VP Sales US
Field Application Specialist - AMERICAS
Sales Specialist - North East
Optimizing transfection process in suspension cells to reduce dramatically the cost of goods of AAV gene therapy.
AAV-based gene therapy products have demonstrated promising results in clinical trials, but their manufacturing costs are so high that these treatments may not be affordable for most of the patients. To increase AAV vector yields, LogicBio optimized the plasmids used for suspension HEK293 cell transfection. When combined with a novel transfection reagent, the vector titers were increased by 20 to 25 times, resulting in a significant drop of the cost of goods.
Presented by Matthias Hebben, PhD, Vice President at LogicBio Therapeutics
Wednesday, August 18th at 9:30 am – 10:00 am EDT- Gene Therapy Manufacturing track
Next-Generation Transfection Reagent for Large Scale AAV Manufacturing
Ashlee Sun, Mathieu Porte, Mégane Denu, Marine Ricordel, Jonathan Havard, Yann Philipson, Malik Hellal, Patrick Erbacher
The number of Advanced Therapy Medicinal Products (ATMPs) for inherited genetic disorders is in constant growth, with a global 32% increase in new clinical trials in the last four years. ATMPs have demonstrated their success with more than 10 products approved for commercialization. AAV is considered the most promising viral vector for gene therapy because of its low immunogenicity, broad tropism and non-integrating properties. One major challenge in translating promising research into clinical development is to manufacture sufficient quantities of AAV. Transient transfection of suspension cells is the most commonly used production platform, as it offers significant flexibility for cell and gene therapy development. However, this method presents some limitations in large scale bioreactors: inadequate transfection protocol, reduced transfection efficiency and low productivity. To address these limitations, Polyplus-transfection presents data on a novel transfection reagent, FectoVIR®-AAV, and its three great benefits: i) increased AAV titers, ii) improved transfection protocol for large scale bioreactors, and iii) reproducibility of AAV titers at different production scales. The aforementioned optimized parameters make this novel transfection reagent the ideal choice for cell and gene therapy developers by combining the flexibility of transient transfection with scalability and speed to market.