Precise, Versatile Editing Platform
Our novel base editing approach uses a chemical reaction designed to create precise, predictable and efficient genetic outcomes at the targeted DNA sequence. By rewriting a single base in the genome, base editors enable a diverse toolkit that could therapeutically intervene to correct disease-causing point mutations, modify genes to create protective genetic variations, activate gene expression, silence gene expression, or “multiplex” and make multiple simultaneous edits.
Leveraging All Clinically Validated Delivery Modalities
Our delivery strategy is to establish a comprehensive suite of clinically validated technologies, including electroporation, non-viral and viral delivery modalities. We understand that no single technology has been able to deliver medicines to different target organs with equal efficacy. As such, we plan to use electroporation for efficient delivery to blood cells and immune cells ex vivo, LNP for in vivo delivery to the liver and potentially other organs in the future, and AAV for in vivo delivery to the eye and CNS.
Broad and Diversified Portfolio
We are utilizing three clinically validated delivery modalities to build a broad and diverse portfolio.