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Welcome to the Clinical
Center Grand Rounds,

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a weekly series of educational
lectures for physicians and

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health care professionals
broadcast from the Clinical

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Center at the National
Institutes of Health in

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Bethesda, MD.

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The NIH Clinical Center is the
world's largest hospital totally

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dedicated to investigational
research and leads the global

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effort in training today's
investigators and discovering

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tomorrow's cures.

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Learn more by visiting us online
at http://clinicalcenter.nih.gov

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TODAY WE'RE HONORED TO HEAR FROM

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2 COLLEAGUES AT THE NATIONAL

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CENTER FOR ADVANCING

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TRANSLATIONAL SCIENCES.

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DR. DONALD C. LOWE AND

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DR. BROOKS WHO WILL PRESENT ON

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GENE THERAPY APPROACHES FOR

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MULTIPLE RARE DISEASES, THE

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PGGTC INITIATIVES.

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DR. LOWE, THE DIRECTOR OF THE

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THERAPEUTIC THERAPEUTIC BRANCH

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FOR PRECLINICAL INNOVATION AT

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NCATS IS OUR FIRST SPEAKER.

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DR. LOWE RECEIVED Ph.D. IN

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MOLECULAR AND CELLULAR

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PHYSIOLOGY IN YALE UNIVERSITY.

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THEN PURSUED POST DOCTORAL

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STUDIES AT THE LUDWIG INSTITUTE

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FOR CANCER RESEARCH AT

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UNIVERSITY OF COLLEGE LONDON.

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IN 1992 HE JOINED THE FACULTY OF

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THE NEWLY ESTABLISHED DEPARTMENT

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OF NEURAL BIOLOGY AT DUKE

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UNIVERSITY AND IN THE LATE

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1990S, DR. LOWE CONFOUNDED A

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BIOTECH CHUSM STUDYING

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NEUROSCIENCE SERVING AS CHIEF

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SCIENTIFIC OFFICER UNTIL 2002,

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THEN RETURNED TO DUKE UNIVERSITY

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TO ESTABLISH DRUG DISCOVERY ON A

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COLLABORATION MODEL THAT

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PARTNERED WITH OVER A DECADE FOR

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40 ACTIVE INSTITUTIONS AND MORE

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THAN 20 BIOTECH AND MAJOR FORM

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SUITICAL FIRMS.

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DR. LOWE'S WORK HELPED TO

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DEVELOP NEW DRUG AND DRUG TARGET

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CANDIDATES THAT FOR DISEASINGS

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THAT AFFECT THE CENTRAL NERVOUS

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SYSTEM, INCLUDING HUNTING TON'S

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DISEASE, ALZHEIMERS GLAUCOMA,

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ESCHEMIC STROKE AND BRAIN

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CANCER.

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DR. LOWE HOLDS 3 PATENTS AND A

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LEADERSHIP MEMBER OF TRANSLATION

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TOGETHER, A COLLABORATION OF

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TRANSLATIONAL RESEARCH

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ORGANIZATIONS FROM AROUND THE

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WORLD.

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HE IS CONFOUNDER OF HD REACH, A

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NONPROFIT ORGANIZATION DEDICATED

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TO BRINGING QUALITY HEALTHCARE,

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EDUCATION AND SOCIAL ASSISTANCE

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TO ALL PATIENTS AND FAMILIES

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AFFECTED BY HUNTING TON'S

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DISEASE ACROSS THE STATE OF

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NORTH CAROLINA.

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DR. LOWE HAS RECEIVED A NUMBER

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OF AWARDS TO INCLUDE THE

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[INDISCERNIBLE] RESEARCH

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FELLOWSHIP, [INDISCERNIBLE]

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FELLOWSHIP AWARD IN

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NEUROSCIENCE, AND THE BIG KNIGHT

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SCHOLAR AWARD AS WELL AS

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NUMEROUS NCATS DIRECTOR'S AWARDS

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AND 2 NIH DIRECTOR'S AWARDS.

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OUR SECOND PRESENTER IS

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DR. PJ BROOKS, ACTING DIRECTOR

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OF THE OFFICE OF RARE DISEASE

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AND RESEARCH AT NCATS.

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DR. BROOKS RECEIVED A MASTER'S

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DEGREE IN PSYCHOLOGY FROM THE

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UNIVERSITY OF TORONTO AND WENT

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ON TO EARN A Ph.D. IN

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NEUROBIOLOGY FROM UNIVERSITY OF

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NORTH CAROLINA AT CHAPEL HILL.

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FOLLOWING A POST DOCTORAL

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FELLOWSHIP AT ROCKEFELLER

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UNIVERSITY, DR. BROOKS BECAME AN

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INVESTIGATOR IN THE NATIONAL

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INSTITUTE ON CONTROL ABUSE AND

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ALCOHOLISM, WHERE HE DEVELOPED

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AN INTERNATIONALLY RECOGNIZED

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RESEARCH PROGRAM FOCUSED ON RARE

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[INDISCERNIBLE] DISEASES RESULT

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FREE RADICALS GENERATED

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DEFECTIVE DNA REPAIR INCLUDING 0

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DERMA PIG MATOSEIN AND

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[INDISCERNIBLE].

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IN 2010 DR. BROCKS JOINED THE

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OFFICE OF RARE DISEASE AND

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RESEARCH WHERE HE IS NOW

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DEVELOPING NOVEL APPROACHES FOR

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CLINICAL TRIALS AND RARE

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DISEASES BY MOVING BEYOND 1

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DISEASE AT A TIME APPROACHES.

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EXAMPLES INCLUDE THERAPEUTICS AT

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SHARE MOLECULAR MECHANISMS

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UNDERLYING MULTIPLE RARE

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DISEASES AND PLATFORM

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TECHNOLOGIES FOR DELIVERY OF

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NUCLEIC ACID THERAPEUTICS

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INCLUDING PAVE-GT AND GBTC THAT

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WE WILL DISCUSS TODAY.

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HE HAS CO-AUTHORED BOOKS AND A

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LEADER IN RESEARCH AND

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ADMINISTRATION, SERVING AS A

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COORDINATOR FOR NIH COMMON--NIH

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COMMON FUND, SOMATIC CELL GENOME

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EDITING PROGRAM AND AS AN

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INTERDISCIPLINARY SCIENTIFIC

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COMMITTEE CHAIR FOR THE

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INTERNATIONAL RARE DISEASES

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RESEARCH CONSORTIUM.

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PLEASE WELCOME OUR FIRST SPEAKER

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DR. LO.

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>> THANK YOU AGAIN FOR THAT VERY

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GENEROUS INTRO.

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MY COLLEAGUE DR. BROOKS AND I

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ARE DELIGHTED AND HONORED TO BE

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INVITED TO SPEAK TODAY ON THE

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GENE THERAPY APPROACHES THAT

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WE'VE BEEN WORKING ON.

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I THINK THESE BIZARRE ACRONYMS

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PAVE GT AND BGTC WILL MAKE A LOT

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MORE SENSE DURING THE COURSE OF

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THE NEXT HOUR.

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TO TART WITH I HAVE NO FINANCIAL

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DISCLOSURES AND THE LEARNING

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OBJECTIVE FOR DR. BROOKS AND I

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TO LET YOU KNOW ABOUT THE

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STRATEGIES AND INITTIAIVE ITS

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WE'RE TAKING TO IMPROVE

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ACCESSIBILITY TO ALL PAIBTS,

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PARTICULARLY RARE DISEASE

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PATIENTS.

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SO I THOUGHT I WOULD START OFF

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WITH JUST A COUPLE OF WORDS

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ABOUT WHO WE ARE, THAT IS WHAT

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IS THE NATIONAL CENTER OF

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ADVANCING TRANSLATIONAL

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SCIENCES?

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AND WHAT HATTER OF IT IS WORKING

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ON THESE PROJECTS.

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SO NCATS AS I HOPED MOST ALL OF

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YOU KNOW IS THE YOUNGEST KID ON

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THE BLOCK AT NIH, WE'RE THE

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NEWEST OF THE 27 ICs JUST

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HITTING OUR FIRST DECADE.

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AND ON OUR EMBLEM, SEAL HERE,

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CAN YOU SEE THAT WE REALLY WORK

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ACROSS THE SPECTRUM OF

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TRANSLATIONAL SCIENCES WITH THE

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ACCEPTION OF BASIC RESEARCH.

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SO THAT'S IN CONTRAST TO MOST

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ALL OF THE OTHER INSTITUTES AT

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NIH AND IT'S BECAUSE THE AMAZING

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BASIC RESEARCH THAT HAS BEEN

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UNDERTAKEN BY NIH AND THE

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RESEARCH COMMUNITY OVER THE PAST

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DECADES AND HUNDRED YEARS IF NOT

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MORE, HAS GENERATED AMAZING

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KNOWLEDGE ABOUT THE CAUSES OF

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DISEASE.

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--NO, THAT WE KNOW OF TODAY.

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AND WITH IMPROVEMENTS IN GENE

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SEQUENCING AND IN MORE

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GERONTOLOGYSTS NME-CE

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CHARACTERIZATION AND ANALYSIS,

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THIS NUMBER KEEPS ON RISING.

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SO THAT'S THE GOOD NEWS ON THE

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BASIC RESEARCH SIDE, WE HAVE A

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MUCH BETTER UNDERSTANDING OF THE

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CAUSES OF DISEASE OF THOUSANDS

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AND PERHAPS OVER 10,000

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DIFFERENT DISEASES BUT IF YOU

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LOOK AT HOW MANY DISEASES, HAVE

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FDA APPROVED TREATMENTS.

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THAT'S STILL LAGS BEHIND AND

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AROUND 500 OR SO.

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AND EACH YEAR, IF YOU FOLLOW,

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WHAT THE FDA APPROVES, IT'S

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REALLY ONLY A HANDFUL OF NEW

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TREATMENT THIS IS YEAR, EACH

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YEAR, SO AT THIS RATE WE WILL

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NOT CATCH UP ANYTIME SOON.

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THIS PROBLEM IS WORSENED BY THIS

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ANALYSIS THAT WAS DONE A FEW

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YEARS AGO, EROOM'S LAW IS FOR

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ALL YOU CHP FANS OUT THERE IS

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THE INVERSION OR BACKWARDS

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VERSION OF MOORE'S LAW WHERE THE

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COST OF MANUFACTURING MICROCHIPS

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GOES DOWN EXPONENTIALLY EACH

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YEAR.

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EROOM'S LAW SHOWS THAT IN THIS

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GRAPH, THE NUMBER OF DRUGS

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APPROVED BY FDA, PER MILLION

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DOLLARS IN R&D SPENDING IN IT

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COUNTRY, THE X-AXIS IS TIME, CAN

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YOU SEE IN THIS LOG SCALE, THE

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LOG GRAPH THAT IN FACT, THE COST

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OF DRUG DEVELOPMENT ARE GOING UP

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EACH YEAR SO THIS CAN'T BE GOOD.

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SO 1 WAY TO INCAPSULATE NCATS

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MISSION IN TERMS OF TRYING TO

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ADDRESS THIS PROBLEM, OF

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TRANSLATING BASIC RESEARCH

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STUDIES INTO NEW DRUG

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CANDIDATES, 1 WAY YOU CAN THINK

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OF NCATS MISSION IS THAT WE'RE

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HERE TO REALLY TRY TO IDENTIFY

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AND TACKLE THE COMMON

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BOTTLENECKS THAT KEEP NEW DRUG

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DEVELOPMENT FROM HAPPENING

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BETTER AND FASTER ACROSS ALL

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INDICATIONS AND REALLY FOR ALL

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PATIENTS AND ESPECIALLY RARE

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DISEASE PATIENTS AS THIS TALK

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AND THE NEXT TALK WILL TRY TO

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COMMUNICATE.

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SO THE PART OF NCATS THAT I WORK

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IS CALLED THE THERAPEUTIC

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DEVELOPMENT BRANCH AND WE WORK

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WITHIN THIS AREA BOUND BY THE

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RED CIRCLE SO MANY OF HAVE YOU

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SEEN THIS CHEVRON DIAGRAM MANY

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TIMES.

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OT LEFT YOU START WITH BASIC

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SCIENCES, THAT HAVE IDENTIFIED

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PERHAPS A NEW MOLECULAR TARGET

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THAT COULD BE USED TO TREAT A

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DISEASE INDICATION AND FROM

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THERE ON, THERE ARE A NUMBER OF

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STEPS BEFORE YOU CAN GET TO A

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POTENTIAL DRUG CANDIDATE, THAT'S

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THE B-COMPOUND THAT HAS TO BE

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OPTIMIZED TO HAVE THE RIGHT

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PHARMACEUTICAL PROPERTIES AND BE

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DEVELOPED FULL SKPE BAKUGAN ALL

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DAILY BASIS THEA SUBMITTED TO

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THE FDA BEFORE THE DATA WILL

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CLEAR FOR THE SAFETY AND

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POTENTIAL EFFICACY OF THE THIS

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DRUG CANDIDATE IN THE CLINICAL

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TRIALS FOR FIRST EVALUATION OF

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SAFETY AND THEN WHETHER OR NOT

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THE DRUG WORKS.

259
00:09:49,760 --> 00:09:54,880
SO IN THIS CIRCLE, IT'S USUALLY

260
00:09:54,880 --> 00:09:56,960
CALLED PRECLINICAL DEVELOPMENT.

261
00:09:56,960 --> 00:09:59,080
IT'S AIM IS TO DEVELOP NEW DRUG

262
00:09:59,080 --> 00:10:01,120
CANDIDATES FOR TIME DECLEARANCE

263
00:10:01,120 --> 00:10:02,880
ZONE, SO AN INVESTIGATION NEW

264
00:10:02,880 --> 00:10:06,320
DRUG FILING AND CLEARANCE WITH

265
00:10:06,320 --> 00:10:09,120
THE FDA AND THIS SEGMENT OF DRUG

266
00:10:09,120 --> 00:10:09,680
DEVELOPMENT USUALLY TAKES

267
00:10:09,680 --> 00:10:11,240
SEVERAL YEARS AND I PUT DOWN

268
00:10:11,240 --> 00:10:13,040
THERE ON THE SLIDE $4 MILLION

269
00:10:13,040 --> 00:10:15,880
BUT USUALLY A LOT MORE THAN THAT

270
00:10:15,880 --> 00:10:17,080
AND THERE'S A BUNCH OF PROBLEMS

271
00:10:17,080 --> 00:10:18,160
WITH THIS, IT'S VERY HARD

272
00:10:18,160 --> 00:10:20,520
SCIENCE BUT THERE ARE ALSO VERY

273
00:10:20,520 --> 00:10:25,480
FEW FUNDING MECHANISMS, IN THIS

274
00:10:25,480 --> 00:10:26,560
AREA, AND COMMERCIAL INVESTMENT

275
00:10:26,560 --> 00:10:29,480
IS VERY HARD TO FIND BECAUSE

276
00:10:29,480 --> 00:10:30,240
HISTORICALLY, THE FAILURE RATES

277
00:10:30,240 --> 00:10:33,400
DURING THIS PART OF THE DRUG

278
00:10:33,400 --> 00:10:34,480
[INDISCERNIBLE] CHAIN SEQUENCE

279
00:10:34,480 --> 00:10:35,280
IS EXTREMELY HIGH.

280
00:10:35,280 --> 00:10:37,240
SO MANY, MANY PEOPLE CALL THIS

281
00:10:37,240 --> 00:10:40,160
STAGE OF DRUG DEVELOPMENT THE

282
00:10:40,160 --> 00:10:42,480
VALLEY OF DEATH AND ACTUALLY,

283
00:10:42,480 --> 00:10:44,200
THAT'S WHAT WE'RE HERE TO DO.

284
00:10:44,200 --> 00:10:47,920
SO OUR BRANCH LIVES AND WE HOPE

285
00:10:47,920 --> 00:10:49,240
THRIVES WITH THAT.

286
00:10:49,240 --> 00:10:49,960
IT'S ADDITIONALLY CALLED THE

287
00:10:49,960 --> 00:10:51,840
VALLEY OF DEATH BECAUSE IF YOU

288
00:10:51,840 --> 00:10:54,680
LOOK AT THIS FROM A BUSINESS

289
00:10:54,680 --> 00:10:56,240
PERSPECTIVE AND IN THIS GRAPH,

290
00:10:56,240 --> 00:10:58,760
THE Y-AXIS IS THE VALUE OF THE

291
00:10:58,760 --> 00:11:01,480
DRUG CANDIDATE, THE MARKET VALUE

292
00:11:01,480 --> 00:11:03,280
COMPUTED,--MARKET VALUE OF THE

293
00:11:03,280 --> 00:11:06,480
DRUG CANDIDATE AND ON THE X-AXIS

294
00:11:06,480 --> 00:11:07,880
S&P TIME OR MONEY, HOW MUCH

295
00:11:07,880 --> 00:11:10,480
MONEY YOU INVEST INTO THE

296
00:11:10,480 --> 00:11:11,800
DEVELOPMENT OF THIS NEW DRUG

297
00:11:11,800 --> 00:11:12,080
CANDIDATE.

