Study ID: AAV2-RPE65-MOUSE-PD-002
Study Title: Evaluation of Retinal Morphology Following Subretinal Administration of Voretigene Neparvovec in the rd12 Mouse Model.

Study Report Body:
Objective: To assess the effect of voretigene neparvovec on preserving retinal structure in the rd12 mouse model of RPE65-mediated retinal degeneration.
Methods: rd12 mice received a single unilateral subretinal injection of voretigene neparvovec. Animals were sacrificed at 3, 6, and 12 months post-injection. Retinal structure was assessed via histology and measurement of the outer nuclear layer (ONL) thickness, which contains the photoreceptor cell bodies.
Results: In untreated control eyes, a progressive loss of photoreceptor cells and thinning of the ONL was observed over 12 months, consistent with the known disease progression. In contrast, eyes treated with voretigene neparvovec showed significant preservation of ONL thickness at all time points compared to controls. The treated retinas maintained a more organized photoreceptor layer structure.
Conclusion: Early intervention with voretigene neparvovec can slow or prevent photoreceptor cell death in a relevant animal model, suggesting a potential disease-modifying effect in addition to vision restoration.