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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.

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.

Reata Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, identifies, develops, and commercializes novel therapeutics for patients with serious or life-threatening diseases. The company is developing Phase 3 clinical trial programs, including bardoxolone methyl (bardoxolone) for the treatment of patients with chronic kidney disease (CKD) caused by Alport syndrome, as well as for a form of pulmonary arterial hypertension associated with connective tissue disease; omaveloxolone to treat Friedreich's ataxia; and conduct various form of CKD, such as, type 1 and type 2 diabetic CKD, type 1 and type 2 diabetic CKD, hypertensive CKD, focal segmental glomerulosclerosis, and others. It is also developing RTA 901 for neurological diseases; and bardoxolone for the treatment of autosomal dominant polycystic kidney disease. In addition, the company has a strategic collaboration agreement with Kyowa Kirin Co., Ltd. to develop and commercialize bardoxolone for renal, cardiovascular, diabetes, and various other related metabolic indications in Japan, China, Hong Kong, Macao, South Korea, Taiwan, Thailand, Singapore, the Philippines, Malaysia, Indonesia, Brunei, Vietnam, Laos, Myanmar, and Cambodia; AbbVie Inc. to jointly research, develop, and commercialize all second- and later-generation Nrf2 activators for all indications other than renal, cardiovascular, and metabolic indications. Reata Pharmaceuticals, Inc. was incorporated in 2002 and is headquartered in Plano, Texas.