Analytical Ultracentrifugation for Profiling AAV Gene Therapies: Where Are We Now?

This webcast features: Yijun Huang, Scientific Fellow, WuXi Advanced Therapies

Adenoassociated virus (AAV) is often the vector of choice for gene therapies due to their low immunogenicity, ability to promote long-term gene expression, and capsid tropism-dependent tissue specificity. Representing around 37% of the current advanced therapies market, there is an ongoing demand for robust quality lot release programs capable of supporting the large patient cohorts involved in clinical trials. Along with critical quality attributes such as titer, identity, and potency, empty/full capsid characterization is an important parameter unique to AAV vectors as it has significant effects on immunotoxicity, transduction efficiency, and anti-AAV antibody generation immune responses. Developing accurate and precise methods for characterizing capsid distribution is important not only at the clinical level where empty capsids need to be minimized for patient safety, but at the development level where significant research is ongoing as to the optimal ratio for maximal gene transfer and readministrating potential.

In this presentation, WuXi Advanced Therapies will demonstrate how analytical ultracentrifugation (AUC) is a critical and robust quality control (QC) method for capsid size distribution capable of supporting clinical and commercial AAV products.

Key Takeaways:

  • AUC is a platform and robust QC method for AAV capsids distribution determination.
  • AUC provides sufficient resolution to distinguish between empty, full, and partial capsids.
  • AUC analysis is independent of the AAV serotype and reagents free.

 

 

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