What is Gene Therapy?
Human 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 1.
Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms:
Introducing a new or modified gene into the body to help treat a disease
Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases.
There are a variety of types of gene therapy products, including:
Bacterial vectors: Bacteria can be modified to prevent them from causing infectious disease and then used as vectors (vehicles) to carry therapeutic genes into human tissues.
Human gene editing technology: The goals of gene editing are to disrupt harmful genes or to repair mutated genes.
Patient-derived cellular gene therapy products: Cells are removed from the patient, genetically modified (often using a viral vector) and then returned to the patient.
Gene therapy products are biological products regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies in humans require the submission of an investigational new drug application (IND) prior to initiating clinical studies in the United States. Marketing a gene therapy product requires submission and approval of a biologics license application (BLA).
1 Federal Register Volume 66, Number 12. Thursday, January 18, 2001