298
00:11:12,080 --> 00:11:13,480
SO ON THE LEFT IS THE STARTING

299
00:11:13,480 --> 00:11:15,080
THE BASIC SCIENCE SIDE, YOU HAVE

300
00:11:15,080 --> 00:11:17,520
THE IDEA, YOU START TO GENERATE

301
00:11:17,520 --> 00:11:18,920
POTENTIAL DRUG PRODUCTS AND DRUG

302
00:11:18,920 --> 00:11:20,680
CANDIDATES AND AS YOU WILL SEE,

303
00:11:20,680 --> 00:11:22,400
THE MORE MONEY YOU INVEST AND

304
00:11:22,400 --> 00:11:26,480
MORE TIME YOU PUT INTO THIS

305
00:11:26,480 --> 00:11:29,080
PROJECT, THE LESS, THE MARKET

306
00:11:29,080 --> 00:11:33,160
VALUE OF THIS DRUG CANDIDATE AND

307
00:11:33,160 --> 00:11:34,240
THAT'S BECAUSE THE MARKET YOU'RE

308
00:11:34,240 --> 00:11:35,800
PUTTING MORE AND MORE CAPITAL AT

309
00:11:35,800 --> 00:11:38,520
RISK AND REALLY AS YOU SEE FROM

310
00:11:38,520 --> 00:11:42,360
THE GRAPH, UNTIL YOU GET FDA

311
00:11:42,360 --> 00:11:44,680
CLEARANCE FOR FIRST IN HUMAN

312
00:11:44,680 --> 00:11:46,400
TESTING, SO IND STATE AND REALLY

313
00:11:46,400 --> 00:11:48,680
THROUGH PHASE 1 SAFETY TRIALS,

314
00:11:48,680 --> 00:11:51,320
THE VALUE OF THE DRUG CANDIDATE

315
00:11:51,320 --> 00:11:53,720
DOESN'T REALLY START TO INCREASE

316
00:11:53,720 --> 00:11:56,120
AND SO THIS VALLEY OF DEATH IS

317
00:11:56,120 --> 00:11:58,160
BOTH A SCIENTIFIC VALLEY OF

318
00:11:58,160 --> 00:11:59,800
DEATH BECAUSE FAILURE RATES ARE

319
00:11:59,800 --> 00:12:02,280
SO HIGH, IT'S ALSO AN ECONOMIC

320
00:12:02,280 --> 00:12:04,800
VALLEY OF DEATH BECAUSE THE

321
00:12:04,800 --> 00:12:07,880
MARKET VALUE OF THESE EARLY

322
00:12:07,880 --> 00:12:09,880
STAGE DRUG CANDIDATES ARE VERY

323
00:12:09,880 --> 00:12:12,600
WELL, VERY TENUOUS.

324
00:12:12,600 --> 00:12:14,800
SO, OUR GROUP, THE THERAPEUTICS

325
00:12:14,800 --> 00:12:15,720
DEVELOPMENT BRANCH HAS PUT

326
00:12:15,720 --> 00:12:17,280
TOGETHER TO TRY TO TACKLE THE

327
00:12:17,280 --> 00:12:18,920
GENERAL PROBLEMS THAT ARE

328
00:12:18,920 --> 00:12:20,720
ENCOUNTERED IN THE VALLEY OF

329
00:12:20,720 --> 00:12:23,040
DEATH, THIS STAGE OF THE VERY

330
00:12:23,040 --> 00:12:25,120
RISKY STAGE OF PRECLINICAL DRUG

331
00:12:25,120 --> 00:12:25,480
DEVELOPMENT.

332
00:12:25,480 --> 00:12:29,280
WE HAVE A NUMBER OF TEAMS, ABOUT

333
00:12:29,280 --> 00:12:30,680
60 SCIENTISTS ALTOGETHER, AND WE

334
00:12:30,680 --> 00:12:33,400
USE A COLLABORATIVE MODEL TO

335
00:12:33,400 --> 00:12:35,120
PARTNER WITH OTHER INVESTIGATORS

336
00:12:35,120 --> 00:12:37,680
AT NIH, WITH ACADEMIC GROUPS,

337
00:12:37,680 --> 00:12:39,280
AND WITH SMALL BIOTECH

338
00:12:39,280 --> 00:12:40,200
COMPANIES, PERHAPS THAT HAVE

339
00:12:40,200 --> 00:12:42,920
JUST BEEN SPUN OUT OF ACADEMIC

340
00:12:42,920 --> 00:12:45,480
GROUPS AND THE IDEA IS TO

341
00:12:45,480 --> 00:12:47,000
PARTNER TOGETHER SO THAT ALL THE

342
00:12:47,000 --> 00:12:48,960
NECESSARY SKILLS CAN BE BROUGHT

343
00:12:48,960 --> 00:12:50,880
TOGETHER TO REALLY AGGRESSIVELY

344
00:12:50,880 --> 00:12:52,960
PUSH FORWARD AND TRY TO IDENTIFY

345
00:12:52,960 --> 00:12:55,200
AND SOLVE BOTTLENECKS THAT ARE

346
00:12:55,200 --> 00:12:56,520
COMMON IN THIS PRECLINICAL

347
00:12:56,520 --> 00:12:56,920
DEVELOPMENT SPACE.

348
00:12:56,920 --> 00:12:58,360
AND YOU CAN SEE WE'RE BROKEN UP

349
00:12:58,360 --> 00:13:00,320
INTO A NUMBER OF TEAMS, THESE

350
00:13:00,320 --> 00:13:01,480
ARE--THESE ARE COMMON, THE

351
00:13:01,480 --> 00:13:03,480
COMMON DIVISIONS, AND THE

352
00:13:03,480 --> 00:13:04,760
BIOTECH COMPANY OR FORM SUITICAL

353
00:13:04,760 --> 00:13:05,840
COMPANY THAT ARE REALLY

354
00:13:05,840 --> 00:13:09,080
NECESSARY TO ADDRESS ALL THE

355
00:13:09,080 --> 00:13:10,880
ISSUES THAT THE FDA WILL BE

356
00:13:10,880 --> 00:13:11,280
CONCERNED ABOUT.

357
00:13:11,280 --> 00:13:13,480
AND THAT STARTS WITH THE BIOLOGY

358
00:13:13,480 --> 00:13:18,920
AND CHEMISTRY OF A DRUG

359
00:13:18,920 --> 00:13:23,880
CANDIDATE THROUGH ITS CONNECTION

360
00:13:23,880 --> 00:13:26,480
WITH PHARMA CODE DMPK, SAFETY,

361
00:13:26,480 --> 00:13:27,280
TOXICOLOGY AND MANUFACTURING AND

362
00:13:27,280 --> 00:13:30,040
A HUGE PART OF THIS IS PROJECT

363
00:13:30,040 --> 00:13:31,320
MANAGEMENT BEING ABLE TO

364
00:13:31,320 --> 00:13:32,240
COORDINATE ALL THESE DIFFERENT

365
00:13:32,240 --> 00:13:58,120
PIECES THAT GO INTO PRECLINICAL

366
00:13:58,120 --> 00:13:58,440
DEVELOPMENT.

367
00:13:58,440 --> 00:14:00,680
IS IT POTENT ENOUGH, IS IT

368
00:14:00,680 --> 00:14:07,160
COLLECTIVE ENOUGH, DOES IT GET

369
00:14:07,160 --> 00:14:09,600
TO THE RIGHT PLACE IN THE RIGHT

370
00:14:09,600 --> 00:14:10,920
TYPES AND TISSUES AND SHOW THAT

371
00:14:10,920 --> 00:14:12,680
THIS EFFICACY IN THE ANIMAL

372
00:14:12,680 --> 00:14:14,600
DISEASE MODEL IF IT EXISTS AND

373
00:14:14,600 --> 00:14:16,400
VERY IMPORTANTLY ESPECIALLY IN

374
00:14:16,400 --> 00:14:19,800
THE INITIAL TRIALS IT'S CAN YOU

375
00:14:19,800 --> 00:14:21,640
SHOW EVIDENCE THAT THAT AT THE

376
00:14:21,640 --> 00:14:23,280
DRUG EXPOSURE LEVEL, YOU NEED

377
00:14:23,280 --> 00:14:30,200
FOR EFFICACY, WILL THAT DRUG BE

378
00:14:30,200 --> 00:14:30,880
SAFE AND NONTOXIC.

379
00:14:30,880 --> 00:14:32,200
THE LAST 2 ARE ESPECIALLY

380
00:14:32,200 --> 00:14:35,640
DIFFICULT AREAS TO FIND IN AN

381
00:14:35,640 --> 00:14:36,600
ACADEMIC STUDY OR BASIC RESEARCH

382
00:14:36,600 --> 00:14:39,000
SETTING AND THAT IS CAN YOU

383
00:14:39,000 --> 00:14:39,680
ACTUALLY MANUFACTURE THIS DRUG

384
00:14:39,680 --> 00:14:44,240
AND PURE ENOUGH FORM AND LARGE

385
00:14:44,240 --> 00:14:46,080
ENOUGH QUANTITIES FOR TESTING IN

386
00:14:46,080 --> 00:14:47,720
HUMANS BECAUSE IN THE LABORATORY

387
00:14:47,720 --> 00:14:50,160
OF COURSE YOU MAY ONLY NEED A

388
00:14:50,160 --> 00:14:52,200
FEW MILLIGRAMS BUT PEOPLE MAYBE

389
00:14:52,200 --> 00:14:54,000
ONLY NEED GRAMS OR KILOGRAMS

390
00:14:54,000 --> 00:14:55,600
MAYBE FOR SOME OF THESE STUDIES

391
00:14:55,600 --> 00:14:56,680
SO MANUFACTURING IS A HUGE

392
00:14:56,680 --> 00:14:58,280
QUESTION AND CAN THE DRUG BE

393
00:14:58,280 --> 00:15:01,680
FORMULATED INTO THE ROOTS OF FOR

394
00:15:01,680 --> 00:15:04,560
THE ROOT OF ADMINISTRATION THAT

395
00:15:04,560 --> 00:15:05,080
YOU NEED.

396
00:15:05,080 --> 00:15:06,680
SO MAYBE A PILL, INJECTABLE,

397
00:15:06,680 --> 00:15:07,880
MAYBE A SPRAY, ALL THESE

398
00:15:07,880 --> 00:15:10,080
PROBLEMS HAVE TO BE WORKED OUT

399
00:15:10,080 --> 00:15:11,480
AND DATA PACKAGED VERY NICELY

400
00:15:11,480 --> 00:15:14,800
FOR SUBMISSION TO THE FDA, AND

401
00:15:14,800 --> 00:15:17,960
INDs AND UNTIL FDA CLEARS THE

402
00:15:17,960 --> 00:15:19,480
IND, OF COURSE 1 CANNOT START

403
00:15:19,480 --> 00:15:21,720
TESTING THIS DRUG CANDIDATE IN

404
00:15:21,720 --> 00:15:21,960
PEOPLE.

405
00:15:21,960 --> 00:15:24,480
SO THAT'S WHAT WE DO.

406
00:15:24,480 --> 00:15:26,280
SO OUR GROUP OF 60 PEOPLE IN A

407
00:15:26,280 --> 00:15:28,800
WAY, YOU CAN THINK OF US AS A

408
00:15:28,800 --> 00:15:31,320
SWISS ARMY KNIFE OF BIOTECH

409
00:15:31,320 --> 00:15:32,480
COMPANIES, SO WE PARTNER ALMOST

410
00:15:32,480 --> 00:15:35,480
ALL OF OUR PROJECTS WITH OUTSIDE

411
00:15:35,480 --> 00:15:37,960
PARTIES AND WE ALLEVIATE THE

412
00:15:37,960 --> 00:15:41,440
OUTSIDE PARTY FROM THE NECESSITY

413
00:15:41,440 --> 00:15:43,160
TO START THEIR OWN BIOTECH

414
00:15:43,160 --> 00:15:43,400
COMPANY.

415
00:15:43,400 --> 00:15:44,960
SO WE WORK TOGETHER WITH

416
00:15:44,960 --> 00:15:46,160
PARTNERS FOR ALMOST ALL OUR

417
00:15:46,160 --> 00:15:48,680
PROJECTS, I THINK WE DO A PRETTY

418
00:15:48,680 --> 00:15:51,440
GOOD JOB, WE CLEARED, HOPED TO

419
00:15:51,440 --> 00:15:54,680
CLEAR OVER 40 NEW DRUG

420
00:15:54,680 --> 00:15:55,800
CANDIDATES, INTO THE PHASE 2

421
00:15:55,800 --> 00:15:58,320
CLINICAL TRIALS OFFER THE LAST

422
00:15:58,320 --> 00:16:00,680
DECADE, AND AS YOU'LL SEE FROM

423
00:16:00,680 --> 00:16:02,440
THE--AS YOU WILL SEE FROM THE

424
00:16:02,440 --> 00:16:03,920
TABLE, UP TOP, MANY OF THESE

425
00:16:03,920 --> 00:16:05,360
DRUGS ARE PROGRESSING THROUGH

426
00:16:05,360 --> 00:16:07,800
THE SEQUENCE OF CLINICAL TRIALS,

427
00:16:07,800 --> 00:16:09,680
PHASE 1, PHASE 2, PHASE 3, AND

428
00:16:09,680 --> 00:16:12,600
WE'RE HOPING THIS YEAR AND NEXT,

429
00:16:12,600 --> 00:16:17,280
PERHAP THIS IS FIRST DRUG WILL

430
00:16:17,280 --> 00:16:17,880
BE RELEASED.

431
00:16:17,880 --> 00:16:21,360
SO IT'S IN THIS CONTEXT THAT WE

432
00:16:21,360 --> 00:16:25,520
CAME TO WORK WITH TJ AND OTHER

433
00:16:25,520 --> 00:16:26,760
COLLEAGUES ON THIS AGTG PROJECT

434
00:16:26,760 --> 00:16:28,440
WHICH I WILL TELL YOU A BIT MORE

435
00:16:28,440 --> 00:16:29,520
ABOUT, SO YOU SEE FROM THE

436
00:16:29,520 --> 00:16:32,640
SAMPLE WE WORK IN A LOT OF

437
00:16:32,640 --> 00:16:33,280
DIFFERENT AREAS, RARE DISEASES

438
00:16:33,280 --> 00:16:36,280
AND WORK AS PART OF THE HEAL

439
00:16:36,280 --> 00:16:37,680
INITIATIVE, WHEN COVID HIT US,

440
00:16:37,680 --> 00:16:40,080
WE TRIED TO MOBILIZE AND DO OUR

441
00:16:40,080 --> 00:16:42,640
BEST TO HELP ADDRESS

442
00:16:42,640 --> 00:16:43,440
TRANSLATIONAL BOTTLENECKS AND

443
00:16:43,440 --> 00:16:45,840
NEW COVID DRUG DEVELOPMENT, I

444
00:16:45,840 --> 00:16:47,120
WON'T HAVE TIME TO TELL YOU

445
00:16:47,120 --> 00:16:48,680
ABOUT ANOTHER PROGRAM WE HAVE

446
00:16:48,680 --> 00:16:50,200
ONGOING, WHICH LOOKSA THE A

447
00:16:50,200 --> 00:16:52,800
DIFFERENT KIND'VE GENE THERAPY,

448
00:16:52,800 --> 00:16:55,880
THAT ADDRESS YOU THINK CAN BE

449
00:16:55,880 --> 00:16:58,960
SENT TO THE OLIGO NUCLEOTIDE

450
00:16:58,960 --> 00:17:00,080
DRUGS WHEN THERE ARE PARTICULAR

451
00:17:00,080 --> 00:17:01,080
PROBLEMS WHEN THE DISEASES ARE

452
00:17:01,080 --> 00:17:02,560
EXTREMELY RARE, DOWN TO PERHAPS

453
00:17:02,560 --> 00:17:15,520
A DISEASE NOT ONLY A SINGLE

454
00:17:15,520 --> 00:17:15,880
PATIENT HAS.

455
00:17:15,880 --> 00:17:18,840
SO THEN WE STARTED THIS

456
00:17:18,840 --> 00:17:19,760
INITIATIVE, THE LINK IS

457
00:17:19,760 --> 00:17:21,400
UNDERNEATH AND THE CORE OF THIS

458
00:17:21,400 --> 00:17:25,920
PROJECT IS LOOKING AT THIS TYPE

459
00:17:25,920 --> 00:17:27,240
OF GENE THERAPY WHERE YOU

460
00:17:27,240 --> 00:17:28,720
ESSENTIALLY AS YOU ALL KNOW I'M

461
00:17:28,720 --> 00:17:30,880
SURE REPLACE PART OF A VIRAL

462
00:17:30,880 --> 00:17:33,120
GENOME, LIKE A THERAPEUTIC

463
00:17:33,120 --> 00:17:35,800
CONSTRUCT THAT YOU THINK WILL

464
00:17:35,800 --> 00:17:37,640
PROVIDE PATIENT BENEFIT AND YOU

465
00:17:37,640 --> 00:17:39,240
PUT THAT, SUBSTITUTE THAT INTO

466
00:17:39,240 --> 00:17:40,320
THE VIRAL GENOME, WITHIN THE

467
00:17:40,320 --> 00:17:43,800
VIRUS CAN BE ESSENTIALLY USEAISE

468
00:17:43,800 --> 00:17:45,480
VECTOR OR AS AN AGENT TO DELIVER

469
00:17:45,480 --> 00:17:52,480
THAT CHAIN TO THE DESIRED CELL

470
00:17:52,480 --> 00:17:52,920
TYPES AND TISSUES.

471
00:17:52,920 --> 00:17:56,280
THIS IS NOW AND INVENTED, THE

472
00:17:56,280 --> 00:17:58,080
IDEA INVENTED SOME DECADES AGO,

473
00:17:58,080 --> 00:18:00,000
THIS OLD JOURNAL COVER MAGAZINE

474
00:18:00,000 --> 00:18:03,080
COVER FROM TIME IS OVER 20 YEARS

475
00:18:03,080 --> 00:18:04,520
OLD, REALLY PROMISING GENE

476
00:18:04,520 --> 00:18:14,720
THERAPY, AS FUTURE OF MEDICINE.

