Document Title: Integrated Summary of Safety (ISS).

This Integrated Summary of Safety provides a comprehensive analysis of safety data pooled from the 41 subjects who received at least one dose of voretigene neparvovec across the Phase 1 (Studies 101/102) and Phase 3 (Study 301) trials. The analysis covers demographics, exposure, adverse events (AEs), serious adverse events (SAEs), and clinical laboratory data. The data show that 66% of subjects experienced ocular AEs, primarily related to the subretinal injection procedure. The most frequent were conjunctival hyperemia (22%), cataract (20%), and increased intraocular pressure (15%). A total of 8 SAEs occurred in 7 subjects, with 2 ocular SAEs (endophthalmitis; retinal atrophy progression) considered related to treatment. This integrated analysis confirms the safety profile observed in individual studies and provides a robust characterization of the risks associated with treatment.