Gene Therapy Potency
This workstream shares and examines members experiences via benchmarking activities and pools feedback from regulators to define practical considerations for building a potency strategy for in vivo gene therapy products.
Gene therapy potency is difficult to demonstrate to regulators
It can be difficult to demonstrate what regulators expect to see for gene therapy potency and translate these requirements into assays for in vivo GTs. Different products have a different mechanism of action (MoA) and for some AAV-based therapeutics the MoA is not yet elucidated for the protein being augmented.
Potency takes a long time in development. There is confusion regarding how potency is defined and translating that into what it means practically, particularly when differentiating between potency and infectivity. Also MoA and phase-based different potency solutions exist.
Reducing risk and time to market
Using identified scenarios this workstream is aiming to provide phase- and MoA-appropriate guidance for potency strategy, helping to reduce risk, specifically for quality/regulatory non-compliance and regulatory delay. Members are also focusing on reducing time to market (time in development) and technology transfer time to implement into the QC environment.
Gene Therapy Potency deliverables
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