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.
Adicet Bio secures FDA Fast Track Designation for ADI-001 to treat refractory SLE
Acrivon Therapeutics receives FDA Breakthrough Device designation for ACR-368 OncoSignature assay
Vistagen secures US patent for AV-101 in neuropathic pain treatment
Thermo Fisher Scientific launches international registry for adolescent alopecia areata
hVIVO successfully completes RSV antiviral trial for Shionogi
Valneva secures UK approval for world's first chikungunya vaccine
Alys Pharmaceuticals' IND for ALY-101 approved in US and Canada
NeuroTherapia's Phase 2 Alzheimer's disease trial receives European regulatory approval
NFL Biosciences confirms superior safety profile of NFL-101 in new toxicity study
Avirmax Biopharma completes first cohort of Wet AMD clinical trial
PhaseV collaborates with Alimentiv to address challenges in GI clinical trials
HOPE Therapeutics and NRx Pharmaceuticals select BTIG as financial advisor
Helix unveils clinico-genomic dataset of over 15,000 patients treated with GLP-1 agonists
Roquefort Therapeutics sells Lyramid for USD10.8m equity deal with Pleiades Pharma