Authors: Alexander E. Yarawsky, Carlo Ciatto, Peter Slade, Natalya Figueroa, John W. Burgner 2nd, Michael DeLion, Lake N. Paul. Quantitation of AAV in a dual-vector system using SV-AUC. Journal of Pharmaceutical Sciences, volume 114, pages 900-910, (2025). DOI: Click Here

About This Research

Gene therapy transgene targets often exceed conventional AAV vector packaging limits, requiring the use of two or more vectors. Such systems are difficult to reliably quantitate with traditional approaches. This study, featuring BioAnalysis team members Natalya Figueroa, John W. Burgner II, Michael T. DeLion, and Lake N. Paul, presents a first-of-its-kind application of SV-AUC for dual-vector AAV systems. By achieving resolution of vectors with only 400-nucleotide differences, this methodology marks a significant leap in analytical precision for complex gene therapy development.

Why This Matters

The study demonstrates accuracy within 8 percent and R² > 0.99 correlation with ddPCR, confirming the method’s robustness for regulatory submissions. This breakthrough opens new possibilities for gene therapy development using multi-vector systems, providing the analytical reliability essential for advancing large transgene therapeutics that exceed single-vector capacity.