Real-world experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration

The article, titled “Real-world experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration,” by Birgit Lorenz et al., was published in the journal Ophthalmology in 2023.

The objective of the study was to assess the impact of baseline data on psychophysical and morphological outcomes of subretinal Voretigene Neparvovec (VN) treatment. The study design was a single-center, retrospective, longitudinal, consecutive case series. Participants included patients with RPE65-IRD who were treated with VN and oral immunosuppression according to the manufacturer’s recommendation.

The main outcome measures were best-corrected visual acuity (BCVA), low-luminance VA (LLVA), Goldmann Visual Fields (GVF), chromatic full-field-stimulus-threshold testing (FST), scotopic and photopic 2-Color-Threshold Perimetry (2CTP), and multimodal retinal imaging.

The results showed that VN therapy was effective, with stable median BCVA and mean retinal thickness, and improvements in LLVA, FST, and 2CTP up to 32 months.

Treatment effects were superior in the pediatric group. However, new chorioretinal atrophy was observed in 50% of the treated eyes. The study concluded that VN therapy is effective in real-world settings but may be associated with the development of chorioretinal atrophy in some cases (Lorenz et al., Ophthalmology, 2023).

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Retina Ward of Farabi Eye Hospital