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Versatile, High-Titer AAV and LV Manufacturing System using cGMP Lipid Polymer Nanocomplexes (LPNCs) for Therapeutic Applications

A Storck(1) J Ludtke(1) H Abdul-Razak(1) L Juckem(1)

1:Mirus Bio LLC

Nearly 60% of cell and gene therapy programs with ongoing clinical trials seek to treat a variety of more prevalent conditions including musculoskeletal disorders, diabetes, CNS disorders, and cardiovascular diseases. These clinical trials and potential commercialization manufacturing require multiple, efficient viral vector production platforms at large-scale in a variety of cell culture platforms. Transient transfection is a flexible and easy methodology that can address this need while obtaining maximum yields and reducing costs. As a response, Mirus developed an advanced and versatile transfection formulation, TransIT-VirusGEN®, consisting of a mixture of lipid and polymer enabling the formation of lipid polymer nanocomplexes (LPNCs). This novel, biomimetic technology is specifically designed for large-scale virus production to support the biopharmaceutical market's need for highly efficient recombinant AAV and LV titers in both suspension and adherent 293-derived cell types. This innovative system increases viral titers 2-10-fold over current technologies with complementary enhancers and/or complex formation solutions that provide better viral vector yields. Our data supporting the VirusGEN® platform shows i) higher titers, ii) better quality vectors (i.e., better AAV percent full capsids), iii) compatibility with various cell culture adherent and suspension systems, iv) adaptability with a turnkey easy-to-follow workflow for scalability, and v) availability in a different configuration, permitting a seamless transition from process development to GMP. These benefits make the TransIT-VirusGEN® Transfection Reagent ideal for cell and gene therapy developers by consolidating versatility with performance and scalability to support cost-effective manufacturing to produce more doses per run.

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