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CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, which targets sickle cell disease and beta-thalassemia with an ex vivo approach whereby cells are harvested from a patient, treated with a CRISPR/Cas9-based therapeutic and reintroduced into the patient. The company is also developing CTX101, a donor-derived gene-edited allogeneic CAR-T therapy targeting CD19-positive malignancies. In addition, it is developing earlier stage allogeneic CAR-T programs targeting B-Cell maturation antigen and CD70; hemoglobinopathies to treat other diseases, including Hurler Syndrome and severe combined immunodeficiency disease; programs that are in preclinical development for indications, including glycogen storage disease Ia and hemophilia; and programs targeting diseases of organ systems outside the liver, such as Duchenne muscular dystrophy and cystic fibrosis. It has a collaboration agreement with Vertex Pharmaceuticals, Incorporated Vertex Pharmaceuticals (Europe) Limited to develop, manufacture, commercialize, sell, and use therapeutics. The company also has research collaboration agreements with Neon Therapeutics for developing neoantigen-based therapeutic vaccines and T cell therapies; Massachusetts General Hospital Cancer Center to develop T cell therapies for cancer; and with the ViaCyte, Inc. for designing allogeneic cell therapies derived from gene edited human stem cells for use in the treatment of diabetes type 1, diabetes type 2, and insulin dependent diabetes. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
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