Generation Bio

Generation Bio is a biotechnology company focused on developing redosable genetic medicines for both rare and prevalent diseases. The company is particularly known for its work on hemophilia A and is exploring expansion into skeletal muscle, central nervous system (CNS) disorders, and oncology. A key aim is to create CAR-T cells directly in the body for treating cancer and autoimmune diseases. Generation Bio employs a cell-targeted lipid nanoparticle (ctLNP) delivery system and a redosable immune-quiet iqDNA platform to provide durable gene expression while evading innate immunity. The company also targets hematopoietic stem cells (HSCs) for genetic therapies, including those for sickle cell disease and beta-thalassemia. Generation Bio uses a rapid enzymatic synthesis (RES) manufacturing process and a cell-free manufacturing platform to ensure scalable production. Their modular ctLNP system allows for targeting new tissues and cell types, and their therapies can be dosed and redosed according to patient needs. With a four-week research cycle, Generation Bio aims to rapidly develop drug-like genetic medicines that can provide life-long treatment and improve global access to these therapies.