CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious human diseases. The company develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a gene editing technology that allows for precise directed changes to genomic DNA. It has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-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. It is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, the company is developing allogeneic CAR-T programs comprising CTX120 targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and CTX130 for the treatment of solid tumors and hematologic malignancies. Further, it engages in the development of regenerative medicine programs in diabetes; and in vivo and other genetic disease programs to treat glycogen storage disease Ia, Duchenne muscular dystrophy, myotonic dystrophy type 1, and cystic fibrosis. The company has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc. It also has a strategic partnership with Nkarta, Inc. to research, develop, and commercialize CRISPR/Cas9 gene-edited cell therapies for cancer. The company was formerly known as Inception Genomics AG and changed its name to CRISPR Therapeutics AG in April 2014. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
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