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, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. The company is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, it is developing allogeneic CAR-T programs targeting B-Cell maturation antigen and CD70; CTX120, a CAR-T cell product candidate for the treatment of multiple myeloma; CTX130 for the treatment of solid tumors and hematologic malignancies; programs to treat Hurler Syndrome and severe combined immunodeficiency disease, as well as glycogen storage disease Ia; and programs targeting diseases, such as Duchenne muscular dystrophy and cystic fibrosis. It has a collaboration agreements with Vertex Pharmaceuticals, Incorporated and Vertex Pharmaceuticals (Europe) Limited to develop, manufacture, commercialize, sell, and use various therapeutics; and StrideBio LLC to develop adeno-associated viral capsids. 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; ViaCyte, Inc. for designing, developing, and commercializing gene-edited allogeneic stem cell therapies for the treatment of diabetes; and ProBioGen AG to develop novel in vivo delivery modalities for CRISPR/Cas9. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
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