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 ↗Solid Biosciences Inc. Common Stock
Solid Biosciences Inc. develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, which is in phase 1/2 for the treatment of Duchenne muscular dystrophy. It also develops SGT-212 for the treatment of Friedreich's ataxia that is in phase 1b clinical trial; SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia that is in preclinical phase; SGT-601 the treatment of TNNT2-mediated dilated cardiomyopathy, which is in preclinical phase; SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy that is in preclinical phase; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, which is in discovery phase; and other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries; genetic regulators, such as promoters, UTRs, and introns; immunomodulation technologies; manufacturing purity; and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc.; and a license agreement with the University of Washington, University of Missouri, University of Florida, and ICS Maugeri S.p.A. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.
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