Disciplined CMC And Rigorous Trial Design Define Credibility In Accelerated CGT Development

In this segment of Cell & Gene Live, “2026 Regulatory Roadmap,” Cell & Gene’s Chief Editor, Erin Harris, Wilson Bryan, M.D., former director of FDA’s Office of Tissues and Advanced Therapies (OTAT) and Daniela Drago, Ph.D., RAC, Partner at NDA Partners emphasized that credible cell and gene therapy programs succeed by integrating CMC as a core, well-controlled part of development, with early but flexible frameworks, strong analytics, clear comparability plans, and potency strategies tied directly to clinical outcomes.

On the clinical side, regulators remain open to single-arm and innovative trial designs in rare diseases, but only when supported by rigorous methodology, bias-resistant endpoints, large effect sizes, and transparent justification rather than reliance on flexibility alone.