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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Neurocrine Biosciences, Inc. discovers, develops, and markets pharmaceuticals for neurological, neuroendocrine, and neuropsychiatric disorders in the United States and internationally. The company's products include INGREZZA for tardive dyskinesia and chorea associated with Huntington's disease; ALKINDI for adrenal insufficiency; Efmody capsules for classic congenital adrenal hyperplasia; Orilissa tablets for endometriosis; Oriahnn capsules to treat uterine fibroids; and CRENESSITY to treat congenital adrenal hyperplasia. Its product candidates in clinical development include valbenazine to treat dyskinetic cerebral palsy and schizophrenia; NBI-1076986 to treat movement disorders; Osavampator for inadequate response to treatment in major depressive disorder; NBI-1117568 for the treatment of schizophrenia; NBI-1070770 to treat major depressive disorder; and NBI-1117570, NBI-1117567, NBI-1117569, and NBI-1065890 for neuropsychiatric and neurological conditions. The company also has license and collaboration agreements with Nxera Pharma UK Limited; Takeda Pharmaceutical Company Limited; Xenon Pharmaceuticals Inc.; Voyager Therapeutics, Inc.; Mitsubishi Tanabe Pharma Corporation; and AbbVie Inc. The company was incorporated in 1992 and is headquartered in San Diego, California.
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