WELCOME TO THE 16th NINDS NON-PROFIT FORUM. THIS IS ALWAYS ONE OF MY FAVORITE DAYS OF THE YEAR WHERE AT THE FORUM, WE PROVIDE AN OPPORTUNITY FOR THE LEADERS OF ALL THE DISEASE ORGANIZATIONS AND OUR PORTFOLIO AND TO NETWORK WITH THEMSELVES AND HAVE DISCUSSIONS WITH NINDS STAFF AND LEADERSHIP. WE HAD HOPED TO RUN A HYBRID EVENT BUT WE ARE IN MONTGOMERY COUNTY SO THE DECISION WAS TO GO VIRTUAL, HOPEFULLY NEXT YEAR WE CAN COME BACK IN-PERSON. WE HAVE OVER 240 REGISTRANTS FOR THE EVENT AND WE WILL BE RECORDING THE MAJORITY OF THE SESSIONS THAT WILL BE AVAILABLE AND TO LOOK WHAT THE WAS DISCUSS. NOW, EACH YEAR WE HAVE AN EXECUTIVE COMMITTEE THAT WE WORK WITH TO DETERMINE WHAT IS THE THEME AND THE AGENDA FOR THE MEETING AND THE AGENDA AND THEME THIS YEAR IS HOW TO WORK WITH QUOTE-UNQUOTE, ANYBODY FROM INDUSTRY OR RESEARCH PARTNERS TO NIH TO IMPROVE AND ADVANCE THE RESEARCH TO GET BETTER TREATMENTS FOR PATIENTS WITH NEUROLOGICAL DISORDERS. AND SO I WANT TO GO THROUGH A COUPLE OF UPDATES FROM NINDS. MISSION OF OUR INSTITUTE IS TO SEEK FUNDAMENTAL KNOWLEDGE ABOUT THE BRAIN AND THE NERVOUS SYSTEM AND TO USE THAT KNOWLEDGE TO REDUCE TH THE PEOPLE HERE KNOW WE'RE UP AGAINST IN TERMS OF THE CHALLENGES AND THERE'S A LOT OF CHALLENGES AND MANY DIFFERENT DISORDERS AND ALL OF WHICH CORE A LOT OF SUFFERING AND TRAGEDY FOR PEOPLE AND MOST OF THEM HAVE LITTLE IN THE WAY OF REALLY EFFECTIVE TREATMENTS. WE HAVE SOME TREATMENTS AND SOME CONDITIONS BUT IN GENERAL, WE'RE STILL KIND OF SCRATCHING THE SURFACE THERE SO RESEARCH IS REALLY WHAT WILL LEAD TO BETTER TREATMENTS ANDTREATMENT AND THAR MISSION TO TRY AND GET US THERE. AS I MENTIONED WHEN I STARTED, THE FOREARM AND THE TOPIC AND THE THEME OF PARTNERSHIP HOW TO WORK WITH FOLKS FDA WAS SET BY THIS COMMITTEE AND PEOPLE ARE SEEING HERE ON THE LEFT AND FROM THE DIFFERENT DISEASE ORGANIZATIONS THAT REALLY SET THE TONE AND THE PEOPLE ON THE RIGHT AND THE NINDS PEOPLE WHO WORK WITH THEM AND THEY TROY TO PUT THIS SESSION, THESE SESSIONS TOGETHER. AND I DO HAVE ONE BITTERSWEET ANNOUNCEMENT TO MAKE AND THAT IS Dr. NINA SHORE, WHO HAS BEEN THE DEPUTY DIRECTOR AT NINDS IS GOING TO LEAVE NINDS AND INSTEAD SHE'S GOING TO ANOTHER HIGHER LEVEL MOVING TO LEAVE FOR ALL OF NIH'S INTRAMURAL RESEARCH AND SHE HAS BEEN THE ACTING SITE OF THE DIRECTOR IN OUR INTRAMURAL PROGRAM OVER THE LAST YEAR OR SO AND WE'LL HAVE A GREAT PARTNER AS WE MOVE INTO OUR NEXT PHASE IN THE INTRAMURAL PROGRAM. NOW, NINDS RECEIVES A BUDGET FROM CONGRESS AND THAT BUDGET IS THEN USED TO ADVANCE THE MISSION AND I MENTIONED THIS BEFORE THAT WE TRY TO HAVE MECHANISMS TO FUND RESEARCH THAT GOES FROM THE MOST BASIC RESEARCH AND ALL THE WAY TO PATIENTS AND EVERYTHING IN BETWEEN AND TRY VERY HARD NOT TO LEAVE FUNDING GAPS WHERE WHERE THERE'S NOT A MECHANISM TO MOVE THINGS FORWARD TO GET TO PATIENTS. THAT BEING SAID, MOST OF OUR FUNDING GOES INTO WHAT WE CALL BASIC RESEARCH AND THAT IS 20% TO 25% UNDER HOW THE BRAIN WORKS, DISEASE AGNOSTIC AND ABOUT 50% IS MECHANISMS OF DISEASES SO, WHAT IS GOING ON IN THE BRAIN AND PEOPLE WHO HAVE EPILEPSIES OR PEOPLE THAT HAVE DIFFERENT NEURODEGENERATIVE CONDITIONS. THAT'S BASIC RESEARCH AND THAT'S WHERE MOST OF OUR MONEY GOES, PROBABLY 75%. THE REST IS SPLIT BETWEEN TRANSLATIONAL RESEARCH WHICH IS MOVING THINGS FROM THE BENCH TO THE BEDSIDE THROUGH THE PATHWAY THAT IS NECESSARY TO BRING A THERAPY TO PATIENTS AND THAT IS NOT TRIVIAL. THERE ARE SAFETY CONCERNS, THERE ARE LOTS OF STEPS THAT YOU HAVE TO GO THROUGH JUST LIKE A COMPANY WOULD TO MOVE THERAPY TO PATIENTS THROUGH ALL THE STEPS, GETTING IT AT THE END, APPROVAL BY THE FDA TO TRY SOMETHING IN PATIENTS AND SO-CALLED IND OR IDE. AND WE HAVE HERE AT NINDS ARE LIKE A VIRTUAL PHARMACEUTICAL COMPANY THAT CAN HELP PEOPLE MOVE THINGS POINT TO THE IND FOR THE FDA. MANY OF OUR WORKS IN THE FARM SIDE KALIN DUSTY AND I MUST SAY MOST OF THE PEOPLE THAT WE WORK IN THIS SPACE ARE GENERALLY TIED TO SOME COMPANY, BIOTECH, SOMETIMES A LARGER PHARMA COMPANY AND OFTEN TIMES AN ACADEMIC ASSOCIATED WITH A COMPANY AND THIS IS WHERE WE DO PARTNERSHIPS WITH OUR INGUS TREE PARTNERS AND THE THEY'RE COMPARE OR EFFECTIVENESS WHERE YOU TEST TWO AGENTS THAT ARE AVAILABLE AND BEEN APPROVED BY THE FDA AND TO SEE WHICH ONE IS BETTER FOR A PARTICULAR DISEASE AND WE ALSO HAVE SO MOVING FROM THE IND STAGE TO PEOPLE WOULD BE A PHYSICIAN ONE STUDY TO SHOW IT'S SAFE AND IN A PHASE 2 STUDY LOOKING FOR SIGNS OF TARGETING GAGEMENT OF PROOF OF PRINCIPLE AND WE HAVE NETWORKS THAT WILL DO THAT AND. WE WOULD MUCH RATHER HAVE THE INDUSTRY TAKE THAT OFF OUR HANDS, BUT UNFORTUNATELY, FOR MANY OF THE SORTERS IN OUR PORTFOLIO, THE INDUSTRY ISN'T READY AND THEY FEEL IT'S TOO RISKY. ALL ACROSS THE BOARD IN OUR TRANSLATIONAL PROGRAMS AND OUR EARLY TESTING, IN PATIENT PROGRAMS, THE IDEA IS TO DERISK THESE THERAPIES SO THAT INDUSTRY WILL EVENTUALLY PICK THEM UP BECAUSE WE DON'T MAKE DRUGS BUT WE'RE TRYING TO DERISK THOSE DRUGS FOR THE INDUSTRY. IF YOU LOOK WHERE OUR MONEY GOES, IT GOES TO INVESTIGATOR INITIATE OR RESEARCH, THE DARK BLUE PIECE. THIS IS THE BEAUTY OF THE AMERICAN SYSTEM, WHERE EVERYONE CAN HAVE THEIR IDEA SENT INTO NIH AND REVIEWED BY A STUDY SECTION AND THE BEST IDEAS FLOAT TO THE TOP AND IT GETS FUNDED AND WE TEND TO BE VERY COGNIZANT OF THE STUDY SECTIONS AND THE CRITIQUES THEY PUT OUT, GUIDE MUCH OF OUR FUNDING DECISIONS IN THIS SPACE. ANOTHER COLORS ARE PARTICULAR AREAS THAT ARE FILLED IN SUCH AS TRAINING OR SOME OF THE PROGRAMI MENTIONED BUT THE MONEY GOES TO INVESTIGATOR INITIATIVE RESEARCH. THERE ARE PRETTY COMPLICATED MECHANISMS HERE TO KNOW WHAT IS THE RIGHT MECHANISM FOR YOUR TYPE OF RESEARCH? WHETHER IT'S WITH AN INDUSTRY PARTNER OR ACADEMIC PARTNER AND WE'VE TRIED TO MAKE THAT TRANSPARENT AS BEST WE CAN AND THIS IS A PROGRAM WHERE WE HAVE A PODCAST THAT GOES THROUGH ALL THE DIFFERENT PROGRAMS WE HAVE AND EXPLAINS HOW ONE MIGHT GET INVOLVED IN ANY OF THESE PROGRAMS. THE NEXT SLIDE IS REALLY COMPLICATED AND I MENTIONED BEFORE THAT WE WANTED TO MAKE SURE WE HAVE A FUNDING MECHANISM FOR EVERY STEP THAT GOES FROM A DISCOVERY STAGE TO AN ACTUAL DRUG OR TREATMENT IN PATIENTS AND SO WE HAVE PROGRAMS THAT ARE WHAT WE CALL PRO CLINICAL READINESS OR PROOF OF CONCEPT PROGRAMS SO THESE ARE EARLY STAGE PROGRAMS. WE HAVE ACTUALLY CLINICAL TRIAL READINESS GRANTS. TO STUDY PATIENTS AND GETTING UNDERSTANDING WHAT THE OUTCOME MEASURE MIGHT BE FOR A COMING CLINICAL TRIAL. THEN WE HAVE THERAPEUTIC DEVELOPMENT PROGRAMS AS THE CLINICAL TRIAL READINESS AND WE HAVE PHASE 1 IN HUMAN STUDIES AND PHASE 2 FIRST AND TARGET POPULATION AND PHASE 2 PROOF OF CONCEPT, BIO MARKERRING AND INFORM STUDIES AND THEN A PHASE 2 PRELIMINARY EFFICACY AND WE RRARELY DOPHASE 3s. WE DON'T DO ANY PHASE 4 POST MARKETING BUT WE DO COMPARATIVE STUDIES AND IMPLEMENTATION STUDIES. IT'S A LONG, COMPLICATED CHART AND I'M FEELING IT IN HERE. IT'S MUCH TOO COMPLICATED TO REMEMBER. THE KEY THING IS WE HAVE PROGRAM DIRECTORS IN CHARGE OF THESE AND THEY CAN GUIDE FOLKS TO KNOW AND WHICH OF THESE PROGRAMS IS THE BEST THING FOR WHAT YOU WANT TO DO. ALL THESE PROGRAMS IN THE TRANSLATIONAL SPACE ARE WITH A INDUSTRY PARTNER AND MUCH OF OUR WORK IS FOCUSED ON SOME COMMON DISORDERS SUCH AS EPILEPSIES OR STROKE OR NEURO DEAGAIN NA TIVE CONDITIONS AND WE HAVE THE GREATEST NUMBER OF OUR DISEASE ARE THE RARE DISEASES AND SO WE HAVE A LARGE INVESTMENT IN RARE DISEASE AND THE CONSORTIUM THAT RUN OUT OF NCATS HAS MANY NEUROLOGICAL CONSORTIUM WITHIN IT AND WE FUND THESE NEUROLOGICAL CON SORRY SUM HAS PART OF THE RARE DISEASE CLINICAL RESEARCH NETWORK AND IT'S BEEN A VERY PRODUCTIVE ENDEAVOR OVER THE YEARS. THE TOPICS VARY AND THEY RUN A PERIOD OF ABOUT FIVE YEARS AND NEW GRANTS COME IN IN THIS RARE DISEASE CONSORTIUM AND WE ARE MOVING INTO THE SPACE OF DEVELOPING GENE THERAPIES FOR OUR RARE NEUROLOGICAL DISORDERS AND MOST OF WHICH ARE RELATED TO A GENETIC ABNORMALITY SO WE ARE ONE OF THE PARTNERS IN THE ACCELERATED MEDICINE PARTNERSHIP WHICH IS RUN BY THE FOUNDATION OF NIH AND WE'LL BE HEARING FROM JULIE WHO IS THE DIRECTOR SHORTLY AND SO THIS IS A PARTNERSHIP WITH MULTIPLE INDUSTRY AND MULTIPLE NON-PROFIT ORGANIZATIONS AND THE IDEA IS TO DEVELOP A PLATFORM THAT YOU CAN PLUG IN DIFFERENT GENE THERAPIES INTO A DELIVERY MECHANISM USUALLY WHAT WE CALL A V VIRUS THAT WOULD DELIVER THE GENE THERAPY TO THE TISSUE AND ORGANIZE OF INTEREST AND THIS PROGRAM IS TO DEVELOP THAT KIND OF PLATFORM AND STREAM LOIN THE DREAM THEY WILL PEE DEVELOPMENT PROCESS AND DO IT IN THE SAMEST MASTER AS POSSIBLE. A LOT OF IT HAS TO DO WITH ADVANCING HOW THIS VIRUS DELIVERS THE DIFFERENT JEANS AND IN THE GENE THERAPY WORLD, THE REAL CRUX IS IN THE DELIVERY SIDE OF THINGS AND THE GOWNS THEMSELVES ARE MORE EASILY DEVELOPED AND UNDERSTOOD THAN THE DELIVERY MECHANISMS AND JUST ONE EXAMPLE IS A WORK THAT'S BEEN COMING OUT OF THE BRAIN INITIATIVE AND TO DEVELOP VIRUSES THAT ARE TARGETING THE BRAIN AND THE NERVOUS SYSTEM AND THERE'S A BOONE BARRIER FROM GETTING INTO THAT THE BRAIN AND THAT IS AN ISSUE FOR THE VIRUSES TOO WHICH LIKE TO GO TO THE LIGE RIVER BUT THEY HAVE SOPHISTICATED STREAMING THAT WILL DELIVER THE GENES TO THE BRAIN AND STAY AWAY FROM THE LIVER WHERE YOU SEE THE TOXICITY SO THIS IS REALLY EXCITING AND FUTURISTIC BUT MAYBE NOT THAT FAR AWAY. IN NINDS WE DECIDED PARTICULARLY UNDER THE LEADERSHIP OF Dr. SHORE TO MAKE A BIG UNUSUAL THIS AREA AND START AT THE ULTRARARE GENE BASED THERAPY NETWORK CALLED THE THIS IS TO DEVELOP GENOMIC THERAPIES AND THEY CAN BE NUCLEO TIDES AND THEY ARE ANTI SENSE KNEW CLEO TIDES THAT CAN BLOCK THE PRODUCTION OF A HARMFUL GENE PRODUCT AND OR VIRAL VECTOR BASED AND WE JUST MENTIONED AND MAYBE EVEN GENOME EDITING PROCESSES SO THESE NOW THE ABILITY TO GO IN AND SNIP OUT AWE MUTATION THAT IS CAUSING TROUBLE AND REPLACE IT WITH THE NORMAL THIS IS BEING DONE IN SICKLE CELL DISEASE WHERE YOU CAN TAKE OUT THE RED BLOOD CELLS, EDIT THEM, WIPE OUT THE GONE MARROW AND REPLACE IT WITH THE EDITED PRO AGAIN TOE CELLS AND CURE SICKLE CELL THAT WAY SO THERE ARE A MATTER OF TIME BEFORE WE WORKOUT HOW TO DO THIS IN THE NERVOUS SYSTEM AND RIGHT NOW, WE HAVE AND THESE ARE USUALLY BIO AND THESE ARE MANUFACTURE THE THE GREEN PRODUCT AND TREAT PATIENTS AND SO THAT IS OUR VERY AMBITIOUS PLAN AND I THINK IT'S WE'RE JUST BEGINNING SO YOU HAVE TO GO SLOW AND CAREFULLY AS WE DO THIS AND THIS IS WHERE I THINK THE FUTURE LIES AND MANY OF OUR DISORDERS. I DID MENTION GENE EDITING AND JUST TALKING ABOUT THE URGENT NETWORK AS SOMETHING THAT IS POTENTIAL IN THE FUTURE AND I'M ALSO CO DIRECTING A COMMON FUND PROJECT DEVELOPING THESE TOOLS THAT I MENTIONED WHICH CAN THEN CUT OUT THE ABNORMAL GENE AND THE NORMAL GENE AND USING THESE REALLY ADVANCED GENOMIC TECHNIQUES CALLED SAY CRISPR CAS 9 AND SO IN THIS COMMON FUND PROJECT, THE FIRST FIVE YEARS, WE FUNDED GROUPS TO DEVELOP REALLY POWERFUL TOOLS AND THE SECOND FIVE YEARS WE'RE HOPING TO ACTUALLY TEST THOSE TOOLS AND REFINE THEM BUT ALSO TO BEGIN, IF SAVED, TO START TESTING THIS THERAPY IN A COUPLE OF GENOMIC DISORDERS BECAUSE THESE ARE FIRST IN HUMAN TYPE OF WORK, THESE WOULD BE DISORDERS THAT ARE OTHERWISE FATAL IF NOT TREATED AND WHERE THERE IS A REAL ETHICAL FOUNDATION TO TAKE A RISK WITH A TREATMENT SUCH AS THIS. AND THE HOPE IS WE LEARN HOW TO DO THIS AND IT BECOMES SAFER OVER TIME AND WE CAN REALLY EXPAND, NOT JUST FOR CRISPR-CAS 9 BUT GONE THERAPY IN GENERAL TO MANY OTHER DISORDERS AND SO IN PHASE 2 WE'RE HOPING TO GET THESE IN THE CLINICS. IT'S A VERY AMBITIOUS PROGRAM. WE TALKED A LITTLE BIT ABOUT OUR THERAPY DEVELOPMENT GROUP, WHICH I MENTIONED IS MADE UP OF VERY SOPHISTICATED PROGRAM OFFICIALS, MANY OF WHICH CAME IN FROM INDUSTRY AND THEY RUN WHAT WE CALL THE BLUE PRINT NEURO THERAPEUTICS NETWORK AND THIS IS ACTUALLY NOT JUST FOR NINDS BUT OUR GROUP ENGAGES ACROSS ALL THE NEUROSCIENCE INSTITUTES SO SOME OF THE THERAPIES MIGHT BE FOR AN EYE DISEASE OR DEMENTIA OR AGING INSTITUTE OR A DISORDER IN THE NATIONAL FOR CHILD HEALTH AND DEVELOPMENT. BUT THEY'RE ALL NEURO-BASED THERAPIES AND THE IP BELONG TO THE GRANTEE SO NIH IS NOT PART, THERE'S NO RISK TO THE INTELLECTUAL PROPERTY WE'RE JUST FUNDING TO DERISK THE THERAPEUTICS OF THE POINT OF THE INDUSTRY INVESTMENTS AND ADVANCES THE NEW DRUGS TOWARDS PATIENTS EFFICIENTLY. AND THERE'S VERY, AS I MENTIONED, THERE'S PROGRAMS FROM THE EXPLORATORY TO THE DRUG AND AND I MENTIONED THESE ARE GENERALLY THESE ARE LINED WITH DISEASE ORGANIZATIONS AND MANY OF THE INDUSTRY FOLKS ARE ALSO ALIGNED WITH INDUSTRY PARTNERS SO THESE ARE REAL EXAMPLES OF THE PARTNERSHIP THAT WE'RE GOING TO TALK ABOUT TODAY AND IN THE BLUE PRINT THERAPEUTICS, THERE ARE A SERIES OF CONTRACTS OUT TO COMPANIES MOST BIO TECHS DO HAVE THAT AND MOST OF THE AK DEEMIA DON'T HAVE THE CHEM ASSIST AND PEOPLE WHO DO TOXICOLOGY AND MANUFACTURING. SO, WE HAVE CONTRACTS OF THE PEOPLE WHO WIN THE AWARDS HAVE ACCESS THOUGH THESE CONTRACTS TO DO THIS WORK AS THEY MOVE TOWARDS THE IND. AND I'M GOING TO END NOW JUST WITH A MENTION ABOUT OUR TRAINING PROGRAMS AND BECAUSE FOR MY VIEW, NINDS, NIH HAS RESOURCES. THERE ARE A HOST OF REALLY GREAT IDEAS AND OPPORTUNITIES. SO, WHAT IS IT THAT REALLY SETS THE FUTURE AND THE PACE OF THE DISCOVERY AND TRAPS LAYING AND THAT IS ACTUALLY PEOPLE. I THINK THAT THE BOTTLENECK IS REALLY THE PEOPLE IN THE PROCESS OF TRANSLATION. THERE ARE SOME GOOD ONES, BUT IT'S NOT THE MOST COMMON CAREER PATH FOR MANY PEOPLE. MANY PEOPLE WHY GREAT SCIENTISTS WHO LATE IN THEIR CAREER DECIDED THEY WANTED TO DEVELOP THEIR IDEAS INTO A THERAPY. THEY'RE STILL NAIVE AT THAT POINT SO WE REALLY NEED TO DO IS TO WORK ON TRAINING PEOPLE IN THIS SPACE AND WE HAVE A TRAINING PROGRAM NOW FOR TRANSLATIONAL RESEARCH AND I THINK IT'S THE DISEASE ORGANIZATIONS THAT CAN REALLY HOOK SOMEONE ON THIS IDEA OF WORKING TOGETHER WITH YOU TO DEVELOP THERAPIES FOR YOUR PATIENTS AND FOR THE PEOPLE THAT YOU REALLY CARE ABOUT AND I THINK YOU ARE MUCH MORE CONVINCING THAN THE GOVERNMENT TO SAY I'M GOING TO WORK ON THIS RARE DISEASE FOR THE REST OF MY CAREER. IT'S SOMETHING THAT NIH WILL MAKE HAPPEN BUT DISEASE ORGANIZATION, YOU CAN MAKE IT HAPPEN. THE NUMBER OF APPLICATIONS HAS BEEN FLAT GOING BACK 22 YEARS AND IF YOU AT THE STAGE OF TRANSLATION AND SO AGAIN, I THINK IT'S A DISEASE ORGANIZATIONS THAT CAN REALLY LIGHT THE FIRE AND FOLKS TO WHO ARE TRAINED TO TAKE CARE OF PATIENTS AND SPEND THEIR CAREER WORKING WITH PATIENTS TO DEVELOP THERAPIES BUT IT'S ONE OF THE BIGGEST BOTTLENECKS WE HAVE. IN TERMS OF HOW THE NINDS PARTNERS, WE ARE ALWAYS LOOKING TO PARTNER. WITH INDUSTRY THAT CAN ADVANCE OUR MISSION WITH DISEASE ORGANIZATIONS AND WE HAVE OUR COUNCIL THAT MAKES ALL THE DECISIONS ON WHAT WE FUND. WE DON'T FUND ANYTHING UNLESS THE COUNCIL GROWS AND THIS HAS PUBLIC MEMBERS AS WELL AS SCIENTISTS AND PHYSICIANS. WE DO STRATEGIC PLANNING WHICH Dr. SHORE LED FOR US BUT RIGHT NOW WE'RE DOING STRATEGIC PLANNING AND ALL THESE DIFFERENT EVENTS COME UP, WE'RE ALWAYS PARTNERING WITH THE DISEASE ORGANIZATIONS AND WE'RE ALSO LOOKING FOR INDUSTRY INPUT BECAUSE WHEN YOU GET TO THE TREATMENTS BECAUSE THAT'S WHAT IS REALLY GOING TO MAKE A DIFFERENCE FOR PATIENTS AND WE HAVE CONFERENCES WHERE THESE ARE ALWAYS INCLUSIVE OF PUBLIC MEMBERS AND THOSE WHO REPRESENT THE DISORDERS THAT WE'RE TRYING TO ATTACK. IT'S A LITTLE BIT ABOUT HOW WE PARTNER AND THE MECHANISMS FOR PARTNERSHIP AND I'M LOOKING FORWARD TO THE REST OF THE DISCUSSIONS WITH REGARDS TO THIS TOPIC AND HOW WE WORK WITH INDUSTRIES AND NIH AND ACADEMIC TO ADVANCE OUR INDIVIDUAL MISSIONS. I WANT TO THANK YOU FOR YOUR ATTENTION AND NOW I'D LIKE TO INTRODUCE OUR KEYNOTE SPEAKER, WHO IS Dr. -- I HAVE MY NOTES HERE. Dr. JULIE GERBERDING THE NEW CHIEF OFFICER FOR THE FOUNDATION OF THE NATIONAL INSTITUTE OF HEALTH SO OTHERWISE KNOWN AS FNIH AND THE FOUNDATION WAS SET UP BY CONGRESS TO MANAGE RESEARCH ALLIANCES WITH PUBLIC AND PRIVATE INSTITUTIONS AND SUPPORT OF THE MISSIONS OF THE NIH AND SO IT IS REALLY A PARTNERSHIP ORGANIZATION AND JULIE COMES WITH EXPERIENCE AND CREDENTIALS AND PASSION FOR THIS TYPE OF PARTNERSHIP. SHE WAS PREVIOUSLY THE EXECUTIVE VOICE PRESIDENT OF THE CHIEF PATIENT OFFICER AT MERK AND SHE JOINED MERK IN 2010 AS A PRESIDENT OF THE THEIR VACCINE PROGRAM AND DIRECTOR OF THE CDC AND SHE LED THE AGENCY BETWEEN 2002 AND 2009 AND SHE'S TRAINED PHYSICIAN. SHE DID HER UNDERGRADUATE MD DEGREES AT CASE WESTERN AND TRAINED IN INTERNAL MEDICINE AND INFECTIOUS DISEASE AT UNIVERSITY OF CALIFORNIA SAN FRANCISCO AND SHE WAS ON THE FACULTY THERE FOR MANY YEARS AND AND ALSO AND UNIVERSITY OF CALIFORNIA BERKELEY AND SHE'S REALLY VERY WELL RESPECTED AND MULTIPLE HONORS AND AWARDS AND WE ARE SO LUCKY TO HAVE JULIE RUNNING THE FOUNDATION FOR NIH AND NINDS HAS HAD SO MANY GREAT RELATIONSHIPS. I'M LOOKING FORWARD TO YOUR COMMENTS. >> THANK YOU, WALTER. I'M REALLY HONORED TO BE THE KEYNOTE OF THIS PARTICULAR PROGRAM AND IT'S REALLY PROBABLY MY FIRST PUBLIC PRESENTATION ON BEHALF OF FNIH AND CONJUNCTION OF THE NIH AND I COULDN'T THINK OF A BETTER AUDIENCE FOR IT SO IT'S TEAR TO MY HEART TO REALLY BE ENGAGING WITH PATIENTS ORGANIZATIONS, CARETAKER ORGANIZATIONS, ET CETERA, BUT PARTICULARLY WITH A PHOTOGRAPHIC HE IS I HAVE TO ADMIT TO YOU I WASN'T REALLY FAMILIAR WITH THE FNIH EVEN WHEN I WAS IN GOVERNMENT AND NOW THAT AND THE ROLE THIS ORGANIZATION PLAYS AND TRYING TO BE A INTERFACE NOT JUT BETWEEN THE NIH AND INDUSTRY THAT YOU'VE BEEN HEARING ABOUT, BUT WITH OTHER ORGANIZATIONS INCLUDING A NUMBER OF PATIENT AND NON PROFITS AND THE GATES FOUNDATION, OTHER FOUNDATIONS, ET CETERA, AND AS WELL AS JUST INDIVIDUAL STAKEHOLDERS AND SOMETIMES PHILANTHROPISTS WHO ARE INTERESTED IN OUR WORK OUR MISSION IS VERY BROAD. IT'S REALLY TO SUPPORT THE MISSION OF THE NIH SO THAT IS A VERY BROAD INTERPRETATION OF OUR CONGRESSIONAL AUTHORITY BUT WE DO TEND TO FOCUS ON THREE MAIN CHANNELS OF ENGAGEMENT. ONE IS SUPPORT FOR SCIENCE AND WE'LL SAY A BIT MORE ABOUT THAT AND THE SECOND FOR SUPPORT FOR SCIENTISTS AND WE DO THAT BY SUPPORTING LABS AT THE NIH, SCIENTIFIC MEETINGS, ET CETERA, ET CETERA, TO TRY TO NOT ONLY BE A FORCE OF THE ACTUAL RESEARCH, BUT SUPPORTING THE PEOPLE AND THE PIPELINE OF PEOPLE NECESSARY TO CONDUCT AND PARTICIPATE IN THAT RESEARCH IN AND OUT OF THE NIH. IN OUR THIRD DOMAIN, WE HAVE A ROLE IN THE THOUGHT LEADERSHIP. FOR ME COMING INTO THIS ROLE, WITH MY BACKGROUND AS THE CHIEF PATIENT OFFICER AT MERK, I REALLY WANT TO BRING IT'S EXCITING FOR ME TO REALLY INITIATE THIS CONVERSATION AND I'M SHARING MY E-MAIL AT THE END OF IT BUT I DO HOPE TO BE ABLE TO MEET WOMAN OF YOU FOR SEVERAL REASONS OF HOW WE MIGHT BE HELPFUL IN THE FUTURE AND I'M INTERESTED UNTIL THE PATIENT ENGAGEMENT AND THE END-TO-END CLINICAL RESEARCH PROCESS. SOMETHING I'VE LEARNED ABOUT FROM THE INDUSTRY SIDE BUT I WOULD LIKE TO LEARN MORE ABOUT IT FROM THE EXTERNAL PERSPECTIVE. I'VE MEET YOU AT THE BIO ANNUAL PATIENT ENGAGEMENT SUMMIT, AND I HOPE I WILL BE INTERACTING WITH SOME OF YOU IN MY ROLE AS A MEMBER OF THE NATIONAL HEALTH COUNCIL BOARD SO I LOCK FORWARD TO LINING, LEARNING AND SEE WHAT FNIH CAN DO TO BE INCLUSIVE OF THE POPULATIONS WE'RE ALL TRYING TO SERVE. I DON'T KNOW WHAT WAS IN THE MINDS OF CONGRESS WHEN THEY CREATED THE FNIH BUT PART OF THAT EFFORT WAS AN ATTEMPT TO BRING AN INTERFACE IN THE SENSE MIDDLE WEAR SO THAT THE GOVERNMENT AND INDUCE TREMENDOUS INDUSTRY CANCOLLABORATION. IT'S NOT POSSIBLE FOR INDUSTRY TO GIVE MONEY TO THE GOVERNMENT OR KIND OF OBVIOUS CONFLICT OF INTEREST REASONS. SO IT'S IMPORTANT TO HAVE A ADJUDICATED, ETHIC-BOUND, MECHANISM SO THAT APPROPRIATE COLLABORATIONS CAN BE CRAFTED AND ALSO THEY CAN BE CONDUCTED IN THE RIGHT WAY. AND I WON'T GO INTO SOME OF THE COMPETENCIES OF THE FNIH BUT I'LL HIGHLIGHT A COUPLE THAT STAND OUT TO ME. ONE IS PROJECT MANAGEMENT AND I'LL USE ACTIVE AS AN EXAMPLE OF THAT. THE MECHANISMS THAT ARE BROUGHT ALL KINDS OF PARTNERS TOGETHER TO ACCELERATE THE PROGRESS OF ANTI VIRALS AND VACCINES AND DIAGNOSTICS DURING COVID. THESE PROJECT MANAGEMENT, KIND OF IN THE BACKGROUND BUT THE BEST PROJECT MANAGEMENT I'VE SEEN IN ANY SETTING, INCLUDING MY ACADEMIC BACKGROUND AND GOVERNMENT BACKGROUND AND INDUCE TREMENDOUS BACKGROUND AND THIS IS JUST A BRILLIANT SET OF SCIENTISTS AND THEIR AND CREATE A WHOLE GREATER THAN THE SOME OF THE PARTS. ONE OF THE STRENGTHS REALLY THE INTERFACE WITH THE FDA AND THE ABILITY TO WHICH IS REALLY IMPORTANT TO INDUSTRY WHEN THEY'RE STRIKING OUT TO HAVE CLARITY ON WHAT THE FDA WILL CONSIDER AS THE KIND OF END POINT OR THE DAY THAT THAT WOULD HELP FACILITATE DECISIONS AROUND DRUG APPROVAL AND LABELING. SO ON THE NEXT SLIDE, JUST TALKING A LITTLE BIT ABOUT THE BIG NUMBERS, TODAY, THE FOUNDATIONS JUST OVER 25-YEARS-OLD, AND IT HAS RAISED ABOUT 1.2 BILLION IT'S MORE IF WE COUNT SOME OF THE ACTIVE RESOURCES AND WE'VE RAISED A SUBSTANTIAL INVESTMENTS AND WE'RE PROUD OF THE FACT THAT THE VAST MAJORITY OF THOSE DOLLAR REALLY DO GO DIRECTLY TO SUPPORT PROGRAMS AND ABOUT 600 OF THEM TO BE EXACT. AND THE ACTIVE RESEARCH PROJECTS WE HAVE GOING ON RIGHT NOW ARE 117 OF THEM, WHICH I THINK IS A TRIBUTE TO THE SMALL FOUNDATION AND WE HAVE PEOPLE WHO ARE VERY ASSERTIVELY INVOLVED IN SUPPORTING AND I'VE DEFINED HOW WE DECIDE WHAT PRIVATE-PUBLIC PARTNERSHIP MAKES SENSE TO US AND WE HAVE A GOVERNANCE STRUCTURE THAT HELPS ADJUDICATE THESE DECISIONS AND WE PUT A GREAT DEAL OF EFFORTS ON POLICY AND COMPLIANCE SO WE ARE NOT INA SITUATION WHERE WE VIOLATE THE PRINCIPLES OF ETHICAL CONDUCT OR CONFLICT OF INTEREST AND WE REALLY OF COURSE ENDORSE AND SORT POLICIES OF THE NIH AS WELL IN THAT REGARD. OUR FUNDRAISING AND RELATIONSHIP MANAGEMENT IS A VERY IMPORTANT PART OF OUR ORGANIZATIONS AND WE ARE PERMIT TODAY SOLICIT FROM FOUNDATION AND FROM TRANSFORMATIONAL DONORS AND FROM GRATEFUL PATIENTS AND TIMES WE'RE NOT ALLOWED TO SOLICIT FROM THEM DIRECTLY OF COURSE BUT WE DO HAVE OPPORTUNITIES TO RESPOND TO THEIR INTERESTS AND SOME OF THAT INTEREST HAS LED TO FUNDING OF CERTAIN HAB TORIES OR CERTAIN INVESTIGATORS OR FELLOWSHIPS AT THE NIH SO THAT'S PART OF OUR SUPPORTING SCIENTISTS COMPONENT OF WHAT WE DO AND I MOVE TO -- I WANT TO MAKE A POINT HERE ABOUT THE LAST ITEM ON THE SLIDE. OUR MECHANISMS OF ENGAGEMENT IS IN THE PRE COMPETITIVE SPACE. SO WE DON'T BRING TWO PHARMA COMPANIES TOGETHER AND SHARE STRAYED SECRETS OR CREATE A SITUATION THAT WOULD BE PERCEIVED AS ANTI COMPETITIVE. WE DO OUR WRACK IN THE MORE SCIENTIFIC PRE COMPETITIVE ENVIRONMENT AND IT ALLOWS PEOPLE TO BE OPEN WITH THEIR INFORMATION AND THEIR DATA AND THE MECHANISMS FOR HANDLING INTELLECTUAL PROPERTY ARE WORKED OUT BEFORE ANY PROJECTS BEGIN AND I THINK THIS THIS IS PLACE. WE HAVE A GOOD TRACK RECORD MAN NOT AGING WITH THE ETHICAL FRAMEWORK AND ON THE NEXT SLIDE, I HAVE HOW THIS PLACE OUT IN REAL-TIME AND ANYONE CAN COME UP WITH A REASON TO HAVE A COLLABORATIVE RESEARCH PROGRAM INCLUDING GOVERNMENT INDUSTRY AND AK A DEM I GUESS AND ESPECIALLY HIGHLIGHT PATIENTS GROUPS AND YES SOME OF THE PATIENT ORGANIZATIONS THAT ARE PARTICULARLY INTERESTED IN CLINICAL RESEARCH ARE A GREAT SOURCE OF IDEAS AND CONNECTIONS THAT HELP BUILD THE CRITICAL MASS OF PEOPLE COMING TOGETHER TO FORMULATE A SPECIFIC IDEA AND THE NIH PROCESS WHERE INSTITUTES ARE THE NIH DIRECTOR SEES THE OPPORTUNITY FOR CROSS CUTTING PROGRAMS OR THE AT ANY TIME A COLLABORATION CAN CREATE AND THERE'S AN INTERNAL PROCESS AT NIH TO MAKE SURE THAT THE PROPOSAL MEETS NIH CRITERIA AND ULTIMATELY THEY END UP AT THE FNIH AND WE HAVE A ROBUST PANEL OF SCIENTIST AND OUR BOARD OF DIRECTORS WHO INCLUDE WORLD-CLASS SCIENTISTS TO REVIEW THE PROPOSALS AND MAKE DECISIONS ABOUT WHETHER IT'S A GO OR A NO-GO PROGRAM. IF IT'S A GO PROGRAM, WE CREATE THE GOVERNANCE, RESTORE THE PARTNERSHIP AS QUICKLY AS WE CAN, WHICH SOMETIMES ISN'T QUICK. WE RAISE THE MONEY IN ORDER TO INITIATE. ONE OF OUR PRINCIPLES BECAUSE WE ARE USING OTHER PEOPLE'S MONEY TO FUND THIS RESEARCH WE GET US TO THE FIRST MILESTONE SO WE HAVE BUT ULTIMATELY THIS PROCESS RESULTS IN A PROGRAM THAT'S BEEN VETTED AND TO HIT THE GROUND RUNNING AND TO MOVE THINGS FORWARD NOT ALL PARTNERSHIPS MAKE IT. FIRST AND FOREMOST WE MAKE SURE WITH WHAT WE'RE DEALING WITH IS SOMETHING THIS