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 understanding of the critical quality attributes essential in ensuring consistent product quality prior to product release and administration to patients.
Potency is one of the most critical quality attributes for GT development and manufacturing and is generally reflective of the product’s therapeutic activity. Potency assays are therefore based on the product’s mechanism of action (MoA). For GTs, understanding the MoA is complex where more than one function is critical to the therapeutic activity; targeting a single attribute for the potency assay is not sufficient.
BioPhorum’s new paper, Challenges for potency assay development for in vivo and ex vivo gene therapies and the matrix approach brings an awareness of the complexity and challenges associated with developing potency assays that adequately reflect the MoA of GTs and proposes a strategy, the matrix approach, to incrementally implement potency tests to meet early development needs and readiness for licensure application. To help the reader, the paper provides practical examples of a potency assay matrix that reflects the different attributes of in vivo and ex vivo GTs.