GenSight Biologics (Euronext: SIGHT) announced on Wednesday that it has published five-year follow-up results in JAMA Ophthalmology for LUMEVOQ, its investigational gene therapy for Leber Hereditary Optic Neuropathy (LHON) caused by the ND4 mitochondrial gene mutation. Data from the RESTORE study show sustained bilateral visual acuity improvements and a favorable safety profile.

Participants from the RESCUE and REVERSE Phase III trials demonstrated durable gains in Best-Corrected Visual Acuity (BCVA), with LUMEVOQ-treated eyes improving by a mean of +22 ETDRS letters and sham-treated eyes by +20 letters from nadir. Over 66% of patients experienced clinically meaningful vision recovery in at least one eye and 80.6% achieved on-chart vision. Quality-of-life metrics also improved significantly, with a 7-point gain in composite scores from baseline.

Safety data indicate the therapy is well-tolerated, with no severe ocular events or systemic safety concerns reported. LUMEVOQ uses a mitochondrial targeting sequence (MTS) technology to deliver the ND4 gene via an AAV vector, restoring mitochondrial function in affected retinal cells.

RESTORE enrolled 62 participants and is among the largest long-term follow-up studies for rare disease treatments. LUMEVOQ has not yet been registered but represents a promising advance in addressing the debilitating effects of LHON, a rare hereditary condition leading to rapid and irreversible vision loss.