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Gene Therapy Potency Strategy Roadmap
Navigating the Path to Robust Potency Assessment
Establishing potency assessments that align with regulatory expectations at different development stages remains a significant challenge for in vivo gene therapies. This complexity is further compounded by variations in regulatory requirements across regions, making it difficult to implement a unified global potency strategy.
Additional challenges include misalignment between scientific justification and worldwide regulatory expectations for the TCID50 assay, as well as inconsistent or insufficient guidance on whether representative or surrogate cell lines are appropriate for potency testing. In response to the need for greater industry alignment, we convened subject matter experts to establish standardized potency approaches for recombinant AAV gene therapy products. The resulting publication provides developers with a structured, consensus-driven framework to support potency strategy development. By addressing the disconnect between scientific principles and varying global regulatory requirements, the roadmap offers practical guidance for implementing potency assays throughout development. Central to this guidance are three visual frameworks covering phase-appropriate potency testing for release, mechanism-of-action-based assay selection, and cell line choice.
This roadmap addresses a key scientific and regulatory challenge in the field by providing guidance on demonstrating appropriate potency for in vivo gene therapy products across development stages while accommodating diverse global regulatory frameworks. It serves as a practical resource for both early-career and experienced developers engaged in potency strategy design.
Ulrike Herbrand (Scientific Director of Global In Vitro Bioassays and Head of Bioassay R&D at Charles River) and Mark Jones (Director of Scientific Portfolio Management at Charles River) are key contributors within a team of authors selected by BioPhorum to present this guide that helps navigate the complexities of potency for gene therapy.