Federal Register #FDA-2026-D-1257

FDA Draft Guidance: Leveraging Prior Knowledge for Human Gene Therapy Products with Genome Editing

The Food and Drug Administration (FDA), part of the Department of Health and Human Services, has released a draft guidance for sponsors developing human gene therapy products that incorporate genome editing. - Government Buyer: - Food and Drug Administration (FDA) - Department of Health and Human Services - Purpose: - Guidance for sponsors developing human gene therapy (GT) products using ex-vivo and in vivo genome editing (GE) of human somatic cells - Recommendations on leveraging prior knowledge (chemistry, manufacturing, controls, nonclinical, and clinical data) to support product development - Aims to increase review efficiency and accelerate development, especially for rare disease applications - Notable Requirements: - Applies to sponsors of human gene therapy products with genome editing components - Focuses on scientific and regulatory guidance, not procurement of products or services - FDA is seeking public comments on the draft guidance - OEMs and Vendors: - No specific OEMs or vendors are mentioned, as this is a regulatory guidance document, not a product or service procurement - Locations: - FDA headquarters and public comment submission address in Rockville, MD

Description

The Food and Drug Administration (FDA) is announcing the availability of a draft guidance document titled "Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing." This draft guidance provides recommendations to sponsors developing human gene therapy products that incorporate ex-vivo and in vivo genome editing of human somatic cells. It outlines the types of prior knowledge that may be scientifically appropriate to leverage to advance product development, including chemistry, manufacturing, controls, nonclinical, and clinical prior knowledge. The guidance aims to increase review efficiency and accelerate product development, particularly for genome editing products intended to treat rare diseases. Comments on the draft guidance are due by September 1, 2026.

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