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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