August 19, 2026
The Food and Drug Administration issued two guidance documents addressing the development of cellular and gene therapy (CGT) products, finalizing one and issuing the other in draft.
The final guidance, "Frequently Asked Questions: Developing Potential Cellular and Gene Therapy Products," compiles the agency's answers to questions sponsors commonly raise across the CGT development lifecycle. These include considerations related to regulatory review; chemistry, manufacturing and controls; pharmacology and toxicology; and clinical topics. The guidance also addresses interacting with FDA on investigational new drug applications and meeting requests, product development considerations, the conduct of nonclinical studies and the design of human trials.
The guidance finalizes a draft issued in November 2024. Those interested in commenting on the final guidance may do so online.
The draft guidance, "Potency Assessment of Active Immunotherapy Products," provides recommendations for developing assays to assess the potency of active immunotherapy products (ACTIMPs), which treat a pre-existing disease or condition by inducing, stimulating, or modulating immune effector cells against a disease-associated antigen.
The guidance covers identifying potency-related critical quality attributes and selecting bioassays or physicochemical methods, with tailored advice for specific product types. It is intended to supplement FDA's December 2023 draft guidance, “Potency Assurance for Cellular and Gene Therapy Products.”
Upon publication, the agency will accept comments on the draft guidance until Nov. 18.
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