Testing two doses of a new gene therapy for inherited vision loss

This trial is testing a new gene therapy called GS010 to see if it can help restore vision in people with ND4 Leber hereditary optic neuropathy, a rare inherited eye condition that causes progressive vision loss. Researchers will give some patients a higher dose of the medication and others a lower dose, delivered through injections directly into both eyes. The goal is to find out whether this treatment is safe and whether it can improve vision and help eye cells work better.

ND4 Leber hereditary optic neuropathy is caused by a genetic mutation that damages the energy-producing structures in eye cells, leading to vision loss that currently has no proven cure. This trial is testing whether the new treatment can work by delivering a healthy copy of the gene to restore normal function in those cells.