Abstract
The development of any medicinal product requires thorough characterization and quality control to assure drug product potency. Adeno-associated virus (AAV)-based gene therapy products (GTPs), a relatively new therapy with limited examples of licensed products, pose a distinctive challenge for measuring the product potency. Unlike conventional drugs, potency for gene therapeutics is a conglomerate of measures of multiple steps that include infectivity of the AAV capsid (vehicle carrying the therapeutic gene), therapeutic gene expression, its protein expression, and ultimately protein function. In this chapter, the authors wanted to accentuate the importance of not only the matrix approach for developing assays to measure potency pertinent to AAV-based gene therapy but also the connection between manufacturing process and product potency. Finally, the challenges and the road ahead for potency assays for AAV-based GTPs are delineated.
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Peters, E.E., Pach, R., Datta, S. (2023). Phase-Appropriate Potency Assay Development for AAV-Based Gene Therapy Products. In: Gautam, S., Chiramel, A.I., Pach, R. (eds) Bioprocess and Analytics Development for Virus-based Advanced Therapeutics and Medicinal Products (ATMPs). Springer, Cham. https://doi.org/10.1007/978-3-031-28489-2_10
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DOI: https://doi.org/10.1007/978-3-031-28489-2_10
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