CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a revolutionary technology for gene editing, which is the process of precisely altering specific sequences of genomic DNA.
It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes. The company also develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 (GPC3) for the potential treatment of solid tumors.
The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies. In this therapy, a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells.
Additionally, it develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications. The company also works on CTX310 and CTX320, which involve in vivo gene editing to address cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein. Furthermore, it is developing CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of type 1 diabetes.
CRISPR Therapeutics AG has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. The company was incorporated in 2013 and is headquartered in Zug, Switzerland.
