRY.

I HOPE MANY OF YOU ARE ABLE TO

PARTICIPATE IN THOSE.

WHAT CAME OUT OF THAT IS SEVERAL

THEMES THAT WE HAVE GROUPED INTO

TWO MAIN BUCKETS.

COMMENTS ON THE NATURE OF

SCIENTIFIC PORTFOLIO AND THE KEY

ASPECT ON WHICH TO FOCUS AS WELL

AS THE ADOPTION OF STREAMLINE

YET NIMBLE PROCESSES FOR

FLEXIBILITY AND BROAD INCLUSION.

I FOR GOT TO SAY -- HERE IS AN

ANIMATION HERE BUT IF WE CAN

MOVE FORWARD TWO NOW.

THAT WOULD BE GOOD BECAUSE I

WILL TALK ABOUT THAT A LITTLE

BIT FURTHER.

SO IF WE MOVE TO THE NEXT SLIDE.

THE AREAS OF FOCUS ON THE

PORTFOLIO IN TERMS OF FEEDBACK

WE RECEIVE REALLY CENTERED

AROUND THE NEED TO COMPLIMENT

NIH RESEARCH PORTFOLIO AND NOT

DUPLICATE IT AND OF COURSE THAT

SOMETHING THAT WE OBVIOUSLY ARE

VERY KEENLY AWARE OF AND WANT TO

TAKE STEPS TO AVOID.

AND REALLY TO ALSO NOT JUST

AVOID DUPLICATION BUT IDENTIFY

AREAS FOR COLLABORATION AND

SYNERGY TO ENHANCE EFFORTS

ACROSS BOTH ORGANIZATIONS

WHETHER ARPA H OR NIH INSTITUTES

AND CENTERS.

WE ALSO HEARD ROBUSTLY ABOUT THE

IMPORTANCE AND CRITICALITY OF

ADDRESSING HEALTH INEQUITIES.

AND PROMOTING HEALTH EQUITY AND

THIS IS IMPORTANT TO THE

ADMINISTRATION BROADLY AND

SOMETHING WE IN THINKING ABOUT

SETTING UP A NEW ORGANIZATION WE

WANT TO BUILD IN TO IT FROM ITS

FOUNDATION.

WE HEARD A DESIRE TO HAVE -- TO

BUILD TECHNOLOGIES, PLATFORMS

THAT ARE REALLY BROADLY

APPLICABLE THAT ARE NOT DISEASE

SPECIFIC.

ALSO WE HEARD BROADLY ABOUT

EVERY SINGLE COMMENT YOU CAN

IMAGINE ABOUT DATA SO THINKING

ABOUT ACCESSIBILITY INTEGRATION

SHARING STANDARDS TOOLS, ET

CETERA.

HOW TO LEVERAGE AI IN CREATIVE

AND UNIQUE WAYS.

AND FOCUS ON COMMERCIALIZATION

AND PUSHING PRODUCT CLOSER TO

MARK TO GET TO PATIENTS.

WE HEARD ABOUT PROCESSES.

AND THE MAIN TAKE HOME HERE IS

THE NEED THE STREAMLINE

PROCESSES TO EXPEDITE DISCOVERY

THROUGH INTEGRATION OF THE

COMMUNITY PATIENTS AND THEIR

PROVIDERS EARLY IN THE PROCESS

AS MENTIONED BEFORE.

BUT BUILDING MULTI-DISCIPLINARY

COLLABORATION AND PARTNERSHIPS,

SO DRAWING IN SCIENTISTS FROM

OTHER DISCIPLINES, HOW WE CAN

PULL IN FOLKS FROM CHEMISTRY OR

MATH OR PHYSICS.

ENGINEERING.

I KNOW THIS IS SOMETHING THAT

THERE IS A PUSH FOR THIS ACROSS

SCIENCE, THAT WAS EMPHASIZED WE

SHOULD FOCUS ON WITH ARPA H AND

WORKING WITH INDUSTRY

NON-PROFITS AND OTHERS IN THE

PRIVATE SECTOR.

SO GO TO NEXT SLIDE.

BACK IN SEPTEMBER WE RELEASED A

REPORT SUMMARIZING THE FEEDBACK,

THAT IS AVAILABLE ON LINE, ON

THE OSTP AND I BELIEVE ALSO

POSTED ON THE HIPPOWEBSITE.

AS I MENTIONED -- NIH WEBSITE.

WE HELD A FOLLOW-UP LISTENING

AND FEEDBACK SESSION TO ENSURE

THAT WHAT WE HEARD INDICATED TO

US WE CAPTURED IN OUR ELEMENTS

AND THAT WE WERE HEARING FOLKS

CORRECTLY.

WE ALSO HOOKED TOGETHER

ADDITIONAL FEEDBACK HOW TO

ENGAGE WITH THE COMMUNITY.

IF YOU MOVE TO THE NEXT SLIDE.

JUST SORT OF UPDATE I GUESS OR

REMINDER MAYBE WHERE THINGS

STAND.

THE PRESIDENT IN HIS FY 22

BUDGET REQUEST INCLUDED

$6.5 BILLION FOR ARPA H, NOW THE

HOUSE INCLUDED ARPA H IN THEIR

DRAFT FY 22 APPROPRIATIONS BILL

AND PUT FORTH 3 BILLION IN

APPROPRIATIONS.

WE SAW SIMILAR LANGUAGE ON THE

SENATE SIDE WITH BUDGET OF

2.4 BILLION SO OF COURSE THE FY

22 APPROPRIATION HASN'T

OFFICIALLY MOVED FORWARD OR

PASSED YET SO WE ARE KIND OF IN

A WAITING STATUS THERE TO SEE

HOW THIS ALL PLAYS OUT AND WHERE

IT ENDS UP GETTING FINALIZED IN

THE MIDDLE.

IF YOU MOVE TO NEXT SLIDE IN

TERMS OF OUR AUTHORIZATION,

THERE ARE COUPLE OF OPTIONS FOR

AUTHORIZATION THAT HAVE BEEN

INTRODUCED.

THERE WAS A BILL REPRESENTATIVE

ESCHEW RELEASED IN OCTOBER AND

THE CARES 2.0 BILL THAT

REPRESENTATIVES UPTON INTRODUCED

IN OCTOBER AND WE HOPE TO SEE

MOVEMENT SOON.

ONE THING I WILL SAY IS IN

ADDITION TO PRESIDENTIAL SUPPORT

THIS INITIATIVE HAS, WE'VE HEARD

GREAT BIPARTISAN CONGRESSIONAL

SUPPORT AND SO OPTIMISTIC ABOUT

IT, ITS SUCCESS FOR HOPEFULLY FY

22 BUT FOR THE FUTURE.

SO YOU GO TO THE FINAL SLIDE I

BELIEVE.

THE TAKE HOME HERE IS THAT WE

KNOW THIS ARPA MODEL WORKS.

AND WE HAVE SEEN HOW SUCCESSFUL

DARPA HAS BEEN.

THERE ARE SO MANY AREAS RIPE FOR

TRANSFORMATION.

WE JUST NEED TO HARNESS AND

FOSTER THEM SO THAT WE CAN TAKE

THESE POTENTIAL OPPORTUNITIES TO

SHAPE THE FUTURE OF HEALTH AND

MEDICINE AND SIGNIFICANTLY

IMPROVE QUALITY OF LIFE FOR ALL

AMERICANS BY LEVERAGING THIS

MODEL WE KNOW WORKS.

SO WITH THAT JONI, I DON'T KNOW

I THINK WE STILL HAVE A FEW

MINUTES OR QUESTIONS OR

COMMENTS?

>> THANK YOU SO MUCH.

REALLY APPRECIATE IT.

WE DO HAVE A FEW MINUTES FOR

QUESTIONS.

PAUL I SEE YOUR HAND FIRST

PLEASE GO AHEAD.

>> TARA GREAT JOB.

THANK YOU FOR THAT PRESENTATION.

WHERE DO YOU SEE OR WHERE DO THE

GROUP THINKING ABOUT THIS MOST

SEE THE IC RECORDS SITTING HERE?

I'M THINKING ONE LEVEL DOWN

MAKING SURE WE DON'T HAVE THAT

DUPLICATION OF EFFORT AND

FOCUSING ON THE RIGHT PROBLEMS.

>> OVER THE SUMMER AND INTO

FALL, THE NIH LEADERSHIP

CONVENED A GROUP OF IC DIRECTORS

THINK THROUGH HOW HAY WORK

TOGETHER AND I CAN'T RECALL JONI

IF YOU WERE ON THAT GROUP.

>> YES.

>> I KNOW I SPOKE AT THEM ONCE

OR TWICE.

THEY PROVIDED SOME REALLY GREAT

FEEDBACK IN THINKING THROUGH HOW

WE CAN COORDINATE, HOW WE COULD

ENSURE THAT THERE WAS AN

EXCHANGE OF IDEAS AND EXCHANGE

OF INFORMATION ACROSS THEM.

GOING FORWARD THERE'S A COUPLE

OF AREAS WE CAN ENOR ENVISION

COLLABORATION BETWEEN ARPA H AND

ICs AND THAT'S FIRST AND

FOREMOST REVIEW PROCESS WILL BE

MOSTLY HEARD OF FEDERAL DRIVEN

PROCESS AND WE'LL NEED EXPERT

REVIEWERS TO REVIEW SOME OF THE

ARPA H APPLICATIONS.

SO THAT TO ME IS A GREAT WAY TO

HAVE INVOLVEMENT AND TO LEVERAGE

EXPERTISE OF DIFFERENT ICs.

WE ALSO KNOW THERE'S GOING TO BE

SOURCES OF IDEAS AND SO SHOULD

-- GOING TO TRY TO THINK THROUGH

A FEEDBACK LOOP IF YOU WILL FOR

DEVELOPMENT OF THOSE IDEAS AND

WHAT EXACTLY THAT MEANS AND LOOK

LIKE.

AND I THINK WITH ARPA H

ESPECIALLY AS THIS IS GETTING

DEVELOPED, THIS IS NOT LIMITED

TO THE OTHER NIH ICs BUT

BROADER ACROSS THE FEDERAL

GOVERNMENT, WE WANT TO MAKE SURE

THAT PEOPLE ARE AWARE OF AND

SUPPORTIVE OF ARPA H AND WHAT IT

IS DOING AND TRYING TO THINK

THROUGH REALLY ROBUST WAYS TO

FACILITATE STAFF INTERACTION AND

EVEN DETAILS AND OTHER WORK KIND

OF EXCHANGE OPPORTUNITIES AS WE

CONTINUE TO BUILD UP THIS EFFORT

AND WE DISCUSSED THESE ALSO ON

THE INTERAGENCY LEVEL TOO

BECAUSE WE HAVE INTERAGENCY

COMMITTEE.

>> THANKS.

RAJESH YOU HAD YOUR HAND UP.

>> AS SOMEONE WHO SORT OF WORKED

INDUSTRY CAME TO NIH THEN LEFT

NIH GO BACK TO ACADEMIA AND

INDUSTRY, I WOULD RAISE A COUPLE

OF THINGS THAT I -- YOU MIGHT

HAVE HEARD ME TALK ABOUT.

THIS MODEL OF ESSENTIALLY HIRING

WORK FORCE THAT IS TERM LIMITED

IS RUNS COUNTER TO THE

COMPENSATION SCHEME AVAILABLE IN

THE GOVERNMENT.

SO IF YOU ARE PLANNING TO HIRE

THE BIGGEST BEST AND BRIGHTEST

INCLUDING THE DIRECTOR OF ARPA

H, YOU CAN PAY A PERSON THESE

PRESIDENT SALARY, NOT SURE YOU

ARE GOING TO GET A LIST

CHARACTERS.

SO I'M VERY SKEPTICAL ABOUT THAT

PARTICULAR PIECE OF HOW YOU WILL

STRUCTURE AND IF IT WILL SIT

WITHIN THE NIH.

THE OTHER PART I GUESS LINKED TO

THE IC QUESTION, WILL THIS BE AN

IC, WILL THIS BE SAME SANDING OF

IC, WILL IT BE OFFICE?

WHAT IS IT STRUCTURE?

I WAS INVOLVED IN THE STANDING

UP OF NCATS, THERE IS A 19

WHATEVER -- 79 OR 89 LAW, 26

INSTITUTES AT NIH.

SO IF ONE NEEDS TO CREATE A NEW

ONE, ONE NEEDS TO DISAPPEAR.

LAST TIME IT WAS NCRR TO CREATE

NCATS.

SO THAT'S BEGS THE QUESTION TO

WHAT LEVEL THIS IS BEING SET UP.

LINKED TO THAT QUESTION IS NCATS

COUNCIL MEMBER, I GUESS I WOULD

JUST RAISE THE PRESENTATION AND

VALUES YOU ESPOUSED AND ETHOS

YOU WANT THERE, THESE ARE THINGS

THAT WE ARE SEEING IN PRACTICE

BEING PUT MANY PLAY BY THE NCATS

STAFF.

SO HOW SHALL I PUT IT, A

VOLKSWAGEN AND AUDI ARE ON THE

SAME CHASSI.

SO IS CAMRY AND INFINITY.

SO IF THERE IS A REQUIREMENT FOR

REBRANDING THAT WOULD MAKE A

DIFFERENCE OKAY WE CAN SEPTEMBER

THAT BUT YOU GOT A FRAMEWORK --

ACCEPT THAT, BUT YOU HAVE A

FRAMEWORK OF SOMETHING WHY NOT

USE WHAT YOU HAVE TO BUILD WHAT

YOU NEED TO DO RATHER THAN

COMPLETELY REINVENT SOMETHING

NEW.

I WORRY A LITTLE BIT ABOUT THE

NIH SYNDROME, CALL IT NOT

INVENTED HERE SYNDROME IN PLAY

HERE.

SO I'M RAISING -- BEING GLASS

HALF EMPTY HERE BUT ON BEHALF OF

N CATS I'M CONCERNED HOW ARPA H

SETS ITSELF UP FOR SUCCESS, THE

GOAL IS LAUDABLE BUT THE -- IN

AN OPERATIONAL WAY TO GET THE

RIGHT PEOPLE YOU GOT TO PAY THEM

THE RIGHT AMOUNT TO GIVE THEM

THE AUTONOMY BUT IN THE

STRUCTURE OF THE NIH HOW ARE YOU

GOING TO MAKE THAT HAPPEN?

>> I THINK A LOT OF IT TIES BACK

TO THE SHORT ANSWER FOR ALL

THREE OF THOSE QUESTIONS, REALLY

TIES BACK TO AUTHORITIES THAT

ARE NEEDED.

THESE ARE THINGS WE HAVE BEEN

WORKING VERY CLOSELY WITH THE

HILL ON IN GIVING TECHNICAL

ASSISTANCE WHERE NEEDED.

TO PROVIDE RATIONALE FOR WHY WE

DO THINK THAT IT IS IMPORTANT

FOR ARPA H TO HAVE FOR EXAMPLE

REALLY ROBUST FLEXIBLE HIRING

AUTHORITIES.

BECAUSE AS YOU INDICATED, THERE

ARE LIMITATIONS THERE.

WE HOPE SOME LEVEL ONE OF THE

THINGS THAT WE WILL ARACKET

FOLKS TO IS THE ABILITY

ESPECIALLY WITH FIRST INSTRUCTOR

TO SHAPE AND MOLD THIS

ORGANIZATION BUT ALSO THE

POTENTIAL OPPORTUNITIES THAT

EXIST SO WE ARE GOING TO BE

CONFINED BY CERTAIN

RESTRICTIONS, OPERATING WITHIN

THE FEDERAL GOVERNMENT BUT

HAVING AS FLEXIBLE OF AUTHORITY

AS POSSIBLE WILL BE REALLY

HELPFUL.

SO HA IS THE FIRST POINT.

THE SECOND QUESTION, I'M FAILING

THIS COGNITIVE TEST, WAS AROUND

REALLY -- REMIND ME.

>> ABOUT THE ICs, WHERE WILL

IT STAND, IS THE STRUCTURE.

>> YEAH.

SO THE STRUCTURAL ORGANIZATION

THAT BEING CONSIDERED RIGHT NOW,

IT WILL HINGE ON THE LANGUAGE IN

THE FINAL BILLS FOR THIS BECAUSE

AGAIN THIS IS STILL PROPOSAL

PHASE, DOESN'T EXIST, IN DRAFT

FORM.

T IT IS BEING PROPOSED AS NOT

CALLING AN INSTITUTE, IT WON'T

BE LOCATED WITHIN THE OD.

I LIKE TO REFER TO IT AS ARPA H

BECAUSE IT IS UNIQUE.

IT IS DIFFERENT BUT IT WOULD BE

AT SIMILAR LEVEL AS AN IC.

I DON'T THINK WE HAVE TO WORRY

AT ALL KIND OF THE ISSUE OF

RESTRUCTURING NIH TO ACCOUNT FOR

THAT CAP, THAT IS SOMETHING THAT

WILL BE ADDRESSED AS NEEDED

WITHIN THE AUTHORIZING LANGUAGE

AND TO YOUR LAST POINT ABOUT THE

SYNERGIES AND COMMONALITIES OF

ARPA H COUPLE OF THINGS TO POINT

OUT, ARPA H DOES NOT INTEND TO

HAVE AN INTRAMURAL PROGRAM, SO

ONE THING THAT WILL MAKE IT

SOMEWHAT DISTINCT.

AMONG THE INSTITUTES AND

CENTERS.

BUT ALSO WHAT WE ARE TALKING

ABOUT HERE TOO IS LEVERAGING

DIFFERENT APPROACH AND MIND SET

TO FUNDING BIOMEDICAL RESEARCH,

THAT DRPA OR ARPA MODEL IF YOU

WILL AND WHILE I DO THINK THERE

ARE ELEMENTS THAT I THINK RING

TRUE AND PROBABLY SOUND SOMEWHAT

FAMILIAR FROM AN NCATS SIDE

THERE ARE DISTINCT DIFFERENCES

AND IT IS SOMETHING JONI AND I

TALK ABOUT HOW TO ENSURE THE

DISTINCTION AND THE COORDINATION

BETWEEN NCATS AND ARPA H THERE

AND AGAIN WE WANT THIS TO BE

COMPLIMENTARY SO WE ARE BUILDING

OFF THE DIFFERENT EFFORTS THAT

NOT JUST NCATS BUT ALL THE

ICs.

