Gene Therapy / Cell Therapy / Stem Cells – Regulations for the "New Biologics"

Gene Therapy / Cell Therapy / Stem Cells – Regulations for the "New Biologics" William R. Tolbert, Ph.D. AOAC-SCS Regulatory & Compliance Conference Friday March 30, 2007 – 10:50 AM

Applications of FDA Authority Through Product-Centric Centers

Products Regulated in OCTGT ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Somatic cell therapies Gene therapies ,[object Object],[object Object]

Autologous cells transduced with vector

Xenotransplantation ,[object Object]

Xenotransplantation – Not Feasible

The Pre-2005 Regulatory Environment for Manufacturing ,[object Object],[object Object],[object Object],[object Object],[object Object]

How are Cell-Based Products Different? ,[object Object],[object Object],[object Object],[object Object]

FDA Driving Forces & Challenges for Cell Products ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Cell and Tissue Characteristics ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

FDA Regulation of Cellular and Tissue-based Products ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

New FDA Regulation ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Regulatory Requirements for HCT/P’s

“ 361” HCT/Ps (Human cells, tissue and cellular and tissue based products) ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

21 CFR 1271.10 (“Below the line”) ,[object Object],[object Object],[object Object]

21 CFR 1271.10 (“Below the line”) ,[object Object]

21 CFR 1271.10 (“Below the line”) ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Minimal Manipulation ,[object Object],[object Object],[object Object],[object Object]

Homologous Use ,[object Object]

“ 351” HCT/Ps (Human cells, tissue and cellular and tissue based products) ,[object Object],[object Object],[object Object],[object Object],[object Object]

21 CFR Part 1271 Establishment Registration and Listing ,[object Object],[object Object],[object Object]

21 CFR Part 1271 Donor Eligibility ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

21 CFR Part 1271 Good Tissue Practices (cGTPs) ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

cGTP Basics ,[object Object],[object Object],[object Object],[object Object]

21 CFR Part 1271 Inspection and Enforcement ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

CBER’s Bioresearch Monitoring Branch ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

CBER is assigning more inspections of ongoing studies under IND/IDE ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

FY 2006 HCT/P Inspections 354 Total 44.1 234 All other HCT/Ps 42.8 36 Cord blood stem cells Peripheral blood stem cells 45.7 87 Reproductive tissues Hours/ Inspection Number of Inspections Type of HCT/P establishment

Human Stem Cells ,[object Object],[object Object],[object Object]

Bone marrow stroma/Mesenchymal stem cells Facilitate hematopoietic reconstitution

Human Embryonic Stem Cell Lines ,[object Object],[object Object],[object Object],[object Object],[object Object]

Human Embryonic Stem Cell Lines ,[object Object],[object Object],[object Object],[object Object]

Cell Therapy Product Characterization ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Product Safety and Efficacy ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Cell Therapy Preclinical Assessment: Activity and Safety ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Cell Therapy Preclinical Assessment: Animal Models ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Cell Therapies: Characterization ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Manufacturing Facilities ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Open vs. Closed Manufacturing Systems ,[object Object],[object Object]

[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Paradigm for Delivery of Patient-Specific Products ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

WR Tolbert & Associates Consultants to the Biopharmaceutical Industry 11483 Cypress Woods Drive San Diego, CA 92131-3535 858-693-8163 wrt@wrtolbert.com / www.wrtolbert.com

Gene Therapy / Cell Therapy / Stem Cells – Regulations for the "New Biologics"

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