CRISPR Therapeutics AG
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, as well as 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 which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
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Lexeo Therapeutics, Inc., a clinical stage genetic medicine company, focuses on hereditary and acquired diseases with high unmet need in the United States. Its product pipeline comprises LX2006, an AAVrh10-based gene therapy candidate, which is in phase 1/2 clinical trial to treat friedreich ataxia cardiomyopathy; LX2020, an AAVrh10-based gene therapy candidate, which is in phase 1/2 to deliver a functional plakophilin-2 (PKP2) gene to cardiac muscle for the treatment of PKP2-ACM; and LX2021, a gene therapy candidate, which is in preclinical trial to deliver the coding sequence for the functional connexin 43, or Cx43, protein for a group of inherited cardiac muscle disorders associated with a high risk of sudden death, including arrhythmogenic cardiomyopathy (ACM) and certain forms of dilated cardiomyopathy. The company also develops LX2022, a gene therapy candidate, which is in preclinical trial to deliver a functional TNNI3 gene to myocardial cells to treat a distinct form of hypertrophic cardiomyopathy. In addition, it is developing LX1001, an AAVrh10-based gene therapy candidate, which has completed phase 1/2 clinical trial for the treatment of APOE4-associated Alzheimer's disease. The company has license agreement with Cornell University to conduct of the Phase 1/2 clinical trial of LX1001, as well as support the development of LX1004 program; research collaboration agreement with Cornell University to conduct preclinical research to develop the licensed technology; and third license agreement which obtained certain rights for FA cardiomyopathy, including rights to current and future clinical data from an ongoing Cornell University investigator-initiated Phase 1A trial of a gene therapy candidate. The company was founded in 2017 and is headquartered in New York, New York.
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