>> MAY I ASK A FOLLOW UP

QUESTION?

>> KELLY WILL HAVE THE LAST

WORD.

GO AHEAD.

>> I GUESS IF I HAIR YOU TALKING

ABOUT THAT HOW NCATS, MAYBE I'M

JUST TOO USED TO THINKING

STRUCTURE AND HOW THEY WORK.

JUST AS BARTA COULD BE PART OF

NIH BUT IT ISN'T.

GIVEN WHAT IT DOES.

IF YOU ARE GOING TO DO THIS WHY

NOT SET IT UP UNDER HHS AS

ANOTHER AGENCY EQUAL TO NIH

RATHERTHER HAHN SUBSUMING NIH,

NOT SURE WHAT -- YOU WANT A

DIFFERENT CULTURE, YOU WANT

SOMETHING DIFFERENT PRESUMABLY

NOT GOING TO PUT ON THE THE SAME

CAMPUS SAME BUILDINGS,

SUFFICIENTLY DIFFERENTLY SO WHY

SHORN IT?

>> SHOE HORN IT?

>> THREE REASONS, IT FITS WELL

WITH NIH.

THE MISSION OF ARPA H FITS WELL

AND SQUARELY WITHIN THE MISSION

OF NIH.

SO THAT'S ONE POINT.

THE SECOND POINT I WOULD SAY IS

TO LEVERAGE EXPERTISE AND

KNOWLEDGE THAT WE HAVE AT NIH

AND REALLY HELP TO FACILITATE

COLLABORATION.

THE FURTHER REMOVED YOU ARE, THE

HARDER TO BUILD THOSE.

WE HAVE SEEN INTERACTIONS WITH

BARTA.

TAKEN A LONG TIME TO GET TO

WHERE WE CURRENTLY ARE.

THE THIRD POINT IS MAYBE THE

MOST PRACTICAL.

THAT IS THAT WE -- STARTING THIS

WITHIN AN EXISTING ORGANIZATION

LIKE NIH ALLOW US TO LEVERAGE

INFRASTRUCTURE THAT EXISTS AND

WHAT I MEAN BY THAT, IS THE IT

NETWORKS, AGREEMENTS AROUND

GETTING SPACE AND REALLY JUST

PRACTICAL THINGS THAT ALLOW US

TO GET UP AND RUNNING IN A MUCH

QUICKER TIME FRAME THAN IF WE

HAD TO START COMPLETELY FROM

SCRATCH AS A NEW ORGANIZATION

WITHIN HHS.

IT WOULD SET US BACK YEARS.

>> I HAVE ONE OTHER QUESTION BUT

WILL LET KELLY GO FIRST.

>> WE COULD PROBABLY TALK ALL

DAY.

>> WE PROBABLY COULD.

IN EX TIME WE'LL HAVE MORE TIME

FOR YOU.

>> THANK YOU, TARA, THAT IS A

NICE PRESENTATION AND EVERYBODY

LIKES TO HEAR THE IDEA OF MORE

SPEED MORE URGENCY AND MORE

IMPORTANTLY MORE BUDGET TO FOCUS

ON BETTER HEALTH AND THE SIDE OF

THIS THAT IS ALSO ABOUT GLOBAL

COMPETITION FOR THE US. YOU

JUST STARTED GOING DOWN A PATH

AND I THOUGHT OH I CAN RETRACT

MY QUESTION I DON'T NEED TO ASK

BECAUSE YOU SAID THE PRACTICAL

REASON IS REALLY ABOUT SHARED

INFRASTRUCTURE.

I WONDER WHAT REDUNDANCIES WILL

BE MANAGED BY ARPA H WHEN YOU

START TO THINK THE BREAK THROUGH

INFRASTRUCTURE ALL THE WORDS

THAT YOU USE TALKING ABOUT THE

PLATFORM FOCUS PARTNERSHIPS AND

COLLABORATIONS, I REALLY WAITING

TO HEAR WHAT IS GOING TO BE

REALLY DIFFERENT I FEEL LIKE

NCATS DOES ALL OF THAT AND I

THINK THE BREAK THROUGH

INFRASTRUCTURE THEY HAVE IN

PLACE AND I DON'T THINK THAT

THAT IS A TERM USED ANYWHERE BUT

THAT IS SORT OF WHAT HAS BEEN

SET UP OVER THE PAST DECADE,

WILL THAT BE LEVERAGED, THERE

WILL BE RECONDITION DANCIES IF

IT IS GOING TO NOT INCLUDE

INTRAMURAL RESEARCH WILL

EXTERNAL PEOPLE WORK AND

COLLABORATE TO HAVE ACCESS TO

SOME OF THESE RESOURCES THIS

INFRASTRUCTURE THAT DOESN'T

EXIST ANY PLACE ELSE IN THE

WORLD EXCEPT BIG PHARMA.

COULD YOU COMMENT ON THAT A

LITTLE BIT?

THAT GETS INTO SOME OF THE

PRACTICAL BRASS TAX THAT HAS NOT

BECOME CLEAR TO ME YET.

>> I THINK EVERYTHING YOU JUST

LAID OUT IS ABSOLUTELY CORRECT.

THE LEVERAGING THE

INFRASTRUCTURE THAT EXISTS HERE

IS GOING TO BE A KEY ELEMENT OF

ARPA H.

I THINK THAT WE WOULD EXPAND

THAT EVEN FURTHER TO TALK ABOUT

SOME OF THE INFRASTRUCTURE THAT

EXISTS ACROSS THE FEDERAL

GOVERNMENT BECAUSE I THINK THERE

IS A WHOLE LOT MORE WE COULD DO

IN OPERATING IN SORT OF MUCH

BETTER COORDINATION AS A WHOLE.

EXACTLY WHAT YOU SAID, THE IDEA

IS IS THAT WE WOULDN'T BE

SETTING UP OUR OWN CLINICAL

TRIAL NETWORKS OR OTHER THINGS

THAT ALREADY EXIST WITHIN NIH

BUT RATHER I VIEW IT AS A

FEEDBACK LOOP.

MAYBE EXAMPLE THIS IS NOT

LIMITING BUT SOME OF THE REALLY

INTERESTING AND EXCITING

FUNDAMENTAL BASIC RESEARCH

THAT'S COMING OUT OF THE

INSTITUTES FEEDS INTO OR ALLOWS

FOR THE IDEA ARPA H HAS TO MOVE

FORWARD AND MAKE STRIDES AND

ADVANCEMENTS AND THEN MAYBE YOU

TAKE THAT AND GET THAT TO PROOF

OF CONCEPT STAGE, AND YOU

LEVERAGE AN ICs CLINICAL TRIAL

NETWORK AND WORK WITH FDA AND

CMS AND OTHERS ACROSS THE

FEDERAL GOVERNMENT TO MOVE THAT

FORWARD IN A ROBUST WAY.

THERE MIGHT BE THEN AN

OPPORTUNITY AT THE END TO

OBVIOUSLY THEN HAVE ANOTHER

MAYBE EVEN ARPA H PROJECT WHERE

WHAT DOES THAT LOOK LIKE TO

ADDRESS SOME MAYBE LARGE SCALE

NATIONAL CHALLENGES TOWARDS

IMPLEMENTATION.

I THINK THE OTHER THING TO POINT

OUT TOO IS THAT WITH ARPA H WHAT

WE ARE TALKING ABOUT IS

BIOMEDICAL AND HEALTH RESEARCH,

WE USE THAT TERMINOLOGY REALLY

BROADLY BECAUSE WE SORT OF VIEW

IT FROM REALLY LARGE SPECTRUM OF

THE TYPE OF RESEARCH THIS WILL

SUPPORT AND GET INTO

IMPLEMENTATION AND DISSEMINATION

AND HOW THESE THINGS GET OUT TO

PATIENTS.

>> THANK YOU.

>> LAST QUESTION OR IMMEDIATE TO

CLOSE OUT?

>> WE NEED TO CLOSE OUT.

GO AHEAD, ASK YOUR QUESTION

PLEASE.

>> I WANT TO COME TO MONEY.

SO YOU ARE MAKING THIS ASK IN

THE BUDGET WHATEVER THE NUMBERS

YOU SAID WHETHER IT IS WE HEARD

DIFFERENT NUMBERS THROWN AROUND,

3 BILLION, 6 BILLION WHATEVER

THAT MIGHT BE.

HARKENING BACK TO EXPERIENCE

STARTING N CATS AND WHAT WE GOT

INITIALLY AND HOW WE GET IT TO

THE POINT AND ONE OF THE THINGS

THAT WAS A THIRD RAIL IN THE

ORGANIZATION WHEN NCATS WAS

STOOD UP IS THE IDEA THAT HE PUT

IN ANY WAY CANNIBALIZE ANY OTHER

IC BUDGET SO IN A SENSE THAT

THERE IS A ZERO SUM CONCERN THAT

THIS HAD TO BE NEW MONEY HA

COMES IN TO DO THIS.

SO ARE WE SAYING THAT THAT IS

WHAT REALLY IS GOING TO HAPPEN

WITH THE POLITICAL CLIMATE

CERTAINLY SUPPORT ONE FIFTH OF

THE NIH BUDGET TO BE ARPA H,

THAT'S THE SIZE OF ESSENTIALLY

THE LARGEST INSTITUTE LIKE NCI

AND NIAID WOULD BE STOOD UP FROM

THE GET GO.

>> I MEAN IF YOU -- LOOK AT THE

DRAFT LEGISLATION, IT IS ALL

DRAFT LANGUAGE IN TERMS OF

APPROPRIATION.

IT IS NEW MONEY, IT IS NOT

CANNIBALIZING FROM THE NIH

BUDGET.

WE WOULD NOT ADVOCATE FOR THAT

IN FACT WE DIDN'T, WE NAVIGATED

HARD THAT THAT SHOULDN'T BE THE

CASE.

IF FACT I THINK BOTH THE HOUSE

AND SENATE GAVE THE NIH

INCREASES ON TOP OF THE FY 21

BUDGET FOR FY 22.

THIS IS ALL DRAFT LANGUAGE,

NOTHING HAS BEEN PASSED YET.

>> THANKS, THAT WAS TERRIFIC.

YOU EXPERTLY ANSWERED A LOT OF

QUESTIONS AND WE REALLY

APPRECIATE YOUR TIME HERE.

THANK YOU FOR THE PRESENTATION.

WE LOOK FORWARD TO SAYING ALONG

WITH YOU HOW WE UPHOLD THIS

GOING FORWARD AND HOPEFULLY WE

CAN HAVE THIS BACK SOON AT

COUNCIL.

>> I APPRECIATE COPY OF THAT

REPORT MENTIONED AT THE

BEGINNING, WOULD LOVE TO SEE

THAT.

THANK YOU FOR INVITING ME.

APPRECIATE THE ROBUST DISCUSSION

AND I'M ASSURE JONI YOU AND I

WILL TALK AGAIN.

TAKE CARE.

>> THANK YOU.

>> THANK YOU SO MUCH.

WITH THAT WE DID GET A LITTLE

OVER IN TIME SO HOPE WE CAN MAKE

UP FOR THAT IN THE NEXT COUPLE

OF PRESENTATIONS.

BUT WE ARE GOING TO MOVE ON TO

LILY PORTILLA I BELIEVE IS NEXT

TO TALK ABOUT THE OFFICE OF

STRATEGIC ALLIANCE.

SO LILY PLEASE GO AHEAD.

>> CAN YOU HEAR ME?

>> YEP.

>> GREAT.

HI, I'M LILY PORTILLA, DIRECTOR

OF STRATEGIC ALLIANCES AT NCATS

AND JUST TO ORIENT TO COUNCIL

OUR OFFICE IS IN CHARGE OF ALL

THE AGREEMENT AND PARTNERSHIP

ESTABLISHMENTS THAT GO ON ACROSS

THE CENTER, THE OTHER THING WE

DO IS ALSO RUN THE SBIR STTR

PROGRAM AND THE FOCUS OF MY

CONVERSATION TODAY OR MY

PRESENTATION TODAY IS GOING TO

BE ON THE SBR STTR PROGRAM AND

OUR EFFORTS AROUND THAT SPECIFIC

PROGRAM, WITH THAT I WILL START.

NEXT SLIDE PLEASE.

SO THE NIH SBIR STTR PROGRAMS

LARGEST SOURCES OF EARLY STAGE

SEED FUNDING IN THE COUNTRY.

BUDGET IS WELL OVER A BILLION

DOLLARS NOW THAT ACROSS THE NIH

THAT HAS TO GO TO U.S. SMALL

BUSINESSES WORKING IN THE

BIOMEDICAL SPACE AND 24

INSTITUTES INCLUDING NCATS HAVE

BUDGETS THAT SUPPORT THE SBIR

STTR PROGRAM.

IT IS A 3.65% SET ASIDE, IT IS A

CONGRESSIONALLY MANDATED

PROGRAM, IT IS NOT MONEY THAT

CAN BE REPROGRAMMED TO OTHER

GRANT LINES OR CONTRACT LINES,

IT HAS TO BE USED IN SUPPORT OF

U.S. SMALL BUSINESS.

LET'S GO TO NEXT SLIDE.

LET'S CLICK ONE MORE TIME.

MAJORITY OF APPLICATIONS THREE

WAYS APPLICATIONS COME IN FOR

THIS PROGRAM.

THE MAJORITY OF THE APPLICATIONS

COME IN WHAT'S CALLED THE

OMNIBUS THAT IS 24 PARTICIPATING

INSTITUTES UTILIZES WELL AS OUR

SISTER AGENCIES AT THE CDC AND

FDA, OTHER PARTS OF HHS.

I WOULD SAY ABOUT 90%

APPLICATIONS THAT WE GET IN ARE

FUNDED THROUGH THE OMNIBUS WHICH

IS BROAD TOPICS ACROSS THE NIH

NCATS HAS LISTED THOSE RESEARCH

PRIORITIES AS PART OF THE

OMNIBUS SOLICITATION.

THE OTHER WAY WE GET

APPLICATIONS IS LIEU TARGETED

SOLICITATION, WE HAVE A FEW OF

THEM WE GENERATED HERE AT N

CATS, WE ALSO PARTICIPATE IN

SOLICITATION OTHER INSTITUTES

HAVE PUT TOGETHER.

ONCE A YEAR THERE IS A CONTRACT

SOLICITATION AND THIS IS WHEN

SPECIFIC DELIVERABLE -- WE

BELIEVE A SPECIFIC DELIVERABLE

CAN BE TARGETED BY SMALL

BUSINESS AND I WANT TO CLARIFY

THAT IT IS NOT LIKE A SERVICE,

WE ARE NOT ASKING FOR A SERVICE

TO BE PROVIDED BY A SMALL

BUSINESS BUT WE ARE SAYING THAT

WE THINK THERE'S A TECHNOLOGY

SOLUTION PROVIDED BY SMALL

BUSINESS AND THE CONTRACT

MECHANISM WHICH HAPPENS ONCE A

YEAR, IS BEST WAY THE GET THOSE,

WE BROAD THOSE TO YOU ALL AND

COUNCIL THE APPROVE.

SBIR STTR HAS SEVERAL BENEFITS

AS I MENTIONED LEAD TO OVERALL

BUDGET SO IT IS STABLE AND

PREDICTABLE,IT IS NON-DILUTIVE.

THE GOVERNMENT DOESN'T TAKE IP

RIGHTS DEVELOPED UNDER THIS

PROGRAM.

ONCE IN THE PROGRAM, ONCE YOU

RECEIVE A GRANT YOU GET ACCESS

TO SOME TECHNICAL ASSISTANCE AND

COMMERCIALIZATION PROGRAMS THAT

THE NIH OFFERS ALONGSIDE OF WITH

THE GRANT WE FOR EXAMPLE OFFER

COMPANIES OPPORTUNITY TO GO

THROUGH INNOVATION CORE TRAINING

PROGRAM.

WE WILL PAY FOR THEM TO DO THAT.

WE ALSO NIH HOST IN LARGE

CONFERENCES LIKE BIO AND ABMED

HAVE SPACE WHERE MANY COMPANIES

CAN DO PITCHES TO POTENTIAL

INVESTORS AS WELL TOO, THAT IS

ANOTHER BENEFIT OF BEING IN THE

PROGRAM.

I BELIEVE I MENTIONED THAT

BEFORE THAT, ONE OF THE OTHER

BENEFITS IS THAT THE PROGRAM --

THE GRANTS AND CONTRACTS GO

THROUGH RIGOROUS PEER REVIEW

PROCESS, WHICH THEY CAN THEN

LEVERAGE FOR ADDITIONAL FUNDING

THROUGH OTHER SOURCES, PRIVATE

INVESTMENT, AND EVEN STATE

ECONOMIC ORGANIZATIONS HAVE

COMPLIMENTARY PROGRAMS ONCE YOU

GET AT NIH SBIR STTR.

IT IS THREE PHASE PROGRAM, PHASE

1 IS FEASIBILITY STUDIES AND

THERE ARE PROGRAM CAPS BUDGET

CAPS THAT THE NIH HAS TO ADHERE

TO THAT ARE SET BY THE SMALL

BUSINESS ADMINISTRATION.

HOWEVER, THERE ARE SOME TOPICS

ACROSS THE NIH THAT WE HAVE

ALLOWED HIGHER BUDGET DOLLAR

AMOUNTS, PHASE 2 IS MORE FULL

ADD PROGRAMS WE CAN GO UP TO 2

MILLION OVER PROJECTS THAT LAST

BETWEEN TWO TO THREE YEARS.

THE FAST TRACK THAT COMBINES

BOTH THE PHASE 1 AND PHASE 2 ONE

REVIEW THE COMPANY HAS TO GO

THROUGH.

DIRECT TO PHASE 2 WHICH ALLOWS

YOU TO SKIP THE PHASE 1 IF YOU

HAVE ENOUGH DATA YOU HAVE GOTTEN

UNDER FEASIBILITY STUDY.