ADDRESSES IMPORTANT UNMET NEEDS AND SOMETHING THAT HAS A HYPO TEN SHALL FOR TRULY IMPACTING PATIENTS AND MAKING A DIFFERENCE FOR THEM AND THAT IS A REALLY IMPORTANT AREA WHERE THE CONCEPT OF EQUITY COMES IN AND OUR WEIGHTED PROBLEMS AND NOT NECESSARILY PROBLEMS THAT ADDRESS ALL COMMUNITIES AND WE ARE GOING TO INCREASE OUR EMPHASIS ON PROGRAMS THAT ADDRESS HEALTH EQUITIES AND MAKE SURE IT'S ONE OF THE SPECIFIC CRITERIA WHAT WE CONSIDER AS AN OPPORTUNITY TO AND THE OTHER CRITERIA ARE A PRETTY OBVIOUS HERE AND LOCK AT NUMBER FOUR BECAUSE SOMETIMES WE HAVE A FANTASTIC IDEA AND WE JUST CAN'T GET IT FUNDED AND THEY'RE JUST ISN'T ENOUGH INCENTIVE FROM THE INDUSTRY PARTNERS AND NIH HAS COMPETING PRIORITIES AND THIS IS WHERE WE'RE GOING TO GO FORWARD AND HOW WE CAN RAISE MONEY IN OTHER DOMAINS THAT WOULD HELP GIVE US AT LEAST THE START UP RESOURCES TO TEST IDEAS OR PILOT IDEAS AND TO MAKE SURE THAT WE HAVE PROOF OF CONCEPT BEFORE WE LULAUNCH INTO A MORE AGGRESSIVE FUNDRAISING OPPORTUNITY. THIS SHOWS YOU THAT, AS RESOURCES BECOME AVAILABLE, SOME OF THAT MONEY GOES TO THE NIH INSTITUTES WHO ADDED TO THEIR OTHER PROGRAMMATIC MONEY AND MOVE IT OUT TO THE PEOPLE THAT ARE GETTING FUNDING THROUGH THE MANY MECHANISMS THAT WALTER JUST SHOWED US AND THAT HAPPENS THROUGH THE USUALLY CUSTOMARY NIH MECHANISMS BUT FNIH ALSO PROVIDES GRANTS TO SCIENTISTS AND WE HAVE CONTRACTS WITH VARIOUS RESEARCH ORGANIZATIONS AND WE SUPPORT DATA COORDINATING CENTERS AND OTHER KINDS OF INVESTIGATORS TO PARTICIPATE IN THE COLLABORATION. ALL OF OUR WORK IS DONE IN AN OPEN PLATFORM MEANING THE DATA ARE SHARED AS PART OF THE ADVANTAGE TO THIS BEING PRE COMPETITIVE THAT WE HAVE THE OPPORTUNITY TO MAKE WHAT WE'RE DOING VISIBLE SO OTHERS CAN LEARN FROM IT AND AND I HAVE A WONDERFUL EXPERIENCE AND RELATED TO THE CONSEQUENCES OF REPEATED EXPOSURES TO CORONAVIRUS VACCINES AND TO TALK AND SHARE THIS ELEGANT WORK. IT WAS JUST REFRESHING TO ME TO PARTICIPATE OPEN EXCHANGE AND SCIENTIFIC COMMUNICATION WASSEN LIGHT EPING AND I THINK THAT VERY INDICATIVE OF THE KIND ATMOSPHERE AND WE TRY TO AND THE NEXT SLIDE, JUST TO GIVE YOU A LITTLE BIT. RIGHT NOW WE HAVE $445 MILLION IN PROGRAMS IN PROGRESS. SO THAT IS A LOT OF PROJECTS THAT ARE GOING ON AND THESE ARE BIG PROJECTS. YOU'VE HEARD WALTER MENTION A COUPLE OF THEM. THE FOUR BIG CATEGORIES FOR US RIGHT NOW ARE THE SO-CALLED AMP PROGRAMS. AND KIND OF AN ACCELERATED OPPORTUNITY TO DEFINE NEW TARGETS OR NEW APPROACHES TAG EIGHT INDICATION AND MEDICATIONS THAT WOULD SELECT THAT TARGET AND THOSE PROGRAMS, THERE ARE EIGHT OF THEM RIGHT NOW THEY ARE LONG STANDING AND THEY HAVE A VERY WONDERFUL TRACK RECORD OF CONTRIBUTION AND THE OTHER LONGSTANDING PROGRAM IS THE BIOMARKERS PROGRAM AND THIS IS A PROGRAM THAT LOOKS FOR THE PROGNOSTIC BIOMARKERS THAT HELP DETERMINE WHO IS AT RISK FOR DISEASE PROGRESSION, FOR EXAMPLE, SOMETHING LIKE NON ALCOHOLIC LIVER DISEASE WHERE IT'S IMPORTANT TO KNOW WHO IS MOST LIKELY TO PROGRESS TO CIRRHOSIS SO THAT THOSE ARE THE PEOPLE THAT YOU WOULD WANT TO FOCUS INTERVENTION ON AND NOT FORCE OTHER PEOPLE TO HAVE A BUY ON SEE OR UNNECESSARY INVASIVE PROCEDURES BECAUSE THEY'RE NOT GOING TO BE AT RISK FOR PROGRESSION. IF YOU WERE STUDYING A PROBLEM WITH A LONG LIFELINE, THE STUDIES WOULD HAVE TO GO ON FOREVER TO SHOW HARD DEMONSTRATIONS OF DECREASED MORTALITY OR DECREASED LONG-TERM MORBIDITY AND IF YOU HAVE A SURROGATE BIOMARKER THAT IS HIGHLY CORRELATED WITH THOSE END POINTS AND YOU HAVE THE RIGHT DATA ON THE RIGHT STANDARDIZATION TO USING THAT END POINT YOU CAN CONDUCT CLINICAL TRIALS AND GET DRUG APPROVAL BASED ON THAT SURROGATE MARKER OF SUCCESS AND THAT'S SOMETHING THAT IS SCIENTIFICALLY SIMPLE MINDED BUT REALLY HARD TO DO AND -- WHICH ARE FOUR CATEGORIES OF PROGRAMS THAT ARE PRIMARILY FUNDED THROUGH THE GATES FOUNDATION AND PARTNERS THAT ARE GLOBAL PORTFOLIO AND I JUST WANT TO MAKE THE POINT THAT WE'RE NOT TOTALLY FOCUSED ON THE DEVELOPED WORLD AND WE'RE THINKING ABOUT HOW WE CAN BRING THE PROCESS NO A EQUITABLE MANNER ACROSS ALL OF OUR DISEASE CATEGORIES AS WE EVOLVED THE FNIH. NEXT SLIDE, PLEASE. AND I THINK WE CAN JUST SKIP THOSE AND I'VE MENTIONED THESE BEFORE WELL MAYBE JUST QUICKLY TO SHOW YOU THE SCALE OF INVESTMENTS IN THESE VARIOUS PROJECTS SO FOR EXAMPLE, I MENTIONED THE BIOMARKERS CONSORTIUM AND WE HAVE THE INVESTED IN THAT AND IT PAYS FOR IMAGING STUDIES THAT MIGHT NOT BE DONE IN THE COURSE OF A CLINICAL TRIAL OR A CLINICAL INVESTIGATION TO DETERMINE IF SOMETHING IS AN EFFECTIVE BIOMARKER OR NOT SO, THREE MOVEMENTS ABOUT WHAT IS THE STANDARD DATA SET AND HOW WE CAN POOL THIS TOGETHER TO ACHIEVE THE KIND OF STATISTICAL POWER THAT WOULD HE CAN SELL RATE OUR DECISION-MAKING. NEXT SLIDE, PLEASE. I'M GOING TO END WITH A LITTLE BIT OF IN-HOUSE PERSPECTIVE BECAUSE I AM DEFINITELY STILL IN THE LEARNING CURVE AND I'M NEW. WE WE HAVE THE SAME CHALLENGES THAT BIG ORGANIZATIONS HAVE AND AND I'M NOT SURE MANY PATIENTS INCLUSION AND NOT JUST IN TERMS OF WHO WE ARE AND IN THE WORK WE DO SO I'M PLEASED SO SAY THE BIOMARKERS CONSORTIUM SPENT A EXECUTIVE COUNTY MEETING TO MAKE SURE OUR GOVERNANCE MODELS ARE EQUITABLE AND HOW DO WE INCLUDE MORE PATIENT ORGANIZATIONS IN THAT PROCESS BECAUSE OBVIOUSLY IN THE SENSE THERE'S A PAY TO PLAY PARTICIPATION REQUIREMENT AND PEOPLE WHO SMALL ORGANIZATIONS REALLY HAVE A STRUGGLE WITH THAT AND YET THEIR PERSPECTIVES AND KNOWLEDGE IS SO RELEVANT TO HOW WE DESIGN THE PROGRAMS AND HOW WE SUFFICE TO SAY WE ARE A SMALL ENOUGH ORGANIZATION THAT WE CAN AFFORD TO TEST PEOPLE SO WE ARE REQUIRING PEOPLE TO BE TESTED EACH WEEK THEY COME TO THE OFFICE AND LIKE EVERYONE, THIS IS BEEN A REALLY DIFFICULT TIME FOR RECRUITING AND RETAINING THE PROFESSIONALS WE NEED OF HIGH PREPORTION OF OUR TEAM HAVE PHDs BUT WE HAVE OTHER PROGRAM SUPPORT, PROFESSIONALS THAT WE CAN'T LIVE WITHOUT AND SO WE ARE BROADENING OUR RECRUITMENT TO A NATIONAL BASIS AND WE ARE LEARNING LIKE EVERYONE ELSE TO WORK REMOTELY AND SO ON AND SO FOURTH SO THIS IS A LITTLE BIT OF A PLUG HERE BECAUSE IF YOU KNOW SOMEONE WHO IS REALLY INTERESTED TO THIS KIND OF COLLABORATIVE RESEARCH, ASK THEM TO CHECK OUT OUR WEBSITE BECAUSE WE ARE RECRUITING AND WE LOVE TO THE THE BEST AND BRIGHTEST TALENT AND THE FINAL SLIDE, THIS IS IMPORTANT. I WANTED TO MENTION THAT I SAID SCIENTISTS AND SCIENCE BUT WE ALSO HAVE THIS LONG STANDING COMMITMENT TO SUPPORTING TRACTOR-TRAILER CARE OF OUR PATIENTS AND ONE OF THE WAYS THAT WE'VE DONE THAT IS THROUGH THE STAFF OR FOUNDATION IS TO SUPPORT THE FAMILY LODGE ON THE NIH CAMPUS WHICH IS LIKE THE CHILDREN'S AND IT'S AN OPPORTUNITY FOR THE FAMILIES OF PEOPLE WHO ARE RECEIVING CARE AT THE NIH CLINICAL CENTERS TO HAVE A COMFORTABLE AND SUPPORTIVE ENVIRONMENT DURING THEIR OTHERWISE DIFFICULT CHALLENGE OF RECEIVING CARE FOR COMPLICATED DISEASES. WE ALSO ANNOUNCED THE ESTATE GIFT WHICH MENTIONED IS ONE OF THE WAYS THAT WE SUPPORT SCIENTISTS BY SUPPORTING Dr. BONOMAN RESEARCH ON MUSCULAR DYSTROPHY AND WE WILL BE WORKING TO INCREASE THIS GIVING GOING FORWARD. AND FINALLY ON MY LAST SLIDE, I JUST MENTIONED MY OWN PRIORITIES AS ANNAN BOARDING PERSON AND PART OF THAT IS LISTENING AND LEARNING AND I HAVE REACHED OUT TO VARIOUS PATIENT ORGANIZATIONS TO HAVE THESE KINDS OF CONVERSATIONS. I'M INTERESTED TOOULD BE HELPFUR CURRENTLY BEING HELP AND WHAT SHOULD WE BE DOING BETTER AND HOW CAN WE PARTICIPATE IN PATIENT ORGANIZATIONS NOT JUST IN THE DC AREA BUT OBVIOUSLY NATIONALLY AND HOPEFULLY AT SOME POINT ORGANIZATIONALLY AND WE ARE REFRESHING OUR STRATEGY AND I'VE MENTIONED THE WORKPLACE AND CULTURE ISSUES THAT ARE COMMON TO US IN THE ORGANIZATIONAL WORLD AND OUR TEAMS ARE EVERYTHING AND IF WE DON'T DO THAT RIGHT WE CAN'T BE SUCCESSFUL AND IN TERMS OF SCIENTIFIC PARTNERSHIP INVESTMENTS OBVIOUSLY WE TEND TO CONTINUE THE AMPS AND THE BIOMARKERS CO CONSORTIUM AS WELS OUR CONTRIBUTION IN THE AREA OF DIAGNOSTICS AND VACCINES AND WE'LL CONTINUE AND IT'S NOT IN THE REAR VIEW MIRROR FOR US SO WE ARE VERY MUCH CONTINUING TO BE INVOLVED. WE JUST HAD THE EXECUTIVE COMMITTEE MEETING THIS MORNING WITH THE DIRECTOR OF THE NIH AND THE COR TEAMS AND WE ARE STILL WORKING ON THIS PANDEMIC DILIGENTLY AND FINALLY, I THINK IN THIS ERA WHERE THE TRUST AND SCIENCE IS AT AN ALL-TIME LOW, IT'S REALLY IMPORTANT THAT ALL OF US, IN THE HEALTH COMMUNITY STANDS STRONG ON THE PRINCIPLES OF SCIENCE AND THE IMPORTANCE OF SCIENCE AND CLINICAL RESEARCH AND THAT WE KIND OF WORK TOGETHER TO FIND WAYS TO EMPHASIZE HOW IMPORTANT ALL OF THIS IS TO PEOPLE AND TO PATIENTS EVERYWHERE. SO I'LL STOP THERE AND ON MY LAST SLIDE I HAVE MY E-MAIL ADDRESS SO IF YOU HAVE SOME IDEAS, COMMENTS CRITICISM OR COMPLIMENTS, GET IN TOUCH WITH ME AND I'LL DO MY BEST TO BE RESPONSIVE. THANK YOU. >> THANK YOU SO MUCH Dr. GERBERDING. UNFORTUNATELY, Dr. KOROSHETZ HAD TO RUN FOR ANOTHER MEETING THAT GOTTA JUSTED BUT HI, NICE TO MEET YOU, I'M Dr. SAMANTHA WHITE OUR RUN OUR BRANCH HERE AND I REALLY ENJOYED YOUR TALK. I MEAN IT REALLY RESINATES WITH ME IN THE ENGAGEMENT AND WITH THE FORUM IN OUR THEME OF PROGRESS THROUGH PARTNERSHIPS, WHICH YOU KNOW VERY MUCH ABOUT. SO, I WANT TO ENCOURAGE EVERYONE TO POST QUESTIONS IN THE CHAT SO THAT I CAN HELP Dr. GERBERDING GO THROUGH A BRIEF Q&A PERIOD. I SEE ONE WE GOT. WHAT ELEMENT MAKES YOU THE MOST EXCITED ABOUT WORK AT THE FNIH IN THE NEXT SIX TO 12 MONTHS? >> WOW. WELL, FIRST, LET ME JUST SAY, THE REASON I AM GOING TO HAVE A HARD TIME ANSWERING THIS IT'S JUST LIKE BEING IN A CANDY STORE OF SCIENCE. OF COURSE I'VE BEEN IN THE SCIENTIFIC ENVIRONMENT MY ENTIRE CAREER BUT THIS IS CLOSE TO THE SCIENTISTS AND THE DISCOVER SO TO SPEAK SO IT'S JUST THRILLING TO BE BACK IN THAT AND BE EXPERIENCING THAT. I THINK IN TERMS OF WHAT CAN WE ACCOMPLISH IN THE NEXT SIX MONTHS, I WOULD HAVE TO SAY THAT THERE ARE FRONTIERS ON THE BIOMARKERS' HORIZON THAT ARE EXCITING AND I PROBABLY DON'T WANT TO SAY SPECIFICALLY WHICH ONCE I'M TALKING ABOUT BECAUSE IT MIGHT NOT BE READY FOR PRIME TIME BUT THERE ARE IMPORTANT ADVANCES IN THOSE SPACES AND I THINK OF COURSE, WE HAVE THE ON GOING WORK AND WHETHER OR NOT WE'LL GET TO A POINT WHERE WE HAVE THEIR PIECE FOR LONG COVID SO STAY TUNED. >> STAY TUNED. WE WILL. SO, ANOTHER QUESTION IS COMING IN FROM ONE OF OUR EXECUTIVE COMMITTEE MEETING MEMBERS AND SHE'S THANKS YOU FOR THE PRESENTATION AND DO YOU SEE THE FNIH HELPING TO ENGAGE YOUNG MINDS IN SCIENCE FOR RESEARCH? >> YOU KNOW, BECAUSE THE PIPELINE OF SCIENTISTS IS SO IMPORTANT TO THE NIH MISSION, IS CLEARLY IN SCOPE FOR US AND WE DO SOME SMALL THINGS IN THAT REGARD BUT WE DON'T HAVE A LARGE PROGRAM THERE AND THIS IS ONE OF THOSE AREAS WHERE AS WE WORK ON OUR STRATEGIES, AND THINK ABOUT HOW WE GROW THE FOUNDATION, FINDING PEOPLE WHO ARE SPECIFICALLY INTERESTED IN THAT AND WANTING TO SUPPORT STEM EDUCATION OR JUST BASIC LIT RA SHE IN SCIENCE I THINK THAT'S A REALLY IMPORTANT OPPORTUNITY. ONE OF MY FANTASIES IS TO PERHAPS TRY TO BE PART OF A CONVENING OF A COALITION 6 OF PEOPLE WHO ARE AS INTERESTED IN THE TRUST ISSUES AS I AM AND THINK ABOUT HOW KIDS CAN BE PART OF THAT CONVERSATION BECAUSE IF WE CAN BRING PEOPLE UP IN A MANNER FOR THEIR HEALTH LITERACY FOR THAT MATTER WE'LL HAVE A NEW GENERATION THAT'S BETTER EQUIPPED TO PARTICIPATE IN THESE BROADER SOCIETAL CHALLENGES THAT WE FACE. >> YEAH, ABSOLUTELY. SO ANOTHER QUESTION THAT'S COME IN FROM LOUIS MAZWAY AND A POLL GOES IF I MISPRONOUNCE ANYONE'S NAME. HE IS LOOKING FOR CLARIFICATION. WHY WOULD SOMEONE GIFT TO FNIH AS OPPOSED TO A PARTICULAR HOSPITAL OR HOSPITAL SYSTEM OR PARTICULAR NON PROFITS. CAN YOU HELP THEM UNDERSTAND KIND OF WHERE FNIH HANGS OUT IN THE LANDSCAPE? >> YEAH, I THINK OUR FOCUS IS ON SCIENCE AND WHERE THAT HAPPENS AND NOT EVERY SCIENTIFIC PROBLEM BENEFITS FROM A COLLABORATION OF MULTI SECTOR COLLABORATION BUT SOME OF THE HARD PROBLEMS DO AND I THINK PEOPLE WHO SOPHISTICATED DONORS SO TO SPEAK, UNDERSTAND THE PROCESS OF SCIENCE IS ULTIMATELY AT ITS CORE A COLLABORATIVE PROCESS. NO ONE MAKES PROGRESS BY THEMSELVES AND SO BY FACILITATING AND BROKERING AN ORGANIZATION THAT HAS A PRETTY GOOD TRACK RECORD OF MOVING THINGS FROM IDEAS THROUGH THE WHOLE TRANSLATIONAL PROCESS TO REGULATORY APPROVAL, IS THE WAY THAT ULTIMATELY MAKE SURE THAT YOUR INVESTMENTS IS GOING TO HAVE A HIGH VALUE AT THE END OF THE DAY AND I THINK THAT'S ONE OF THE ASPECTS OF OUR ENGAGEMENT THAT WE NEED TO MAKE MORE VISIBLE, THIS IS A HIGH POS AND WE HAVE A REALLY GOOD TRACK RECORDS OF MOVING THINGS THROUGH THE PIPELINE. >> YEAH, ABSOLUTELY. SO ANOTHER QUESTION HAS COME IN HOW DO YOU SEE TECHNOLOGIES I OBJECT INNOVATION AND IN INITIATIVES AND FOCUS AND DIGITAL BIOMARKERS. >> WELL, ABSOLUTELY. THIS IS A STRATEGIC QUESTION THAT MY TEAM IS ENGAGING ON AND SO THAT IS A FARE OF INNOVATION THAT IS A NO-BRAINER RIGHT NOW SO WE'LL BE THINKING ABOUT WHERE THAT MIGHT BE USEFUL AND EVEN IN MENTAL HEALTH DISORDERS AND NEWER LOGIC DISORDERS THE DIGITAL MONITORING IS PATIENT FRIENDLY AND PROVIDES A UNIQUE KIND OF INFORMATION THAT ULTIMATELY SPEAKS TO PATIENT REPORTED OUTCOMES AS MUCH AS THE BIOLOGICAL INDICATORS OF SUCCESS SO I THINK IT'S TIME HAS COME. >> YES, ABSOLUTELY. SO ANOTHER QUESTION FROM SARAH, I'M GOING TO KIND OF PIVOT A LITTLE BIT IN TERMS OF HOW THIS IS FRAMED AND YOU KNOW, AS YOU ARE AWARE ABOUT 10 INSTITUTE AND HAVE AT NIH THAT A NEUROSCIENCE FOCUS NINDS IS ONE LEADER AND WEST NATIONAL INSTITUTE OF MENTAL HEALTH OR THE NATIONAL EYE INSTITUTE AND SO ON AND SO CAN YOU HELP US FIGURE OUT HOW IT WORKS WITH INDIVIDUAL NEUROSCIENCE AND INITIATIVES ACROSS NIH BUT AS WELL A INITIATIVE LIKE FOR EXAMPLE A CROSS INSTITUTE INITIATIVE IN NEUROSCIENCE IS THE BRAIN INITIATIVE FOR ADVANCING NEURO TECHNOLOGIES. >> YEAH, THIS IS PART OF I THINK, WHERE THE CROSS DISCIPLINARY APPROACH BECOMES REALLY IMPORTANT AND OFTEN, THE AREAS OF COLLABORATION ARE DRIVEN BY THE NIH DIRECTOR OR A SUBSET OF INSTITUTES THAT SEE THE NEED BUT DO ALWAYS HAVE THE MECHANISMS -- DON'T ALWAYS HAVE THE MECHANISMS TO CREATE A COLLABORATION IF YOU THINK ABOUT PARTICIPATING IN EVERYBODY THINKS WELL GEE, MAYBE IT WOULD BE GREAT TO HAVE THESE INDUSTRY PARTNERS COME INTO PLAY. YOU CAN'T HAVE EACH INSTITUTE REACH OUT TO EACH PARTNER TO THE COORDINATION OF ALL THAT FIRST AND FOR MOST HAPPENS WITHIN THE NIH BEFORE WE SEE IT AND THEN THE GOVERNANCE PROCESS REALLY PUTS AT THE TABLE THE LEAD INSTITUTE OR THE LEAD SCIENTISTS TO BE THE KIND OF PRINCIPLE OF THE PROCESS BUT WE WOULD MAKE SURE THAT THE RELEVANT INSTITUTES ARE INCLUDED ON THE STEERING COMMITTEES AND PARTICIPATING IN THE ACTUAL DESIGN AND CONDUCT OF THE THE SCIENCE. THAT'S WHAT I THINK IS REALLY THE AMAZING THING ABOUT THIS. COLLABORATIONS HAVE A HIGH TRANSACTION AT CLASS BUT THEY PLAN HORIZONTAL LOW AND THEN EXECUTE VERTICALLY AND I THINK THAT IS THE SECRET TO THIS KIND OF PROJECT MANAGEMENT. ABSOLUTELY. >> I'M GOING TO ASK YOU A QUESTION THAT I'M INTERESTED IN AND ALSO AS I DO, I'M GOING TO PUT THE FNIH WEBSITE INTO THE CHAT. IT'S VERY SIMPLE. ONE OF THE MY QUESTIONS WAS, I REALLY APPRECIATED THAT FNIH IS VERY EXPLICIT OF PUBLIC-PRIVATE PARTNERSHIP CRITERIA SO I'M CURIOUS FOR THE DIVERSE ATTENDEES OF THE FORUM WHO MIGHT BE ACTUAL LEADERSHIP IN DIFFERENT SIZE NON-PROFIT ORGANIZATIONS AND THEY MIGHT BE MEMBERS OF THE ORGANIZATION AND MIGHT BE THE PATIENTS AND THEIR CAREGIVERS WHO ARE SUPPORTED BY THE ORGANIZATION AND LOOKING TO THEM. WHAT ARE THE STEPS THAT THEY COULD TAKE, IF THEY'RE TRYING TO UNDERSTAND WHERE THEY FALL IN KIND OF YOUR PIPE LYNN AT FNIH? IS THERE A FLOW CHART ON THE WELCOME BACK SIGHT OR A PERSON THEY SHOULD E-MAIL? YOU DON'T WANT ALL OF THESE E-MAILS GOING TO BE DIRECTLY BUT REVIEWING THE CRITERIA, IF THERE ARE GROUPS VERSUS INDIVIDUALS WHO WANT TO TRY TO ENGAGE IN THESE PARTNERSHIPS, WHAT WOULD YOU RECOMMEND THEY DO? >> SO, RIGHT NOW, THE FIRST ANSWER IS I DON'T KNOW. SO THAT IS AN HONEST ANSWER AND I'M STILL FIGURING THIS OUT BUT I WILL SAY THAT ONE OF THE FIRST THINGS I DID WAS TO CREATE A CONTRACT FOR A PERSON THAT I WORKED WITH AT MERC WHO LED OUR PATIENT ENGAGEMENT ORGANIZATION BUT JANICE RETIRED BUT SHE WILL CONDUCT AN ASSESSMENT. FIRST OF ALL, WHAT ARE WE DOING RIGHT NOW WITH PATIENT ORGANIZATION THIS IS WHERE ARE THE PERSPECTIVES AND INSIGHTS FROM PATIENTS COMING NO THE PLAY AND WHERE COULD OR THEY SHOULD AND WHAT IS THE BEST PRACTICE AND WHAT ARE WE MISSING? JANUARY WILL MISS OUT TO ORGANIZATIONS TO GET A POINT OF VIEW SO WE'LL HAVE CONVERSATIONS AND INTERVIEWS WITH THOSE FOLKS AND COMING OUT OF THAT WE'LL PROVIDE CLARITY AROUND THIS AND TRUST ME IT'S AN INVITATION AND I WANT THIS TO HAPPEN? AND. >> FANTASTIC. >> I'M GOING TO ASK YOU ANOTHER QUESTION BECAUSE IT'S AN AREA THAT IS IMPORTANT NON PROVE I CAN PARTNERS AND AND YOU HAD MENTIONED ON THE STATUS OF FNIH AND WORKING TOWARDS ONE OF THE ONES YOU MENTIONED IS EQUITY DIVERSITY AND INCLUSION AND I KNOW IN RESEARCH AND WE'RE ALL WORKING TO MAKE SURE THAT IT HAPPENS AND THERE'S A SESSION AT THE FORUM AND HOW TO BE INCLUSIVE AND SO I'M CURIOUS LIKE YOU DO GIVE US A FEW MORE ON HOW GOALS AND AREAS THAT YOU ARE PAYING FOR IN SIGHT ON FROM OUR COMMUNITY MEMBERS. >> I WANT TO START WITH THE AGO NOMMENT THAT A LOT HAS BEEN DONE AND SO I DON'T MEAN TO IMPLY THAT SOMETHING WAS WRONG AND RAISING INTO FIX IT AND FROM A GRASSROOTS LEVEL BECAUSE OF THE IMPOSED ISOLATION OF THE PANDEMIC AND HALF OF OUR ORGANIZATION GOT TOGETHER SO I THINK THAT IT CAN GO PIR IN THE SPACE AND SOME OF THE AREAS WHERE WE NEED TO IMPROVE JUST DON'T THE DIVERSITY OF WORKFORCE AND THE INCLUSION AND COMFORT THAT WE CREATE FOR THE BROAD SPECTRUM OF EMPLOYEES THAT WE HAVE SO ONE OF THE WORKGROUPS IS SPECIFICALLY LOOKING AT OUR WORK AND NOT JUST WHO WE ARE AND HOW WE INTERACT WITH EACH OTHER BUT DOES OUR COMMITMENT TO SCIENCE AND THE MISSION OF THE NIH BUT ULTIMATELY ARE WE INCLUSIVE IN THAT SENSE? THAT'S A HARDER QUESTION. I DON'T KNOW THE ANSWER BECAUSE WE HAVE COMPLEX SET OF PROGRAMS AND THAT IS WHAT I TEND TO FIND OUT. AND WE KNOW THE HEALTH DISPARITIES ARE SO PARAMOUNT AND WHAT DO WE GET TOGETHER WITH OUR PARTNERS AND HOW DO WE SELECT PARTNERS THAT ARE GOOD AT THIS SO THERE'S AN EXPECTATION THAT IT'S NOT JUST THAT THEIR THIS BUT EVERYBODY THAT COMES INTO KIND OF THIS PART AND IT'S COMMITTED AND THAT IS WHAT IS SO GREAT ABOUT THE RECENT MEETING AND SO WE ARE ADDRESSING THAT THEY TOOK THE ENTIRE MEETING TO FOCUS ON THIS ISSUE. >> IT'S FANTASTIC AND I LOVE THAT POINT ABOUT IT IS COLLABORATIVE AND IT'S NOT JUST ALL FNIH OR ALL NIH IT'S THE WHOLE COMMUNITY AND TAKING EXPERTISE SO WE CAN MOVE FORWARD IN A POSITIVE WAY. WE HAVE IS LOOKING ANOTHER THE FUNDRAISING PARTICULARS SO IN TERMS OF YOU HAD MENTIONED FNIH AND JUST A LITTLE BIT MORE TWO THINGS MEAN AND WHAT DOES IT NOT VERSED IN KIND OF THE WORLD OF DONATIONS AND HOW GET ARTICULATE IN THE SYSTEM. >> EARLY DAYS FOR ME IN THIS REGARD AND ONE IS THE TRADITIONAL FILL AN THROW I CAN APPROACH GOING WAY BACK 25 YEARS AGO AND THE PEOPLE WHO WERE PART OF THE ORIGINAL BOARD OF THE FOUNDATION HAD STRONG PHILANTHROPIC TENDENCIES AND WE HAVE SUPPORT THAT WAS LONGSTANDING AND WE ALSO HAVE SPECIALEN COUPLES AND AND THEY SUPPORT IN WAYS AND THE I SKIPPED OVER THE SLIDE AND I DESCRIBED OUR ANNUAL AWARDS DINNER AND WE NOMINATE PARTICULARLY TALENTED AND PRODUCTIVE SCIENCES AND AN AWARD AND THAT IS SOMETHING THAT HAS BEEN SUPPORTED BY FOR A NUMBER OF YEARS OR GRATEFUL AND IT'S 10 YEARS AND SO THAT IS THE KIND OF THING THAT WE TRY TO BUILD ON GOING FORWARD AND WE HAVE A DIFFERENT FUNDRAISING AND THAT'S PROJECT FUNDRAISING AND THE FOLKS THAT ARE INTERESTED IN ARE PROJECT AND THOSE WHO ARE STAKEHOLDERS AND INCLUDES INDUSTRIES AND BIOTECH OR PARTNERS FOR THE MOR DIGITAL APPROACH TO WHAT WE'RE DOING AND IN ADDITION TO THIS WE DO RECEIVE SIGNIFICANT RESOURCE 0 FOUNDATIONS AND I MENTIONED GATES BUT THERE ARE OTHER FOUNDATIONS WHO HAVE A SPECIAL INTEREST IN A PROJECT AREA OR JUST THE ADVANCEMENTS AND THE HARTEST THINGS FOR US TO ACCELERATE THE START UP OF SOME OF OUR FUNDS SO NOT HAVING TO WAIT UNTIL UNTIL THE MONEY WAS THE SPTO --WE'VE LEARNED THAT AO NOW WE HAVE TO FIGURE OUT HOW CAN WE ARE PART OF THAT ENGINE AND IT DRIVES THE PROGRESS FORWARD AND MORE EXPEDITION US MANNER. >> FANTASTIC. SO WE DID JUST RECEIVE ONE LAST QUESTION THAT WE DO HAVE A COUPLE MINUTES FOR BUT I WANT TO JUST ACKNOWLEDGE AGAIN THAT YOU HAVE RECENTLY TAKE NOT ON THIS FANTASTIC ROLE SO IT'S CONCEIVABLE YOU KNOW THE ANSWER TO THIS BUT THAT'S OK WE CAN PUT A PIN IN IT IF NOT. ONE OF OUR GROUP WAS SO DELIGHTED BY YOUR TALK BECAUSE THEY DIDN'T EVEN KNOW NIH EXISTED SO THIS WAS FANTASTIC AND WHAT WE WERE TRYING TO DO. THE QUESTION IS, KIND OF, ABOUT MORE OF A TARGETED RESEARCH PROJECT IN THE NEUROSCIENCES AND SO YOU MENTIONED ONE OR TWO AND IS THERE ANYTHING THAT FNIH HAS ITS FINGERS IN WITH WITH REGARD TO BRYN INJURY AND THE AND OF THAT AND I KNOW THAT THE HISTORY AND THE PROCESS AGENCIES AND I HAVE TO CHECK ON THAT WOULD BE SURE AND I DO JUST REALLY WANT TO SAY IN CLOSING THAT I APOLOGIES FOR NOT BEING FULLY INFORMED ABOUT EVERYTHING, IT'S JUST BEEN DRINKING FROM A FIRE HORSE AND I WOULD ALSO JUST SAY WHEN YOU THINK ABOUT FNIH AND THINK ABOUT IT IN THE SENSE OF THE MOUSE THAT ROARED AND WE WANT TO STAY IN THE GOLDILOCKS STAGE OF BEING VISIBLE ENOUGH TO DO OUR WORK WELL AND BE HELPFUL TO THE NIH AND OUR PATIENTS BUT AT THE SAME TIME, WE'RE NOT GET BIG AND GO HIGHWAYLY VISIBLE AND FRONT-RUNNER HERE AND WE'RE VERY HAPPY TO BE CATALYST OF GREAT SCIENCE AND ULTIMATE LOW TO DO WHAT I HOPE WILL BE BETTER HEALTH FOR THE PEOPLE THAT REALLY FEET N AND HOPEFULLY I'LE MORE INFORMED. >> YOU DID AN AMAZING JOB AND THANK YOU FOR GIVING US YOUR TIME. I KNOW IT'S BEEN DRINKING FROM THE FIRE HORSE AND I APPRECIATE ALL THE INFORMATION YOU SHARED AND WE ARE GOING TO ARCHIVE THIS SO THAT THOSE WHO WANT TO REFERENCE IT AGAIN IN THE NEWT ARE ABLE TO THANK YOU SO MUCH AND HAVE A WONDERFUL DAY AND I HOPE YOU ARE ABLE TO JIN OUR SESSIONS IN THE NEXT TWO DAYS. >> ENJOY THE REST OF THE MEETING, EVERYONE. THANK YOU. >> THANK YOU. >> SO WITH THAT, I'M GOING TO SEG WAY US INTO OUR NEXT SESSION AND THIS IS A SESSION THAT IS GOING TO BE LED BY Dr. GERALD DEAN ACUNA SUNSHINE AND PATIENT ADD VOW SEE IN COMMERCIALIZATION HOW TO WORK WITH INDUSTRY SO I JUST WANT TO GIVE A FEW REMARKS ABOUT GERALDINE SHE'S THE PRESIDENT OF THE SUNSHINE CARE FOUNDATION FOR NEUROLOGICAL CARE AND RESEARCH AND A CO-FOUNDER OF THE CENTER FOR DIS TONE YAR PARKINSON'S AT THE MASSACHUSETTS GENERAL HOSPITAL. THESE ARE TWO INTERNATIONAL NON-PROFIT ORGANIZATIONS, TWO, DEDICATING TO FINDING NEUROSCIENCE RESEARCH AND FINDING WAYS OF DELIVERING FRO CLINICAL CARE AND HUMANITARIAN AID TO INDIGENOUS ENTER PATIENTS IN RURAL AREAS OF AISH AMOUNT SHA.SHE WAS THE FIRST BRAIN BANN THE PHILIPPINES NOW PRIOR, SHE ENTERED NO THE NON-PROFIT AND SOCIAL IMPACT SECTORS, SHE PRACTICED CORPORATE LAW FULL TIME AS SENIOR COUNCIL IN ALTERNATIVE INVESTMENTS AT BRACE BRIDGE CAPITAL IN BOSTON AND SERVED ON THE HARVARD UNIVERSITY OF OVERSEERS AND THE PRESIDENT COUNCIL OF MGH AND SHE SITS ON THE VISITING COMMITTEE FOR THE HARVARD SCHOOL OF PUBLIC-HEALTH AND THE HARD SAD INITIATIVE AND SHE LIVES IN BOSTON AND AS WELL AS MANILA IN THE PHILIPPINES. I'LL TAKE IT AWAY AND I DO WANT TO NOTE, I'M SO SORRY OTHER, ONE LAST THING, THAT I WOULD LIKE TO ENCOURAGE OUR REGISTRANTS TO DO. WE'RE GOING TO PASTE IN THE CHAT THE OPPORTUNITY TO CONTINUE TO SCHEDULE ONE-ON-ONE MEETINGS WITH NINDS PROGRAM STAFF OVER THE NEXT TWO DAYS AND ON WEDNESDAY AND I ALSO WANT TO ENCOURAGE ALL OF OUR NON NIH PARTICIPANTS TO CONSIDER FILLING OUT A SURVEY THAT WAS CREATED BY THE NON-PROFIT MEMBERS OF OUR EXECUTIVE BOARD FOR A BREAKOUT SESSION THAT IS AT THE END OF DAY TWO. APOLOGIES. THE FLOOR IS YOURS -- >> AWESOME. THANK YOU SO MUCH, SAM. GOOD MORNING, EVERYONE, WELCOME TO OUR FIRST PANEL DISCUSSION OF THE MORNING AND THANK YOU TO THE ENTIRE NINDS TEAM FOR THIS AMAZING OPPORTUNITY FOR US TO GATHER IN THIS FORUM, AS WAS MENTIONED MODERATOR FOR THIS AND AND COMMERCIALIZATION AND HAVE TO WORK WITH INDUSTRY AND AS MENTIONED I CO FOUNDED THE CENTER FOR DIS TONIA AND IT'S A RARE ADULT ONSET NEURODEGENERATIVE DISEASE FOUND IN PATIENTS WITH ANCESTRY FROM THE