Gene Therapy Analytical Development Summit USA
Optimisation of the AAV expression and purification using dedicated HPLC system
Speaker: Ales Strancar, Managing Director
Adeno associated virus (AAV) is the leading vector in the field of gene therapy because of its low toxicity, good overall safety profile, and ability to maintain stable expression for long periods of time. It is therefore crucial to develop a robust and high efficiency platform for its manufacturing. One of the key challenges in manufacturing viral vectors is to increase the ratio between empty and full capsids. The most efficient way is to design the USP to result in less than 10% of empty capsids. This can be realised by process optimisation using at-line HPLC to allow for analysis of the full and empty capsids ratio during the virus expression. Fast and reliable in HPLC methods to allow for process optimisation USP and DSP, and assessing the purity of the final product using PATfix system will be presented.