477
00:18:14,720 --> 00:18:17,440
SO THE AV VECTORS HAVE BECOME

478
00:18:17,440 --> 00:18:18,920
THE MAIN STAY.

479
00:18:18,920 --> 00:18:21,040
AND IN FACT THIS FIELD IS

480
00:18:21,040 --> 00:18:24,320
MATURING VERY NICELY SO THE

481
00:18:24,320 --> 00:18:30,120
FIRST FEW FDA APPROVALS OF GENE

482
00:18:30,120 --> 00:18:33,160
THERAPY DRUGS BASED ON AAV, CAN

483
00:18:33,160 --> 00:18:36,680
YOU SEE A COUPLE FIRST 2

484
00:18:36,680 --> 00:18:38,240
APPROVALSOT RIGHT AND SO WE'RE

485
00:18:38,240 --> 00:18:43,800
NOT LOOKING SO MUCH THE SCIENCE

486
00:18:43,800 --> 00:18:45,680
OF GENE THERAPY OR EVEN THE

487
00:18:45,680 --> 00:18:47,800
FUNDAMENTAL DRUG PROCESSES FOR

488
00:18:47,800 --> 00:18:50,880
GENE THERAPY, THOSE WELL ENOUGH

489
00:18:50,880 --> 00:18:52,960
TO HAVE APPROVED GENE THERAPY

490
00:18:52,960 --> 00:18:58,920
PRODUCTS AND MANY MORE IN LINE

491
00:18:58,920 --> 00:19:00,800
FOR WHAT WE'RE LOOKING FOR IS

492
00:19:00,800 --> 00:19:02,680
THE ISSUE OF ACCESSIBILITY.

493
00:19:02,680 --> 00:19:04,320
WHAT WILL IT TAKE FOR GENE

494
00:19:04,320 --> 00:19:07,480
THERAPY TO BE AVAILABLE FOR MANY

495
00:19:07,480 --> 00:19:09,280
MORE DISEASE, NOT A FEW AT A

496
00:19:09,280 --> 00:19:10,480
TIME.

497
00:19:10,480 --> 00:19:13,080
BUT TO REALLY MAKE A DENT AND

498
00:19:13,080 --> 00:19:14,640
THOUSANDS OF DISEASES THAT COULD

499
00:19:14,640 --> 00:19:15,760
BENEFIT FROM GENE THERAPY.

500
00:19:15,760 --> 00:19:16,640
SO THE PROBLEM STATEMENT REALLY

501
00:19:16,640 --> 00:19:20,120
IS WHAT CAN WE DO TO LOWER THE

502
00:19:20,120 --> 00:19:20,680
BARRIERS TO ACCESSIBILITY,

503
00:19:20,680 --> 00:19:24,520
ESPECIALLY TO RARE DISEASE

504
00:19:24,520 --> 00:19:27,400
PATIENTS AND THE MARKET COSTS

505
00:19:27,400 --> 00:19:29,280
FOR THESE GENE THERAPIES SUCH AS

506
00:19:29,280 --> 00:19:32,520
READ ON THE RIGHT GIVE YOU SOME

507
00:19:32,520 --> 00:19:34,520
CLUE AS THE PAIR BERNEA DEETS

508
00:19:34,520 --> 00:19:38,840
THAT YOU MIGHT ENCOUNTER AND THE

509
00:19:38,840 --> 00:19:40,480
FIRST 1 REALLY THE FIRST 1 IS AN

510
00:19:40,480 --> 00:19:43,600
ECONOMIC 1 SO THIS IS AN

511
00:19:43,600 --> 00:19:45,160
HISTOGRAPH SHOWING THE

512
00:19:45,160 --> 00:19:47,200
DISTRIBUTION OF THE PREVALENCE

513
00:19:47,200 --> 00:19:49,280
OF RARE DISEASES.

514
00:19:49,280 --> 00:19:50,600
SO THEY'RE--THERE ARE THOUSANDS

515
00:19:50,600 --> 00:19:53,440
AND THOUSANDS OF RARE DISEASES

516
00:19:53,440 --> 00:19:54,680
COLLECTIVELY AND IT CONSTITUTES

517
00:19:54,680 --> 00:19:56,480
SOMETHING LIKE 20 OR 30% OF ALL

518
00:19:56,480 --> 00:19:58,520
DISEASE BURDEN WHICH YOU WILL

519
00:19:58,520 --> 00:20:02,800
SEE IN THE HISTOGRAM WHERE THE

520
00:20:02,800 --> 00:20:04,440
X-AXIS, THIS HISTOGRAM IS AN

521
00:20:04,440 --> 00:20:05,680
ESTIMATED PREVALENCE OF RARE

522
00:20:05,680 --> 00:20:07,360
DISEASES AND THE Y-AXIS IS THE

523
00:20:07,360 --> 00:20:09,760
NUMBER OF RARE DECEASES OF THAT

524
00:20:09,760 --> 00:20:10,240
PREVALENCE.

525
00:20:10,240 --> 00:20:12,880
YOU CAN SEE THAT THE VAST

526
00:20:12,880 --> 00:20:14,080
MAJORITY OF THE RARE DISEASES

527
00:20:14,080 --> 00:20:15,400
HAVE VERY LOW PREVALENCE AND

528
00:20:15,400 --> 00:20:18,440
WHAT I'VE SHOWN IN RED, IS THAT

529
00:20:18,440 --> 00:20:20,280
FROM A COMMERCIAL PERSPECTIVE OF

530
00:20:20,280 --> 00:20:22,800
A PHARMACEUTICAL COMPANY OR

531
00:20:22,800 --> 00:20:23,600
BIOTECH COMPANY GENERALLY

532
00:20:23,600 --> 00:20:28,840
SPEAKING WOULD NOTED CONSIDER

533
00:20:28,840 --> 00:20:30,360
ANY ANY RARE DISEASE WITH

534
00:20:30,360 --> 00:20:31,720
PREVALENCE LOWER THAN A FEW

535
00:20:31,720 --> 00:20:32,960
THOUSAND PATIENTS TO BE

536
00:20:32,960 --> 00:20:33,840
COMMERCIALLY VIABLE.

537
00:20:33,840 --> 00:20:39,120
SO A MAIN STAY OF WHAT DRIVES

538
00:20:39,120 --> 00:20:40,160
THE DEVELOPMENT AND MARKETING

539
00:20:40,160 --> 00:20:41,400
DISTRIBUTION OF DRUGS IN OUR

540
00:20:41,400 --> 00:20:48,080
COUNTRY THAT IS THE

541
00:20:48,080 --> 00:20:48,560
PHARMACEUTICAL INDUSTRY,

542
00:20:48,560 --> 00:20:49,280
BIOPHARMACEUTICAL SECTOR IS

543
00:20:49,280 --> 00:20:51,280
REALLY UNABLE TO ADDRESS THE

544
00:20:51,280 --> 00:20:52,160
VAST MAJORITY OF DISEASES

545
00:20:52,160 --> 00:20:53,480
BECAUSE OF THIS COMMERCIAL

546
00:20:53,480 --> 00:21:01,320
LIMITATION, AND SO THAT'S AND

547
00:21:01,320 --> 00:21:03,640
THAT'S BECAUSE THE PREVALENCE

548
00:21:03,640 --> 00:21:04,760
CONSITUTE A VIABLE BUSINESS

549
00:21:04,760 --> 00:21:06,080
PROMULGATE CISION, THERE ISN'T A

550
00:21:06,080 --> 00:21:09,480
TYPICAL KIND OF COLLABORATION

551
00:21:09,480 --> 00:21:14,480
WITH INDUSTRY THAT WE CAN COUNT

552
00:21:14,480 --> 00:21:14,640
ON.

553
00:21:14,640 --> 00:21:16,280
SO THIS IN TURN DRIVES A SECOND

554
00:21:16,280 --> 00:21:18,680
CLASS OF PROBLEMS SO IF THESE

555
00:21:18,680 --> 00:21:20,320
DISEASES ARE NOT THAT COMMERCIAL

556
00:21:20,320 --> 00:21:24,280
VIABLE AND YOU CAN SEE THAT IS

557
00:21:24,280 --> 00:21:25,440
REFLECTED IN THE COST OF

558
00:21:25,440 --> 00:21:27,120
TREATMENT FOR THESE DISEASES,

559
00:21:27,120 --> 00:21:28,640
THERE ARE A BUNCH OF FOLLOW ON

560
00:21:28,640 --> 00:21:30,000
EFFECTS AND 1 OF THESE, SO SOME

561
00:21:30,000 --> 00:21:32,880
OF THESE ARE LISTED IN THE

562
00:21:32,880 --> 00:21:36,560
CENTER BOX, SO 1 THING THAT

563
00:21:36,560 --> 00:21:37,400
HAPPENS IS THAT INTELLECTUAL

564
00:21:37,400 --> 00:21:40,960
PROPERTY, THERE IT IS ARE VERY

565
00:21:40,960 --> 00:21:44,480
STRONGLY ENFORCED SO THAT IS

566
00:21:44,480 --> 00:21:46,000
PROPRIETARY KNOW HOW AND THESE

567
00:21:46,000 --> 00:21:47,200
ARE NOT GENERALLY SHARED BECAUSE

568
00:21:47,200 --> 00:21:54,720
OF COMMERCIAL MARKET IS SO TIGHT

569
00:21:54,720 --> 00:21:57,480
THAT MEANS IF YOU'RE AN

570
00:21:57,480 --> 00:21:59,080
RESEARCHER, NIH RESEARCHER,

571
00:21:59,080 --> 00:21:59,720
PATIENT, FOUNDATION WORK NOTHING

572
00:21:59,720 --> 00:22:01,360
A RARE DISEASE, YOU WILL HAVE

573
00:22:01,360 --> 00:22:02,520
VERY LITTLE ACCESS, PROBLEMATIC

574
00:22:02,520 --> 00:22:04,280
ACCESS TO THE KINDS OF EXPERTISE

575
00:22:04,280 --> 00:22:08,400
AND RESOURCES YOU NEED TO

576
00:22:08,400 --> 00:22:14,280
DEVELOP THE GENE THERAPY. 

577
00:22:14,280 --> 00:22:15,240
CONSEQUENTLY BECAUSE OF THESE OF

578
00:22:15,240 --> 00:22:18,880
WHAT IT COSTS TO MANUFACTURE

579
00:22:18,880 --> 00:22:21,000
THIS, GENES CAN BE EXTREMELY

580
00:22:21,000 --> 00:22:22,240
HIGH TO GENERATE ENOUGH AV GENE

581
00:22:22,240 --> 00:22:30,680
VECTOR EVEN TO CONDUCT PHASE 1

582
00:22:30,680 --> 00:22:32,200
CLINICAL TRIAL CURRENT MARKET

583
00:22:32,200 --> 00:22:33,400
COSTS ARE 5 MILLION OR MORE.

584
00:22:33,400 --> 00:22:36,080
AND EVEN IF YOU HAVE 5 MILLION

585
00:22:36,080 --> 00:22:37,960
OR MORE, IT'S TIGHTLY HELD AND

586
00:22:37,960 --> 00:22:39,920
IT'S DIFFICULT TO GET A

587
00:22:39,920 --> 00:22:44,440
MANUFACTURING SLOT THAT

588
00:22:44,440 --> 00:22:45,080
IS--MANUFACTURING FACILITIES ARE

589
00:22:45,080 --> 00:22:46,480
SO TIED UP IN COMMERCIAL

590
00:22:46,480 --> 00:22:48,360
PRODUCT, VERY HARD TO GET A

591
00:22:48,360 --> 00:22:51,280
NONCOMMERCIAL PRODUCT MADE EVEN

592
00:22:51,280 --> 00:22:52,640
IF YOU HAVE TO FUND IT.

593
00:22:52,640 --> 00:22:53,520
AND THEN THE THIRD PROBLEM IS

594
00:22:53,520 --> 00:23:04,520
BECAUSE OF THESE ISSUES IN

595
00:23:04,520 --> 00:23:05,480
ACCESSIBILITY IN THE GENERAL

596
00:23:05,480 --> 00:23:06,680
COMMUNITY THERE'S A LACK OF

597
00:23:06,680 --> 00:23:08,080
THESE IN MAKING GENE VECTORS AND

598
00:23:08,080 --> 00:23:09,960
CERTAINLY A LACK OF EXPERIENCE

599
00:23:09,960 --> 00:23:12,680
INTO THE PRECLINICAL DEMANDS IN

600
00:23:12,680 --> 00:23:15,440
TERMS OF GOING FROM THE AAV GENE

601
00:23:15,440 --> 00:23:16,880
CONSTRUCT THAT WORKS IN THE LAB

602
00:23:16,880 --> 00:23:17,920
THROUGH THE DEVELOPMENT PROCESS

603
00:23:17,920 --> 00:23:22,520
TO A POINT WHERE MAYBE GENE

604
00:23:22,520 --> 00:23:23,440
CANDIDATE, GENE THERAPY

605
00:23:23,440 --> 00:23:25,000
CANDIDATE CAN BE EVALUATED BY

606
00:23:25,000 --> 00:23:26,040
THE CLEARANCE AND THE TESTING SO

607
00:23:26,040 --> 00:23:29,080
IT'S A FOLLOW ON, SET OF

608
00:23:29,080 --> 00:23:33,400
PROBLEMS AND SO, WHAT WE'RE

609
00:23:33,400 --> 00:23:36,160
SEEKING TO ADDRESS IN THE AGT

610
00:23:36,160 --> 00:23:38,840
PROGRAM IS TO MAKE THAT AN OPEN

611
00:23:38,840 --> 00:23:41,560
BOOK, PROCESS INSTEAD SO INSTEAD

612
00:23:41,560 --> 00:23:51,240
OF HAVING THE ENTIRE METHODOLOGY

613
00:23:51,240 --> 00:23:53,480
AND ROADMAP THROUGH AAV GENE

614
00:23:53,480 --> 00:23:56,360
THERAPY IS A HELD TIGHT CLOSE

615
00:23:56,360 --> 00:23:58,080
SECRET, PUT IT TOGETHER AND MAKE

616
00:23:58,080 --> 00:23:59,320
A PUBLIC VERSION, OPEN BOOK,

617
00:23:59,320 --> 00:24:01,520
PUBLIC VERSION OF HOW TO

618
00:24:01,520 --> 00:24:03,080
CONSTRUCT AN AAV GENE THERAPY

619
00:24:03,080 --> 00:24:04,720
AND HOW TO THAT I CAN THAT GENE

620
00:24:04,720 --> 00:24:11,600
THERAPY CANDIDATE ALL THE WAY

621
00:24:11,600 --> 00:24:12,680
THROUGH PRECLINICAL DEVELOPMENT

622
00:24:12,680 --> 00:24:13,840
AND SUBMITTING THAT DATA PACKAGE

623
00:24:13,840 --> 00:24:17,880
TO FDA FOR THAT IND PACKAGE FOR

624
00:24:17,880 --> 00:24:31,280
CLEARANCE INTO PHASE 1 TRIALS.

625
00:24:31,280 --> 00:24:32,800
MORE OVER WE WILL TRY TO SHOW

626
00:24:32,800 --> 00:24:33,880
SOME ECONOMY OF SCALE.

627
00:24:33,880 --> 00:24:35,160
THE WHOLE PROMISE OF GENE

628
00:24:35,160 --> 00:24:37,000
THERAPY IS THAT IT'S A PLATFORM

629
00:24:37,000 --> 00:24:40,680
THAT IS THE VERY SAME OUTSIDE

630
00:24:40,680 --> 00:24:42,400
VECTOR, THE CAPSID, THE AAV

631
00:24:42,400 --> 00:24:44,680
VECTOR CAN BE THE SAME BUT CAN

632
00:24:44,680 --> 00:24:47,520
BE USED TO TREAT DIFFERENT

633
00:24:47,520 --> 00:24:48,960
DISEASES BY SWAPPING IN

634
00:24:48,960 --> 00:24:51,080
DIFFERENT GENE THERAPY PAY

635
00:24:51,080 --> 00:24:52,280
LOADS, DIFFERENT GENE CONSTRUCTS

636
00:24:52,280 --> 00:24:55,080
INTO THE PAY LEAD SIDE UP IN AAV

637
00:24:55,080 --> 00:25:00,840
GENE VECTOR SO WE CAN SHOW THAT

638
00:25:00,840 --> 00:25:02,040
BY DEVELOPING 4 THERAPIES AT

639
00:25:02,040 --> 00:25:02,480
ONCE.

