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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.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California.

Mylan N.V., together with its subsidiaries, develops, licenses, manufactures, markets, and distributes generic, branded-generic, brand-name, and over-the-counter (OTC) pharmaceutical products in North America, Europe, and internationally. It offers active pharmaceutical ingredients and finished dosage forms; and antiretroviral medicines to treat HIV/AIDS. The company also provides prescription products, such as EpiPen Auto-Injector; Perforomist Inhalation Solution; Dymista; Creon; and Influvac, as well as YUPELRI, an inhalation solution for the maintenance treatment of patients with chronic obstructive pulmonary diseases. In addition, it markets OTC products, including Cold-EEZE, MidNite, Vivarin, Brufen, CB12, and EndWarts. The company offers its products to therapeutic areas, such as cardiovascular, CNS and anesthesia, dermatology, diabetes and metabolism, gastroenterology, immunology, infectious disease, oncology, respiratory and allergy, and women's health. Its customers include retail pharmacies, wholesalers and distributors, payers, and insurers and governments, as well as institutions, such as hospitals. Mylan N.V. has collaboration and license agreements with Pfizer Inc.; Momenta Pharmaceuticals, Inc.; Theravance Biopharma, Inc.; Biocon Ltd.; Fujifilm Kyowa Kirin Biologics Co. Ltd; and Revance Therapeutics, Inc. The company was founded in 1961 and is based in Hatfield, the United Kingdom.

Ono Pharmaceutical Co., Ltd., together with its subsidiaries, produces, purchases, and sells pharmaceuticals and diagnostic reagents worldwide. It offers Opdivo Intravenous Infusion and Kyprolis Intravenous Injection for malignant tumors; Emend capsules/Proemend intravenous injections for chemotherapy-induced nausea and vomiting; Demser capsules for the symptoms in patients with pheochromocytoma; and Mektovi, Velexbru, and Braftovi capsules for malignant tumors, as well as ADLUMIZ tablets for cancer cachexia. The company also provides Glactiv tablets for type 2 diabetes; Forxiga tablets for diabetes; Onoact Intravenous Infusion for tachyarrhythmia; Opalmon tablets to treat peripheral circulatory disorder; Coralan for chronic heart failure; Orencia injections for rheumatoid arthritis; Rivastach patches for Alzheimer's disease; Ongentys tablets for Parkinson's disease; Parsabiv, an intravenous infusion for dialysis patients; Staybla tablets for overactive bladder; Onon capsules and Dry Syrups for bronchial asthma and allergic rhinitis; and JOYCLU intra-articular injection for the improvement of joint function, as well as Recalbon tablets for osteoporosis. In addition, it develops products for hepatocellular and urothelial carcinoma; ovarian, bladder, prostate, pancreatic, gastric, esophageal, colorectal, thyroid, cell lung, and breast cancer; acute myeloid leukemia; solid tumors; myelodysplastic syndrome; melanoma; T-cell lymphoma; tachyarrhythmia; pemphigus; scleroderma; seizures; diabetic polyneuropathy; neurodegenerative diseases; autoimmune diseases; narcolepsy; and thrombosis. The company has a collaboration with Adimab, LLC to discover novel antibody drugs; a research collaboration with Turbine Ltd. to identify and validate novel oncology targets; Harvard University for validating novel therapeutic targets; and a strategic drug discovery collaboration with Sibylla Biotech in central nervous system disorders. The company was founded in 1717 and is headquartered in Osaka, Japan.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.