Streamlined and affordable gene therapy manufacturing through advanced rAAV production platforms

Jose Miguel Escandell.

Instituto de Biologia Experimental e Tecnológica, Portugal.

Gene therapies have the potential to transform treatment for rare diseases. However, the viral vectors used to deliver them remain expensive, slow and difficult to manufacture at scale, limiting patient access to potentially life-changing therapies. This project aims to make gene therapy more affordable and accessible by developing a more effective platform for producing recombinant adeno-associated viral vectors.

The HERO platform combines advances in cell engineering with bioprocess development to increase production yields while maintaining quality. The platform can reduce manufacturing costs by up to 80% and shorten development timelines from several months to just a few weeks. These improvements make large-scale production more feasible.

The project could expand access to advanced gene therapies, particularly for people with rare diseases for whom high production costs often limit treatment options. By enabling faster, more efficient and cost-effective manufacturing, this platform could also accelerate the translation of new therapies from the laboratory to the clinic and into real-world patient care.

STAGE 2