GBIO

Generation Bio Co is a genetics medicine company 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 differentiated therapeutic applications. Its first platform is a potent, 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 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.