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

The number of ATMP therapeutic-based medicines for inherited genetic disorders is in constant growth, with a global 32% increasein new clinical trials in the last 4 years. ATMPs have demonstrated their success with already more than ten approved for commercialization. The success of AAV as the most promising viral vector for gene therapy is due to low immunogenicity, broad tropism and non-integrating properties. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV. Transient transfection of suspension cells is the most commonly usedproduction platform, as it offers significant flexibility for cell and gene therapy development. However, this method presents some limitations in largescale bioreactors: inadequate transfection protocol, reduced transfection efficiency and lower productivity. To address this concern, we present data on a novel transfection reagent showing: i) increased AAV titers, ii) Cost-effectiveness with reduced cost per dose for therapy affordability, iii) improved transfection protocol for large scale bioreactors and iv) reproducibility of viral titers at different production scale. Withour continuous commitment in supporting CGT manufacturers, FectoVIR®-AAV is available at GMP grade, the highest quality grade for ancillary materials aligned with quality standards recommended by health authoritiestoensure patient safety.