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 ↗Kiniksa Pharmaceuticals International, plc Class A Ordinary Shares
Kiniksa Pharmaceuticals International, plc, a biopharmaceutical company, developing and commercializing novel therapies for diseases with unmet need and focuses on cardiovascular indications worldwide. Its product candidates include ARCALYST, an interleukin-1alpha and interleukin-1beta, for the treatment of recurrent pericarditis, which is an inflammatory cardiovascular disease; Vixarelimab, a monoclonal antibody, that is in Phase 2b clinical trial for the treatment of prurigo nodularis, a chronic inflammatory skin condition; and KPL-387, an investigational, that is in Phase 2/3 clinical trial for human immunoglobulin G2 monoclonal antibody that binds human interleukin-1 receptor 1, inhibiting IL-1a, IL-1ß-mediated signaling for the treatment of advance recurrent pericarditis by providing the added convenience of monthly subcutaneous dosing with a liquid formulation. Its preclinical products include KPL-116, a Fc-modified immunoglobulin G2 monoclonal antibody. The company was formerly known as Kiniksa Pharmaceuticals, Ltd and changed its name tpKiniksa Pharmaceuticals International, plc in June 2024. The company was incorporated in 2015 and is based in London, the United Kingdom.
Open KNSA dashboard ↗