CRISPR Therapeutics AG Company profile
CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious diseases. The company develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a revolutionary 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. 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 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, the company 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, cystic fibrosis, and hurler syndrome. It partners with biopharma companies, academic centers, universities, and other organizations. The company has a strategic collaboration and license agreement with Vertex Pharmaceuticals Incorporated to develop and commercialize products for the treatment of Duchenne M. CRISPR Therapeutics AG is headquartered in Zug, Switzerland. 
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