Abstract

Gene and cell therapy-based medicines are experiencing resurgence due to the introduction of “next generation” transfer viral vectors, which have demonstrated improved safety and efficacy. Adeno Associated Virus (AAV) and Lentivirus are very commonly used in therapeutics and often produced using PEI-mediated transient transfection in HEK-293 or HEK-293T cells. The critical raw materials needed for cGMP vector production must be sourced from approved suppliers and should have gone through a rigorous testing program to reduce the risk of introducing adventitious agents into the production process. Polyplus-transfection now provides PEIpro®, the unique PEI-based transfection reagents available in different quality grades, allowing a seamless transition from process development with PEIpro®-HQ to cGMP biomanufacturing with PEIpro®-GMP. Here, we describe an optimized PEI-based virus production process for high-yielding viral vector production, compatible with different cell culture adherent and suspension systems. We further demonstrate the robust viral vector production yields, as well as the adaptability and reliability of the PEI-based transient gene expression approach to efficiently manufacture GMP-grade viral vectors at a sufficiently large scale for more advanced clinical trials, and in fine to drive commercialization of therapeutic vectors.