FDA issues draft Q&A guidance on cell and gene therapy

The U.S. rolled out draft guidance intended to provide industry with answers to frequently asked questions that arise during the development of cell and gene therapies, with the end goal of facilitating the development of safe, effective and high-quality CGT products.

The FAQs compile common questions directed to the Office of Therapeutic Products (OTP) from various sources, such as FDA interactions with sponsors in development programs, questions received following public presentations by FDA staff or questions received from public stakeholders via email. The questions span multiple disciplines from IND submission and quality to trial design to manufacturing changes.

The 44-page guidance, created as part of FDA’s response to the PDUFA VII commitment to increase efficiency in the development of CGT products, is open for commentary until February 18, 2025.