AND THE PHASE 2B COMPETING

RENEWAL PROGRAM WE OFFER TO ONLY

COMPANIES THAT WE FUND IN PHASE

2.

UNLIKE OTHER AGENCIES WITH STIR

SBIR, WE DO NOT HAVE A PHASE 3

PROGRAM BUT WE PRESUME COMPANIES

ARE GRADUATING OUT OF THE

PROGRAM AND PSYCHING PRIVATE

SOURCES OF FUNDING, OR

DEVELOPING STRATEGIC

PARTNERSHIPS.

HOPEFULLY THE GRANT HAS GOT TO

THAT KEY INFLECTION POINT WHERE

THEY CAN DO THAT.

NEXT SLIDE.

THERE ARE -- I WON'T GO THROUGH

THE DIFFERENCES BETWEEN

GUIDELINE AND ELIGIBILITY

REQUIREMENTS BUT THERE ARE --

THE MAIN DIFFERENCE BETWEEN THE

TWO OF SBIR AND STTR IS THE STTR

HAS -- MUST HAVE A RESEARCH

INSTITUTION COMPONENT TO IT.

IN THAT INSTANCE THE PI CAN

EITHER WORK FOR THE RESEARCH

INSTITUTION OR CAN WORK FOR THE

SMALL BUSINESS.

BUT KEEP IN MIND THAT THE GRANT

ALWAYS HAS TO GO TO THE SMALL

BUSINESS.

THERE ARE WORK REQUIREMENTS

DIFFERENCE BETWEEN PROGRAMS NOT

ALL GRANT CAN BE OUTSOURCED,

MANY TIMES WE HAVE VIRTUAL

COMPANY ASKING IF THEY WANT TO

PARTICIPATE IN THE PROGRAM BUT

THERE HAS TO BE WORK DONE WITHIN

THAT SMALL BUSINESS IN ORDER TO

BE ELIGIBLE TO RECEIVE THE

AWARD.

NOW I WILL GET INTO INITIATIVES

AND PROGRAM CHANGES ACROSS THE

NIH.

NEXT SLIDE.

THERE HAVE BEEN CHANGES TO THE

BUDGET CAPS, THERE WAS A PERIOD

OF TIME THERE WHERE THE BUDGET

CAP STAYED STATIC OF SMALL

BUSINESS ADMINISTRATION OVER

SEVERAL YEARS HAS INCREASED THAT

WE ARE NOW UP TO 275 AND CHANGE

FOR PHASE 1 AND 1.8 MILLION FOR

PHASE 2.

THERE ARE TOPICS AT THE NIH DOES

ALLOW FOR HIGHER BUDGET AMOUNTS

AND THEY ARE CALLED WAIVER

TOPICS.

EACH INSTITUTE HAS THEIR WAIVER

TOPICS THAT YOU CAN APPLY UNDER

AND FOR US THE WAIVER TOPICS

ALLOW YOU TO GO UP TO 325,000

FOR PHASE 1.

2 MILLION FOR PHASE 2.

WE THINK THAT THAT IS WARRANTED

FOR SOME TOPICS BUT WE BELIEVE

OUR BUDGET CAN SUPPORT THESE

HIGHER BUDGET DOLLAR AMOUNTS.

NEXT SLIDE PLEASE.

THERE'S ALSO CHANGES AROUND

TECHNICAL AND BUSINESS

ASSISTANCE PROGRAM.

COMPANIES NOW REQUEST IN

ADDITION TO THE -- THEIR BUDGET

THEY CAN ASK FOR 6,500 PER YEAR

PHASE 1 AND 50,000 FOR PHASE 2,

IT WOULD ALLOW THE COMPANY TO DO

THINGS LIKE MARKET ANALYSIS, OF

TECHNOLOGY, PATENT LANDSCAPE OR

FILING PATENTS BUT THIS IS A NEW

INITIATIVE THAT IS STARTED NIH

OFFERS COMPANIES THAT ARE

INTERESTED CONSULTING SERVICES

TO FIGURE TECHNOLOGY TO A

INFLECTION POINT TO GET

STRATEGIC PARTNERSHIP OR

INVESTMENT BUT THE TECHNICAL

ASSISTANCE FUNDING IS VERY

HELPFUL IN TERMS OF DOING THAT.

NEXT SLIDE PLEASE.

I ALSO WANT TO POINT OUT THAT

LIKE OTHER NIH GRANT PROGRAMS

SBIR STTR TAKES ADMINISTRATIVE

SUPPLEMENTS TO PROMOTE

DIVERSITY.

IN THIS INSTANCE THE -- THIS

PARTICULAR ADMINISTRATIVE

SUPPLEMENT IS IN SUPPORT OF

INDIVIDUALS WHO WANT TO WORK IN

SMALL BUSINESS AND GET

EXPERIENCE AS PART OF THE C

SUITE OR DOING RESEARCH FOR THE

SMALL BUSINESS AND I WANT TO

NOTE THIS IS A SPECIAL

ADMINISTRATIVE SUPPLEMENT ONLY

FOR SBIR STTR COMPANIES.

WE ALSO PARTICIPATE WITH NINE

OTHER INSTITUTES IN THE

APPLICANT ASSISTANCE PROGRAM AND

HERE APPLICANTS THAT HAVE EITHER

NEVER APPLIED TO THE NIH OR

APPLIED NOT SUCCESSFUL AND GIVEN

A MENTOR TO WORK WITH PUTTING

APPLICATION TOGETHER, HONING

TOPICS AND THE CONSULTANT THAT

WORKS WITH THEM HAS A LOT OF

EXPERIENCE WORKING WITH --

HELPING COMPANIES WITH NIH

GRANTS AND WHAT IT DOESN'T DO,

IT DOESN'T WRITE THE GRANT FOR

THEM BUT GIVES THEM THE TOOLS

AND KNOWLEDGE NEEDED TO BE

SUCCESSFUL IN GETTING NIH

FUNDING.

THE PROGRAM DOES HAVE A FOCUS ON

SUPPORTING WOMEN AND MINORITY

OWNED SMALL BUSINESSES AS WELL.

TO THE NEXT SLIDE.

HERE WE KIND OF TALK I THINK I

MENTIONED A FEW THINGS HERE,

THAT IF YOU ARE TO BE ELIGIBLE

YOU HAVE NEVER GOTTEN NIH GRANT,

COMPANY DOESN'T HAVE CURRENT

APPLICATION UNDER REVIEW, WE

AGAIN TRY TO MAKE SURE THAT

THESE INTERESTS OF FOCUS OF THE

APPLICATIONS ALIGN WITH THOSE

INTEREST OF THE PARTICIPATING

INSTITUTES.

MENT WE ARE LOOKING AT THE

PROGRAM OVERALL TO DETERMINE

WHETHER IT IS SUCCESSFUL AND

HOPEFULLY NEXT TIME WE MEET I

WILL BE ABLE TO PROVIDE MORE

DATA AROUND THAT.

WE FOLLOW SOMETHING LIKE REALLY

WOULD BE A GOOD INVESTMENT OF

TIME AND ENERGY TO TRY TO FIGURE

OUT HOW TO HELP CERTAIN

APPLICANTS GET OVER THIS HUMP OF

GITTING FUNDING FOR UNDER THIS

SPECIFIC PROGRAM.

SO INITIATIVES WE ARE DOING

AROUND OUTREACH AND DRIVING

AWARENESS TO THE NCATS SBIR STTR

PROGRAM, NEXT SLIDE SO OUR

OBJECTIVES ARE GETTING PEOPLE TO

BE AWARE OF WHAT TYPES OF

PROJECTS WE ARE FUNDING UNDER

THE PROGRAM, TO INCREASE THE

NUMBER OF APPLICATIONS THAT

WOMEN OWN AND MINORITY OWNED

SMALL BUSINESSES APPLY TO US

INCREASING THOSE NUMBERS OF

QUALITY APPLICATIONS, ALSO SMALL

BUSINESSES IN STATES THAT

TYPICALLY DON'T GET A LOT OF NIH

FUNDING EITHER TRYING TO FOCUS

EFFORTS AND OUTREACH AROUND THAT

TOO, IN GENERAL ADVANCING

INVASION AND DRUG DEVELOPMENT

DISCOVERY RESEARCH TOOLS AND

TECHNOLOGIES THAT IMPROVE

PATIENT CARE AND THOSE RESEARCH

TOPICS THAT ARE OF GREAT

IMPORTANCE AND PRIORITY TO

NCATS. NEXT SLIDE.

THIS PAST YEAR EVEN THOUGH WE

WEREN'T ABLE TO DO IN PERSON

OUTREACH WE DID A FAIR AMOUNT OF

WEBINARS WE WERE QUITE BUSY AND

PARTNERED WITH VARIOUS GROUPS TO

GET WEBINARS IN PLACE, STATE BIO

ORGANIZATIONS, AS WELL AS

ACADEMIC TECH TRANSFER OFFICES,

INNOVATION HUBS AND ACCELERATORS

AND GOVERNMENTAL PARTNERS WE

WORKED WITH TO DO JOINT OUTREACH

EVENTS.

I WANT TO POINT OUT WE HAVE BEEN

BUSY THOUGH NOT ABLE TO DO IN

PERSON OUTREACH.

THIS PAST YEAR WE WERE ABLE TO

GET PARTICIPATION TO WEBINARS

FROM 45 STATES, AND 45 SATES

WERE IDEA STATES, WHICH WE WERE

PURPOSEFULLY TARGETING.

THIS PAST YEAR WE TALKED TO A

HUNDRED APPLICANTS AND -- I WILL

TALK ABOUT HOW WE TRACK WHAT

HAPPENS AFTER THOSE

CONVERSATIONS WHAT HAPPENS TO

THE APPLICANTS BUT THIS YEAR WE

WANT TO TARGET OTHER STATES WE

WEREN'T ABLE TO MAKE IN ROADS IN

AND THERE'S ONLY A FEW OF THEM

HERE THAT WE HAVE LISTED.

BUT THAT IS PRIORITY NUMBER ONE

GOING INTO FY 22.

NEXT SLIDE.

WE HAVE A SOCIAL ENGAGEMENT

CALENDAR WE PUT TOGETHER EVERY

MONTH WITH OUR COMS TEAM.

WE HAVE GOTTEN TRAFFIC AS A

RESULT OF THE EFFORTS WE HAVE

BEEN DOING EVERY TUESDAY WE HAVE

A TIP TUESDAY SOCIAL ENGAGEMENT

THAT GOES OUT ABOUT HOW TO PUT

IN AN APPLICATION TOGETHER,

ADVISING APPLICANTS WHAT TO DO

FOR PARTICULAR FUNDING

ANNOUNCEMENT.

WE HIGHLIGHT NCATS STAFF

INVOLVED WITH THE PROGRAM

UPCOMING WEBINARS.

ANOTHER THING THAT IS VERY

SUCCESSFUL ARE THESE QUOTE CARDS

HIGHLIGHT SOME OF OUR GRANTEES

AND SPECIFICALLY SOME HIGHLIGHT

AROUND COMPANIES, MILESTONE THEY

REACHED IF WE CAN GO TO THE NEXT

SLIDE.

BY WAY OF EXAMPLE, THIS PAST

YEAR WE HAVE WORKED CLOSELY WITH

OUR PROGRAM OFFICERS AND ALSO

HAVE ALERTS SO WE KNOW WHEN

COMPANIES WE HAVE FUNDED HAVE

GOTTEN ACANNINGAL FUNDING OR

CREATED A STRATEGIC PARTNERSHIP,

WE TALKED TO THAT GRANTEE, TO

FIND OUT WHAT IS GOING ON.

AND AMPLIFY THE FACT THAT THEY

HAVE REACHED A MILESTONE.

GREAT WAY ENGAGING COMPANY BUT

ALSO OTHERS ABOUT GOOD WORK WE

ARE DOING WITH PROGRAM AND

ENTREPRENEURS THAT WE ARE

FOCUSING AS PART OF THE SOCIAL

ENGAGEMENT OPPORTUNITIES HERE.

NEXT SLIDE.

/TALK ABOUT FACT WE HAVE HAD --

WE DID 100 INDIVIDUALCALS WITH

INDIVIDUALS WHO NEEDED TO TALK

TO US ABOUT PROJECTS AND WE --

WHEN WE FIRST STARTED OUT WE HAD

A HARD TIME TRACKING WHO WE

SPOKE TO BUT MORE IMPORTANTLY

WHAT HAPPENED AFTER THAT

CONVERSATION.

SO NOW WE ARE UTILIZING SALES

FORCE TO GIVE US AN IDEA OF WHEN

THESE CONVERSATIONS HAPPEN, HOW

LONG DOES IT TAKE THE APPLICANT

TO I APPLY TO THE PROGRAM AND

ONCE THEY APPLY, HOW SUCCESSFUL

WERE THEY.

ARE THEY APPLYING FOR OTHER

GRANTS AND NOW THIS -- OUR SALES

FORCE APPLICATION IS ABLE TO

TALK TO THE NIH SYSTEM SO WE ARE

ABLE TO SEE REAL TIME GRANTS AT

THESE POTENTIAL APPLICANTS HAVE

AND TRACK WHAT CONVERSATIONS

HAVE LED TO.

IT IS A GREAT TOOL TO KEEP TRACK

OF CERTAIN CAMPAIGNS THAT WE ARE

DOING WEBINARS, WHAT'S BEEN THE

INTEREST AROUND PARTICIPATION

POST WEBINAR WITH FOLKS COMING

TO NCATS, APPLYING TO THE

PROGRAM.

I WILL GIVE DATA, I TALKED

INITIATIVES AROUND THE PROGRAM

SO NOW THIS TIME THE TALK TO

DATA SO LET'S DO THAT.

QUICKLY TO GIVE YOU SENSE OF

PROGRAM, IT IS 25 MILLION THAT

GOES TO FUNDING SMALL BUSINESSES

SBIR STTR.

AT ANY GIVEN YEAR E WE HAVE

ABOUT I WOULD SAY LIKE A 15 TO

80 -- 15 OR 20% OF OUR BUDGET

GOES TO FUNDING THOSE SBIR

CONTRACTS THAT I MENTIONED

EARLIER AND THE REST REALLY GOES

TOWARDS SUPPORTING THE GRANT

PROGRAM.

TO THE NEXT SLIDE.

IF YOU WERE TO LOOK AT OUR

PORTFOLIO THIS PACIFIST WALL

YEAR I WOULD SAY THAT THEY FALL

INTO FOUR BUCKETS THE MAJORITY

ARE AROUND TOOLS AND

TECHNOLOGIES FOR DRUG DISCOVERY

AND DEVELOPMENT, IT IS A BROAD

TOPIC, WE ALSO HAVE DEVICE

PORTION OF OUR PORTFOLIO A LOT

OF OUR TISSUE CHIP FALLS UNDER

THAT SLIVER OF DEVICE.

GENE THERAPY BECAUSE WE HAVE A

FOCUS PROGRAM ANNOUNCEMENT

AROUND GENE THERAPY AND THEN THE

REST AROUND BIOINFORMATICS.

SO THIS IS SHOWING OVER TIME THE

NUMBERS OF APPLICATIONS THAT WE

HAVE BEEN GETTING IN AND COUPLE

OF OBSERVATIONS.

ONE WE DON'T TALK ABOUT THIS

PROGRAM WE DON'T SEE -- PEOPLE

DON'T APPLY SO YOU HAVE TO IN

ORDER TO GET PEOPLE/NEW FRESH

GRANTEES TO THE MIX YOU NEED TO

BE TALKING ABOUT THE PROGRAM AND

DOING OUTREACH ON A PRETTY

CONSISTENT BASIS.

THE OTHER THING WE NOTICED WAS A

DROP IN 2020 OF APPLICATIONS AND

MANY APPLICANTS THOUGH HOME

BOUND HAD TIME, THEY HAD OTHER

RESPONSIBILITIES, I KNOW THAT

ANECDOTALLY SEVERAL APPLICANTS

SAID IT WAS HARD TO WRITE A

GRANT WHEN THEY WERE HAVING TO

TAKE CARE OF KIDS OR PARENTS

DURING COVID.

SO WE DID SEE A DROP IN

APPLICATIONS HAPPENING AND ALSO

SOME OF OUR COMPANIES WERE

IMPACTED BY THE FACT THAT THEY

COULDN'T HIRE DURING THE

PANDEMIC SO THAT DELAYED THEM

PERHAPS MOVING FROM THE PHASE 1

TO THE PHASE 2.

IN 2021 BESTARTED TO SEE AN

INCREASE BACK -- WE STARTED TO

SEE AN INCREASE BACK AGAIN IN

NUMBER OF APPLICATIONS THAT CAME

TO US.

NEXT SLIDE.

HERE WE ARE FOCUSING ON AWARDED

APPLICATIONS.

TYPICALLY AT ANY GIVEN TIME I

THINK WE RUN ANYWHERE BETWEEN 25

TO 35 APPLICATIONS THAT WE --

NEW APPLICATIONS WE FUND EVERY

YEAR.

NEXT SLIDE.

HOW DO -- WHAT ARE -- DOES OUR

PRIORITY SCORE, WHAT ARE WE

FUNDING THE QUESTION WE GET

OFTEN AND I THINK OVER TIME WE

HAVE SEEN IMPROVEMENT IN THE

AVERAGE PRIORITY SCORES NCATS

FUNDS IN SBIR, AGAIN PRETTY

PREDICTABLE WHAT OUR BUDGET IS

GOING TO BE SO WE KNOW WHETHER

KIND OF OUTREACH THAT WE HAVE TO

DO IN OUR APPLICANTS -- WE HAVE

REPEAT GRANTEES WHO COME BACK TO

US AND FUND VARIOUS PROJECTS.

SO I DO THINK THAT IT IS

LEVELING OFF AND THIS IS KIND OF

WHAT FY 20 -- FY 21 WAS VERY

COMPETITIVE YEAR.

BECAUSE WE HAVE SEEN OUR

APPLICANTS NOT COME IN FOR

ADDITIONAL FUNDING BECAUSE THEY

HAVEN'T BEEN ABLE TO FINISH

THEIR AIMS AND GRANT BECAUSE OF

COVID.

