CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious human diseases. It 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 multiple myeloma; and CTX130 for the treatment of solid tumors and hematologic malignancies. Further, it engages in developing regenerative medicine programs in diabetes; and in vivo and other genetic disease programs to treat glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. The company has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
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