PHILIPPINES. SO, WE HAVE AN AMAZING GROUP OF PANELISTS TODAY. BUT BEFORE WE BEGIN, THEY DID WANT ME TO CLARIFY BECAUSE OF THE SESSION TITLE, THAT OUR DISCUSSION WILL NOT BE FOCUSED ON THE COMMERCIALIZATION OF THERAPEUTICS, PER SE, BUT RATHER ON HOW PATIENT ADVOCACY GROUPS AND COMMERCIAL ENTITIES CAN WORK TOGETHER TO FIND THERAPEUTICS SOLUTIONS FOR THEIR RELEVANT DISEASES. AFTER THE PANELIST DISCUSSION, WE WILL HAVE 10 TO 15 MINUTES IN THE END FOR YOUR QUESTIONS AND IF YOU WITH PUT THEM IN THE CHAT, YOU WILL CALL ON YOU AT THE END OF THE MEETING. SO, JOINING US SO JOINING ME IS CATHERINE BEAVERSON, AND CONNECTS PATIENTS AND INDUSTRY PARTNERS AND FOCUSED ON ULTRARARE NEURO DEVELOPMENTAL DISORDERS IN THEIR JOURNEY TO DRUG DEVELOPMENT AND LAURA MITIC CHIEF SCIENTIFIC OFFICER AT BLUEFIELD PROJECT TO CURE FTD. AND I BELIEVE WE ARE ALL THERE, WE HAVE NO SLIDES SO HERE WE ARE. ALL OF US ON THE SCREEN. SO, WELCOME, THANK YOU AND LET'S START WITH CATHERINE, WELCOME, GOOD MORNING. >> GOOD MORNING. >> YOU KNOW, AS WE DISCUSSED DURING ONE OF OUR THREE CONFERENCE CALLS, I COME ACROSS A 2015 STUDY CONDUCTED BY SOME RESEARCHERS AT DUKE ON THE HURDLES TO COLLABORATION AMONG PATIENT GROUPS, INDUSTRY PARTNERS AND AK DEEM I CAN AND WHILE INDUSTRY IDENTIFIED INTERNAL BUREAUCRACY AS ITS GREATEST HURDLE TO COLLABORATION, PATIENT GROUPS FOCUSED ON THE LACK OF TRANSPARENCY AND UNDERSTANDING OF HOW TO ENGAGE WITH INDUSTRY AS THEIR GREATEST HURDLE. AND IN THAT DISCUSSION, YOU SHARED WITH ME A LINK TO FRAMEWORK THAT I FOUND REALLY HELPFUL DEVELOPED BY THE CLINICAL TRIALS TRANSFORMATION INITIATIVE. IT'S A GROUP OF ACADEMICS, PEOPLE AT THE FDA, PATIENT ADVOCATES AND INDUSTRY INCLUDING PEOPLE AT PFIZER, THAT DEVELOPED THIS FRAMEWORK WHICH I FOUND REALLY HELPFUL. AND I BELIEVE THAT WE HAVE THIS LINK IN THE CHAT AND I WONDERED IF YOU COULD LEAD US THROUGH AND GIVE A BRIEF OVERVIEW OF THE CTTI FRAMEWORK BECAUSE I FOUND IT VERY HELPFUL. >> THANK YOU IT'S A GREAT QUESTION AND I'M HAPPY IT TALK ABOUT THIS FRAMEWORK AND THE MODEL THAT CTTI HAS PUT TOGETHER AND LET'S QUALIFY THAT WITH THERE ARE A SHARED PURPOSE IN HOW DO WE ENHANCE THE QUALITIES AND THE EFFICIENCY OF THE CLINICAL TRIAL PROCESS AND EXPERIENCE. THE STUDY THAT YOU REFERENCED REALLY SAID OK, WELL, WHO ARE THE STAKEHOLDERS INVOLVED IN THIS PROCESS AND WHO DO SHARE IN THIS PURPOSE AND THIS MISSION AS YOU MENTIONED, ACADEMICS, SPONGESSORS OF CLINICAL RESEARCH AND PATIENT COMMUNITIES HAVE A SHARED INTEREST BUT YOU HAVE TO START FROM PLACE OF WELL, YOU KNOW, HOW WELL DO WE KNOW EACH OTHER AND HOW WELL DO WE KNOW OUR CAPABILITIES AND HOW WELL DO WE KNOW OUR OWN CAPABILITIES AND THAT INITIAL STUDY WRAPPED OUT WHERE ENTITIES SAW THE CHALLENGES TO ENGAGING WITH EACH OTHER BUT THEIR PERCEIVED VALUE NAD THAT DISCUSSION AND COMING OUT OF THAT WORK THIS MODEL THAT FOLKS HAVE BEEN ABLE TO OPEN UP AND TAKE A LOOK AT WAS REALLY PUT FORWARD TO SAY HERE THE OPPORTUNITIES AND PATIENT GROUPS CAN TAKE TO ENGAGE IN THE CLINICAL TRIAL CONTINUUM RECOGNIZING WHAT ARE SOME OF THE SCIENTIFIC MILESTONES THAT NEED TO BE ACHIEVED IN ORDER TO MOVE FORWARD IN THE PROCESS AND SO THE MODEL NICE LOW LAYS OUT AT A HIGH LEVEL, THAT THIS STAGE OF DEVELOPMENT LOOKING AT KIND OF DISCOVER RODISCOVERY AND PRE CLK AND THE REASON TO BELIEVE FROM A SCIENTIFIC RATIONALE STANDPOINT AND MOVING INTO THOSE CLINICAL PHASES OF DEMONSTRATING SAFETY AND EFFICACY AND WHAT DOES A REGULATORY REVIEW LOOK LIKE AND THE DECISION MAKERS AND THE DATA PACKAGE THAT IS SUBMIT AND WHAT NEEDS TO HAPPEN IN A POST APPROVAL PROCESS. SO NICELY LAYS OUT WHAT ARE THOSE MAJOR MILESTONES THAT NEED TO BE ACHIEVED ALONG THE DEVELOPMENT PATHWAY AND WITHIN THOSE STAGES WHAT RECOGNIZES TO MOVE IT FORWARD AND SO, IN THAT DISCOVERY AND PRE CLINICAL SPACE, IT'S REALLY AROUND WHAT ARE THE STUNTS TO DERISK THE SCIENCE THAT ENABLES THE ADVANCEMENT AND TO CLINICAL RESEARCH AND SOME OF THE OPPORTUNITIES THERE ARE GRANT FUNDING OF RESEARCH AND IDENTIFYING WHAT ARE SOME OF THE SCIENTIFIC NETWORKS, WHERE THE MOUSE MODELS AND WHERE ARE THE AS SAYS BEING DEVELOPED AND ALL THOSE KINDS OF THINGS AND WHEN YOU GO INTO OPERATIONALIZING CLINICAL RESEARCH, HOW CAN PATIENT GROUPS BE INFORMING INDUSTRY TO OPTIMIZE THE FEASIBILITY AND THOSE OPERATIONAL ASPECTS OF THE TRIAL WITH A SHARED PURPOSE OF REDUCING BURDEN ON THE PATIENT COMMUNITY, RIGHT. ENHANCING RECRUITMENT AND RETENTION. ALL OF THESE THINGS IMPORTANT TO BOTH PARTICIPANTS AND TO SPONSORS AND HOW DO WE LEVERAGE THE EXPERTISE THESE ENTITIES BRING TO THE TABLE AND FURTHER ALONG IN THE PROCESS AND HOW DO PATIENTS ENGAGE IN THAT DECISION-MAKING AND I THINK WHY IS THIS THAT PEOPLE CAN COME TOGETHER AND SPEAK TO EACH OTHER WHAT IS IMPORTANT FROM A PATIENT ADVOCACY ORGANIZATION STAND POINT AND WE MIGHT NOT BE ABLE TO DO EVERYTHING BUT WE CAN CHECK AND WE CAN SET OUR MISSIONS AND OUR CAPE A LOTS AGAINST WHAT IS NEEDED FOR OUR COMMUNITY AND HOW WE K WE FIT INTO THIS FRAMEWORK SO A LONG WINDED ANSWER, I HOPE THAT WAS HELPFUL. >> YES, AND I THINK IT'S GREAT AND AS STATED IN THE CHAT, PEOPLE WILL SEE THAT IF THERE'S MORE ON THE WEBSITE AND I WOULD JUST ENCOURAGE EVERYONE TO GO ON THERE AND FIND WHAT MIGHT BE USEFUL FOR YOU. SO, LET'S MOVE ON TO YAEL. YOU'VE SEEN THIS FRAMEWORK AND THERE'S A EUROPEAN FRAMEWORK SIMILAR THAT CAN HELP GUY PATIENTS THROUGH AND MAHZI THERAPEUTICS AND YOU HAVE A GROUP OF PEOPLE THAT RUN A BOOTCAMP THAT GUIDES PATIENTS THROUGH THIS PROCESS, RIGHT, TO INTRODUCE AND BRING PATIENT GROUPS AND INDUSTRY TOGETHER. CAN YOU TALK ABOUT THAT AND OF COURSE AFTER THAT, WHAT YOU DO WITH MAHZI. >> SURE. THANK YOU FOR HAVING ME. SO, I'D LICK TO FOLLOW-UP ON WHAT CATHERINE WAS DISCUSSING, THE FRAMEWORK. I THINK THE FRAMEWORK IS EXCELLENT AND IT'S A GREAT STARTLING POINT. WHAT WE REALIZED DURING MY TIME AT ULTRA-GENICS AND OF COURSE NOW WITH MAHZI, THERE ARE A LOT OF NEW FOUNDATIONS THAT ARE COMING TO LIFE IN THE LAST FEW YEARS AND SINCE THAT PUBLICATION IN 2015, THE LANDSCAPE HAS CHANGED SIGNIFICANTLY AND THERE ARE A LOT OF ESTABLISHED FOUNDATION THAT'S HAVE BEEN THROUGH INTERACTIONS WITH INDUSTRY AND THEY HAVE TAKEN DRUGS AND DEVELOPED THEM AND HANDED THEM OVER TO PARTNERS BUT THERE ARE MANY, MANY NEW FOUNDATION AND SOME OF THEM I SEE HERE IN THE AUDIENCE AND ARE REALLY STARTING OUT AND MANY OF THEM ARE NOT BIOLOGISTS BY EDUCATION OR DRUG DEVELOPMENT BY PROFESSION AND THE GAP BETWEEN THE FRAMEWORK AND ACTUALLY PUTTING IT TO PRACTICE IS PRETTY SIGNIFICANT BECAUSE YOU LOOK AT IT AND NOT UNDERSTAND THE STEPS BECAUSE YOU'VE NEVER BEEN THROUGH IT BEFORE SO AFTER RESPONDING TO MANY INDIVIDUAL FOUNDATIONS AND PARENTS AND PATIENTS ON ALMOST A WEEKLY BASIS AT ULTRA-GENICS, WE DECIDED TO PUT TOGETHER AN EVENT THAT WOULD EDUCATE PEOPLE THAT ARE NEW TO THE DRUG DEVELOPMENT SCENE OR AREA AND EDUCATE THEM ABOUT WHAT IT'S ALL ABOUT SO WE ORGANIZED A THREE-DAY EVENT WHERE PEOPLE COME TO THE ULTRA-AGAIN -- WE TRYTO COMPLAT STARTING FROM HOW TO COLLABORATION WITH ACADEMICS AND NEGOTIATE A LICENSE AGREEMENT ALL THE WAY THROUGH REGULATORY CLINICAL END POINTS TALKING ABOUT REGISTRIES AND NATURAL HISTORY STUDIES FOR THOSE WHO ARE NEW TO THIS OR MAY BE STARTED OUT AND KNOW WHAT IS COMING ON THE HORIZON IT'S A GREAT OPPORTUNITY TO NOT ONLY LEARN BUT ALSO INTERACT WITH EXPERTS AND GIVE TALKS WITH THE BOT CAMP AND PEERS AND SHARE BEST PRACTICES. WE KEEP REFINING AND IMPROVING THE PROGRAM BASED ON FEEDBACK FROM PARTICIPANTS AND HOPEFULLY THEY'LL OPEN A FOLLOW-UP FOR PEOPLE THOUGH WANT TO KEEP ENGAGING WITH THE GROUPS THAT PARTICIPATE TO HELP THEM THROUGHOUT THEIR JOURNEY ON A SPECIFIC HURDLES THAT THEY COME UP WITH AND I SEE HERE IN THE AUDIENCE THAT THERE ARE A LOT OF FOUNDATIONS AND DIFFERENT LEVELS OF MATURITY AND WE DO CATER TO THE ONES THAT ARE NEWER AND LESS EXPERIENCED BUT WHEN ANYONE KNOCKS ON OUR DOOR AND HAS A SPECIFIC PROBLEM OR ISSUE THEY NEED HELP WITH WE ENGAGE WITH THE DIFFERENT EXPERTS AND THE SPONSORS OF THE BOOTCAMP THAT LEND THEIR EXPERTISE AND WE TROY TO HELP ON A CASE-BY-CASE BASIS AS WELL. THIS IS GIVEN US ACCESS AND BETTER UNDERSTANDING OF WHAT THE ISSUES THAT IS THE FOUNDATIONS HAVE TO DEAL WITH WHEN THEY'RE STARTING OUT AND IT COULD BE LOOKING AT A CHINESE PICTURE BOOK FOR A NON CHINESE SPEAKER. WE HAVE OUR OWN TERM ON OLE GOUGH AND WE TERMINOLOGY SOWE'RS POSSIBLE. TO SIMPLIFY THE PROCESS SO PEOPLE WILL FEEL MORE COMFORTABLE AS THEY DIVE INTO DRUG DEVELOPMENT. THAT'S THE BOOTCAMP. POST COVID WE'RE REFORM ATING BUT WE HOPE TO RUN IT TWICE AS YEAR AND WE DO IT WITH THE SUPPORT OF GLOBAL GENES AND THE REGISTRATION GOES TO THEM SO IF ANYONE WANTS MORE DETAILS, CONTACT ME AN I'LL CONTACT. MAHZI I AM INNATED FROM THE BOOTCAMP BECAUSE I SAW GROUPS THAT ARE FUNDING BASIC RESEARCH AND LAURA CAN TALK A LITTLE BIT MORE ABOUT HOW BLUE FIELD PROJECT IS HANDLING IT AND A LOT OF GROW GROUPS ARE FUNDING IT AD TAKING THE -- THEY DON'T HAVE THE KNOW HOW OR EXPERTISE TO TAKE IT FURTHER OR THE FUNDING INFORM TAKE IT FURTHER BECAUSE A LOT OF THESE ARE GENETIC BASED THERAPIES AND THESE ARE NOT CHEAP AND WE NEED RESOURCES IN ORDER TO TAKE THOSE INTO THE CLINIC AND FURTHER AND I STARTED THE COMPANY TO TRY AND FIRST OF ALL BE A SOURCE OF INFORMATION TO GROUPS THAT WANT TO DO THIS AND CAN COME TO US AND SEEK OUR ASSISTANCE AND PUTTING TOGETHER A RESEARCH PLAN AND ALSO WE TOOK ON THREE PROGRAMS TO GENE THERAPY AND ONE FOR SPECIFIC NEURO DEVELOPMENTAL DISORDERS AND HOPING TO BRING ON MORE AS TIME GOES BY AND HOPEFULLY FUNDING WILL COME TO ENABLE US TO TAKE ON MORE PROGRAMS AND REALLY WORK HAND AND HAND WITH THE PATIENT GROUPS WHO BRING THEIR OWN EXPERTISE, THE OUTREACH TO THE COMMUNITY AND THE DEEP UNDERSTANDING OF THE DISEASE AND THE DISORDER AND ALSO THE ABILITY TO COLLECT INFORMATION FROM THE COMMUNITY AND HELP ENROLL THESE AND BRING OUR EXPERTISE AND DRUG DEVELOPMENT AND HOPEFULLY TOGETHER TAKE A DRUG INTO THE CLINIC AND MAYBE EVEN COMMERCIALIZE AND IN THE CHAT, YOU ARE CONTACT INFORMATION OR WHERE PEOPLE -- >> I'M HAPPY TO DO THAT. THAT'S A GREAT SEG WAY TO LAURA BECAUSE YOUR CHIEF SCIENTIFIC OFFICER OF BLUEFIELD FOUNDATION FOCUS OZ ON FRONTING A CURE FOR FTD AND YOU HAVE WORKED WITH INDUSTRY AND NOW I HAVE TWO DRUGS IN CLINICAL TRIALS AND YOU'VE ALSO SEEN THIS FRAMEWORK AND HOW DID YOU GUYS START DOING YOUR WORK WITHOUT THIS KIND OF ROADMAP AT THAT TIME? CAN YOU SHARE WITH US A LITTLE BIT ABOUT YOUR EXPERIENCE? >> SURE. THANK YOU. YES, SO, THE BLUEFIELD PROJECT WAS FOUNDED IN 2009 AND WE HAVE A PRE DETERMINED SUNSET DATE OF 2025. WE'RE NEARING THE END OF OUR EXISTENCE AND ACTUALLY THERE ARE FOUR COMPANIES HAVE DRUGS IN CLINICAL TRIALS FOR THE DISEASE WE WORK ON WHICH IS FTD CAUSED BY MUTATIONS IN AN ADULT ONSET NEURODEGENERATIVE DISEASE STRIKING IN THE MID-60S WITH A SIX TO 10-YEAR TIME UNTIL DEATH. SO, WE ARE VERY FORTUNATE TO HAVE THE POSITION WHEN WE DO SUNSET, WE HOPEFULLY WILL HAVE AN APPROVED DRUGS OR DRUGS IN TRIALS AND WILL BE APPROVED OR AT LEAST HAVE READOUTS SOON AND I THINK THAT WHEN I LOOK BACK OVER THIS, EACH FRAMEWORK, I WAS PLEASED TO SEE THAT INDEPENDENTLY BLUE FIELD HAD SINCE 2009, REALLY DONE MANY OF THE THINGS ON THAT FRAMEWORK BUT I THINK IF YOU ASKED ME IN 2009 ABOUT THAT FRAMEWORK I WOULD NOT HAVE MANY TERMS ON IT EXPLAINED TO ME AND SO I THINK THAT BRINGS ME TO THE POINT IN MANY FOUNDATIONS THAT ARE ATTENDING TODAY, IF YOU HAVE NOT ALREADY AND PROBABLY MANY OF YOU HAVE AND WHICH BENEFITED FROM THE EXPERTISE OF OTHER COLLEAGUES AT OTHER FOUNDATIONS WHO WERE GENEROUS IN SHARING ALL KINDS OF INFORMATION AND EVEN A TEMPLATES FOR FUNDING AGREEMENTS AND ALL KINDS OF THINGS AS WE STARTED ON AND IN VARIOUS STAGES ALONG THE ROAD AND SO I WOULD ENCOURAGE ALL OF US TO JUST CONTINUE TO BE AS COLLEGIAL AND COLLABORATIVE AS WE ARE IN SHARING OUR INFORMATION AND OUR EXPERIENCES AND ALONG THIS WAY BECAUSE THERE'S NO REASON TO REINVENT THE WHEEL AND I THINK MANY OF THE ITEMS ON THIS CTI FRAMEWORK CAN BE CONDUCTED SIMULTANEOUSLY AND BECAUSE TIME IS ALWAYS OF THE MOST URGENCY IN THESE DISEASES AND BEING ABLE AND SMALL FOUNDATION TO DO THINGS SIMULTANEOUSLY REALLY MEANS PIGGING BACKING ON THE EFFORTS OF OTHERS. I THINK THAT THE ROADMAP, WE'RE AN AT THE FOURTH PART OF THAT ROADMAP AND I HOPE WE WILL BE AND I HOPE I CAN REFER TO IT FOR GUIDANCE AS WE HAVE OUR FORTUNATE ENOUGH TO BE IN THAT POST APPROVAL SITUATION BUT I DO THINK THE FRAMEWORK IS HELPFUL AND A GO AHEAD THING TO REFER BACK TO OVER TIME. >> THAT'S AWESOME. CATHERINE, YOU WANTED TO ADD ABOUT THE TOOLS. THIS IS ALL ABOUT TOOLS AND WHAT WE CAN USE TO HELP OUR -- I JUST WANT TO BUILD ON THE COMMENTS IN THAT THIS IS A HIGH-LEVEL FRAMEWORK THAT IS DIFFICULT TO TRANSLATE TO THE TOOLS THAT WE NEED TO ACTUALLY HAVE IT AND THE BOOTCAMP IS JUST A GREAT OFFERING AND FOR FOLKS UNDERSTANDING THIS AND THERE ARE NEW COMPANIES ALWAYS COMING INTO THE RARE DISEASE SPACE, THAT NEED INTERNALLY, TO BE ABLE TO HAVE TOOLS FOR SYSTEMATIC EASTBOUND GAGEMENT RIGHT THAT IS WHAT THE PATIENT COMMUNITIES WANTS AND HOW THEIR COMPANY FEELS THEIR FREE TO OPERATE AND I KNOW PFIZER HAS TAKEN GREAT EFFORT TO BUILD INTERNAL TOOLS FOR COLLEAGUES INVOLVED IN CLINICAL RESEARCH TO DO THIS APPROPRIATELY, SYSTEMATICALLY AND CONSISTENTLY AND IN A WAY THAT IS PALLETABLE BY THE PATIENT COMMUNITY. SO I JUST WANTED TO ADD THAT INTERNAL TOOLS FOR COMPANIES IS JUST AS IMPORTANT AS BEING ABLE TO SHARE AND DEVELOP TOOLS EXTERNAL LOLY FOR FOLKS. >> CATHERINE, THERE ARE PEOPLE IN ATTENDANCE WHO ARE JUST BEGINNING THEIR JOURNEYS HERE AND ALL OF THIS IS TRYING TO FIND THE RIGHT PARTNER, RIGHT, TO ACHIEVE OUR GOALS. SO, WHAT WOULD YOU SAY PATIENT ADVOCATE GROUPS WHO ARE STARTING OUT OR TRYING TO FIND THEIR WAY SHOULD MAKE SURE THEY DO? TO TRY TO FIND THE RIGHT PARTNER AND WHAT DOES THE RIGHT FIT LOCK LIKE? FROM YOUR PERSPECTIVE? >> YEAH, I DON'T KNOW I'M AN EXPERT ON THE RIGHT FIT. YOU KNOW, IN TERM OF WHAT ORGANIZATIONS PATIENT COMMUNITIES SHOULD BE LOOKING FOR IN THE INDUCE THROW PARTNER OF CHOICE OR MULTIPLE PARTNERS, RIGHT, IS A, DO THEY REALLY UNDERSTAND THE FOCUS OF THE COMPANY? IS IT BROAD TO RARE DISEASES? IS IT SPECIFIC TO SERVING THERAPEUTIC AREAS? DO YOU REALLY UNDERSTAND, IS IT EVEN SPECIFIC TO A PLATFORM OF OUR PLATFORM TECHNOLOGY AND SO DO YOU UNDERSTAND WHAT THE COMPANY IS TRYING TO ACCOMPLISH WITH REGARD TO ITS DISEASE AREAS OF FOCUS AND IT'S OWN CAPABILITIES AND MAYBE UNDERSTANDING WHAT IS THE LIFE CYCLE PROCESS? IS IT TO ENABLE PRE CLINICAL DEVELOPMENT AND BASIC RESEARCH OR IS IT REALLY TO MOVE INTO THE CLINIC, RIGHT? IT COULD BE BOTH. AND THEN SO DO YOU UNDERSTAND WHO YOU ARE REACHING OUT TO AND THEIR PRIORITIES AND THEIR CAPABILITIES? DO YOU UNDERSTAND THE DRUG DEVELOPMENT PROCESS ITSELF? AGAIN, THE BOT CAMP DO YOU HAVE A REALLY GOOD UNDERSTANDING OF WHAT IT TAKES TO GET TO GOAL AND WHERE YOUR ORGANIZATION WANTS TO FOCUS THEIR EFFORTS, RIGHT, BECAUSE NONE OF US CAN DO EVERYTHING. SO IT'S REALLY, DO YOU HAVE, CAN YOU FIND THAT MISSION MATCH, RIGHT, AND THEN I THINK THE CHALLENGE IS NAVIGATING THAT ORGANIZATION THAT YOU ARE THEN TRYING TO REACH OUT TO, RIGHT. AND THAT CERTAINLY IS SOMETHING THAT PFIZER HEARS A LOT OF, RIGHT. BECAUSE WE'RE SUCH A LARGE COMPANY AND TRYING TO LOCK FOR THOSE FOLKS WHO ARE FOCUSED ON PATIENT ADVOCACY, WE'RE PROBABLY BEING A GREAT PLACE TO START IN TERMS OF THAT INITIAL CONTACT. >> MAYBE I COULD SE EVEN ADD TO THAT. I WANTED TO GIVE THE PERSPECTIVE FROM OUR FOUNDATION PERSPECTIVE, WE DIDN'T -- WE WANTED AS MANY PEOPLE AS POSSIBLE TO BE INTERESTED IN OUR DISEASE AND SO WE WERE REALLY QUITE AGNOSTIC TO PRETTY MUCH ANY ASPECT OF THE COMPANY. I MEAN, WE WERE SUPER EXCITED WITH COMPANIES THAT WERE LARGER ESTABLISHED COMPANIES IN THE SPACE AND TOOK A INTEREST IN FTD BUT SMALL, GRAND NEW START UP BIO TECHS THAT WERE INTERESTED IN AND WE TRIED TO GIVE THEM EQUAL ACCESS AND EQUAL OPPORTUNITIES TO WHATEVER WE COULD DO TO HELP THEM WAS OUR MODEL BECAUSE WE DIDN'T KNOW WHO MIGHT SUCCEED AND IN FACT, WE HAD INTERESTING EXPERIENCES I WOULD SAY OVER THE LAST 10 PLUS YEARS WE'VE SPOKEN WITH 75 COMPANIES RANGING FROM ALL OVER THE WORLD AND ALL KINDS OF STAGES AND FUNDING LEVELS AND SOMETIMES WE WOULD GIVE A PRESENTATION AND THINK OH THAT'S GOING TO GO NOWHERE AND THEN TWO YEARS LATER THE COMPANY MIGHT CIRCLE BACK AND SAY BY THE WAY, HERE IS SOME DATA THAT WE HAVE YOU JUST NEVER KNOW AND IN FACT SOME OF THE COMPANIES, SOME EARLY PLAYERS IN OUR SPACE EVENTUALLY DROPPED OUT FOR A VARIETY OF REASONS AND PLAYERS WE DIDN'T KNOW HAD ACTIVE PROGRAMS ARE FARTHER ALONG THAN OTHERS SO I THINK THAT IN HINDSIGHT IT'S OBVIOUSLY VERY DEPENDENT ON THE RESOURCES AND THE FOUNDATION BUT BECAUSE IT'S SO UNPREDICTABLE WHO CAN GET A DRUG INTO THE CLINIC, AND THEY MIGHT COME YOUR WAY AS POSSIBLE. >> AND IF ANYTHING COMES OUT OF IT IMMEDIATELY YOU NEVER KNOW WHAT HAPPENS IN THE FUTURE AND IT'S GOOD FOR THEM TO KNOW ABOUT YOU AND THE MORE RESOURCES THAT YOU HAVE ABOUT YOUR SPECIFIC DISORDER, THE BETTER EQUIPPED YOU ARE TO HAVE THIS DIALOGUE WITH A COMPANY AND TELL THEM WE HAVE A REGISTRY AND A NATURAL HISTORY STUDY . BANK, WHATEVER YOU HAVE YOU KNOW THIS DISORDER BETTER THAN ANYONE ELSE. YOU ARE THE ONE THAT MIGHT TRIGGER STARTING A PROGRAM IN THIS COMPANY. DO IT EARLY AND DO IT AS MANY TIMES AS YOU CAN. BIO MEETINGS, THE BIGGEST PARTNERING MEETING FOR IN DOES TREE. IN THE PAST WE HARDLY SAW PATIENT GROUPS AND THE LAST BIO THERE WERE SO MANY PATIENT GROUPS REQUESTING MEETINGS WITH COMPANIES, IT'S AMAZING TO SEE HOW MUCH ACTIVITIES THERE IS AND HOPEFULLY THESE INTERACTIONS WILL GENERATE AN ENERGY THAT WILL START A PROGRAM AND HELP SOMEONE START A PROGRAM SO DO IT DEFINITELY. >> THAT'S GREAT. CAN YOU TALK ABOUT GROUPS AND WE HAVE OUR SPECIFIC INTERESTS AND WE THE ALIGNMENT HAS TO BE BETWEEN A PATIENT GROUP AND A GOAL WHICH IS CURE MY DISEASE AS SOON AS POSSIBLE, RIGHT. AND USUALLY YOU WORK WITH AN ACADEMIC WHO NEEDS TO PUBLISH AND GET GRANT MONEY AND TUN YOU'RE ATENUREAT THE UNIVERSITYD THEY NEED TO BE SUCCESSFUL AND GENERATE REVENUE. IT DOESN'T ALWAYS ALIGN. MY RECOMMENDATIONS TO PATIENT GROUPS THAT ARE WORKING WITH ACADEMICS AND I KNOW THERE'S A SESSION ABOUT WORKING WITH ACADEMICS LATER SO I WON'T SPEND TOO MUCH TIME AND GOING TO PHARMA AND UNDERSTANDING THE GOALS DON'T ALWAYS ALIGN BUT THERE CAN BE ALIGNMENT IF THERE'S VALUE BROUGHT IN BY THE PATIENT GROUP OR BY THE FOUNDATION THAT HAS ALL THE INFORMATION THAT COULD HELP ACCELERATE A PROGRAM OR WORKING PARALLEL TO THE PRE CLINICAL OR CLINICAL DEVELOPMENTS SO THIS THINGS CAN BE EXPEDITED AND NOT TROY TO DO IT STEP WISE ONE AFTER THE OTHER BUT WORK PARALLEL ON MANY THINGS. ALSO, INTRODUCE THE COMPANIES TO YOU AND YOUR FAMILY AND YOUR STORY. A LOT OF TIMES IT SWITCHES SOMETHING IN THE BRAIN OF PERSON THAT YOU ARE YOU CANNING TO AND ALL OF A SUDDEN THEY UNDERSTAND WHY THERE'S A SENSE OF URGENCY. ON MY PH.D I WORKED ON DOWN SYNDROME AND THE FIRST YEAR AND THE LAB I'VE NEVER MET A FAMILY THAT HAD A CHILD EFFECTED BY DOWN SYNDROME AND NEVER MET A PATIENT WITH DOWN SYNDROME AND THE MOMENT IT HAPPENS, MY PERSPECTIVE COMPLETELY CHANGED AND A LOT OF TIMES, PEOPLE DON'T KNOW AND HAVEN'T BEEN ABLE TO GET THIS EXPOSURE SO HELP THEM GET THAT EXPOSURE. >> THAT'S REALLY IMPORTANT. WHEN WE TALK ABOUT I.P. AND LAURA, CAN YOU SHARE WITH US HOW BLUE FIELD HAS DEALT WITH THE IP ISSUES TO GET WORK ON THE FTD? >> SO I THINK OUR FOUNDATION FROM THE START, DECIDED THAT WE WEREN'T GOING TO FUND PROGRAMS WITHIN INDUSTRY AND SO THAT MAKES IT A LOT EASIER IN THE SENSE THAT YOU DON'T NEED TO NEGOTIATE MORE COMPLICATED CONTRACTS SO JUST THAT'S THE STARTING POINT SO THEN WHAT WE DID DO WAS WE FUNDED ACADEMIC RESEARCH AND ESPECIALLY BECAUSE WHEN OUR FOUNDATION WAS FOUNDED THERE WAS LITTLE KNOWN ABOUT OUR DISEASE OR THE MECHANISM OF WHY MUTATIONS CAUSE FTD AND WHEN THE ACADEMICS WERE SUCCESSFUL IN CREATING TOOLS AS THEY DID THAT, WHAT WE DECIDED TO DO WAS TROY TO LOWER THE BARRIER TO GET THOSE TOOLS INTO COMPANIES FOR ANY COMPANY THAT MIGHT BE INTERESTED IN WORKING ON FTD AND SO, WE LICENSED SOME OF THE TOOLS IN PARTICULAR MOUSE MODEL AND PATIENT DERIVED CELL LINES FROM DIFFERENT ACADEMIC INSTITUTIONS WHERE INVESTIGATORS HAD CREATED AND WORKED ON THEM AND THEN WE HAD THE A LOT ABILIO GET THEM IN THE HANDS OF INTERESTED COMPANIES FOR EXPLORE TORI RESEARCH AS QUICKLY AS POSSIBLE BECAUSE OFTEN TIMES, ACADEMIC INTERESTS, THEY MUST BE BOGGED DOWN AND IN THIS WAY, WE CAN HAVE A MASTER AGREEMENT THAT WE CAN CONTROL THE PROCESS OF SENDING OUT THE REAGENTS VERY QUICKLY AND MAKE IT AS STREAMLINE AS POSSIBLE AND ITS DO THINK THAT HELPED. IF COMPANIES HAD INITIAL INTEREST AND THEY THOUGHT, MAYBE I WOULD LIKE TO TROY AN IDEA OR HYPOTHESIS, SO VERY, VERY EX MOR EXPLORATORY RESEARCH AND IT MIGHT JUMP START A PROGRAM SO WE FELT THAT TIME WAS OF THE ESSENCE AND THE WAY WE CAN DO THAT WAS TO OFFER THIS SUB LICENSING OPPORTUNITIES. >> GREAT. CATHERINE. >> THAT'S BRILLIANT. RIGHT, BECAUSE I CAN'T SPEAK IN DETAIL AROUND THE COMPLEXITIES OF IT BUT I DO KNOW THAT WHEN WE TRY TO OPERATIONALIZE COLLABORATIONS, THROUGH CONTRACTING, WHETHER IT'S WITH AN EPIDEMIC OR ADVOCACY GROUP, THE ISSUES BECOME VERY, VERY COMPLEX AND VERY FAST AND IT DOES SLOW DOWN THE PR PROCESS TT IDENTIFY AND ALIGN AGAINST A SHARED SENSE OF URGENCY AND IT BREAKS DOWN WHEN YOU TRY AND EXECUTE THESE CONTRACTS BECAUSE YOU HAVE VERY BUSY ACADEMIC INSTITUTIONS AND YOU HAVE YOUR LEGAL PARTNERS AT A COMPANY THAT ARE REALLY WANTING THAT FREEDOM TO OPERATE AND CLARITY. >> MAYBE I CAN JUST CLARIFY, OUR ABILITY TO SUB LICENSE WAS REALLY FOR EXPLORATORY RESEARCH. IF A COMPANY PERSUADE A PURSUE M THEY NEED TO GET A COMMERCIAL LICENSE. WE REALLY JUST WANTED TO INCENTIVIZE THAT FIRST STEP. LET'S GET A HOOK INTO THE COMPANY TO GET THEM INTERESTED ON THAT PROGRAM AND THEN IF IT GOES FORWARD, THEN THEY WILL STILL HAVE TO DO THE COMMERCIAL NEGOTIATIONS BUT LET'S GET THEM A LITTLE BIT INTERESTED BEFORE TO SEE WHETHER OR NOT IT'S WORTH THEIR TIME. >> I DO HEAR FROM SOME FOUNDATIONS THAT THEY ARE NOT GOING TO BOTHER WITH FILING I.P. OR BOTHERING WITH I.P. BECAUSE IT'S SUCH A DISTRACTION AND I THINK IF YOU UNDERSTAND HOW VALUABLE THIS INTELLECTUAL PROPERTY IS IF YOU WANT TO PARTNER WITH A COMPANY THAT WILL LATER COMMERCIALIZE IT, IT'S VERY IMPORTANT FOR THE PARTNER. SO YOU MAY NOT THINK IT'S IMPORTANT FOR YOU AND IT COST MONEY TO FILE IP AND TO MAINTAIN IT, IT IS IMPORTANT IF YOU PARTNER WITH A COMPANY THAT WANTS IT GENERATE REVENUE AND HAVE AN ADVANTAGE WITH IT GETS TO THE MARKET WITH A PRODUCT THAT'S VERY IMPORTANT TO HAVE I.P. SO DON'T UNDERESTIMATE THE VALUE OF IT AND FOR PATIENT GROUPS THAT ARE WORKING WITH ACADEMICS AND FUNDING RESEARCH, IT'S VERY IMPORTANT THAT YOU GET ACCESS TO THAT I.P. THAT IS FILED BASED ON THE MONEY THAT YOU FUNDED THE PROGRAM WITH AND LICENSE THIS I.P. WHEN THE RESEARCH IS DONE AND THE RESULTS LOOK PROMISING BECAUSE IF YOU FUNDED IT, YOU DESERVE TO CONTROL IT EVER AT RESEARCH IS DONE. >> YEAH, I'M ASSUMING THIS I.P. SECTION WILL BE DISCUSSED THERE AS PART OF THE BOOTCAMP. >> OH, YEAH. WE STUDY HALF A DAY ON THE BUSINESS OF SCIENCE WHICH IS INTELLECTUAL PROPERTY AND NEGOTIATING LICENSES WITH THE UNIVERSITIES AND WHAT CAN YOU ASK FOR AND HOW TO STRUCTURE AND WE'VE SEEN SEVERAL GROUPS THAT FUNDED RESEARCH WITHOUT GETTING ACCESS TO THE RESULTS OF THE RESEARCH AND THE FRUIT THAT CAME OUT THE RESEARCH AND IT WAS FRUSTRATING TO THEM. >> OH MY GOSH. THAT'S A GOOD SEG WAY TO MY NEXT QUESTION WHICH IS YOU GUYS HAVE BEEN IN THIS SPACE AND THE SCIENCE WIELD FIELD FOR A LONG TIME. YOUR WHOLE CAREERS. I WANT INFORMED ASK YOU, YOU KNOW, IF YOU COULD THINK OF SOME CASES WHERE THE RELATIONSHIPS HAVE WORKED REALLY WELL BETWEEN PATIENTS GROUP AND INDUSTRY. NO NAMES BUT WHAT ARE SOME CASES THAT YOU MIGHT CITE AS SORT OF MODELS FOR GOOD RELATIONSHIPS IN PARTNERSHIPS? >> I HAVE A GREAT EXAMPLE BECAUSE A PRESS RELEASE JUST CAME OUT YESTERDAY AND THAT IS A RELATIONSHIP BETWEEN ULTRA-GENICS AND THE ANGLE FOUNDATION AND THE COMPANY THE FOUNDATION SPUN OUT AND IT'S A RELATIONSHIP THAT STARTED OVER W YEARS AGO ULTRA-GENICS SUPPORTED THE GENETICS, THE COMPANY SPUN OUT OF THE FOUNDATION AND ULTRA-GENICS ENTERED A COLLABORATION FOR