Alnylam Pharmaceuticals
NASDAQ:ALNYAlnylam 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.
Biogen
NASDAQ:BIIBBiogen Inc. discovers, develops, manufactures, and delivers therapies for treating neurological and neurodegenerative diseases in the United States, Europe, Germany, Asia, and internationally. The company provides TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI, and FAMPYRA for multiple sclerosis (MS); SPINRAZA for spinal muscular atrophy; ADUHELM to treat Alzheimer's disease; FUMADERM to treat plaque psoriasis; BENEPALI, an etanercept biosimilar referencing ENBREL; IMRALDI, an adalimumab biosimilar referencing HUMIRA; FLIXABI, an infliximab biosimilar referencing REMICADE; and BYOOVIZ, a ranibizumab biosimilar referencing LUCENTIS. It offers RITUXAN for treating non-Hodgkin's lymphoma, chronic lymphocytic leukemia (CLL), rheumatoid arthritis, two forms of ANCA-associated vasculitis, and pemphigus vulgaris; RITUXAN HYCELA for non-Hodgkin's lymphoma and CLL; GAZYVA to treat CLL and follicular lymphoma; OCREVUS for relapsing MS and primary progressive MS; LUNSUMIO to treat relapsed or refractory follicular lymphoma; glofitamab for non-Hodgkin's lymphoma; and other anti-CD20 therapies. In addition, the company is developing various products for the treatment of MS, Alzheimer's disease and dementia, neuromuscular disorders, Parkinson's disease and movement disorders, neuropsychiatry, genetic neurodevelopmental disorders, and biosimilars, which are under various stages of development. It has collaboration and license agreements with Acorda Therapeutics, Inc.; Alkermes Pharma Ireland Limited; Denali Therapeutics Inc.; Eisai Co., Ltd.; Genentech, Inc.; Neurimmune SubOne AG; Ionis Pharmaceuticals, Inc.; Samsung Bioepis Co., Ltd.; Sangamo Therapeutics, Inc.; and Sage Therapeutics, Inc. The company was founded in 1978 and is headquartered in Cambridge, Massachusetts.
BioMarin Pharmaceutical
NASDAQ:BMRNBioMarin 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.
Incyte
NASDAQ:INCYIncyte Corporation, a biopharmaceutical company, engages in the discovery, development, and commercialization of therapeutics for hematology/oncology, and inflammation and autoimmunity areas in the United States and internationally. The company offers JAKAFI (ruxolitinib) for treatment of intermediate or high-risk myelofibrosis, polycythemia vera, and steroid-refractory acute graft-versus-host disease; MONJUVI (tafasitamab-cxix)/MINJUVI (tafasitamab) for relapsed or refractory diffuse large B-cell lymphoma; PEMAZYRE (pemigatinib), a fibroblast growth factor receptor kinase inhibitor that act as oncogenic drivers in liquid and solid tumor types; ICLUSIG (ponatinib) to treat chronic myeloid leukemia and Philadelphia-chromosome positive acute lymphoblastic leukemia; and ZYNYZ (retifanlimab-dlwr) to treat adults with metastatic or recurrent locally advanced Merkel cell carcinoma, as well as OPZELURA cream for treatment of atopic dermatitis. Its clinical stage products include retifanlimab under Phase 3 clinical trials for squamous cell carcinoma of the anal canal and non-small cell lung cancer; axatilimab, an anti-CSF-1R monoclonal antibody under Phase 2 that is being developed as a therapy for patients with chronic GVHD; INCA033989 to inhibit oncogenesis; INCB160058, which is being developed as a disease-modifying therapeutic; and INCB99280 and INCB99318 for the treatment solid tumors. The company also develops INCB123667, INCA32459, and INCA33890, as well as Ruxolitinib cream, Povorcitinib, and INCA034460. It has collaboration out-license agreements with Novartis and Lilly; in-license agreements with Agenus, Merus, MacroGenics, and Syndax; and collaboration and license agreement with China Medical System Holdings Limited for the development and commercialization of povorcitinib. The company sells its products to specialty, retail, and hospital pharmacies, distributors, and wholesalers. The company was formerly known as Incyte Genomics Inc and changed its name to Incyte Corporation in March 2003. Incyte Corporation was incorporated in 1991 and is headquartered in Wilmington, Delaware.