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Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of indolent SM, and other mast cell disorders. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered thyroid carcinoma, and medullary thyroid carcinoma; BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.

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.

Horizon Therapeutics Public Limited Company, a biotechnology company, focuses on the discovery, development, and commercialization of medicines that address critical needs for people impacted by rare, autoimmune, and severe inflammatory diseases. Its portfolio comprises 12 medicines in the areas of rare diseases, gout, ophthalmology, and inflammation. It markets TEPEZZA (teprotumumab-trbw) for intravenous infusion; KRYSTEXXA (pegloticase injection) for intravenous infusion; RAVICTI (glycerol phenylbutyrate) oral liquid; PROCYSBI (cysteamine bitartrate) delayed-release capsules and granules for oral use; ACTIMMUNE (interferon gamma-1b) injection for subcutaneous use; BUPHENYL (sodium phenylbutyrate) tablets and powder for oral use; QUINSAIR (levofloxacin) solution for inhalation; and UPLIZNA (inebilizumab-cdon) injection for intravenous use. The company also markets PENNSAID (diclofenac sodium topical solution) for topical use; DUEXIS (ibuprofen/famotidine) tablets for oral use; RAYOS (prednisone) delayed-release tablets for oral use; and VIMOVO (naproxen/esomeprazole magnesium) delayed-release tablets for oral use. It has collaboration agreements with HemoShear Therapeutics, LLC; Alpine Immune Sciences, Inc.; Halozyme Therapeutics, Inc.; Arrowhead Pharmaceuticals, Inc.; Q32 Bio Inc.; and Xeris Biopharma Holdings, Inc. The company was formerly known as Horizon Pharma Public Limited Company and changed its name to Horizon Therapeutics Public Limited Company in May 2019. Horizon Therapeutics Public Limited Company was founded in 2005 and is headquartered in Dublin, Ireland.

The Medicines Company, a biopharmaceutical company, focuses on developing therapeutics for the treatment of therosclerotic cardiovascular disease. The company is developing Inclisiran, an investigational RNA interference therapeutic that inhibits production of proprotein convertase subtilisin/kexin type 9, which controls LDL-cholesterol levels. It has collaboration agreement with Alnylam Pharmaceuticals, Inc. The Medicines Company was founded in 1996 and is headquartered in Parsippany, New Jersey.