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.
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Mesoblast Limited, together with its subsidiaries, engages in the development of regenerative medicine products in Australia, the United States, Singapore, and Switzerland. The company's proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. It offers Remestemcel-L, which is in Phase III clinical trials for the treatment of systemic inflammatory diseases, including steroid refractory acute graft versus host disease and biologic refractory inflammatory bowel disease, as well as ulcerative colitis and Crohn's disease; and Remestemcel-L, which is in Phase III clinical trials to treat chronic heart failure and chronic low back pain due to degenerative disc disease. The company is also developing MPC-300-IV to treat biologic refractory rheumatoid arthritis diabetic nephropathy. It has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for the treatment or prevention of chronic heart failure; MPC-25-IC for the treatment or prevention of acute myocardial infarction; and Ryoncil for the treatment of pediatric SR-aGVHD; JCR Pharmaceuticals Co. Ltd. to treat wound healing in patients with epidermolysis bullosa and for the treatment of neonatal hypoxic ischemic encephalopathy; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. The company was incorporated in 2004 and is headquartered in Melbourne, Australia.
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