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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.

Biogen 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 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.

Seagen Inc., a biotechnology company, develops and commercializes therapies for the treatment of cancer in the United States and internationally. It offers ADCETRIS, an antibody-drug conjugate (ADC) for the treatment of patients with Hodgkin lymphoma or CD30-positive T-cell lymphomas; PADCEV, an ADC targeting Nectin-4 for the treatment of adult patients with advanced or metastatic urothelial cancer; and TUKYSA, an oral small molecule tyrosine kinase inhibitor for the treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer. The company also develops TIVDAK for metastatic cervical cancer and other solid tumors; Ladiratuzumab Vedotin, an ADC targeting LIV-1 for metastatic breast cancer and solid tumors; Disitamab Vedotin, a novel HER2-targeted ADC; and SEA-TGT and SEA-CD70 for various cancer diseases. It has collaboration agreements with Takeda Pharmaceutical Company Limited; Agensys, Inc.; Genmab A/S; Merck; and RemeGen, Co. Ltd. The company was formerly known as Seattle Genetics, Inc. and changed its name to Seagen Inc. in October 2020. Seagen Inc. was incorporated in 1997 and is headquartered in Bothell, Washington. As of December 14, 2023, Seagen Inc. operates as a subsidiary of Pfizer Inc.