Issue Date: May 5, 2026
Issued by: Center for Biologics Evaluation and Research
Topic(s): Chemistry, Manufacturing, and Controls (CMC), Gene Therapy
Status: Final
Chemistry, Manufacturing, and Controls Flexibilities for Developing Human Cellular and Gene Therapy Products for a Biologics License Application: Guidance for Industry
For a high-level overview of this guidance document, please watch this recorded webinar featuring Kimberly Schultz, PhD (Office of Therapeutic Products, FDA) and LCDR Jessica Dunn, PhD (Office of Compliance and Biologics Quality, FDA). This guidance describes how FDA applies flexibility to the chemistry, manufacturing, and controls (CMC) requirements for human cellular and gene therapy (CGT) products being developed for biologics license applications (BLAs) under Title 21 of the Code of Federal Regulations (CFR) Part 601 (21 CFR 601). Consistent with the statutory and regulatory requirements for biological products, FDA uses a flexible approach to ensuring applicable CMC requirements are met for CGT products. FDA’s flexible approach serves to help expedite development, review, and patient access to safe and effective CGT products to treat serious or life-threatening conditions that represent significant unmet medical needs.FDA has issued several guidances that provide CMC recommendations to sponsors developing CGT products. Sponsors developing CGT products should consider the CMC recommendations in this guidance in addition to those guidances for industry, as applicable. This guidance provides information on when CMC flexibilities may be appropriate for the development of CGT products and does not comprehensively address the CMC information and data necessary to support CGT product licensure under section 351 of the Public Health Service Act (42 U.S.C. 262).
Download the Guidance Document