GENETICS AND FUNDED SOME OF THE RESEARCH THAT THEY DID AND TOOK IT INTO THE CLINIC WITH AN OPTION TO ACQUIRE THE COMPANY AND THEY ENDED UP YESTERDAY ANNOUNCING THAT THE COMPANY WAS ACQUIRED SO IT TOOK TIME AND IT WAS A VERY INTERESTING COLLABORATION FROM WHEN WE SIGNED THE AGREEMENT AND THE ACQUISITION OF THE COMPANY. IT WAS A VERY PROLIFIC RELATIONSHIP AND ENDED WITH HOPEFULLY WITH A THERAPY IN THE CLINIC AND WE'LL GET TO MANY PATIENTS SO I THINK THE LEARNING FROM IT AND NOT THAT I RECOMMEND EVERY FOUNDATION TO SPIN OUT COMPANIES AND NOT EVERY FOUNDATION CAN DO IT, YOU NEED RESOURCES FOR IT BUT IT GOES BACK TO WHAT I SAID BEFORE, ENGAGE EARLY WITH A PHARMA PARTNER AND GET AS MUCH INFORMATION AS YOU CAN AND TRY TO GET THEM TO HELP WITH EXPERTISE, EVEN IF THEY'RE NOT INVESTING MONEY, SOMEONE YOU CAN TAP INTO AND ASK QUESTIONS AND MAKE THEM AWARE OF YOUR PROGRAM AND GET THEM TO LIKE THE PROGRAM AND EVENTUALLY IT MIGHT END WITH A GREAT BUSINESS RELATIONSHIP AS WELL. >> AND THAT DISEASE IS ULTRARARE, RIGHT. LIKE WHAT IS? >> THERE ARE A FEW THOUSAND PATIENTS IN THE WORLD AT THE MOMENT. >> HOW LONG DID IT TAKE TO GET FROM STARTING A BUSINESS TO THE ACQUISITION? >> SO THEY STARTED THE COMPANY IN 2019 OR 2018. AFTER THEY SHOPPED IT AROUND WITH MANY IN THE STREET AND PARTNERS AND THERE WAS AN INTEREST AT THE TIME BECAUSE THEY HAD VERY LOW DATA SO THEY DECIDED TO CONTINUE FUNDING THE PROGRAM AND THEY GOT A FULL AND VERY COM ROW HENCE I HAVE PROOFD SO THEY DECIDED TO LOOK FIRE PARTNER AS WELL AND THEY ACTUALLY WERE LOOKING FOR VENTURE FUNDING OR A PARTNER AND WE MET WITH THEM AND WE LIKED THE PROGRAM AND WHAT WAS PRESENTED TO US AND DECIDED TO ENGAGE IN THIS COL A COLLABORAT. THEY START INSIDE 2017 AND WE STARTED THE RELATIONSHIP WITH THEM IN 2019 AND THREE YEARS LATER THE COMPANY WAS ACQUIRED. >> AMAZING. AMAZING. ANY OTHER SUCCESS STORIES YOU CAN THINK OF? >> I'M HAPPY SHARE AN EXPERIENCE BECAUSE I THINK IT MIGHT BE A DIFFERENT APPROACH TO CONSIDERING WHAT SUCCESS LOOKS LIKE, RIGHT. TO THE COMMENTS OF MY PANEL COLLEAGUES EARLIER, I THINK OUR GOAL WHERE I SIT IN MY ORGANIZATION IS REALLY FOCUSES ON THAT DISCOVERY PIECE AND CONCEPTS SO OUR MODEL IS TO ENGAGE VERY EARLY ON TO CO CREATE THE RESEARCH QUESTIONS, RIGHT, BACK WHERE ADVANCED HOPEFUL LOW THERAPEUTIC DEVELOPMENT AND I THINK THE CON SEX OF WORKING WITH A PARTICULAR PATIENT SAD ROW KA SEE GROUP WAS REALLY FOCUSED ON A SPECIFIC RESEARCH QUESTION WITH A DEFINED OUTCOME THAT COULD TIE TO A DECISION, RIGHT. OBVIOUSLY YOU DON'T KNOW WHAT THE OUTCOME IS GOING TO BE AT THE BEGINNING BUT YOU KNOW REGARDLESS OF OUTCOME YOU ARE IN A POSITION TO MAKE A DECISION AND THE PROJECT THAT WAS CO FUNDED, YOU KNOW WAS A TARGET IDENTIFICATION SCREENING AND FOR THE ADVOCACY ASSOCIATION, THE OUTCOME WOULD GIVE THEM CONFIDENCE IN HOW THEY WERE GRANT FUNDING GIVEN A PARTICULAR MECHANISTIC APPROACH TO ADDRESSING THEIR CONDITION. FOR PFIZER, IT WAS YOU KNOW, DO WE PURSUE THIS MECHANISTIC APPROACH OR NOT? AND SO THERE WAS CLARITY ON WHAT WAS THE RESEARCH QUESTION AND WHAT DECISIONS COULD ENTITIES MAKE GIVEN THE OUTCOME AND I THINK WE WEREN'T ABLE TO IDENTIFY ANY HITS AND I THINK THE CONCERN THERE WAS HOW IS THIS GOING TO IMPACT THE RELATIONSHIP BUT WE ALREADY DISCUSSED THE POTENTIAL OUTCOMES SO FOR THE ASSOCIATION, THEY WERE VERY SATISFIED BECAUSE NOW THEY KIND OF COULD STRATEGICALLY REORIENT THEIR STRATEGY AROUND GRANT FUNDING, RIGHT, AND PHASE PFIZER HADCLARITY AND COULD REPE SOME PROGRAMS FOR THAT INDICATION AS WELL AND SO WAS IT SUCCESSFUL IN FINDING A HIT, NO, BUT IT WAS SUCCESSFUL IN ANSWERING THE RESEARCH QUESTIONS AND SUSTAINING THAT RELATIONSHIP BECAUSE AGAIN YOU WERE IN THERE ALIGNED WITH THE SHARED PURPOSE. >> GREAT, LAURA, ANY STORIES YOU CAN SHARE? >> WELL, I GUESS JUST GENERALLY I WOULD SAY THAT I THINK THERE ARE THESE PRACTICAL -- THE CONCEPT OF OUR NEED TO HAVE A STRONG RELATIONSHIP WITH THE COMPANIES IS SO PARAMOUNT AND SOMETIMES IT CAN BE FRUSTRATING WHEN IT DOESN'T WORKOUT AND I THINK THAT THERE ARE SO MANY DIFFERENT FACTORS AND INFLUENCES FROM THE PRACTICAL SUCH AS HOW MANY PATIENTS HAVE THIS DISORDER AND HOW MUCH MONEY MIGHT HAVE FOUNDATION CONTRIBUTE AND TO REALLY THESE INTANGIBLES LIKE INTERPERSONAL RELATIONSHIPS WHICH ARE HARD TO DEFINE. THEY CAN BE VERY IMPORTANT AND HAVING A CHAMPION WITHIN A COMPANY FOR YOUR DISORDER CAN BE REALLY, REALLY TRANSFORMATIVE BECAUSE AS A COMPANY LOOKED AT REPORT PORTFOLIO OF ALL THE DIFFERENT THINGS THEY'RE WORKING ON, HAVING A VOCAL AND COMPELLING CHAMPION WHEN YOU HAVE FIVE PROJECTS THAT ARE KIND OF HAVING TROUBLE AND THE COMPANY IS TRYING TO DECIDE WHERE TO DOU DEVOTE RESOURCES SO DEVELOPING THOSE PERSONAL RELATIONSHIPS WHICH IS EASIER SAID THAN DONE BUT I DO THINK THAT IS AN IMPORTANT COMPONENT TO CONSIDER. >> GREAT. THANK YOU. I'M GOING TO ENCOURAGE OUR AUDIENCE NOW TO START PUTTING THEIR QUESTIONS IN THE CHAT. AS WE WAIT FOR PEOPLE TO PUT THEIR QUESTIONS IN, I WANT TO ASK THE OTHER SIDE OF THE QUESTION I ASKED, WHICH IS, AGAIN, NO NEED FOR -- TO GO ABLE TO TAKE THE PROGRAM AND EITHER CONTINUE DEVELOPING AND DO TON YOUR OWN OR PLACE IT AND THE FATE OF THE PROGRAM IS NOT POSSIBLE AND NOT EVERY COMPANY IS HAPPY TO DO THIS BUT IT HELPS IF YOU ARE ABLE TO NEGOTIATE THIS SO THERE HAVE BEEN CASES WHERE THIS WAS AND DON'T HAVE ANY CONTROL OVER THE PROGRAM IF IT'S SHELVED AND PEOPLE DON'T WANT TO HAND IT OVER TO SOMEONE ELSE FOR COMPETITIVE REASONS AND IS IT JUST DIES A SLOW DEATH AND THAT'S REALLY THE WORST-CASE SCENARIO SO THE SECOND THING IF YOU ARE PARTNERING WITH A COMPANY TO TRY TO UNDERSTAND WHERE YOUR PROGRAM IS AND IN THEIR PIPELINE IS IT GOING TO BE THE FIRST OR SECOND OR THIRD PRIORITY OR IS IT GOING TO BE THE FIFTH OR 2:00 PLACE IN THE LIST AND NOW WHEN THE MARKET WAS GREAT A LOT OF PEOPLE HAD REALLY PIPELINES AND NOW WHEN TIMES ARE HARDER AND PEOPLE NEED TO GET BACK IF YOU ARE NUMBER 15 THEY ARE HIGHER WHEN THE TIMES ARE GREAT AND THERE'S A LOT OF RESOURCES AROUND. >> AGREE OF EVERYTHING. WHEN YOU THINK ABOUT RELATIONSHIPS FAILING, WHETHER IT'S BETWEEN AN ADVOCACY GROUP AND AN INDUSTRY OR HUSBAND AND WIFE, RIGHT, IT BREAKS DOWN AROUND COMMUNICATION AND MANAGING EXPECTATIONS, RIGHT. IF YOU ARE SOMETIMES OUR CHALLENGE IS NOT BEING DIRECTED AND NOT SAY HERE IS THE PRIORITY OF THIS PROGRAM AND THIS IS THE STAGE IT'S AT AND THIS IS WHAT WE NEED TO DEMONSTRATE TO MOVE IT FORWARD AND THIS IS WHERE IT SITS IN RELATIONSHIP TO RESOURCES ACROSS THE COMPANY AND WE MIGHT TEND TO SAY WE'RE COMMITTED TO THIS DISEASE AND WE'RE IN IT FOR THE LONG HALL AND WHEN AND THE OTHER PARTNER. >> LAURA DO YOU HAVE ANYTHING ELSE TO ADD TO THAT? >> WE'RE STILL WAITING ON QUESTIONS BUT THERE'S A QUESTION FROM DARLENE. SHE SAID AND I DON'T KNOW IF THERE'S SHOULD THEY FUND A SEPARATE MOUSE. >> YEAH, I THINK IT'S A GREAT QUESTION. FIRST TRY AND UNDERSTAND WHY HE IS NOT WILLING TO SEND OUT THE MOUSE AND MAYBE IT'S SOMETHING RELATED TO HIS PUBLICATION AND HE WANTS TO BE ON THE PUBLICATIONS OR HE WANTS FUNDING AND THERE'S ALWAYS THE REASON WHY THIS IS NOT WHAT PEOPLE ARE NOT WILLING TO SHARE AND TRY TO UNDERSTAND BETTER AND MAYBE ADDRESS THE SPECIFIC REASONS IF NOT IF THERE'S NO OTHER CHOICE AND NO OTHER MOUSE MODEL THEN YEAH THEY WOULD. YOU HAVE TO MAKE YOUR OWN MOUSE MODEL THAT TAKES TIME AND IT COST MONEY AND IT'S DEFINITELY WORTH HAVING CONTROL OVER A KEY COMPONENT OF ANY RESEARCH PLAN THAT YOU ARE GOING TO BE FUNDING. >> YEAH. THAT IS REALLY HARD. LAURA, YOU GUYS HAVE DONE A GREAT JOB. I KNOW YOU MENTIONED YOU HAVE A SUNSET DATE OF 2025, YOU GUYS HAVE DONE A REALLY GREAT JOB OF LEVERAGING FUNDING FROM OTHER SOURCES AND YOUR PARTNERSHIPS, CAN YOU TALK ABOUT THAT SUNSET PROVISION AND ARE YOU GUYS STICKING TO THAT? I MEAN YOU HAVE FOUR DRUGS NOW GOING INTO CLINICAL TRIAL BUT CAN YOU TALK A LITTLE BIT ABOUT HOW YOU LEVERAGED YOUR PARTNERSHIPS, ESPECIALLY IN THE FUNDING SIDE? >> SURE, I DO THINK IT UNUSUAL T OUR FOUNDERS WERE INTENTIONAL IN IT BECAUSE IT FORCES US TO BE HIGHLY FOCUSED AND I THINK TO MAKE SOME -- I THINK LAURA FROZE. HOPEFULLY SHE CAN COME BACK. >> YES, EVERYONE ELSE SEEMS FINE. >> OK. >> AS WE WAIT FOR LAURA TO COME BACK, LET'S JUST MOVE ON TO THIS QUESTION ON WHAT CAREGIVERS AND PATIENTS CAN DO TO ADVANCE SCIENCE OUTSIDE OF FUNDING FOUNDATIONS. TIME IS MONEY TO INDUSTRY AND I THINK THAT WE HAVE INDIVIDUALS AND SHE'S REPRESENTING ALS AND THEY HAVE A GREAT DEAL TO OFFER TO INDUSTRY THAT CAN HELP THERAPY DEVELOPMENT MOVE FORWARD FASTER. I THINK CATHERINE, YOU MIGHT BE ABLE TO ANSWER THAT QUESTION. >> YOU KNOW, IT DOESN'T HAVE TO BE A FUNDING AND IT'S TO IDENTIFY WHAT ARE SOME OF THE BARRIERS THAT ARE IN PLACE TO DRUG DEVELOPMENT AND MAYBE IT'S AN ISSUE OF ACCESS TO DATA RIGHT AND I THINK THAT THE QUESTION WAS ACCESS TO A MOUSE MODEL, WELL, YOU KNOW, I THINK WE ALL EXPERIENCE CHALLENGES WHERE SOME DATA IS SILOED AND IT'S REALLY HARD TO ACCESS IT AS AN INDUSTRY SPONSOR AND WHEN WE HAVE TO UNDERSTAND WHAT IS NATURAL HISTORY AND SO ARE THERE THINGS THAT YOU AS AN ORGANIZATION CAN BE DOING THAT ENABLED THAT IN YOU ARE JOINING CO CONSORTIA SOT DOESN'T HAVE TO BE SPECIFICALLY FUNDING BUT A GOOD SENSE OF SOME OF THE EXISTING CHALLENGES IN THE RESEARCH DEVELOPMENT SPACE FOR YOUR COMMUNITY. >> GREAT. LAURA WE LOST YOU FOR A LITTLE BIT. I DON'T KNOW WHERE ENDED. >> I'M SORRY. WORKING WITH OTHER FOUNDATIONS IF YOU HAVE THEM IN YOUR SPACE IS GREAT. THAT WAS MY POINT. LET'S TAKE THIS QUESTION FROM SARAH FROM THE SPINA BIFIDA ASSOCIATION. AND SHE SAID OUR ORGANIZATION PUBLISHED OUR FIRST COMMUNITY AND THEY WANT ANSWERED AND IT'S CONSIDERED AND COME SHOULD PATIENT GROUPS DO FIRST? DO YOU WANTED TO RESTATE THAT FOR THIS? >> I'M SORRY, I THOUGHT LAURA WAS ADDRESSING THAT QUESTION. >> ANYONE. LAURA, YOU CAN. I THINK IS MAYBE A LITTLE UNDER APPRECIATED AND UNDERSTANDING THE SINCE IS OF COURSE THE FOUNDATION OF ANY DRUG AND IT'S EASIER TO ADDRESS AND ALTHOUGH WE CERTAINLY FEEL LIKE WE HAVE DONE A LOT TO INFLUENCE DRUG DEVELOPMENT IN THE SPACE WE REALIZE BECAUSE OF THE MECHANISM OF DISEASE MUTATIONS CAUSE A DEFICIENCY SO IT'S STRAIGHT FORD THERAPEUTIC APPROACH INDIVIDUALS HAD HALF THE AMOUNT OF NORMAL THAN THEY SHOULD SO IT'S A REPLACEMENT STRATEGY AND INCREASING REALLY VERY STRAIGHT FORWARD AND HOPEFULLY, RIGHT AND I THINK THAT IT HAS BEEN ENORMOUS BENEFIT AND SO I THINK SOMETIMES UNFORTUNATELY, IT'S JUST A LONGER STRUGGLE IF IT'S NOT A CLEAR STRAIGHT FORWARD THERAPEUTIC APPROACH OR IF THERE'S NOT AS MUCH BASIC SCIENCE UNDERSTOOD ABOUT THE MECHANISM OF DISEASE AND THESE THINGS TAKE TIME. FUNDING BASIC SCIENCE AND DISCOVERY RESEARCH AS NECESSARY TO HELP MAKE CLEAR PER THERAPEUTIC APPROACHES. >> WE HAVE ANOTHER QUESTION HERE. WE HAVE A QUESTION AT END POINTS AND DO YOU HAVE ANY SUGGESTIONS ABOUT HOW TO PER WAYED INDUSTRY TO ACCEPT CLINICAL END POINTS WITH PATIENTS AND CAREGIVERS, OTHER THAN WHAT THEY, THE INDUSTRY MIGHT BELIEVE ARE THE APPROPRIATE END POINTS. >> YES, THERE'S A BALANCE AND A DISCUSSION BETWEEN THE END POINTS THAT ARE MEANINGFUL FOR PATIENTS, RIGHT, AS DEFINED BY WHAT YOU HAVE TO DEMONSTRATE BY THE FDA AND HOW THEY CAN BE MEASURED AND THAT IS SOMETIMES A CHALLENGE. AND THEN THE LANGUAGE OF DATA ABOUT THE END POINT THAT THE FDA IS GOING TO ACCEPT. >> ASSUME CERTAIN RISKS IN ORDER TO GET A BENEFIT FROM AN INVESTIGATIONAL THERAPY AND THE TRADE OFFS IS THEY WILL USE PERHAPS, IN SOME OF THOSE CLINICAL END POINTS SO DOING SOME OF THAT STRUCTURED RESEARCH TO SAY, THIS IS WHAT THE COMMUNITY IS WILLING TO DO. RESPONSORS WILL LOOK AT THAT AND SAY OH MY GOSH, I HAVE NO IDEA AND IN TERMS OF PREFERENCES WHAT I'M PRO FOESING IS SO FAR AT THE BOTTOM OF THE LIST AND AND REGULATORS WILL SAY THE COMMUNITY IS SPEAKING AS A WHOLE AND WE CAN SEE A DIFFERENT RISK BENEFIT UNDERSTATED PATIENT PREFERENCE RESEARCH, RIGHT, SO KIND OF COUPLE POINTS TO THAT QUESTION. >> YOU COVERED EVERYTHING I WANTED TO SAY BUT THE PATIENT-FOCUSED MEETINGS ARE USEFUL FOR THAT SO IF YOU HAVE DONE YOUR RESEARCH AND YOU HAVE INVESTIGATED THE COMMUNITY ON WHAT END POINTS WOULD MATTER TO THEM AND WHAT THE RISK IS THEY'RE WILLING TO TAKE, WHEN YOU TALK TO THE FDA YOU BRING IT UP IN THESE PATIENT FOCUSED MEETING AND FAMILIARIZE THEM WITH YOUR LIMITS AS A PATIENT GROUP AND IT'S DIFFICULT BECAUSE WITHIN A PATIENT GROUP, THERE WILL BE FAMILIES THAT WILL TAKE MORE RISK THAN OTHERS SO THEY WILL ENROLL FIRST IN THE CLIP CAL TRIALS? PROBABLY. BUT WHAT HAPPENS WITH ALL THE OTHER FAMILIES THAT ARE NOT WILLING TO TAKE THAT RISK? IT'S A BIG DISCUSSION YOU ARE TALKING ABOUT WHICH IS ONE AND DONE AND YOU CAN'T REVERSE IT AND THE BENEFITS YOU GET WITH IT THERE MIGHT BE SOME OTHER THINGS THAT YOU GET WITH IT AS WELL BUT MAY NOT HAVE SIGNED UP FOR. YOU DID SIGN UP FOR BUT HOPING IT WON'T HAPPEN. >> WE HAVE JUST A COUPLE MINUTES LEFT. AND I HAVE TWO QUESTIONS FOR YOU, ONE, AND EACH OF YOU CAN ANSWER THIS, THE GREATEST MISCONCEPTION THAT YOU THINK A PATIENT GROUPS HAVE OF INDUSTRY AND INDUCE TREMENDOUS HAS I PATIENTS AND YOUR LAST TAKEAWAY YOU WOULD LIKE TO SHARE AND ADVICE YOU WOULD GIVE PATIENT ADVOCACY GROUPS SEEKING WITH STRONG RELATIONSHIP WITH PHARMA AND INDUSTRY. >> ANYONE CAN START. LAURA, WHY DON'T YOU START. >> I GUESS THE MISCONCEPTION WOULD BE PERHAPS I DO THINK THERE CAN BE A PERCEPTION OF PHARMA BEING SOLELY MOTIVATED BY MONEY AND ALTHOUGH OF COURSE THEY'RE A MONEY-MAKING ENTERPRISE THEY'RE VERY HUMAN SCIENTISTS WORKING WITHIN PHARMA WHO ARE DEDICATING THEIR LIVES AND WORKING VERY LONG HOURS TO HELP CREATE DRUGS TO HELP FIGHT DISEASES AND I THINK MUCH TIMES ONE TO ONE BUILT THESE RELATIONSHIPS WITH FARTHER SUED CAL SCIENTIST TO SEE THEIR LEVEL OF DEVOTION AND THE WORK THAT THEY DO IS REALLY GRATIFYING AND I THINK FROM THE SECOND QUESTION ABOUT WHAT IS THE BIGGEST ADVICE, I THINK NOT TO UNDER VALUE YOUR OWN CONTRIBUTIONS AND WHAT YOU CAN DO AND TO GET STARTED AS EARLY AS POSSIBLE ON CREATING PATIENT REGISTRIES AND. >> IT COMES FROM THE PATIENT GROUPS THAT THIS IS DEFINITELY CHANGING AND PEOPLE LIKE MYSELF, WE'RE A SMALL COMPANY AND WE ALL KNOW THE VALUE OF WHAT THE PATIENT GROUP HAS BEEN THROUGH THE TABLE BUT A BIG COMPANY LIKE PFIZER HAVING A PERSON LIKE CATHERINE, AND OBVIOUSLY SHE UNDERSTANDS THE VALUE AND SHE IS THE SEG WAY INTO THE COMPANY IN MANY CASES AND IT'S THE GREAT JOB THAT EACH AND EVERY ONE OF YOU ARE DOING IN MAKING SURE WE UNDERSTAND WHAT EXPERTISE YOU BRING TO THE TABLE. >> LAST ADVICE, KATHRYN. >> OH MY GOODNESS, I WOULD SAY, I'M STILL REFLECTING ON THE MISCONCEPTIONS AND AGREE THAT INDUSTRY TENDS TO UNDER APPRECIATE THE EXPERTISE IN THE PATIENT COMMUNITY AND IN REVERSE, THERE'S PROBABLY A MISCONCEPTION AROUND INDUSTRY THAT WE'RE KIND OF WITHHOLDING INFORMATION AND WE KNOW THAT THE PATH WAY WITH CERTAINTY IN DEVELOPING A DRUG AND THAT'S JUST NOT THE CASE BECAUSE YOU KNOW, THE INTEREST IS REALLY EMPLOYING INNOVATIVE MECHANISMS AND DRUGS WHERE THE PATH ISN'T MAPPED OUT FOR US AND WE'RE ALL KIND OF LEARNING AS WE GO AND APPLYING OUR EXPERTISE BUT IT'S NOT SOME MAGIC BULLET THAT WE'RE HOLDING ON TO AND NOT PAYING ATTENTION TO. THANK YOU FOR THIS WONDERFUL INSIGHTFUL DISCUSSION. THANK YOU FOR ATTENDING OUR SESSION AND WE LOOK FORWARD TO THE NEXT. THANK YOU, EVERYONE. >> THANK YOU ALL SO MUCH FOR MODERATING THAT FANTASTIC SESSION. SO, WITH THAT, I AM GOING TO TAKE US INTO OUR NEXT SESSION WHICH IS HOW TO FIND INNOVATIVE METHODS TO FUND RESEARCH IN AN EFFORT TO SUPPORT SCIENCE AS A SMALL BUSINESS SO THIS SESSION WILL BE MODERATED BY OUR NON-PROFIT FORM EXECUTIVE ME GERALDINE BLISS. THE PRESIDENT AND CO-FOUNDER OF CURESHANK, A NON-PROFIT ORGANIZATION WITH ACCELERATED DRUG IN PMS AND RELATED SHARINGK DISORDERS. SHE SERVED ON THE BOARD OF DISTRICTORS FOR TWO YEARS AND RESEARCH CHAIRPERSON FOR SEVEN. AND THAT ROLE SHE CHAIRED THE FIRST SCIENTIFIC STRATEGIC PLANNING INITIATIVE SUCCESSFULLY A REPLIED FOR GRANTS TO FURTHER PMS RESEARCH SUPPORT INITIATIVES AND ORGANIZED SIM POSIA IN 2012, 14 AND 18 A MEETING IN '13 AND MULTIPLE WORKSHOPS ON PMS AND THE 2016 ASK 2018 AND FAMILY CONFERENCE RESEARCH SESSION THIS IS 2010, 2012 AND 2014 AND ESTABLISHED A PMS RESEARCH REPOSITORY PARTNERSHIP WITH AUTISM BRAIN AND THE NINH AMONG MANY OTHER ACCOMPLISHMENTS I'M NOT SURE WHEN SHE SLEEPS AND SHE HAS SERVED AS A LAY REVIEWER FOR THE FDA AND HAS A BACHELORS FROM TRINITY AND MASTER FROM THE UNIVERSITY OF HOUSE ON AND HER PERSONAL MISSION IS TO HAVE CURES FOR PMS, SHE AND HER HUSBAND BRAD HAVE TWO SONS, CHARLES AND NATHAN AND CHARLES DOES HAVE PMS CAUSED BY THE SHANK 3 GENE SO WITH THAT, PLEASE TAKE IT AWAY FOR OUR NEXT SESSION. >> GREAT, THANK YOU FOR THAT NICE INTRODUCTION. FOR THOSE OF YOU WHO PARTICIPATED IN THE NINDS NON-PROFIT FORUM LAST YEAR, YOU MIGHT REMEMBER ALISON WHO GAVE A TALK ABOUT THE FOUNDATION FOR PMS THERAPEUTICS AND THE COMPANY THEY STARTED CALLED GENETICS AND THEY ULTIMATELY PARTNERED WITH YOU WILL INTEREST GENICS TO DEVELOP A ASL TREATMENT FOR PATIENTS AS YOU JUST HEARD MENTIONED IN THE LAST PANEL, YESTERDAY, ULTRA-AGA IT WAS ACQP FRONT PLUS ROYALTY PAYMENTS SO IT'S A REALLY EXCITING OUT AND AND JUST REALLY SUPER EXCITING FOR THE COMMUNITY. SO TODAY, WE'RE GOING TO CONTINUE THIS DISCUSSION ABOUT HOW NON-PROFIT GROUPS CAN SUPPORT SCIENCE AS A SMALL BUSINESS AND HOW TO FIND INNOVATIVE METHODS TO FUND RESEARCH AND OUR FIRST SPEAKER IS JILL WOOD FROM PHOENIX NEST. JILL IS THE FOUNDER AND C.E.O. PHOENIX NEST WHICH IS THE BIOTECH COMPANY FOCUSED ON TREATING THE SYNDROME AND YOU MIGHT KNOW JILL AS THE MOM TO JONAH AND THE FOUNDER OF JONAH JUST BEGUN A NON-PROFIT ORGANIZATION TO RAISE AWARENESS IS SUPPORT FOR INDIVIDUALS. JILL WILL TALK TO US ABOUT PHOENIX NEST AND HER SUCCESS AND OBTAINING SMALL BUSINESS FUNDING FROM THE NINDS. CAN WE GO AHEAD AND GET JILL STARTED, THEN. >> I UNMUTED MYSELF. I'LL TAKE IF FROM HERE. THANK YOU FOR THAT INTRODUCTION. AND THANK YOU FOR HAVING ME HERE TODAY. I AM JILL WOOD AND I AM THE C.E.O. OF PHOENIX NEST AND I AM JONAH'S MOM AND MY SON JONAH HAS TYPE C YOU MIGHT HEAR ME REFER IT AS SAM C AND I'LL REFEX TO P PHOENIX NEST AS PN. I'LL GIVE YOU A BACKGROUND ON HOW PN CAME TO BE AND WORKING WITH THE NINDS TO SUPPORT PHOENIX NEST PROGRAMS. SO, MY OBLIGATORY STACKS AND THERE ARE FOUR DIFFERENT SUB TYPES CAUSED BY MUTATION FOR DIFFERENT GENES AND THERE ARE THE INCIDENTS RATES ARE ARBITRARY NO ONE REALLY KNOWS AND THE TRUE INCIDENTS OF SANPHILLIPPEO, THEY'RE RARE AND TYPE C AND D BEING THE RAREST TOGETHER THEY'RE OCCUR 1/70,000 PATIENTS. BUT WHAT WE DO KNOW ABOUT SANFILIPPO IT'S OF A STORAGE DISEASE BECAUSED BY A SINGLE GENE DETECT AND NO APPROVED TREATMENTS FOR THE SUB TYPES. SAN PHILLIPPE OWE WE STARTED WIS JUST BEGUN AND WE DID CLOSE OUT AND SENT OUR FAMILIES AND SUPPORTERS THE SANFILIPPO FOUNDATION SO I CAN FOCUS ON PHOENIX NEST. WHEN JOE NA WAS DIAGNOSED WE FOUND THERE WAS NO TREATMENTS AND THERE WAS NO ONE EVEN WORKING ON A GRAHAM FOR SAN SO WE CREATED JJB AND FOUND OUR RESEARCHES AND CREATED A MOUSE MODEL AND JOINED FORCES WITH THE FAMILIES AROUND THE WORLD. TOGETHER WE CREATED A TYPE C SPECIFIC CONSORTIUM HAND H.A.N.D.S. AND PERFORMED PRE CLINICAL RESEARCH AND FOUNDED THE FIRST AND ONLY GLOBAL PATIENT REGISTRY CONNECT MPS. I DON'T HAVE TO TELL THE FAMILIES JUST HOW HARD IT IS TO START FROM SCRATCH BUT FOR THOSE OF YOU THAT DON'T KNOW, DOING THIS AND GOING THROUGH TH THIS JOURNEY WILL BE THE HARDEST JOB OF YOUR LIFE. NOTHING IS HARDER THAN WATCHING YOUR CHILD DIE. I'VE WILL BE DOING THIS FOR 12 YEARS NOW AND IF THINGS GO PERFECTLY, I MIGHT BE IN THE CLINIC IN ANOTHER FOUR YEARS. MY SON WILL BE 18 BY THEN AND I KNOW THAT HE MIGHT NOT MAKE IT. I DO KNOW THAT JONAH'S LEGACY WILL LIVE ON IN THE LIVES OF CHILDREN THAT HE HAS SAVED. PHOENIX NEST IS A TWO-MAN SHOW THAT'S MY PARTNER IN CRIME THERE WHO I HAVE TO GIVE CREDIT FOR A LOT OF OUR GRANT WRITING EFFORTS TO AND I CREATED PHOENIX NEST AFTER WATCHING THE TRIALS AND THE TRIBULATIONS THAT THE SAM A AND SAM B GROUPS WERE GOING THROUGH WITH THEIR TREATMENTS THEY HAD GONE TO THE CLINIC AND JUST REALLY HAD NOT FOUND THE CORRECT END POINTS AND GOTTEN ACROSS THE FINISH LINE. SO, I REALIZED THAT BECAUSE OF THE RARITY OF S SANFILIPPO WE HA SPONSOR PICK US UP AND I CO FOUNDED PHOENIX NEST IN 2012 TO HELP BRING THE TREATMENTS THAT ARE NON PROFITS FUNDRAISED TO THE CLINIC. WE HAVE THREE DRUG PROGRAMS AND TWO NATURAL HISTORY STUDIES IN OUR TYPE LINE. WHAT WE CAN'T DO IN-HOUSE WE USE THE EXPERTISE OF OUR CONSULT APARTCONSULTANTSAND CONTRACTORS. SO SOME OF THE PROS AND CONS OF WRITING SBR GRANTS ARE THE PRIMARY FOCUS OF THE NIH GRANTS THAT WE WRITE GRANTS FOR AND A EMPLOYING FOR GRANTS IS EXTREMELY ARDUOUS AND HEARTBREAKING. YOU PUT SO MUCH INTO WRITING THESE GRANTS AND IT TACKS A LONG TIME. AND SOMETIMES YOU DON'T WIN. ACTUALLY A LOT OF TIMES YOU DON'T WIN. IT'S HEARTBREAKING. YOU CAN COME INTO A LOT MORE PROBLEMS TOO WITH IF YOU ARE FUNDING IS FROZEN AND YOU DIDN'T MAKE A MILESTONE AND IF THERE'S A GOVERNMENT SHUT DOWN, THERE'S ALL KINDS OF THINGS THAT CAN HAPPEN TO YOU ON THIS ROAD OF BEING FUNDED BY NIH GRANTS BUT FOR THOSE OF YOU WHO KNOW HOW HARD IT IS TO DO GRASSROOTS FUNDRAISING I TAKE THE WRITING THE GRANTS ANY DAY. ALSO, IN ORDER TO GET YOU TO THE POINT WHERE YOU CAN WRITE OR WIN A GRANT, YOU MUST GO THROUGH THIS LONG LIST OF LONG-TO DO LIST OF APPLYING AND REGISTERING FOR ALL THESE INSTITUTIONS. AND EACH REGISTRATION IS CONTINGENT ON THE INSTITUTION ACCEPT YOU IN THE PROCEEDING LIST OF THINGS TO DO IF THAT MAKES SENSE. THEY ALL FLOW TOGETHER. YOU CAN'T HAVE ONE WITHOUT ANOTHER AND IT TAKES A REALLY LONG TIME TO GO THROUGH ALL OF THIS. GIVE YOURSELF FOUR TO SIX MONTHS. THIS IS OUR GRANT HISTORY AND WE GO FOR THE SBIR GRANTS AND WHICH SIDE AM I ON? THEY HAVE LONG COMPLICATED NUMBERS HERE WHICH REPRESENT THE TYPE OF GRANT THAT YOU'VE GONE FOR. AND ALMOST IN CLOSING HERE, HERE IS MY FAVORITE SLIDE. I KNOW IT'S SUPER BUSY HERE BUT HERE IS AN OUTCOME OF A GRANT WE'RE WORKING ON RIGHT NOW AND CALLED FROM THE COMMERCIALIZATION READINESS PILOT PROGRAM GRANT WHICH I HOPE IS NOT GOING TO JUST BE A PILOT THAT IT WILL BE AROUND TO STAY. THIS IS THE THIRD IN A SERIES OF GRANTS AND THIS GRANT IS FOCUSED ON A NATURAL HISTORY STUDY FOR TYPE D, I THINK IN OUR LAST CONVERSATION WE HAD A LOT OF GOING ON AROUND NATURAL HISTORY STUDIES AND THE PORT APPS OF NATURAL HISTORY STUDIES AND I CAN'T ECHO THIS ENOUGH, ESPECIALLY FOR THE TALL RARE ULE DISEASES WHERE THERE'S NOTHING. IN THE SPACE OF SANFILIPPO AND I CANNOT WAIT TO KICK IT OFF AND SEE THIS GO LIVE AND IN ACTION. THERE'S -- WE ARE THE LAST TYPE C AND D ARE THE LAST TO BE GOING TO THE CLIP I CAN HERE AND THAT IS KIND OF A SILVER LINING WHEN YOU HAVE ARE THE LAST, YOU GET TO SEE WHERE THE OTHER SPONSORS MAY HAVE NOT NECESSARILY MESSED UP BECAUSE YOU NEVER KNOW WHEN YOU START THERE BUT GAPS. THIS NATURAL HISTORY STUDY WILL FILL IN THE GAPS. WE HAVE DESIGNED AN APP WHICH IS EXACTLY WHAT IT SOUNDS LIKE. AN APP YOU CAN DOWN LOAD ON TO YOUR PHONE AND THE FAMILY WILL BE GIVEN SEVERAL TASKS THAT ARE SANFILIPPO-RELATED AND THESE TASKS WILL ECHO WHAT IS HAPPENING IN THE CLINIC BUT IN THE CLINIC, YOU DON'T GET A GOOD SENSE OF THE CHILDREN, WHERE THE KIDS DO NOT COOPERATE AND THEY WALK INTO THE CLINIC AND THEY RUN FOR THE HILLS, THEY'RE OUT THE DOOR. THAT'S THE KIDS THAT CAN RUN. WE'RE GOING TO HAVE FAMILIES GO THROUGH AND DO IN A VERY STANDARDIZED FORM SAT, CONTROLLED FORMAT, GO THROUGH AND VIDEOTAPE SEVERAL ACTIVITIES THAT REVOLVE DAILY LIVING ACTIVITIES AND WE ARE ALSO DESIGNING OUR NEW CLINICAL OUTCOME ASSESSMENTS AND I DON'T KNOW IF WE'VE TALKED ABOUT CLINICAL OUTCOME ASSESSMENTS IF PEOPLE ARE AWARE OF WHAT CLIP CAL OUTCOME ASSESSMENTS ARE BUT IF YOU ARE STARTING OUT, I REALLY ENCOURAGE YOU TO GET A GOOD FEEL OF WH WHAT THESE MEASUREMENT RIGHT SIDE AND WHAT IS APPROPRIATE FOR OUR PATIENT POPULATION. I SEE ON THE CHAT PEOPLE ARE ASKING ABOUT END POINTS AND THE IMPORTANCE OF END POINTS AND YEAH I CAN'T BEHIND THAT ENOUGH, YOU ARE ON THE RIGHT TRACK ASKING THOSE QUESTIONS AND IT'S PERFECT TIMING THE FDA JUST CAME OUT WITH A DRAFT GUIDANCE FOR PATIENT-FOCUSED DRUG DEVELOPMENT AND SELECTING AND DEVELOPING CLINICAL OUTCOME ASSESSMENTS SO THIS IS TIMELY FOR THIS CONVERSATION AND I FEEL LIKE I JUST SPED THROUGH 12 YEARS OF MY LIFE IN EIGHT MINUTES AND I HOPE IT WAS HELPFUL. THE FAMILIES ARE THE FUEL THAT KEEPS ME GOING AND TO OUR WONDERFUL PROGRAM OFFICERS JILL AND EMILY WHO YOU WILL MEET NEXT. THANK YOU AND I LOOK FORWARD TO THE QUESTIONS AND THE ANSWERS. >> IT'S AMAZING THAT YOU HAVE BEEN ABLE TO ACCOMPLISH IT AND IT'S CREDIBLE YOU HAVE ADDED $10 MILLION OF FUND TO GO SANFILIPPO RESEARCH AND SUFFICIENT THIS CLINICAL OUTCOMES CEASEMENT PROJECT FOR NERLY THREE AND A HALF MILLION DOLLARS AND IT'S INCREDIBLE. AS JILL MENTIONED, THE WORK THAT THEY'VE BEEN ABLE TO ACCOMPLISH HAS BEEN FUNDED THROUGH THE NINDS RESEARCH TRANSFER PROGRAM AND THE PROGRAM DIRECTOR IN THE DIVISION OF TRANSLATIONAL RESEARCH AT NINDS IS Dr. EMILY AND SHE JOINED FROM INDUSTRY BYWAY OF THE DEPARTMENT OF DEFENSE AND SHE'S A FOUND I REMEMBER OF A SMALL BUSINESS HERSELF SO SHE WILL TALK ABOUT THE NIH SMALL BUSINESS PROGRAMS WHICH HAPPEN TO BE THE LARGEST SOURCE OF SPECIFICALLY TO SUPPORT SMALL BUSINESS RESEARCH AND DEVELOPMENT. >> THANK YOU SO MUCH. I THINK I HAVE CONTROL OVER THE SLIDES AND I'LL WALK THROUGH AND IT'S A PLEASURE TO FOLLOW OUR PROGRAM THAT IS VERY PROUD OF THE SUPPORTING HER AND HER EFFORTS AND REALLY I THINK SHE HAS BEEN SO SUCCESSFUL WITH SMALL BUSINESS FUNDING AND SO TODAY I'M GOING TO TALK A LITTLE BIT ABOUT WHAT IS IT LIKE TO APPLY FOR BRAVE SOULS WHO WANT TO FOLLOW JILL DOWN THE PATH OF STARTING A SMALL BUSINESS OF THERAPEUTIC DEVELOPMENT AND EDUCATE THE COMMUNITY, THE NON-PROFIT COMMUNITY ABOUT WHAT SMALL BUSINESS FUNDING GRANTS ARE LIKE WHAT'S OUT THERE AND SOME WAYS THAT NON PROFITS CAN HELP THESE EARLY STABLE SMALL BUSINESSES AND ACHIEVE THEIR GOALS. OK, LET'S DO IT. SO WHAT IS SBIR AND STTR THIS ACRONYM WE'RE REFERRING TO THIS REFERS TO CONGRESSIONAL REVIEW DATED SMALL BUSINESS PROGRAMS AND THE SMALL BUSINESS INNOVATION RESEARCH PROGRAMS AND THE SMALL BUSINESS TECHNOLOGY TRANSFER PROGRAM. BOTH OF THESE PROGRAMS REQUIRED THAT FEDERAL FUNDERS LIKE NIH SEND THEIR FUNDING DOLLARS ON SMALL BUSINESSES AND THERE ARE SOME SMALL DIS TANKION BETWEEN WHAT MAKES AN SBIR VERSUS AN STTR IT HAS TO DO WITH PARTNERING WITH THE UNIVERSITY AND I'M NOT GOING TO DO INTO IT TODAY AND IT'S NOT RELEVANT TO UNDERSTAND THE BASICS BUT IF YOU ARE INTERESTED IN A EMPLOYING FOR THIS FUNDING I'LL WALK THROUGH WHICH MECHANISMS MAKE THE MOST SENSE. THE PORTAGE THING TO UNDERSTAND THESE ARE GRANTS AND THEY'RE NOT LOANS SO YOU DO NOT NEED TO PAY THIS MONEY BACK. IT'S GRANT FUNDING SO YOU ARE NOT LOSING ANY PERCENT OF OWNERSHIP OR EQUITY IN YOUR COMPANY AND SMALL BUSINESS AWARDS, ESPECIALLY I WITH SAY NIH AWARDS WHICH GO THROUGH IT RIGOROUS PEER REVIEW PROCESS ARE RECOGNIZED AS BEING A GOLD STAR ON YOUR COMPANY AND IT CAN BE USED TO HELP LEVERAGE ADDITIONAL WE SUPPORT THE VERY EARLY STAGE OF RESEARCH TYPICALLY MAYBE RYE AFTER A LICENSING AGREEMENT HAPPENED WITH THE UNIVERSITY OR A A VERY EARLY PROTOTYPING STAGE AND WE'LL FUND THROUGH EARLY STAGE CLINICAL TRAILS AND REALLY OUR GOAL IS TO HELP DERISK THAT TECHNOLOGY AND TO A POINT WHERE IT'S EITHER READY FOR PRIVATE FUNDING AND MUCH MORE LARGER DOLLAR AMOUNT THAN WE'RE ABLE TO PROVIDE FOR GRANT FUNDING OR FOR SOME TECHNOLOGIES LIKE AS SAY OR RESEARCH TOOLS THEY MAY GET TO THAT POINT OF BEING COMMERCIAL READY THROUGH GRANT FUNDING SO WHAT DOES IT TAKE TO BE ELIGIBLE FOR SBIR AND STTR FUNDING, WE HAVE TO BE ABLE FORMED AND REGISTERED U.S.