Gene therapy for degenerative retinal disease has no safety concerns but fails to improve vision loss

By | June 1, 2022
A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, with even the highest dose. LHON affects the optic nerve, which carries visual signals from the light-sensing retina to the brain.