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Overview: Division of Cellular and Gene Therapies, Office of Cellular,
Tissue and Gene Therapies
Raj K. Puri, M.D., Ph.D.
Director, DCGT
Office of Cellular, Tissue and Gene Therapies
FDA, CBER
CTTB Site Visit Report to CTGTAC February 10, 2012
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OutlineOrganization Mission and ActivitiesRegulatory Scientist and Researcher
Reviewer Model DCGT Research Projects
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CBER Office of Cellular, Tissue, and Gene Therapies (OCTGT)
Office of the DirectorCelia M.Witten, Ph.D., M.D., Director
Stephanie Simek, Ph.D. Deputy DirectorSuzanne Epstein, Ph.D. Associate Director of Research
Richard McFarland, M.D., Ph.D., Associate Director of Policy
Division of Cellular and Gene TherapiesRaj Puri, M.D., Ph.D., Director
Kimberly Benton, Ph.D., Deputy Director
Division of Human TissuesEllen Lazarus, M.D., Director
Division of Clinical Evaluation and Pharmacology/ToxicologyWilson Bryan, M.D., Director
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Division of Cellular and Gene Therapies (DCGT)Raj Puri, M.D., Ph.D., Division Director
Kimberly Benton, Ph.D., Deputy Director
Cellular and Tissue Therapy Branch
Steven Bauer, Ph.D., Chief
Tumor Vaccines and Biotechnology Branch
Raj Puri, M.D., Ph.D. Chief
Cell Therapies BranchKeith Wonnacott, Ph.D., Chief
Gene Therapies BranchDaniel Takefman, Ph.D., Chief
Gene Transfer and Immunogenicity Branch
Andrew Byrnes, Ph.D., Chief
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OCTGT Regulated Products
Cellular therapies Tumor vaccines and immunotherapyGene therapiesTissue and tissue based productsXenotransplantation productsCombination products Devices used for cells/tissuesDonor screening tests (for use
with cadaveric blood samples)
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MissionEvaluate investigational new drug, device, biological
license, and pre-market applications for OCTGT products
Protect patients from infectious disease transmission from tissue transplantation.
Strongly committed to partnership between FDA, government agencies, industry, patient advocates, scientists, and the public to promote and develop new therapies for the 21st Century, while protecting human subjects and maximizing biological product safety
Plan and conduct FDA’s Mission relevant research to support development of Cutting edge medical products
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OCTGT regulatory portfolio
Over 1260 active INDs and IDEs, over 5000 INDamendments in 2011, plus consult reviews
Four licensed products, a growing number of IND products in advanced development
Devices: 510ks, PMAs, HDEs Tissue regulations Pre-INDs, pre-pre-IND advice Policy and guidance, advisory committee
meetings Enforcement actions, international activities
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DCGT ActivitiesEnsure the safety of cellular, gene therapy and other products through:
Development and implementation of a comprehensive risk-based regulatory framework
Evaluation of new technologies for product characterization and rapid assessment of product safety
Development of FDA Policies and Guidances for the regulation of cellular and gene therapy and other products
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DCGT Activities continued..
Inspections:of manufacturing facilities, regulatory site visits
Consultation and Education:Provide scientific and technical advice to other CBER
Offices, FDA Centers, and Government AgenciesInformation sharing and discussion with sponsors
Counterterrorism:COOP Coordination and Laboratory Red Alert Plan Participation in FDA’s CT exercise/simulations
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DCGT Activities continued..
Community Outreach: (seminars, panel discussions, round table, workshops)Cell Therapy including stem cell Tumor Vaccines and ImmunotherapyGene Therapy Tissue Engineering Xenotransplantation
Round Table (CIRM, JDRF, ARM)OCTGT Learn Webinar Series
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DCGT Activities continued..Partnerships:Development of Retrovirus and Adenovirus
reference materialASTM representation and STR profiling for cell line
authenticationWorkshops in collaboration with NIH “Pluripotent
Stem Cells in Translation: Early Decisions” (March 21-22, 2011)
MOU with NIH NINDS and NHLBI for sharing of information and expertise; collaboration with CRM
ERCC and Fluorescence standards for microarray and flow technologies
Inter Agency Oncology Task Force between NCI and FDA for joint fellowship training program
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Current DCGT Research AreasVirology
Retroviruses, adenovirusImmunology
Immune responses to viral and plasmid vectors, autoimmunity and immune regulation
Cell and developmental biologyControl of differentiation in animal models: cell fate and survival, stem cell biology
Cancer biologyMolecular biomarkers, cancer vaccines, animal
modelsBiotechnology
Genomics, flow cytometry, proteomics, transgenicsMicrobiology of tissue safety
Pyrosequencing and whole genome sequencing
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Researcher Reviewer Model Cellular, tissue engineering, and gene therapies evolve
rapidly and continually present new regulatory challenges
These novel products raise extraordinarily complex issues
DCGT seeks to foster a cadre of Researcher Reviewer scientists who : perform regulatory review and identify Critical Path
research needs to enhance and promote product development and patient safety
perform research in key areas to support the FDA mission and help sponsors solve product development problems to advance cellular, gene therapy and other products to the market place
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Types of Researcher Reviewers
Principle investigators (PIs) – tenured or tenure track researcher reviewers
Staff Scientists – tenured researcher reviewers supporting PIs program: do both review and research
Technicians: do primarily research, some do limited review work
Staff Fellows: do both review and research work Postdoctoral fellows funded as ORISE: do primarily
research
Note: Resources are provided to PIs
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Responsibilities of PIsProduct review INDs, IDEs, PMA, 510k, HDEs, licenses, master files,
inspections - regulatory mentoring by branch chiefs
Policy development working groups, guidance development, advisory
committeesOutreach
presubmittal advice, scientific and regulatory talks, refereeing and editing for journals, chairing sessions at scientific conferences, scientific collaborations relevant to the regulatory science
Research lab management, training/mentoring/supervising,
publishing papers, grant writing, leveraging/collaboration
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Research Assessment/Management
Site visit and CBER Advisory Committee recommendations
Promotion and Conversion Evaluation (PCE) Committee review
Regulatory workload and qualityPublications (including guidance documents,
research articles and regulatory articles) Success in securing external funding
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Thank you