Design Therapeutics, Inc. Common Stock
Design Therapeutics, Inc. a clinical-stage biopharmaceutical company, research, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.
Open DSGN 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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