6th Gene Therapy for Rare Disorders

Chromatography Toolkit for Enhanced Purity of AAV Products

Speaker: Maja Leskovec, Head of PC3, Process Development Viruses

Adeno-associated virus is go-to vector for gene therapy treatment of rare diseases. Even with a good overall safety record to date in human patients, some recent clinical trials revealed that immune response to AAV vector is likely to be a bottleneck in gene therapy. Improved manufacturing is therefore mandatory for future safer products.

Chromatography on monolith columns offers efficient and scalable downstream processes.  Case study using Design-of-experiment approach for improved separation of empty and full capsids will be presented. In addition, residual host cell proteins, host cell DNA, plasmid DNA, and endotoxin removal will be discussed.

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