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Luncheon Seminar
Day 2, June 23（Mon.） 13:00-14:00
Room B (Maesato Center)
- 2B-L-1300
Impact of viral protein stoichiometry on the biological activity of virus vector for gene therapy
(The Univ. of Osaka)
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. In addition, research and development is being actively conducted, particularly in the USA, and approximately 250 clinical trials are currently underway. 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 Japan, a governmental project to develop manufacturing technology for AAV vectors was carried out from 2018 to 2024, with the speaker mainly responsible for the development of vector characterization methods.
In this presentation, heterogeneity of capsid protein stoichiometry revealed by orthogonal biophysical methods including charge detection mass spectrometry is introduced. The impact of the heterogeneity on the biological function is then explained with the direction of future AAV quality control.