HEDGE-A: High cell density engineering for the production of recombinant adeno-associated virus (rAAV)

HEDGE-A will address one of the current main challenges of biopharmaceutical production: The scalability of the recombinant adeno-associated virus (rAAV) production process. Big Data approaches like transcriptomic and proteomic analyses will be used together with synthetic biology tools to engineer cell cultures and improve rAAV production. The objectives of HEDGE-A are to design (1) a suitable cell platform to sustain transfection at high cell densities and (2) a scalable upstream bioprocess to improve rAAV biomanufacturing. Access to gene therapy is currently extremely limited due to the high cost of producing rAAVs, leading to expensive treatments and making it virtually inaccessible for the general population or even for diseases requiring high dosage. The main limitation is the poor scalability of the current rAAV production process. HEDGE-A will delve into the molecular limitations of the cellular platform and provide a solution to reduce costs, increase titers and help rAAV-based gene therapies reach the wider population