AAV.GMU034

AAV.GMU034 is an adeno-associated viral (AAV) vector-based gene therapy candidate being developed by Sanofi's Genomic Medicine Unit. The program is currently in the analytical characterization phase to support its progression through Phase 1 to Phase 3 clinical studies. Research presented at ASGCT 2026 highlights the development of a robust multi-attribute Size Exclusion Chromatography (SEC) method utilizing UV, fluorescence (FLD), refractive index (RI), and multi-angle light scattering (MALS) detection. This comprehensive analytical approach is designed to quantify and characterize high molecular weight species (HMWS) and aggregates, ensuring the safety and efficacy of the AAV vector drug product during clinical development and manufacturing.