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Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes Plozasiran, which is in Phase 2b and one Phase 3 clinical trial to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome; Zodasiran that is in Phase 2b clinical trial for the treatment of dyslipidemia and hypertriglyceridemia; ARO-PNPLA3, which is in Phase 1 clinical trial to treat patients with non-alcoholic steatohepatitis; ARO-RAGE that is in Phase 1/2a clinical trial to treat inflammatory pulmonary conditions; and ARO-MUC5AC, which is in Phase 1/2a clinical trial to treat muco-obstructive pulmonary diseases. It also develops ARO-MMP7 that is in Phase 1/2a clinical trial for treatment of idiopathic pulmonary fibrosis; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-SOD1 for the potential treatment of amyotrophic lateral sclerosis; and ARO-C3, which is in Phase 1/2a clinical trial for the treatment of patients with various complement mediated or complement associated renal diseases. In addition, the company is involved in the development of JNJ-3989, which is in Phase 2 clinical trial to treat chronic hepatitis B virus infection; Olpasiran that is in Phase 3 clinical trial to reduce the production of apolipoprotein A; GSK-4532990 that is in phase 2 clinical trial to treat liver diseases; HZN-457, which is in phase 1 clinical trial to treat uncontrolled gout; and Fazirsiran that is in Phase 3 clinical trial for the treatment for liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead Pharmaceuticals, Inc. has license and research collaboration agreements with Janssen Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Limited; Horizon Therapeutics Ireland DAC; Amgen Inc.; and Glaxosmithkline Intellectual Property (No. 3) Limited. The company was founded in 2003 and is headquartered in Pasadena, 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.

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.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.