I DO EXPECT IT TO BE A DIFFERENT

NUMBER THIS NEXT IN THISY 2022.

WE WILL SEE WHAT HAPPENS SO WHAT

DO WE LOOK LIKE COMPARED TO THE

REST OF NIH?

NEXT SLIDE.

WE ARE CLOSE.

SOME YEARS WE ARE DOING BETTER

THAN WHAT NIH AVERAGE PRIORITY

SCORES ARE FOR SBIR AND SOME

YEARS WE ARE DOING A LITTLE BIT

WORSE.

CONSISTENTLY WE ARE STAYING

ALONG THE LINES OF WHAT NIH

FUNDS FOR SBIR.

TO THE NEXT SLIDE.

EYE A LITTLE BIT DIFFERENT.

COUPLE OF REASONS.

ONE IS THAT OUR BUDGET IS NOT

THAT HIGH FOR STTR SO OUR

APPLICANTS COME IN AND FIGURE

AND SAY I WILL TAKE A CHANCE AND

GOING TO APPLY FOR SBIR BECAUSE

I FEEL LIKE I HAVE A BETTER

CHANCE THERE.

SOME YEARS WHAT WE SEE IS DROP

OFF IN NUMBER OF APPLICATIONS

THAT COME IN UNDER SPECIFIC --

UNDER STTR.

OTHER YEARS WE SEE A FEW PHASE

2s TAKE UM THE BUDGET WHICH

LIMITS WHAT WE CAN FUND. SO

FREE YEAR-TO-YEAR THERE IS

FLUCTUATION WITH WHAT WE FUND

UNDER THE PROGRAM.

SHOWING HOW WE ARE DOING WITH

REST OF NIH, I THINK IT HAS BEEN

A LITTLE ALL OVER PLACE.

DEPENDS ON WHETHER THERE'S A

STREAM OF APPLICATIONS COMING

IN, WHETHER THERE'S PHASE 2s

TO FUND.

APPLICANTS COMING IN SAYING I'M

GOING TO PUT TOGETHER ABIR

VERSUS STTR.

ANOTHER THING WE HAVE BEEN

TRACKING TO IS WOMEN OWNED AND

MINORITY OWNED SMALL BUSINESSES

COMING AND APPLYING.

DROVES A DROP OFF IN 2019.

WE ACCOUNT THAT TO CHANGE IN HOW

DATA IS COLLECTED.

THE DATA USED TO BE COLLECTED AT

THE NIH LEVEL WITHIN YOU PUT

YOUR APPLICATION IN, YOU WOULD

TAKE WHETHER YOU WERE MINORITY

OWNED OR WOMAN OWNED SMALL

BUSINESS VERY DEPENDENT ON SBA

GUIDELINES.

NOW THAT DATA IS CAPTURED ON THE

SBA SITE SIDE WHEN YOU REGISTER,

YOU DO YOUR SBA REGISTRATION.

AND WE HAVE SEEN A SHIFT IN THE

NUMBERS.

IF YOU LOOK LOOK AT HER PARTS OF

NIH YOU SEE SIMILAR SHIFTS AS

WELL TOO IN TERMS OF DATE

CAPTURE.

TO THE NEXT SLIDE.

I WILL END WITH SUCCESSORRIES.

AS PART OF THE TEN YEAR

ANNIVERSARY WE TALKED ABOUT WHAT

WE DID ON I BELIEVE DECEMBER 7

WE HAD THIS BIG LOT OF

PARTICIPATION AROUND THE TEN

YEAR ANNIVERSARY BUT ALSO

DECIDED TO DO SOMETHING WITH

SBIR TOO AND HIGHLIGHT THREE

SUCCESS STORIES WITH COUPLE OF

OF OUR GRANTEES.

LET ME GO THROUGH SOME OF THE

STORIES.

I WANT O GET A GOOD

REPRESENTATION OF DIFFERENT

PARTS OF THE ENTREPRENEURIAL

CYCLE TO GIVE ILLUSTRATE HOW

THESE COMPANIES USE THE GRANT

MORE IMPORTANTLY HOW THEY HAVE

GOT TO KEY INFLECTION POINTS

ALONG THE WAY -- MOVING THEIR

TECHNOLOGY FOR

COMMERCIALIZATION.

LET'S GO TO NEXT SLIDE.

WE HAD DR. LIN PARKS WU WHO

DEVELOPED THIS PORTABLE SYSTEM

CALLED THE BLAZE SYSTEM WHICH

COMBINES MASS SPEC WITH PROTEIN

IMAGING AND LEONA WAS ABLE TO DO

THIS WITH DEVELOPMENT WITH SBIR

BUT SHE SOLD HER COMPANY TO

(INAUDIBLE) AS A RESULT OF THAT

SO THE COMPANY WAS INTEREST

MISDEMEANOR THE PRODUCT AND HER

EXIT STRATEGY WAS TO SELL THE

ASSET WHICH IS WHAT SHE DID SHE

WAS SUCCESSFUL IN DOING THAT BUT

SBIR HELPED WITH THE EARLY

DEVELOPMENT OF THE TECHNOLOGY.

THE NEXT PERSON WAS CHRIS

GIBSON, CHRIS SURE YOU HAVE

HEARD ABOUT RE,CURSION.

ONE OF THE FIRST COMPANIES WE

FUNDED WHEN WE STARTED THE

CENTER.

IT EAT A GREAT STORY BECAUSE

THAT INITIAL GRANT THAT CHRIS

WENT FOR OVER TIME COMPANY WAS

ABLE TO LEVERAGE INVESTMENT,

CHRIS'S COMPANY WAS SUPPORTED BY

NIH GOING TO VARIOUS PITCH

PETITION THAT BIO HAD AND OTHER

CONFERENCES HE COULD SHOWCASE

WHAT HE WAS DOING AND TECHNOLOGY

BUT ONE OF THE KEY THINGS WITH

RECURSION WAS THEY MEET THE

PINNACLE MILESTONE LISTED AS

NASDAQ COMPANY THIS PAST YEAR

WHICH IS A WONDERFUL STORY AND

YES THE GRANT HELPED BUT CHRIS'S

VISION AND WHAT HE'S DONE IS

REALLY PAVED THE WAY TO SUCCESS

OF THE COMPANY.

THEN THE OTHER GRANTEE WE

FOCUSED ON WAS DR. CHANG KIM

BASED IN MILWAUKEE, WISCONSIN.

HE HAD THIS POINT OF CARE

TECHNOLOGY THAT WE WERE FUNDING

FOR DIARRHEAL DISEASE AND WHEN

COVID CAME IN HE SAID I THINK I

CAN PIVOT AND START ADDRESSING

COVID USING THE SAME PLATFORM.

AND THAT IS WHAT HE DID, HE GAVE

A SUPPLEMENT TO DO THAT.

AND CHANG HE IS WORKING

DILIGENTLY TO GET AUTHORIZATION

FOR THE TECHNOLOGY TO BE USED,

HE HAS A PORTAL VAN HE GOES

AROUND MILWAUKEE USING HIS POINT

OF CARE TECHNOLOGY TO GET COVID

TESTS AND HE'S BEEN A GREAT

SUCCESS STORY FOR US AND SHOWING

HOW YOU CAN PIVOT PLATFORM

TECHNOLOGIES AND IN THIS CASE WE

OFFER THE SUPPORT TO GET HIM TO

THAT NEXT CRITICAL STAGE.

GO TO NEXT SLIDE.

FUTURE DIRECTIONS FOR SBER STTR,

FOCUS ON INCREASING

PARTICIPATION OF WOMEN AND

MINORITY OWNED SMALL BUSINESS,

WE WANT TO CONTINUE TO IDENTIFY

SUCCESS STORIES IN WAYS OF

AMPLIFYING OUR COMPANIES

REACHING PIVOTAL MILESTONES THAT

SHOW EFFECTIVENESS OF THE

PROGRAM, PROVIDING EDUCATIONAL

PROGRAMMING TO GRANTEES HOW TO

APPLY TO THE PROGRAM, WHAT WE

ARE LOOKING FOR SPECIFICALLY

HERE AT NCATS AND MORE

IMPORTANTLY, HOW TO LEVERAGE

WHAT WE ARE DOING HERE AT NCATS

IN TERMS OF SCIENTIFIC

PRIORITIES AND SEEING SBIR STTR

AS A WAY OF ALSO GETTING THERE.

SO IN TERMS OF ASPR COUNCIL

MEMBERS I WE ARE ALWAYS LOOKING

FOR OPPORTUNITIES TO DO OUTREACH

FOR THE PROGRAM, IF YOU HAVE

SUGGESTION WE WELCOME THOSE.

AND IDEAS HOW TO LEVERAGE SBIR

WITH OTHER NCATS INITIATIVES AND

PROGRAMS, WE AGAIN HAPPY TO

LISTEN AND I'M GOING TO OPEN IT

UP FOR QUESTIONS.

>> I WAS GOING TO FILE IN

OUTREACH RECOMMENDATIONS --

GOING TO TIME IN OUTREACH

RECOMMENDATIONS.

I DON'T HAVE ANY QUESTIONS.

JUST A COUPLE OF LEADS FOR HER.

>> FANTASTIC.

THANK YOU SO MUCH KELLY,

APPRECIATE THAT.

>> HI, LILY.

ONE OF THE THINGS YOU MIGHT

THINK ABOUT IN EXTENDING THIS IS

REALLY IN THAT ACT AND USING

MENTORS IS TO ALSO PARTNER

TOGETHER WITH THE CTSA PROGRAMS

AND OTHERS WHICH COULD REALLY

USE LOCAL MENTORS SUCCESSFULLY

TRAVERSES BOTH FROM ACADEMIC AND

BUSINESS SIDE THAT COULD WORK

TOGETHER WITH EXTENDING REACH,

ALSO MAKE IT RELEVANT WITHIN THE

OWN COMMERCIALIZATION SYSTEMS

WITHIN OUR ECONOMIC DEVELOPMENT

THINGS WITHIN THE STATES AND

MIGHT HELP THAT SUSTAINABILITY

AS YOU GO PHASE 1 TO PHASE 2 AND

THIS LESSONS LEARNED THAT I

THINK MAKE TEAMS MORE

SUCCESSFUL.

>> THANKS, BECKY.

WE HAVE HAD VERY SUCCESSFUL

ENGAGEMENT WITH VARIOUS CTSAs

THAT HAVE PARTNERED WITH US TO

TALK ABOUT THE PROGRAM.

IN FACT LAST WEEK WE HAD A

WEBINAR WITH A COMPANY THAT WAS

STARTED FROM A K 1 SCHOLAR AT UT

HOUSTON AND HE LICENSED

TECHNOLOGY INTO THE COMPANY.

ALSO PARTNERED WITH AN EXPERT

WITHIN LOCAL CTING ISA.

WE TALK ABOUT HOW TO ENGAGE WITH

THE CTSA PROGRAM IF YOU ARE

SMALL BUSINESS HOW YOU GO ABOUT

DOING THAT.

THE WEBINAR WAS VERY SUCCESSFUL

IN TERMS OF MAKING TALKING THE

STORY HOW TO BRIDGE THE TWO

PROGRAMS, I WOULD LOVE TO SEE

MORE OF THAT OVER TIME AS WELL

TOO.

MAYBE WE CAN TALK SEPARATELY AND

GIVE ME MORE IDEAS ON WHERE TO

GO.

>> LOVE TO, WE CAN TALK OFFLINE.

>> ANY OTHER QUESTIONS FOR LILY?

>> LILY HOW PRESCRIPTIVE CAN YOU

MAKE THE RFAs IF YOU WILL FOR

THESE?

IS IT JUST SORT OF OPEN

EVERYTHING OR CAN YOU DO

TARGETED ANNOUNCEMENTS?

>> ABSOLUTELY WE CAN DO TARGETED

ANNOUNCEMENTS.

YES.

OUR OMNIBUS TOPICS ARE ON THE

BROADSIDE BECAUSE WE WANT TO

CAPTURE AS MUCH AS WE POSSIBLY

CAN WITH THAT.

BUT WE DO TARGET SOLICITATION,

ORDR IS VERY SUCCESSFUL IN

LEVERAGING WHAT THEY -- THEY ARE

DOING USING THE TARGETED

SOLICITATIONS.

THEY HAD A BASKET TRIAL RFA

USING A U MECHANISM ABOUT WE

USED A U 44 MECHANISM ON THE

SBIR AS COMPLIMENTARY WAY OF

GETTING SMALL BUSINESS ENGAGED.

SO IT IS VERY MUCH SOMETHING

THAT WE WANT TO SEE MORE OF

GOING FORWARD AND FIGURING HOW

TO LEVERAGE CURRENT PROGRAMS AND

INITIATIVES USING SBIR STTR.

>> COUPLE OF FOLLOW UPS.

DOES THE END RESULT NEED TO BE A

PRODUCT OR IT -- CAN IT BE A

SUSTAINABLE SET OF SERVICES?

>> TYPICALLY A PRODUCT.

IN TERMS OF SUSTAINABLE SERVICE,

IF THERE IS SOMETHING UNIQUE

ABOUT INNOVATIVE ABOUT THAT

SERVICE, THAT REQUIRES PERHAPS A

PLATFORM OF SOME SORT TO DO, WE

HAVE MANY COMPANIES THAT OFFER

DRUG DEVELOPMENT SUPPORT AND DO

IT AS PART OF SERVICE BUT IT IS

THE TECHNOLOGY THEY USE BEHIND

THE SCENES THAT IS THE

INNOVATION THAT WAS FUNDED BY

SBIR, THAT WOULD BE POSSIBLE.

>> WHERE MY HEAD IS ON YOUR

SECOND QUESTION THINKING BACK TO

JONI'S AUDACIOUS GOALS, LOOKING

AT WHERE THE GAPS ARE THAT MAY

WELL NOT BE EASY TO FILL USING

TRADITIONAL METHODS, SEEMS LIKE

TARGETED ANNOUNCEMENTS MIGHT

OFFER A GOOD OPPORTUNITY.

>> WE ARE UP TO EXPLORING HOW TO

MAKE THAT HAPPEN.

IN SOME INSTANCES HAVING THE

TARGETED ANNOUNCEMENTS IS GOING

TO GET YOU MORE WHERE YOU

IMMEDIATE TO GO, MAYBE THAT IS

EXACTLY IN TERMS OF BRIDGING

THOSE WITH JONI'S GOALS SHE

MENTIONED, SPECIFICITY AROUND

TARGETED SOLICITATION IS REALLY

THE WAY TO GO OPPOSED TO BEING

BROAD.

>> LOVE THAT IDEA.

ANY OTHER QUESTIONS FOR LILY?

OKAY.

HEARING NONE WE HAVE MADE UP

SOME TIME.

ANNA, QUICK PROCESS QUESTION

HERE, SHOULD WE TAKE A BREAK?

OR PROCEED WITH THE LAST BIT OF

THE COUNCIL CONCEPT CLEARANCES?

>> JONI, I THINK FOLKS WOULD

APPRECIATE A SHORT BREAK.

WE DO HAVE TIME, WE ARE 20

MINUTES AHEAD OF SCHEDULE.

SO TEN MINUTE BREAK WOULDN'T BE

PROBLEMATIC AT ALL.

>> SOUNDS GREAT.

EVERYBODY IN TEN MINUTE BREAK.

ALL RIGHT.

LET'S DO IT.

SEE YOU BACK IN ABOUT TEN A

LITTLE AFTER TEN OF 4.

SEE YOU SOON.

SO NEXT UP IS THE CLEARANCE OF

CONCEPTS AND WE HAVE A COUPLE

FOR TODAY AND WE HAVE A COUPLE

FOR TOMORROW.

THE FIRST ONE WITHINOR WILL BE

WITH OFFICE OF POLICY

COMMUNICATIONS AN EDUCATION

CONCEPT AND PENNY BURGOON WILL

KICK OFF AND THEN WE WILL HEAR

FROM JESSICA TAGLE BADGER.

>> THANK YOU, GOOD AFTERNOON,

EVERYBODY.

HERE TO OVERVIEW OFFICE OF

POLICY COMMUNICATIONS AND

EDUCATION.

I WILL ALSO WEAVE INTO A BRIEF

INTRODUCTION ABOUT THE CONCEPT

FOR THE EDUCATION BRANCH.

NEXT SLIDE PLEASE.

THE OFFICE OF POLICY

COMMUNICATIONS AND EDUCATION

OTHERWISE KNOWN AS OPIC PROVIDES

GUIDANCE AND SUPPORT FOR NCATS

ON NUMBER OF ACTIVITIES IN THE

PURVIEW OF SCIENCE POLICY.

COMMUNICATIONS AND OUTREACH.

THE NEWEST BRANCH IS OUR

EDUCATION AN TRAINING BRANCH.

THIS IS A GROUP THAT WAS -- WE

HAD THE ORGANIZATIONAL APPROVAL

PROBABLY AROUND 2017 BUT WE WERE

NOT ABLE TO STAFF THE -- THIS

BRANCH UNTIL 2019.

NEXT SLIDE PLEASE.

NOW, THE GOALS OF THE EDUCATION

BRANCH ARE TO DISSEMINATE AND

DEVELOP EVIDENCE BASED TOOLS AND

BEST PRACTICES.

TO IMPROVE THE UNDERSTANDING OF

TRANSLATIONAL SCIENCE.

AND IN THAT GOAL THEY ALSO HOPE

TO EXPAND AND DIVERSIFY

TRANSLATIONAL SCIENCE WORK

FORCE.

THE BRANCH AIMES TO ACHIEVE

THESE GOLDSBY COORDINATING AND

PROVIDING CENTRAL LEADERSHIP TO

NCATS TRANSLATIONAL EDUCATION

ACROSS THE CENTER.

SO THEY WORK WITH ALL THE CENTER

DIVISIONS AND OFFICES, IN

DEVELOPING NEW INITIATIVES LED

BY THIS BRANCH AND LEADING

CENTER WIDE AND ALSO LEAVE A

CENTER WIDE COMMITTEE FOCUSED ON

TRANSLATIONAL SCIENCE EDUCATION.

