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AAVNova –Nextgen Platform for AAV Production

AAV Gene therapy allows for curing of previously incurable diseases. We aim to make it more accessible and scalable by improving how AAV are produced.

AAVNova

Gene therapy allows for a new way of delivering effect via DNA into the cells. This way protein therapeutics can be produced inside the cells Luxturna is an approved AAV drug treating inherited blindness. One treatment per eye with the AAV-drug is enough for restoring vision Zolgensma is another approved AAV drug treating a muscular disorder called SMA. This drug restores the expression of a key protein needed for control of the arms and legs of children. The AAV is a small and simple viral vector which allows for packaging of cargo DNA (the therapy) Different AAV serotypes have different tropism and are enriched in different organs The delivered cargo is typically maintained extrachromosomaly in the nucleus without integration into the host genome.
Some highlights which we have learned sofar since the program start Apr 2019 are: 1) Behaviour and structure similarities of 10 common AAV serotypes 2) How to rationally design and experimentally produce novel AAV 3) How human cells react on AAV production when transfected with viral DNA in a bioreactor 4) A first process for AAV production in high cell density per fusion
New products, tools and software have been generated in relation to the program 1) Genetic volume controls for precise control of protein translation rate 2) AlfaLaval has launched a series of single-use products for bioprocesses 3) Vironova has developed novel ways to prepare AAV sampels and software and statistical methods for automated analysis of quality parameters

Frågor?

Prof. Johan Rockberg

KTH - Protein Science

Webbplats: www.rocklab.se

E-post johanr@biotech.kth.se

Senast uppdaterad 3 mars 2021