On August 20, 2026, the FDA published guidance intended to provide answers to frequently asked questions about the development of cellular and gene therapy (“CGT”) products to help facilitate the development of safe, effective, and high-quality CGT products. The FDA’s guidance is in response to the Prescription Drug Use Fee Act VII commitment to increase the efficient development of CGT products.
The frequently asked questions are grouped into four main categories: (1) submissions and FDA review; (2) product development considerations; (3) conducting nonclinical studies; and (4) conducting human trials.
The Submissions and FDA Review section answers frequently asked questions related to Investigational New Drug (“IND”) applications, including what must be included in an original IND submission and the general process for evaluating original INDs for CGT investigational products.
The Product Development Considerations section covers, among other things, donor eligibility, product characterization testing and release testing, analytical methods, manufacturing changes, the needed stability information to support first-in human studies, and process characterization/validation.
The Conducting Nonclinical Studies section addresses frequently asked questions about the selection of animal models, product selection, tumorigenicity, proof-of-concept studies, toxicity, whether sponsors can submit INDs without nonclinical toxicology studies, design of cell distribution and biodistribution studies, and recommended methods for dose level extrapolation from animals to humans.
Lastly, the Conducting Human Trials section answers frequently asked questions such as important considerations when designing clinical trials, considerations when using a surrogate endpoint as a primary outcome measure for a later phase clinical trial, and what sponsors should consider for short- and long-term safety monitoring in trials of investigational CGT products.
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