PatheonTM Quick to ClinicTM: A Serotype Agnostic AAV Vector Manufacturing Platform

Recombinant adeno-associated viral (rAAV) vectors are currently one of the most widely used gene therapy products in development due to their lack of pathogenicity, gene expression persistence and presence of various serotypes that enable a diverse cell tropism. The rapidly growing demand combined with challenges arising from long process development timelines, lack of cGMP-suitable fit-for-purpose components and uncertainty in regulatory approval has led to a critical need for scalable and cost- effective standardized manufacturing platforms. Thermo Fisher Scientific has created a high quality scalable robust end-to-end manufacturing rAAV platform that uses fit-for-purpose materials with matched analytics. Despite the subtle biochemical and biophysical properties of various AAV therapeutics/ serotypes, we were able to develop a purification platform that would be tolerant of these differences. Utilizing this characterized platform will enable clients to bring their products to the clinic on a de-risked and accelerated pathway.