-BASED SMALL BUSINESS AND IT HAS TO BE A FOR PROFIT BUSINESS AND IT'S WISE TO START THOSE FOUR MONTHS BEFORE YOU PLAN ON APPLYING BECAUSE YOU ARE BUSINESS HAS TO BE INCORPORATE AND REGISTERED BEFORE YOU APPLY. THE COMPANY NEEDS TO BE LESS THAN 500 PLOY SKIS THIS CRITERIA IS NOT HARD THE COMPANIES ARE MAYBE FIVE OR SO PEOPLE AND IT'S PROBABLY ABOUT AVERAGE AND IT NEEDS TO BE U.S. INDIVIDUAL OWNED. MULTIPLE U.S. INDIVIDUALS AND IT NEEDS TO BE MAJORITY OWNED BY U.S. INDIVIDUALS. THERE'S A LOT OF LEGAL EASE BEHIND THIS DEFINITION AND I'M NOT GOING INTO THIS TIME BUT THIS INFORMATION IS PUBLIC AND I'M HAPPY TO WALK THROUGH ANY QUESTIONS FUR NOT QUITE SURE WHETHER YOU ARE -- IT'S GOING TO BE UP TO THE BUSINESS TO CERTIFY THAT YOU ARE ELIGIBLE FOR SMALL BUSINESS FUNDING AT THE TIME THAW RECEIVE YOUR AWARD. SO AT NINDS THE AMOUNT OF FUNDING DEDICATE TODAY SMALL BUSINESS IS OVER $80 MILLION A YEAR AND IT SUPPORTS ALL OF THE INDICATIONS WITHIN THE MISSION SPACE AND SO THAT IT INCLUDES LARGE INDICATIONS LIKE STROKE AND ALSO MANY OF THE RARE DISORDERS THAT ARE REPRESENTED HERE TODAY AND WHICH PHOENIX NEST IS WORKING TO SUPPORT. WE HAVE MODALITIES OF TECHNOLOGIES SO ABOUT PROBABLY OVER HALF OF OUR PORTFOLIO IS SMALL MOLECULES OR ANNA RARE OF DIFFERENT BIOLOGICAL APPROACHES TO GENE THERAPY, THEY WILL THERAPY AND ET CETERA A REALLY BROAD SCOPE THERE BUT WE FUND DIAGNOSTICS, RESEARCH TOOLS, BIOMARKERS, DEVICES, ANYTHING THAT WILL MAKE ITS WAY THROUGH THE COMMERCIALIZATION PATH TO BE A PUBLIC THAT WILL HELP BENEFIT THE PATIENTS AND RESEARCH COMMUNITIES THAT NINDS SUPPORTS. THIS STAGE OF RESEARCH CAN BE BROAD AS WELL SO WE SUPPORT THE VERY EARLY STAGE WORK IN THE TRANSLATION ALDO MAIN THROUGH IND TO BE READY FOR CLINICAL TRIALS IN THE FUND PHASE 1 AND EVEN PHASE 2 CLINICAL TRIALS UNDER OUR FUNDING. >> THERE'S A NEED TO DO FOREIGN BASED WORK AND IT CAN'T BE UNDER THE CLASS OF A SMALL BUSINESS OR SBIR AWARD. IT'S A FEE THAT GOES TO THE COMPANY AND IT CAN BE USED HOWEVER YOU LIKE BUT IT'S LIMITED AND I MENTIONED THIS BECAUSE ONE WAY WE FOUND THAT SMALL BUSINESSES REALLY STRUGGLING TO DEVELOP THEIR THERAPEUTICS IN THE EARLY STAGES CAN BENEFIT FROM ENGAGEMENT WITH NON PROFITS IS SMALL AMOUNTS OF FUNDING THE COMPANIES CAN BE BUT NOT GETTING THEIR PATENT FEES OR THEIR LICENSE FEES OR THEIR OTHER EXPENSES AND THESE ARE ESSENTIALS TO SECURING THE IP AND FOR THEIR TECHNOLOGY BUT THEY REALLY STRUGGLED TO FIND THAT FUNDING SO I'LL JUST MENTION THAT. AFTER YOU SUBMIT THAT APPLICATION IN AND AS JOE MENTIONED I'M NOT GOING TO SHY AWAY FROM IT IT'S AN OWNER US APPLICATION THEY'RE 90 TO 100 PAGES AND THEY'RE INTENTION AND AFTER YOU SUBMIT THAT IT'S GOING TO GO THROUGH PEER REVIEW AND THAT PEER REVIEW IS SIMILAR TO NIH TYPICAL NIH RESEARCH ACADEMIC RESEARCH PER REVIEW AND THESE PANELS DO INCLUDE MEMBERS OF INDUSTRY AND CLINICIANS AND OTHER PEOPLE WITH TRANSLATION ALEX PER TEASE. THE STANDARD THINGS HAVE YOU ON ANY RESEARCH ARE EVALUATED WITH THE ADDITIONAL AN ALLEY EVALUATION OF THE COMMERCIALIZATION POTENTIAL. PRODUCT AND I WANT TO STRESS HERE THAT IT'S A COMPETITIVE PROGRAM AND FUNDING PERCENTAGES HAVE HOVERED BETWEEN 15% TO 20% AND I WANT TO ACKNOWLEDGE THAT A LOT OF TIMES YOU ARE COMPETING AGAINST SMALL BUT ESTABLISHED BIOTECH COMPANIES AND SO BRIDGING IN A TEAM AND YOU WANT TO SHOW THAT YOU ARE A CAPABLE COMPANY AND YOU CAN TAKE THIS FORWARD TO THE POINT WHERE IT COULD BE FURTHER WHERE GROUPS CAN REALLY THOUGH SUPPORT SMALL BUSINESSES IS PROVIDING THOSE LETTERS OF SUPPORT AND IF YOU CAN PROVIDE LETTERS OF SUPPORT FROM PATIENTS AND FAMILIES THEMSELVES, FROM CLINICIANS AND WHO WORKED WITH AND OR IF YOU HAVE EARL STAGE CONVERSATIONS WITH PARTNERS HAVING THAT SUPPORT OF THEM, IT DOESN'T HAVE TO BE A COMMITMENT BUT STATING THEIR INTEREST SHOWS THAT COMMERCIALIZATION POTENTIAL OF YOUR TECHNOLOGY. AND ANOTHER ONE IN THIS ONE IS REALLY HARD AS JILL MENTIONED IT'S NOT UNCOMMON FOR APPLICATIONS NOT TO BE PURCHASED ON THEIR FIRST TRY YOU WILL GET A LIST OF COMMENTS WE'LL GET A LOT OF FEEDBACK AND I OR YOUR PROGRAM OFFICER WE CAN GO THROUGH HOW WE CAN STRENGTHEN THE PROPOSE AM AND IMPROVE THIS TO GET TO A FUNDABLE RANGE. I ALSO WANT TO NOTE OUR PROGRAM DOES HAVE ADDITIONAL SUPPORT PROGRAMS FOR NEW APPLICANTS SPECIFICALLY AND THE APPLICANT ASSISTANCE PROGRAM IS INTENDED TO PROVIDE GRANT-WRITING SUPPORT FOR A PHASE 1SBIR TO ANY COMPANY WHO HAS NEVER BEFORE RECEIVED NIH, SBIR OR STTR FUNDING AND IN BEING, WE HIGHLY ENCOURAGE DIVERSE APPLICATIONS TO A PLOY TO THIS PROGRAM DIVERSITY AND SMALL BUSINESS INCLUDES WOMEN-LED COMPANIES. IF YOU ARE INTRODUCED IN PURSUING SMALL BUSINESS FUNDING AND STARTING YOUR COMPANY AND PURSUING THIS PATH WOULD BE TO REACH OUT TO OUR TEAM IT'S HELPFUL TO HAVE A DESCRIPTION OF THE PROJECT YOU ARE THINKING ABOUT EVEN IF IT'S A DRAFT OR NOT CLEAR SO WE CAN SORT OF GET A SENSE OF WHERE YOUR READINESS IS AND WHAT WE SHOULD RECOMMEND BUT WE'RE HAPPY TO TALK WITH YOU AND OFTEN TIMES A LOT OF THE QUESTIONS ARE VERY SPECIFIC SO IF IT'S BEST TO JUST GET ON A CUL AND TALK THROUGH THAT. I'M GOING TO PAUSE THERE AND I'M HAPPY TO ANSWER ANY QUESTIONS. >> THANK YOU, EMILY. THAT WAS REALLY HELPFUL OVERVIEW. OUR NEXT SPEAKER IS KEN FROM SHANEER AND LUBITZ. IT'S A FIRM THAT REPRESENTS TAX INCENTIVE ORGANIZATIONS. WHETHER YOU ARE A PATIENT ADVOCACY GROUP CONTEMPLATING STARTING A COMPANY OR YOU ARE THINKING OF WORKING WITH AN ACADEMIC CENTER OR EVEN JUST THINKING ABOUT LIKE THE ABILITY TO SUB LICENSE RESOURCES OR AS SAYS YOU HELPED TO FUND LIKE THEY WERE DISCUSS NORTH THE PREVIOUS PANEL, YOU WILL WANT TO BE MINDFUL ABOUT PROJECTING YOUR 501C3 STATUS AND SO KEN WORKS WITH GROUPS AND HE HAS SERVED AS COUNCIL TO CYSTIC FIBROSIS IN THEIR TRANSACTION WITH -- HE IS OUTSIDE COUNCIL TO NON-PROFIT GROUPS ADVISING ON A WIDE RANGE OF ISSUES. ARE YOU READY? >> YOU ARE MUTED. >> ALL RIGHT, I'M UNMUTED. MAYBE I WOULD BE MORE INTERESTING MUTED. WHO KNOWS. THANK YOU FOR HAVING ME. I REPRESENT ABOUT 80 DIFFERENT DISEASE RELATED CHARITIES AND CYSTIC FIBROSIS IS ONE OF THEM, MICHAEL J. FOX -- A BUNCH. SOME BIG AND SOME SMALL AND MOST GIVE AWARDS TO COMPANIES TO DEVELOP PRODUCTS SO THE PURPOSE TODAY IS TO TALK ABOUT WHAT KIND OF AGREEMENTS THAT THEY, THE CHARITIES EXPECT FROM THE APPLICANTS AND WHAT FORMS THEY WILL TAKE SO LET ME SAY I'VE DONE THE CYSTIC FOUNDATION AND EVERYONE TALKS ABOUT IT AS THE MOTHER OF ALL VENTURE PHILANTHROPY AND WHEN TRANSACTIONS THAT WAS FIRST PIECE WAS IN 2002 AND THE COMPANY CALLED BIO SCIENCE AND THEY MADE THE FIRST AND WE DID SUPPLEMENTAL FUNDING WITH VER TEXT THAT ADDED UP TO $125 MILLION OR SO AND ULTIMATELY IT'S ALL PRODUCTS AND CYSTIC FIBROSIS FOUNDATION SOLD IT'S ROYALTY INTEREST UNDER THOSE AGREEMENTS AND REAPED ABOUT FOUR AND A HALF BILLION DOLLARS OF FUNDING, REALLY AN EXCITING AND SHOULD HAPPEN TO ALL OF US-TYPE EXPERIENCE AND ULTIMATELY THAT MONEY WILL ALLOW THEM TO CURE THE DISEASE. AND I THINK I PROBABLY HAVE DONE FIVE TO 600 OF THESE TRANSACTIONS AND ONE AND MAYBE THE TOTAL IN MAYBE COMING UP WITH A BILLION DOLLARS OF FUNDING WITH YOU DON'T GET SCARED BECAUSE A LOT OF THEM ARE SMALL, SMALL START UP COMPANIES AND THAT'S WHAT I WANT TO FOCUS ON TODAY BECAUSE THE FUNDING IN THOSE TRANSACTIONS CAN GO FROM 100,000 TO A MILLION AND I WANTED TO TALK ABOUT THE KINDS OF AGREEMENTS THAT YOU WILL ENTER INTO. SO LET ME START WITH THE FIRST ONE. AT THE BEGINNING, LEADING THE PACK, THE THOUGHT IT COULD BE A CONFLICT 20 INVEST IN STOCK AND EQUITY. SO MOST OF THE EARLY TRANSACTIONS AND A BUNCH OF THEM STILL ARE BASED ON ROYALTIES AND THE ROYALTIES FOR SMALL TRANSACTIONS ARE USUALLY NOT A PERCENTAGE OF NET SALES BUT THEY ARE A MULTIPLE OF THE AWARD AND SO IF AT WARD IS $100,000 AND THE DRUG A APPROVED, THE CHARITY WOULD GET BACK THREE TO 6X CALLED ROYALTY OR UPON FIRST COMMERCIAL SALE OF THE PRODUCT AND N ARE POTENTIAL MILESTONES WHERE THE CHARITY PARTICIPATES WITH THE COMPANY IN SUCCESS AT CERTAIN LEVELS SO IF AGGREGATE SALES REACH WHAT IS DEFINED IN THE AGREEMENTS AS A SUCCESS LEVEL, THEN THE CHARITY GETS AN EXTRA ONE TO TWO SO THOSE ARE NICE TO THINK ABOUT. NICE TO THINK ABOUT APPROVALS AND MOST OF TIME ON THESE TRANSACTIONS THE PAYMENTS OCCUR AT EXITS. THAT IS WHEN THE SMALL COMPANY SELLS ITSELF AND LICENSE IS THE PRODUCT SELLS ITSELF TO A LARGER PHARMA THAT HAS THE A LOT TO TAKE A PRODUCT TO MARKET. SO THE WAY THAT WORKS IS THE CHARITY IN SUCH CASES GETS A PERCENTAGE OF THE CONSIDERATION RECEIVED BY THE COMPANY UP TO THE ROYALTY CAP WHICH WE TALKED ABOUT WHICH WAS THE GREATEST SUCCESS. AND THE CHARITY IS THEN PAID OFF ITS AMOUNT AND THE COMPANY HOPEFULLY TAKES THE PRODUCT TO MARKET. AND I THINK THE PRIOR PANEL I SAT IN FOR THE TAIL END OF THE PRIOR PANEL, I ALSO INCLUDE WHAT I CALL AN INTERRUPTION LICENSE. THERE'S ALWAYS THE WORRY AND I'LL TELL YOU THE ONE DAY A LONG, LONG TIME AGO NOW, AFTER THE FIRST VER TEXT AND SO WHAT I PUT INTO ALL THESE AGREEMENTS NOW, THE COMPANY STOPS WORKING AND A PARTICULAR OF THE FUND THAT COMPOUND OR DIAGNOSTIC OR DEVICE AND THE LICENSE OCCURS AND THE COMPANY AND THE CHARITY TAKES OVER AND BRINGS IT TO MARKET. FROM A COMPANY'S STANDPOINT, ONE CAN, THERE CAN BE A LOT OF REASONS WHY THE COMPANY STOPS. THEY RUN OUT OF MONEY, ET CETERA. AND I THINK THE CHARITIES ARE SAYING IF THIS HAPPENS TO YOU, WE'LL SHARE REVENUE WITH REVENUE WITH YOU AT A CERTAIN SELF AND ROYALTIES I WOULD SAY OF THE 15 YEARS OR SO AND THEY'VE BEEN DOING THIS 20 YEARS, 20 YEARS. AND ROYALTY BASED AGREEMENTS WERE BUY AND LARGE THE MAJORITY OF THE FIRST 10 YEARS OR SO AND THEN CHARITIES THEY CAN BE DIRECT EQUITY. THIS SIDE IS OUT OF ORDER BUT THEY CAN BE DIRECTLY INEQUITY AND I'M GOING TO SWITCH TO -- THEY'RE OUT OF ORDER SO I'M GOING TO SWITCH TO THE NEXT ONE. IT ANTICIPATES A LARGER FUNDING FOR THROW YEARS AND THEY TAKE THE TAKE THE FORM OF A SAFE WHICH IS THE SIMPLE AGREEMENT FOR EQUITY OR CONVERTIBLE AND WHEN THE COMPANY DOES ITS LARGER FUNDING AND THE CONVERSION RATE IS AT A 10% TO 30% DISCOUNT OVER WHAT THE LARGER FUNDING PEOPLE WHO PARTICIPATED IN THE LARGER FUNDING PAY AND THE MAJOR DIFFERENCE BETWEEN A SAFE AND CONVERTIBLE NOTICE, THE CONVERTIBLE NOTE BEARS INTEREST USUALLY AT THE SIX TO 12% DEPENDING UPON THE NEGOTIATIONS AND THE SAFE DOES NOT I'M NOT CRAZY ABOUT SAVES FOR THAT REASON BECAUSE IT'S UNFAIR, I THINK, FOR THE CHARITIES THAT HAVE THE MONEY OUT THERE WITHOUT HAVING AN INTEREST ON THEM. SO, EQUITY AGREEMENTS THAT RELATE TO OFTEN HAVE A SIDE LIST SO THE CHARITIES ARE INVESTING IN A COMPANY THAT MAY HAVE MORE THAN ONE PARTICULAR PRODUCT AND WHILE THE CHARITY IS ONLY FOCUSING ON A PRODUCT THAT RELATES TO ITS DISEASE SO THE SIDE AGREEMENT REQUIRES THAT THE MONEY THAT THE CHARITY PUTS IN IS FOCUSED AND USED PRIMARILY FOR THE PARTICULAR DISEASE SO FOR EXAMPLE, IF I'M REPRESENTING NATIONAL KIDNEY WHICH IS A REALLY GOOD GROUP THAT DOES EQUITY FUNDING AND LET'S SAY IT INVESTS UP $200,000, IT WOULD REQUIRE THAT THE COMPANY USE ITS MONEY SOLELY FOR THE PARTICULAR THERAPY THAT THE NATIONAL IS FOCUSING ONMENT A COUPLE OTHER REASONS YOU WOULD FOCUS ON USE OF SIDE AGREEMENT WOULD BE MOST FAVORED NATION AGREEMENT SAYS THE CHARITY IS GETTING THE BEST DEALS AND SOMETIMES THE CHARITY LIKES AN OBSERVER SEAT ON THE BOARD OF THE DIRECTORS AND THE SIDE AGREEMENT REQUIRES THAT AND IF NEITHER OF THOSE POSSIBLE, THE CHARITY REQUIRES REPORTING AND IT'S PARTICULAR BECAUSE THEY'RE TAKING A RISK AND INVESTING AT THE EARLIEST STAGES AND THEY MAY WISH TO INVEST SUBSEQUENTLY AND YOU MAY WISH TO HAVE THEM INVEST SUBSEQUENTLY. SO I'M GOING TO SWITCH TO THE LAST SLIDE AND SO SOMETIMES THE CHARITY IS COMING IN AT A FUNDING ROUTE AND SO THERE WON'T BE THIS INTERVENING SAVOR A CONVERTIBLE NOTE THEY'LL INVEST DIRECTLY AND THERE WILL BE SHAREHOLDER AGREEMENTS THAT YOU THE COMPANY WILL BE ASKED TO PREPARE FOR THE CHARITY DOCUMENT REGISTRATION RIGHTS AND SALES AND TRANSFER RESTRICTIONS AND ET CETERA, ET CETERA. AND INCLUDING RIGHT TO PARTICIPATE IN FUTURE ROUNDS. AND SO THESE TOO ARE OFTEN THE CHARITY OFTEN REQUIRES THE SIDE AGREEMENT TO MAKE SURE THAT IT'S FUNDING WAS USED FOR THE TARGET PRODUCT. SO THAT'S ALL. THERE'S BEEN A COUPLE QUIZ ABOUT THE SLIDES. THEY'RE AVAILABLE AND I'M HAPPY TO SMALL COMPANIES WISH TO CALL ME TO CHAT WITH THEM ABOUT WHAT THEIR AGREEMENTS SHOULD SAY. SAY JUST WANT TO SAY I SPENT A 35 YEAR CAREER AND BILLING LAW AND REPRESENTING COMPANIES AND THAT WAS FINE AND I ENJOYED IT AND STARTED A FIRM WITH A FEW FRIENDS THAT GROUP TO 300 AND ET CETERA BUT THIS LAST 10 YEARS OF WORKING WITH DATA AND CHARITIES AND FUNDING THERAPIES AND DEVICES IN DIAGNOSTICS FOR DISEASE CHARITIES HAVE BEEN THE BEST 10 YEARS OF MY PRACTICE. THE DAY WE FOUND OUT THAT THE FIRST CF DRUG WORKED AND GOING TO MARKET, I'M NOT A SCIENTIST SO I COULDN'T SAY I HAD CREDIT FOR IT BUT IT WAS A GREAT POINT IN MY CAREER. SO THANK YO FOR HAVING ME. >> THANK YOU SO MUCH. WE HAVE A FEW MINUTES WE CAN TAKE SOME QUESTIONS RIGHT NOW IF ANYBODY HAS ANY QUESTIONS YOU ARE WELCOME TO JUST TYPE THEM INTO THE CHAT. >> I CAN BE REACHED AT THE WEBSITE. >> THANK YOU. >> KEN, HOW OFTEN DO YOU SEE A SITUATION SORT OF LIKE PHOENIX NEST? WHERE A PATIENT ADD CO KIT SAYS I'M GOING TO START A COMPANY. >> I SEE IT FAIRLY OFTEN AND SO WE DON'T -- WE GENERALLY REPRESENT THE FOUNDATION NOT THE COMPANY. WE GET INVOLVED SOMETIMES THERE'S STILL ACADEMIC GRANTS. THAT GO WAY BACK TO THE EARLY 2000s AND THE FIRST QUESTION CYSTIC FIBROSIS ASKED ME, NOT THE FIRST QUESTION BECAUSE I REPRESENTED YEARS BEFORE BUT ONE OF THE KEY QUESTIONS WAS COULD IT FUND INDUSTRY? COULD IT FUND THE COMPANY? AND UP TO THEN, MOST FOUNDATIONS WERE THOUGHT THEY COULD ONLY MAKE ACADEMIC GRANTS. THEY DIDN'T THINK THEY COULD FUND COMPANIES AND SO WE GOT PAST THAT ISSUE EARLY ON AND SEE HOW IT STARTED FUNDING COMPANIES AND NOW SOMEONE CAME UP WITH THE MAIN WHICH WAS QUITE CLEVER AND IT'S BECOME APP AREA THAT THE FOUNDATIONS HAVE FOCUSED ON. >> WE HAVE A COUPLE QUESTIONS. HOW LARGE DOES A CHARITY NEED TO BE TO MAKE THESE TYPES OF ATTEMPTS FEASIBLE? >> REALLY SMALL. I DON'T WANT TO PRETEND THAT WITH THE FUNDING WAS $25 MILLION. THERE'S NOT GOING TO BE MANY OF THOSE AND THE MILLION DOLLARS OR SO OF FUNDING MOST OF THEM IF YOU TOOK AWAY THE BIG BIG AMOUNTS, EVEN PFIZER AND FUND LILLY, ET CETERA AND THE REASON FOR THAT IS THERE'S A GUY WOULD WORKS FOR THEM AND WHO WANTS TO GO TO HIS WINDOWS AND SAY I HAVE THIS IDEA FOR A PROGRAM AND I CAN GET FUNDING FOR A CHEETAH THAT HELPS HIM JUMP START HIS OWN PROJECT AND MOST OF THE THESE FUNDINGS TAKE NATIONAL KIDNEY WHICH I'VE BEEN DOING SIX OR SEVEN DEALS FOR IT AND THOSE ARE BETWEEN 100,000 AND 250 AND I'LL DONE DEALS AS LOW AS 50,000 AND LET ME JUST SAY I ENJOYS THOSE JUST AS MUCH AND I HOPE THEY'RE SUCCESSFUL AND YOU KNOW, JILL'S STORY WAS FABULOUS. THERE ARE SMALLER DEALS WITH SMALL START UP COMPANIES THAT WORK FOR BOTH PARTS. >> ARE THERE ANY TYPES OF ININDUCE TREMENDOUS INVESTMENTS THAT YOU DO NOT RECOMMEND? WHO ORGANIZATIONS ENGAGE WITH? >> >> WELL, I DON'T THINK I -- OBVIOUSLY, FROM THE CHARITY STANDPOINT, THEY'RE LOOKING FOR THERAPY OR DIAGNOSTIC OR ADVICE THAT IS GREAT FOR THE PATIENTS THAT IS NOT AVAILABLE AND YOU ARE OBVIOUSLY LOOKING FOR A COMPANY THAT'S NOT GOING TO GO BROKE USE YOUR MONEY AND THEN GOT TO GO GET WHATEVER PROGRESS WILL BE LOST BY THE FACT THEY GO BANKRUPT SO SOMETIMES WHEN WE LOOK AT THE BALANCE SHEET THEY'RE THIN AND THEY'RE THIN SO I WOULD SAY IT'S MAINLY WITH PEOPLE WHO HAVE A GOOD IDEA WHO HAVE SOME EXPERIENCE AND CREDIBILITY AND ALL KINDS OF THINGS AND. >> ARE THERE ANY RESOURCES FOR PATIENT AT VOW KIDS OR ACADEMIC INVESTIGATORS CAN LEARN MORE ABOUT A EMPLOYING FOR SMALL BUSINESS GRANTS? >> YEAH, ABSOLUTELY SO I CAN PUT INTO THE CHATBOX THE SEED AT NIH WEBSITE AND THIS IS SMALL BUSINESS AND SUPPORT OFFICE FOR ALL OF NIH. THEIR WEBSITE GOES THROUGH ELIGIBILITY AND REQUIRED REGISTRATION AND FAQs AND HELP POINT YOU TO THE RIGHT FUNDING TICKETS TO THAT'S A GOOD PLACE TO RAISE BACKGROUND TO UNDERSTAND MORE ABOUT THE PROGRAM AND IF IT'S SOMETHING YOU WANT TO PURSUE YOU WOULD ADVISE HAVING A DISCUSSION WITH A PROGRAM OFFICER AND MYSELF OR CONTACTS CAN DIRECT YOU TO THE RIGHT PROGRAM OFFICER WE MAKE SURE THAT YOU ARE TALK TO GO THE PERSON THAT YOU WOULD BE WORKING IF YOU APPLY AND WE ASK FOR THAT PROJECT DESCRIPTION BUT WE'RE HAPPY TO TALK ABOUT THE SPECIFICS OF YOUR APPLICATION WHAT IT LOOKS LIKE MY DECISION IS THAT MOST OF THE APPLICATIONS YOU GET FOR SMALL BUSINESS GRANTS ARE COMING FROM THEIR COMPANIES THAT ARE BEING FORMED BY ACADEMICS LABS IS THAT CORRECT? WHAT IS THE DIFFERENCE? >> >> YEAH, THEY TAKE A LOT OF DIFFERENT FORMS BECAUSE THE ELIGIBILITY IS BROAD. I WOULD SAY IT'S COMMON THAT WE ARE FUNDING VERY EARLY STAGES ACADEMIC SPIN OUTS AND WE HAVE FILL AND AND COMPANIES THAT WE ARE FUNDING AS WELL THAT WERE FORMED BY PARENTS OF CHILDREN WITH THOSE DISORDERS AND OTHERWISE HAVE BEEN PERSONALLY EXPERIENCED DISORDERS THAT MOTIVATES THEM AND I WOULD SAY IT'S SOMETHING THAT YOU NIDS IS LOOKING FOR COMPANIES THAT ARE DEDICATED TO AREAS SO THAT IS IMPORTANT FACTOR IN SORT OF THE COMPOSITION AND I THINK IT'S VERY FREQUENT THAT WE SEE ACADEMICS PARTNER WITH NON PROFITS OR FAMILIES IN SOME WAY AS WELL AS BUSINESS EXPERTS SO IT'S NOT LIKE YOU HAVE TO HAVE ALL THE EXPERTISE WITH YOU AS ONE INDIVIDUAL AND IT'S ABOUT RECOGNIZING WHAT STRENGTHS YOU HAVE AND WHAT YOU ARE BRINGING TO THE TABLE AND THEN LOOK FOR THOSE GAPS AND TRY TO FIND AND WHAT SHE THINKS OF BUILDING A TEAM THAT'S BEEN SUCCESSFUL. >> I WAS READING THE CHAT ON THE SIDELINES. WHAT DID YOU WANT ME TO ANSWER? >> I WAS SPEAKING TO THE KINDS OF TEAMS THAT WE SEE AND HOW OFTEN THESE ARE VERY EARLY COMPANIES AND A LOT OF TIMES ANNAN ACADEMIC IS SPITTING OUT THE COMPANY AND NOT ALWAYS. YOU DO NEED TO BE EVERYTHING IN ONE AS THE INDIVIDUAL STARTING THE COMPANY AND IT'S ABOUT KIND OF RECOGNIZING YOUR SKILLS SET AND THE OTHER SKILLS SETS IN THE SCIENCE AREA AND BUSINESS AREA THAT YOU NO HE HAD TO BRING IT TOGETHER AND I THINK IT'S ABOUT BUILDING THE RIGHT TEAM IN THAT IS SUCCESSFUL. >> SO THAT WAS ONE OF MY BEGINNING SLIDES. IT'S A MASSIVE AMOUNT OF WORK AND YOU JUST HAVE TO IMMERSE YOURSELF IN IT AND I TRAVEL QUITE EXTENSIVELY AND I BROUGHT MY SON WITH ME AND I MET WITH EVERYBODY AND TOOK A LOT OF NOTES AND KEPT THE PHONE NUMBERS AND CALLED THEM BACK AND THAT'S ONE GOOD THING ABOUT BEING A PARENT IS THAT PEOPLE LIKE WE TALKED ABOUT ULTRA-GENICS, I'LL THROW SOMEONE ELSE OUT THERE JUDE SMALL SKI, STEVE GRAY, THESE ARE GREAT ADVOCATES OUT THERE THAT CAN STEER YOU IN THE RIGHT DIRECTION AND THAT'S WHAT YOU NEED. MY LOCAL HOSPITALS, I'M IN BROOKLYN, NEW YORK SO I WAS LUCKY TO BE AROUND NYU AND EINSTEIN, COLOMBIA, AND GOING AND TALKING TO THE NEUROLOGISTS AND THE GENETICIST THAT HAVE NIH GRANTS AS WELL WHO WORK WITH ACADEMIA AND OTHER SPONSORS AND THESE GUYS REALLY GAVE MY MARCHING ORDERS AND THEY CAN PROMOTE YOU AND HOOK YOU UP WITH OTHER PEOPLE AND THAT CAN BRING YOU FURTHER AND YEAH, YOU JUST HAVE TO IMMERSE YOURSELF IN IT AND IT'S HARD AND IT'S A 24/7 JOB AND IT'S YOU KNOW, ESPECIALLY WHEN YOU ARE TAKING CARE OF YOUR CHILD. IT'S NOT RECOMMENDED. ALSO THE OTHER PATIENT ADVOCATES AND FAMILIES ARE SUPPORTIVE. >> WE HAVE A GOOD QUESTION AND THIS IS IMPORTANT FOR PEOPLE TO THINK ABOUT BEFORE THEY CREATE A COMPANY. AND ANY GUIDANCE ON BALANCING BETWEEN WANTING THE UPSIDE TO JUST ROYALTIES ON COMMERCIAL SUCCESS AND LIMITING FRICTION? HOW MUCH LEVERAGE DOES IT ARE AND IT'S A SMART QUESTION BECAUSE IF YOU ARE HAVING A COMPANY AND IT MAY BE COMPETING AGAINST OTHER COMPANIES IN THE SPACE, HOW DOES IT AFFECT YOUR ROLE AS A PATIENT ADVOCATE AND YOUR ABILITY TO SORT OF BE AN HONEST BROKER WITH ALL OF THE STAKEHOLDERS IN YOUR DISEASE SPACE? >> FOR ANYBODY. >> SO, LET ME JUST SAY THAT WE STRUGGLED WITH BALANCING WHAT A ROYALTY RATES SHOULD BE FOR CHARITY AND I MEAN, OBVIOUSLY THE CHARITY'S GOAL IS TO CREATE A THERAPY FOR THEIR DISEASE AND ON THE OTHER HAND, THEY HAVE RAISING MONEY IS HARD AND JILL TALKED ABOUT IT EASIER AND IT'S SO YOU HAVE TOUGH TO RAISE MONEY AND GETTING RETURN IF THERE'S SUCCESS IS HELPFUL AND SHARING WITH COMPANIES SO, GENERALLY, I SORT OF USE A RULE OF THUMB AND MY RULE IS THUMB IS ANY FUNDING BELOW LET'S SAY, $8 MILLION I USE A MULTIPLE OF THE AWARD AND MULTIPLES RANGE FROM THR THREE O SIX TIMES AND THEY'RE PAID AT SUCCESS AT APPROVAL BUT THERE'S AN EARLY PAYMENT IF THERE'S AN EXIT TO IT TO A LARGER COMPANY AND SO WHY IS IT THREE AND NOT SIX? DEPENDS IF IT'S EARLY STAGE AND IN THE RISK IS HIGHER THE MULTIPLE IS OBVIOUSLY HIGHER SO IT'S IN THE FIVE-PERSON, FIVE-X RANGE AND ABOVE THE 8 MILLION MARK WE COME UP WITH A PERCENTAGE OF NET SALES. EVEN IF YOU HAVE A