SLDB

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 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.

Funcionários

121

Sede

Charlestown, United States

Site oficial

solidbio.com

Descrição da empresa fornecida pelo Yahoo Finance. A descrição é publicada pela fonte apenas em inglês.