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Using a matrix approach to address challenges for potency assay development for in vivo and ex vivo gene therapies.

The gene therapy field is advancing rapidly and though there has been significant progress, there are yet relatively few commercial products approved globally reflecting the infancy of this modality. Some therapies have suffered setbacks due to adverse side effects in patients and limited knowledge about product quality.   With more than 400 gene therapies currently under development, it is more important than ever to improve our...

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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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Current mission of the High-Level Analytics Workstream

The High-Level Analytics Workstream has been running since September 2018, with representatives from 21 member companies. The workstream is now progressing several deliverables: Using initial survey data to elucidate the different approaches being taken to measure potency of cell and gene therapies (CGTs), the Potency sub-team, are actively working on a publication that promotes and discusses the adoption and acceptance of a matrix approach...

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