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Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

Primary:To assess the effect of bilateral treatment with AAV5-hRKp.RPGR on functional vision as measured by vision-guided mobility assessment.Secondary:To assess changes after treatment administration in retinal function, functional vision, visual…

A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss

Primary • To evaluate the efficacy of QR-421aSecondary• To evaluate the safety and tolerability of QR-421a • To evaluate changes in Patient-Reported Outcome (PRO) measures in subjects treated with QR-421a• To evaluate systemic exposure of QR-421a