The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft document entitled "Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing." The draft guidance document provides sponsors engaged in the development of human gene therapy (GT) products incorporating ex-vivo and in vivo genome editing (GE) of human somatic cells (GE products) with FDA's recommendations on the type of prior knowledge that may be scientifically appropriate to leverage to advance product development.
US Federal Register - FDA Guidance Notices · June 03, 2026
Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing; Draft Guidance for Industry
Original title: Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing; Draft Guidance for Industry; Availability
FDA draft guidance on using prior knowledge to advance development of human gene therapy products with ex vivo and in vivo genome editing.
Published by US Federal Register - FDA Guidance Notices. HealthSignal links to the original document; the summary above is extracted from the source text.