Gene Therapy for Rare Disorders US

Talk

Supporting Viral Vector Therapies from PD to Commercialization

• Current bottlenecks in the Industrial AAV production process
• Improvement of the upstream process is critical for cost effective gene therapy manufacturing

Presented by Roel Gordijn, CCO at Polyplus-transfection

Next-Generation Transfection Reagent for Large Scale AAV Manufacturing

Mathieu Porte, Mégane Denu, Marine Ricordel, Jonathan Havard, Coralie Stritt, Yann Philipson, Malik Hellal, Patrick Erbacher

ATMPs have demonstrated their success with already more than ten approved for commercialization. A major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV. While transient transfection of suspension cells is the most commonly used production platform, scalability of the process is a bottleneck. Here, we present data on novel transfection reagent that combines flexibility of transient transfection with scalability for manufacturing of AAVs in up to 2000L bioreactors.