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Generation Bio Co is a genetics medicine company that is focused on creating gene therapies to provide durable and redosable treatments for patients suffering from rare and untreated diseases. The company focuses on liver and retina-related diseases. It is developing two distinct and complementary platforms that It believes will enable highly differentiated therapeutic applications. Its first platform is a potent, highly selective ctLNP delivery system for nucleic acids, which is designed to avoid off-target clearance by the liver and spleen, enabling ctLNPs to persist in systemic circulation and allowing for highly selective and potent ligand-driven targeting to specific tissues and cell types. Its second platform is its novel iqDNA, which is an optimized variant of its ceDNA.
Cardiol Therapeutics Inc is a clinical-stage life sciences company focused on the research and clinical development of anti-inflammatory and anti-fibrotic therapies for the treatment of heart diseases. The company's lead product candidate, CardiolRx, is a pharmaceutically manufactured oral cannabidiol formulation that is being clinically developed for use in heart diseases. Cardiol has received Investigational New Drug Application authorization from the United States Food and Drug Administration to conduct clinical studies to evaluate the efficacy and safety of CardiolRx in two diseases affecting the heart which are a Phase II multi-national trial, the ARCHER trial, in acute myocarditis; and a Phase II multi-center open-label pilot study in recurrent pericarditis.