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U.S. Department of Health and Human Services

Vaccines, Blood & Biologics

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Letter to Sponsors / Researchers - Human Cells Used in Therapy Involving the Transfer of Genetic Material By Means Other Than the Union of Gamete Nuclei

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:

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

 

Contact FDA

(800) 835-4709
(240) 402-8010
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Food and Drug Administration

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Building 71 Room 3103

Silver Spring, MD 20993-0002