PRODUCT APPROVED WE PUT SOME GOVERNOR ON IT AND SOME LIMITATIONS SO WE SAY IN NO CASE SHALL THE ROYALTY, SHALL THE FIVE X OR SIX X IS PAID WITHIN THROW YEARS AND AFTER A FDA APPROVAL AND USUALLY WE PUT IN THE NO CASE SHELL THE PAYMENT EXCEED X. >> TWO MINUTE WARNING. >> I WANT TO HEAR YOUR PERSPECTIVE ON THIS BECAUSE YOU WERE THE PATIENT ADVOCATE THAT AND THE HONEST BROKER AND YOU HAVE TO. >> >> YOU TRIGGERED MY MIND ON SO MANY DIFFERENT THINGS AND WORKING WITH ACADEMIA CAN BE BROUGHTALLY CARD AND THEIR TECH TRANSFER DEPARTMENTS ARE SO HAD HE BOUGHT BACK AND I GOT ON A FLIGHT AND BANGED ON THE DOORS AND SAY NO WAY AND THIS IS ULTRARARE DISEASE AND THERE'S NO WAY AND I NEED TO AND YOU HAVE TO YOU PROBABLY ARE ARE THEIR ONLY SOURCE AND YOU HAVE OTHER OPTIONS AND THIS A MATTER OF GOING OUT FINDING THE RESEARCH AND IT'S GETTING MARRIED AND YOU REALLY BEFORE YOU MARRY YOUR PARTNER AND THEY HOLD THE STRINGS THAT MAKE LIFE DIFFICULT FOR YOU AND BROKERING BEING THE PATIENT ADD CO VISIT AND IT'S THE THE SPONSOR AS WELL AND IT'S REALLY NOW CHANGED FOR ME AND I DON'T DO AK DAMIA RESEARCH SO MUCH AND WE BRING THEM ON AS CONSULTANTS SO THEY'RE NOT TIED INTO THE IF YOU BANK ROLL YOUR MOUSE MODEL AND DO AND THERE ARE LOTS OF COMPANIES OUT THERE AND BUT YOU HAVE TO HAVE CONSULT APARTMENT WHO IS GOING TO DESIGN THE ENTIRE STUDY FOR YOU AND BE WITH YOU ALL THE ENTIRE STEP OF THE WAY AND I'M SORRY I'M GOING TO HAVE ALL MY PROFESSOR AND MY ACADEMICS HELP ME WITH PITCHFORKS BUT I THINK IF YOU CAN DON'T THAT AND I WOULD DEFINITELY DO IT BECAUSE YOU GET WHAT YOU PAY FOR AND IT'S SOMEONE GOES ON MATERNITY LEAVE AND THERE'S SOMEONE TO ACCEPT IN THE PLACE AND GET MARRIED ALL THE TIME AND IT'S TOUGH, GUYS. >> GOOD FOR YOU, JILL. >> WE HAVE RUN OUT OF TIME AND EMILY I WANT TO SUGGEST THAT YOU AT SOME POINT ORGANIZE A WHOLE DAY WORKSHOP ON SMALL BUSINESS THERE'S SO MUCH WE NEED TO KNOW. THANK YOU SO MUCH TO EMILY, KEN, WE'RE SO GRATEFUL AND JILL FOR TELLING YOUR STORY AND THANK YOU FOR JOINING THE PANEL AND SAM, THINK WE'RE GOING TO A BREAK NOW, RIGHT. I'M GOING TO HAND IT BACK OFF TO YOU. >> YES, WE ARE. THANK YOU ALL FOR THAT SESSION AND THANK YOU FOR MODERATING SO SHE JUST MENTIONED WE'RE HEADED INTO A HOUR LONG BREAK UNTIL 2:15. WELCOME BACK, EVERYONE, THE AFTERNOON SESSIONS OF OUR 2022NINDS NON-PROFIT QUORUM. THE SESSIONS THIS AFTERNOON ARE GOING TO KICKOFF WITH THE ONE ON YOUR SCREEN. HOW TO WORK WITH ACADEMIC RESEARCHERS. THIS SESSION WILL BE MODERATED BY Dr. PENNY DACKS FROM THE ASSOCIATION OF FRONTTOMEPORAL DEGENERATION WHERE SHE FUNDS RESEARCH, RECRUIT AND RETAIN TAL ANT, ENGAGE IN THE COMMUNITY WITH LIVED EXPERIENCE. SHE IS PRESIDENT OF THE FTD DISORDERS REGISTRY LLC WHICH IS RESPONSIBLE FOR PARTNERSHIPS SUSTAINABILITY OF THIS ORGANIZATION AFFILIATE. PREVIOUSLY, SHE WORKED AT THE AMERICAN EPILEPSIES SOCIETY OVERSEEING MISSION RELATED PROGRAMS IN RESEARCH, MEDICAL EDUCATION AND CLINICAL ACTIVITIES AND AS WELL AS AS THE ALZHEIMER'S DRUG DISCOVERY PROGRAM WHERE SHE LED COGNITIVE VITALITY.ORG AND THE AGING PROGRAM TO SOURCE AND EVALUATE POTENTIAL THERAPIES. PENNY TRAINED IN NEUROSCIENCE WITH A PH.D AND THE ICON SCHOOL OF MEDICINE IN MOU MOUNT SINAI. I HOPE PENNY IS ON. >> YES, I'M HERE. >> I CAN GO TO YOU AND LET YOU TAKE IT AWAY. >> THANK YOU, SAMANTHA. I'M HONORED TO BE HERE WITH EVERYBODY TODAY. SO THINKING ABOUT THE TITLE OF THIS SESSION LEVERAGING PROGRESS As COT SCIENTIFIC PIPELINE, HOW TO WORK WITH ACADEMIC RESEARCHERS, THAT WE'RE GOING TO SHOWCASE ARE FEW IS THERE'S NO ONE ANSWER TO THAT QUESTION. IT VARIES WHAT IS HAPPENING WITHIN YOUR SPACE. THERE IS SOME NON-PROFIT AND ACADEMIC RESEARCHERS ON WHAT ARE THE RIGHT STEPS WITH DIFFERENT ADVISORY BOARDS AND WE FUND RESEARCH. HOW DO WE MAKE SURE OUR FUNDING IS MEETING UNMET NEEDS. ARE WE ATTRACTING PEOPLE TO THE SPACE AND ARE WE TRYING TO BRIDGE FUNDING BECAUSE FUNDING AVAILABILITY IS BECOME RELATIVELY WEAK AND ARE WE SEEING RESEARCH TO GET IT IN OUR SPACE AND HOW WE WORK CONVENING THOUGHT LEADERS TO HELP CATALYZE INNOVATION AND ARE WE CREATING RESOURCES AND PARTNERING SO THAT WE CAN FOR EXAMPLE HELP ACADEMIC RESEARCHERS ACCESS OR UNDERSTAND THE PATIENT COMMUNITY THAT WE LIVED WITH AND SO THE RIGHT ANSWERS TO ALL OF THESE THINGS DEPENDS ON WHAT IS HAPPENING IN YOUR FIELD AND WHAT IS THE STAGE OF RESEARCH AND WHAT IS ON THAT SCIENTIFIC PIPELINE IN THAT LONG-TERM JOURNEY WHERE IS IT? WHERE IS EVERYONE? AND WHAT IS THE STAGE OF THAT RESEARCH. SO WE'RE GOING TO HEAR FROM FOUR DIFFERENT SCIENTIFIC LEADERS TODAY ABOUT THE SUCCESS STORIES THAT THEY'VE SEEN IN THEIR WORK WITH HOW FAR NON PROFITS CAN REALLY MAKE A DIFFERENCE AND HOW THEY CAN HAVE REALLY ROBUST RELATIONSHIPS WITH ACADEMIC RESEARCHERS. WE'RE GOING TO HEAR FROM FROM KENNETH FISCHBECK AND SABRINA PAGANONI AND FROM DAVID LIMBRIKK FROM THE WASHINGTON SCHOOL UNIVERSITY OF MEDICINE AND MIGUEL SENA-ESTEVES. FIRST ON THE LIST IS KEN. >> CAN YOU HEAR ME OK? >> SO, I AM A INVESTIGATOR IN THE INTRAMURAL PROGRAM, THE DIVISION OF INTRAMURAL RESEARCH AT NINDS AND THIS IS KIND OF A UNIQUE SITUATION WE HAVE HERE IN BETHESDA ON THE NIH CAMPUS. WE ARE FUNDED TO DO RESEARCH WE DON'T HAVE TO A REPLY FOR GRANTS AS OTHER INVESTIGATORS DO IN THE REST OF THE COUNTRY. WE ARE FUNDED AND WE GET PAID TO DO OUR RESEARCH AND WE ARE REVIEWED EVERY THREE TO FOUR YEARS BY OUTSIDE REVIEWERS AS LONG AS WE'RE DOING WELL, WE CONTINUE TO GET RESEARCH FUNDING. WE'RE TRYING TO PUT THAT MONEY TO GOOD USE AND THE BEST POSSIBLE USE IN REDUCING THE BURDEN OF NEWER LO NEURO LOGIC . I USUALLY SHOW THIS AS DISCLOSURE AND POTENTIAL CONFLICTS OF INTEREST BUT FOR THIS FORUM, I'VE LISTED NOT CONFLICT OF INTEREST BUT MY TRUE INTEREST LOWS AND IT'S NOT JUST ENDURING RESEARCH HERE AT THE NIH, IN BETHESDA BUT IT'S ENGAGING IN INTERACTING WITH THE LARGE NUMBER OF NON-PROFIT ORGANIZATIONS AND FOR PROFIT ORGANIZATIONS AND I'VE LONG OVER THE COURSE OF MY CAREER INTERACTS WITH IN THE PAST TWO OR THREE YEARS I'VE INTERACTED WITH THE ORGANIZATIONS LISTED HERE AND I'VE BEEN FORTUNATE TO BE ABLE TO DETHREE SA BAT TICKALS IN INDUSTRY AND I'M CONTINUING THE NON-PROFIT ARE FORECASTED IN OUR INTEREST OF INTEREST, NEURODEGENERATIVAL DISEASE AND THE FRENCH EQUIVALENT OF THE NDA ASK TREAT-NMD FOUNDATION AND TARGET ALS, PACKARD CENTER FOR ALS AND THE KENNEDY'S DISEASE ASSOCIATION AND RECENTLY AND FOR SOME YEARS NOW WITH THE SMA FOUNDATION AND A COUPLE OF DISEASES THAT FOCUSING ON HUNTING TON'S DISEASE AND ONE NON-PROFIT IS THE N-LOREM FOUNDATION SET UP A COUPLE OF YEARS AGO AS A PIN SPIN OFF BY E IONIS FOUNDATION AND IT'S SET UP LIKE ULTRARARE AND SO ON AND THIS IS STAN CROOK CAME UP WITH NANO RARE IT'S A FOUNDATION THAT IS SET UP TO PROVIDE ALL THE NUCLEOTIDE THERAPY FOR PATIENTS WITH DISEASES WHERE THEY'RE THE ONLY PATIENTS WITH THE DISEASE IN THE WORLD OR MAYBE UP TO 30 OR SO ARE AFFECTED BY THIS THE DISEASE SO THEY'RE MUCH LESS COMMON THAN THE ULTRARARE DISEASES AND THE FOUNDATION MAKE THE NUCLEO TIDES AVAILABLE AS TREATMENT FOR INDIVIDUALIZED HAD THESE PATIENTS AND PROVIDE THEM FOR FREE SORRY FAR THEY WILL DO THAT WITH NEW TREATMENT FOR THESE PATIENTS THIS YEAR. AND I MENTIONED THE INTERACTIONS I'VE HAD WITH THE COMPANIES LISTED HERE. SO, NEXT SLIDE. THE RESEARCH REEF DONE OVER THE YEARS IS ON THIS PROGRAM WHICH A SLIDE I'VE USED FOR THIS TIME NOW AND OVER THE LAST 35 YEARS NOW, I LARGE NUMBER OF DISEASE GENES HAVE BEEN IDENTIFIED AND AT LAST COUNT 7100 DIFFERENT DISEASES THAT HAVE AN IDENTIFIED GENETIC CAUSE AND THIS IS BEEN CONTRIBUTED TO AT THE NIH AND SEEING PATIENT CHARACTERIZING THE DISEASE MANIFESTATIONS AND COLLECTING SAMPLES DURING SEQUENCING AND ANALYZING THE VARIANTS AND MAPPING AND IDENTIFYING THE DISEASE CHAINS AND CAUSES OF BEGAN ET TICK DISEASES BUT IT STARTS US ON THE BOTTOM PART OF THIS CLUB TO CHARACTERIZE THE GENES AND FIGURE OUT THE NORMAL FUNCTION OF THE GENE AND HOW THOSE NORMAN FUNCTION IS ALTERED IF A LOSS OF FUNCTION AND TO DEVELOP SUB CULTURE IN ANIMAL MODELS WE USE A LOT OF CELLS AND MOUSE MODELS AND TO USE TO TEST TO SCREEN FOR NEW TREATMENTS AND THEN TO ASSESS NEW TREATMENTS FOR EFFICACY AND SAFETY AND THEN FINAL LIGHT LAST STEP TO CLOSE HERE, WHERE THERE HAS BEEN SOME SUCCESS IT'S BEEN A LOT OF WORK AND WE CAN POINT TO A DISEASE THAT'S WE HAVE WORKED ON THAT HAVE SUCCESSFULLY CLOSED THE GAP THROUGH CLINICAL TRIALS AND UP TO HAVE SAFE AND EFFECTIVE TREATMENT FOR THE DISEASE AND THE POSTER CHILD FOR OUR FIELD IS DISEASE WE'VE BEEN INVOLVED WITH FOR A NUMBER OF YEARS AND DEFINING THE DISEASE AND HELPING THE IDENTIFICATION AND CHANGE AND WORKING WITH OTHERS TO SHOW BENEFIT IN THE FIRST BENEFITS IN MOUSE MODELS AND INTERVENTION TO CORRECT THE UNDERLINE DEFECT OF DEFICIENCY AND IN MOUSE MODELS WITH THE DISEASE AND WE HAVE THROW TREATMENTS AT LEAST THAT T OF AND BENEFIT WHEN STARTED EARLIER ENOUGH BEFORE THE DISEASE ONSET AND THERE'S AN A NATIONWIDE SCREENING AND TO IDENTIFY NEWBORN INFANTS WHO WERE AT RISK. THE WORK THAT WE DO IS ON THE NIH CAMPUS HERE IN BETHESDA AND IT'S DIVIDED BETWEEN BUILDINGS AND BOTH VERY PORT AT TO BE USING THESE FACILITIES FOR LABORATORY RESEARCH AND THE NEURO SIGN RESEARCH CENTER SHOWN ON THE NIH CAMPUS LOOKS LIKE A NIGHT WHEN PEOPLE FORGOT TO TURN THE LET'S OFF AND IT'S MY OFFICE IN THE CORNER BUT IT'S WONDERFUL FACILITY TO DO BABY SEARCH AND APPLIED RESEARCH RELATED TO NEUROLOGICAL DEDISEASES AND THE NIH CLINICAL CENTER OUR BUILDING 10 WHICH IS ONE OF THE LARGEST IF NOT THE LARGEST RESEARCH HOSPITALS IN THE WORLD AND IT'S A WONDERFUL FACILITY TO DO CLINICAL STUDIES AND INTERCEPTIONSAL STUDIES PHASE ONE AND PHASE TWO AND IN PARTICULAR, AND CLINICAL PROOF CONCEPT STUDIES WITH THE DISEASES THAT WE WORK ON AND WE DON'T HAVE TO APPLY FOR FUNDING WE HAVE TO GET APPROVAL AND START THE STUDY AND WE ALSO HAVE THE OPPORTUNITY TO COLLABORATE WITH EXTRA MUR AVENUE INVESTIGATORS AND GETTING THOSE STUDIES DONE AND TO SHARE IS AND PROVIDE FUNDING FOR THEM. I JUST LISTED HERE SOME OF THE INTRAMURAL CLINICAL INVESTIGATORS WITH THEIR DISEASE AREAS AND I THINK ALL OF US CAN BE VERY USEFUL PARTNERS FOR NON-PROFIT GROUPS SUCH AS A REPRESENTATIVE IN THIS FORM AND COLLABORATORS FOR ACADEMIC INVESTIGATORS AS WELL THROUGH SHARED FUNDING AND THIS IS A SPECIALIST IN PETE ADD TRICK NEWER LOGIC DISEASES AND MYSELF FOCUSING ON A RANGE OF NEURO MUSE KULAR AND PARTICULARLY RARE HEREDITARY MOTOR NUR ON DISEASES LIKE KENNEDY DISEASE AND ALS4 AND 8 AND ILS2 AND DAVID GOLDSTEIN WORKS ON AUTONOMIC DISORDERS AND THERE'S A STROKE BRANCH AND MARK HAS A LONG AND VERY PRODUCTIVE CAREER IN MOVEMENT DISORDERS RESEARCH AND NEW RECRUIT WORKS ON PARK INSINCE DISEASE AND JUVENILE VARIANTS AND THE CLINICAL DIRECTOR SO HE IS IN CHARGE OF ALL THIS GROUP AND HAS A PARTICULAR INTEREST IN NEURON INFECTIOUS DISEASES SUCH AS COVID AND HIV. DAVE REICH HAS BEEN IN THE BRANCH WITH A FOCUS ON MULTIPLE SCLEROSIS AND SONIA SCHOLZ WORKS ON LEWY BODY DEMENTIA BETWEEN ALZHEIMER'S AND PARKINSON'S AND MICHAEL WARD AND A NEW UP-AND-COMING INVESTIGATOR IN OUR GROUP WHO WORKS ON ALS AND IT'S INTIMATELY CONNECTED WITH THE INTRAMURAL PROGRAM CALLED CARVE A CENTER FOR ALZHEIMER'S AND RELATED DISORDERS AND AND HE RUNS A SUB CULTURE PROGRAM PROJECT THROUGH CARVE AND HE IS INVOLVED WITH THAT WHICH IS VERY WELL FUNDED THROUGH THE NATIONAL INSTITUTE OF AGING AND KAREEM A STAR NEUROSURGEON AND WHO IS GROUP WORKS ON BRAIN TUMORS AND HE IS ALSO INVOLVED IN SURGICAL TREATMENT FOR EPILEPSIES SO AWFUL THESE PEOPLE ARE PEOPLE YOU CAN GET IN TOUCH WITH AND I ENCOURAGE YOU TO FORM A WORKING RELATIONSHIP WITH THIS INVESTIGATORS TO MOVE THE FORWARD. THERE ARE A NUMBER OF OTHERS WHO ARE STAFF CLINICIANS AND ASSISTANT CLINICAL INVESTIGATORS AND IT'S A USEFUL RESOURCE TO TAP INTO. SO HOW DO YOU FIND IN GENERAL, HOW DO YOU FIND CLINICAL ININTEREST MURAL THAT YOU CAN WORK AND LOOKING AROUND AT DIFFERENT WEB SITES I THOUGHT MOST USEFUL IS A WEBSITE ON PATIENT RESEARCH AT NINDS AND I GIVE THE LINK HERE BUT IF YOU JUST GOOGLE ON OUR SEARCH ON PATIENT RESEARCH YOU WILL GET THE LIST I SHOWED YOU AND THE CONTACT INFORMATION HOW TO GET IN TOUCH WITH PEOPLE O'ER YOU CAN GO TO THE OFFICE OF THE CLINICAL DIRECTOR THAT IS Dr. AL BYE AND HIS OFFICE CAN SEND YOU FURTHER AND JUST SEARCH ON CLINICAL DIRECTOR OR SPEAKING OF SEARCHES A TRICK I PICKED UP A FEW YEARS AGO IS YOU CAN JUST PUT IN THE NAME OF YOUR DISEASE AND A SEARCH AND THE ZIP CODE 20892 WHICH IS A UNIQUE ZIP CODE FOR THE NIH CAMPUS AND IT'S GOING TO TAKE YOU TO PUBLICATIONS AND HOME PAGES FOR RESEARCHERS AT THE NIH AND WHATEVER AREA OF YOUR INTEREST AND THEN FINALLY, ALL ELSE FAILS, CONTACT ME AND YOU CAN SEARCH ON MY NAME. AND YOU WILL GET MY E-MAIL THE NIH IS A PIECE OF THE JIGSAW PUZZLE BUT ANY OTHERS THAT ARE SHOWN HERE. PARTICULARLY THE DISEASE FOUNDATION IS A REPRESENTATIVE THEY PUT THE WIND IN OUR SALES AND YOU ARE CUSTOMERS AND OUR BOSSES BECAUSE OUR FUNDING COMES THROUGH CONGRESS WITH HELP FROM PATIENT ADVOCACY GROUPS AND WE DEPEND ON YOU THROUGH CONGRESS AND CONGRESSIONAL APPROPRIATIONS AND YOU ARE ALSO DEVOTED TO SERVING AND MEETING YOUR NEEDS, EFFECTING THOSE EFFECTING YOURSELVES OR YOUR CHILDREN AND YOUR FAMILY MEMBERS AND WE ALSO HAVE TO ENGAGE IN THIS AND I SEE IT'S LIKE A RELAY RAISE AND I THINK THIS IS TRUE OF ACADEMIC RESEARCHERS IN GENERAL AND SHOWING EVIDENCE AND THEY'RE GOING TO WORK AND THAT STAGE WE HAVE TO PASS THE BATON ON TO INDUSTRY TO CARRY IT THROUGH TO A DRUG YOU CAN GO TO THE DRUGSTORE AND GET AND GET THE ADMINISTERED IN THE HOSPITAL 20 GET FDA APPROVAL TO PAY FOR IT BUT INTER MIGHT WITH INDUSTRY AND IT'S JUST A POINT ON THIS SLIDE BUT IMPORTANT RELATIONSHIP WITH OUTSIDE ACADEMIC MEDICAL CENTER AND NIH IS THE MAIN SOURCE OF FUNDING AND MANY OF THESE RESEARCHERS AND WE'RE HAPPY TO COLLABORATE WITH OPPORTUNITIES FOR CO FUNDER AND I HOPE I WAS ON TIME. I DIDN'T RUN OVER TOO MUCH. >> THANK YOU. WE'RE GOING TO HOLD ON QUESTIONS UNTIL ALL FOUR SPEAKERS HAVE A CHANCE TO CHAT AND WHILE YOU ARE LISTENING TO ANY OF THESE SPEAKERS ININCLUDEDDING, PLEASE GO AHEAD AND PUT QUESTIONS IN THE CHATBOX AND WE'LL MAKE THOUSAND THOSE GET ADDRESSED AS MUCH AS TIME ALLOWS AT THE END OF THE SESSION AND OUR NEXT SPEAK SER Dr. DAVID DIM BRICK AND THE CHIEF OF PEDIATRIC NEURO SURGERY AND EXECUTIVE VICE-CHAIR AT WASHINGTON UNIVERSITY. HE ALSO SERVES AS THE NEUROSURGEON AND CHIEF AT ST. LOUIS CHILDREN'S FONT AND GO RIGHT A HEAD, PLEASE, LET ME FOE IF YOU HAVE ANY TECHNICAL ISSUES WITH MY DECISION AND TODAY MY TOPIC IS GOING TO BE THE ROLE AND INVOLVEMENT OF NON-PROFIT ORGANIZATIONS IN PATIENT ORIENTED RESEARCH AND SO I'M HAPPY TO TALK TO YOU ABOUT THIS TODAY. JUST TRYING TO ADD THESE SLIDES AND I DON'T KNOW IF I DID THAT BUT I HAVE SOME DISCLOSURES THAT ARE DEVELOP RANT TODAY AND THIS SLIDE JUST KIND OF GOES THROUGH FUNDING SOURCE AND SO FOURTH BUT THE RELEVANT DISCLOSURES WHICH I'LL TRY TO GET BACK TO AND KEEP MY STREAM OF THOUGHT INCLUDED AND WHEN EXECUTIVE COMMITTEE AND AND FOUNDATION AND I JUST WANT TO SHOUT OUT TO THEM BECAUSE THEY'RE ON THE LINE TODAY AND MARY, KAITLYN AND DORTHY THANK YOU FOR ATTENDING ONE OF THE MOST CONTROVERSIAL QUESTIONS IN PEDIATRIC NEURO SURGERY AROUND THE THEY ARE DEPLOYED FOR THAT AND ONE IS IS I BONE AND BIGMENT AND THE OTHER ONE IS A STEP FURTHER WHERE WE OPEN UP WE APPLIED THROUGH THE INSTITUTE AND IN THE FUNDING WAS TO PERFORM A RANDOMIZED CONTROL TRIAL COMPARE THESE TWO DIFFERENT OPERATIONS WHICH HAVE A DIFFERENT RISK PROFILE AND A DIFFERENT EFFICACY PROFILE AND I'M GOING TO SAY I'M HAVING TROUBLE MOVING THEM FORWARD AND THANK YOU. AND THIS TRIAL WAS SUPPORTED THROUGH ALL THE MAJOR AT TRICK ORGANIZATION AND IT'S A CLUSTER RANDOMIZED AND A HIGH-QUALITY LAST YOU CAN SEE THE ENROLLMENT IN BLUE AND THERE ARE A LOT OF FACTORS INVOLVED IN THAT AND ONE KEY THING AND THIS IS WHAT I WANT TO HIGHLIGHT HERE IS THAT THEY ARE DEVELOP RANT FROM THE PATIENT COMMUNITY AND DO YOU MIND ADVANCE INTERESTING IF YOU DON'T MIND I'M GOING 20 TRY THIS METHOD. AND THANK YOU AND SO WE WENT THROUGH A NUMBER OF DIFFERENT ENGAGEMENT ACTIVITIES TO TRY TO FOSTER SUPPORT IN THE TRIAL AND CAN YOU ADVANCE THE SLIDES MORE AND THIS IS ONE EXAMPLE OF THIS WAS PUT ON BOBBIE JONES AND OUR SAINT AND FLOOR THIS AND WE REALLY WORKED ON THE LEGAL SUPPORT FOR FAMILIES AND DEALING WITH A CHILDHOOD IS DIS ABILITY AND THE EDUCATIONAL SUPPORT AND YOU CAN GO FORWARD ONE NOW AND ONE OF THE THINGS THAT TURNED OUT TO BE ABSOLUTELY KEY IN OUR ENROLLMENT AND OUR RELATIONSHIP WITH BOBBIE JONES AND WHAT HAPPENED EARLY IN THE TRIAL ON THUR SOCIAL MEDIA ACCOUNTS THEY STARTED TO SEE SOME NEGATIVE OR NEAR NEGATIVE COMMENTS ABOUT PARTICIPATING IN THE CLINICAL TRIAL AND WHAT THEY DID WAS THEY REALLY HELPED US TO DISPEL NEGATIVE FEELINGS REGARDING CLINICAL RESEARCH AND THROUGH ON-LINE CAMPAIGNS HERE IS AN EXAMPLE AND YOU CAN ADVANCE AND THEY HELPED US TO CREATE PATIENT BROCHURES TO REASSURE PATIENTS THAT THERE'S POINTS BETWEEN THESE TWO DIFFERENT PROCEDURES AND WE REALLY NEEDED TO SUPPORT THE RESEARCH THERE AND THERE'S AN EXAMPLE OF PATIENT PRO SURE IF YOU CAN ADVANCE ONE MORE SLIDE, WITHOUT THEM WE WON HAVE MET OUR IT WOULD HAVE BEEN UNDER-POWERED SO YOU CAN MOVE FORWARD, PLEASE. THE SECOND EXAMPLE IS ONE IN HYDROCEPHALUS. YOU KNOW THE TWO KEY TREATMENTS FOR HYDROCEPHALUS IN CHILDREN IS YOU MAY BE AWARE IS SHUNTING, AS YOU CAN SEE ON THE LEFT WHICH IS IS A LITTLE TUBE OR A NEW PROCEDURE WHICH WE'LL USE THE A ABBREVIATION OF ETV-CPC FOR AND YOU CAN ADVANCE ONE MORE SLIDE THERE AND PATIENT POPULATIONS FOR THIS AND WHAT IS HAPPENING IS DESPITE A LACK OF EVIDENCE THAT ONE IS BETTER THAN THE OTHER THERE'S A GLOBAL RISE OF THE USE OF THIS PROCEDURE AND YET, THE VEN TRICK ALS, THE FLUID CAVITIES REMAIN LARGE WITH ATV SO WE'RE NOT SURE WE WORKED WITH THE HYDROCEPHALUS AND BEFORE WE DESIGNED THE TRIAL WHAT'S MOST IMPORTANT TO FAMILIES WITH HYDROCEPHALUS AND THE MOST IMPORTANT FACTOR WAS COGNITIVE FUNCTION. SO THERE WAS A STRONG DESIRE FOR FAMILIES TO HAVE THEIR CHILDREN BE FREE OF SHUNTS BUT THEY WERE EVEN WILLING TO UNDERGO MORE THAN ONE SURGICAL PROCEDURE TO BE SHUNT FREE AND IF IT DIDN'T EFFECT COGNITIVE FUNCTION AND IT'S A KEY FACTOR IN THE DECISION-MAKING HERE SO WE DESIGNED AN RCT TO HELP US TEST THIS AND THIS LIGHT ON THE RIGHT I JUST WANT TO SAY THAT ONE THING THAT HA HAS DONE REALLY WELL IS THEY'VE CREATED A SERIES OF INTER LACED EFFORT. ONE IS CRITICAL RESEARCH NETWORKS AND A DISCOVERY SCIENCE NETWORK AND THERE'S THE PATIENT POWERED REGISTRY WHICH IS HYDROCEPHALUS ASSOCIATION PATIENT-POWERED I'M NOT SURE OF THE INE ARE SO YOU CAN GO FORWARD, PLEASE. SO WE DO HAVE THIS RCT NOW THAT REALLY TAKES THE INFORMATION THAT WE GOT FROM THE HYDROCEPHALUS SU SURVEY TO DESIN THIS RCT WHICH IS U01 FUNDED THROUGH THE NINDS AND THE PRIMARY OUTCOMES WHICH WAS GENERATED WITH THAT INFORMATION FROM HA IS THE PRIMARY OUTCOME IS COGNITIVE OUTCOME AT 12 MONTHS SO FOR NEUROSURGERY WE MIGHT SAY THE PRIMARY OUTCOME WOULD BE SHUNT REVISION OR INFECTION OR SO FOURTH BUT SO I DO WANT TO SAY THANK YOU FOR ALL THE SUPPORT FOR THE HYDROCEPHALUS ASSOCIATION AND I CAN SEE SOME OF THE MEMBERS ARE HERE O AN AS WELL I'M GRATEFUL TO BE INVOLVED WITH THIS INFORMATION WITH THIS ORGANIZATION AND IN FACT I'M ON THE BOARD OF DIRECTORS AS I SAID BEFORE AND WE JUST HAD OUR NATIONAL CONFERENCE THE LAST WEEKEND WHICH IS A SUCCESS SO CONGRATULATIONS SO YOU CAN MOVE FORWARD, PLEASE. I WANT TO SAY IN 2022, YOU JUST SAW A SIMILAR IMAGE IN KEN'S PRESENTATION BUT IN 2022, TO MAKE THE KINDS OF ADVANCES AND SCIENCE AND CLINICAL MEDICINE THAT WE NEED TO, IT REALLY TAKES A TEAM AND SO THAT TEAM IS PEOPLE WITH MULTIPLE DIFFERENT SPECIALITIES WITH DIFFERENT EXPERIENCES AND DIVERSE SKILLS AND ALL FOCUSED ON A COMMON GOAL AND AND THEY ARE KEY PLAYNESS OUT WE DO TEAM SIGNS AND WE HAVE CLINICAL TRIALISTS AND CLINICAL EXPERTS AND THEN A KEY PIECE OF THE PUZZLE IS INPUT FROM PATIENTS AND ADVOCACY GROUPS AND WITH THAT I'LL STOP AND I'LL WAIT FOR THE QUESTIONS TO COME UP DURING QUESTION PERIOD LATER AND THANK YOU SUCH FOR YOUR ATTENTION. >> IT'S INTERESTING TO SEE THE EXACT SAME META FOR FOR YOURSELF ACROSS DIFFERENT TYPES OF STAKEHOLDERS AND I THINK IT'S GOING TO BE SOMETHING WE SHOULD CIRCLE BACK TO ABOUT THE WAYS OF NON PROFITS CAN HELP WITH THOSE DIFFERENT PIECES FITTING TOGETHER. WE HAVE THE CO DIRECTOR THE NEUROLOGICAL INSTITUTE AT THE MASSACHUSETTS GENERAL HOSPITAL AND HARVARD MEDICAL SCHOOL HER RESEARCH IS ON CLINICAL TRIALS AND THERAPY DEVELOPMENT. TAKE IT AWAY. >> THANK YOU SO MUCH AND IT'S SUCH A PLEASURE TO BE HERE WITH ALL OF YOU AND AS YOU SAID, A CLINICAL TRIAL IS FOCUSING ON ALS AND TODAY I WAS ASKED TO REFLECT AN THE IMPORTANCE OF OUR RELATIONSHIPS WE FOUNDATIONS SO I WANT TO FOCUS ON THAT AND START FROM THE BEGINNING OF MY CAREER WHERE IT'S BUILDING ON THE SAME META FOR WE'RE HERE BECAUSE OF OUR AND FOUNDATIONS WE ARE ALL WORKING FOR THE SAME GOAL SO THE WORK I'VE BEEN DOING DID REALLY COLLABORATIVE BETWEEN THE FDA SCIENTISTS AND FOUNDATIONS HAVE BEEN SUPPORTING MANY OF OUR ACTIVITIES AND I WANT TO TALK ABOUT A DIFFERENT CONTEXT AND DIFFERENT WAYS THAT FOUNDATIONS CAN SUPPORT ACADEMIC PHYSICIAN INVESTIGATORS LIKE MYSELF. SO GOING BACK TO THE FINING, I'M DOING CLINICAL TRIALS AS MY MAIN OCCUPATION GOES BACK TO A CLINICAL SCIENTIST DEVELOPMENT AWARD I GOT IN 2017 AND I'M THERE IN THE MIDDLE YOU MAY NOTICE DIFFERENT HAIR COLOR BUT I CHANGE THAT FREQUENTLY SO I'M IN THE MIDDLE WITH MANY OTHERS ARE STILL GREAT FRIENDS AND COLLABORATORS AND WE REALLY HAVE A CLASS OF FUNDED INVESTIGATORS AND STARTING TOGETHER IN 2017 AND EVERYONE HAS BEEN SPECIALIZING IN DIFFERENT AND NEUROLOGICAL AREAS AND THE DEVELOPMENT AWARD I GOT WAS SUPPORTED BY THE ALS ASSOCIATION IN COLLABORATION WITH THE AMERICAN ACADEMY OF NEUROLOGY AND THE AMERICAN BRAIN FOUNDATION AND IT'S REALLY WONDERFUL THAT DIFFERENT ASSOCIATIONS COME TOGETHER TO FUND THIS SPECIFIC RESEARCH AND IN MY CASE SPECIFICALLY, I FINISHED MY TRAINING INCLUSIVE RESIDENTIAL BOARD CERTIFICATES, ET CETERA, WHEN SOMEONE FINISHES THE TRAINING AS A PHYSICIAN SCIENTIST IT'S A TIME IN ONES' CAREER WHERE YOU HAVE TO GET PROTECTIVE TIME TO REALLY DEVELOP EXPERTISE AND A PHARMACEUTICAL COMPANY IN THE BOSTON AREA BUT THE FUNDING, THE INITIAL FUNDING FOR SOME OF THE INITIAL EXPERIMENT THE SUCH AS TOXICOLOGY AND SOME OF THE INITIAL INFRASTRUCTURE GRANTS FOR THE TRIAL AND WAS FUNDED ACTUALLY BY A FOUNDATION AND THIS TRIAL WOULD HAVE NEVER HAPPENED WITHOUT THE HELP AND NOW AFTER THAT, AFTER THE PROVIDED THE FUNDING, THE COMPANY WENT ON TO SECURE ADDITIONAL FUNDING OF VENTURE CAPITOL, ET CETERA, THEY DID A GREAT JOB EXPANDING THE TRIAL AND HERE WE ARE AT FIVE YEARS LATER WHERE THE DRUG THAT WAS TESTED IN THAT TRIAL WAS ACTUALLY APPROVED BY HEALTH CANADA THAT HAPPENED A FEW WEEKS AGO AND WE EXPECT THE FDA DECISION BY SEPTEMBER AND THERE'S AN ADDITIONAL CONFIRM TORI TRIAL THAT'S ON GOING IN EUROPE. IT HELPED GET THIS PROGRAM OFF THE GROUND AND NOW WE'VE BEEN APPROVED DRUG IN CANADA AND HOPEFULLY APPROVED DRUGS IT'S THE FIRST PLATFORM TRIAL FOR ALS SO IT'S A LARGE-SCALE TRIAL WHERE YOU TUFT DRUGS STATEMENT SO RIGHT NOW WE'RE TESTING FIVE DRUGS FOR ALS USING THE SAME INFRASTRUCTURE AND WE'RE ADDING MORE DRUGS TO THE SAME PLATFORM AND THE TRIAL IS NOT DIRECTOR THE CENTER FOR ALS AND GENERAL WHERE I'M SPEAKING FROM AND THE REASON THERE ARE LOGOS ON THE SLIDE IS BECAUSE THIS IS TEAM SCIENCE AND WE ARE DOING THIS IN COLLABORATION WITH MULTIPLE STAKEHOLDERS AND SOME OF THEM ARE ACADEMIC DOLL COLLABORATORT AS YOU SEE THERE'S A NUMBER OF FOUNDATIONS THAT ARE SUPPORTING PIECES OF THE TRIAL AND THIS IS CRITICAL FOR THE CONDUCT OF THE TRIAL ITSELF AND REALLY BEING ABLE TO TEST SO MANY DRUGS AT THE SAME TIME IN THE SAME DISEASE AREA THEY MAY NOT BE THEY ARE FOR ANT FOR PATIENTS AND ACADEMIC RESEARCHERS SO FOR EXAMPLE, FUNDED BY FOUNDATIONS, SPECIFIC FOUNDATIONS WE ARE FOCUSING ON INCLUSION AND NOW WE HAVE AND HAVE PEOPLE WHO CAN ANSWER QUESTIONS FROM PATIENTS LIFE AT ALL TIMES AND BECAUSE OF THEIR HELP, WE HAVE CONNECTED WITH THOUSANDS OF PATIENTS OF ALS AND THIS IS REALLY RESPONSIBLE IN THE LARGE PART IN 2020 WE'VE HAD GREAT ENROLLMENT RATES AND WITH THE PATIENT COMMUNITY AND