Regulatory Strategy

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Feedback on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial

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document summarizes consolidated feedback and recommendations from a collaboration of BioPhorum members on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial consultation.

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Challenges for potency assay development for in vivo and ex vivo gene therapies and the matrix approach

Challenges for potency assay development for in vivo and ex vivo gene therapies and the matrix approach

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.

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