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New research · Ophthalmology
Ophthalmology · 5d
Cohort studyOphthalmology · 2026

Interim Results From a Multicenter Observational Safety Study of Patients Treated With Voretigene Neparvovec-rzyl in the United States.

Aaron Nagiel, Stephen R Russell, Byron L Lam … VN PASS Study Group
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OphthalmologyCohort study

Chorioretinal atrophy developed in over a quarter of eyes after gene therapy injection

Interim Results From a Multicenter Observational Safety Study of Patients Treated With Voretigene Neparvovec-rzyl in the United States.

Aaron Nagiel … VN PASS Study Group
Ophthalmology · 2026
Purpose

To assess long-term safety and exploratory ophthalmic outcomes in patients treated with voretigene neparvovec-rzyl (VN) in US clinical practice.

Methods

Eighty-seven patients (169 eyes) receiving VN at 10 ocular gene therapy treatment centers.

n = 169 eyes
Results

eyes developed patchy thinning/scarring of the retina's back layers, worse than before

RR 5.33 (95% CI 1.27 to 22.40)
null = 11.2722.40
CI excludes the null - significant
More results

With a median 3.7 years of follow-up (range, 2.9-4.9), most patients (95%) reported ≥1 treatment-emergent adverse events (387 events); 67% were procedure related, 9% were attributed to VN, and 82% were mild.

More results

The area of chorioretinal atrophy mostly localized to the retinotomy or bleb area, with 40% of eyes with chorioretinal atrophy having atrophy outside the bleb area and highly correlated between fellow eyes (r=0.97; P<0.0001).

“
Conclusion · 1 of 3

This interim analysis supports the long-term safety of VN in a real-world US cohort, consistent with clinical trial results.

Conclusion · 2 of 3

chorioretinal atrophy did not appear to diminish sustained functional and anatomical benefits with a minimum follow-up of nearly 3 years.

Conclusion · 3 of 3

Ongoing data collection will further clarify the occurrence, impact, and risk factors for chorioretinal atrophy while continuing to monitor VN safety in clinical practice.

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