ARM-FDA Roundtable on Potency Assays White Paper

Addressing potency-assay related development delays for cell and gene therapies: Results of a scientific exchange between FDA and developers

Potency assays present a significant challenge for developers and regulators of gene and cell therapy products. Regulators require developers to measure the potency of all biologics, including gene and cell therapies to ensure that a consistent product is delivered to all patients. As a new and complex field of therapeutics, cell and gene therapies require a bespoke and multi-faceted approach to demonstrating potency. For developers, this represents a significant investment with uncertain returns, as achieving regulatory acceptance of a developer’s approach to demonstrating potency has often led to delays1,2 . On October 19th, an all-day meeting of regulators, developers, and other concerned parties was held to address the challenge of potency assay development and validation for cell and gene therapies (see the Appendix for a full list of attendees).

Read and download the full paper.