Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use 1.

Gene therapy is a technique that modifies a persons genes to treat or cure disease. Gene therapies can work by several mechanisms:

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:

Gene therapy products are biological products regulated by the FDAs 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).

Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry, January 2020

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What is Gene Therapy? | FDA - U.S. Food and Drug Administration