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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Regeneron Pharmaceuticals, Inc. discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide. The company's products include EYLEA injection to treat wet age-related macular degeneration and diabetic macular edema; myopic choroidal neovascularization; diabetic retinopathy; neovascular glaucoma; and retinopathy of prematurity. It also provides Dupixent injection to treat atopic dermatitis and asthma in adults and pediatrics; Libtayo injection to treat metastatic or locally advanced cutaneous squamous cell carcinoma; Praluent injection for heterozygous familial hypercholesterolemia (HoFH) or clinical atherosclerotic cardiovascular disease in adults; REGEN-COV for covid-19; and Kevzara solution for treating rheumatoid arthritis in adults. In addition, the company offers Inmazeb injection for infection caused by Zaire ebolavirus; ARCALYST injection for cryopyrin-associated periodic syndromes, including familial cold auto-inflammatory syndrome and muckle-wells syndrome; and ZALTRAP injection for intravenous infusion to treat metastatic colorectal cancer; Evkeeza for treatment of HoFH; Ordspono for Follicular lymphoma and Diffuse large B-cell lymphoma; and Veopoz for CD55-deficient protein-losing enteropathy. Further, it develops product candidates for treating patients with eye, allergic and inflammatory, cardiovascular and metabolic, infectious, and rare diseases; and cancer, pain, and hematologic conditions. It has collaboration with Mammoth Biosciences, Inc. to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types; and with Sonoma Biotherapeutics, Inc. to discover, develop, and commercialize engineered regulatory T cell therapies. The company was incorporated in 1988 and is headquartered in Tarrytown, New York.
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