REGULATORY UPDATE: CBER Seeks Feedback on Opportunities to Advance CGT Development

September 27, 2023

The Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) is requesting information from stakeholders about scientific challenges and opportunities to advance the development of individualized cellular and gene therapies (CGTs). The feedback will inform potential planning of future town halls, workshops or discussion papers that could ultimately facilitate the development of additional regulatory science tools, standards or guidance.

The Sept. 21 Federal Register notice (FRN) outlines the following four topic areas on which the agency is seeking feedback, along with sample questions:

  • Manufacturing (e.g., product quality).
  • Nonclinical development (e.g., toxicology, proof of concept and biodistribution).
  • Clinical development (e.g., assessing safety and efficacy).
  • Additional questions to consider (e.g., additional scientific needs, best practices and opportunities for collaborations).

FDA is accepting written or electronic comments on from the public through 11:59 p.m. ET on Monday, Nov. 20. Instructions are available in the FRN.