CRISPR Therapeutics AG

CRISPR Therapeutics AG is a gene editing company that develops therapeutic medicines using CRISPR/Cas9 technology, a precision gene-editing platform that allows researchers to alter specific DNA sequences in human cells. The company's therapeutic pipeline spans multiple disease areas, with a focus on hemoglobinopathies, CAR T cell therapies, and metabolic disorders. The company's lead product candidate, CASGEVY, is an ex vivo gene-edited cell therapy designed to treat patients with transfusion-dependent beta-thalassemia and severe sickle cell disease by modifying hematopoietic stem cells to produce elevated levels of fetal hemoglobin.

The company's clinical pipeline includes CTX112 and CTX131, CAR T cell therapies targeting CD19 and CD70 for oncology and autoimmune indications, as well as in vivo gene editing programs CTX310 and CTX320 aimed at cardiovascular disease. CTX211, an allogeneic gene-edited cell product, targets type 1 diabetes. CRISPR Therapeutics maintains a strategic partnership with Vertex Pharmaceuticals, which includes co-development and commercialization agreements.

Incorporated in Switzerland in 2013 and headquartered in Zug, the company is listed on Nasdaq. As of the available data, CRISPR Therapeutics operates primarily as a clinical-stage development company focused on advancing its CRISPR-based therapeutic candidates through regulatory approval and commercialization.