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

Gene therapies (GTs) are a complex class of biopharmaceuticals that offer the prospect of curative treatments. Critical quality attributes such as potency, should be identified and controlled, to ensure desired product quality, and thus safety and efficacy. Potency is one of the most critical quality attributes for GT development and manufacturing that is related to the therapeutic product’s MoA, however, developing and validating appropriate potency assays that reflect the mechanism(s) of action acceptable to regulators, is a process fraught with challenges.

This paper highlights some of the challenges to develop potency assays for GTs and promotes a potential solution; a ‘matrix’ approach that involves capturing two or more mechanisms of action and compiling the data to draw a collective conclusion on a product’s potency. Such an approach includes the combination of complementary potency assays based on product specific attributes covering biological or non-biological/orthogonal methods. This paper 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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Challenges for potency assay development for gene therapies and the matrix approach September 2021(1).pdf
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