The project addresses the critical need for standardized, robust analytical methods to detect and quantify AAV aggregation, which is essential for ensuring the safety, potency, and quality of gene therapy products. By generating reproducible data across multiple serotypes and stress conditions, it mitigates manufacturing risks and supports regulatory compliance, ultimately enabling scalable and reliable gene therapy production in the U.S. biopharmaceutical industry.
This project will reduce risk and increase product quality in gene therapy manufacturing by generating critical data on AAV aggregation across varied stress conditions and by developing robust, standardized analytical methods for AAV aggregate quantification. The use of multiple AAV serotypes, gene sizes, and sources (including commercial and in-house produced) will deliver a comprehensive, high-reproducibility dataset that will directly inform process development and quality control across the NIIMBL community and the broader U.S. biopharmaceutical ecosystem.
The project will deliver a detailed, well-defined set of stress conditions to study AAV2 and AAV9 aggregation and stability.
Fully documented and validated analytical assay(s) for AAV aggregate quantification will be developed, complete with step-by-step procedures, instrument calibration, and data analysis methods to facilitate method transfer across the NIIMBL community.
This $205,778 investment will deliver validated AAV aggregation testing methods that could reduce batch failure rates by 20–30%, saving $2–3 million annually in wasted materials and rework for large-scale gene therapy manufacturers. By accelerating process development and improving quality control, the project can shorten commercialization timelines by 3–6 months, generating $10–15 million in additional revenue per therapy while lowering regulatory risk and enhancing reproducibility across the U.S. biopharmaceutical ecosystem.
A full SOP for AAV aggregation analysis including any sample preparation, instrument calibration, data analysis
A manuscript to be submitted to a peer-reviewed journal and production of educational resources on AAV aggregation analysis
Login to the NIIMBL member portal to access additional project information, including presentations, progress updates, reports, and more.
Not yet a member? Learn more about which level of NIIMBL membership is right for you and your organization.
Michigan Technological University
