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Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. Its marketed products include ONPATTRO (patisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; AMVUTTRA for the treatment of hATTR amyloidosis with polyneuropathy in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria; and OXLUMO for the treatment of primary hyperoxaluria type 1. In addition, the company develops patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; Belcesiran for the treatment of alpha-1 liver disease; Elebsiran to treat chronic HBV infection; Zilebesiran to treat hypertension; ALN-APP to treat Alzheimer's disease and cerebral amyloid angiopathy; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Roche to develop pharmaceutical products containing zilebesiran. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

Alexion Pharmaceuticals, Inc. develops and commercializes various therapeutic products. The company offers ULTOMIRIS for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS); and SOLIRIS for the treatment of PNH, aHUS, generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). It also provides Strensiq for patients with hypophosphatasia; Kanuma (sebelipase alfa) for the treatment of patients with lysosomal acid lipase deficiency; and Andexxa, a reversal agent for patients treated with rivaroxaban or apixaban. In addition, the company is developing ALXN1210 (Intravenous) that is in Phase III clinical trials for the treatment of gMG, NMOSD, ALS, COVID-19, and HSCT-TMA; ALXN1210 (Subcutaneous), which is in Phase III clinical trials for PNH and aHUS; ALXN1820 that is in Phase I clinical trial, a therapeutic antagonist of properdin; and ALXN1720, which is in Phase I clinical trial for the treatment of disease states involving dysregulated terminal complement activity. Further, it is developing ALXN1840 that is in Phase III clinical trials for the treatment of Wilson disease; ALXN1830, which are in Phase I clinical trials for neonatal Fc receptor; ALXN2040 and ALXN2050 to treat diseases associated with dysregulation of the complement alternative pathway; ALXN1850, an enzyme replacement therapy; ALXN2060 for treating transthyretin amyloidosis; and ALXN2075 for treatment of relapsed/refractory chronic lymphocytic leukemia. The company serves distributors, pharmacies, hospital, hospital buying groups, and other healthcare providers in the United States and internationally. Alexion Pharmaceuticals, Inc. has collaboration and license agreement with Halozyme Therapeutics, Inc.; and agreements with Dicerna Pharmaceuticals, Inc., Zealand Pharma A/S, Caelum Biosciences, Inc., Stealth BioTherapeutics Corp., and Affibody AB. The company was founded in 1992 and is headquartered in Boston, Massachusetts.

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc. The company was incorporated in 1996 and is headquartered in San Rafael, California.

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.