SPECIFIC ASPECTS OF THE TRIAL THAT ARE FUNDED BY SPECIFIC ORGANIZATIONS AND I'M NOT GOING INTO THE DETAILS BUT ACCESS IS AND INCLUSION IS BIOMARKERS AS OTHER SO I THINK FOR THE WORK WE DO COLLABORATING WITH FOUNDATIONS AND PATIENTS CENTER GROUPS, IT'S CRITICAL AND FOR ACADEMIC INVESTIGATORS LIKE MYSELF IT'S CRITICAL WITH SPECIAL EXPERTISE IN ONE DISEASE AREA. THAT WAS ALL I HAD TO SHARE OUR LAST SPEAKER IS Dr. MIGUEL HE IS A ASSOCIATE PROFESSOR IN THE GENE THERAPY CENTER AT U.S. CHAN MEDICINE SCAL SCHOOL AND AND AS WELL AS GENE THEY ARE FEE FOR DISEASES AND I WILL LEAVING TO HIM TO EXPLAIN MORE. GOOD AFTERNOON, EVERYONE. I HAVE NO SLIDES TO SHOW THIS AFTERNOON SO I DON'T RUN THE RISK OF REPEATING SOME OF THE ASPECTS THAT MY COLLEAGUES HAVE ALREADY TALKED ABOUT IT'S COMPLICATED AND THERE'S NO QUESTION THAT IN SCIENCE AS WE BECOME MORE AND MORE COMPLICATED WITH BRINGING TO BEAR LOTS OF DIFFERENT TECHNOLOGIES SCIENCE IS PART OF A LARGE TEAM TO BRING THE TECHNOLOGIES WE HAVE AT HAND TO BEAR IN PARTICULAR QUESTIONS AND SO, INSTEAD OF GOING ON A TANGENT THAT I DID WHEN I SPOKE TO PENNY BEFORE, I AM ACTUALLY GOING TO TELL YOU MORE ABOUT MY EXPERIENCE ON WORKING WITH ONE FOUNDATION TO DEVELOP A GENE THERAPY FOR A DISEASE THAT IS A WELL-KNOWN INHERITED DISEASE DESCRIBED MANY YEARS AGO. WHEN I STARTED ON THIS PROJECT, ACTUALLY IT STARTED FROM THE VERY BEGINNING BY BRINGING TOGETHER QUITE A FEW SCIENTISTS. IT HAD DIFFERENT PIECES OF THE PUZZLE, IF YOU WILL, THAT WE'RE WORKING ON MOUSE MODELS, LARGE ANIMAL MODELS FROM THE BEGINNING IT WAS GOAL THE BRINGING EVERYONE TOGETHER AND THEY HAD PLAY A CENTRAL ROLE BECAUSE I WAS ABLE TO BRING TOGETHER INDIVIDUALS AND PROVIDE SEED FUNDING TO GET THE PROJECT STARTED. THERE WAS NOTHING IN THE OR EYES ON THAT COULD CHANGE THE AND SO WE STARTED OUT RESEARCH WITH SOME OF THE FUNDING FROM NTSAB AND WAS ME AND A BUNCH OF DIFFERENT COLLEAGUES ACTUAL WILL HE FROM ABROAD AS WELL AND AT BEGINNING EVENTUALLY WE WERE ABLE TO WRITE A GRANT TO NIH AND TO SUPPORT MOVING OUR FINDING FROM THE BEACH TOWARDS THE CLINIC AND DOING TOXICOLOGY STUDIES, ET CETERA. WHAT WE FOUND OVER THE COURSE OF A COUPLE OF YEARS, IN OUR WORK, IS THAT WHILE SOMETHING THAT FAPHARMACOLOGISTS KNEW, IF YOU GIVE CELLS IN THE BRAIN, ASK THEM TO MAKE TOO MUCH OF A GOOD THING, IT'S A BAD THING AND WE KNOW THAT FROM TILE INOLE OR ADVIL BUT IF YOU TAKE TOO MUCH IT'S A MAJOR PROBLEM. WE FOUND THAT OUT AT THE GENETIC LEVEL AND THAT IS WHERE THE PROGRAM COULD HAVE DIED. THAT'S WHERE IT COULD HAVE STOPPED AND THEY RAISED A LOT OF MONEY AND WAS ABLE TO PROVIDE FUNDING NECESSARY TO CONTINUE THE PROJECT TO THE POINT WE SAW THOSE TOXICOLOGY ISSUES WE HAD ENCOUNTERED AND FIND AND DEVELOP NEW VECTORS THAT EXPRESS OR MAKE THE NORMAL PROTEINS AT LOWER LEVELS BUT DON'T KILL THE CELLS WHICH IS THE PRIMARY GOAL THAT DEVELOPED A TREATMENT THAT DOES NEED TO PROCESS KILL CELLS IN THE BRAIN BECAUSE THEY DON'T COME BACK WHEN THEY'RE DEAD AT LEAST FOR THE MAJORITY OF THE CASES. WITH THAT IN MIND, WE SPEND YEARS AND THAT WERE CONSULTED FOR THESE FOUNDATIONS FROM INDUSTRY TELLING US THAT WE ABSOLUTELY NO ABILITY TO TAKE THIS TO A CLINICAL TRIAL AND IT MAY HAVE BEEN TRUE BUT MONEY WAS NOT AVAILABLE AND WE'RE NOT INTERESTED IN GREEN THERAPY IT'S THIS MAYBE IT WORKS BUT WE NEED LARGER INDICATIONS BECAUSE IT FALLS INTO DISEASE AND IT'S HARD TO MAKE A CASE FOR ULTRARARE DISEASES AND WE'RE STUCK IN THAT CYCLE OF YOU CAME TO IT BUTNTTEN DOING IT SO, A FOUNDATION CAME ON BOARD, THE BLUE JEANS FOUNDATION THAT ALLOWED US TO TAKE THE FINAL STEPS TO GET TO CLINICAL TRIAL AND WE WERE ABLE TO GENERATE CLINICAL TRADE MATERIAL WHICH ARE EXPENSIVE AND WITH ADVENTURES THEY'RE SINGLE COULD LAST FOR A LONG TIME AND WE SEE WHAT HAPPENS IN MODELS AND WE DID ALL THE STUDIES FOR THE FDA TO APPROVE THE PILE AND WE HAVE TWO PATIENTS AND WE SAW BENEFITS AND WE STARTED WITH VERY LOW AND SINGLE TO THE TREATMENT THAT CAN'T BE TAKEN AWAY WHEN YOU PUT IN IT STAYS IN THE CELLS AND EVENTUALLY THE TECHNOLOGIES WERE LICENSED AND ALL OUR DATA THAT HAD SUPPORTED THAT INITIAL CLINICAL TRIAL WAS A PHASE TO STUDY THAT IS STILL ON GOING TODAY SO ONE OF THE THINGS WE'VE DONE IS PARTICIPATED IN THE ANNUAL MEETING OF THE FONDATION IT'S CALLED THE FAMILY MEETING TO ACTUALLY MEET THE PATIENTS AND NOT ONLY MEET THE PATIENTS ACTUALLY EDUCATE THEM AS WELL IN WHAT WE'RE TRYING TO DO WITH THE TECHNOLOGY. OVER THE YEARS, YOU WOULD START TO GET QUESTIONS THAT WERE EXTREMELY SPECIFIC TO THE TECHNOLOGY SO THERE'S VERY, VERY CLEAR THAT INDIVIDUALS THAT PARTICIPATED IN THOSE MEETINGS ON A YEARLY BASIS STARTED TO REALLY UNDERSTAND THE BASICS OF THE TECHNOLOGY AND THAT IS ALSO IMPORTANT FROM THE POINT OF VIEW OF THE PATIENT COMMUNITY TO REALLY HAVE A GOOD SENSE OF WHAT IS IT THAT IS BEING DONE? ON THE SURFACE IF YOU LOOK AT T THE IDEA OF PUTTING GENES BACK INTO YOUR BODY CAN BE SCARY. NOW A LAYS A LITTLE BIT LESS BUT NONE THE LESS, IT'S STILL A LITTLE BIT OF A DAUNTING IDEA. IT'S NECESSARY THE COMMUNITY GETS EDUCATED, LEARNS THROUGH THE RESEARCH SOURCE THAT THERE'S A DEGREE OF TRUST THAT IS PRETTY IMPORTANT TO DEVELOP OVER TIME AND WE CONTINUE TO GO TO THE FAMILY MEETINGS AND ARE COMPLETELY TRANSPARENT WITH THE FAMILIES AND THE FOUNDATIONS WHERE WE ARE TODAY AND WHAT ARE THE PROBLEMS THAT WE HAVE AND FORTUNATELY NOT MANY NOWADAYS AND WE CONTINUE TO WORK VERY, VERY CLOSELY WITH THIS FOUNDATION SO BASED ON THIS EXPERIENCES, IT'S A SECOND PROGRAM FOR ANOTHER STORAGE DISEASE THAT ACTUALLY WE COLLABORATE WITH A COLLEAGUES THAT WORKS IN THE BUILDING 10, THE CLINICAL RESEARCH CENTER AT NIH SO THIS IS A NICE STARTED WITH AND SO BASED ON THIS EXPERIENCE, WE TOOK THAT PROGRAM AND DEVELOPED IT ALL ON OUR OWN SO WE WORD THAT YES ACADEMICS CAN AND DEVELOP AND LEAD PROGRAMS TOWARDS CLINICAL TRIALS AND IT REQUIRES A LOT OF PERSONAL AND THERE'S NO QUESTION ABOUT THAT AND YOU HAVE IF HE TAKE YOUR OWN HANDS AND DO-IT-YOURSELF AND YOUWORK WITH THE THINGS ARE DONE PROPERLY AND OF COURSE THEY HAVE TO FIT THE CRITERIA AND THE REQUIREMENTS THAT THE FDA HAS BASED ON THIS EXPERIENCE TO START THE TRANSLATIONAL INSTITUTE FOR THERAPEUTICS UMAS SCHOOL TO TRANSLATE THAT EXPERIENCE TO TRANSLATE FROM THE BENCH TO THE CLINIC IN ANNIE MISH MANNER AND WHAT WE UNDER UP WORKING ON ARE RARE DISEASES AND WE'RE CONTACTING BY PATIENTS AND THE PARENTS AND WE STARTED TO FORM PARTNERSHIPS WITH THE FAMILIES AND WE ALWAYS ADVISE THEM TO FORM A FOUNDATION AND THERE CAN BE A FORMAL THEY STRUGGLED TO RAISE MONEY BECAUSE WE'RE TALKING ABOUT AGAIN LIKE DISEASE WHERE THERE MAY BE 10 PATIENTS KNOWN WORLD WILD AND WE CAN MAN MAKING A COMMERCIAL CASE IS A BIT DAUNTING AND I DON'T MAKE THAT COMMERCIAL CASE SO WE'RE WORKING VERY CLOSELY WITH THE FOUNDATION THAT WAS STARTED BY A FAMILY AND THIS IS ACTUALLY EXAMPLES OF THAT WE HAVE NOW ON GOING AND TO RAIL' TAKE IT TO THE SMALL CLINICAL TRIALS AND HOPEFULLY SHOW EFFICACY AND MAYBE THERE'S SOME THAT WANTS TO TAKE IT OVER AND O NEVER IT OVER AND THESE THERAPIES HAVE MADE IT AVAILABLE AND THAT SUFFER FROM RARE CONDITIONS THAT MAY IN FACT HAVE THE CATEGORY OF THE RARE DISEASES AND ULTRARARE DOESN'T A PLOY TO THIS ONE SO OUR EXPERIENCE HAVE BEEN BROAD AND I WORK WITH FOUNDATIONS THAT HAVE SUFFICIENT FUNDS THAT REALLY CAN THROW MANY ERRORS AT LODGE OF TECHNOLOGY. ONE IS THE FAMILY FOUNDATION THAT FUNDS N F-1 AND THEY HAVE ENOUGH RESOURCES THAT THEY CAN ACTUALLY FUND A BROAD ARRAY OF MOLECULAR TECHNIQUES TO FIND THERAPY FOR NEURO FIBRO MITO SIS SO WE WORKED FROM FOUNDATIONS WHERE OF COURSE YOU KNOW, YOU HAVE TO BE VERY, VERY CAREFUL WITH THE EXPERIMENTS ASK MAKE SURE NOT THAT YOU ARE NOT DOING MORE THAN ARE NECESSARILY NEEDED TO GO TOWARDS THE CLINICAL TRIAL AND ALWAYS OF COURSE MAINTAINING IN MIND THE SAFETY AND AT LEAST HAVE A GOOD CHANCE OF HAVING SOME EFFICACY AND WHATEVER IT IS THAT BEER DOING SO IT'S A BROAD EXPERIENCE AND I THINK DEVELOPING VERY CLOSE COLLABORATION AND CONTACT WITH THE FOUNDATIONS AND TO BE COMPLETELY TRANSPARENT ABOUT THE GOOD AND THE BAD AND THE TERRIBLE AND IT'S REALLY IMPORTANT FOR THE FOUNDATIONS OF THE PATIENTS SO THAT THEY REALLY UNDER THE SCIENTIFIC PROGRESS AND AT THE END OF THE DAY THEY TRUST WHAT YOU ARE DOING CAN MAKE A DIFFERENCE AND MOVE THE FIELD FORWARD FOR PARTICULAR DISEASES FOR WHICH NOWADAYS WE DON'T HAVE THERAPIES AND SO I DID NOT KNOW HOW TO PUT THIS ALL IN SLIDES SO I JUST GAVE YOU A FAIRLY LONG MONOLOGUE ON THIS AND I'M HAPPY TO ANSWER QUESTIONS LATER ON. THANK YOU FOR YOUR ATTENTION. >> THANK YOU. COULD ALL OF THE SPEAKERS PLEASE JOIN ME ON VIDEO. WE HAVE ABOUT 10 MINUTES LEFT AND I HOPE, I SEE ONE QUESTION AND I'LL GET US STARTED AND ENCOURAGE OTHER QUESTIONS TO COME IN. I THINK THAT JIG SA METAPHOR RESINATES WITH MANY OF YOU AND WHERE A NON-PROFIT CAN HELP UNDERSTANDING THE LANDSCAPE AND HELPING ALL THE DIFFERENT TYPES OF STAKEHOLDERS FIT TOGETHER. DURING THE OTHER WAYS, ONE THING THAT I DIDN'T HEAR AS MUCH ABOUT WAS CONVENING, AND I THINK YOU MENTIONED THE FAMILY FOUNDATIONS ANNUAL MEETING, FOR EXAMPLE, AND HAVE YOU GUYS SEEN EXAMPLES WHERE YOU FEEL THAT THE NON PROFITS WORK IN CREATING SETTINGS TO CONVENE DIFFERENT TYPES OF STAKEHOLDERS WITHIN ACADEMIA OR ACROSS ACADEMIA HAS BEEN PIVOTAL TO THE ADVANCES IN YOUR FIELD AND I'LL LEAVE IT TO YOU WHICH OF YOU TO RESPOND? >> CERTAINLY. IT'S GOOD TO INVITE ACADEMIC INVESTIGATORS OF COURSE BUT COMPANIES THAT MIGHT SHALL INTERESTED TO COME TO YOUR ANNUAL MEETINGS. IT'S BEEN REALLY KAI I THINK WITH SOME OF THE DISEASES WE WORK ON TO CONVINCE THE COMPANIES THAT THERE ARE PEOPLE WHO REALLY WANT TO HAVE A TREATMENT AND ARE WILLING TO DO WHAT THEY CAN TO HELP I THINK IN PARTICULAR TO SEE WHAT THE DISEASES ARE LIKE. I ENCOURAGE THAT FROM PATIENTS TO INDUSTRY ARE VERY USEFUL. >> I JUST WANT TO CALL OUT TO ME, IT RESINATED SO MUCH TO SEE DIFFERENT STAKEHOLDERS PUT ON TO EACH OF YOUR DIFFERENT JIG SA PUZZLE AND I WORK AT THREE DIFFERENT FOUNDATIONS OR ASSOCIATIONS AND IT'S SUCH AN EXAMPLE OF ABOUT HOWDY PENDING ON THE FIELD OF SCIENCE AND THE MISSION OF THE ORGANIZATION AND THE STAKEHOLDERS YOU NEED TO WORK WITH AND UNDERSTANDING WHERE THE STAKEHOLDERS ARE COMING FROM AND BEING EVOLVING OVER TIME. WONDERFUL QUESTION IN THE CHATBOX. THE FOUNDATION ARE ALTRUISTIC WANTING TRIALS TO BE SUCCESSFUL, WHAT IS A FAIR ASK OF FUNDERS OR NIH OR PHARMA AND INDUSTRY TO HELP WITH FOUNDATIONS WHO ARE TRYING TO SUPPORT RECRUITMENT IN RESEARCH, FOR EXAMPLE. IS IT REASONABLE FOR FOUNDATIONS TO MONETIZE THE ACCESS TO PATIENTS THAT THEY HAVE CULTIVATED AND I THINK THIS IS A IMPORTANT QUESTIONS THAT NEEDS STAKEHOLDERS ANSWERING IT BECAUSE THERE'S ANSWERS IN BIOETHICS THAT WE DON'T HAVE IN THE PANEL RIGHT NOW BUT I WOULD LOVE TO HEAR THOSE OF YOU FROM THE ACADEMIC RESEARCH WORLD AND THE INTRAMURAL RESEARCH WORLD'S. WHAT DO YOU THINK? >> WELL, I WAS STARTING TO RESPONDED IN THE CHAT. I WOULDN'T PUT ANY BARRIERS TO RECRUITMENT. I THINK IT'S GOOD FOR THE FOUNDATIONS AND MAYBE I'M NOT READING THIS CORRECTLY. IT HELPS FOR THE FOUNDATIONS TO CONVINCE THE ACADEMIC INVESTIGATORS FIRST AND THE COMPANIES THAT THEY'RE GUNG HO GETTING AN EFFECTIVE TREATMENT AND WILLING TO DO WHAT THEY CAN WITHOUT CHARGING FOR IT AND MAYBE I'M READING THIS BACKWARDS? >> FOUNDATION NOWADAYS AND THERE'S A BIG TREND IN THIS IS THAT IT USED TO BE THAT FOUNDATIONS ARE PROVIDING FUNDING AND THAT'S ABOUT IT AND NOWADAYS MOST FOUNDATIONS WANT PART OF THE IP THAT IS GENERATED. I THINK IT'S COMPLETELY FAIR. NOW, THE NOTION OF MONETIZING ACCESS TO PATIENTS, WHILE YOU PUT A BARRIER THERE AND YOUR REPRESENTING THE PATIENTS AND IN MY OPINION, THE REAL OF THE FOUNDATION AND TO FACILITATE AS MUCH AS POSSIBLE AND RECRUITING PATIENTS THAT'S THE GOAL AND THE PROCESS THEY CAN HAVE THE INDUSTRY SUPPORT NATURAL HISTORY STUDIES AND IT'S FABULOUS AND I PERSONALLY DON'T THINK THAT CHARGING COMPANIES TO GET ACCESS TO A LIST OF PATIENTS IS SOMETHING THAT IT WILL WORK BECAUSE COMPANIES WILL BE ABLE TO GET AROUND THAT SO IT FALLS OUT OF THE MISSION OF A FOUNDATION TO DO SO AND IT'S A WAY TO RAISE MUND FOR THE FOUNDATION BUT THERE'S OTHER WAYS IT CAN BE DONE SO RESHAPING HAVE A DIM VIEW OF THAT. >> I'M HAPPY TO MAKE A COMMENT ALSO. I KNOW PAUL WELL AND I'VE HAD CONVERSATIONS WITH THIS IN THE PAST AND ONE OF THE GROUPS WITH PAUL, ONE OF THE GROUPS THAT WE COLLECT DATA AND IT'S GOING ON IN A PERSON WAY FOR YEARS AND FIREFIGHTERS CONSISTENT WITH BEING ABLE TO DO THAT AND IN OTHER WORDS DO WE HAVE TO RECONSENT OR THINK ABOUT IT IN A DIFFERENT ETHICAL WAY AND IT'S AN 'EM PEDESTRIANMENT WITH A NETWORK OR THAT YOU ALL WORK WITH. >> WE'VE ANOTHER QUESTION COME IN FROM KAITLYN AND I'M GOING TO PARAPHRASE 2 AND I HOPE I DO IT JUSTICE, KAITLYN, ARE ROLES AROUND THE NON-PROFIT AND THE ACADEMIC RESEARCHERS AND SOMETIMES THE NON-PROFIT HAS CONTRIBUTION OF SCIENTIFIC SUPPORT LIKE STUDY DESIGN, ANALYSIS AND DISSEMINATION AND I'VE SEEN OTHER NON PROFITS CURATE DATABASE IS OR INVEST IN DO YOU HAVE A DEEPER RELATIONSHIP WITH SCIENTISTS AT DIFFERENT NON PROFITS? TO BE HONEST, THERE ARE SO MANY EXAMPLES THAT I'M HAVING A HARD TIME COMING UP WITH JUST A FEW BUT JUST TO START, WITH THE MAJOR SOME OF THE MAJOR ORGANIZATIONS AND THERE'S MANY ALS GROUPS AND NON PROFITS THAT HAVE ENGAGE WITH RESEARCHERS AND ACADEMIC CENTERS AND AND THE ALS ASSOCIATION HAS BEEN FUNDING MANY COMPLEX RESEARCH STUDIES AND I MENTIONED A FEW WHEN I PRESENTED THIS AND THERE ARE SO MANY MORE AND YOU MAY RECALL AND THE CHALLENGE AND THAT WAS GREAT OPPORTUNITY FOR THE ALS ASSOCIATION TO HAVE MORE FUNDS AND IT'S PHENOMENAL HOW THEY DISTRIBUTED THAT TO ALLOW IMPORTANT RESEARCH TO HAPPEN AND AGAIN, JUST ONE OF THE MANY EXAMPLES THAT COULD MAKE BASED ON ALS ASSOCIATION FUNDING AND MANY NEW JEANS FOR ALS HAS BEEN DISCOVERED BECAUSE OF THE FUNDING THAT THEY DISTRIBUTE SOD CLEARLY ASSOCIATIONS LIKE THIS ONE LIKE THE ASSOCIATION HELPED NOT JUST FOR RECRUITMENT OR COLLABORATION WITH THE PATIENT COMMUNITY BUT TO ACTUALLY FUND RESEARCH THAT HAS PROVEN TO BE TRANSFORMATIVE AND IT'S ONE OF THE EXAMPLES AND OTHER ASSOCIATIONS LIKE FOR EXAMPLE THE MDA OR MUSCULAR DYSTROPHY ASSOCIATION HAVE A MOVING REGISTRY THAT HELPS AMOUNT IN TERMS 6 KNEW OWE MUSCULAR DISEASE AND PROVIDES INFRASTRUCTURE FOR RESEARCH FOR NATURAL HISTORY STUD EYES AND IT COULD GO ON AND ON SO ESSENTIAL LOW THERE'S SEVERAL EXAMPLES AND I WOULD LIKE TO EMPHASIZE THE IMPORTANCE OF THIS RESEARCH SOMETIMES FOUNDATIONS FUND HIGH-RISK AND HIGH REWARD PROJECTS THAT OTHERWISE WOULD TAKE A LONG TIME TO GET FUNDED THROUGH MORE TRADITIONAL SOURCES SO THEY HE CAN SELL RATE THE PROGRESS. >> ONE MINUTE WARNING. >> YOU ARE MUTED. >> ONE EXPERIENCE TO JUST DESCRIBE REALLY BRIEFLY IS CONVINCING BIOGEN TO TAKE ON SPINAL AND I WENT THERE WITH BY OUR INSTITUTE DIRECTOR AT THE TIME AND SAY COUPLE FOUNDATION AND HAVE I WELL-INFORMED COUPLE AND GETTING OUT TO BUY ONTO THE IDEA AND WORKING ON A RELATIVELY RARE DISEASE LIKE SPINAL MUSCULAR ATROPHY IS GETTING THERAPEUTICS GOING FOR THAT DISEASE AND SO IT WAS A REAL TURNING POINT AND IT WAS FUN TO BE THERE FOR THAT. >> IT WAS ASPIRING. WE'RE ALMOST OUT OF TIME. >> I THINK THE INCOMPETENTER ACTION WITH THE PATIENTS OF MULTIPLE IS IMPORTANT AS A REALITY CHECK BECAUSE SOMETIMES CERTAIN TESTS AND FREQUENCIES ARE PERFECTLY LOGICAL AND GIVEN THE CONDITIONS OF PATIENTS OFTEN THAT LEADS TO VERY HIGH RATES OF ATTRITION AND CLIP CAL TRAILS AND SO IT'S IMPORTANT TO HAVE THE SENSE OF WHAT PATIENTS ARE LIKELY TO DO AND WHAT WOULD LIKE TO BE DONE AND THOSE TWO COULD BE VERY DIFFERENT. >> IT WAS REALLY WONDERFUL TO HEAR FROM ALL OF YOU WITH ALL OF YOUR REMARKS. WE ARE AT TIME. IT'S BEEN AN HONOR TO HAVE YOU WITH US TODAY AND AN INSPIRATION TO HEAR THE SUCCESS STORIES YOU'VE BEEN ABLE TO SHARE. >> THANK YOU. THANK YOU. >> THANK YOU ALL. THANK YOU ALL OF OUR SPEAKERS FOR THAT AMAZING SESSION. SO, I AM GOING TO TAKE US INTO DAY ONE AND THE MEETING BEFORE I DO THAT AND I WANT TO REMIND EVERYONE THERE'S AN OPPORTUNITY TO SCHEDULE ONE-ON-ONE MEETINGS WITH NIH STAFF WE'VE BEEN PLACING THE LENGTH IN THE CHAT AND YOU CAN CHECK YOUR E-MAIL IF YOU ARE REGISTERED FOR THE EVENT AND IF YOU ARE NOT REGISTERED, YOU CAN CERTAINLY STILL REGISTER NOW OR TOMORROW AND ADDITIONALLY TOMORROW AVERAGE, THERE'S BREAKOUT SESSIONS FOR NON NIH PARTICIPANTS OF THE MEETING AND THERE'S A SURVEY THAT WE WOULD LOVE FOR AWFUL YOU TO FILL OUT TO HELP US FIGURE OUT TO BREAK OUT ROOMS SO WITHOUT FURTHER ADIEU OUR NEXT SESSION IS TITLED ENGAGING TRAINEES IN EARLY STAGE SCIENTISTS HOW TO WORK WITH ACADEMIC RESEARCHERS AND THESE ARE KIND OF LIGHTENING TALKS, IT'S A LITTLE BIT MORE OF A ROUND-ROBIN FIRESIDE CHAT WE'RE GOING TO AND WE'RE DELIGHTED TO HAVE Dr. NINA SCHOR MODERATE THE SESSION AND NINA IS THE SCIENTIFIC DIRECTOR OF NINDS BEFORE COMING TO NIH, SHE SPENT 20 YEARS ON FACULTY AT THE UNIVERSITY OF PITTSBURGH BECOMING THE CAROLE PROFESSOR THE PEDIATRIC RESEARCH AND THE CHIEF OF DIVISION OF CHILD NEUROLOGY AND THE DEPARTMENT OF PEDIATRIC AND DEAN FOR MEDICAL RESEARCH AND THE MEDICAL SCHOOL AND IN 2006, THE CHAIR OF THE AND AND AT UNIVERSITY OF ROCHESTER AND CHILD IN JANUARY 2018 AT WHICH POINT SHE CAME TO AND FOR 27 YEARS IT WAS CONTINUOUSLY FUNDED BY NIH AND SHE CURRENTLY LEADS THE DIVISION OF INTRAMURAL RESEARCH AND THE ULTRARARE GENE TARGETING THERAPY OR URGENT NETWORK AND SHE'S ALSO DONE TONS OF WORK FOR NINDS ON OUR PLAN FOR THE NEXT FEW YEARS AS WELL AS CAREER DEVELOPMENT PROGRAMS AT NINDS AND SHE SERVES AS A NEUROLOGY DIRECTOR FOR THE AMERICAN BOARD OF PSYCHIATRY AND ONCE WE HIT AUGUST, Dr. SHORE WILL MOVE ON AND UP IN NIH TO BE THE DEPUTY DIRECTOR FOR ENTIRE TEE OF NIH SO WITH THAT I WOULD LIKE TO PASS THE TORCH TO Dr. SHORE SO SHE CAN PICK US OFF WITH THIS NEXT SESSION. THANK YOU INFORM ALLFUL YOU FOR JOINING US THIS AFTERNOON AND I KNOW TIME IS JUST VERY THE SESSION THAT FOLLOWS WILL BE ON NOMINALLY ENGAGING TRAINEES AND EARLY STAGE SCIENTISTS BUT I'M HOPING WE GET A DISCUSSION GOING WELL BEYOND THAT AND THAT ENGAGES ALL OF YOU BECAUSE THIS IS A ROLE AND FUNCTION THAT NON PROFITS PLAY NOT ONLY FOR THE SCIENTIFIC COMMUNITY BUT ESPECIALLY FOR THE PATIENTS AND FAMILIES THAT THEY SERVE, ONE OF THE MOST CHALLENGING TIMES IN A SCIENTIST LIFE IS AT THE BEGINNING, HOW DO YOU CONVINCE ANYBODY THAT YOU ARE HOPING TO ANSWER AN IMPORTANT QUESTION AND IMPACTFUL QUESTION AND HOW DO YOU CONVINCE ANYBODY THAT YOU, WHOSE NAME IS NOT YET KNOWN, TO THE SCIENTIFIC COMMUNITY, THAT YOU ARE THE BEST PERSON TO DO THAT WORK AND I THINK MOST OF US AT THAT STAGE IN OUR CAREERS HAVE DEPENDED IN VERY LARGE MEASURE ON FUNDING FROM VERY DEDICATED NON-PROFIT ORGANIZATION AND THERE'S ANOTHER TIME IN A SCIENTIST LIFE THAT IS CHALLENGING AS WELL AND THAT IS RIGHT AROUND MID CAREER WHEN YOU'VE HAD YOUR INDEPENDENT GRANT FUNDING PERHAPS A FEDERAL AGENCY OR LARGER NON PRIVILEGE ITS AND YOU LOOK TO RENEW IT AND AGAIN, THEY CAN PLAY A ROLE TO HELP WITH PRELIMINARY D FOR THE RENEWAL OF A GRANT WITH HELPING TO DIRECT INDIVIDUALS IN A DIRECTION THAT IS MOST HELPFUL, IMPACTFUL, MEANING TO TO THE LIVES OF PATIENTS AND FAMILIES SO TO ADDRESS THIS WE HAVE FOUR SPEAKERS TODAY. THEY'RE EACH GOING TO GIVE RELATIVELY BRIEF TALKS AND NONE OF THEM HAVE SLIDES SO THIS WILL BE VERY IMPROMPTU KIND OF A DISCUSSION TWO OF OUR SPEAKERS ARE FROM ACADEMIA AND TWO ARE FROM NON PROFITS AND I'M GOING TO INTEREST THEM EACH BEFORE THEIR TALKS AND OUR FIRST SPEAKER IS Dr. DANIEL OKO BYE. HE GOT HIS PH.D IN NEUROSCIENCE AT NYU AND HIS MD ALSO FROM NYU AND HIS CLINICAL TRAINING HE DID AT THE UNIVERSITY OF MIAMI AND AT UCLA AND HE STAYED ON AT UCLA WHERE HE IS NOW FACULTY AND SPECIALIZES IN BEHAVIORAL NURROLOGY SO I WILL HAND THE FLOOR OVER TO Dr. OKOBI. >> GOOD AFTERNOON. I'M A BEHAVIORAL NEUROLOGY FELLOW AND CLINICAL STRICT OR AND JUNIOR ATTENDING IN SOME WAY AND I WORK WITH THE GROUP THAT WORKS ON DIFFERENT TYPES OF NEUROLOGICAL DISEASES AND TRADITIONALLY IT'S BEEN AT OF GENETIC BASED AWE AT THE SAME TIME AND EPILEPSIES AND I WANTED TO CONTINUE A LITTLE BIT TOWARDS DISEASE THAT A LOT OF PEOPLE HAVE BEEN THINKING ABOUT AND IT'S DEMENTIA AND WHEN I CAME OVER, TO WORK IN RESIDENCY AND I TALKED TO THE CHAIR AND I TALKED ABOUT LOOKING AT THE PROJECTS ABOUT WHAT KIND OF THE BEST QUESTIONS TO ANSWER AND WHAT THE FIELD IS KIND OF LOOKING FOR BECAUSE I ACTUALLY WORKED ON BEHAVIOR AND I HAD NOT REALLY THOUGHT ABOUT BEHAVIORAL NEUROLOGY AND BASICALLY, DEVELOPMENTAL DISABILITY IN THE ADULT POPULATION IT'S DEMENTIA AND NEURO PSYCHIATRY AND I HAD BEEN A MEMBER OF THE SOCIETY FOR NEUROSCIENCE AND I WANTED TO ATTACK IT TO LOOK AT THE CIRCUITRY AND DEMENTIA WHICH NOT HAS MANY PEOPLE HAVE AND MY IDEA OF LOOKING AT THE DEMENTIA WAS LOOKING AT NOT EVERYONE IS ACTUALLY LOOKING INTO SO ONE OF THE DEMENTIAS I WAS INTERESTED IN IS AT UCLA WE HAVE A SPECIALIST EXPERT THAT RUNS A FELLOWSHIP I'M A PART OF AND FOR TEMPORAL DEMENTIA IS MORE GENETIC THAN THE AVERAGE DEMENTIA AN AND IT TENDS TO BE E BEHAVIORAL VANT WHICH IS WHEN PEOPLE NOTICE THAT THEIR LOVED ONES CAN BE THEY MIGHT EATING SWEETS AND THEY MIGHT START TO GET VERY SEXUALLY FRANK IF THEY WERE LIKE A RESERVED PERSON SO LIKE A CHANGE IN-PERSON A LOT AND JUST BEHAVIORAL AUD TEES AND THAT PRE DATES WHAT WE THINK ABOUT DEMENTIA WHICH IS MEMORY ISSUES WHERE PEOPLE HAVE LANGUAGE ISSUES AND TO BE ABLE TO COMMUNICATE IS TO THOSE ARE THE TWO TYPES OF DEMENTIA AND WHAT I'M BUILDING ON IS THE A LOT OF PEOPLE HAVE BEEN WORKING ON THIS AND IN SOME RESPECT SO A LOT OF PEOPLE HAVE BEEN WORKING ON THE GENETICS AND UNDERSTANDING HOW THIS IS IN THE BRAIN CAUSES LOTS OF DAMAGE AND I WANT TO BE ONE OF THE PEOPLE O TO UNDERSTAND IT IS AS THE DEMENTIA IS RAIN BECAUSE WE ACTUALLY CAN RESTRAIN CIRCUITS AND PEOPLE HAVE BEEN LOOK AT SO I WANT TO SEE ALSO THERE'S SOMETHING THERE IN TERMS OF THE ACTUAL CIRCUITRY BEING BROKEN DOWN SO I CAN AT LEAST ESTABLISH THAT IF WE TROY TREATMENTS OF ACTUAL LOW CAN REWIRE OR FIX WHAT IS BEING BROKE EASTBOUND DOWN AS THE D DEMENTIA IS HELPIG IT'S A WAY TO BRING DECELERATION TO THE PATTERN OF DEMENTIA AND THE THING THAN IS NOT UNIQUE TO THE FRONTAL TEMPORAL DEMENTIA IS THERE IS NOT REALLY A TREATMENT THAT IS REVERSING THE FUNDAMENTAL DAMAGE THAT IS HAPPENING YET AND AS WE HEARD EARLIER ABOUT FDA APPROVALS FOR MEDICATIONS JUST BECAUSE THE FD APPROVALS THE MEDICATION DOESN'T MEAN THAT EVERYONE WHO NEEDS IT OR GETS ACCESSIBLE THERAPEUTIC RESOLUTION SO WE NEED TO DO A LOT OF DIFFERENT THINGS 245 WE DON'T KNOW WHAT WILL WORK BUT IF WE FIND SOMETHING, WE SHOULD TRY IT. SO I'LL STOP THERE BECAUSE I KNOW THERE ARE OTHER PEOPLE AND YOU MIGHT BE ON MUTE. >> THANK YOU AND OUR NEXT SPEAKER IS Dr. ERIC MORROW. Dr. MORROW GOT HIS PH.D IN GENETICS AND NEURO DEVELOPMENT AT HARVARD AND OF MOLECULAR CELL BIOLOGY AND Dr. MORROW. >> THANK YOU, VERY MUCH, Dr. SCHOR. WE GET THUMBS UP, I'M THRILLED TO BE PARTICIPATING IN THIS CONFERENCE AND I'VE ENJOYED THE SESSIONS. SO AS Dr. SCHOR SAID, I'M AT BROWN UNIVERSITY AND I'M A PHYSICIAN SCIENTIST AND I WORK IN A BASIC SCIENCE DEPARTMENT BUT I ALSO WORK IN CLINICAL DEPARTMENTS WHERE YOU DO RESEARCH WITH FAMILIES WITH NEURO GENETIC DISORDERS AND IT'S MY CLINICAL RESEARCH SO THE PATIENTS ORIENTED WORK WHERE I'VE COME INTO CONTACT WITH DIFFERENT FAMILY-LED NON PROFITS SO ONE GENETIC DISORDER IS CHRISTIANSON SYNDROME AND WE NAMED IT AFTER THE SPEED TRICK AN WHO NEWER LOGIC DISORDERS CAUSES DEVELOPMENTAL DISABILITY AND AS WELL AS NEURODEGENERATIVE FEATURES AND I WAS WORKING ON THIS CONDITION IN MY BASIC SCIENCE LAB AND HAD NOT STARTED WORKING WITH THE FAMILIES AND I GOT AN E-MAIL OF AN EFFECTED, FROM THE MOTHER OF A EFFECTED PATIENTS PERSON, MALE WITH THIS CONTINUE AND SAYING THAT SHE JUST GOT THE GENETIC DIAGNOSIS AND SHE DOESN'T KNOW ABOUT THE CONDITION BUT THEY READ A PAPER FROM READING THE INTERNET AND WHAT I SPEAK WITH HER ABOUT IT AND THIS WAS 2010 OR 2011 AND HER NAME WAS DEBBIE NASH AND I'VE HAD THE HONOR OF WORKING WITH DEBBIE WHO FOUNDED THE CHRISTIANSON SYNDROME ASSOCIATION AND IT'S A NON-PROFIT TO RAISE AWARENESS ABOUT CHRISTIANSON SYNDROME AND SUPPORT RESEARCH AND SO I'VE HAD THE HONOR AND PRIVILEGE OF WORKING WITH FAMILIES WHO RUN NON PROFITS FROM THE VERY EARLY STAGES AND