(1329-B) LabChip GX II Touch system for gene therapy applications: High-throughput AAV empty/full characterization
Monday, February 5, 2024
2:00 PM – 3:00 PM EST
Location: Exhibit Halls AB
Abstract: Non-viral and viral vectors are gaining success in gene therapy due to low immunogenicity and the ability for long-term gene expression in vivo. There are several different delivery vectors that require optimized characterization protocols including lipid nanoparticles (LNP) and adeno-associated virus particles (AAV). During AAV development, testing strategies for viral protein stoichiometry, capsid product purity, and ssDNA loading efficacy are all used to monitor and assess critical quality attributes (CQAs) for AAV particles. Here we show improvements in microfluidic capillary electrophoresis methods enabling high-throughput (up to 96 samples at a time) multi-parameter analysis of AAV particles. The methods optimize for the genomic peak height and allow full 96 well plate runs. The resulting AAV empty/full CV obtained was within ~ 5% during 96-well plate experiments. For research use only. Not for use in diagnostic procedures.