The State of Gene Therapies: The FDA Perspective

Clinical development of gene therapy began over 20 years ago. Currently there are more than 320 ongoing gene therapy clinical trials that are regulated by the Office of Cellular, Tissue, and Gene Therapies (OCTGT) in the Center for Biologics Evaluation and Research of the US Food and Drug Administration (FDA). The FDA's mission is to advance the public health by helping to speed innovations that make medicines safer and more effective. The diverse gene therapy products in clinical development for a wide range of disorders offer an opportunity for the FDA to fulfill that mission.