Regulatory Considerations for Genetically Manipulated Cell-Based Products

Abstract

Gene and cell therapy products are amongst the most complex of products currently under regulatory oversight. They can be administered by vectors introduced directly into the body or can be developed by changing the constitution of cells removed from allogeneic or autologous donors. GCT products are defined by FDA's Office of Cellular, Tissue and Gene Therapies (OCTGT) as both human gene therapy and cellular therapy products. As drugs, they fall under the purview of the Food, Drug and Cosmetic Act because they mitigate, treat and prevent disease, but as biologics they also fall under the Public Health Service Act, Sections 351 and 361 specifically, because they can convey infectious disease. This overview describes some of the special regulatory considerations that are associated with gene therapy products, with an emphasis on the regulatory concerns related to their production and testing.