Advancing AAV Standards in Gene Therapy

As gene therapy advances toward broader clinical applications, the need for robust quality standards and harmonized regulatory frameworks has become paramount. This session will explore the quality assessment of gene therapy, specifically the standardization of analytical methods for adeno-associated virus (AAV) vectors. For AAV-based therapies, the session will delve into analytical standardization challenges for raw materials, titer determination, empty-full analysis and process impurities. It will highlight efforts to reduce variability across laboratories and ensure data comparability, supporting the development of consensus-based standards for vector identity, purity, and potency.

In this symposium, attendees will hear perspectives about shaping quality expectations and analytical methodologies, including how standardized, fit-for-purpose testing approaches can be integrated into existing workflows and how to navigate the interface between innovation and regulatory compliance. There is a lack of harmonized analytical standards for AAV products, leading to inconsistent quality data and regulatory uncertainty. This session addresses the implementation of standardized approaches in line with regulatory expectations.