640
00:25:02,480 --> 00:25:08,040
WE WILL USE THE SAME OUTSIDE

641
00:25:08,040 --> 00:25:10,000
GENE THERAPY VECTORS SO AAV 9,

642
00:25:10,000 --> 00:25:13,320
WHICH IS A CAP SID TYPE OF AAV

643
00:25:13,320 --> 00:25:16,040
THAT HAS VERY BROAD TROAPIC--SO

644
00:25:16,040 --> 00:25:24,480
THAT IS AAV 9 OR CAN BE USED TO

645
00:25:24,480 --> 00:25:27,880
TRANSDUCE SO IN THE CNS, IN THE

646
00:25:27,880 --> 00:25:29,320
PERIPHERY, WE WILL MAKE THESE

647
00:25:29,320 --> 00:25:31,320
VECTORS SO THAT CAN BE

648
00:25:31,320 --> 00:25:33,160
ADMINISTERED THROUGH THE SAME

649
00:25:33,160 --> 00:25:35,040
ROUTE OF AND ADMINISTRATION, AND

650
00:25:35,040 --> 00:25:38,440
IMPORTANTLY FOR THE FDA, AND FOR

651
00:25:38,440 --> 00:25:40,680
THE PRECLINICAL REGULATORY

652
00:25:40,680 --> 00:25:43,240
PROCESS, USE THE IDENTICAL

653
00:25:43,240 --> 00:25:44,400
PRODUCTION MANUFACTURING AND

654
00:25:44,400 --> 00:25:45,520
PURIFICATION METHODS SO THE ONLY

655
00:25:45,520 --> 00:25:46,960
THING THAT WILL BE DIFFERENT AS

656
00:25:46,960 --> 00:25:48,800
I WAS MENTIONING IN THE CONTEXT

657
00:25:48,800 --> 00:25:54,400
OF A PLATFORM IS THAT THESE 4

658
00:25:54,400 --> 00:25:56,920
DRUGS WILL HAVE DIFFERENT GENE

659
00:25:56,920 --> 00:25:57,880
CONTRUCTS INSIDE OTHERWISE THEY

660
00:25:57,880 --> 00:25:59,400
WILL BE IDENTICAL AND THIS

661
00:25:59,400 --> 00:26:01,960
SHOULD SHOW THE ECONOMY OF SCALE

662
00:26:01,960 --> 00:26:02,880
USING IDENTICAL PRECLINICAL

663
00:26:02,880 --> 00:26:04,320
PLGHTS BUT USING THAT TO CARRY

664
00:26:04,320 --> 00:26:06,800
DIFFERENT PAY LOADS TO ADDRESS

665
00:26:06,800 --> 00:26:08,960
DIFFERENT DISEASES.

666
00:26:08,960 --> 00:26:11,640
SO, THE GOAL, NUMBER 1, IS TO

667
00:26:11,640 --> 00:26:14,640
MAKE THIS REALLY AN OPEN BOOK, A

668
00:26:14,640 --> 00:26:17,480
PUBLIC METHODOLOGY THAT CAN BE

669
00:26:17,480 --> 00:26:19,280
WIDELY DISSEMINATED AND ANYBODY

670
00:26:19,280 --> 00:26:21,240
COULD FOLLOW ALONG WITH THEIR

671
00:26:21,240 --> 00:26:24,120
OWN GENE THERAPY STRATEGIES

672
00:26:24,120 --> 00:26:24,800
WITHOUT ANY STRINGS ATTACHED SO

673
00:26:24,800 --> 00:26:27,000
IT'S PART OF THAT GOAL TO USE

674
00:26:27,000 --> 00:26:28,160
REAGENTS AND PROCESSES INCLUDING

675
00:26:28,160 --> 00:26:31,720
MANUFACTURING PROCESSES THAT ARE

676
00:26:31,720 --> 00:26:35,160
FREE INTELLECTUAL PROPERTY

677
00:26:35,160 --> 00:26:36,280
CONSTRAINTS, SO THEREFORE ON TOP

678
00:26:36,280 --> 00:26:37,680
OF SCIENCE, THERE PONENT BE ANY

679
00:26:37,680 --> 00:26:40,080
ISSUES ABOUT HAVING TO PAY VERY

680
00:26:40,080 --> 00:26:41,360
COSTLY LICENSING FEES WHICH

681
00:26:41,360 --> 00:26:44,520
CURRENTLY IS QUITE COMMON.

682
00:26:44,520 --> 00:26:45,760
AND SO IMPORTANTLY, AS I SAY, WE

683
00:26:45,760 --> 00:26:49,000
WILL MAKE ALL THE METHODS AND

684
00:26:49,000 --> 00:26:50,400
PROTOCOLS, THROUGH THE AAV

685
00:26:50,400 --> 00:26:52,360
PACKAGES AND SUBMIT TO THE FDA

686
00:26:52,360 --> 00:26:53,680
PUBLIC AND WE WILL BE POSTING 

687
00:26:53,680 --> 00:26:57,240
ON OUR WEBSITE IN REALTIME AS

688
00:26:57,240 --> 00:26:59,120
WELL AS FDA FEEDBACK.

689
00:26:59,120 --> 00:27:02,480
THIS IS A REALLY IMPORTANT

690
00:27:02,480 --> 00:27:03,120
COMPONENT OF NEW DRUG

691
00:27:03,120 --> 00:27:04,880
DEVELOPMENT THAT IS GETTING FDA

692
00:27:04,880 --> 00:27:08,640
FEEDBACK ALONG THE WAY, AS YOU

693
00:27:08,640 --> 00:27:10,320
PRIMATES PAIR TO FILE AN IND

694
00:27:10,320 --> 00:27:13,600
PACKAGE WITH THE FDA AND THAT

695
00:27:13,600 --> 00:27:16,120
FEEDBACK AND VERY UNCOMMON TO

696
00:27:16,120 --> 00:27:18,240
HAVE THAT SHARE, AND WE ARE

697
00:27:18,240 --> 00:27:20,240
DELIGHT THAD FDA AGREED TO ALLOW

698
00:27:20,240 --> 00:27:22,960
US TO SHARE THEIR FEEDBACK TO

699
00:27:22,960 --> 00:27:24,280
OUR REGULATORY FILINGS ON OUR

700
00:27:24,280 --> 00:27:28,360
WEBSITE AS WELL.

701
00:27:28,360 --> 00:27:30,040
THAT'S THE GENERAL IDEA OF THE

702
00:27:30,040 --> 00:27:34,840
PAVE GT PROGRAM AND I COULDN'T

703
00:27:34,840 --> 00:27:36,400
RESIST THIS SLIDE, THIS JOKING

704
00:27:36,400 --> 00:27:38,160
SHOWS MY AGE, NO 1 YOUNGER THAN

705
00:27:38,160 --> 00:27:40,240
I WOULD KNOW WHAT THIS IS BUT IF

706
00:27:40,240 --> 00:27:41,240
THERE'S ANYONE AS OLD AS I AM

707
00:27:41,240 --> 00:27:43,800
THAT USED TO WORK ON CARS YOU

708
00:27:43,800 --> 00:27:45,320
WOULD RECOGNIZE THE CHILTON

709
00:27:45,320 --> 00:27:46,720
MANUEL OR MANUELS OF THIS SORT.

710
00:27:46,720 --> 00:27:50,520
SO IN THE OLD DAYS BEFORE CARS

711
00:27:50,520 --> 00:27:51,480
AND TRUCKS HAD MICROCHIPS AND

712
00:27:51,480 --> 00:27:55,640
SUCH, YOU BEING WORK ON YOUR OWN

713
00:27:55,640 --> 00:28:00,280
CAR AND SO--SO WE OFTEN THINK OF

714
00:28:00,280 --> 00:28:02,960
PAVE GT PROGRAM AS NOT OF THE

715
00:28:02,960 --> 00:28:04,360
MOST ADVANCED MOST AMAIDZING

716
00:28:04,360 --> 00:28:05,560
GENE THERAPY VECTOR GENOME OR

717
00:28:05,560 --> 00:28:07,120
BUT RATHER A GENE THERAPY VECTOR

718
00:28:07,120 --> 00:28:09,320
WILL THAT BE GOOD FOR

719
00:28:09,320 --> 00:28:09,640
EVERYTHING.

720
00:28:09,640 --> 00:28:11,400
SO OR FOR EVERYONE ARE, SO THAT

721
00:28:11,400 --> 00:28:13,320
THE GENE THERAPY VECTOR IS A

722
00:28:13,320 --> 00:28:15,320
DRUG THAT ALMOST EVERYONE CAN

723
00:28:15,320 --> 00:28:18,280
AFFORD, IT CAN TAKE ANY PAY LOAD

724
00:28:18,280 --> 00:28:19,040
TO ALMOST ANYWHERE AND YOU CAN

725
00:28:19,040 --> 00:28:21,600
GET ALL THE PARTS DOWN AT THE

726
00:28:21,600 --> 00:28:23,960
CORNER STORE, AND YOU WILL HAVE

727
00:28:23,960 --> 00:28:26,080
A MANUEL LIKE THE CHILTON MANUEL

728
00:28:26,080 --> 00:28:30,160
THAT SHOWS YOU EXACTLY HOW TO

729
00:28:30,160 --> 00:28:32,920
DEVELOP, MAKE AND REPAIR EVERY

730
00:28:32,920 --> 00:28:34,520
SINGLE ASPECT OF THIS GENE

731
00:28:34,520 --> 00:28:35,520
THERAPY VECTOR.

732
00:28:35,520 --> 00:28:43,040
SO AGAIN, REALLY THE WATCH WORD

733
00:28:43,040 --> 00:28:44,520
FOR AGT, IS ACCESSIBILITY

734
00:28:44,520 --> 00:28:45,480
PARTICULARLY TO THE COMMERCIAL

735
00:28:45,480 --> 00:28:48,760
SECTOR WOULD BE VERY UNLIKELY TO

736
00:28:48,760 --> 00:28:50,760
PLAY A MAJOR ROLE.

737
00:28:50,760 --> 00:28:52,680
SO I KNOW WE'RE RUNNING OUT OF

738
00:28:52,680 --> 00:28:52,880
TIME.

739
00:28:52,880 --> 00:28:56,080
JUST GIVE YOU A COUPLE MINUTES

740
00:28:56,080 --> 00:28:57,080
ABOUT THE SCIENCE AND MORE AND

741
00:28:57,080 --> 00:28:59,200
MORE OF THIS WILL COME OUT IN

742
00:28:59,200 --> 00:29:00,720
PUBLICATIONS AND OUR WEBSITE,

743
00:29:00,720 --> 00:29:02,160
BUT AS YOU IMAGINE, ALTHOUGH I

744
00:29:02,160 --> 00:29:05,560
TRIED TO CONVEY THE IDEA OF THE

745
00:29:05,560 --> 00:29:06,480
PAVE GT INITIATIVES, YOU KNOW AS

746
00:29:06,480 --> 00:29:08,480
THOUGH IT WAS A SIMPLE IDEA,

747
00:29:08,480 --> 00:29:10,000
IT'S ACTUALLY QUITE A

748
00:29:10,000 --> 00:29:13,280
COMPLICATED PROCESS AND QUITE A

749
00:29:13,280 --> 00:29:17,360
LARGE SCALE PROJECT SO AS I

750
00:29:17,360 --> 00:29:18,000
MENTIONED, WE'RE DEVELOPING 4

751
00:29:18,000 --> 00:29:19,480
GENE THERAPIES AT THE SAME TIME

752
00:29:19,480 --> 00:29:22,280
IN PARALLEL, AND THESE ARE

753
00:29:22,280 --> 00:29:24,560
DIVIDED UP INTO 2 PAIRS.

754
00:29:24,560 --> 00:29:29,880
SO IT'S 2 PLUS 2 MODEL AND THE

755
00:29:29,880 --> 00:29:32,840
REASON IS THAT THERE ARE A

756
00:29:32,840 --> 00:29:35,600
NUMBER OF DISEASE STATES,

757
00:29:35,600 --> 00:29:36,240
INDICATION THAT ESSENTIALLY

758
00:29:36,240 --> 00:29:38,920
COULD COME TO BE, COULD ARISE

759
00:29:38,920 --> 00:29:40,880
FROM DIFFERENT KINDS OF

760
00:29:40,880 --> 00:29:42,160
MUTATIONS IN DIFFERENT GENES

761
00:29:42,160 --> 00:29:43,360
THAT GIVE YOU MORE OR LESS THE

762
00:29:43,360 --> 00:29:43,720
SAME DISEASE.

763
00:29:43,720 --> 00:29:47,280
SO THIS IS WHAT WE'RE TRYING TO

764
00:29:47,280 --> 00:29:48,120
ACHIEVE IN TERMS OF

765
00:29:48,120 --> 00:29:50,080
DEMONSTRATING THE ECONOMY OF

766
00:29:50,080 --> 00:29:53,480
SCALE AND DEVELOPING GENES FROM

767
00:29:53,480 --> 00:29:54,680
THE VERY INCEPTION THROUGH

768
00:29:54,680 --> 00:29:56,360
FREQUENT DEVELOPMENT ALL THE WAY

769
00:29:56,360 --> 00:29:58,360
INTO CLINICAL STUDIES.

770
00:29:58,360 --> 00:30:00,280
SO, WE'RE LOOKING AT 2 LIVER

771
00:30:00,280 --> 00:30:04,400
DISEASES AND 2 ORGANIC

772
00:30:04,400 --> 00:30:06,200
[INDISCERNIBLE], AND 2 CNSs, 2

773
00:30:06,200 --> 00:30:07,440
CONGENITAL MY O SINNIAS, THOSE

774
00:30:07,440 --> 00:30:12,320
ARE IN COLLABORATIONS, THOSE ARE

775
00:30:12,320 --> 00:30:14,320
CLOS COLLABORATORS CHUCK

776
00:30:14,320 --> 00:30:18,200
VENDITTI, AT THE HUMAN GENOME

777
00:30:18,200 --> 00:30:21,480
AND CARSON BONNEMANN AT NINDS,

778
00:30:21,480 --> 00:30:24,680
SO THEY'RE WORKING WITH US AND

779
00:30:24,680 --> 00:30:28,120
DOING ALL THE HEAVY LIFTING FOR

780
00:30:28,120 --> 00:30:30,680
THE GENE THERAPY CONSTRUCTS FOR

781
00:30:30,680 --> 00:30:41,960
THESE 4 DISEASES, THE 2

782
00:30:41,960 --> 00:30:47,160
MYASTHENIAS, AND THE 2 IND

783
00:30:47,160 --> 00:30:47,440
PACKAGES.

784
00:30:47,440 --> 00:30:48,560
SO DOING ALL THE

785
00:30:48,560 --> 00:30:50,480
CHARACTERIZATIONS FOR THE PHARMA

786
00:30:50,480 --> 00:30:51,720
CODE, KINETIC PROPERTIES AND

787
00:30:51,720 --> 00:30:53,880
PROPERTIES OF THE GENE VECTORS,

788
00:30:53,880 --> 00:30:56,280
SCALING THEM UP FOR

789
00:30:56,280 --> 00:30:58,040
MANUFACTURING AND SHOWING THAT

790
00:30:58,040 --> 00:31:03,400
THEY CAN BE MANUFACTURED HERE

791
00:31:03,400 --> 00:31:04,680
AND FORMULATED INTO THE PRODUCTS

792
00:31:04,680 --> 00:31:07,760
THAT THEY NEED TO BE AND

793
00:31:07,760 --> 00:31:10,120
IMPORTANTLY TO SHOW IN

794
00:31:10,120 --> 00:31:11,520
TOXICOLOGY STUDIES THAT THESE

795
00:31:11,520 --> 00:31:12,760
GENE VECTORS SHOULD BE SAFE AND

796
00:31:12,760 --> 00:31:14,200
ALONG THE WAY, BUILDING THE

797
00:31:14,200 --> 00:31:16,400
REGULATORY FILINGS THAT WILL BE

798
00:31:16,400 --> 00:31:17,760
NECESSARY FOR INITIAL

799
00:31:17,760 --> 00:31:19,440
DISCUSSIONS WITH THE IND AND

800
00:31:19,440 --> 00:31:23,120
THEN FOR A FINAL SUBMISSION OF

801
00:31:23,120 --> 00:31:28,400
THE IND PACKAGE TO THE FDA, SO

802
00:31:28,400 --> 00:31:30,760
IN OUR SHOP, [INDISCERNIBLE]

803
00:31:30,760 --> 00:31:32,600
SCIENTISTS AND PROJECT MANAGERS,

804
00:31:32,600 --> 00:31:35,080
IN OUR GROUP THAT ARE LEADING

805
00:31:35,080 --> 00:31:36,880
THIS PROJECT ALONG WITH THE

806
00:31:36,880 --> 00:31:39,200
OTHER 60 SCIENTISTS WE HAVE IN

807
00:31:39,200 --> 00:31:42,600
OUR GROUP AND ALONG WITH A

808
00:31:42,600 --> 00:31:44,880
NUMBER OF COMMERCIAL RESEARCH

809
00:31:44,880 --> 00:31:47,280
ORGANIZATIONS AND COMMERCIAL

810
00:31:47,280 --> 00:31:48,280
MANUFACTURING OPERATIONS, CROs

811
00:31:48,280 --> 00:31:51,800
AND CMOs THAT ARE DOING THE

812
00:31:51,800 --> 00:31:55,880
BULK OF THE LARGE SCALE

813
00:31:55,880 --> 00:31:57,040
MANUFACTURING WORK.

814
00:31:57,040 --> 00:31:58,680
AS THESE VECTORS ARE FINISHED,

815
00:31:58,680 --> 00:32:03,480
THEN WE GO BACK TO CHUCK AND

816
00:32:03,480 --> 00:32:04,800
CARSTEN, AND SO AS PART OF THE

817
00:32:04,800 --> 00:32:06,880
ECONOMY OF SKILL, THEY ARE GOING

818
00:32:06,880 --> 00:32:08,680
TO BE CONDUCTING UMBRELLA TRIALS

819
00:32:08,680 --> 00:32:12,280
THAT IS TO USE THE SAME CLINICAL

820
00:32:12,280 --> 00:32:14,800
PROTOCOL WITH THE SAME CLINICAL

821
00:32:14,800 --> 00:32:16,080
END POINTS TO EVALUATE BOTH

822
00:32:16,080 --> 00:32:20,280
PAIRS OF THESE VECTORS IN THE PC

823
00:32:20,280 --> 00:32:22,120
CA, AND MAAD, ARE DIFFERENT

824
00:32:22,120 --> 00:32:23,880
GENES AND DIFFERENT GENE

825
00:32:23,880 --> 00:32:25,200
MUTATIONS BUT THEY CAUSE

826
00:32:25,200 --> 00:32:27,680
DISEASES THAT MANIFEST SIMILAR

827
00:32:27,680 --> 00:32:28,160
FASHION.

