Gene therapy with Adeno-associated virus (AAV) vectors is on a steep rise, but efficient production of safe vectors remains a challenge. The ATIVAA project develops a next generation vector production platform - for the gene therapeutics of tomorrow.
Gene therapy is the substitution of a patients defective gene with a functional one. The therapeutic gene is delivered by a gene ferry, also called vector, which is often an engineered virus. The most prominent virus vector is the Adeno-associated virus (AAV). Two AAV-based therapeutics are currently approved for treatment and the clinical pipeline holds dozens more. However, a limited production capacity and production-based impurities lead to high therapy costs and impede a wide-spread application beyond rare genetic diseases. The ATIVAA project aims to address these problems with an innovative technology platform including up- and downstream processing of AAV vectors.