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Oral Sessions
Day 3, June 24（Tue.） 13:55-14:10
Room A (Maesato West)
- 3A-O2-1355
Biophysical characterization of virus vectors for gene therapy by mass spectrometry
(Osaka Univ.)
oSusumu Uchiyama
Adeno-associated virus (AAV) vectors, with a molecular weight of 5 MDa and a particle diameter of 25 nm, are a promising platform modality for gene therapy, and products developed and approved in the USA and Europe are being used to treat genetic diseases. AAV vectors are manufactured using cultured cells with recombinant biotechnology, whereas in order to be used as a drug, quality in terms of physicochemical, biochemical and biological properties related to efficacy and safety should be well identified to mitigate the risks when administered to patients. In this presentation, the quality attributes of AAV vectors that need to be analyzed and the appropriate mass spectrometry (MS) based analytical methods will be explained, including peptide mapping by liquid chromatography MS and MS/MS, higher order structure and dynamics by hydrogen deuterium exchange MS, and size distribution analysis by charge detection MS. These MS based characterization of AAV vectors will contribute to ensure safety and efficacy of AAV vectors for gene therapy.