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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.
Vigil Neuroscience Inc is a microglia-focused therapeutics company treating both rare and common neurodegenerative diseases by restoring the vigilance of microglia, the sentinel cells of the brain's immune system. It is utilizing the tools of modern neuroscience drug development across multiple therapeutic modalities to rapidly deliver precision-based therapies to improve the lives of patients and families. Its clinical candidate, iluzanebart, is currently being studied in IGNITE, a Phase 2 PoC trial and the first-ever interventional trial in ALSP patients. The company is also developing VG-3927, an orally available small molecule TREM2 agonist for the treatment of common neurodegenerative diseases associated with microglial dysfunction.