Interpreting the new FDA draft potency guidance: an RNA cell therapy perspective

The FDA’s new draft guidance on Potency Assurance for Cellular and Gene Therapy Products offers a structured framework for developing a robust potency assurance plan to ensure that therapies consistently achieve their intended biological effects. As with all guidance documents, its effectiveness will depend, at least in part, on how it is interpreted and applied—an undertaking with unique challenges in the complex and diverse cell and gene therapy sector. This article examines the application of the new draft guidance, exploring how its key principles can be practically implemented using a novel gene-modified cell therapy as a case study.