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OVERVIEW

CRISPR Therapeutics is a leading gene-editing company focused on the development of transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. Our multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Our lead program, CTX001, aims to treat Sickle cell disease and ß-thalassemia using an ex vivo approach. We are also pursuing oncology indications with our allogeneic CAR-T platform and liver, lung, and neuromuscular indications using in vivo approaches. In addition, we have established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in other diseases with high unmet need. 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. We are headquartered in Zug, Switzerland with R&D operations in Cambridge, Massachusetts, USA and select business operations in London, United Kingdom.








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