We are pioneering the development of precision gene therapies based on our proprietary AAV vectors. We believe our customized AAV vectors have the potential to enable targeted delivery, efficient transduction, reduced inflammation and resistance to pre-existing antibodies. As a result, we believe we will be able to develop gene therapies with improved therapeutic profiles and also address previously untreatable diseases and patient populations.
Our Therapeutic Vector Evolution platform enables our “disease first” approach to product discovery, design and development. We use our platform to discover custom and proprietary AAVs designed for specific tissues and diseases, which we believe will allow us to overcome known limitations of conventional AAV vectors and potentially address a broad range of both rare and large market diseases. Based on our Target Vector Profile for a disease, we select customized capsids in non-human primates from an estimated over one billion vector capsid sequences in our 37 proprietary and diverse AAV vector libraries as of August 1, 2019. We subsequently characterize each vector through extensive studies in non-human primates and human cell and organotypic tissue assays.
Targeted delivery to specific tissues by routine clinical routes of administration
Improved transduction of target cell types and tissues
Lower toxicity with reduced inflammation
Ability to avoid neutralization by pre-existing antibodies in humans
How it works: Through our proprietary Therapeutic Vector Evolution platform, we rigorously screen our vectors to select for the optimal AAV for a specific disease, or class of diseases. We focus on discovering and selecting vectors that we believe have the potential to display superiority to conventional vectors.