REGULATORY UPDATE: FDA Draft Guidance Addresses Gene Therapy Product Development

March 16, 2022

The Food and Drug Administration issued a new draft guidance on Wednesday for sponsors of gene therapy products that incorporate genome editing (GE) of human somatic cells. The guidance provides recommendations regarding the information that sponsors should include in an Investigational New Drug (IND) application to assess the safety and quality of the investigational GE product, as required in Title 21 of the Code of Federal Regulations 312.23 (21 CFR 312.23). This includes information on product design, product manufacturing, product testing, preclinical safety assessment and clinical trial design.

AABB is evaluating the draft guidance and soliciting comments for a submission and, to ensure a timely process, encourages all interested individuals to submit comments by May 13 to As noted in the Federal Register notice, interested parties may submit electronic or written comments on the draft guidance directly to FDA by June 14.