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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Insmed Incorporated develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally. The company offers ARIKAYCE for the treatment of refractory nontuberculous mycobacterial lung infections, as well as is in phase 3 clinical trial for the treatment of mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It also devlops brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1(DPP1) that is in phase 3 clinical trial for the treatment of bronchiectasis; and in phase 2 clinical trial for the treatment of chronic rhinosinusitis without nasal polyps and hidradenitis suppurativa. In addition, the company is developing treprostinil palmitil inhalation powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil, which is in phase 3 clinical trial for the treatment of pulmonary hypertension associated with interstitial lung disease; and phase 2 clinical trial for the treatment of pulmonary arterial hypertension. Further, it develops gene therapy, a microdystrophin adeno-associated virus gene replacement therapy which is in phase 1 clinical trial for the treatment of Duchenne muscular dystrophy, as well as is in pre-clinical development for gene therapy, next generation DPP1 inhibitor, deimmunized therapeutic protein, and synthetic rescue. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.
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