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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OPKO Health, Inc., a healthcare company, engages in the diagnostics and pharmaceuticals businesses in the United States, Ireland, Chile, Spain, Israel, Mexico, and internationally. The company's Diagnostics segment operates BioReference Laboratories that offer laboratory testing services for the detection, diagnosis, evaluation, monitoring, and treatment of diseases, including esoteric testing, molecular diagnostics, anatomical pathology, genetics, women's health, and correctional healthcare to physician offices, clinics, hospitals, employers, and governmental units; and 4Kscore prostate cancer test services. Its Pharmaceutical segment offers Rayaldee to treat secondary hyperparathyroidism in adults with stage 3 or 4 chronic kidney disease, and vitamin D insufficiency. This segment also develops multi-specific immune therapies focused on oncology, immunology, and infectious diseases; OPK88004, an orally administered selective androgen receptor modulator; OPK88003, a once-weekly administered peptide for the treatment of type 2 diabetes and related obesity; Somatrogon (hGH-CTP), a once-weekly human growth hormone injection; and Factor VIIa-CTP, a novel long-acting coagulation factor to treat hemophilia. In addition, it develops and commercializes longer-acting proprietary versions of already approved therapeutic proteins or peptides; develops and produces specialty APIs; develops, manufactures, markets, and sells pharmaceutical, nutraceutical, veterinary, and ophthalmic products; commercializes food supplements and over the counter products; manufactures and sells products primarily in the generics market; and markets, distributes, and sells pharmaceutical products for a range of indications, including cardiovascular products, vaccines, antibiotics, gastro-intestinal products, hormones, and others. The company also operates pharmaceutical platforms in Spain, Ireland, Chile, and Mexico. OPKO Health, Inc. was founded in 2007 and is headquartered in Miami, Florida.
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