The project addresses a critical need for scalable, cost-effective rAAV manufacturing by reducing the production of empty capsids, improving vector quality and yield, lowering purification requirements, and enabling broader access to gene therapies.
The project will develop and scale the DRIVE rAAV manufacturing platform, which increases production of genome-filled viral vectors by controlling vector replication and capsid assembly. The team will optimize the process using enhanced transfection technologies, validate performance across multiple manufacturing scales and sites, and assess economic benefits relative to conventional rAAV production methods.
Increasing the percentage of filled capsids to greater than 90% in cell culture would simplify downstream processing by eliminating steps, improve final product quality by reducing empty capsids, reduce product losses in downstream, and potentially reduce costs.
The project will improve rAAV manufacturing by achieving >90% filled capsids, increasing functional vector yields, reducing purification burden and production costs, and demonstrating scalability to 50 L bioreactors. The resulting process will provide a validated, transferable manufacturing approach that enhances industry capabilities and supports more affordable, accessible gene therapies.
Pilot-scale DRIVE optimization and vector quality analysis by MIT
High-throughput screening of viral sensitizers by Virica
Intermediate-scale end-to-end process validation by Landmark Bio
Industrial-scale (50L) production and analysis by MilliporeSigma
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Massachusetts Institute of Technology
EMD Millipore Corporation
Landmark Bio
Virica Biotech Inc.