HERE IS AN EXAMPLE OF THE

ACTIVITIES THEY STOOD UP IN THE

SHORT TIME THEY HAVE BEEN IN

EXISTENCE. THE 501 COURSE IS

ONLINE COURSE INITIATED BY

EDUCATION BRANCH AND THIS WAS TO

BUILD ON EXISTING EFFORTS BY N

CATS COURSES TO TEACH PRINCIPLES

OF TRANSLATIONAL SCIENCE.

THEY ARE WORKING WITH OUR

COMMUNICATION BRANCH TO IMPRINT

SOME OF THESE IMPORTANT

PRINCIPLES IN TERMS OF BADGES TO

HELP EXEMPLIFY SOME OF THE IDEAS

AROUND TRANSLATIONAL SCIENCE

THAT WE THINK NEED TO HAVE A

PARTICULAR EMPHASIS.

HERE ARE THINGS WE ARE WORKING

ON, THIS IS IN DEVELOPMENT, IT

IS NOT OUT THERE YET BUT THIS IS

AN EXAMPLE OF THINGS WE ARE

TALKING ABOUT IN TERMS OF HOW WE

LOOK AT THE SCIENCE OF

TRANSLATION AND WHAT THE WORK

FORCE, WHAT NEED TO BE EXPERT

GAINED EXPERTISE IN.

WITH THAT THIS WILL LEAD TO NEW

CONCEPT CLEARANCE AND I WILL

HAND THIS OVER TO DR. JESSICA

BADGER EDUCATION BRANCH CHIEF.

>> THANK YOU, THIS IS A PROPOSAL

TO DEVELOP A PORTFOLIO OF

EXTRAMURAL RESEARCH EDUCATION

AWARDS THAT RESULT IN A SUITE OF

ONLINE EDUCATION ACTIVITIES THAT

CENTER TEACHING EFFECTIVE

PRACTICES AND CORE PRINCIPLES OF

TRANSLATIONAL SCIENCE.

WITH KNOW THERE IS A NEED FOR

CURRICULAR ACTIVITIES THAT

DISSEMINATE EFFECTIVE PRACTICES

AND TRANSLATIONAL SCIENCE BASED

ON OUR ACTIVITIES INCLUDING

EXTENSIVE LIT REVIEW AND

EXPERIENCE WITH OUR INTERNAL

TRAINING PROGRAM AND MOST

IMPORTANTLY WE RECEIVED THIS

FEEDBACK FROM THE COMMUNITY.

AS YOU HEARD FROM DR. BERRIGAN

THE EDUCATION BRANCH -- BURGOON

THROUGH DEVELOPMENT OF TWO ON

LION COURSES THAT USE EXAMPLES

OF SUCCESSFUL NCATS LED EFFORTS

TO DEMONSTRATE HOW SPECIFIC

RESEARCH ADVANCES CAN BE

EXAMINED TO OFFER INSIGHT TO

TRANSLATIONAL PROCESS IDENTIFY

COMMON CHALLENGES THAT STYMIE

TRANSLATIONAL PROJECTS AND

SOLUTIONS THAT ARE APPLICABLE

NOT ONLY TO THIS PARTICULAR CASE

BUT ALSO MORE BROADLY TO

RESEARCH INITIATIVES ACROSS THE

TRANSLATIONAL SPECTRUM.

THROUGH EXAMINING CASES N CATS

STAFF ALSO IDENTIFIED THE SEVEN

INITIAL PRINCIPLES FOR EFFECTIVE

TRANSLATIONAL SCIENCE SHARED ON

THE PRIOR SLIDE AND HAVE BEEN

INCORPORATED INTO THESE COURSES.

WE NOW WANT TO EXPAND UPON AND

EXTEND THIS APPROACH THROUGH

STIMULATING ADDITIONAL

TRANSLATIONAL SCIENCE EDUCATION

ACTIVITIES, OR WE CAN CALL THESE

EDUCATION EXPERIMENTS, THAT

FURTHER DEVELOP AND FORMALIZE

TRANSLATIONAL SCIENCE FOR

CONCEPTS APPROACHES AND

STRATEGIES DERIVED FROM THE

EXPERIENTIAL KNOWLEDGE IN THE

FIELD.

WE ALSO WANT TO DO THIS WITH AN

EYE TOWARDS EXPANDING

TRANSLATIONAL SCIENCE WORK FORCE

ENHANCING WORK FORCE DIVERSITY

ENSURING BROAD ACCESS TO

ACTIVITIES AS WELL AS

ENCOURAGING INNOVATION AND

ONLINE TRANSLATIONAL SCIENCE

EDUCATION.

THESE AWARDS FUND FULL CYCLE

FROM DEVELOPMENT TO

IMPLEMENTATION TO EVALUATION OF

THESE EDUCATIONAL ACTIVITIES.

THIS PROPOSAL ALIGNS WITH TWO

NCATS STRATEGIC GOALS, THAT IS

ADVANCING TRANSLATIONAL SCIENCE

BY UNCOVERING TRANSLATIONAL

SCIENCE PRINCIPLES TO CATALYZE

ADVANCES IN HEALTH INTERVENTIONS

SO UNCOVERING PRINCIPLES AND

TEACHING THOSE TO CURRENT FUTURE

MEMBERS OF THE TRANSLATIONAL

SCIENCE WORK FORCE.

AND TO DO THAT, MEET STREAM GOAL

THREE DEVELOPING FOSTERING

INNOVATIVE TRANSLATIONAL SCIENCE

TRAINING AND HIGHLY SKILLED

CREATIVE AND DIVERSE

TRANSLATIONAL SCIENCE WORK

FORCE.

WE REALLY SEE THIS AUGMENTING

CURRENT EDUCATION TRAINING

PROGRAMS.

WE KNOW SOME CURRENT ACTIVITIES

FOCUS ON THE CHARACTERISTICS OF

THE TRANSLATIONAL SCIENCE BEING

SKILLED COMMUNICATOR, DISTINCT

PHASES OF TRANSLATION OR CONTENT

AREAS RELATED TO TRANSLATION.

BUT NOW WE WANT TO ADD TO THIS

HOW DO WE EXPLORE TRANSLATIONAL

SCIENCE BY LOOKING AT THE WHOLE

SYSTEM.

HOW DO WE IDENTIFY THESE

EFFECTIVE PRACTICES AND TEACH

THEM TO OTHERS, AND ARTICULATE

THE BENEFITS OF A TRANSLATIONAL

SCIENCE APPROACH.

AND USING THESE PRINCIPLES COULD

INVESTIGATE A SCIENCE MIND SET.

THIS IS ESTABLISHING

TRANSLATIONAL SCIENCE AS

DISTINCT AREA OF STUDY.

SO I MENTION WE HAVE DONE LIT

REVIEW, I WILL SHARE ABOUT THE

DATA HERE AS IT IS INFORMED OUR

THINKING ON THIS.

BUT WE WERE SPECIFICALLY LOOKING

AT TRANSLATIONAL SCIENCE

EDUCATION WHERE THERE WAS FORMAL

CURRICULUM COMPONENT, EDUCATION

OR COURSE, A WORKSHOP, SEMINAR

SERIES AND THEN WHAT WAS TAUGHT

OR CONVEYED WITHIN THOSE COURSES

WORKSHOPS, ET CETERA.

THIS IS SHOWING YOU CAN IMAGINE

LIT REVIEW FOR TRANSLATIONAL

SCIENCE EDUCATION YOU GET

ARTICLES WHEN YOU NARROW DOWN

SPECIFIC CURRICULUM COMPONENTS

IT BECOMES FEW ARTICLES IN THE

LITERATURE OVER THE SPAN WE LOOK

AT FROM ABOUT 2005 THROUGH 2021.

BASICALLY THE TAKE HOME MESSAGE

IN THESE SLIDES THERE ARE

CERTAIN SEGMENTS OF

TRANSLATIONAL SCIENCE WORK FORCE

WE ARE MISSING OR PEOPLE WHO WE

WANTS BRING INTO THE WORK FORCE.

AGAIN THIS WAS PRE-COVID WE KNOW

A LOT HAPPENED WITH ONLINE

EDUCATION IN THIS TIME.

THERE WAS -- THERE WAS FEW

ARTICLES MENTIONED ANYTHING

ABOUT ONLINE COMPONENT.

FROM OUR LITERATURE RESEARCH.

WE ALSO ORGANIZED ARTICLES BASED

ON FRAME WORK OF TRANSLATIONAL

PRINCIPLES TO SEE WHERE GAPS OR

ADDITIONAL EDUCATION NEEDS COULD

EXIST AND AGAIN THIS SHOWS YOU

THERE'S SOME THINGS THAT WE ARE

TOUCHING ON FREQUENTLY LIKE TEAM

SCIENCE BUT OTHER THINGS WE CAN

BUILD OUT MORE.

SO GIVE YOU SOME OF THE

HIGHLIGHTS FOR THIS PROPOSAL,

WHAT WE ARE PROPOSING IS FUNDING

OPPORTUNITY FOR THE EXTRAMURAL

COMMUNITY TO DEVELOP NEW

EDUCATION OPPORTUNITIES TEACHING

CORE TRANSLATIONAL SCIENCE

CONCEPT, INCLUDING SCIENTIFIC

OPERATIONAL PRINCIPLES KEY

STRATEGIES AND APPROACHES USED

EFFECTIVELY TO ADVANCE

TRANSLATION LOOKING FOR THIS TO

EXPAND AND DIVERSIFY

TRANSLATIONAL SCIENCE WORK FORCE

TO REACH PEOPLE NOT CURRENTLY

REACHING WITH OUR CURRENT

EDUCATION TRAINING PROGRAMS AND

WE REALLY WANT TO STIMULATE

INNOVATION AND EDUCATION CONTENT

DESIGN.

SO WHILE WE WANT THIS AVAILABLE

IN AN ONLINE FORMAT WE ARE NOT

FOCUSED IN ON THIS HAS TO BE

TYPICAL ON LINE COURSE, THERE'S

WAYS THIS CONTENT COULD BE

DELIVERED.

KEY PART OF THIS WILL BE

EVALUATION.

WE WANT THESE EDUCATION

ACTIVITIES EVALUATED TO USE TO

ESTABLISH EVIDENCE BASED FOR

EFFECTIVE APPROACHES IN TEACHING

TRANSLATIONAL SCIENCE CONCEPTS.

SO AT NCATS WE SAY EVERYTHING IS

AN EXPERIMENT WE ARE NOT AFRAID

FOR THIS TO BE AN EXPERIMENT AND

FOR US TO LEARN WHAT WORKS.

THE NEXT SLIDE SPEAKS MORE TO

SOME OF THE THINGS WE WOULD LIKE

TO SEE COLLECTED AS THE

EVALUATION COMPONENT.

THAT INCLUDES DIVERSITY OF THE

PARTICIPANTS IN THESE EDUCATION

ACTIVITIES AND DIVERSITY IN ALL

AREAS TRAINING AND CAREER STAGE,

EMPLOYMENT SECTOR GENDER RACE

ETHNICITY INSTITUTION TYPE

GEOGRAPHY WE WANT TO KNOW

ADHERENCE OR DEGREE OF

PARTICIPATION IN A COURSE, THAT

WOULD BE IMPORTANT.

FOR THEM TAKING THIS TO THE NEXT

LEVEL AND UNDERSTANDING HOW

PEOPLE ARE UTILIZING THIS

KNOWLEDGE AND HOW DOES IT

INFLUENCE THEIR RESEARCH OR

CAREER GOALS GOING FORWARD.

SO WHAT DO WE EXPECT AN OUTCOME

FROM THIS EDUCATION PROGRAM?

BROADEN EXPAND FOUNDATIONAL

TRANSLATIONAL SCIENCE KNOWLEDGE.

DOING THROUGH VARIETY OF ONLINE

EDUCATION ACTIVITIES TO INCREASE

UNDERSTANDING OF TRANSLATIONAL

SCIENCE PRINCIPLES AND EFFECTIVE

APPROACHES AND TRANSLATIONAL

SCIENCE.

WE KNOW ONLINE LEARNING ONLINE

EDUCATION ACTIVITIES ARE HERE TO

STAY, PEOPLE APPRECIATE THE

FLEXIBILITY OF THIS, THEY

APPRECIATE ACCESSIBILITY, SO WE

WANT TO MAXIMIZE ONLINE LEARNING

AND EVALUATE THIS TO LEAD TO

EVIDENCE INFORMED TEACHING

APPROACHES FOR ONLINE EDUCATION

AND TRANSLATIONAL SCIENCE

REACHING DIVERSE AUDIENCE OF

LEARNERS.

WE WANT TO CATALYZE NEW

TRANSLATIONAL SCIENCE CURRICULA.

SO ULTIMATELY THESE EDUCATION

OPPORTUNITIES OR ACTIVITIES

SHOWN TO BE EFFECTIVE COULD BE

INFORM RATED INTO A WIDE RANGE

OF BIOMEDICAL RESEARCH AND

TRAINING, RESEARCH EDUCATION AND

TRAINING PROGRAMS INCLUDING

THOSE FUNDED BY NCATS BUT MORE

BROADLY, ACROSS NIH.

IN SUMMARY THIS PROPOSAL WILL

ADVANCE TRANSLATIONAL SCIENCE

EDUCATION FOR DEVELOPMENT

IMPLEMENTATION AND EVALUATION OF

ONLINE EDUCATION ACTIVITIES

UNDERSTANDING TRANSLATIONAL

SCIENCE PRINCIPLES AN

APPLICATION TO RESEARCH ACROSS

THE TRANSLATIONAL SCIENCE

SPECTRUM.

NOW IS THE TIME TO AGGREGATE

THIS EXPERIENCIAL KNOWLEDGE WE

AMASS AND WE HEARD ABOUT MANY OF

THESE ADVANCES AT OUR TENTH

ANNIVERSARY AND WE KNOW THERE

ARE MORE THAN THAT TO DRAW ON.

TIME TO EXPANDS TRANSLATIONAL

SCIENCE KNOWLEDGE BASE AND

CONDITION SRI TO BROAD RANGE OF

INDIVIDUALS TO EXPAND AND

DIVERSIFY THE WORK FORCE AND

STIMULATE INNOVATION AND

TRANSLATIONAL SCIENCE EDUCATION

AND HOW WE ARE DEVELOPING THAT

FORCE.

ON THE NEXT SLIDE I WILL

TRANSITION TO TAKING QUESTIONS

AND COMMENTS FROM THE COUNCIL,

COUNCIL MEMBERS WE LOOK FORWARD

TO HEARING FROM YOU WHAT IMPROVE

THE INITIATIVE AND OTHER THINGS

WE SHOULD BE CONSIDERING.

>> JESSICA, BEFORE YOU MOVE ON

WE HAVE COUPLE OF COUNCIL

MEMBERS DISCUSSANTS FOR THIS

CONCEPT.

I WILL LET BEBECKY GO FIRST AND

RAJESH AFTER THAT AND HEAR YOUR

THOUGHTS AND WE WILL OPEN FOR

BROADER DISCUSSION.

BECKY, LEAD US OFF?

>> THANKS, JONI.

I LIKE TO START THIS OFF BY

SAYING THAT I'M INCREDIBLY

ENTHUSIASTIC ABOUT SEEING REALLY

LEVERAGE OF BROADER EXTRAMURAL

COMMUNITY, THAT INCLUDES PUBLIC

PRIVATE PARTNERSHIPS AND OTHERS

THAT CAN PULL TOGETHER BOTH

BASIC AS WELL AS MORE ADVANCE

TRAINING IN THE UNDERLYING

PRINCIPLES OF TRANSLATIONAL

SCIENCE AND REALLY SHARING

WITHIN THAT SOME OF THE TOOLS

THAT WE CAN UTILIZE FOR DOING

THAT.

I DO THINK IT IS IMPORTANT

SHOULD BE EMPHASIZED THAT THIS

SHOULD REALLY SUPPORT THAT FULL

SPECTRUM OF TRANSLATIONAL

SCIENCE REALLY FROM TAKING

DISCOVERY TO THE BEDSIDE ALL THE

WAY INTO DISSEMINATION AND

IMPLEMENTATION SCIENCE BUT THERE

ARE COUPLE OF THINGS THAT I

THINK COULD IMPROVE THIS I HAVE

A COUPLE OF SUGGESTIONS TO

RESPOND TO.

THE FIRST I THINK IS THAT AS THE

FIELD OF TRANSLATIONAL SCIENCE

CONTINUES TO EXPAND AND AS WE

GET MORE CLARITY AROUND SOME OF

THE THINGS THAT WILL ADVANCE

GOALS THAT HAD BEEN TALKED ABOUT

TODAY I THINK THAT IS GOING TO

BE CRITICAL THAT ANY TYPE OF ON

LION OPPORTUNITIES ARE KEPT UP

TO DAY SO HAVING THAT

SUSTAINABILITSUSTAINABILITY PLAN REVIEW AN D

UPDATE OF MATERIALS OR SUN

DOWNING THEM AT SPECIFIC PERIODS

OF TIME ARE CRITICAL IN ORDER

FOR THIS TO ACTUALLY HAVE A

SIGNIFICANT IMPACT.

I THINK THE SECOND THING WHICH

YOU MENTIONED IN YOUR SLIDE,

REALLY TO INNOVATE CURRICULAR

YOU KNOWVATION AND I USE THIS AS

A OPPORTUNITY TO SEE WHICH

APPROACHES ARE MOST EFFECTIVE

FOR ADULT LEARNERS IN TERMS OF

ACTUALLY CHANGING BEHAVIOR AND

CULTURE AND BEING MORE ENGAGED

AND TRANSLATIONAL SCIENCE

APPROACHES.

SO ONE OF THE PROBLEMS AS ALL OF

US KNOW WHO CREATED ONLINE

LEARNING, IS THAT IT DOES NOT

FOLLOW THE FIELD OF DREAMS WHICH

YOU TALKED ABOUT EARLIER SO JUST

BECAUSE YOU BUILD IT DOESN'T

MEAN PEOPLE WILL COME.

