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ChemoCentryx, Inc., a biopharmaceutical company, focuses on the development and commercialization of new medications for inflammatory disorders, autoimmune diseases, and cancer in the United States. It offers TAVNEOS (avacopan), an orally administered selective C5aR inhibitor for the treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis. The company also develops TAVNEOS for the treatment of patients with severe hidradenitis suppurativa, as well as patients with complement 3 glomerulopathy, and lupus nephritis. In addition, it develops CCX559, an orally administered inhibitor for programmed death protein 1/programmed death-ligand 1 for the treatment of various cancers; and CCX507, an orally administered inhibitor of the chemokine receptor known as CCR9, which has completed Phase I clinical trial for the treatment of inflammatory bowel disease. Further, the company has early-stage drug candidates that targets Th17 driven diseases and CCR6. ChemoCentryx, Inc. was incorporated in 1996 and is headquartered in San Carlos, California.

GW Pharmaceuticals plc, a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics from its proprietary cannabinoid product platform in various disease areas. Its lead product is Epidiolex, an oral medicine for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. The company also develops and markets Sativex for the treatment of spasticity due to multiple sclerosis. In addition, it develops various product candidates for the treatment of schizophrenia, autism spectrum disorder, neuropsychiatric symptom, and neonatal hypoxic ischemic encephalopathy. It primarily operates in Europe, the United Kingdom, the United States, and internationally. GW Pharmaceuticals plc was founded in 1998 and is based in Cambridge, the United Kingdom.

Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product is ARIKAYCE® (amikacin liposome inhalation suspension), which is approved in the United States for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a rare and often chronic infection that can cause irreversible lung damage and can be fatal. Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.

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 offers 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. 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, infectious diseases, vaccines, and immunology; 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 being developed to treat hemophilia. In addition, it develops and commercializes longer-acting proprietary versions of already approved therapeutic proteins; 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 in a range of indications, including cardiovascular products, vaccines, antibiotics, gastro-intestinal products, hormones, and others. The company also operates pharmaceutical platforms in Ireland, Chile, Spain, and Mexico. The company was founded in 1991 and is headquartered in Miami, Florida.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.