2022 - Process Intensification in AAV Gene Therapy

Gene therapy holds the potential to dramatically change healthcare for the better. Many of these viral therapies address patient populations that have no other options for treatment. AAV is one of the leading viral vectors for gene therapy applications, with a well-characterised safety profile, and (relatively) low immunogenicity. The initial approved gene therapy products on the market, such as Luxturna (2017) and Zolgensma (2019) utilize AAV as the delivery vehicle.
However, while new AAV therapies are being constantly developed, the manufacturing capacity for viral vectors is not able to address the demand. Some estimates indicate, for example, that the entire current capacity of the viral manufacturing industry is only capable of supplying a fraction of the viral vector doses needed to address Duchenne muscular dystrophy patients, a single disease indication. Novel solutions need to be applied to bottlenecks within the manufacturing process to supply the needs of patients waiting for these therapies. One of the major bottlenecks, addressed in this webinar, is the problem of low manufacturing yield.
Historically, viral manufacturers will focus on three major categories to increase yield: cell line and vector engineering, media optimization and bioreactor design. These methods often provide substantial increases to yield. However, none of them address antiviral defences in production cells. These defences actively attack and degrade foreign genetic material used to make viruses, as well as the viral products made by the cell. As a result, viral yield on a per-cell basis can be significantly affected.
Register for this webinar to learn about process intensification and the role of antiviral defences in AAV gene therapy systems.