IT IS IMPORTANT WE THINK ABOUT

REALLY COMING UP WITH MORE

ENGAGING APPROACHES, AND

INNOVATIVE APPROACHES WHETHER

THIS IMPLICATION CASE STUDIES,

EXTENDED MORE IN DEPTH READINGS

ASSOCIATED WITH THIS OR OTHER

TYPES OF ACTIVITIES THAT COULD

BE PURSUED THAT WILL TAKE THAT

LEVEL FROM BASIC TO NEXT LEVEL

MASTERY ALL THE WAY TO ADVANCE.

ONE OF THE THINGS TO THINK ABOUT

THIS IS YOU ALSO WANT TO SUPPORT

OTHER ASSOCIATED ACTIVITIES.

SO DISCUSSION FORMS PEOPLE WHO

ARE INVOLVED IN THIS, OR WAYS OF

STARTING TO DEVELOP A COMMUNITY

OF PRACTICE OR WAYS TO FURTHER

EXPAND THE FIELD.

I THINK THE OTHER THINGS THEN IS

TO THINK ABOUT HOW DO WE REALLY

DRIVE SUCCESS OF THIS.

I THINK IT WOULD BE WORTHWHILE

IN THIS IS RATHER THAN ASKING OR

HAVING A MEETING JUST BETWEEN

THOSE THAT ARE FUNDED IS TO

REALLY THINK ABOUT WHETHER IT

MIGHT BE MORE NIMBLE COST

EFFECTIVE AND GET YOU REALLY

MORE SIGNIFICANT RESULTS IF YOU

CONSIDER THIS AS MORE FORMALIZED

CONSORTIA WHERE YOU BRING

TOGETHER AND MAKE SOME LEVEL OF

CONSISTENCY OF STRUCTURE.

YOU CAN DO THIS THEN THROUGH

PORTAL, DEVELOPMENT OF

EDUCATIONAL ONTOLOGIES THAT

ALLOW PEOPLE TO FIND BOTH

CONTENT THAT BEST MET THEIR

NEEDS AT THE TIME THEY HAD THEM

AS WELL AS MARKETING AND

COMMUNICATION PLANS THAT DRIVE

TO THE RESOURCE AND CONSISTENT

AND ACTIONABLE EVALUATION PLAN

SO YOU ARE COMPARING ACROSS ALL

THE DIFFERENT PROGRAMS THE SAME

KIND OF EVALUATION IN TERMS OF

LOOKING AT LONG TERM IMPACT.

SO DOES THAT CHANGE LEVEL OF

TRAJECTORY OR ENGAGEMENT OF

RESEARCH TRANSLATION, ARE MOVING

THE FIELD MORE RAPIDLY AND ARE

WE BEING MORE EFFICIENT.

I THINK IF YOU THINK ABOUT

EXPANDING THIS PROGRAM BEYOND

JUST THE SERIES OF ON LINE

COURSES TO REALLY SERIES OF

ONLINE ENGAGING ACTIVITIES, AND

LEARNING BETWEEN DIFFERENT

FUNDED PROGRAMS YOU WILL HAVE

GREATER IMPACT ON THE

TRANSLATIONAL SCIENCE WORK FORCE

AND SIMILARLY CAN REALLY THEN

FOCUS ON APPROACHES THAT ALSO

ENHANCE DIVERSITY AND INCLUSION

MULTIPLE PERSPECTIVES AS WELL AS

INDIVIDUALS.

>> THANK YOU, THOSE ARE ALL

FANTASTIC COMMENTS, THINGS WE

CAN REALLY TAKE TO HEART AS WE

CRAFT MORE SPECIFIC LANGUAGE.

I REALLY APPRECIATE YOUR POINT

TO HAVE OUTCOMES WE NEED PEOPLE

TO BE ENGAGED HOW DO WE ENHANCE

THAT ENGAGEMENT.

>> THANK YOU, RAJESH NEXT

PLEASE.

>> SURE.

JESSICA I'M SORRY WE DIDN'T HAVE

A CHANCE TO MEET EARLIER THIS

WEEK YOU CAN CONNECT WITH ME A

COUPLE OF TIMES.

SO I DESIGN/I TEND TO SPEAK MY

MIND SO I WILL HERE.

I'M NOT ENTHUSIASTIC ABOUT THIS,

TELL YOU WHY.

DOES BECAUSE OF MY PAST

EXPERIENCE HAVING TO DO THIS, I

WAS HEAD OF EDUCATION OF

NOVARTIS FOR SEVEN YEARS

MULTI-NATIONAL COMPANY WE

BASICALLY THE ORIGINAL IDEA WAS

DO IT ALL ONLINE, YOU HAVE SEX

CENTERS PUT IT ONLINE, WILL

LEARN DRUG DISCOVERY AND

EVERYTHING WILL BE HUNKY DORY,

ADULTS DON'T LEARN THAT WAY.

THIS IS -- THIS IS NOT THE MODEL

BY WHICH YOU ARE GOING TO GET

THE BIGGEST BANG FOR THE BUCK IN

MY OPINION.

THAT IS WHY THE COURSE IS SUCH

AS THE ONES YOU DEVELOPED

WHETHER THEY ARE ONLINE IN

CLASSROOM SESSIONS SPEAKING WITH

EXPERTS AND WHAT HAVE YOU REALLY

CASE STUDY METHODOLOGY AND OTHER

THINGS THAT MAKE A DIFFERENCE IS

PREFERRED TO JUST PUTTING THAT

OUT THERE BECAUSE IT IS TOO

PASSIVE.

THE OTHER CONCERN IS ONLINE

COURSE IF YOU PUT THIS RFA OUT

THERE OR WHATEVER APPROACH YOU

TAKE CALL FOR APPLICATION MY

CONCERN IS THE COMMUNITY WILL

JUST DEVELOP A WHOLE BUNCH OF

LECTURES, THEY WILL DO A SERIES,

FIND A BUNCH OF PEOPLE WHO GIVE

SERIES OF LECTURES ONE AFTER

ANOTHER, PART ONCOLOGIST

CLINICIAN TALK ABOUT SOMETHING

AND THEN THAT WILL GET POSTED AS

ONE AND THAT WILL GET POSTED AS

OTHER.

QUALITY CONTROL WILL VARY, THERE

WON'T BE ANY STRUCTURE

NORMALIZATION OF PRINCIPLES THAT

WILL BE TAUGHT IN THERE.

IT WILL BE A BOON TOGGLE, I'M

JUST REALLY PRESENTING YOU THE

OTHER CONCERN THAT I WOULD HAVE

IN PUTTING THIS OUT THERE.

YOU SHOULD THINK CAREFULLY ABOUT

THIS IS WHERE YOU WANT TO INVEST

YOUR RESOURCES IN THE

EDUCATIONAL REALM,S WITH THE

ONLINE ARENA.

I I'M NOT -- I WILL THROW

POSITIVES OUT THERE NOT JUST TO

BE NEGATIVE.

ONE THING THAT ACTUALLY WORK

WHICH TOOK A LOT TO DEVELOP, OUT

THERE AS FREE WARE, I KNOW A

BRAZILIAN GROUP, THEY CALL THEIR

GAME SCREENER, WE DEVELOP A GAME

IN PHARMA, IN THE NOVARTIS

CALLED PHARMA GAME OR SOMETHING

LIKE THAT.

FORGET HOW THEY BRANDED IT BUT

YOU CAN DEVELOP A GAME WHICH IS

WHETHER OUR TEENAGERS DO IS PLAY

A GAME ALL THE TIME, PLAY A GAME

AND LEARN DRUG DISCOVERY THAT

MIGHT HAVE MORE IMPACT AND MIGHT

BE A COOL WAY TO MEET APPROACH

OF CURRENT GENERATION DRUG

DISCOVERY.

IF YOU CAN INCLUDE IT MIGHT BE

INTERESTING TO SEE IF

PROGRAMMERS OUT THERE CAN THINK

ABOUT THE COMPLEXITY OF THE

WIRING DIAGRAM IN LAST SLIDE

MEANT TO HAPPEN FASTER AND TAKE

THAT WIRING DIAGRAM AND FIGURE A

WAY TO DO INPUT OUTPUT CIRCUIT

GAIN YOU CAN THEN AS GROUP

DECISION MAKING MAY TRICK.

SO -- MATRIX.

SO PK IS NOT GOOD.

WHAT THE HELL DO YOU DO NEXT?

THAT'S WHAT IT COSTS ANOTHER

MILLION DOLLARS.

DO YOU HAVE THE MONEY, YOU DON'T

HAVE THE MONEY SO HOW DO YOU

KNOW HUH WHOM TO EMPERIMENT.

YOU COLLECT -- YOU DO A

DIFFERENT EXPERIMENT TAKE HELO

CELLS AND THAT DOESN'T WORK.

WHAT IS NEXT EXPERIMENT?

THAT MIGHT TEACH THEM MORE THAN

A BUNCH OF DRY LECTURES THAT

ESSENTIAL WILL I PUTTING TO ON A

WEBSITE.

SO THAT WOULD BE I WILL STOP

WILL.

TRILLION READING WHAT YOU SAY,

I'M NOT SURE WHETHER THE

COMMUNITY THAT IS GOING TO

RESPOND TO THIS I HATE TO SAY

WILL BE OUR GENERATION.

OURS IS GOING TO (INAUDIBLE)

SOMETHING NOT GOING TO WORK FOR

THE NEXT GENERATION IN ONLINE

SPACE.

>> THANK YOU.

ANYTHING TO RESPOND?

WE WILL OPEN IT UP.

>> I APPRECIATE THE COMMENTS AND

I THINK THERE ARE MANY CAVEATS

HERE AND THINGS TO THINK ABOUT.

ONE OF THE THINGS SO HERE ARE

SOME OF THE CHALLENGES THAT I

SEE.

ONE OF THE REASONS WE DID THE --

WE START WITH THE ONLINE COURSE

WHICH AGAIN MAY -- IS VERY

TRADITIONAL, I HOPE WE CAN DO

THIS IN A LOT OF WAYS.

WAS TO REACH MORE PEOPLE MORE

UNDERGRADS AND THINK ABOUT THOSE

SEGMENTS THAT AREN'T HEARING

TRANSLATIONAL SIEBS OR MIGHT NOT

BE AT A HUB IN A FORMAL TRAINING

PROGRAM.

SO REALLY THINKING HOW DO WE

PUSH TRANSLATIONAL SCIENCE

EDUCATION OUT WILL TO MORE

PEOPLE AND EXPAND THE WORK FORCE

AND DI VERY PHI WHO WE RECRUIT

TO THE WORK FORCE.

THAT IS ONE PRINCIPLE UNDERLYING

THIS.

IS GETTING TRANSLATIONAL SCIENCE

EDUCATION TO MORE PEOPLE.

I UNDERSTAND SOME OF THE

CONCERNS YOU MAZE RAISE, WITHIN

NIH WE HAVE DIFFERENT WAYS WE

CAN DI SIGN FUND MECHANISMS

WHERE THERE'S GREATER

INVOLVEMENT FROM M CATS STAFF

AND WE CAN HELP SHAPE THINGS

LITTLE MORE AS DR. JACKSON SAID

THROUGH CONSORTIUM PROCESS AND

SETTING CENTRALIZED EVALUATION

MATRIX -- MEASURES AND BEING

ABLE TO HAVE THAT SORT OF I

DON'T KNOW IF I WANT TO USE

OVERSIGHT BUT MORE CENTRALIZED

FUNCTION ACROSS ALL THESE

ACTIVITIES.

WE COULD HELP SHAPE THAT AS

WELL.

SO I UNDERSTAND WHAT YOU ARE

SAYING.

(OVERLAPPING SPEAKERS)

>> THIS MAY NOT FIT INTO YOUR

SCHEME OF THINGS.

SEND THIS OUT AS THINGS FOR HIGH

SCHOOL STUDENTS AND COLLEGE

STUDENTS TO BUILD RATHER THAN

YOUR PI IN SOME UNIVERSITY AND

THEN GIVE HEM THE EXPERTISE THEY

NEED TO LEARN BUT THEY CAN BUILD

WHAT NEEDS TO BE BUILT TO HELP

PEERS LEARN.

I THINK WHAT WE NEED IN THIS

SITUATION IS SOME THOUGHTFUL

EXPERTISE IN EDUCATIONAL

METHODOLOGY THAT WORKS FOR THE

GENERATION THAT WE ARE TRYING TO

TEACH.

NOT THE SAME YOU AND I LEARNED.

WE MAKE THIS MISTAKE ALL THE

TIME, IF WE TAKE IT THE WAY WE

LEARN IT THEY WILL LEARN IT.

I DON'T THINK THAT WORKS.

>> GOOD POINT.

GO I A HEAD.

>> IT IS CLEARLY A SIGNIFICANT

UNMET NEED THERE IS A FAIR

ENOUGH ACTIVITY IN THE SPACE

WHICH IF YOU CAN REACH INTO

CONCEPT OF ONTOLOGY, FRAMEWORK

WHAT HAS BEEN DEVELOPED

INCLUDING IN OUR FORM OF

TRAINING CERTIFICATE WHERE

TRANSLATIONAL ELEMENTS ARE

TAUGHT, THEIR DISEASE SPACE BUT

MIGHT BE GENERALLY APPLICABLE IN

SEVERAL CTSA SITES, TRANSLATION

TRAINING PROGRAMS ONLINE SO THEN

I THINK WOULD BE DELIVERABLE

WHICH WOULD HAVE IMMEDIATE

IMPACT EXTENDING ON RAJESH

CONCEPT, WE ARE DEVELOPING

COMPETITION FOR SPECIFIC NEEDS

TO SOFTWARE DEVELOPMENT FOR

MOLECULAR BIOLOGY DATA MINING

TOOLS.

THIS COULD BE ADOPTED AS WELL

DEFINE EDUCATIONAL PROBLEM AND

YOU LET COMPETITION COMPETE

LARGE COMMUNITY OF DIVERSE

STAKEHOLDERS AND THEN YOU HAVE A

BENCHMARKING PROCESS AND THAT IS

A KEY PIECE TO DEVELOP

INNOVATIVE CREATIVE EFFECTIVE

BENCHMARK,OBVIOUSLY THE COUNTER

COMPETITIONS BUT MIGHT BE A WAY

TO PULL IN UNEXPECTED IN A WAY

THAT YOU ARE NOT CONSTRAINING TO

ONLY ONE OR TWO BUT YOU GET WIDE

SPECTRUM OF SOLUTIONS, YOU CAN

SET UP COMPETITION THAT YOU HAVE

STAGGERED RESOURCE DISTRIBUTION

TO MAIN PERFORMANCE.

WHICH IS OUT AND COULD BE

ENGAGED.

VERY YOUNG COMPETITORS ENTERING

THESE SPACE AND SOMETIMES THEY

ARE NOT TRAINING LEADING EDGE.

>> YOU MADE COMMENTS IN CHAT.

SAY ANYTHING MOTHER?

>> I THINK WHAT YOU DESCRIBE THE

GOAL OF PROFESSIONALIZING

TRANSLATIONAL SCIENCE AS A

DOMAIN OF EXPERTISE IS

NECESSARY.

IF IF GOAL IS TO REACH YOUNGER

GENERATION I AGREE WITH RAGESH

DO AN EXPERIMENT ON THE SIDE AND

GUILTY IT BUT IF YOU ARE GOING

TO GAMEFY IT DON'T DO IT IN A

WEB 2.0, GO ALL THE WAY.

GO ALL IN AND DO IT IN A WEB 3.0

ENVIRONMENT THEN REACH THE

WORLD.

>> MAKE IT VR.

>> AGAIN, I DON'T WANT TO TAKE

UP ALL THE REST OF THE TIME, IT

IS LIKE I WAS JUST TEXTING

BECAUSE I WAS GETTING REALLY

INTO IT BECAUSE I'M INTO THIS

SORT OF THING AND WE BUILD THIS

STUFF.

SO IT JUST HIT A NERVE IF THE

GOAL IS THE YOUNGER GENERATION,

I THINK IT IS WORTH THINKING

CONTENT DELIVERY IN NEW WAY BUT

YOU HAVE BEEN SO THOUGHTFUL WHAT

THAT CONTENT IS.

IT WAS JUST WRAPPING.

>> I WOULD SECOND KELLY, I THINK

THE CONTENT IS NOT THE ISSUE.

IT WAS REALLY ABOUT HOW YOU WANT

TO GET THIS ACROSS.

WHO YOUR AUDIENCE IS.

IF YOUR AUDIENCE IS 30

SOMETHINGs AND BELOW WHO YOU

ARE WANTING TO BE THE NEXT

GENERATION HAVE GROWN UP

DIFFERENTLY THAN WE HAVE.

>> RIGHT.

YOU DID COME ACROSS AS SOUNDING

LIKE A LITTLE BIT LIKE YOU WERE

CRITICIZING THE CONTEMPT.

JUST MY OPINION.

IT IS GOOD YOU CLARIFIED THAT

YOU DIDN'T MEAN THAT.

>> GOOD.

THANK YOU.

KEITH I SEE YOUR HAND THEN WE

WILL LET BECK KY HAVE THE LAST

WORD HERE.

KEITH GO AHEAD.

>> I THINK YOU ARE MUTED.

>> YOU ARE ON MUTE STILL.

>> THANK YOU.

I'M INTRIGUED BY THE VALUATION

COMPONENT AND ESPECIALLY

INTERESTED IN BEING SURE WE WILL

LEARN HOW TO REACH DIFFERENT

AUDIENCES DIFFERENTLY, I DON'T

MEAN THE YOUTH AUDIENT.

THAT IS MADE ALREADY.

I'M THINKING IN TERMS OF

DIVERSITY, IT IS A CHALLENGE FOR

ME AS AN STROBING TORR EVEN IN A

LIVE ENVIRONMENT TO THINK HOW

DID I SAY THAT AND DID I SAY IN

A WAY THAT RESONATES WITH

DIVERSE AUDIENCE BY RACE

ETHNICITY AGE, ET CETERA.

RATHER THAN JUST GETTING BACK

COUNTS OF WHO AND WHAT TYPES OF

PEOPLE WENT THROUGH WHETHER

GETTING BACK GENERAL IMPRESSIONS

I WOULD LOVE TO SEE INNOVATION.

