Fulcrum Therapeutics, Inc. Common Stock
Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops small molecules to improve the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its clinical-stage product candidate is pociredir, a fetal hemoglobin inducer, which is in phase 1b clinical trial for the treatment of sickle cell disease and beta-thalassemia. The company also discovers drug targets for potential treatment inherited aplastic anemias, such as of diamond-blackfan anemia, shwachman-diamond syndrome, and fanconi anemia, as well as novel fetal hemoglobin inducers, fibrotic disorders, and cardiomyopathies which is in preclinical trial. It has collaboration and license agreement with MyoKardia, Inc. to identify biological targets that are capable of modulating genes of interest with relevance to cardiomyopathies; and licensce agreement with CAMP4 which includes certain small molecule compounds, composition of matter and method of use patent rights, and know-how to research, develop, manufacture, use, and commercialize; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
Open FULC dashboard ↗Jazz Pharmaceuticals, Inc.
Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products in the United States, Europe, and internationally. The company offers Xywav to treat cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia (IH); Xyrem for the treatment of cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC); Rylaze for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Zepzelca for the treatment of metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy; Ziihera to treat HER2-positive biliary tract cancers; Defitelio to treat severe veno-occlusive disease; and Vyxeos for the treatment of newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab in Phase 3 trial to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Epidiolex in Phase 3 trial to treat LGS, DS, and TSC; Suvecaltamide in Phase 2 trial to treat Parkinson's disease tremor; JZP815, a pan-RAF kinase inhibitor in Phase 1 trial that targets components of the mitogen-activated protein kinase; JZP898, a conditionally activated interferon alpha molecule in Phase 1 trial; JZP441 in Phase 1 trial to treat narcolepsy, IH, and other sleep disorders; JZP324, an extended-release low sodium oxybate formulation in Phase 1 trial for patients with sleep disorders; and JZP150 to treat post-traumatic stress disorder. The company has licensing and collaboration agreements with Redx Pharma plc, Autifony Therapeutics Limited, Zymeworks Inc., Sumitomo Pharma Co., Ltd., and Werewolf Therapeutics, Inc. Jazz Pharmaceuticals plc was founded in 2003 and is headquartered in Dublin, Ireland.
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