Cellular & Gene Therapy Products
The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.
Cellular therapy products include cellular immunotherapies, and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.
Although some cellular therapy products have been approved, CBER has not yet approved any human gene therapy product for sale. However, the amount of cellular and gene therapy-related research and development occurring in the United States continues to grow at a fast rate. CBER has received many requests from medical researchers and manufacturers to study cellular and gene therapies and to develop cellular and gene therapy products. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development.
Compliance & Enforcement
Gene Therapy Patient Tracking
Genetic Modification Clinical Research Information Sytem (GeMCRIS) (National Institutes of Health) Gene Therapy Patient Tracking System(PDF - 100KB) NIH and FDA Launch New Human Gene Transfer Research Data System (National Institutes of Health) Office of Biotechnology Activities: Recombinant DNA (National Institutes of Health)
Product Development & Approval
Application of Current Statuatory Authorities to Human Somatic Cell Therapy Products and Gene Therapy Products(PDF - 489KB) Cellular & Gene Therapy Guidances References for the Regulatory Process for the Office of Cellular, Tissue and Gene Therapies Proposed Approach to Regulation of Cellular and Tissue-Based Products(PDF - 433KB) Reinventing the Regulation of Human Tissue