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.
Galapagos
NASDAQ:GLPGGalapagos NV, an integrated biopharmaceutical company, engages in the discovery, development, and commercialization of?various medicines for high unmet medical need. Its pipeline products include filgotinib, a JAK1 inhibitor that is in various phases of clinical trials for the treatment of rheumatoid arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis, and uveitis. The company's pipeline products also comprise GLPG3667 that has completed Phase 1b trial; and GLPG5101 and GLPG5201, CD19 CAR-T product candidate manufactures at point-of-care, that is in Phase I/II in relapsed/refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia. The company has collaboration agreements with Gilead Sciences, Inc.; AbbVie S.à r.l.; and Novartis Pharma AG. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.
Ono Pharmaceutical
OTCMKTS:OPHLFOno 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.
Sage Therapeutics
NASDAQ:SAGESage Therapeutics, Inc., a biopharmaceutical company, develops and commercializes brain health medicines. Its product candidates include ZULRESSO, a CIV injection for the treatment of postpartum depression (PPD) in adults; and ZURZUVAE, a neuroactive steroid, a positive allosteric modulator of GABAA receptors, targeting both synaptic and extrasynaptic GABAA receptors, for the treatment of postpartum depression. Its product pipeline also comprises SAGE-324, a compound that is in Phase II clinical trial to treat essential tremors, as well as has completed Phase I clinical trial for epilepsy and Parkinson's diseases; and SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor, which is in Phase II clinical trial for the treatment of depression, Huntington's disease, Parkinson's diseases, Alzheimer's disease, attention deficit hyperactivity disorder, schizophrenia, and neuropathic pain. The company has a strategic collaboration with Shionogi & Co., Ltd. for the development and commercialization of zuranolone in Japan, Taiwan, and South Korea; and a collaboration and license agreement with Biogen MA Inc. to jointly develop and commercialize SAGE-217 and SAGE-324 products. The company was formerly known as Sterogen Biopharma, Inc. and changed its name to Sage Therapeutics, Inc. in September 2011. Sage Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.
Sarepta Therapeutics
NASDAQ:SRPTSarepta 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.