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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Novartis AG engages in the research, development, manufacture, distribution, marketing, and sale of pharmaceutical medicines in Switzerland and internationally. The company offers Entresto, an angiotensin receptorneprilysin inhibitor for the treatment of symptomatic chronic heart failure with reduced ejection fraction (HFrEF); Cosentyx to treat plaque psoriasis, pso riatic arthritis, ankylosing spondylitis, and nonradiographic axial spondy loarthritis; Kisqali, a selective oral cyclin dependent inhibitor of kinases 4 and 6 (CDK4/6); Promacta/Revolade to treat immune thrombocytopenia (ITP), thrombocytopenia, and patients with severe aplastic anemia (SAA); Tafinlar+Mekinist, an oral combination therapy to treat patients with certain types of cancers; and Jakavi for the treatment of myelofibrosis, polycythemia vera, and acute or chronic graftversushost disease (GvHD). It also provides Tasigna to treat philadelphia chromosomepositive chronic myeloid leukemia in the chronic and/or accelerated phase; Xolair for the treatment of allergic asthma and nasal polyps or severe chronic rhinosi nusitis with nasal polyps; Ilaris to treat fever syndromes, Still's disease, and acute gouty arthritis; Pluvicto to treat prostatespecific membrane anti genpositive metastatic castrationresistant prostate cancer; Sandostatin SC and Sandostatin LAR to treat acromegaly carcinoid tumors and other types of functional gastro intestinal and pancreatic neuroendocrine tumors; Zolgensma for the treatment of genetic root cause of spinal muscular atrophy; Lucentis; Leqvio to reduce LDL cholesterol; Lutathera; Scemblix; and Fabhalta. The company focuses on therapeutic areas, such as cardiovascular, renal and metabolic, immunology, neuroscience, oncology, and hematology. It has a license and collaboration agreement with Alnylam Pharmaceuticals, Inc. to develop, manufacture, and commercialize Leqvio (inclisiran), a therapy to reduce LDL cholesterol. Novartis AG was incorporated in 1996 and is headquartered in Basel, Switzerland.
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