ceDNA Factor IX

ceDNA Factor IX is an investigational non-viral gene therapy being developed by Generation Bio for the treatment of hemophilia B. The therapy utilizes the company's proprietary closed-ended DNA (ceDNA) technology, which consists of a double-stranded DNA construct with covalently closed ends. This structure is designed to provide stable, episomal gene expression in the liver without the risks associated with viral vector integration or pre-existing viral immunity. The ceDNA is delivered using a cell-targeted lipid nanoparticle (ctLNP) system, which is engineered to specifically target hepatocytes. This approach is intended to allow for higher therapeutic doses and the potential for repeat administration (re-dosing) to maintain or titrate Factor IX levels over time. Preclinical studies have demonstrated the ability of the ceDNA-ctLNP system to achieve functional Factor IX protein expression in animal models and maintain tolerability upon multiple doses.

Other names

closed-ended DNA Factor IXceDNA-ctLNP Factor IX