THE FOUNDING OF THE KRISTEN SON FOUNDATION WENT LIKE THIS AND DEBBIE HAVE YOU MET OTHER FAMILIES AND ONE OF THE FAMILIES STARTED A Facebook GROUP AND WOAD OFFERED AT BROWN UNIVERSITY TO DONATE SPACE OR TO THE FIRST CONFERENCE CAME TOGETHER AT THAT FIRST MEETING IN 2013 AND AT BROWN UNIVERSITY AND THE RESEARCH HAS GROWN AND DEVELOPED AND IN MY GROUP AND IN OTHER SCIENTIFIC OVER THE LAST DECADE AND IN 2015 AND WE WERE FORTUNATE TO GET THEY HAVEN'T MET FAMILIES THAT ARE EFFECTED AND THESE CONFERENCES ARE WONDERFUL TO HAVE THE OPPORTUNITY TO BRING THEY'RE MORE AND MORE CLINICAL AND MORE AND MORE INTERNATIONAL AND THERE'S AN INTERNATIONAL AND THERE'S A SURE PEE AN WHERE TIVE FAMILIES AND LANGUAGES AND AND ONE POINT THAT I WILL MAKE THAT I THINK HAS BEEN MADE AND ON TRAINEES AND ALSO FOR HELPING THE SO FOR EXAMPLE AND TO CHRISTIANSON DIN DROP WE'VE RECEIVED FUNDING AND CHRISTIANSON AND AND LARGER ONE SUCH AS THE OUR WORK OF OTHER PEOPLE IN THE WORLD THE MORE ESTABLISHED NON PROFITS AND AUTISM AND EP DEPOSIT SEE AND LARGER AREAS WHERE THE RARE GENETIC DISORDERS COME TOGETHER AND REALLY CRITICAL AND SO I THINK THAT RELATIONSHIP HAS BEEN CRITICAL AND BEFORE I WRAP-UP IS THERE HAVE BEEN I THINK SOME VERY, VERY INTERESTING FUNDING GRANTS AND MAYBE WE CAN DISCUSS THIS IN THE DISCUSSION PERIOD AND FROM NEW FOUNDATION AND MONDAY PROFITS THAT REPRESENT COP AGO RACES AND BETWEEN THE NON PROFITS AND THE RESEA RESEAS THAT WOVE HAD PLIED FOR IS THE ZUCKERBERG INSTITUTE HAS FUNDED A SERIES OF GRANTS AND THE AREA OF RARE GENETIC DISORDERS ONE CALLED RARE IS ONE AND THE SECOND ONE LAST MAY AND ON THE NON PROFITS ARE OWE ON CANNES TOGETHER ON A SCIENTIFIC ENDEAVOR WHICH REALLY ORGANIZES THE COLLABORATION BETWEEN THE ACADEMIC RESEARCHERS AND THE NON-PROFIT IN THE APPLICATIONS I THINK THESE CONFERENCES IN THE EXCHANGE WHETHER IT'S E-MAIL, SOCIAL MEDIA, ARE CRITICAL IT'S A TWO-WAY STREET AND INSPIRING COLLABORATION AND THE RELATIONSHIPS BETWEEN THE EARLY FORMING NON PROFITS AND THE MORE ESTABLISHED IS I THINK THE THEME IS WE HAVE A LOT TO LEARN FROM EACH OTHER AND THANK YOU. >> THANK YOU, Dr. MORROW. OUR NEXT SPEAKER IS Dr. DIANNE BOVENKAMP. SHE GOT HER PH.D FROM QUEEN'S UNIVERSITY IN KING STON, ONTARIO, AND WENT ON TO DO A POSTDOC TO BAL FELLOWSHIP. SHE'S VICE PRESIDENT FOR SCIENTIFIC AFFAIRS AT BRIGHT FOCUS FOUNDATION. Dr. BOVENKAMP. >> GREAT, CAN EVERYBODY HEAR ME BECAUSE I WAS TOLD MY VOLUME WAS LOW BEFORE? >> SOUNDS GOOD. >> OK. SO I'M WITH BRIGHTFOCUS, 12 YEARS NOW IN VARIOUS CAPACITY BUT RIGHT NOW I'M ALMOST ALL ASPECTS OF OUR GRANTING. LIKE OTHER ORGANIZATIONS, THAT WE -- OTHER NON PROFITS, THE SCIENCE HELPS WITH THE FUNDRAISING AS WELL AS THE COMMUNICATIONS AS WELL AND SO WE'VE BEEN NEXT YEAR IS GOING TO BE OUR 50th ANNIVERSARY ACTUALLY AND WE USED TO BE CALLED AMERICAN HEALTH ASSISTANCE FOUNDATION AND WE REBRANDED IN 2013 SO SOME OF YOU WHO HAVE HAD AN AWARD THAT WAS US AND SO YEAH SO WE'VE BEEN AROUND A WHILE AND YOU KNOW, FOR THE LAST TWO YEARS, WE'VE GIVEN $25 MILLION IN GRANTS EACH YEAR IN OUR THREE PROGRAMS SO WE FUND ALZHEIMER'S DISEASE, GLAUCOMA AND MACULAR DEGENERATION AND WHAT'S THE CONNECTION? SO WHEN WE STARTED OUT SMALL IN 1973, IT WAS REALLY THE THING THAT BINDS IT ALL TOGETHER IS THEIR DISEASES OF AGING, RIGHT. AND A LOT OF PEOPLE GET THEM WHEN THEY GET OLDER AND A LOT OF THE FOUNDERS FAMILY MEMBERS HAD THEM AND NOW ACTUALLY THERE'S A LOT OF SCIENTIFIC TIES SO THEY'RE ALL GENERATIVE DISEASES AND AT THE FOUNDATION WE HAVE OUR OFFICIAL JOURNAL IS MOLECULAR DEGENERATION AND A LOT OF PUBLICATIONS THERE WITH BOTH THE VISION AND THE BRAIN CONNECTION THAT WE FUND. WE FUND RESEARCH AS WELL AS WE'RE CONNECTIONING WITH I THINK IT'S ABOUT 500,000 PEOPLE ACROSS THE U.S. AND BEYOND BECAUSE WE'RE AN INTERNATIONAL FUNDER AND WE GIVE NOT ONLY GRANTS TO SCIENTISTS AND OTHER NON MONETARY SUPPORT AS I'LL JUST BRIEFLY ALIGN LATER, BUT WE ALSO COMMUNICATE SCIENCE TO INDIVIDUALS AND ACTUALLY WE HAVR COMMUNICATIONS OF ALL OF THE RESEARCH THAT WE FUND AND THE BIG ACHIEVEMENTS IN THE FIELD TO EFFECTED FAMILIES AND WE HAVE A 1-800 NUMBER SO WE STRADDLE THE LINE BETWEEN I WOULD SAY THERE'S FOUR WAYS THAT I'LL BREAK IT DOWN THAT WE ENGAGE TRAININGS IN EARLY STAGE SCIENTISTS AND I KNOW THAT EVERY NON-PROFIT IS DIFFERENT AND SOME PEOPLE DO MORE AND ONE OF THE FINANCIAL SO WE HAVE GRANTS IF ALL THROW PROGRAMS AND FELLOWSHIPS AND ALL OF THEM AND PEOPLE WHO ARE WE HAVE NEW INVESTIGATOR AWARDS AS WELL AS SEASONED INDIVIDUALS AWARDS OR STANDARDS AWARDS SO WE FUND THE WHOLE GAMUT AND WE ARE MORE OF A TRADITIONAL, WE TEND TO NOT NECESSARILY SAND OUT SPECIFIC IDEAS OR SPECIFIC RFPs IT'S INVESTIGATOR INITIATED AND THIS IS THIS COULD BE WHERE WE WE ALSO REALLY TRY AND ENCOURAGE PEOPLE LIKE DANIEL TO COME IN AND WHO ARE EXPERTS IN ONE FIELD TO COME IN AND APPLY AND LIKE WITH ERIC WE ACTUALLY DO AGREE WE DO FUND IT'S GOOD FOR PEOPLE TO COME IN AND GET FUNDING TO SHOW THE CROSS OVER BETWEEN ALZHEIMER'S AND OTHER DEMENTIA OR MAYBE A RARE FORM OF MACULAR DEGENERATION SO PEOPLE SHOULD TRY AND AND WE'VE PARTNERED WITH OTHER ORGANIZATIONS BEFORE TOO SO YOU ARE HITTING ALL THE OF THE -- HOW DO YOU FIND THE FOUNDATION THAT IS IMPORTANT AND SO WE ACTUALLY DO HEALTH WITH NO HOUSE AND SOME OF THE OTHER THINGS THAT WE DO. WE HAVE SIGNATURE CONFERENCES WE CALL FAST TRACK AND IT'S A BOOTCAMP AND HEAR FROM INVESTIGATORS IN THE FIELD AND NETWORK WITH THEIR COLLEAGUES AND WITH SEASONED INDIVIDUALS AND AS WELL AS WE HAVE BREAKFAST AT MAJOR CONFERENCES WHERE PEOPLE CAN NETWORK AND WEBINARS AND THOSE ARE LEARNING HOW TO CONNECT. THERE'S OTHER WAYS THAT WE CAN HELP TO CONNECT TOO SO A LOT OF PEOPLE WHILE TRYING TO MAKE CONNECTIONS IN THEIR FIELD IT MIGHT NOT THINK ABOUT THE NON-PROFIT GRANTERS BUT NOT ONLY CAN PEOPLE CONNECT WITH US FOR FEEDBACK ON SPECIFIC GAINS WHEN THEY'RE APPLYING BUT ALSO WE KNOW A LOT OF PEOPLE IN THE FOR PROFIT AND OTHER NON PROFITS SO WE CAN INTRODUCE YOU TO OTHERS OR MAYBE WE MIGHT BE ABLE TO TELL YOU, YOU KNOW, HAVE YOU CONSIDERED APPLYING TO THIS ORGANIZATION SO IT'S A GOOD WAY TO TRY AND CONNECT AND I THINK THAT THE FINAL THING SO THERE'S THE NO HOW AND THE FINANCIAL AND THERE'S THE THE FOURTH WOULD BE WE HAVE AN ALLUMNI NETWORK SO DON'T STOP THINKING ABOUT THE NON-PROFIT ORGANIZATIONS WHEN THE FUNDING ENDS. WHEN WE'VE FUNDED YOU, A LOT OF TIMES WE'RE YOUR BIGGEST CHAMPIONS AND SO WE HAVE THINGS AND I THINK THAT THERE ARE SOME DOCUMENTS THAT I DID SHARE ASPECTASEXAMPLES. WE HAVE YEAR BOOKS WITH PEOPLE'S MUGSHOTS WITH DESCRIPTIONS AND YEAR BOOKS AND ANNUAL REPORTS AND RESEARCH AND BRIEF AND WE'LL BRING PEOPLE IN FOR TALKS AND TALK WITH MAJOR DONORS SO AND INTRODUCE YOU TO PEOPLE IN THE FIELD SO THERE'S A LOT MORE BUT WE SHOULD GET ON TO HAVING JENNIFER CHAT AND WE CAN HAVE DISCUSSIONS. >> GREAT, THANK YOU. AND OUR FINAL SPEAKER IS JENNIFER BRUMMET. SHE GOT HER BACHELORS DISEASE IN PSYCHOLOGY AT THE ILLINOIS WESLEY ANNE UNIVERSITY. HER PH.D IN PSYCHOLOGY AND BEHAVIORAL NEUROSCIENCE AT MICHIGAN STATE AND WORKED IN GOVERNMENT AGENCIES AND ORGANIZATIONS AND SOCIETY OF NEURAL SCIENCE, NSF, THE AMERICAN BAR FOUNDATION AND SHE'S CURRENT LEE THE MANAGER FOR RESEARCH AT THE AMERICAN EP LHEEPILEPSIES SOCIETY. >> I'M THE CLEAN UP HITTER HERE TODAY SO I'M GOING TO SPEND A FEW MINUTES GIVING A HIGH-LEVEL OVERVIEW OF HOW THE AMERICAN EPILEPSIES SOCIETY WORKS WITH EARLY CAREER INVESTIGATORS AND I WANT TO USE THE LAST PART OF MY TIME TO KIND OF DISCUSS A FEW OVERARCHING THEMES AND SOME LESSON LEARNED BEFORE WE LAUNCH INTO THE Q&A. SO FIRST, A QUICK OVERVIEW OF THE DIFFERENT WAYS THAT WE WORK WITH EARLY CAREER INVESTIGATORS AT THE AMERICAN EPILEPSIES SOCIETY. I WORK WITH SOME OF OUR VOLUNTEER COMMITTEES TO SUPPORT EARLY KA RO CAREER INVESTIGATORS INCLUDING NETWORKING OPPORTUNITIES AND WE DO THIS THROUGH ON-LINE TRAINING MODULES AND WEBINARS, EPILEPSIES, SPECIFIC ASSESSMENTS ON-LINE AND TARGETED CONTENT FOR EARLY CAREER AUDIENCE AND OUR ANNUAL SCIENTIFIC CONFERENCE AND INCLUDING CAREER PATH WAY SESSIONS AND ADDITIONAL INFORMATION SESSIONS TO HELP ON BOARD THEM TO THE SOCIETY IN OUR COMMUNITY. ONE OF OUR FLAGSHIP PROGRAMS THAT WE OFFER IS CALLED THE FELLOWS PROGRAM AND THOSE PROGRAMS PROVIDES SUPPORT FOR CLINICAL FELLOWS AND POSTDOC TO BAL RESEARCH FELLOWS TO ATTEND OUR ANNUAL SCIENTIFIC CONFERENCE AND DURING THIS PERIOD AT OUR MEETING, THEY'LL GET PROFESSIONAL DEVELOPMENT CONTENT AND THEY GET CONNECTED WITH MENTOR SO REALLY THE GOAL THERE IS TO PROVIDE THEM THIS NETWORKING AND ONBOARDING OPPORTUNITY TO OUR COMMUNITY. IN ADDITION TO THESE OTHER OPPORTUNITIES, WE ALSO PROVIDE GRANT FUNDING WHICH EVERYONE KNOWS IS CRITICAL FOR EARLY CAREER SCIENTISTS AND ABOUT 85% OF OUR GRANT FUNDS GO TO EARLY CAREER INVESTIGATORS AND CLINICIAN SCIENTISTS RANGING FROM THE PH.D STAGE 20 THE AND OF COURSE OUR GOAL IS TO MENTORING AND NETWORKING AND OPPORTUNITIES AND WE HAVE ALUMNI EVENTS INCLUDING A RECEPTION AT OUR CON YOU'LL CONFERENCE AND TO HELP CONNECT SOME OF OUR GRANTEES WITH OTHER NON-PROFIT PARTNERS THAT WE WORK WITH THAT MIGHT FOCUS ON VARIOUS EPILEPSIESES ARE GENETIC EPILEPSIES AND THE NINDS PROGRAM OFFICERS AND THAT'S REALLY PART OF OUR COMMITMENT TO YOU KNOW, WE WITHIN TO FUND THEM AND WE WANT THEM TO HAVE GREAT GRAND OUTCOMES WHEN SO BECAUSE OF THAT GOAL AS WELL WE TROY TO HELP TRAINEES AND LAUNCH THE GRA CORE AND WE SERVE AS GRANT REVIEWERS WHICH HELPS US BUT HOPEFULLY ALSO HELPING THEM CONTINUE TO DEVELOP THEIR OWN. SO I'LL USE THE NEXT THREE MINUTES TO TALK ABOUT SOME OF MY LESSON LEARNED OVER THE YEARS SO I'LL BE INTERESTED TO SEE WHAT THE OTHER PANELISTS AND TO ENCOURAGE THE NON-PROFIT ON THE CALL TO REALLY PUT A LOT OF WORK NO IDENTIFYING THE NEEDS OF YOUR COMMUNITY AND IT'S REALLY IMPORTANT TO UNDERSTAND THEIR TRADING ENVIRONMENTS AND CHARGES FACE AND CAREER TRAJECTORY AND ABOUT WHAT RESOURCES THEY NEED TO SUITE AND THIS CHANGES OVER TIME, RIGHT. SO THIS ISN'T NECESSARY ONE OFF TASK AND YOU KNOW THE FUNDING NEEDS MIGHT CHANGE OVER TIME. AND IN ADDITION TOP UNDERSTANDING THE NEEDS OF YOUR COMMUNITY, I WOULD ALSO PUT ONE POINT OUT THERE WHICH IS TO UNDERSTAND WHAT THE EARLY KA RAILROAD INVESTIGATORS WOULD LOOK TO YOUR ORGANIZATION FOR. NONE OF US IS NON PROFITS HAVE UNLIMITED CAPACITY OR FUNDS SO IT'S JUST REALLY HELPFUL TO MAKE SURE ONE EXAMPLE IS WE HAVE TO PUT A LOT OF IT WAS WELL ALIGNED WITH THE NEEDS OF OUR COMMUNITY AND UNDER UTILIZED SO WITH A LITTLE BIT DIGGING REAL REALIZED WHAT WE WERE OFFERING WAS OFFERED BY THE INSTITUTIONS AND SO WHAT T ALLOWED US TO TAKE A STEP BACK AND WORK TO REFINE OUR PROGRAM IN A WAY THAT REALLY HONED IN ON OR ORGANIZATION UNIQUE EX PER HE'S AND REALLY HELPED GET THOSE RESOURCES OUT THERE TO OUR EARLY CAREER INVESTIGATORS. AND THE SECOND BIG LESSON IS TO IDENTIFY WAYS TO MAKE WORKING WITH EARLY CAREER INVESTIGATORS MUTUALLY BENEFICIAL AND IT DOESN'T NEED TO BE EQUALLY BENEFICIAL BUT I THINK THE BEST RELATIONSHIP TEND TO BRING SOMETHING OF BENEFIT TO BOTH SIDES AND SO EVEN A GRANT PROGRAM CAN BE BENEFICIAL RIGHT AND OBVIOUSLY EARLY CAREER INVESTIGATORS AND HOCH YOU CAN TORCHBEARER TO PULL IN YOUR PAST GRANT TEES AS A VOLUNTEER FOR OTHER PROGRAMS OR A GRANT REVIEWERS AND OR BUILD AN ONGOING COMMUNITY AND PEOPLE WORKING ON A SPECIFIC DISEASE AREA OR REALLY ANYTHING OF THEY HAVE HAD A LOT OF CHANG HES FUNDING AND RESEARCH AND THE SO IN MY OPINION THE POSITIVE SPIN ON THIS IS THERE'S A LOT OF FUNDS ARE NOT PROFIT TO STEP IN AND IDENTIFY SOME EMERGING NEEDS AND OPPORTUNITIES TO WORK WITH TRAINEES WHETHER IT'S FUNDING PROGRAMS AND OR PROVIDING OTHER RESEARCHES AND SO I WILL END THERE AND I LOOK FORWARD TO THE Q&A. >> THANK YOU. SO, THIS IS YOUR TIME, YOU ARE AUDIENCE TO ASK US ANYTHING YOU WANT AND TO PERHAPS SUGGESTING THINGS THAT YOU WOULD LIKE TO HEAR DISCUSSION ABOUT. I'M HAPPY TO GET THINGS STARTED BECAUSE AS I HEARD EACH OF THE TALKS THE THING THAT STRUCK ME THE MOST, AND I SUSPECT THIS WILL COME AS NO SURPRISE AND IT IS INTERESTING TO ME THAN A NON-PROFIT ORGANIZATION CAN PLAY MANY, MANY DIFFERENT ROLES EVEN IN THE SPACE OF FUNDING EARLIER MID CAREER INVESTIGATORS AND IT SEEMS LIKE SOMETIMES YOU ARE ACTUALLY FUELING THEIR ABILITY TO CARRY OUT A BUNCH OF STUDIES AND THEY PERHAPS WANT TO DO TRY SO YOU GET THEM OFF THE GROUND AND KEEP THEM ENGAGE IN WHAT IT IS YOU ARE SOCIETY IS DEDICATING TO DOING AND I THINK SOMETIMES ALSO YOU HAVE THE OPPORTUNITY TO DIRECT WHERE THE FIELD AND MAKING FUNDING OPPORTUNITIES AVAILABLE THAT ARE TARGETED IN SOME WAY OR THAT RESPONDED TO SOME NEED OF YOUR PATIENT POPULATION AND FAMILIES OR OF YOUR ORGANIZATIONS SO IF I'M THE DIRECTOR OF A NON-PROFIT THAT HAS JUST MOVED INTO THE FINANCIAL SITUATIONS WHERE WE CAN THINK ABOUT FUNDING YOUNG INVESTIGATORS LET'S SAY WERE IN THAT SPECTRUM SHOULD I THINK ABOUT BEING? DO I PUT OUT SOMETHING VERY TARGETED THAT I KNOW IS EXACTLY WHAT OUR PATIENTS NEED IN THE NEAR TERM OR DO I PUT OUT SOMETHING THAT WILL HELP ME INVEST IN THE LONG-TERM FOR THE CAREER OF SOMEONE WHO IS JUST GETTING STARTED. >> I PUT MY HAND UP HERE. IF I YOU MAY, I THINK ALL OF THE ABOVE IF WE CONSIDER FINANCE AND ALL OF THE NON FINANCIAL SUPPORT THAT YOU CAN DO AND IF I LIKE IT IT TO ICE CREAM, MANY DIFFERENT FLAVORS OF ICE CREAM COME FROM MANY DIFFERENT ORGANIZATIONS AND ICE CREAM IS STILL DELICIOUS SO SO ONE OF THE EXAMPLE ARE WE HAVE A SMALL PERCENTAGE OF OUR PORT GOAL THAT GETS DIRECTED BUT I THINK THAT ONE OF THE THINGS WE DO SO OVER THE LAST COUPLE OF YEARS WE'VE HAD YOU KNOW JUST TO MAKE SURE WE HELP TO SUPPORT EVERYTHING AND WE LET THEM DECIDE WHICH ARE THE MOST, THE HIGHEST REACHING ABSTRACT AND ALSO I WANTED TO ADDRESS WHAT JENNIFER WAS SAYING ABOUT THE COVID I THINK THAT A LOT OF OUR ORGANIZATIONS HAVE BEEN HELPING RESEARCHERS AND WE'VE GIVEN OUT WHEN POEM COME HAVE TO DIRE NEED AND WE'VE ALSO ALLOWED REBUDGETING SO THERE'S A LOT OF THINGS THAT WE CAN DO AND THE ADD VOICE TO THE SCIENTISTS ARE YOU CAN'T GET IT IF YOU DO ASK SO KEEP IN CONTACT WITH YOUR NON-PROFIT GRANT ADMINISTRATOR AND ANYBODY ELSE WANT TO ANSWER IN HERE? I JUST SAID EVERYTHING. ALL OF THE ABOVE AND ALL FLAVORS OF ICE CREAM ARE DELICIOUS. >> ONE THING I WOULD SAY IS IT WAS SOMETHING THAT WAS MENTIONED OFF HAND AND ONE OF THE EARLIER SPEAKERS IN THE DAY AND IT'S JUST IF YOU START A DISEASE THAT YOUR KID HAS AND SOMEONE I THINK JUST MENTIONED A FEW MINUTES AGO, YOU START WORKING WITH OTHER PEOPLE ON Facebook AND RAISING MONEY AND THAT CAN BE THE THING THAT BRINGS AWARENESS FOR SCIENTISTS AND RESEARCH AND I MEAN Dr. MORROW KNOWS THIS BUT THERE'S A LOT OF MEDICINE OUT THERE SO LIKE EVEN IF YOU WANTED TO JUST DO NEUROSCIENCE, IT'S HARD TO GUARANTEE THAT YOUR DISEASES OF TAUGHT AND LIKE THE OVERVIEWS AND OF NEUROSCIENCE FOR MEDICAL STUDENTS AND GRADUATE STUDENTS SO WHAT PEOPLE ACTUALLY END UP DOING AND IN TERMS OF THE RESEARCH CAREER CAN BE ALMOST LIKE A RANDOM WALK LIKE SOME PEOPLE MIGHT JUST END UP WORKING ON RED SYNDROME BECAUSE YOU KNOW, OTHER PEOPLE WERE WORKING ON IT BUT IF YOU HAVE A RARE DISEASE AND YOU WANT TO BE FUNDED, IF YOU ARE ABLE TO SEE START GOING TO CONFERENCES LIKE AAS BECAUSE THAT IS A TERM EPILEPSIES IS AND YOU CAN GET PEOPLE'S ATTENTION AND PEOPLE ARE WORKING IN IT AND YOU HAVE THAT MONEY THAT CAN BE AND THERE WILL BE DOING AT LEAST EARLY STAGE IF NOT LATER STAGE RESEARCH ON THE DISEASE AND THEY FILE AWARE THE MONEY IS SO IF THAT MEANS THAT THEY END UP TURNING THEIR GENETIC KNOWLEDGE TO THIS KIND OF RARE DISEASE AND AS LONG AS SOMEONE IS GOT LIKE A PASSIONATE COMMITMENT TO THE PATIENTS OF THAT DISEASE AND PEOPLE WILL ACTUALLY START TO DO THAT RESEARCH. >> WE HAVE A MINUTE LEFT BUT THERE'S A QUESTION I WANT TO POSE THAT PENNY DACKS PUT IN THE CHAT. HOW CAN A NON-PROFIT MEASURE SUCCESS IN ITS INVESTMENTS AND EARLY CAREER AND IF A FUNDED TRAINEE LEAVES ACADEMICS RESEARCH A SUCCESS OR A FAILURE? >> JENNIFER OR ERIC, DO YOU WANT TO ANSWER? >> I CAN GIVE MY THOUGHTS AND IT'S NOT NEARLY MUFF FOR THIS LOAD OF OF A QUESTION BUT IT'S SUCH AN IMPORTANT ONE AND THE SHORT ANSWER I GIVE IT REALLY DEPENDS ON YOUR MISSION AND YOUR GOAL IS AND FOR AN ORGANIZATION LIKE MINE, OUR GOALS REALLY TO FURTHER RESEARCH AND TO TREAT AND CARE EPILEPSIES SO FOR US, THAT CAN HELP IN A LOT OF DIFFERENT VENUES, RIGHT, THEY'RE NON PROFITS DOING THAT AND THEY'RE INDUSTRY COMPANIES DOING THAT AND CERTAINLY ACADEMIC RESEARCH IS IF THAT COULD HAPPEN IN TERMS OF HOW IT IMPACTS OUR MISSION. >> ERIC? >> I DON'T KNOW THAT I HAVE MORE TO SAY. I THINK THAT WAS A GOOD DISCUSSION OF THAT SO MEASURING OUTCOMES AND I CAN'T SPEAK TO -- PEOPLE THE PEOPLE FROM THE NON-PROFIT AND LIKE JENNIFER AND DIANNE SHOULD SPEAK T TO THAT. NATURALLY THERE ARE STANDARD MEASURES AND OF ULTIMATELY TO DEPENDS ON WHAT THE GOALS ARE AND TREATMENTS AND GETTING RAISING AWARENESS AND INFORMATION AND AS WELL AS PAPERS AND FUNDING AND IF I CAN SPEAK TO GENERALLY I DON'T THINK IT'S A FAILURE IF SOMEONE LEAVES ACADEMIC RESEARCH BECAUSE THERE ARE IMPORTANT THINGS TO GO TO AND MANY IMPORTANT THINGS TO GO TO SO IT DEPENDS -- OR TO SAD ROW KA SEE AND POLICY AND WE ARE SEEING MORE PEOPLE COME BACK AND HAVE A BLEND EXISTENCE AND SO WITH THOSE FIELDS SO I THINK IT SPEAKS TO DO THEY MAINTAIN THE FIRE AND THE INSPIRATION AND THE FIGHT TOWARDS THE GOALS AND ONE OF THE THINGS THAT I WOULD JUST LIKE TO PICK UP IS THE ISSUE OF COVID SINCE I DO THINK THAT IS A SESSION ON TRAINEES AND EARLY CAREER AND I WAS JUST HAD TWO CONVERSATIONS THIS MORNING ONE WITH THE JUNIOR FACULTY TRYING TO GET HIS FIRST GRANT AND HOW THE COLLECTION OF THE PRELIMINARY DATA HAS BEEN INTERRUPTED REPEATEDLY BY COVID AND DIFFERENT WAYS MORE ADVANCED FACULTY MEMBERS WHO SHE'S THINKING ABOUT PROMOTION AND YOU KNOW AND HOW IT'S BEEN HARDER TO MAKE WITH POEM OUTSIDE ARE INSTITUTE AND VIRTUAL MEETINGS ARE GREAT AND ZOOM IS HELPFUL IN MANY WAYS BUT IT'S NOT CONNECTING IN-PERSON WITH PEOPLE AND SO MY FINAL COMMENT IS I LIKED THAT 'EM THINKING HARD ABOUT HOW WE ALL CAN SUPPORT THOSE PEOPLE WHOSE CAREERS ARE MOST VULNERABLE BY THE PANDEMIC. >> IT CAN ADD WHAT ERIC AND JENNIFER SAID SO TO ANSWER THE SECOND QUESTION, SO IT DOESN'T MATTER FOR US, JUST LOOK AT NON PROFITS BECAUSE WE ALLOW A P.I. TO BE FROM ACADEMIA OR INTRAMURAL GOVERNMENT OK SO TAKE A LOOK AT INSTITUTIONS AND LOOK AT THAT. >> RIGHT. NIH TAKES EXACTLY THE SAME VIEWPOINT THAT IN FACT IT IS OUR JOB TO KEEP SCIENCE HAPPENING AND TO KEEP EXCELLENCE AS ROBUST AS IT CAN BE AND WE'VE MADE MANY NEW MECHANISMS AND VARIATIONS ON OLD MECHANISMS TO ADAPT TO THE CIRCUMSTANCES FROM WHICH NONE OF US CAN HIDE AT THIS POINT. >> REFER BACK TO PENNY'S SESSION. IT'S A PUZZLE. A JIG SA PUZZLE. >> MANY. MANY THANKS BOTH TO THE SPEAKERS FOR THIS SESSION AND TO OUR AUDIENCE AND I'M GOING TO TURN THE THERE WERE OVER TO Dr. KOROSHETZ. >> THANK YOU VERY MUCH, NINA AND THANK YOU SO MUCH ERIC AND JENNIFER, DAN AND DIANNE. GREAT SESSION. I THINK JUST A COUPLE OF REFLECTIONS FROM THE DAY. THE ONE THING WE SHOULD ALL TAKEAWAY IS THERE'S A LOT OF REALLY GOOD POEM IN THIS SPACE AND A LOT OF ENERGY IN THIS VIRTUAL ZOOM ROOM AND I WOULD SAY THAT WITH REGARD TO THE TRAINING, DON'T UNDERSTAND ESTIMATE THE POWER OF THE CONTAGION OF THIS ENERGY WHEN PEOPLE ARE LOOKING TO A KA ROAR CAREERAND MAKING A DIFFERENCE IE WORLD AND LOOKING HAT A DISEASE. THERE'S NOT MUCH MORE THAT IS MORE REWARDING THAN THAT AND SO THAT DIMENSION IN THE OPENING REMARKS, THAT COMES FROM THE DISEASE ORGANIZATIONS AND NOT USUALLY FROM THE GOVERNMENT SO YOU HAVE A LOT OF POWER IN MORPHING THE FUTURE BY INFLUENCING PEOPLE. I THINK AFTER THE KIND OF PIE IN THE SKY, I THINK WE GOT SOME HEAVY DOSES OF REALITY THROUGHOUT THE DAY AS PEOPLE TALK ABOUT THE DIFFERENT PATHWAYS AND THE NEED FOR PERSISTENCE AND TO KIND OF CONVERT THAT ENERGY AND PASSION TO ACTUAL GETTING PAST THE GOAL LINE WHICH IS DEVELOPING TREATMENTS FOR PEOPLE. LOTS OF DIFFERENT THINGS ARE NECESSARY AND IT'S ALL ABOUT THE PATIENTS IN THE END SO THE DISEASE ORGANIZATIONS THAT ARE IN TOUCH WITH THE PATIENTS WITH THE DISEASES HAVE INCREDIBLE VALUE AND THE PROCESS OF MOVING TREATMENTS FORWARD, ESPECIALLY WHEN IT COMES TO CLINICAL TRIALS, THAT'S REALLY WHERE THINGS HAPPEN AND I THINK WE'VE HEARD THAT FROM THE HYDROCEPHALUS TALK AND THE JONAS FOUNDATION AND THEIR ABILITY TO MOVE THEIR ENROLLMENT TO WHERE IT NEEDED TO BE AND IT'S TRUE ABOUT SO MANY DIFFERENT SPECIFICALLY THE MORE LESS COMMON DISEASES. WE HEARD A LOT ABOUT THE FOUNDATION FOR NIH WHICH IS SET UP BY CONGRESS TO DEVELOP THESE KIND OF PARTNERSHIPS. THEY KEEP THE GOVERNMENT PEOPLE IN THEIR SWIM LANES SO THEY DON'T END UP IN JAIL AND THINK MANAGEMENT AND DEVELOPING PARTNERSHIPS WITHIN THE LEGAL FRAMEWORK THAT THE GOVERNMENT FOLKS CAN WORK IN AND WE HEARD ABOUT THE BUSINESS OF DEVELOPING TREATMENTS AND THE PROGRAMS MA WE HAVE HERE IN NIH WHICH FUND WHETHER THEY'RE TRYING TO DEVELOP TREATMENT AND PARTICULARLY THE SMALL BUSINESSES ARE OFTEN TIMES THEY'RE SWIM LANE IS THE RARE DISEASES AND THERE'S REAL IMPORTANT LEGAL AND MANAGEMENT FRAMEWORKS THAT YOU HEARD ABOUT TODAY AND EXAMPLES WHERE THEY HAVE HAD SUCCESS IN GAINING FUNDS FROM INDUSTRY OR FROM NIH AS WE HEARD FROM THE CASE. WE TALKED ABOUT THE ACADEMIC PARTNERS WHERE PEOPLE ARE IN TRAINING AND YOU CAN PUT YOUR HOOKS INTO PEOPLE AND GET THEM INTERESTED IN YOUR DISEASE AND WE HAVE INVESTIGATORS WHO HAVE DECIDED TO MAKE THEIR CAREER IN UNDERSTANDING SO DEVELOPING RELATIONSHIPS WITH THOSE PEOPLE IS BENEFICIAL TO BOTH SIDES THEY GET A LOT OF FULFILLMENT OUT OF BEING PART OF THIS BIGGER COMMUNITY AND THEY'RE WORKING IN THEIR LABS DAY AND NIGHT AND A LITTLE BIT OF AN ISOLATION AND WHEN THEY COME TO A MEETING BY DISEASE ORGANIZATION, LISTEN TO THE PATIENTS AND LISTEN TO THEIR CAREGIVERS AND LISTEN TO THE OTHER INVESTIGATORS WHO ARE ALL ENGAGE AND I THINK THAT'S A VERY FULFILLING PART OF THEIR CAREER AND THEY'RE THE PEOPLE WHO ARE GOING TO HAVE THIS DISCOVERIES THAT MAKE THINGS HAPPEN. AND THE FUTURE IS REALLY IN THE NEW PEOPLE WHO ARE COMING INTO THE FIELD OF THE POOLS THAT ARE COMING OUT AS I MENTIONED IN MY TALK AND I THINK THEY'LL BLOW AWAY WHAT WE HAVE NOW AND SO THE ABILITY TO INFLUENCE AND GET YOUNG PEOPLE TRAINED IN TRANSLATION WHETHER IT'S THROUGH A BOOTCAMP OR WHETHER IT'S DISEASE MEETINGS AND I THINK IT'S SO IMPORTANT IN GENERATING THE ARMY THAT IS GROUND ZERO TO SOLVE THE PROBLEMS THAT WE'VE BEEN ABLE TO PUT OUT ON THE TABLE AND SO DEVELOPING THE WELCOME COMMUNITY FOR YOUNG PHYSICIANS FOR YOUNG PHD SCIENTISTS AND FOR ALL THE OTHER PROFESSIONALS AND IS REALLY WHAT IS A BIG INVESTMENT AND OFTEN TIMES IT'S NOT SO EXPENSIVE. A LITTLE MORE EXPENSIVE HAN WHEN I WAS YOUNG BUT IT'S STILL A FAIRLY INEXPENSIVE WAY TO REALLY BUILD YOUR KADRI OF INVESTIGATORS MOVING FORWARD SO I WAS REALLY IMPRESSED. EVERY YAR WE THINK OH BOY, NON-PROFIT FORUM CAN'T BE BETTER BUT IF SEEMS LIKE ONCE AGAIN YOU HAVE EXCELLED AND IT GETS BETTER AND BETTER AND IT'S BETTER THIS YEAR THAN EVER AND SO THANK YOU TO ALL THE PEOPLE WHO PUT THEIR WORK IN. TOMORROW WE'LL HEAR ABOUT SUCCESS STORIES AND DIFFERENT ORGANIZATIONS HAVE HAD AND WE LIKE TO HAVE THESE SHOWERED AND ALSO WE'LL HAVE TALKS AND DISCUSSIONS ABOUT HOW WE MOVE TO BE MORE INCLUSIVE IN THE RESEARCH THAT WE DO AND THE PATIENTS THAT WE SEARCH AND THEN THERE'S A SESSION AS ME SAID TALK AMONGST YOURSELVES TO LEARN FROM EACH OTHER AND THESE BREAK OUTS AND SO THERE'S GOING TO BE IN THE LINK IN THE CHATBOX OF PEOPLE TO TAKE THIS SURVEY THAT WILL INFORM THE BREAK OUT SESSION AND ASSIGNMENTS TOMORROW SO IF YOU HAVEN'T DONE THAT, PLEASE DO THAT NOW AND IT WILL HELP US TOMORROW AND THERE'S OPPORTUNITIES STILL AVAILABLE TO TALK TO THE PROGRAM DIRECTORS AND EVERY SINGLE DISEASE HAS A PROGRAM DIRECTOR IN CHARGE OF THAT DISEASE AND SOME PROGRAM DIRECTORS HAVE ONE DISEASE LIKE THE STROKE FOLKS AND SOME LIKE JOE MORRIS PROBABLY HAVE 30 DIFFERENT DISEASES IN THEIR PORTFOLIO AND MOSTLY RARE DISEASES AND THE PROGRAM DIRECTORS ARE HERE TO HELP YOU ACCESS THE SN NIH RESEARCHERS OS IN YOUR FIELD BECAUSE IT'S REALLY DOVE TAILS WITH OUR MISSION AND ALMOST ALL THE CASES THAT YOU HAVE. THANK YOU AGAIN TO THE GROUP AND LOOK FORWARD TO DISCUSSIONS TOMORROW. HAVE A GOOD EVENING. I WISH WE WERE IN-PERSON.