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Regulus Therapeutics Inc is a biotechnology company focused on the development of microRNA therapies. MicroRNA therapies target diseases such as cancer, metabolic diseases, fibrosis, and inflammatory diseases. The product candidate of the company is RGLS8429, an anti-miR next-generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), and is in Phase 1b clinical development.
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.