Generation Bio

Gene Therapy

7 undisclosed programs RD PKU, Wilson disease, Gaucher disease, Leber congenital amaurosis 10, Stargardt disease, wet AMD, ETAP

Generation Bio is developing a new class of genetic medicines. Using their proprietary non-viral genetic medicine platform, Generation Bio is creating scalable and redosable medicines. The platform uses closed-ended DNA (ceDNA) encased in cell-targeted lipid nanoparticles. ceDNA has 3x the carrying capacity of AAV capsids and has the potential to offer durable expression. The medicines designed by Generation Bio are efficiently manufactured - ceDNA can be consistently produced with high purity using their cell-free rapid enzymatic synthesis approach. Generation Bio is building a portfolio initially targeting diseases of the liver and retina before expanding into skeletal muscle and the CNS.