Gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use. Gene therapies offer the potential to treat diseases or conditions for which no or few treatments exist. They are being studied to treat cancer as well as genetic, infectious, and other diseases. FDA considers any use of CRISPR/Cas9 gene editing in humans to be gene therapy.
Gene therapy products are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies of gene therapy in humans require the submission of an investigational new drug application (IND) prior to their initiation in the United States, and marketing of a gene therapy product requires submission and approval of a biologics license application (BLA). Clinical trials of gene therapies can be found at www.clinicaltrials.gov. FDA has also approved certain gene therapy products.
FDA is aware that gene therapy products intended for self-administration and “do it yourself” kits to produce gene therapies for self-administration are being made available to the public. The sale of these products is against the law. FDA is concerned about the safety risks involved.
Consumers are cautioned to make sure that any gene therapy they are considering has either been approved by FDA or is being studied under appropriate regulatory oversight.