This member document summarizes consolidated feedback and recommendations from a collaboration of BioPhorum members on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial consultation.
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This paper highlights some of the challenges to develop potency assays for gene therapies and promotes a potential solution. It seeks to establish Industry alignment on the benefits of a matrix approach and provides high level guidance on how to adopt this strategy towards potency assay development and validation with examples for in vivo and ex vivo GT processes.