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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Supernus Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes products for the treatment of central nervous system (CNS) diseases in the United States. The company offers Qelbree, a non-stimulant indicated for the treatment of attention-deficit hyperactivity disorder (ADHD); GOCOVRI for the treatment of dyskinesia and levodopa/carbidopa in patients with Parkinson's Disease (PD); Oxtellar XR, an extended-release oxcarbazepine product indicated for the monotherapy treatment of partial onset epilepsy seizures; and APOKYN for the acute and intermittent treatment of hypomobility or off episodes in patients with advanced PD. It also provides Trokendi XR, an extended release topiramate product indicated for the treatment of epilepsy, as well as for the prophylaxis of migraine headache; XADAGO for the treatment of levodopa/carbidopa in patients with PD experiencing off episodes; and MYOBLOC, a Type B toxin product indicated for the treatment of cervical dystonia and chronic sialorrhea. In addition, the company develops ONAPGO, which received FDA approval, for the treatment of motor fluctuations in adults with advanced PD; SPN-817, a first-in-class selective acetylcholinesterase inhibitor, which is in Phase 2 clinical trial, for the treatment of epilepsy, partial seizures, Dravet syndrome, and Lennox-Gaustaut syndrome; SPN-820, a small molecule in Phase 2 clinical trial for treating resistant depression; SPN-443, a stimulant in Phase 1 trial for the treatment of ADHD/CNS; and SPN-446, a preclinical product for CNS. It markets and sells its products through pharmaceutical wholesalers, specialty pharmacies, and distributors. It has a development agreement with Navitor Inc. for the conduct of Phase 2 clinical program of SPN-820. Supernus Pharmaceuticals, Inc. was incorporated in 2005 and is based in Rockville, Maryland.
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