Study ID: AAV2-RPE65v2-101/102
Study Title: An Open-Label, Phase 1 Dose-Escalation and Contralateral-Eye Treatment Study of the Safety and Tolerability of Voretigene Neparvovec.

This report describes the first-in-human Phase 1 study. Study 101 was a dose-escalation study in 12 subjects with RPE65-LCA, where three doses were administered to the first eye to determine a safe and biologically active dose. The highest dose (1.5 x 10^11 vg) was found to be well-tolerated and was associated with the most consistent improvements in exploratory efficacy measures. Study 102 was a follow-on study in which 11 of the 12 subjects received the high dose in their contralateral eye. The study's primary objective of safety was met. A serious adverse event of endophthalmitis in one subject led to the discontinuation of prophylactic ocular corticosteroid use in the Phase 3 trial in favor of a systemic regimen. Efficacy assessments, including mobility testing and FST, showed improvements that were durable for at least three years, providing the initial proof-of-concept and supporting evidence for durability of effect.