828
00:32:28,160 --> 00:32:29,320
AND SO WE'RE HOPING THAT IN THE

829
00:32:29,320 --> 00:32:34,760
NEXT YEAR OR 2, TO START THOSE

830
00:32:34,760 --> 00:32:35,320
CLINICAL STUDIES EXPW WE'RE

831
00:32:35,320 --> 00:32:36,800
EXCITED ABOUT WHERE WE ARE,

832
00:32:36,800 --> 00:32:38,880
WHICH IS A LITTLE BIT ON THE

833
00:32:38,880 --> 00:32:41,480
EARLY END BUT REALLY HITTING OUR

834
00:32:41,480 --> 00:32:42,040
STRIDE.

835
00:32:42,040 --> 00:32:44,320
I ONLY SHOWED 4 PICTURES THERE,

836
00:32:44,320 --> 00:32:46,520
AND IN THIS SLIDE, STILL ONLY

837
00:32:46,520 --> 00:32:48,160
SHOWING A FRACTION OF ALL THE

838
00:32:48,160 --> 00:32:50,280
PEOPLE THAT ARE CONTRIBUTING

839
00:32:50,280 --> 00:32:52,680
VERY CRITICALLY, TO THIS WORK.

840
00:32:52,680 --> 00:32:57,560
OUR GROUP AT NCATS, OUR VARIOUS

841
00:32:57,560 --> 00:33:00,200
GROUPS ADDED NCATS, NOT ONLY OUR

842
00:33:00,200 --> 00:33:02,600
BRANCH THAT I TOLD YOU ABOUT BUT

843
00:33:02,600 --> 00:33:04,240
THE OFFICE OF RARE DISEASES

844
00:33:04,240 --> 00:33:06,200
RESEARCH, YOU CAN SEE PJ'S PHOTO

845
00:33:06,200 --> 00:33:08,240
IN THE MIDDLE OF THAT BOX IN THE

846
00:33:08,240 --> 00:33:10,880
UPPER CENTER, AND A LOT OF THIS

847
00:33:10,880 --> 00:33:13,080
AS I MENTIONED, OR HINTED AT HAS

848
00:33:13,080 --> 00:33:16,440
TO DO WITH LICENSING ISSUES,

849
00:33:16,440 --> 00:33:17,600
INTELLECTUAL PROPERTY, SO, THE

850
00:33:17,600 --> 00:33:20,000
ROLL OF OF OUR OFFICE OF

851
00:33:20,000 --> 00:33:24,080
STRATEGIC ALLIANCES AND THAT AND

852
00:33:24,080 --> 00:33:26,800
NHGRI AND NINDS HAVE BEEN QUITE

853
00:33:26,800 --> 00:33:30,680
CRITICAL, VERY CRITICAL AS WELL.

854
00:33:30,680 --> 00:33:33,880
THERE ARE CHUCK AND CARSTEN AND

855
00:33:33,880 --> 00:33:35,440
FOLKS FROM THEIR GROUP AS WELL

856
00:33:35,440 --> 00:33:37,520
AS OUR CLINICAL DIRECTOR AT

857
00:33:37,520 --> 00:33:38,080
NICHD.

858
00:33:38,080 --> 00:33:40,320
SO AGAIN, THIS IS JUST A SUBSET

859
00:33:40,320 --> 00:33:42,680
OF PLAYERS THAT HAVE TO COME

860
00:33:42,680 --> 00:33:43,680
TOGETHER TO MAKE THIS BIG

861
00:33:43,680 --> 00:33:44,800
PROJECT WORK AND WE'RE HOPING

862
00:33:44,800 --> 00:33:48,760
THAT IN THE END BY CONDUCTING

863
00:33:48,760 --> 00:33:50,560
ALL OF THIS, DEVELOPMENT OF 4

864
00:33:50,560 --> 00:33:52,080
GENE THERAPY VECTORS IN THE

865
00:33:52,080 --> 00:33:54,160
PUBLIC DOMAIN, AND TO MAKE ALL

866
00:33:54,160 --> 00:33:57,480
THE FILINGS AND EVEN THE FDA

867
00:33:57,480 --> 00:33:58,880
FEEDBACK PUBLIC, THIS WILL

868
00:33:58,880 --> 00:34:01,680
REALLY SERVE TO FACILITATE A

869
00:34:01,680 --> 00:34:08,000
MUCH BROADER AND CHEAPER AND

870
00:34:08,000 --> 00:34:09,080
APPROACHABLE PROPOSITION FOR THE

871
00:34:09,080 --> 00:34:12,600
RARE DISEASE COMMUNITY SO THAT

872
00:34:12,600 --> 00:34:14,080
OVER TIME GENE THERAPY AND IN

873
00:34:14,080 --> 00:34:15,520
THIS PARTICULAR CASE WILL BE

874
00:34:15,520 --> 00:34:17,920
MUCH MORE ACCESSIBLE TO ACADEMIC

875
00:34:17,920 --> 00:34:20,680
SCALE WORK AND TO RARE DISEASES

876
00:34:20,680 --> 00:34:23,640
BUT LOW, LOW, NUMBERS OF

877
00:34:23,640 --> 00:34:24,720
PATIENTS AND IN THE END,

878
00:34:24,720 --> 00:34:26,320
OBVIOUSLY THE GOAL IS TO REALLY

879
00:34:26,320 --> 00:34:30,280
TRY TO MOVE THIS GRAPH A BIT, SO

880
00:34:30,280 --> 00:34:33,560
THAT THE 500 DRUGS THAT ARE

881
00:34:33,560 --> 00:34:35,240
APPROVED CURRENTLY THAT IS

882
00:34:35,240 --> 00:34:36,880
ALMOST CREEPING UP YEAR BY WREER

883
00:34:36,880 --> 00:34:38,520
THIS COULD BE 1 OF THE WAYS IN

884
00:34:38,520 --> 00:34:40,080
WHICH WE COULD MAKE A REAL DENT

885
00:34:40,080 --> 00:34:42,920
IN THE FRACTION OF DISEASES THAT

886
00:34:42,920 --> 00:34:46,200
HAVE FDA APPROVED TREATMENTS.

887
00:34:46,200 --> 00:34:48,840
SO THAT'S A THUMB NAIL SKETCH OF

888
00:34:48,840 --> 00:34:49,480
THE PAVE GT PROGRAM.

889
00:34:49,480 --> 00:34:52,080
I HAND IT OVER TO MY COLLEAGUE

890
00:34:52,080 --> 00:34:53,520
T. J. BROOKS NEXT AND HAPPY TO

891
00:34:53,520 --> 00:34:55,400
ANSWER ANY QUESTIONS AT THE END

892
00:34:55,400 --> 00:34:57,720
OF THE HOUR.

893
00:34:57,720 --> 00:34:57,960
THANK YOU DON

894
00:34:57,960 --> 00:34:59,160
THESE ARE LEARNING OBJECTIVES I

895
00:34:59,160 --> 00:35:05,160
WILL TALK ABOUT OR THE BGTC AND

896
00:35:05,160 --> 00:35:07,000
BRIEFLY AT THE END IS TALKING

897
00:35:07,000 --> 00:35:12,400
ABOUT OPPORTUNITIES FOR THE NIH

898
00:35:12,400 --> 00:35:13,080
AND THE SOMATIC CELL GENOME

899
00:35:13,080 --> 00:35:14,800
EDITING PROGRAM THEY THINK WILL

900
00:35:14,800 --> 00:35:15,160
BE OF INTEREST TO THIS SLIDE.

901
00:35:15,160 --> 00:35:22,920
SO THIS IS THE BGTC AND

902
00:35:22,920 --> 00:35:24,480
ORGANIZED BY THE FOWBDATION FOR

903
00:35:24,480 --> 00:35:25,840
THE NIH WHICH VERY IMPORTANTLY

904
00:35:25,840 --> 00:35:27,800
IS NOT PART OF THE NIH AND THAT

905
00:35:27,800 --> 00:35:34,880
HAS IMPLICATIONS FOR HOW WE CAN

906
00:35:34,880 --> 00:35:38,840
DO THIS THERE YOU CAN SEE THE

907
00:35:38,840 --> 00:35:41,160
GROUP, MIERS, PETER MARKS, AT

908
00:35:41,160 --> 00:35:42,960
FDA WHO WAS REALLY INVOLVED IN

909
00:35:42,960 --> 00:35:44,600
SPEARHEADING THIS PROGRAM AND

910
00:35:44,600 --> 00:35:46,320
GETTING IT OFF THE GROUND WITH

911
00:35:46,320 --> 00:35:48,200
GREAT MANAGEMENT AND

912
00:35:48,200 --> 00:35:49,120
PARTICIPATION FROM COURTNEY AND

913
00:35:49,120 --> 00:35:50,720
BRAD FROM THE NIH.

914
00:35:50,720 --> 00:35:51,360
NEXT SLIDE.

915
00:35:51,360 --> 00:35:55,520
SO THE GOALS OF THE BGTC ARE IN

916
00:35:55,520 --> 00:35:56,920
SOME WAYS SIMILAR TO THOSE OF

917
00:35:56,920 --> 00:35:59,680
PAVE GT WHICH IS TO AAV

918
00:35:59,680 --> 00:36:00,320
TECHNOLOGY MORE ACCESSIBLE

919
00:36:00,320 --> 00:36:02,760
PARTICULARLY FOR DISEASES OF NO

920
00:36:02,760 --> 00:36:04,120
COMMERCIAL INTEREST.

921
00:36:04,120 --> 00:36:06,560
ACCELERATE THE POTENTIAL OF THE

922
00:36:06,560 --> 00:36:08,320
STREAMLINE OF CLINICAL AND

923
00:36:08,320 --> 00:36:10,240
PREPRODUCT TESTING AND

924
00:36:10,240 --> 00:36:10,760
FACILITATE SCIENTIFIC AND

925
00:36:10,760 --> 00:36:11,720
REGULATORY ADVANCE THAT WILL

926
00:36:11,720 --> 00:36:14,320
BENEFIT THE WHOLE FIELD OF AAV

927
00:36:14,320 --> 00:36:16,720
GENE THERAPY AND ULTIMATELY

928
00:36:16,720 --> 00:36:18,320
BRING GENE THERAPIES TO

929
00:36:18,320 --> 00:36:20,480
INDIVIDUALS IN NEED SOONER.

930
00:36:20,480 --> 00:36:21,800
NEXT SLIDE.

931
00:36:21,800 --> 00:36:24,480
SO THERE'S BASICALLY 2

932
00:36:24,480 --> 00:36:26,720
COMPONENTS TO THE BGTC AND WE

933
00:36:26,720 --> 00:36:28,680
ALL A BASIC BIOLOGY COMPONENT ON

934
00:36:28,680 --> 00:36:30,440
THE TOP WHICH IS FOCUSED ON NOT

935
00:36:30,440 --> 00:36:32,680
JUST A BASIC BIOLOGY OF AAV IN

936
00:36:32,680 --> 00:36:39,280
GENERAL, BUT MORE SPECIFICALLY

937
00:36:39,280 --> 00:36:41,400
TO ENHANCE VECTOR GENERATION AND

938
00:36:41,400 --> 00:36:42,600
ALSO ENHANCE THERAPEUTIC GENE

939
00:36:42,600 --> 00:36:44,120
EXPRESSION IN PEOPLE AND THERE'S

940
00:36:44,120 --> 00:36:47,600
ALSO A CLINICAL COMPONENT THAT

941
00:36:47,600 --> 00:36:50,720
IS FOCUSED ON WHAT WE CALL

942
00:36:50,720 --> 00:36:51,520
BESPOKE CLINICAL APPLICATIONS,

943
00:36:51,520 --> 00:36:52,720
IN THIS CASE, IT'S REALLY

944
00:36:52,720 --> 00:36:54,800
ANOTHER WAY OF SAYING DISEASE OF

945
00:36:54,800 --> 00:36:57,200
NO COMMERCIAL INTEREST, WE'RE

946
00:36:57,200 --> 00:36:59,720
TRYING TO STANDARDIZE VARIOUS

947
00:36:59,720 --> 00:37:02,280
ASPECTS OF THE PROJECT AND TO

948
00:37:02,280 --> 00:37:04,880
COME UP WITH A PATHWAY TO START

949
00:37:04,880 --> 00:37:06,560
UF AAV GENE THERAPY CLINICAL

950
00:37:06,560 --> 00:37:10,720
TRIALS MORE EFFICIENTLY.

951
00:37:10,720 --> 00:37:11,560
NEXT SLIDE.

952
00:37:11,560 --> 00:37:13,920
AND THESE 2 EFFORTS ARE RELATED

953
00:37:13,920 --> 00:37:16,280
TO EACH OTHER, ULTIMATELY WE

954
00:37:16,280 --> 00:37:19,240
ANTICIPATE THAT THEY ENHANCING

955
00:37:19,240 --> 00:37:22,320
VECTOR GENERATION WILL IMPACT

956
00:37:22,320 --> 00:37:24,120
VECTOR MANUFACTURING AND

957
00:37:24,120 --> 00:37:24,840
ANALYTICS IN THE CLINICAL

958
00:37:24,840 --> 00:37:25,320
COMOPPOSITE BEHAVIORIAL

959
00:37:25,320 --> 00:37:26,120
PHENOTYPE AND THEN THE SIMILARLY

960
00:37:26,120 --> 00:37:28,520
WE HOPE THAT ENHANCING GENE

961
00:37:28,520 --> 00:37:29,400
EXPRESSION IN PATIENTS CAN BE

962
00:37:29,400 --> 00:37:31,600
INCLUDED IN SOME OF OUR CLINICAL

963
00:37:31,600 --> 00:37:32,160
TRIALS.

964
00:37:32,160 --> 00:37:34,880
TO THE EXTENT THAT WE CAN

965
00:37:34,880 --> 00:37:37,480
IDENTIFY WAYS TO IMPROVE BOTH OF

966
00:37:37,480 --> 00:37:46,000
THOSE STEPS.

967
00:37:46,000 --> 00:37:46,520
NEXT SLIDE.

968
00:37:46,520 --> 00:37:52,160
SO AS WE'RE SETTING UP BCGT,

969
00:37:52,160 --> 00:37:54,880
WITH THIS, WE HAD A LOT OF

970
00:37:54,880 --> 00:37:56,840
PARTICIPANTS WITH INDUSTRY AND

971
00:37:56,840 --> 00:37:58,640
ACADEMIA INVOLVED AND SOME HICK

972
00:37:58,640 --> 00:37:59,520
UPS WITH COVID, BUT WE THOUGHT

973
00:37:59,520 --> 00:38:03,000
ABOUT A WAY TO,A PROVE AAV

974
00:38:03,000 --> 00:38:04,320
VECTOR PRODUCTION WOULD BE TO

975
00:38:04,320 --> 00:38:09,520
FOCUS ON THE INDIVIDUAL STEPS

976
00:38:09,520 --> 00:38:11,080
INVOLVED IN MAKING AAV GENE

977
00:38:11,080 --> 00:38:12,520
THERAPIES AND THAT WOULD INCLUDE

978
00:38:12,520 --> 00:38:14,320
THE STEPS INVOLVED FROM PUTTING

979
00:38:14,320 --> 00:38:18,040
THE THERAPEUTIC GENE CONSTRUCT

980
00:38:18,040 --> 00:38:18,960
AND THE HELPER VIRUSES AND

981
00:38:18,960 --> 00:38:21,720
THINGS INTO CELLS AND THEN WHAT

982
00:38:21,720 --> 00:38:22,520
HAPPENS TO THOSE CONSTRUCTS IN

983
00:38:22,520 --> 00:38:25,600
THE CELLS AND KIND OF IDENTIFY

984
00:38:25,600 --> 00:38:26,960
EACH INDIVIDUAL STEP AND SEE IF

985
00:38:26,960 --> 00:38:31,120
WE CAN OPTIMIZE EACH 1 OF THOSE

986
00:38:31,120 --> 00:38:31,680
INDIVIDUAL STEPS SEPARATELY.

987
00:38:31,680 --> 00:38:36,840
AND WE PUT OUT A REQUEST FOR

988
00:38:36,840 --> 00:38:37,480
PROPOSALS FOR APPLICATIONS IN

989
00:38:37,480 --> 00:38:39,280
THE COMMUNITY ON HOW TO DO THIS

990
00:38:39,280 --> 00:38:42,040
THAT REQUEST WAS CLOSED ON

991
00:38:42,040 --> 00:38:45,760
FEBRUARY 18th.

992
00:38:45,760 --> 00:38:46,160
NEXT SLIDE.

