Waters

Produce, Purify, and Characterize Adeno-associated Virus Particles for Gene Therapy

Students Thomas Cheung, Jordan Cruz, Matthew Guzman, Charles Koli, Eric Son, Rachel Wehrman

Corporate Liaison Parker Lee

Faculty Advisor Saurav Datta

Waters Corporation, a global leader in analytical instruments and software, recently completed its acquisition of Wyatt Technology, a leader in light scattering and field-flow fractionation instruments, software, accessories, and services. This acquisition enhances Waters' capabilities in bioanalytical characterization, particularly for large molecule applications such as cell and gene therapies.

The company introduced the first commercial light scattering detectors incorporating lasers and has since expanded its product line to include instruments for measuring molar mass, size, particle concentration, charge, and biomolecular interactions. These tools are widely used across various industries, including pharmaceuticals, biotechnology, and academia.

The goal of the Waters-Wyatt TMP with KGI was to study adeno-associated virus (AAV) particle serotype 2, as they are considered one of the most promising vectors for gene therapy. Currently there are challenges that include the low yield of viral vectors, the lack of characterizing undesirable capsid forms such as empty and partial capsids. The team had three main objectives: production of AAV using triple-transient transfection of HEK293T cells, purification of AAV using chromatography and filtration techniques, and characterization of AAV quality attributes using real time multi-angle light scattering (RT-MALS) using equipment provided by Waters-Wyatt. Completing this project will allow Waters-Wyatt to have the data we collected from the project to have a better understanding of the performance of their equipment in an academic setting and how they can use this data for their current and future clients.