Generation Bio aims to create genetic medicines with drug-like properties that enable people born with genetic diseases to live long, full lives, utilizing our proprietary closed-ended DNA (ceDNA) technology. ceDNA technology has a unique combination of properties that derive from its ability to translocate from the cytoplasm to the nucleus without a viral capsid. Once in the nucleus, ceDNA forms stable, non-integrating episomes that result in durable, high levels of gene expression.
This capsid-free technology enables repeated dosing and allows us to deliver transgenes of unprecedented size (>20 kb) . The unique closed ended nature of ceDNA allows it to be delivered using multiple formulations and routes of delivery including lipid nanoparticles, polymers, and novel fusion molecules. This enables access to multiple tissues and therapeutic areas. We have so far demonstrated durable expression with ceDNA in liver, eye, brain, and muscle.