993
00:38:46,160 --> 00:38:48,960
AND WE THOUGHT OF DOING

994
00:38:48,960 --> 00:38:49,960
SOMETHING SIMILAR FOR IMPROVING

995
00:38:49,960 --> 00:38:50,960
AAV GENE EXPRESSION TO THINK OF

996
00:38:50,960 --> 00:38:52,400
ALL OF THE STEPS THAT HAPPEN

997
00:38:52,400 --> 00:38:54,520
AFTER TARGET CELL IS INFECTED

998
00:38:54,520 --> 00:38:58,440
WITH A THERAPEUTIC AAV CONSTRUCT

999
00:38:58,440 --> 00:39:00,280
ALL THE WAY THROUGH THE PROCESS

1000
00:39:00,280 --> 00:39:01,880
OF PRODUCING THE THERAPEUTIC

1001
00:39:01,880 --> 00:39:03,320
GENE WHICH OF COURSE IS WHAT IS

1002
00:39:03,320 --> 00:39:05,000
NEEDED TO GET TO THERAPEUTIC

1003
00:39:05,000 --> 00:39:06,640
EFFICACY, IF YOU LOOK AT EACH 1

1004
00:39:06,640 --> 00:39:08,880
OF THOSE STEPS INDIVIDUAL LEE,

1005
00:39:08,880 --> 00:39:11,440
CAN WE OPTIMIZE EACH 1 OF THEM.

1006
00:39:11,440 --> 00:39:13,640
AND HERE AGAIN, WE HAD FUNDING

1007
00:39:13,640 --> 00:39:16,440
OPPORTUNITIES THAT WERE OPENED

1008
00:39:16,440 --> 00:39:21,360
UP AND NEXT SLIDE.

1009
00:39:21,360 --> 00:39:24,560
AND SO THE WHYED WE HAD IS TO

1010
00:39:24,560 --> 00:39:26,720
ASK THE COMMUNITY FOR HIGH

1011
00:39:26,720 --> 00:39:28,600
THROUGH PUT SCREENING COMPATIBLE

1012
00:39:28,600 --> 00:39:32,400
ASSAYS TO OPTIMIZE EACH OF THOSE

1013
00:39:32,400 --> 00:39:33,680
MECHANISTIC STEPS, WITH THE IDEA

1014
00:39:33,680 --> 00:39:35,720
THAT ONCE WE'VE GOTTEN THOSE

1015
00:39:35,720 --> 00:39:37,400
ASSAYS WE WOULD CARRY OUT HIGH

1016
00:39:37,400 --> 00:39:39,240
THROUGH PUT SCREENING EFFORTS

1017
00:39:39,240 --> 00:39:41,280
USING FOR EXAMPLE, WHOLE GENOME

1018
00:39:41,280 --> 00:39:43,600
SCREENS TO IMPROVE AAV VECTOR

1019
00:39:43,600 --> 00:39:44,480
GENERATION, OR SCREENING

1020
00:39:44,480 --> 00:39:46,440
APPROVED DRUGS IN TARGET CELLS

1021
00:39:46,440 --> 00:39:49,880
FOR THE CLINICAL TRIALS, FOR THE

1022
00:39:49,880 --> 00:39:51,520
PROJECTS ON ENHANCING

1023
00:39:51,520 --> 00:39:52,440
THERAPEUTIC GENE EXPRESSION AND

1024
00:39:52,440 --> 00:39:56,120
WE PLAN TO DO THIS WORKING WITH

1025
00:39:56,120 --> 00:39:57,400
THE NCATS INTRAMURAL HIGH

1026
00:39:57,400 --> 00:39:58,320
THROUGH PUT SCREENING FACILITY

1027
00:39:58,320 --> 00:40:01,640
BUT WE ALSO WROTE THESE FUNDING

1028
00:40:01,640 --> 00:40:03,320
OPPORTUNITIES TO BE OPEN TO

1029
00:40:03,320 --> 00:40:04,520
OTHER NOVEL APPROACHES AS WELL

1030
00:40:04,520 --> 00:40:06,840
TO CAST A VERY WIDE NET TO THE

1031
00:40:06,840 --> 00:40:09,840
COMMUNITY REALIZING THAT A GOAL

1032
00:40:09,840 --> 00:40:11,240
TO THESE--THE ULTIMATE RESULTS

1033
00:40:11,240 --> 00:40:12,320
OF THESE EFFORTS WERE ALL GOING

1034
00:40:12,320 --> 00:40:14,880
TO BE PUT INTO THE PUBLIC DOMAIN

1035
00:40:14,880 --> 00:40:16,680
AND THAT'S A VERY IMPORTANT

1036
00:40:16,680 --> 00:40:18,920
ASPECT OF THIS PUBLIC PRIVATE

1037
00:40:18,920 --> 00:40:19,240
PARTNERSHIP.

1038
00:40:19,240 --> 00:40:21,480
WE TRIED TO MAKE THE WHOLE THING

1039
00:40:21,480 --> 00:40:22,320
IN THE PRECOMPETITIVE SPACE SO

1040
00:40:22,320 --> 00:40:23,880
WE COULD PUT ALL THE DATA AND

1041
00:40:23,880 --> 00:40:26,320
ALL THE RESULTS INTO THE PUBLIC

1042
00:40:26,320 --> 00:40:27,240
DOMAIN TO BENEFIT THE WHOLE

1043
00:40:27,240 --> 00:40:28,920
COMMUNITY AND THAT OF COURSE IS

1044
00:40:28,920 --> 00:40:30,680
OBVIOUSLY GOING TO LIMIT THE

1045
00:40:30,680 --> 00:40:34,200
KIND OF APPLICATIONS WE GET TO

1046
00:40:34,200 --> 00:40:35,520
THE EXTENT THAT PEOPLE ARE

1047
00:40:35,520 --> 00:40:37,720
WORKING ON THINGS WHERE THEY'RE

1048
00:40:37,720 --> 00:40:38,240
VERY CONCERNED ABOUT

1049
00:40:38,240 --> 00:40:39,120
INTELLECTUAL PROPERTY AND THEY

1050
00:40:39,120 --> 00:40:40,320
WOULD OBVIOUSLY NOT APPLY AND WE

1051
00:40:40,320 --> 00:40:46,320
DID UNDERSTAND THAT AND DO

1052
00:40:46,320 --> 00:40:46,960
UNDERSTAND THAT.

1053
00:40:46,960 --> 00:40:47,600
NEXT SLIDE.

1054
00:40:47,600 --> 00:40:48,840
SO THE APPLICATIONS WERE

1055
00:40:48,840 --> 00:40:50,320
RECEIVED IN FEBRUARY AND WE'RE

1056
00:40:50,320 --> 00:40:53,160
STARTING THE REVIEW PROCESS OF

1057
00:40:53,160 --> 00:40:53,360
THEM.

1058
00:40:53,360 --> 00:40:56,320
AS WE GO THROUGH THEM WE'RE

1059
00:40:56,320 --> 00:41:00,280
IDENTIFYING YOU KNOW DIFFERENT

1060
00:41:00,280 --> 00:41:02,240
PROJECTS AND PLAN TO HAVE A

1061
00:41:02,240 --> 00:41:05,000
FORMAL REVIEW IN THE NEXT FEW

1062
00:41:05,000 --> 00:41:06,720
WEEKS OR SO, I WOULD LIKE TO

1063
00:41:06,720 --> 00:41:07,760
MENTION THERE IS A POSSIBILITY

1064
00:41:07,760 --> 00:41:11,440
THAT WE MAY END UP RELEASING

1065
00:41:11,440 --> 00:41:12,240
ADDITIONAL FUNDING

1066
00:41:12,240 --> 00:41:12,560
OPPORTUNITIES.

1067
00:41:12,560 --> 00:41:14,480
SO THOSE OF YOU WHO THINK YOU

1068
00:41:14,480 --> 00:41:16,120
MIGHT HAVA AN INTEREST IN THIS

1069
00:41:16,120 --> 00:41:18,480
AND MAY HAVE MISSED THE FIRST 1

1070
00:41:18,480 --> 00:41:20,000
THERE'S POSSIBILITY OF

1071
00:41:20,000 --> 00:41:23,680
ADDITIONAL FUNDING OPPORTUNITIES

1072
00:41:23,680 --> 00:41:24,440
IN THE FUTURE.

1073
00:41:24,440 --> 00:41:24,760
NEXT SLIDE.

1074
00:41:24,760 --> 00:41:27,120
SO 1 OF THE THINGS WE'RE DOING

1075
00:41:27,120 --> 00:41:29,120
WITHIN THE MANUFACTURING WORK

1076
00:41:29,120 --> 00:41:31,720
STREAM OF THE BGTC IS TO SEE IF

1077
00:41:31,720 --> 00:41:35,680
YOU CAN DEVELOP A MINIMAL SET OF

1078
00:41:35,680 --> 00:41:38,320
QUALITY ATTRIBUTES THAT WOULD BE

1079
00:41:38,320 --> 00:41:56,520
USED TO DETERMINE IF A AN AV,'S

1080
00:41:56,520 --> 00:42:02,400
MANUFACTURED IN PHASE, YOU MIGHT

1081
00:42:02,400 --> 00:42:05,320
THINK THAT THE FACT THAT THE

1082
00:42:05,320 --> 00:42:13,760
IDEA OF DIFFERENT SETS OF DATA,

1083
00:42:13,760 --> 00:42:15,520
DEMONSTRATE SAFETY, OF SAFETY

1084
00:42:15,520 --> 00:42:19,480
ATTRIBUTES, SO, WORKING WITH A

1085
00:42:19,480 --> 00:42:21,440
GROUP OF INDIVIDUALS FROM BOTH

1086
00:42:21,440 --> 00:42:26,040
PUBLIC AND PRIVATE ENTITIES AT

1087
00:42:26,040 --> 00:42:26,520
THIS MINIMAL SET.

1088
00:42:26,520 --> 00:42:27,880
MEETING EVERY OTHER WEEK NOW TO

1089
00:42:27,880 --> 00:42:32,360
KIND OF NARROW IT DOWN, IT'S A

1090
00:42:32,360 --> 00:42:32,920
SURPRISINGLY, COMPLICATED

1091
00:42:32,920 --> 00:42:35,200
PROCESS BUT I THINK WE'RE MAKING

1092
00:42:35,200 --> 00:42:37,320
GOOD PROGRESS ON IT, AND WE'RE

1093
00:42:37,320 --> 00:42:39,280
REALLY FOCUSED ON THINGS THAT

1094
00:42:39,280 --> 00:42:41,040
WOULD BE INDEPENDENT OF THE

1095
00:42:41,040 --> 00:42:42,320
TYPE, SORT OF STANDARDIZED TYPES

1096
00:42:42,320 --> 00:42:45,080
OF ASSAYS YOU DOA THAT WOULD BE

1097
00:42:45,080 --> 00:42:46,920
RELEVANT ANY AAV THAT WOULD GO

1098
00:42:46,920 --> 00:42:49,400
INTO HUMANS, THINGS LIKE FOR

1099
00:42:49,400 --> 00:42:53,600
EXAMPLE, DETERMINING THAT IT'S

1100
00:42:53,600 --> 00:42:53,920
STERILE.

1101
00:42:53,920 --> 00:42:55,400
THE HOPE IS THAT WE CAN

1102
00:42:55,400 --> 00:43:00,240
ULTIMATELY COME UP WITH THIS

1103
00:43:00,240 --> 00:43:02,120
MINIMAL SET OF ATTRIBUTES AND

1104
00:43:02,120 --> 00:43:03,520
THEN WE WILL HAVE A DISCUSSION

1105
00:43:03,520 --> 00:43:05,120
WITH THE F, DIDA TO GET THEIR

1106
00:43:05,120 --> 00:43:06,160
FEEDBACK, AND AGAIN WITH THE

1107
00:43:06,160 --> 00:43:08,040
GOAL OF DEVELOPING SORT OF A

1108
00:43:08,040 --> 00:43:10,920
STANDARD HERE THAT CAN BE USED

1109
00:43:10,920 --> 00:43:12,440
BY OTHER INVESTIGATORS, NOT ONLY

1110
00:43:12,440 --> 00:43:13,880
WITHIN THE UNITED STATES, BUT

1111
00:43:13,880 --> 00:43:17,920
EVEN IN ON THE COUNTRIES AS

1112
00:43:17,920 --> 00:43:18,200
WELL.

1113
00:43:18,200 --> 00:43:19,640
AND THEN OF COURSE WE WILL USE

1114
00:43:19,640 --> 00:43:21,720
THOSE ATTRIBUTES IN OUR OWN

1115
00:43:21,720 --> 00:43:23,320
CLINICAL TRIALS WITHIN THE GGTC

1116
00:43:23,320 --> 00:43:24,320
AND THIS IS 1 TIEWNLT WHERE I

1117
00:43:24,320 --> 00:43:26,480
THINK WE CAN HAVE SOME ACTION

1118
00:43:26,480 --> 00:43:31,080
WITH PAVE GT AS WELL. 

1119
00:43:31,080 --> 00:43:31,520
THE NEXT SLIDE.

1120
00:43:31,520 --> 00:43:34,640
SO THE IDEA OF THE CLINICAL

1121
00:43:34,640 --> 00:43:36,480
PROGRAM IS REALLY AGAIN SIMILAR

1122
00:43:36,480 --> 00:43:39,680
TO PAVE GT IS TO NOT TRY TO DO

1123
00:43:39,680 --> 00:43:41,720
ANYTHING NEW AND CUTTING EDGE

1124
00:43:41,720 --> 00:43:43,120
WITH REGARD TO THE VECTORS, WE

1125
00:43:43,120 --> 00:43:44,240
WANT TO SPECIFICALLY USE THE

1126
00:43:44,240 --> 00:43:46,960
VECTORS THAT HAVE BEEN USED

1127
00:43:46,960 --> 00:43:50,080
PREVIOUSLY FOR AAV GENE THERAPY

1128
00:43:50,080 --> 00:43:51,280
TRIALS WHERE THERE'S APPROVED

1129
00:43:51,280 --> 00:43:52,800
INDs, WE WANT TO WORK WITH

1130
00:43:52,800 --> 00:43:54,360
MANUFACTURES WHO HAVE MADE

1131
00:43:54,360 --> 00:43:55,880
VECTOR AND GOTTEN INDs AND

1132
00:43:55,880 --> 00:43:58,400
BASICALLY TAKE THOSE SAME

1133
00:43:58,400 --> 00:44:01,920
PROCESSES, AND PUT DIFFERENT

1134
00:44:01,920 --> 00:44:02,960
THERAPEUTIC GENES IN AND

1135
00:44:02,960 --> 00:44:06,960
ULTIMATELY COME UP WITH A

1136
00:44:06,960 --> 00:44:10,040
THERAPEUTIC STREAMLINE PLATFORM

1137
00:44:10,040 --> 00:44:11,760
TO GET TO CLINICAL TRIALS AND

1138
00:44:11,760 --> 00:44:13,520
REALLY TO TRY TO ALSO TAKE

1139
00:44:13,520 --> 00:44:15,320
ADVANTAGE OF MASTER FILES AND

1140
00:44:15,320 --> 00:44:18,800
PREVIOUS DATA THAT THESE

1141
00:44:18,800 --> 00:44:19,520
DIFFERENT MANUFACTURES WILL HAVE

1142
00:44:19,520 --> 00:44:22,800
IN EXPERIENCE WITH THE FDA.

1143
00:44:22,800 --> 00:44:25,040
AND SO WE ANTICIPATE THAT VECTOR

1144
00:44:25,040 --> 00:44:26,080
MANUFACTURING WITHIN THE

1145
00:44:26,080 --> 00:44:27,920
VTT WILL BE DONE BY SOME OF THE

1146
00:44:27,920 --> 00:44:29,720
COMMERCIAL PARTNERS THAT ARE

1147
00:44:29,720 --> 00:44:31,840
PARTICIPATING IN THE PROGRAM.

1148
00:44:31,840 --> 00:44:34,320
AND WE ENVISION HAVING A LIMITED

1149
00:44:34,320 --> 00:44:35,960
NUMBER OF AAV SEROTYPES, THESE

1150
00:44:35,960 --> 00:44:37,520
WILL BE 1S THAT HAVE ALREADY

1151
00:44:37,520 --> 00:44:40,280
BEEN USED IN CLINICAL TRIALS AND

1152
00:44:40,280 --> 00:44:43,320
A LIMITED NUMBER OF DELIVERY

1153
00:44:43,320 --> 00:44:44,600
MODALITIES TO TRY TO GET

1154
00:44:44,600 --> 00:44:48,720
DIVERSITY OF TISSUES TO INSURE

1155
00:44:48,720 --> 00:44:53,000
THAT THIS CLINICAL TRIAL

1156
00:44:53,000 --> 00:44:54,720
PIPELINE IS AS BROAD AS WE CAN

1157
00:44:54,720 --> 00:45:00,240
POSSIBLY MAKE IT.

1158
00:45:00,240 --> 00:45:00,680
NEXT SLIDE.

1159
00:45:00,680 --> 00:45:02,920
SO TO DO THE CLINICAL TRIAL

1160
00:45:02,920 --> 00:45:07,200
WOULD BE LOOKING FOR PERHAPS 5

1161
00:45:07,200 --> 00:45:09,760
OR 6 DISEASES THAT HAVE A HIGH

1162
00:45:09,760 --> 00:45:11,720
LIKELIHOOD OF CLINICAL SUCCESS

1163
00:45:11,720 --> 00:45:12,560
FOR AAV GENE THERAPY TO BE

1164
00:45:12,560 --> 00:45:13,920
SELECTED BY THE STEERING

1165
00:45:13,920 --> 00:45:16,520
COMMITTEE AND SO, WE WOULD BE

1166
00:45:16,520 --> 00:45:18,480
THINKING BASICALLY OF YOU KNOW

1167
00:45:18,480 --> 00:45:19,320
SINGLE GENE DISORDERS, VERY

1168
00:45:19,320 --> 00:45:20,560
IMPORTANTLY THESE HAVE TO BE

1169
00:45:20,560 --> 00:45:24,320
DISEASE WHERE THERE'S NO

1170
00:45:24,320 --> 00:45:24,880
COMMERCIAL INTEREST.

