The US Food and Drug Administration (FDA) has announced increased flexibility for cell and gene therapy (CGT) manufacturers, loosening quality requirements in an effort to expedite patient access.
The agency released its new requirements on 11 January 2026, described by FDA commissioner Marty Makary as “common-sense reforms that will address the unique characteristics of CGTs and foster innovation”. Looser and alterable quality requirements for CGT manufacturing may now be allowed in certain cases, both during clinical trials and post-marketing.
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Requirements have been eased in three areas. First, manufacturers may be subject to looser quality controls during later efficacy-focused trials, more typical of earlier safety-focused studies and may implement minor changes to production between Phase I and Phase II trials if supported by sufficient data.
Second, more flexible post-approval manufacturing controls will be considered for Biologics License Applications (BLAs) for CGTs. Quality specifications may also be revised after approval. Finally, the FDA has dropped the requirement for three rounds of Process Performance Qualification (PPQ) before commercial production in certain cases, while also allowing the details of PPQ to be designed batch-by-batch.
According to Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research (CBER), these changes reflect “an explosive growth of CGT submissions, many of which target serious or life-threatening conditions with an unmet medical need”. This is despite several developers abandoning CGT projects in recent months; Belgian biotech Galapagos and Japanese pharma Takeda both dropped their cell therapy efforts in late 2025.
New requirements support the ‘plausible mechanism pathway’ envisioned by Makary and Prasad in an article published in the New England Journal of Medicine in November 2025. This outlined their desire to accelerate approvals for new, personalised therapies in cases where well-characterised, underlying abnormalities could be addressed amid unmet need, provided targets could be confirmed through modelling and improved clinical outcomes could be established.
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By GlobalDataThe pathway was inspired by the case of Baby KJ, a newborn with carbamoyl phosphate synthase 1 (CPS1) deficiency, a rare disorder disabling protein digestion. In May 2025, Baby KJ’s care team manufactured a custom CRISPR treatment to repair the underlying mutation in just one week, making him the world’s first to receive a bespoke CRISPR therapy. Health and Human Services secretary Robert F Kennedy (RFK) Jr held a roundtable of CGT experts on 5 June, where he committed to fast-track approvals for rare disease treatments.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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