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Adeno-associated virus production platform performance for2L and 50L scales

A Anwar(1) B Ozdoganoglu(1) H Chen(1) K Farukshina(1) J Guenat(1) L Giner Robles(1) P Dashwood(1) N Sweeney(1) D Marsh(1)

1: Cell and Gene Therapy Catapult, 12th Floor Tower Wing, Guys Hospital, Great Maze Pond, London, SE1 9RT

Adeno-Associated viruses (AAV) are targeted delivery methods for gene therapies. One of the challenges when manufacturing AAV is to optimise production for the full viral particle which contains the therapeutic gene. Catapult has developed a full upstream and downstream platform at a 2L and 50L production scale. This has been characterised by internal and external assays to assess AAV2 total particle titre, full AAV2 particle titre and impurity levels. Critical quality attribute (CQA) limits were met at both 2L and 50L scale for the TFF2 pool and external analytics show over 49% full viral particles post TFF2. Catapult's AAV downstream platform shows robustness at 2L and scalability to 50L.  Future work will involve the implementation of PAT to ensure product quality, process intensification to further improve yields and assay development using novel techniques for full characterisation of AAV vectors.

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