Adeno-Associated Virus (AAV) Production

There are ever increasing numbers of AAV gene therapies reaching clinical trials, as such Pharmaron requires a robust upstream process at GMP scale to deliver highly efficacious drugs to their clients. The transfection step, which is critical to upstream performance, was identified for assessment to optimise GMP production. Utilisation of PEI – a chemically defined, linear polyethylenimine transfection reagent – allowed Pharmaron to transfect their HEK293 suspension cell line with in-house plasmids to generate AAV viral vectors.

In order to assess the suitability of their transfection step, Pharmaron utilised a multi-faceted approach to process

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