Scaling AAV Manufacturing: A Blueprint For Clinical-Grade Gene Therapy Production

Explore a comprehensive approach to overcoming key challenges in AAV9 gene therapy manufacturing, with a focus on scalability, reproducibility, and accessibility. The speakers outline an end-to-end process that integrates upstream optimization, downstream purification, and rigorous analytical testing to produce clinical-grade viral vectors. Emphasis is placed on addressing common industry bottlenecks, including low transfection efficiency, high empty capsid content, and yield–purity trade-offs during purification. By refining critical steps such as triple plasmid transfection and chromatography-based separation, the presented strategy significantly enhances viral genome yield and full capsid enrichment while maintaining scalability from small to pilot-scale production. The process is designed to be flexible and transferable, enabling broader adoption across organizations without restrictive licensing barriers. Overall, this blueprint aims to standardize AAV9 manufacturing workflows and accelerate the development and delivery of gene therapies to clinical settings.