Optimizing AAV Workflows to Accelerate Gene Therapy Development

Adeno-associated viruses (AAVs) are crucial tools in gene therapy. A major bottleneck during research and development of AAV-based gene therapies is the losses of sample during purifications, especially chromatographic purification steps. These critical steps, essential for isolating large quantities of functional viral vectors, often suffers from significant losses. These losses directly translate to poor yields, hindering the development of these promising gene therapies.

Employing density gradient preparatory ultracentrifugation using systems such as the Beckman Coulter Life Sciences Optima XPN Ultracentrifuge, for isolation can limit the loss of viral vectors during this step. The process efficiently separates empty capsids from full ones, ensuring high-quality samples for downstream processes. Density-gradient preparatory ultracentrifugation is viral serotype-independent, allowing for flexibility between viral batches.

Characterization of viral proteins is yet another crucial step in developing gene therapies involving AAV. In-depth characterization of viral proteins using spectrometry is challenging, especially when working with samples of poor quality. Limited sample quality often impedes method optimization and detection of low-abundance species during the characterization of viral proteins.

High-sensitivity mass spectrometry can eliminate the problems associated with limited sample quality during viral protein characterization. Spectrometers such as the SCIEX ZenoTOF 7600 Mass Spectrometer facilitate in-depth analysis of differences in capsid proteins, including critical indicators of viral vector binding, behavior, and localization within a host.

SCIEX OS automated data acquisition software further improves data quality and aids in incorporating optimized workflows for rapid and hassle-free classification of AAV capsid proteins.

Cutting-edge gene therapies involving AAV may help tackle several currently incurable diseases. Streamlined workflows and improved instruments can help fast-track the development of these therapies. We can partner with you to create customized solutions that optimize your workflows and expedite gene therapies to market. Contact an expert at the Life Sciences companies of Danaher today.