CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing.
Our multi-disciplinary team of world-class researchers and drug developers is working to translate CRISPR/Cas9 technology into breakthrough human therapeutics. Our lead program in the blood diseases β-thalassemia and sickle cell disease will soon enter clinical testing. In addition, our immuno-oncology franchise, focused on CRISPR-based allogeneic CAR-T cell therapies to treat cancers, offers potential therapeutic advantages over the current generation of therapies.