Our therapeutic programs are focused on exploiting our proprietary capsid technology and scalable manufacturing platform to advance AAV-based therapies via three primary approaches: gene replacement, vectorized antibody, and vectorized micro-RNA modulation. Several programs include research partnerships with Sarepta Therapeutics, University of Florida, University of Southern California, and the National Institutes of Health.
AAV Vector Platform
Our proprietary process uses multiple methodologies to enhance both naturally occurring and engineered AAV capsids to improve tissue tropism and provide immune escape profiles. Our strategy has the advantage of targeting the variable regions of capsids, resulting in libraries with superior complexity compared to traditional shuffled serotype libraries.
OneBac Manufacturing Platform
Our proprietary OneBac platform uses a baculovirus expression system to infect cells derived from the Spodoptera frugiperda larval moth. Using a chemically defined medium, our system provides a platform to deliver superior yields and purification of infectious AAV for use in gene therapy applications. These key advantages are accompanied by a reduction in the requirement for manufacturing components, providing a cost-effective system to develop the highest quality material for patients.