CORDIS Project
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This project seeks to create a scalable production platform for recombinant adeno-associated virus (rAAV) gene therapies, aiming to reduce manufacturing complexity and costs. It also plans to develop a gene therapy candidate for treating steroid-resistant nephrotic syndrome, a severe childhood condition.
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.
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