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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Rallybio Corporation, a clinical-stage biotechnology company, engages in development and commercialization of life-transforming therapies for patients suffering from severe and rare diseases. Its lead product candidate is RLYB212, a monoclonal anti-HPA-1a antibody which is in Phase II clinical trial for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT); and RLYB211 for the prevention of FNAIT. The company is also developing RLYB114, a pegylated complement factor 5 (C5)-targeted Affibody molecule in preclinical development for the treatment of complement-mediated ophthalmic diseases; RLYB116, an inhibitor of complement component 5 (C5) to treat several diseases of complement dysregulation which has completed phase 1 trial; and RLYB332, a preclinical antibody, for the treatment of severe anemia with ineffective erythropoiesis and iron overload. It entered into a strategic alliance with AbCellera to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. Rallybio has collaboration with Exscientia for the development of small molecule therapeutics for rare diseases; and collaboration agreement with Johnson & Johnson to provide pregnant individuals therapeutic solutions at risk of fetal and neonatal alloimmune thrombocytopenia. The company was founded in 2018 and is headquartered in New Haven, Connecticut.
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