HOW DO WE LEARN HOW TO TAILOR

OUR PEDAGOGUE NO MATTER WHAT IT

IS TOWARD THE DIFFERENT

AUDIENCES.

AND FINALLY VERY USE -- THEIR

USE OF THAT IN THEIR

TRANSLATIONAL WORK AS THEY REACH

OUT, TO A MORE DIVERSE AUDIENCE.

>> WELL TAKEN.

THANK YOU.

BECKY.

>> IT SOUNDS LIKE THERE IS A

CONSENSUS AMONG ALL OF US THAT

TRADITIONAL LECTURE WITH

POWERPOINT SLIDES, PROBABLY AN

INEFFECTIVE STRATEGY FOR

CERTAINLY CAN BE BACKGROUND

MATERIAL BUT IT CLEARLY ISN'T

THE MOST EFFECTIVE WAY I THINK

IF YOU REALLY EXPAND THIS TO

THINK OF HOW CAN WE USE OTHER

ONLINE THAT INCLUDES LOTS OF

DIFFERENT APPROACH AND THINK HOW

THAT ACTUALLY BRINGS ACROSS

PROCESS GUILTYCATION SOMETHING

WE USE A LOT OF.

SOUNDS LIKE YOU DO TOO.

IT IS SIMPLY MORE ENGAGING BUT

BRINGING TOGETHER IN A WAY THAT

YOU HAVE MORE INTEGRATED SYSTEM

AND THEN THE WAY TO BE ABLE TO

SEARCH WHAT YOU NEED AT THE TIME

YOU NEED IT.

WOULD ADVANCE ALONG AS YOU MAKE

IT AN AVAILABLE SOURCE.

>> THANK YOU FOR THAT.

ANY FINAL THOUGHTS HERE TO

RELAY?

>> THIS IS HELPFUL I'M EXCITED

TO HEAR THE THEWS AM AROUND THE

CONTENT PIECE AND SOUNDS LIKE

THE OTHER SUGGESTIONS GO FURTHER

AS FAR AS FOSTERING MORE

CREATIVE APPROACH HERE.

THAT WOULD BE EXCITING FORWARD

THING FOR NCATS TO DO LIKE

ALWAYS.

>> THANK YOU SO MUCH.

WE HAVE A LOT TO THINK ABOUT.

WBEWILL MOVE TO THE NEXT CONCEPT

CLEARANCE, PRESENTATION.

>> WE NEED TO VOTE.

>> NO FURTHER DISCUSSION.

MOTION TO ADVANCE CONCEPT WITH

THEIR COMMENTS UNDER ADVISE.

>> SO MOVED.

>> SECOND?

>> SECOND.

>> ALL IN FAVOR

>> AYE.

AYE.

AYE.

>> ANY ABSTENTIONS?

NEXT CONCEPT PLEASE.

>> I WANT THE MAKE SURE YOU GOT

THE ONE EXTENSION THERE.

DID YOU GET IT, ANNA?

>> I ABSTAINED BUT I WAS ON

MUTE.

>> GOT IT.

WANT TO MAKE SURE.

>> WE WILL GO AHEAD AND MOVE TO

THE NEXT CONCEPT CLEARANCE.

THIS IS WITH OFFICE OF SPECIAL

INITIATIVES.

I WILL TURN IT OVER TO DAN

TAGLE.

PLEASE TAKE IT AWAY.

>> THANK YOU, GOOD AFTERNOON

EVERYONE, THANK YOU FOR HANGING

IN FOR THE LAST PRESENTATION OF

THE DAY.

I'M DAN TAGLE, DIRECTOR OF

OFFICE OF SPECIAL INITIATIVES

HERE TO GIVE A BACKGROUND ABOUT

THE OFFICE AS WELL AS SOME

BACKGROUND ABOUT THE INITIATIVE.

NEXT SLIDE PLEASE.

BRIEFLY AGAIN, I MENTIONED IN

CLOSE SESSION THE MISSION OF

OFFICE OF SPECIAL INITIATIVES IS

REALLY TO DEVELOP INNOVATIVE

SOLUTIONS THROUGH DISRUPTIVE

TECHNOLOGY AND OTHER

PARTNERSHIPS IN ORDER THE

ADDRESS THE MAJOR TRANSLATIONAL

PROBLEMS THAT THAT PERVADE IN

THE FIELD.

SO THIS IS A LISTING OF PROGRAMS

AND ACTIVITIES WITHIN OFFICE OF

SPECIAL INITIATIVES.

SO THE CONCEPT THAT IS UP FOR

YOUR DISCUSSION AND CONCURRENCE

IS THE -- SO THAT EXTREME STANDS

FOR EXOSOME BASED THERAPIES FOR

REGENERATIVE MEDICINE, THIS WILL

BE A NEW INITIATIVE WITHIN THE

OFFICE BUT I WANT TO MEMBERS OF

THEY JURY IT LEVERAGES PREVIOUS

INVESTMENTS FROM THE NIH COMMON

FUND.

EXTRA CELLULAR RNA COMMUNICATION

INDICATED IN ONE BULLET ABOVE

WHICH LOOKS AT SECRETED RNA

PRIMARILY FOR BIOMARKER THERAPY

DEVELOPMENT.

SO THERE IS FROM COMMON FUND IN

TERMS OF TOOLS AND RESOURCES

DEVELOPED IN ORDER TO ISOLATE

EXOSOMES.

SO NEXT SLIDE.

WHAT ARE EXSOME?

THEY HAVE BEEN RECOGNIZED AS AN

ARTIFACT OR PERHAPS CELL DEBRIS,

IT HAS ONLY BEEN RECOGNIZED

WITHIN THE LAST DECADE OR SO

THAT IT ACTUALLY CARRIES FORTH

AN IMPORTANT FUNCTION WITHIN THE

BODY.

THAT FUNCTION IS NOVEL MESSENGER

FOR INTERCELLULAR COMMUNICATION.

EXOSOMES ARE ESSENTIALLY

DIFFERENT SHADED FROM OTHER

MICROVESICAL MS. THE SENSE

MICROVESICALS FROM PLASMA

MEMBRANE WHEREAS ENDOSOMES ARE

RELEASED TO THE EXTRA CELLULAR

MATRIX WHEN VESICULAR BODIES

FUSE WITH CELL MEMBRANE SO

ENDOSOMAL ORIGIN.

THEY ARE PRETTY SMALL ORGANELLES

THEY ARE ABOUT 40 TO 150

NANOMETERS.

AND CARRY IMPORTANT BIOACTIVE

MOLECULES WHICH ARE IMPORTANT

FOR SIGNALING AS WELL AS FOR

OTHER CELLULAR FUNCTION.

SO THIS BIOACTIVE CARGO INCLUDES

LIPIDS PROTEINS, MESSENGER RNA

AND IMPORTANTLY ALSO REGULATORY

RNA SUCH AS MICRORNA AND LONG

NON-CODING RNAs.

THESE EXSOMES CONTAIN SURFACE

PROTEINS, ALLOWING IT TO TARGET

TO SPECIFIC CELL TYPES TO ALLOW

SPECIFIC COMMUNICATION BETWEEN

DIFFERENT CELLS.

AND DIFFERENT MILIEU.

I THINK WE HEARD FROM TARA

SCHWETZ EARLIER ABOUT ARPA H AND

WANT OF THE INTERESTS IN

MOLECULAR ZIP CODES, WE HAVE

EXOSOMES THAT ARE NOT POPULATED

WITH CELL SPECIFIC OR TISSUE

SPECIFIC MOLECULAR ZIP CODES

THAT ALLOW TO ZOOM IN WITH

SPECIFIC TARGETS.

IT HAS BEEN RECOGNIZED IMPORTANT

AS INTRACELLULAR SIGNALING

MOLECULES EXSOMES CAN BE USED TO

REPAIR TISSUE INHERENTLY IN ITS

OWN OR BECAUSE YOU CAN ACTUALLY

MANIPULATE NOT ONLY SURFACE

MARKERS BUT ALSO MANIPULATE

CARGO, YOU CAN LOAD UP WITH

VARIOUS THERAPEUTIC CARGOES SUCH

AS PROTEINS, GENE THERAPIES AN

VACCINES TO ALLOW SPECIFIC

TARGETING WITH MINIMIZING ANY

UNDUE EFFECTS IN THE TISSUE.

MENTION SLIDE.

NEXT SLIDE.

THIS IS A IN U CONCEPT

CLEARANCE, IT SAYS EXOSOME

THERAPEUTICS FOR REGENERATIVE

MEDICINE, DR. HAPPEL WILL DO THE

PRESENTATION.

SO SHE HAS A Ph.D. IN GENETICS

FROM THEM IT WILL UNIVERSITY

SCHOOL OF MEDICINE, SHE RECEIVED

THE RUTH KIRSTEIN POST-DOCTORAL

FELLOWSHIP AT HOPKINS WORKING ON

MICRORNA REGULATION AS WELL AS

DOING SECOND POST-DOC AT NCI.

SO CHRISTINE WILL BE TALKING A

LITTLE BIT MORE IN DETAIL ABOUT

THIS CONCEPT CLEARANCE.

SO I WILL TURN IT TO YOU,

CHRISTINE.

>> THANK YOU.

THIS IS A NEW CONCEPT THAT WE

ARE PROPOSING FOR NICELY NAMED

EXTREME.

NEXT SLIDE PLEASE.

DESPITE SUCCESS IN REGENERATIVE

MEDICINE MAJOR CHALLENGES ARE

MAIN IN THE FIELD.

STEM CELL TRANSPLANTATION

CURRENTLY MAIN METHOD FOR TISSUE

REGENERATION PROGRESS IS LIMITED

HURDLES INCLUDE RELIABLE CELL

SOURCE TUMOR FORMATION,

INAPPROPRIATE STEM CELL

MIGRATION, IMMUNE REJECTION OF

TRANSPLANTED STEM CELLS,

COMPLICATIONS DURING SURGERY AND

POST-OPERATIVE INFECTION.

WITH THIS CONCEPT NCATS PROPOSES

A NOVEL EXSOME BASED

THERAPEUTICS PROGRAM TO CATALYZE

REGENERATIVE MEDICINE CREATING A

THERAPEUTIC SO AS SEEN ON THE

SCHEMATIC ON THE RIGHT EXOSOMES

ARE SMALL ENDOGENOUS MEMBRANE

ENCLOSED CARRIERS.

ABOUT THE SIZE OF VIRUS

PARTICLE.

AND AS DAN SAID THEY CONTAIN

BIOACTIVE PROTEIN LIPIDS AND

NUCLEIC ACIDS, FOR INTRACELLULAR

COMMUNICATION.

EXOSOME BASED COMMUNICATION

OCCURS IN A BIDIRECTIONAL MANNER

AND TAKE PLACE DURING NORMAL

CELL HOMEOSTASIS AND CONSEQUENCE

OF PATHOLOGICAL DEVELOPMENT.

SO THE SCHEMATIC SHOWN HERE

SHOWS SIGNALING BETWEEN DISEASE

CELL AND STEM CELL WHERE DISEASE

CELL IS INDICATING DISTRESS.

THE STEM CELL THEN RESPONDS BY

RELEASING SPECIFIC REPAIR

EXSOMES.

WHAT MAKES THIS CONCEPT PARADIGM

SHIFT IS THAT UTILIZES

NON-LIVING CELLULAR PRODUCT

RATHER THAN STEM CELLS

THEMSELVES TO PROMOTE TISSUE

REGENERATION.

THERAPIES TO PROMOTE TISSUE

REPAIR A WOUND HEALING

TRANSLATED TO THE CLINIC.

EXTREME KNOWLEDGE RESOURCES

DEVELOPED BY COMMON FUND EXTRA

CELLULAR RNA COMMUNICATION

PROGRAM WHICH LOOK AT EXTRA

CELLULAR RNA CARRIERS SUCH AS

EXSOMES, AS CRITICAL MEDIATORS

OF INTRACELLULAR COMMUNICATION.

ALSO BUILD OFF RECENTLY

CONCLUDED COMMON FUND

REGENERATIVE MEDICINE PROGRAM,

AND LEVERAGE EXPERTISE OF NCATS

STEM CELL TRANSLATION

LABORATORY.

NEXT SLIDE.

SO THE OBJECTIVE OF THIS PROGRAM

IS TRANSFORM REGENERATIVE

MEDICINE THROUGH NOVEL EXOSOME

BASED THERAPEUTIC AND TISSUE

REPAIR WOUND HEALING.

AS SOON IN THE SCHEMATIC ON THE

RIGHT THERE ARE TWO WAYS THIS

CAN BE ACHIEVED.

THE PERSON HAS DIRECT EXOSOME

THERAPY WHICH UTILIZES EXOSOMES

ISOLATED STEM CELLS STORED AS

OFFICE THERAPY TO BE INFUSED IN

PATIENTS.

SO AN EXAMPLE OF DIRECT EXOSOME

THERAPY, WE FOUND RECENT

PUBLICATION WHERE THEY

DEMONSTRATED ENDOTHELIAL CELL

DERIVED EXOSOMES WERE ABLE TO

INDUCE CARDIAC TISSUE REPAIR,

FOLLOWING ISCHEMIC REPROFUSION

INJURY BY SUPPLEMENTING NEARBY

CELLS BY PROTEINS WITH CRITICAL

CELLULAR PROCESSES.

THE SECOND WAY THIS CAN BE

ACHIEVE IS THROUGH DESIGN

EVERYBODY EXOSOMES WHICH IS

PERSONALIZED ACCORDING TO

INDIVIDUAL PATIENT NEEDS.

THIS MEANS STEM CELL CELLS ARE

HARVESTED FROM PATIENT EXPANDED

AND MODIFIED BASED ON NEEDS

FOLLOWED BY GENERATION OF

EXOSOMES THAT ARE INFUSED BACK

INTO THE SAME PATIENT.

MODIFICATIONS INCLUDE GENETIC

MODIFICATION, ADDITION OF

THERAPEUTIC SMALL MOLECULES OR

CELL SURFACE RECEPTORS FOR

SPECIFIC EXOSOME TARGETING.

WE ANTICIPATE EXTREME ENCOMPASS

KEY AREAS OF EXOSOME

THERAPEUTICS.

DIRECT EXOSOME THERAPY, WOULD BE

MORMORE ACHIEVEABLE, DESIGNER

EXOSOMES PUSH ENVELOPE IN WHAT

CAN BE ACHIEVED.

IMPLEMENTATION OF THIS CONCEPT

INCLUDES FOCUS ON IND ENABLING

THERAPEUTICS AND ENTAIL EARLY

ENGAGEMENT WITH THE FDA TOWARD

REGULATORY APPROVAL.

UTILIZE A MILESTONE DRIVEN

APPROACH TOWARD THESE

THERAPEUTICS TRANSLATED TO

CLINIC, A CONSORTIUM OF FUNDED

INVESTIGATORS TO FACILITATE

SHARING RESOURCES AND EXPERTISE.

METRICS INCLUDE DEMONSTRATION OF

ABILITY TO REGENERATE DISEASE

ORGANS, FROM MANY HUMAN

DISORDERS AND THE VALIDATION OF

THESE THERAPEUTICS BENEFITS IN

VIVO OR EXVIVO.

SO THIS WOULD INCLUDE A

KNOWLEDGE OF SPECIFIC STEM CELL

DERIVED EXOSOME TO ENDEUCE

REPAIR AND WOUND HEALING AT

SPECIFIC TARGET ORGANS.

FINALLY WE EXPECT THAT THE

RESEARCH ACTIVITIES AND RESULTS

SPUR NUMBER OF COMMERCIAL

ACTIVITIES BEYOND INITIAL NCATS

INVESTMENTMENINVESTMENT.

NEXT SLIDE.

IN SUMMARY, EXOSOMES REPRESENT

THE THERAPY DUE TO NATURAL

FUNCTION AS MEDIATORS OF TISSUE

REPAIR AND WOUND HAILING.

NCATS PROPOSES THIS CONCEPT TO

TRANSFORM REGENERATIVE MEDICINE

APPROACHES THROUGH THESE NOVEL

EXOSOME BASED THERAPEUTICS,

CREATING A BUT THERAPEUTIC

PARADIGM.

THIS CONCEPT WOULD DEVELOP CELL

DERIVED HE CANSOME BASED

THERAPEUTICS FOR REGULATORY

APPROVAL AND TRANSLATION INTO

THE CLINIC TO THE BENEFIT OF

PATIENTS. WITH THAT WE WILL

BRING UP OUR QUESTIONS FOR

COUNCIL FOR DISCUSSION.

THANK YOU.

T

>> WE HAVE A COUPLE OF

DISCUSSANTS.

ANNIE WOULD YOU LIKE TO GO

FIRST?

>> I DON'T KNOW THAT I CAN

COMMENT ON THE SCIENCE ITSELF

THAT WAS DESCRIBED BUT I THINK

ONE OF THE THINGS AS I WAS

READING THIS PROPOSAL OVER THE

WEEKEND STRUCK BY WAS FIRST I

APPRECIATE THE INNOVATION NATURE

OF PERSONALIZED MEDICINE.

ONE OF THE THINGS THAT WE TALKED

ABOUT HERE ALREADY TODAY WE

TALKED ABOUT PREVIOUSLY, WAS AS

YOU ARTICULATED THE METRICS

DEMONSTRATION ABILITY TO REPAIR

DISEASED ORGANS FROM HUMAN

DISORDERS AND VALIDATION OF

THERAPEUTIC BENEFITS.

AND I JUST WONDERING IF SPECIFIC

TO THIS PROJECT AND PROGRAM IF

WE SHOULDN'T BE THINKING ABOUT

NCATS CHARGE AS BEING BEYOND

JUST REGULATORY APPROVAL AT THE

FDA?

BUT THINKING ABOUT DECISIONS NOW

HAPPENING WITHIN CMS.

AND HOW IF YOU THINK ABOUT

CONSORTIUM YOU BRING TOGETHER

STAKEHOLDERS AND BUILDING ON THE

PARADIGM YOU ARE THINKING ABOUT

WHAT THE IMPLICATIONS FOR THIS

WILL BE WITHIN THE ENVIRONMENT,

HOW -- WHAT OUTCOMES YOU ARE

LOOKING FOR AS YOU STRUCTURE

THIS TO SHOW THAT BENEFITS IN

VIVO EXVIVO ARE BETTER THAN WHAT

IS AVAILABLE NOW REIMBURSED

SUCH.

