CRISPR Therapeutics AG is a biopharmaceutical company specializing in the development of gene-editing therapies based on the CRISPR/Cas9 platform. The company applies its proprietary technology to modify genes in human cells, aiming to create durable treatments for a range of serious diseases. Its research and development efforts focus on both ex vivo and in vivo applications, enabling targeted correction or disruption of disease-causing genes.
Among its lead programs is CTX001, an ex vivo edited cell therapy designed to treat sickle cell disease and transfusion-dependent β-thalassemia in collaboration with Vertex Pharmaceuticals. In addition to hematology, CRISPR Therapeutics is advancing oncology candidates such as CTX110, CTX112 and CTX120, which use engineered immune cells to target various blood cancers. The company also maintains partnerships with organizations including ViaCyte for potential treatments in type 1 diabetes, further diversifying its pipeline across therapeutic areas.
Founded in 2013 and headquartered in Zug, Switzerland, CRISPR Therapeutics maintains a significant presence in the United States, with research and development facilities in Cambridge, Massachusetts. The company’s global footprint supports clinical trials across North America and Europe, reflecting its commitment to addressing unmet medical needs in key healthcare markets worldwide.
Under the leadership of Chief Executive Officer Samarth Kulkarni, CRISPR Therapeutics continues to expand its manufacturing capabilities and prepare for potential regulatory filings. Co-founder Emmanuelle Charpentier, a pioneer in CRISPR science, serves on the board, guiding the company’s strategic direction as it advances a novel class of genetic medicines toward commercialization.