Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled ``Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry.'' The draft guidance provides recommendations to stakeholders developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. The draft guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products.