The Mass Spectrometry society of Japan - The 68th Annual Conference on Mass Spectrometry, Japan

Abstract

Oral Sessions

Day 3, May 13(Wed.) 15:10-15:30 Room B (1202)

Enhancing the characterization of adeno-associated virus (AAV) vectors by improved UPLC and MS methodology

(1Nihon Waters, 2Waters)
oKenji Hirose1, Ximo Zhang2, Stephan Koza2, Hua Yang2, Weibin Chen2

Adeno-associated viruses (AAVs) have become the most widely used vectors in gene therapy development for their low toxicity and long-term expression ability. To ensure the safety and quality of gene therapy products, the critical quality attributes (CQAs) of AAVs must be well understood and monitored throughout the product development process. While conventional analytical methods such as analytical ultra-centrifugation (AUC) and SDS-PAGE can provide valuable information regarding the CQAs of AAVs, the results are often laborious to acquire and difficult to validate. Here, we report the development of LC and MS based methods for comprehensive characterization of AAV capsids in order to deliver efficient, accurate, and reproducible measurement of CQAs including capsid protein stoichiometry and modifications, aggregation, and empty/full ratio.