Breakthrough Gene Therapy Restores Vision in Children with Rare Genetic Blindness
• A groundbreaking gene therapy developed by Moorfields Eye Hospital and MeiraGTx has successfully restored vision in children born with LCA-AIPL1, a severe inherited eye disorder affecting 2-3 in 10 million newborns.
• Clinical trials demonstrated significant vision improvement in all eleven treated children, with some achieving 20/80 vision, enabling them to identify pictures, safely navigate, and engage in visual activities for the first time.
• The treatment must be administered before age four due to irreversible retinal degeneration, with regulatory approval expected within 1-2 years following successful safety and efficacy demonstrations.
LUMEVOQ Gene Therapy Shows Sustained Vision Improvement in LHON Patients After 5 Years
• GenSight Biologics' LUMEVOQ demonstrates sustained visual acuity improvement in Leber Hereditary Optic Neuropathy (LHON) patients five years post-treatment.
• Bilateral injections of LUMEVOQ resulted in a higher rate of clinically relevant visual recovery compared to unilateral treatment in the REFLECT Phase III trial.
• The gene therapy maintained a favorable safety profile over the five-year study period, with no serious ocular adverse events reported.
• RESTORE study showed that Lumevoq-treated eyes achieved a mean improvement against nadir of +22 letters equivalent and sham-treated eyes demonstrated a mean improvement of +20 letters equivalent.
Lenadogene Nolparvovec Shows Sustained Vision Improvement in LHON Patients After 5 Years
• A 5-year follow-up study (RESTORE) showed that lenadogene nolparvovec gene therapy provides sustained visual acuity improvement in both eyes of LHON patients with the MT-ND4 mutation.
• The study demonstrated a mean change in BCVA of -0.4 logMAR (more than +4 lines) in treated eyes, similar to sham-treated eyes, indicating a potential bilateral therapeutic effect.
• Clinically relevant recovery, defined as a BCVA improvement of at least -0.3 logMAR (+3 lines), was observed in 66.1% of participants, with improvements in quality of life.
• The gene therapy demonstrated a favorable long-term safety profile, with manageable intraocular inflammation and no permanent complications reported.
Clearside Biomedical Highlights Suprachoroidal Delivery Technology at Global Medical Meetings
• Clearside Biomedical presented its suprachoroidal delivery technology and pipeline program data at medical meetings across Asia, Europe, and the United States.
• ODYSSEY trial topline results for CLS-AX in wet AMD were presented at the Ophthalmology Innovation Summit and the Asia-Pacific Vitreo-Retina Society (APVRS) Congress.
• Clearside hosted suprachoroidal injection procedure demonstrations at APVRS, showcasing the SCS Microinjector® technology and novel delivery approach.
• Arctic Vision is advancing XIPERE towards potential approval in China, Australia, and Singapore, increasing interest in Clearside's delivery approach.
© Copyright 2025. All Rights Reserved by MedPath