Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset [INFOGRAPHIC]

This infographic summarizes the RESCUE Trial on the efficacy and safety of intravitreous gene therapy (rAAV2/2-ND4) for Leber Hereditary Optic Neuropathy (LHON) patients treated within 6 months of vision loss onset. Conducted across seven centers in France, Germany, Italy, the UK, and the USA, the randomized, double-masked, sham-controlled Phase III study evaluated 39 participants aged 15+ with the m.11778G>A mutation.

The primary outcome measured best-corrected visual acuity (BCVA) change over 96 weeks, with secondary outcomes including contrast sensitivity, mean deviation, and quality of life (VFQ-25). Results showed improvements in BCVA by +10 to +9 ETDRS letters in both treated and control groups, though no significant differences were observed between arms.

Limitations included lack of a placebo arm and predominantly male participants. The infographic highlights the importance of understanding natural LHON progression, trial design challenges, and the potential implications of gene therapy for inherited optic neuropathies.

Source: https://www.nature.com/articles/s41433-025-03950-8/figures/1