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RevITTM A Universal AAV Enhancer and TransIT-VirusGEN® transfection reagent for Improved AAV Manufacturing.

B Reese(1) J Swanson(1) A Storck(1) H Abdul-Razak(1) L Juckem(1)

1:Mirus Bio LLC, Madison, Wisconsin USA

With an exponential increase in the number of registered clinical trials using recombinant adeno-associated virus (AAV) for gene therapy, efficient viral vector manufacturing by transient transfection is now in high demand to ensure maximum yields and reduced costs. To support AAV production platforms, Mirus Bio developed RevIT™, an innovative small molecule-based AAV enhancer, which is compatible with TransIT-VirusGEN® Transfection Reagent or other transfection reagents. RevIT™ increases higher genome titers 2-4X and improves percentages of full capsids in a range of suspension HEK 293 types and cell culture media when used to produce AAV2, AAV5, AAV8, and AAV9. Additionally, when combined with the TransIT-VirusGEN® Transfection Reagent, this enhancer not only yields the highest titers but also allows for the use of reduced amounts of plasmid DNA which is considered a key cost-saving improvement. We also optimized RevIT™ with TransIT-VirusGEN® at a 2L benchtop bioreactor which shows linear scalability. These studies also did not negatively impact cell viability. Overall, we demonstrate that the TransIT-VirusGEN® Transfection Reagent in combination with RevIT® AAV enhancer offers scalable transfection solutions with simplified, cost-effective workflows to produce high AAV titers without sacrificing quality. Moreover, since RevIT™ can be seamlessly dropped into other transfection workflows, it can serve as a turnkey solution for established platforms requiring higher titers. Overall, Mirus offers solutions for cell and gene therapy developers by consolidating high performance and scalability to support cost-effective production to support more patient doses per manufacturing run.

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