Evaluating Current Manufacturing Platforms For Recombinant AAV Production

By Samira Shore, Director Technical Program Design, Thermo Fisher Scientific

With the ability of gene therapies to cure a wide range of chronic, rare, and genetic diseases, the opportunities in this space are significant. Viral vectors are a key component of gene therapies and an excellent means to transport nucleic acid cargo into the cell to express the therapeutic protein. A commonly used viral vector in the industry today with great success in gene delivery is adeno-associated virus (AAV).

Recombinant AAV vectors are based on human parvoviruses that are not known to cause serious diseases, so there is no obvious pathogenicity after transduction. There are currently 13 AAV serotypes and greater than 100 variants. This does not include the novel serotypes and the engineered-capsid serotypes that are currently being developed to ensure broader and/or specific tissue tropism. AAV vectors are most desirable because they can infect both dividing and non-dividing cells, and within these cells, they do not integrate into the genome but rather form episomes that have long-lived transgene expression.

Realizing the full potential of viral vector-based therapies requires a successful manufacturing platform for recombinant AAV vectors. As one explores options in this growing and exciting corner of the market, it is important to understand the platforms currently available to properly evaluate their fitness for the unique product needs.