The US Food and Drug Administration (FDA) has issued draft guidance intended to help developers of cell and gene therapies make greater use of existing scientific and regulatory knowledge when advancing new products. The guidance, released June 2, describes how sponsors may leverage publicly available information and established platform knowledge—including chemistry, manufacturing and controls (CMC) data, nonclinical findings, and clinical evidence—to support regulatory submissions for human gene therapy products that use genome editing in somatic cells.

According to the agency, the approach is intended to reduce duplicative testing and streamline development programs while maintaining existing standards for safety and effectiveness. FDA officials said the framework could be particularly relevant for patients with rare and life-threatening diseases who have limited treatment options.

The draft guidance complements several recent FDA initiatives in genome editing and gene therapy, including the agency’s Plausible Mechanism Framework and a separate draft guidance on the use of next-generation sequencing to assess off-target editing risks. The agency emphasized that sponsors must provide a scientific rationale demonstrating that leveraged data are applicable to their specific product.

The draft guidance is available on the agency’s website for review and public comment. Comments must be submitted within 90 days following publication in the Federal Register.