About GBIO

Generation Bio Co. develops genetic medicines to provide treatments for rare and prevalent diseases. The Company through its non-viral genetic medicine platform, is engaged in developing genetic medicines to provide genetic payloads, which include large and multiple genes to a range of cell types across a range of diseases. Its platform is comprised of three component technologies: ceDNA, ctLNP and ceDNA manufacturing process. ceDNA is an engineered, double-stranded, linear, covalently closed-ended deoxyribonucleic acid (DNA) construct, which includes the gene of interest and associated regulatory sequences. It has produced ceDNA constructs of approximately eight kilobases. Its ctLNP delivery system builds upon clinically validated lipid nanoparticles and is designed to allow for repeat dosing of a genetic payload without stimulating an immune response, such as antibody production. Its ceDNA manufacturing process uses its own cell-free rapid enzymatic synthesis (RES) to produce ceDNA.