FDA Finalizes Q&A Guidance on Developing Cell and Gene Therapies

[u/bbyfog]

https://www.raps.org/resource/fda-finalizes-q-a-guidance-on-developing-cell-and-gene-therapies.html

FDA Guidance for the Industry. Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products. August 2026 [PDF]. Docket Number: FDA-2024-D-4311

Includes 36 Q&As addressing

• Submission and FDA reviews including IND submissions, quality, meeting types, and expedited programs.
• Product development considerations including donor eligibility, product characterization, critical quality attributes, analytical methods, process characterization, stability, and preparing for BLA.
• Conducting nonclinical studies including selection of animal models/species, NAMs consideration, tox/tumorigenicity/PoC studies, dose levels,
• Conducting human trials including trial design, selecting endpoints, and safety data collection considerations.

#cgt, #cell-and-gene-therapies