SO JUST SOME THINGS FOR

CONSIDERATION AS YOU ARE

BUILDING OUT THE SCOPE OF THIS

AND WE ARE THINKING WHICH

PARTNER SO THAT WOULD BE

DEPENDENT WHICH DISEASE

COMMUNITIES WHETHER YOU ARE

THINKING ABOUT MEDICARE

STRUCTURE OR MEDICAID STRUCTURE,

OR MORE COMMERCIAL ENTITIES.

SO JUST SOMETHING TO THINK ABOUT

MORE BROADLY.

AND THEN OBVIOUSLY DATA SHARING

COMPONENTS WHICH WE ALSO TALK

ABOUT WOULD BE INCREDIBLY

IMPORTANT TO CONSIDER.

THOSE WERE MY ONLY COMMENTS ALSO

EXTEND MORE BROADLY TO OTHER

PROGRAMS WE ARE CONSIDERING AS

WELL.

WE ARE THINKING ABOUT ACCESS

NOW.

NEVER TOO EARLY.

WE ARE THINKING ABOUT PROGRAM

AND HA IS A FANTASTIC WAY TO

KEEP OUR EYE ON THAT BALL.

I APPRECIATE THAT COMMENT.

>> VERY INSIGHTFUL QUESTIONS AND

THINGS WE WILL KEEP IN MIND AS

WE MOVE FORWARD.

THEY ARE VERY IMPORTANT BIG

PICTURE SUGGESTIONS, THANK YOU,

THAT'S HELPFUL.

>> KRISTINA, I THINK I CAN

COMPLIMENT THIS I WILL BE A

LITTLE MORE ON THE TAKING A

MOLECULAR BIOLOGY ASPECT.

I THINK VERY HIGHLY, RNA

CONSORTIUM HAS DONE A TREMENDOUS

JOB IN DEFINING ALL THE METHOD

LOGICAL CONSTRAINT IN THAT

SPACE, AND ASSETS LEVERAGE --

OBVIOUSLY WE HAVE SEEN SPECIFIC

SUCCESSES WITH THESE TYPE OF

INTERVENTIONS, FROM BASIC

SCIENCE APPROACH, MY CONCERN

STRATEGIES THAT IT IS BLACK BOX

APPROACH.

YOU TRANSFER BIOLOGICAL ACTIVITY

WITHOUT CHARACTERIZATION OF

BIOLOGICAL ACTIVITY INTO VARIOUS

DISEASE SETTINGS.

AND WHY THIS APPROACH CAN BE

SUCCESSFUL A NEPHROLOGIST WHO

HAS DONEPHORESES TREATMENTS AND

STUDIES, IT REMAINS BLACK BOX.

YOU WILL HAVE RESPONDERS AND

NON-RESPONDERS AND IT WILL BE

DIFFICULT OR IMPOSSIBLE TO

DEFINE WHO IS RESPONDING AND FOR

WHAT REASON.

THE SCIENCE HAS MOVED FORWARD AS

WE DISCUSSED EARLIER WE HAVE NOW

AN OPPORTUNITY TO ACTUALLY KNOW

THE BAR CODES, YOUR VESICLES ARE

HEADING TOWARDS.

YOU CAN SELECT AND DESIGN YOUR

VESICALES, YOUR VESICLE SORT AND

TYPE GETS PREDEFINED AND

CONTENTS CAN BE DEFINED AS WELL.

WHILE THIS MIGHT BE NOT IN SCOPE

OF THE SPECIFIC OPPORTUNITY I AT

LEAST WOULD DESIGN OPPORTUNITY

SUCH THAT THIS MORE TARGETED

APPROACH CAN BE DEVELOPED OUT OF

CLINICAL IMPLEMENTATION STRATEGY

SO WILL IS A AN ARM BUILT IN

WHEN THE VESICALES ARE

TRANSFERRED AND DEFINED

RESPONSES WHICH THE TECHNOLOGIES

I THINK ARE THERE NOW BASED ON O

CONSORTIUM EFFORT YOU CAN

DAPTURE WHO IS RESPONDING AND

WHAT VESICAL -- CAPTURE WHO

RESPONDING AND WHAT VESICLE

RESPONDS TO MENTION STAGE

TOWARDS MOLECULAR DEFINED

INTERVENTIONS.

OBVIOUSLY VESICALLY TARGETING

TRANSCRIPTS WE HAVE SOME OF

THOSE INTO OUR SHOULDERS

RECENTLY.

AND THEY HAVE BEEN MAJOR BREAK

THROUGH AND FULL DISCLOSURE WE

ARE WRITING WITH SUCH ENTITIES

TO TARGET VESICALES TOWARDS

SPECIFIC CELLS AND SPECIFIC

DISEASES.

SO IN A SINGLE MOLECULE DEFINE

APPROACH ALREADY MOVING FORWARD

BUT YOU STRATEGY MIGHT BE

INTERMEDIATE SPACE WHERE YOU CAN

UNDERSTAND WHERE BIOLOGY

LEVERAGING PRINCIPLE TO COMMENT

TOWARDS MOLECULAR CHEMICALLY

DEFINED APPROACH.

SO CERTAINLY MOVE FORWARD.

BUT TRY TO BUILD DISCOVERY

COMPONENT IN TO THE

TRANSLATIONAL ASPECT

INCENTIVIZING.

>> THANK YOU FOR THOSE COMMENTS.

CHRISTINE.

DO YOU WANT TO --

>> YEAH.

THANK YOU FOR THOSE COMMENTS.

I THINK YOU ARE CERTAINLY RIGHT

IN THAT RIGHT NOW THESE --

PEOPLE ARE STARTING TO GET TO

THIS AREA AND RIGHT NOW IT IS A

LITTLE BIT LIKE BLACK BOX BUT

THAT IS WHY I THINK NCATS IS SO

WELL-POSITIONED TO DIVE IN AND

MAKE SIGNIFICANT IMPROVEMENTS.

BECAUSE WE ARE LEVERAGING A LOT

OF VERY NEW TECHNOLOGIES THAT

HAVE BEEN DEVELOPED.

SO THE M CATS STEM CELL

LABORATORY THEY ARE WELL -- VERY

WELL EXPERIENCED IN STEM CELL

ISOLATION.

SO WE CAN UTILIZE THEIR

EXPERTISE TO GENERATE WELL

CHARACTERIZED SOURCES OF THESE

STEM CELL EXOSOMES AND UTILIZE

KNOWLEDGE FROM EXTRA CELLULAR

RNA COMMUNICATION PROGRAM AN

EXOSOME LYSE ICE LAKES METHODS

THEY DEVELOPED, TO REALLY

CONTRIBUTE TO MAKING SOME VERY

WELL CHARACTERIZED EXOSOME

SOURCES BY PIECING ALL THESE

DIFFERENT TECHNOLOGIES THAT HAVE

BEEN DEVELOPED SO UTILIZING THE

PRODUCTION OF WELL CHARACTERIZED

STEM CELLS FROM A STEM CELLS

TRANSLATION LABORATORY UTILIZING

EXOSOME ISOLATION TECHNOLOGY

FROM EXC CELLULAR COMMUNICATION

PROGRAM AND ALSO SOME OF THE

RESOURCES FROM THE REGENERATIVE

MEDICINE SONOR CONSORTIUM SO IT

IS THIS WHEN YOU FIT PIECES

TOGETHER I THINK REALLY

EVERYTHING IS THERE TO HELP A

MAKE LESS A BLACK BOX AND MORE

WELL DEFINED.

EXACTLY WHAT THE MECHANISM

ACTION IS FOR THESE EXOSOMAL

THERAPIES SO WE UNDERSTAND

EXACTLY WHAT IS TAKING PLACE AND

WHAT IS DERIVING THE THERAPEUTIC

EFFECTS.

>> IF YOU MAKE SURE THE ACTIVITY

IS NOT ONLY LOOKING AT CLINICAL

EFFICACY BUT ALSO BUILT IN

SPECIFIC AIMS TOWARD TO BUY

GENERATING EFFICACY SIGNALS ALSO

ALLOWING TO UNDERSTAND WHICH

MOLECULAR COMPONENT EFFICACY IS

TARGETING TO.

THAT IS MY MAIN COMMENT.

>> GREAT COMMENT.

THANK YOU.

SO

>> SO WE WILL OPEN FOR

DISCUSSION.

I SEE MARSHALL AND RAGESH.

>> ONE OF MY COLLEAGUES IS DOING

WORK IN THIS.

WHAT IS IT ACTUALLY DOING?

IT GETS HAND WAVING QUICKLY.

WHAT ARE THE COMPONENTS HAVING

THERAPEUTIC EFFECT?

WE DON'T KNOW.

THERE'S SOMETHING THERE AND

THINGS LIKE THAT ONE THING I

MIGHT SUGGEST ANYWAY, IS THINK

ABOUT PARTNERING WITH ANOTHER

INSTITUTE WHETHER NSF OR

SOMEBODY ELSE, WHO THE DO THE

BASIC SCIENCE ON THIS.

YOU WILL HAVE WELL CHARACTERIZED

EXOSOMES FROM WELL CHARACTERIZED

STEM CELLS, THAT DOESN'T MEAN

YOU KNOW WHAT IS IN THEM.

AND WHICH HAVING BIOACTIVE

PROPERTIES.

SO WHEN WE WERE DOING PEOPLE

TRANSPLANTS, TAKING ONE TO THE

OTHER PATIENTS WERE SEEING

IMPROVEMENT, I DIDN'T DO THOSE

IN GENETICS.

WHEN YOU ASKED WHAT IT WAS

TRANSPLANTED NO ONE KNEW WHAT IT

WAS.

YOU ARE IN A MUCH BETTER

POSITION BUTTONLY RECOMMEND

PARTNERSHIP WITH TO GET A GOOD

ABASIC SCIENCE COMPONENT OF

THIS.

>> SO PEOPLE DON'T THINK I WOKE

UP ON THE WRONG SIDE OF THE BED,

I DO LIKE THIS PROPOSAL AND

WANTED TO SAY SO.

>> THANK YOU, RAJESH.

I CAN JUMP IN, I THINK THAT THE

ISOLATION TOOLS OF EXTRA

CELLULAR RNA COMMUNICATION

PROGRAM HAS DEVELOPED AS WELL AS

THE ANALYTICAL TOOLS.

I THINK ARE REALLY GOING TO BE

ABLE TO ADDRESS A LOT OF THE

BASIC CONCERNS ABOUT MECHANISM

OF ACTION BECAUSE HISTORICALLY

HE IS SMALL VESICALES ARE

DIFFICULT TO PURIFY.

BUT SOME OF THESE TECHNOLOGIES A

LOT ARE MICROFLUIDIC BASED OR --

THERE'S A LOT OF DIFFERENT

TECHNOLOGIES BUILT THROUGH THIS

PROGRAM.

THEN USING THESE TOOLS WILL HAVE

MUCH MORE HOMOGENOUS PRODUCTION

OF THESE EXOSOMES THAT ARE MORE

WELL CHARACTERIZED.

SO I THINK THAT WILL GO A LONG

WAY INTO REALLY ANSWERING A LOT

OF THOSE QUESTIONS MANY TERMS OF

MECHANISM OF ACTION AND WHAT IS

REALLY RESULTING THIS IN

THERAPEUTIC EFFECT.

>> YOU WILL HAVE A PURE SOURCE

DEEP DIVING EVERYTHING -- EVEN

IF SMALL MOLECULES, DOESN'T HAVE

TO BE BIOLOGIC.

GOD KNOWS WHAT IS IN THESE

THINGS.

>> EXACTLY.

ANALYTICAL TOOLS THAT GO WITH

THESE ISOLATION TECHNOLOGIES,

SHOULD REALLY ALLOW US TO

PINPOINT THE EXACT CARGO

RESPONSIBLE FOR THESE

ACTIVITIES.

SO I'M CONFIDENT THAT THE NEW

TECHNOLOGIES WHICH I KNOW ARE

LOT OF THESE TECHNOLOGIES ARE

JUST GETTING TO THE POINT WHERE

BEING PUBLISHED, BUT I THINK

THEY ARE GOING TO BE A VERY

HELPFUL IN TERMS OF MAKING THESE

-- EXOSOMES USEFUL IN TERMS OF

THERAPEUTICS.

>> STEVE WHAT IS CHANGING IS

THAT WE NOW ARE LEARNING ZIP

CODES WHERE THEY ARE HEADED.

AND WHERE WHAT IS DIFFERENT FROM

THREE FOUR YEARS AGO WHERE THE

EXOSOMAL RNA CONSORTIUM HAD TO

BROADEN THE -- CONCERNING THAT,

THAT'S THE OPPORTUNITY YOU

SHOULDN'T MISS.

SO THAT COUPLE THAT IN DIFFERENT

WAYS YOU CAN CAPTURE SIMILAR

COMPONENTS SIMULATING RNAs IF

YOURY ARE ASSOCIATING WITH

INVASIVE -- TO GET THESE IN YOU

CAN CAPTURE THE CELLS IN THE

NEIGHBORHOOD, YOU ARE EXPOSING

TO YOUR EXOSOMES SO CAN

UNDERSTAND DRUG AND OBVIOUSLY

THERE IS A TIME ON CHIP AND N

CATS BEAUTIFUL PLATFORM YOU CAN

HAVE EXVIVO STUDIES TO SEE IF

YOU BUILD THESE PROCESSES UP IN

HUMANS IN VIVO STUDIES YOU CAN

FOLLOW-UP MECHANISTICALLY AS

WELL SO VERY NEATLY CROSS

FERTILIZATION IN MANY ONGOING N

CATS AND COMMON FUND ACTIVITIES.

>> I WANT TO MAKE ONE MORE

SUBSTANTIVE COMMENT BECAUSE OF

SOMETHING SAID SINCE KEN TO

MINE, WHICH IS WE ARE LOOKING

FOR PURIFIED SOURCES TO DO

BIOLOGY, ONE OF THE COOL THINGS

I HEARD MORE RESEASON WILL I IN

THIS ARENA IS THINKING ABOUT

EXOSOMES IN THE CONTEXT OF GUT

BRAIN ACCESS.

SO THE POSSIBILITY THAT THE

EXOSOMES MIGHT BE DERIVED FROM

PARTICULAR BACTERIAL STRAIN IN

YOUR GUT BUT HAS MEANINGFUL

EFFECT IN THE NERVOUS SYSTEM

MIGHT BE OUTLANDISH TO BELIEVE,

ALLOWS YOU A TESTABLE HYPOTHESIS

BECAUSE IT GIVES YOU A SOURCE OF

EXOSOMES THAT YOU CAN ACTUALLY

THEN PERHAPS MAKE IN LARGE

CULTURES PURIFY, IDENTIFY, MIX

AND MATCH, DO THOSE THINGS THAT

MIGHT GIVE YOU MORE TRACTION

INTO WHAT THE UNDERLYING

MECHANISM MIGHT BE FOR

THERAPEUTICS ATTENTION.

>> THE MAIN AREAS FOR

THERAPEUTIC APPLICATIONS RIGHTS

NOW ARE HEART LUNG KIDNEYS

NEURODEGENERATIVE DISORDERS, AND

EVEN SUCH THING WOUND HEALING

AND CANCER SO THOSE ARE THE MAIN

CURRENT APPLICATIONS THE LOW

HANGING FRUIT BUT THERE IS

TREMENDOUS AMOUNT OF

OPPORTUNITIES IN THIS AREA TO

SEE WHAT THIS THERAPEUTICS TO

DO.

AND THE SYNERGISM WITH CHIP,

WITH THAT WE CAN EVALUATE THAT

THERAPEUTIC POTENTIAL AND BREAK

THINGS DOWN A LITTLE BIT MORE.

THERE IS SYNERGY BEAN THOSE TWO

PROGRAMS AS WELL THAT MAKES

EXCITING.

THE TISSUE CHIP PROGRAM DOES

HAVE BLOOD BRAIN BARRIER CHIP SO

THAT WOULD BE A WAY TO LOOK AT

THAT AND ASSESS THAT.

>> THANK YOU SO MUCH CHRISTINE.

I BELIEVE I WILL TURN TO ANNA

NOW AS WE GET CLOSE TIME HERE.

NEED TO TAKE A VOTE.

I REMEMBERED.

THROW IT OVER TO YOU.

>> THANK YOU.

HEARING NO FURTHER DISCUSSION, I

WOULD LICK TO ASK YOUR APPROVAL

FOR THIS CONCEPT, DO I HAVE A

NOMINATION?

>> MOVED.

>> SECOND.

>> SECOND?

>> SECOND.

>> ALL IN FAVOR?

>> AYE.

AYE.

AYE.

AYE.

>> ANY OPPOSED?

ANY ABSTENTIONS?

WITH THAT THE SECOND CONCEPT OF

THE DAY IS APPROVED.

THANK YOU VERY MUCH.

BACK TO YOU, JONI.

>> THANK YOU SO MUCH ANNA AND

REST OF THE TEAM FOR THE

PRESENTATIONS TODAY.

I THINK THIS IS THE MOMENT WE

JUST WRAP UP OUR FIRST DAY HERE.

AND I WANT TO INVITE YOU TO

PROVIDE ANY COMMENTS OR

QUESTIONS HERE BEFORE WE CLOSE

OUT TODAY.

JUST QUICKLY MAKE SURE I'M NOT

SEEING ANY.

ANY LAST MINUTE QUESTIONS PLEASE

RAISE YOUR HAND.

IN THAT CASE WE WILL RECONVENE

TOMORROW AFTERNOON AT 1 P.M.

EASTERN AND I LOOK FORWARD THE

SEEING YOU BACK THEN.

I DON'T NEED THE GAVEL OUT NOW

BECAUSE WE ARE STILL IN OPEN

SESSION UNTIL TOMORROW AFTER 5.

STAY TUNED WE WILL SEE YOU

TOMORROW, THANK YOU FOR YOUR

ATTENTION AN ENGAGEMENT TODAY.