Synthetic Viral Nanosystem for Highly Efficient AAV Manufacturing for Gene Therapy

Gene therapy is one of the most innovative and fastest growing fields in the pharmaceutical industry.

The first approved gene therapy utilized a recombinant AAV vector (rAAV), and dozens of additional rAAVs for gene therapy are presently in clinical trials. rAAV gene therapy drugs have been priced in the region of € 500’000 and above, which is partially a result of them being manufactured by highly complex processes combining multiple components, requiring 5-7 separate GMP production runs.

We intend to introduce the first scalable, single-virus rAAV production platform to resolve this bottleneck.

The resulting significant reduction of manufacturing complexity will both lower the price of future rAAV gene therapies, and also deliver additional, currently unaddressed or unaffordable rAAV treatments for genetic diseases into the clinic by providing scientists operating at the laboratory R&D stage with more user friendly and productive tools.

The proposed project also develops for PoC purposes an rAAV gene therapy candidate to treat the devastating childhood congenital disease known as steroid resistant nephrotic syndrome SRNS.