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Department of Health and Human Services Public Health Service Food and Drug Administration 1401 Rockville Pike Rockville, MD 20852-1448 July 6, 2001 Dear Sponsor / Researcher: We want to advise you that the Food and Drug Administration (FDA) has jurisdiction over human cells used in therapy involving the transfer of genetic material by means other than the union of gamete nuclei. Examples of such genetic material include, but are not limited to: cell nuclei (e.g., for cloning), oocyte nuclei, ooplasm, which contains mitochondrial genetic material, and genetic material contained in a genetic vector, transferred into gametes or other cells. The use of such genetically manipulated cells (and/or their derivatives) in humans constitutes a clinical investigation and requires submission of an Investigational New Drug application (IND) to FDA. We wish to inform you of the FDA regulatory process governing clinical investigations, which includes requirements applicable to manufacturing processes, the study of the safety and efficacy of such cells, and the protection of human participants in such studies. We can advise you whether or not your activities require submission of an IND. If what you are doing or plan to do does require an IND, we would be pleased to provide you with information and guidance regarding filing such an application. FDA's regulations on investigational new drugs, including those for the submission and review of an IND are described in Title 21 of the Code of Federal Regulations (CFR), Parts 50, 56, and 312. The agency has provided notice of the applicability of these requirements to cellular and tissue-based products in many public forums and in various published documents available at http://www.fda.gov/cber/, including the following: A Federal Register (FR) notice describing FDA's authority over cell and gene therapy products ("Application of Current Statutory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products," October 14, 1993, 58 FR 53248). A comprehensive regulatory program for the regulation of human cellular and tissue-based products, based on a tiered, risk-based assessment ("A Proposed Approach to the Regulation of Cellular and Tissue-Based Products," (PDF), (Text), March 4, 1997, 62 FR 9721). A final rule that establishes the criteria for regulation of human cells, tissues, and cellular and tissue based products (HCT/Ps), including reproductive cells and tissues, published on January 19, 2001 (66 FR 5447) (PDF), (Text). This rule also describes which HCT/Ps will be regulated solely under 21 CFR Part 1271, the regulations to prevent communicable disease transmission authorized by section 361 of the Public Health Service Act. HCT/Ps that do not meet the criteria under Part 1271 will be regulated as biological products, drugs, or devices under section 351 of the Public Health Service Act and/or the Food Drug and Cosmetic Act. "Suitability Determination for Donors of Human Cellular and Tissue-Based Products; Proposed Rule" (PDF), (Text). September 30, 1999 (64 FR 52696). "Current Good Tissue Practice for Manufacture of Human Cellular and Tissue-Based Products; Inspection and Enforcement; Proposed Rule" January 8, 2001 (66 FR 1508). If you are unable to access the internet to obtain information on submitting an IND to the FDA, please call or write and we'll supply you with the needed information: Center for Biologics Evaluation and Research Office of Communication, Training & Manufacturers Assistance Manufacturers Assistance and Technical Training Branch 1401 Rockville Pike, HFM-44 Rockville, MD 20852-1448 800-835-4709 or 301-827-1800 matt@cber.fda.gov The specific information required in an IND will depend upon the cells under investigation and on the phase of study. For assistance in determining whether you need to file an IND submission and in preparation of a submission, please contact Wendy Aaronson, Application Administration Branch Chief, Division of Application Review and Policy, at 301-827-5101. Sincerely, --- signature --- Kathryn C. Zoon, Ph.D. Director Center for Biologics Evaluation and Research Food and Drug Administration

Updated May 13, 2002

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