1171
00:45:24,880 --> 00:45:26,840
WE WOULD LIKE TO HAVE DISEASES

1172
00:45:26,840 --> 00:45:27,840
WITH THE CLINICAL OUTCOMES CAN

1173
00:45:27,840 --> 00:45:30,760
BE ASSESSED IN A FAIRLY SHORT

1174
00:45:30,760 --> 00:45:33,400
PERIOD OF TIME, ALREADY

1175
00:45:33,400 --> 00:45:34,320
ASSEMBLED PATIENT GROUPS,

1176
00:45:34,320 --> 00:45:40,280
CLINICAL INVESTIGATORS, ET

1177
00:45:40,280 --> 00:45:40,760
CETERA.

1178
00:45:40,760 --> 00:45:42,520
AND TO RUN THESE PROGRAMS AND

1179
00:45:42,520 --> 00:45:43,800
BASED ON DEVELOPING THEM,

1180
00:45:43,800 --> 00:45:45,440
ULTIMATELY COME UP WITH THE

1181
00:45:45,440 --> 00:45:46,680
STANDARDIZED PROCESS FOR

1182
00:45:46,680 --> 00:45:49,920
STARTING OFF CLINICAL TRIALS

1183
00:45:49,920 --> 00:45:51,520
EMPLOY AND SO THE WAY WE'VE DONE

1184
00:45:51,520 --> 00:45:53,120
THIS IS BASICALLY PUT OUT A

1185
00:45:53,120 --> 00:45:54,440
FUNDING OPPORTUNITY AND ASK THE

1186
00:45:54,440 --> 00:45:56,280
COMMUNITY TO SUBMIT THEIR

1187
00:45:56,280 --> 00:45:59,680
PROPOSED DISEASES FOR

1188
00:45:59,680 --> 00:46:00,320
CONSIDERATION.

1189
00:46:00,320 --> 00:46:01,120
NEXT SLIDE.

1190
00:46:01,120 --> 00:46:05,360
AND SO, YOU OPEN THAT PROCESS,

1191
00:46:05,360 --> 00:46:06,320
WE'VE GOTTEN MULTIPLE

1192
00:46:06,320 --> 00:46:08,520
SUBMISSIONS AND WE'RE NOT GOING

1193
00:46:08,520 --> 00:46:09,840
TO BE TRYING TO DOWN SELECT TO

1194
00:46:09,840 --> 00:46:14,920
GET TO A SMALLER NUMBER OF

1195
00:46:14,920 --> 00:46:16,120
DISEASES AND ULTIMATELY CHOOSE

1196
00:46:16,120 --> 00:46:18,880
THE 5 OR 6 OR SO THAT WE WILL

1197
00:46:18,880 --> 00:46:20,400
ULTIMATELY STUDY AND

1198
00:46:20,400 --> 00:46:21,320
IMPORTANTLY, THESE WILL HAVE TO

1199
00:46:21,320 --> 00:46:22,800
BE PAIR WTD VECTOR MANUFACTURING

1200
00:46:22,800 --> 00:46:25,920
FOR DOING THE FIRST IN HUMAN

1201
00:46:25,920 --> 00:46:31,040
CLINICAL TRIALS. 

1202
00:46:31,040 --> 00:46:32,000
NEXT SLIDE.

1203
00:46:32,000 --> 00:46:33,280
AND ULTIMATELY COME OUT OF THIS

1204
00:46:33,280 --> 00:46:36,120
IS SOMETHING LIKE A MANUEL FOR

1205
00:46:36,120 --> 00:46:40,680
AAV GENE THERAPY CLINICAL

1206
00:46:40,680 --> 00:46:41,480
DEVELOPMENT, INSIGHTS AND

1207
00:46:41,480 --> 00:46:42,760
LEARNINGS THAT FACILITATE THE

1208
00:46:42,760 --> 00:46:45,600
SUCCESS OF FUTURE GENE THERAPYS,

1209
00:46:45,600 --> 00:46:47,520
INCLUDING IMPROVEMENTS IN AAV

1210
00:46:47,520 --> 00:46:48,280
TARGET GENE EXPRESSION,

1211
00:46:48,280 --> 00:46:50,840
IMPROVEMENT NEGLIGENCE THE WAY

1212
00:46:50,840 --> 00:46:53,200
WE MAKE CLINICAL AAV VECTORS

1213
00:46:53,200 --> 00:46:55,040
THIS HARMONIZED AND VALIDATED

1214
00:46:55,040 --> 00:46:56,960
SET OF CLINICAL QUALITY

1215
00:46:56,960 --> 00:46:58,120
ATTRIBUTES AND STANDARDIZED

1216
00:46:58,120 --> 00:47:02,360
REGULATORY SUBMISSION PACKAGE

1217
00:47:02,360 --> 00:47:04,080
AND TEMPLATES, IN PARTICULARLY

1218
00:47:04,080 --> 00:47:05,400
REALLY UTILIZING EXPLOITING

1219
00:47:05,400 --> 00:47:07,800
VECTOR MASTER FILE SO THAT WHEN

1220
00:47:07,800 --> 00:47:11,880
NEW DISEASES NEED TO BE

1221
00:47:11,880 --> 00:47:13,000
ADDRESSED AND THERAPIES FOR NEW

1222
00:47:13,000 --> 00:47:15,240
DISEASE NEED TO BE ADDRESSED

1223
00:47:15,240 --> 00:47:16,320
MANUFACTURES CAN RELAY AS MUCH

1224
00:47:16,320 --> 00:47:19,160
AS POSSIBLE ON PREVIOUSLY

1225
00:47:19,160 --> 00:47:22,320
GENERATED DATA ON THE PRODUCTION

1226
00:47:22,320 --> 00:47:23,640
OF THOSE AAV VECTORS.

1227
00:47:23,640 --> 00:47:25,600
AGAIN, A VERY SIMILAR IDEA TO

1228
00:47:25,600 --> 00:47:31,000
WHAT WE HAVE IN PAVE GT.

1229
00:47:31,000 --> 00:47:31,400
NEXT SLIDE.

1230
00:47:31,400 --> 00:47:34,320
SO THESE ARE THE PARTNERS WE

1231
00:47:34,320 --> 00:47:36,920
HAVE IN THE BGTC, YOU CAN SEE WE

1232
00:47:36,920 --> 00:47:38,240
HAVE PARTICIPATION IN MULTIPLE

1233
00:47:38,240 --> 00:47:40,920
NIH INSTITUTES AND CENTERS, 11

1234
00:47:40,920 --> 00:47:42,520
DIFFERENT ENTITIES, VERY

1235
00:47:42,520 --> 00:47:44,160
IMPORTANTLY, WE HAVE THE ACTIVE

1236
00:47:44,160 --> 00:47:46,360
PARTICIPATION OF THE FTA CENTER

1237
00:47:46,360 --> 00:47:48,000
FOR BIOLOGICS, AND AS I SAID,

1238
00:47:48,000 --> 00:47:50,360
PETER MARK SYSTEM INTIMATELY

1239
00:47:50,360 --> 00:47:51,240
INVOLVED IN INITIATING THIS

1240
00:47:51,240 --> 00:47:53,440
PROGRAM WHICH WAS CRUCIAL FOR

1241
00:47:53,440 --> 00:47:54,920
ITS SUCCESS ON THE RIGHT YOU CAN

1242
00:47:54,920 --> 00:47:59,680
SEE OUR VARIOUS OTHER PARTNERS

1243
00:47:59,680 --> 00:48:00,600
INCLUDING MULTIPLE FOR PROFIT

1244
00:48:00,600 --> 00:48:02,720
ENTITIES AS WELL AS NONPROFITS

1245
00:48:02,720 --> 00:48:04,600
AS WELL.

1246
00:48:04,600 --> 00:48:10,320
SO OVERALL QUITE LARGE

1247
00:48:10,320 --> 00:48:10,600
CONSORTIUM.

1248
00:48:10,600 --> 00:48:11,120
NEXT SLIDE.

1249
00:48:11,120 --> 00:48:14,040
SO THE WAY THE STRUCTURE THAT

1250
00:48:14,040 --> 00:48:17,680
WORKS WE HAVE AN EXECUTIVE

1251
00:48:17,680 --> 00:48:20,240
COMMITTEE THAT OVERSEES THE BGTC

1252
00:48:20,240 --> 00:48:23,600
AND THIS COMMITTEE SEES A LOT OF

1253
00:48:23,600 --> 00:48:25,680
OTHER FNIH ACCELERATED MEDICINES

1254
00:48:25,680 --> 00:48:26,840
PARTNERSHIP, OUR STEERING

1255
00:48:26,840 --> 00:48:30,160
COMMITTEE IS MADE UP OF 50% NIH

1256
00:48:30,160 --> 00:48:31,800
AND 50% PRIVATE SECTOR

1257
00:48:31,800 --> 00:48:38,600
INDIVIDUALS, AND WE'VE GOT

1258
00:48:38,600 --> 00:48:40,520
DIFFERENT AND TRY TO ADDRESS ALL

1259
00:48:40,520 --> 00:48:43,120
THE DIFFERENT ASPECTS THAT WE'RE

1260
00:48:43,120 --> 00:48:47,520
TRYING TO ACHIEVE AND THEN THE

1261
00:48:47,520 --> 00:48:48,080
BGTC.

1262
00:48:48,080 --> 00:48:48,640
NEXT SLIDE.

1263
00:48:48,640 --> 00:48:50,000
AND SO THIS KIND OF GOES THROUGH

1264
00:48:50,000 --> 00:48:52,000
SOME OF THE DIFFERENT COMMITTEE

1265
00:48:52,000 --> 00:48:52,880
ROLES AND I WON'T GO THROUGH

1266
00:48:52,880 --> 00:48:54,560
THIS IN A LOT OF DETAIL, BUT YOU

1267
00:48:54,560 --> 00:48:57,120
CAN SEE HOW WE'RE TRYING TO

1268
00:48:57,120 --> 00:48:59,320
DEVELOP THE OVERSIGHT OF THE

1269
00:48:59,320 --> 00:49:00,960
BGTC TO INCLUDE THE PERSPECTIVES

1270
00:49:00,960 --> 00:49:02,720
AS BOTH THE DEFENDANT AND THE

1271
00:49:02,720 --> 00:49:05,320
PRIVATE SECTOR, SINCE THIS IS OF

1272
00:49:05,320 --> 00:49:07,160
COURSE A PRIVATE PUBLIC PRIVATE

1273
00:49:07,160 --> 00:49:10,160
PARTNERSHIP, AND THERE ARE

1274
00:49:10,160 --> 00:49:13,720
ASPECTS OF THIS THAT

1275
00:49:13,720 --> 00:49:14,400
WILL--ULTIMATELY ALL OF THE DATA

1276
00:49:14,400 --> 00:49:16,920
WILL COME OUT TO THE PUBLIC

1277
00:49:16,920 --> 00:49:18,320
DOMAIN BUT WE ANTICIPATE HAVING

1278
00:49:18,320 --> 00:49:19,280
ANNUAL MEETINGS AND INCLUDING

1279
00:49:19,280 --> 00:49:20,280
SOME THAT WILL OPEN TO THE

1280
00:49:20,280 --> 00:49:23,000
PUBLIC SO THE PUBLIC AND THE

1281
00:49:23,000 --> 00:49:23,720
COMMUNITY CAN SEE THE PROGRESS

1282
00:49:23,720 --> 00:49:30,240
THAT WE'VE BEEN MAKING. 

1283
00:49:30,240 --> 00:49:30,720
NEXT SLIDE.

1284
00:49:30,720 --> 00:49:32,240
AND THESE ARE SOME OF THE

1285
00:49:32,240 --> 00:49:33,200
SPECIFIC ROLES AGAIN, I WON'T

1286
00:49:33,200 --> 00:49:35,200
DWELL ON THIS FOR THE SAKE OF

1287
00:49:35,200 --> 00:49:35,400
TIME.

1288
00:49:35,400 --> 00:49:37,160
BUT I WOULD ALSO NOTE DOWN

1289
00:49:37,160 --> 00:49:41,440
THERE, WE'VE ALSO INCLUDED IN

1290
00:49:41,440 --> 00:49:43,120
THE BGTC, GROUPS REPRESENTING

1291
00:49:43,120 --> 00:49:44,080
PATIENTS INCLUDING THE NATIONAL

1292
00:49:44,080 --> 00:49:45,240
ORGANIZATION FOR RARE DISEASES,

1293
00:49:45,240 --> 00:49:48,160
AND WE'VE GOT A LOT OF THOUGHT

1294
00:49:48,160 --> 00:49:50,520
TO THE COMMUNICATIONS INVOLVED

1295
00:49:50,520 --> 00:49:52,320
IN THE BGTC SO THAT AS WE DO

1296
00:49:52,320 --> 00:49:53,120
MAKE PROGRESS, WE COMMUNICATE

1297
00:49:53,120 --> 00:49:56,320
THAT PROGRESS IN WAYS THAT MAKE

1298
00:49:56,320 --> 00:49:57,480
SENSE TO THE DIFFERENT

1299
00:49:57,480 --> 00:50:04,200
STAKEHOLDERS WHO WILL BE

1300
00:50:04,200 --> 00:50:04,520
RECEIVING IT.

1301
00:50:04,520 --> 00:50:04,840
NEXT SLIDE.

1302
00:50:04,840 --> 00:50:09,600
SO IN TERMS OF THE TIMELINES,

1303
00:50:09,600 --> 00:50:11,680
WE'RE NOW IN 2022 AS I SAID

1304
00:50:11,680 --> 00:50:13,960
WE'VE GONE PAST, GOTTEN

1305
00:50:13,960 --> 00:50:17,720
APPLICATIONS FOR THE DIFFERENT

1306
00:50:17,720 --> 00:50:19,920
WORK ALREADY REVIEWING THOSE AND

1307
00:50:19,920 --> 00:50:21,520
HOPE TO HAVE PROGRESS IN TERMS

1308
00:50:21,520 --> 00:50:23,240
OF IDENTIFYING DISEASES WE WANT

1309
00:50:23,240 --> 00:50:27,960
TO STUDY, BY THE END OF APRIL AS

1310
00:50:27,960 --> 00:50:29,680
WELL AS MORE PROGRESS ON SOME OF

1311
00:50:29,680 --> 00:50:32,120
THE OTHER INITIATIVES, TO

1312
00:50:32,120 --> 00:50:33,120
ULTIMATELY GET TO THE POINT

1313
00:50:33,120 --> 00:50:34,720
WE'RE STARTING TO SEE RESULTS

1314
00:50:34,720 --> 00:50:36,840
AND OF COURSE, WE WILL BE

1315
00:50:36,840 --> 00:50:38,480
KEEPING THE COMMUNITY UPDATED AS

1316
00:50:38,480 --> 00:50:44,320
WE MOVE THROUGH THIS EFFORT.

1317
00:50:44,320 --> 00:50:46,320
NEXT SLIDE.

1318
00:50:46,320 --> 00:50:48,920
JUST TO KIND COMPARE THE BGTC

1319
00:50:48,920 --> 00:50:51,520
AND PAVE GT BECAUSE THEY SOUND

1320
00:50:51,520 --> 00:50:53,120
SIMILAR, THERE ARE CERTAIN

1321
00:50:53,120 --> 00:50:55,320
SIMILARITIES BUT THERE ARE

1322
00:50:55,320 --> 00:50:57,280
DIFFERENCES IN THE BGTC, WE

1323
00:50:57,280 --> 00:50:59,720
CHOSE 4 DISEASES OF INTEREST AND

1324
00:50:59,720 --> 00:51:03,520
THE NIH CLINICAL CENTER, AND THE

1325
00:51:03,520 --> 00:51:08,600
BGTC AS WE'RE SING PROBABLY 5 OR

1326
00:51:08,600 --> 00:51:11,400
6 OR SO WE WILL HAVE A SINGLE

1327
00:51:11,400 --> 00:51:13,600
MANUFACTURE OF MAKING ALL 4

1328
00:51:13,600 --> 00:51:15,320
VECTORS AND THE BGTC WILL HAVE A

1329
00:51:15,320 --> 00:51:17,760
CONSORTIUM IN BOTH CASES WE PLAN

1330
00:51:17,760 --> 00:51:21,200
TO MAKE THE INFORMATION AS

1331
00:51:21,200 --> 00:51:22,320
PUBLIC ULTIMATELY WITH PAVE GT,

1332
00:51:22,320 --> 00:51:24,200
WE PLAN TO DO THAT A LOT MORE, A

1333
00:51:24,200 --> 00:51:26,640
LOT SOONER AND A LOT MORE

1334
00:51:26,640 --> 00:51:32,080
EXPLICITLY, AND WE HOPE THAT THE

1335
00:51:32,080 --> 00:51:35,680
BGTC WE'LL SEE HOW THE TIMING

1336
00:51:35,680 --> 00:51:36,240
GOES ON THAT.

1337
00:51:36,240 --> 00:51:38,320
AND PAVE GT WILL DO ALL THE WORK

1338
00:51:38,320 --> 00:51:40,960
OF THE NIH CLINICAL CENTER,

1339
00:51:40,960 --> 00:51:42,440
WHEREAS IN THE BGTC WE

1340
00:51:42,440 --> 00:51:44,000
ANTICIPATE DIFFERENT CLINICAL

1341
00:51:44,000 --> 00:51:45,920
SITES WHICH MAY IN FACT INCLUDE

1342
00:51:45,920 --> 00:51:47,320
THE NIH CLINICAL CENTER, AND

1343
00:51:47,320 --> 00:51:51,360
AGAIN THE PAVE GT IS A SINGLE

1344
00:51:51,360 --> 00:51:56,120
SEROTYPE AND BGT IS MULTIPLE

1345
00:51:56,120 --> 00:51:56,400
SEROTYPES.

