Highlight
• Voretigene neparvovec-rzyl (VN) demonstrated a favorable long-term safety profile in a large US cohort with inherited retinal dystrophies.
• Procedure-related adverse events were common but mostly mild, with chorioretinal atrophy (CRA) occurring in about 28% of patients, typically near treatment sites.
• Sustained improvements in best-corrected visual acuity (BCVA) and full-field stimulus threshold (FST) were observed at 3 years, irrespective of CRA presence.
• Risk factors for CRA included use of automated foot pedal delivery and myopia, necessitating ongoing monitoring and technique optimization.
