Real-world study shows positive outcomes for gene therapy in patients with RPE65-IRD

Treatment with voretigene neparvovec (VN, Luxturna) in patients with RPE65-IRD (retinal pigment epithelium 65-inherited retinal dystrophy) shows promising outcomes, according to a real-world study.

The retrospective study conducted at a single center included 30 eyes from 19 patients, with a median follow-up of 15 months.

The treatment demonstrated stability in median best-corrected visual acuity (BCVA) and mean retinal thickness, as well as significant improvements in various visual parameters, particularly in the pediatric population.

Goldmann visual fields (GVF) demonstrated a marked improvement in pediatric cases, showing a 20% enhancement for target V4e and an impressive 50% increase for target III4e. However, the study identified novel chorioretinal atrophy in 50% of treated eyes, a phenomenon worth further investigation.

Reference
Lorenz B, Künzel SH, Preising MN, et al. Real-world experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration. Ophthalmology. 2023;S0161-6420(23)00631-0. doi: 10.1016/j.ophtha.2023.09.006. Epub ahead of print. PMID: 37704110.