Next-Generation Transfection Reagent for Large Scale AAV Manufacturing

Ashlee Sun, 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.

Poster

Improving transient CHO and HEK-293 Expression Systems with a powerful transfection solution for high protein production yields: FectoPRO®

Ashlee Sun, Mathieu Porte, Jonathan Havard, Valérie Moreau, Fabrice Stock, Patrick Erbacher

Development process for biotherapeutic protein production usually begins with generating a high-performing stable cell line which can be used for manufacturing. As this step takes a lot of time, transient transfection offers a great alternative to quickly produce milligram to gram quantities of recombinant proteins and antibodies. A various number of culture media are available for performing transient protein production in both CHO and HEK cells, but the limiting factor often remains the transfection reagent. Therefore, Polyplus-transfection has developed a novel technologically advanced transfection solution named FectoPRO®. Here we show that FectoPRO® outperforms currently available PEI-based and lipid-based transfection reagents in all the transient expression systems tested, offering great transfection efficiency and amazing protein yields.