FDA Releases Draft Guidance on Use of Prior Knowledge in Gene Therapy Development

The Food and Drug Administration issued a draft guidance on Tuesday describing how sponsors developing human gene therapy products that incorporate genome editing can leverage existing scientific and regulatory knowledge to support product development and regulatory submissions. The guidance is intended to help accelerate the development of cell and gene therapies, particularly for patients with rare and life-threatening diseases.

The document outlines how developers may use publicly available information and established platform knowledge, including chemistry, manufacturing and controls (CMC) data, nonclinical findings and clinical information, to reduce unnecessary duplication of research. The agency still expects sponsors to provide a scientific rationale demonstrating the applicability of leveraged data to their specific product.

The draft guidance is available for public comment until Sept. 1. Submission instructions are available in the Federal Register notice.