General considerations

In the USA the authority to regulate gene therapy products rests with the Center for Biologies Evaluation and Research (CBER) in the Food and Drug Administration (FDA). At present, all gene therapy products are at the investigational stage and are regulated under the standards for Investigational New Drugs (INDs) in 21 Code of Federal Regulations (CFR) part 312 (Taylor, 1993). Certain existing guidelines for the regulation of biologic therapeutics (21 CFR parts 210, 211, and 600) apply to the production of gene therapy products. Specifically, since biologicals cannot be completely defined, quality control on the starting materials and production process is equally as important as on the finished product to avoid the introduction of harmful contaminants. To outline the requirements for production of gene therapy products and to address issues that are more specific to the concerns unique to gene therapy approaches (e.g., genetic consequences), CBER has issued a series of ‘Points to Consider’ (PTC) documents to aid investigators with IND submissions.

Points to consider in human somatic cell therapy and gene therapy

At the time this document was drafted (Center for Biologies Evaluation and Research Staff, 1991) the majority of the protocols being submitted were ex-vivo cell therapies or cell-marking studies, thus its emphasis is on characterization of modified cell populations.