1. Office of Cellular Tissue and Gene Therapies Celia M.Witten, Ph.D., M.D. Director, Office of Cellular, Tissue, and Gene Therapies Cellular, Tissue, and Gene Therapies Advisory Committee Meeting February 10, 2006

2. OCTGT Mission Facilitate development, approval, and access to safe and effective medical products

3. Outline Science in Research and Review Products Regulated Office Overview

4. Science in Research and Review: Critical Path Initiative Bring scientific advances to medical product development process (simulation models, validated biomarkers, new clinical trial designs) Stimulate development of applicable research programs in critical path scientific areas, aim to develop techniques that address challenges encountered during product development Regulatory guidance/practice and standards to reflect best available science, integrate FDA involvement

5. Patients Research Community FDA PublicSponsors Practitioners/Societies Payors

6. OCTGT Regulation Cellular therapies Tumor vaccines Gene therapies Tissue and tissue based products Xenotransplantation products Combination products Devices used for cells/tissues Anti-idiotype antibodies

7. Office of Cellular, Tissue, and Gene Therapies Celia M.Witten, Ph.D, M.D. Office Director Eda Bloom, Ph.D. Acting Deputy Director Suzanne Epstein, Ph.D., Associate Director for Research Richard McFarland, M.D., Ph.D. Acting Associate Director for Policy Division of Cellular and Gene Therapies Raj Puri, Ph.D., M.D., Director Stephanie Simek, Ph.D., Deputy Director Division of Human Tissue Products Ruth Solomon, M.D., Director Division of Clinical Evaluation and Pharmacology/Toxicology Ashok Batra, M.D.

8. DCGT Reorganization Consolidate research-reviewer PIs with similar background Branch names reflect regulatory responsibilities Critical mass and efficient operations

9. Division of Cellular and Gene Therapies Raj Puri, Ph.D., M.D., Division Director Stephanie Simek, Ph.D., Deputy Director Cellular and Tissue Therapy Branch Steve Bauer, Ph.D., Acting Chief Tumor Vaccines and Biotechnology Branch Raj Puri, Ph.D., M.D., Acting Chief Cell Therapies Branch Kimberly Benton, Ph.D., Chief Gene Therapies Branch Maritza McIntyre, Ph.D., Chief Gene Transfer and Immunogenicity Branch Eda Bloom, Ph.D., Chief New Structure