1346
00:51:56,400 --> 00:51:56,720
NEXT SLIDE.

1347
00:51:56,720 --> 00:51:58,320
AND THEN JUST AT THE LAST FEW

1348
00:51:58,320 --> 00:52:00,200
MINUTES HERE, I WANTED TO

1349
00:52:00,200 --> 00:52:02,440
BRIEFLY MENTION SOME PROGRESS ON

1350
00:52:02,440 --> 00:52:03,840
THE NIH SOMATIC CELL GENOME

1351
00:52:03,840 --> 00:52:05,720
EDITING PROGRAM, THIS IS 1 THAT

1352
00:52:05,720 --> 00:52:09,360
IS FUNDED THROUGH THE NIH COMMON

1353
00:52:09,360 --> 00:52:11,720
FUND THAT NCATS IS INVOLVED IN,

1354
00:52:11,720 --> 00:52:15,760
COORDINATE ALONG THAT WITH

1355
00:52:15,760 --> 00:52:17,720
NINDS, AND MANY OTHER NIH

1356
00:52:17,720 --> 00:52:18,600
INSTITUTES AND CENTERS

1357
00:52:18,600 --> 00:52:19,440
PARTICIPATING AS WELL.

1358
00:52:19,440 --> 00:52:21,320
SO THE PHASE 1 OF THE PROGRAM

1359
00:52:21,320 --> 00:52:23,440
WAS DESIGNED TO LOWER THE

1360
00:52:23,440 --> 00:52:26,000
BARRIERS FOR THE GENOME EDITING

1361
00:52:26,000 --> 00:52:28,640
THERAPIES BY DEVELOPING BETTER

1362
00:52:28,640 --> 00:52:30,640
ANIMAL MODELS FOR TESTING GENOME

1363
00:52:30,640 --> 00:52:34,480
EDITING READS AND DELIVERY

1364
00:52:34,480 --> 00:52:35,080
SYSTEMS.

1365
00:52:35,080 --> 00:52:36,480
BETTER WAYS TO ASSESS THAN

1366
00:52:36,480 --> 00:52:38,360
BIOLODGEICAL EFFECTS OF GENOME

1367
00:52:38,360 --> 00:52:41,520
EDITING A BIG COMPONENT ON

1368
00:52:41,520 --> 00:52:43,280
IMPROVING GENOME EDITING OF THE

1369
00:52:43,280 --> 00:52:43,680
MACHINERY.

1370
00:52:43,680 --> 00:52:45,720
EXPANDING THE HUMAN GENOME

1371
00:52:45,720 --> 00:52:48,040
EDITING REPERER TWOAR AND

1372
00:52:48,040 --> 00:52:50,320
COORDINATING AND DISSEMINATION

1373
00:52:50,320 --> 00:52:52,720
FUNCTION THAT WOULD PUT UP INTO

1374
00:52:52,720 --> 00:52:58,320
A PUBLIC TOOL KIT ALL OF THE

1375
00:52:58,320 --> 00:53:00,080
DISCOVERIES THAT HAVE BEEN MADE

1376
00:53:00,080 --> 00:53:10,360
UNDER THE SCG PHASE 1 PROGRAM. 

1377
00:53:10,360 --> 00:53:10,960
NEXT SLIDE.

1378
00:53:10,960 --> 00:53:12,080
AND IF YOU JUST CLICK THROUGH

1379
00:53:12,080 --> 00:53:14,320
THAT, YOU CAN CLICK IT AGAIN,

1380
00:53:14,320 --> 00:53:17,040
WE'VE HAD MULTIPLE PUBLICATIONS

1381
00:53:17,040 --> 00:53:19,920
THAT WE'VE SUPPORTED THROUGH THE

1382
00:53:19,920 --> 00:53:21,320
SCG PHASE 1, MOST OF THEM

1383
00:53:21,320 --> 00:53:23,720
FOCUSED ON DEVELOPING BETTER

1384
00:53:23,720 --> 00:53:25,160
TOOLS AND INCLUDING THE PRIME

1385
00:53:25,160 --> 00:53:26,920
EDITOR THAT WE PROVIDED SOME OF

1386
00:53:26,920 --> 00:53:30,720
THE FUNDING FOR AND BETTER

1387
00:53:30,720 --> 00:53:37,720
DELIVERY TECHNOLOGIES BUT, NEXT

1388
00:53:37,720 --> 00:53:37,920
SLIDE.

1389
00:53:37,920 --> 00:53:38,760
THERE IS AN OUTPATIENT SETTING

1390
00:53:38,760 --> 00:53:40,440
FOR COMPLON FUND PROGRAMS TO

1391
00:53:40,440 --> 00:53:42,760
HAVE MULTIPLE PHASES, UP TO

1392
00:53:42,760 --> 00:53:44,920
25-YEAR PHASES SO WE HAVE IN

1393
00:53:44,920 --> 00:53:45,680
FACT PROPOSED THE SECOND PHASE

1394
00:53:45,680 --> 00:53:47,440
WHICH WILL BE MORE FOCUSED ON

1395
00:53:47,440 --> 00:53:49,080
GETTING INTO THE CLINIC WHERE

1396
00:53:49,080 --> 00:53:50,920
PHASE 1 WAS REALLY ALL ABOUT

1397
00:53:50,920 --> 00:53:52,000
TECHNOLOGY DEVELOPMENT AND 1 OF

1398
00:53:52,000 --> 00:53:54,320
THE INITIATIVES WE PROPOSED IN

1399
00:53:54,320 --> 00:53:57,320
PHASE 2, AND BETTER DEVELOPMENT

1400
00:53:57,320 --> 00:54:01,640
OF BETTER GENOME EDITING ASSAYS

1401
00:54:01,640 --> 00:54:02,400
THAT WOULD BE ESSENTIALLY

1402
00:54:02,400 --> 00:54:10,440
QUALIFIED OR AT THE LEVEL OF

1403
00:54:10,440 --> 00:54:12,680
PRECISION THAT WOULD BE

1404
00:54:12,680 --> 00:54:13,800
ULTIMATELY REQUIRED FOR

1405
00:54:13,800 --> 00:54:14,680
REGULATORY APPROVAL.

1406
00:54:14,680 --> 00:54:17,000
WE WOULD LIKE TO THINK THAT SOME

1407
00:54:17,000 --> 00:54:18,520
OF THESE WOULD ULTIMATELY BECOME

1408
00:54:18,520 --> 00:54:21,360
PART OF THE STANDARD FDA

1409
00:54:21,360 --> 00:54:22,200
APPROVAL PROCESS ALTHOUGH WE

1410
00:54:22,200 --> 00:54:24,200
CAN'T KNOW THAT BUT THAT'S A

1411
00:54:24,200 --> 00:54:24,520
GOAL.

1412
00:54:24,520 --> 00:54:27,200
SO THESE WILL BE 3 YEAR AWARDS

1413
00:54:27,200 --> 00:54:31,800
TO DEVELOP THESE NEW ASSAYS.

1414
00:54:31,800 --> 00:54:33,040
NEXT SLIDE.

1415
00:54:33,040 --> 00:54:34,920
INITIATIVE 2 WILL BE FOCUS

1416
00:54:34,920 --> 00:54:36,800
SPECIFICALLY ON IND ENABLING

1417
00:54:36,800 --> 00:54:39,240
STUDIES IN DIFFERENT DECS WITH

1418
00:54:39,240 --> 00:54:40,200
DIFFERENT TARGET TISSUES.

1419
00:54:40,200 --> 00:54:42,760
AND THE GOAL OF THESE AWARDS

1420
00:54:42,760 --> 00:54:44,400
WILL BE TO DEVELOP PACKAGES OR

1421
00:54:44,400 --> 00:54:46,320
BRING THINGS ALL THE WAY UP TO

1422
00:54:46,320 --> 00:54:47,840
THE POINT OF GETTING AN IND

1423
00:54:47,840 --> 00:54:48,920
WITHOUT GOING INTO THE CLINICAL

1424
00:54:48,920 --> 00:54:53,560
TRIAL WITH THE HOPE THAT ONCE AN

1425
00:54:53,560 --> 00:54:55,160
IND IS ATTAINED, THAT WILL MAKE

1426
00:54:55,160 --> 00:54:56,720
IT MORE ATTRACTIVE FOR OTHER

1427
00:54:56,720 --> 00:54:59,440
ENTITIES TO PICK UP THE IND AND

1428
00:54:59,440 --> 00:54:59,960
ULTIMATELY SUPPORT THOSE

1429
00:54:59,960 --> 00:55:00,520
CLINICAL TRIALS.

1430
00:55:00,520 --> 00:55:03,480
THESE WILL BE 5 YEAR AWARDS.

1431
00:55:03,480 --> 00:55:07,360
NEXT SLIDE.

1432
00:55:07,360 --> 00:55:09,520
AS YOU TALKED A LOT ABOUT

1433
00:55:09,520 --> 00:55:11,720
THERAPEUTIC PLATFORMS, GENOME

1434
00:55:11,720 --> 00:55:14,360
EDITING IS BY DEFINITION ALMOST

1435
00:55:14,360 --> 00:55:15,880
THERAPEUTIC PLATFORM, SO

1436
00:55:15,880 --> 00:55:18,520
INITIATIVE 3 OF THE COMMON FUND

1437
00:55:18,520 --> 00:55:21,320
THAT WE ENVISION WILL FOCUS

1438
00:55:21,320 --> 00:55:21,920
ACTUALLY ON PLATFORM CLINICAL

1439
00:55:21,920 --> 00:55:25,920
TRIALS AND THE IDEA OF

1440
00:55:25,920 --> 00:55:28,320
BEING--THIS WILL BE A BI PHASIC

1441
00:55:28,320 --> 00:55:29,800
PROGRAM, THE FIRST FACE WILL BE

1442
00:55:29,800 --> 00:55:32,320
TO GET AN IND AND THE SECOND

1443
00:55:32,320 --> 00:55:34,360
PHASE TO CARRY OUT A CLINICAL

1444
00:55:34,360 --> 00:55:35,320
TRIAL WITH THE IMPORTANT

1445
00:55:35,320 --> 00:55:35,960
REQUIREMENT THAT THESE CLINICAL

1446
00:55:35,960 --> 00:55:37,320
TRIALS WILL HAVE TO INVOLVE MORE

1447
00:55:37,320 --> 00:55:38,320
THAN 1 DISEASE, SO THE IDEA

1448
00:55:38,320 --> 00:55:40,680
WOULD BE, YOU WOULD HAVE 1

1449
00:55:40,680 --> 00:55:45,400
EDITOR, ON 1 DELIVERY SYSTEM,

1450
00:55:45,400 --> 00:55:46,400
BUT MORE THAN 1 DISEASE.

1451
00:55:46,400 --> 00:55:48,080
AND TO REALLY SEE IF WE CAN

1452
00:55:48,080 --> 00:55:50,320
ESTABLISH A CLINICAL TRIAL

1453
00:55:50,320 --> 00:55:57,320
PLATFORM FOR GENOME EDITING.

1454
00:55:57,320 --> 00:55:59,440
NEXT SLIDE.

1455
00:55:59,440 --> 00:56:02,360
COORDINATION CENTER THROUGHOUT

1456
00:56:02,360 --> 00:56:05,720
THE SCGE PROGRAM THAT WILL

1457
00:56:05,720 --> 00:56:07,760
SUPPORT SEMIANNUAL MEETINGS BUT

1458
00:56:07,760 --> 00:56:08,960
ALSO IMPORTANTLY DATA AND

1459
00:56:08,960 --> 00:56:10,720
PROTOCOL SHARING AND ULTIMATELY

1460
00:56:10,720 --> 00:56:12,920
ACROSS THE BROADER COMMUNITY SO

1461
00:56:12,920 --> 00:56:13,800
THAT THE RESULTS OF THESE

1462
00:56:13,800 --> 00:56:17,000
EFFORTS CAN BE EXTENDED THROUGH

1463
00:56:17,000 --> 00:56:20,840
THE MUCH BROADER COMMUNITY

1464
00:56:20,840 --> 00:56:21,920
FOCUSED ON DEVELOPING TREATMENTS

1465
00:56:21,920 --> 00:56:23,880
FOR FOR SINGLE GENE DISORDERS,

1466
00:56:23,880 --> 00:56:27,880
BOTH RARE AND COMMON IN FACT.

1467
00:56:27,880 --> 00:56:28,360
úNEXT SLIDE.

1468
00:56:28,360 --> 00:56:30,600
VERY LAST THING I WILL SAY IS I

1469
00:56:30,600 --> 00:56:33,160
POINTED THIS OUT, BECAUSE WE

1470
00:56:33,160 --> 00:56:34,120
ANTICIPATE--I CAN TALK ABOUT

1471
00:56:34,120 --> 00:56:36,120
THIS BECAUSE WE PUBLISHED NOW

1472
00:56:36,120 --> 00:56:37,720
THESE NOTICES OF INTENT TO

1473
00:56:37,720 --> 00:56:39,040
PUBLIC FUNDING OPPORTUNITIES SO

1474
00:56:39,040 --> 00:56:40,400
THESE ARE THESE FORMAL NOTICES

1475
00:56:40,400 --> 00:56:41,360
TO,A LETTER THE COMMUNITY THAT

1476
00:56:41,360 --> 00:56:45,360
WE WILL BE HAVING THESE FUNDING

1477
00:56:45,360 --> 00:56:47,320
OPPORTUNITIES COMING UP AND I

1478
00:56:47,320 --> 00:56:51,000
POINT OUT HERE BECAUSE IN THE

1479
00:56:51,000 --> 00:56:51,720
PAST NIH INTRAMURAL

1480
00:56:51,720 --> 00:56:53,720
INVESTIGATORS HAVE BEEN ABLE TO

1481
00:56:53,720 --> 00:56:55,840
APPLY FOR A COMMON FUND PROGRAMS

1482
00:56:55,840 --> 00:56:57,400
AND WE ANTICIPATE THAT WILL BE

1483
00:56:57,400 --> 00:56:59,920
THE CASE FOR THESE 1S AS WELL,

1484
00:56:59,920 --> 00:57:02,000
SO THESE ARE THE NOTICE OF

1485
00:57:02,000 --> 00:57:03,280
INTENTS TO PUBLISH THAT PEOPLE

1486
00:57:03,280 --> 00:57:06,920
CAN LOOK THROUGH IF THERE'S

1487
00:57:06,920 --> 00:57:07,360
INTEREST.

1488
00:57:07,360 --> 00:57:12,080
AND I THINK THAT'S MY LAST

1489
00:57:12,080 --> 00:57:12,320
SLIDE.

1490
00:57:12,320 --> 00:57:14,960
SO I WILL THANK YOU ALL FOR

1491
00:57:14,960 --> 00:57:19,880
LISTENING ON BEHALF OF DON AND

1492
00:57:19,880 --> 00:57:20,120
MYSELF.

1493
00:57:20,120 --> 00:57:22,480
I THINK WE HAVE QUESTIONS

1494
00:57:22,480 --> 00:57:24,760
SUBMITTED LATER WE CAN TRY TO

1495
00:57:24,760 --> 00:57:25,400
ADDRESS THEM VIA E-MAIL BUT

1496
00:57:25,400 --> 00:57:26,240
THANK YOU VERY MUCH.

1497
00:57:26,240 --> 00:57:28,760
>> YEAH, THANK YOU DR. BROOKS

1498
00:57:28,760 --> 00:57:35,360
AND DR. LO, FOR SHARING REALLY

1499
00:57:35,360 --> 00:57:36,000
THOUGHT-PROVOKING PRESENTATIONS

1500
00:57:36,000 --> 00:57:38,440
ON THE PAVE GT AND THE BGTC

1501
00:57:38,440 --> 00:57:39,920
INITIATIVES, YOU KNOW TO ADVANCE

1502
00:57:39,920 --> 00:57:41,640
THESE TECHNOLOGIES WITH THE GOAL

1503
00:57:41,640 --> 00:57:45,720
OF PURSUING, THE GOAL OF YOU

1504
00:57:45,720 --> 00:57:47,560
KNOW EXPANDING REACHING GENE

1505
00:57:47,560 --> 00:57:49,120
THERAPIES FOR MANY MORE PATIENTS

1506
00:57:49,120 --> 00:57:50,120
WITH RARE DISEASES, YOU ARE

1507
00:57:50,120 --> 00:57:52,880
RIGHT WE HAVE SEVERAL QUESTIONS

1508
00:57:52,880 --> 00:57:53,840
BECAUSE OF YOUR PRESENTATIONS,

1509
00:57:53,840 --> 00:57:56,560
AND I WILL FORD THEM TO YOU SO

1510
00:57:56,560 --> 00:57:57,520
I'M SORRY WE CAN'T ANSWER THEM

1511
00:57:57,520 --> 00:57:59,320
HERE BUT I WANT TO THANK THE

1512
00:57:59,320 --> 00:58:00,880
BOTH OF YOU AND THE NIH

1513
00:58:00,880 --> 00:58:01,600
COMMUNITY FOR JOINING US TODAY

1514
00:58:01,600 --> 00:58:03,000
AND I WISH EVERYONE A GREAT

1515
00:58:03,000 --> 00:58:03,280
AFTERNOON.

1516
00:58:03,280 --> 00:00:00,000
THANK YOU FOR JOINING US.