Neurogene, Inc. Common Stock
Neurogene Inc., a clinical stage biotechnology company, develops genetic medicines for rare neurological diseases. The company's product candidates include NGN-401 that is packaged in an adeno-associated virus 9, which is in Phase 1/2 clinical trial for the treatment of Rett syndrome; and NGN-101, a conventional gene therapy candidate that is in Phase 1/2 clinical trial to treat CLN5 Batten disease. It has a license agreements with The University of Edinburgh, Virovek, Inc., Sigma-Aldrich Co. LLC., and Leland Stanford Junior University. The company was founded in 2018 and is headquartered in New York, New York.
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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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