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A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber*s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Primary: To evaluate the efficacy of QR-110 administered by intravitreal (IVT) injection.Secondary:1. To evaluate the safety and tolerability of QR-110 administered via IVT injection.2. To evaluate changes in patient-reported outcome (PRO) measures…

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

A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease

To determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)1. To evaluate the safety and tolerability of emixustat compared to placebo2. To…

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability,
and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis
Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration
Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in
Intron 26 (IVS26) of the CEP290 Gene (*LCA10-IVS26*)

Primary-To evaluate the safety and tolerability of a single dose of EDIT-101 when administered to participants with LCA10-IVS26 mutationSecondary-To evaluate different doses of EDIT-101 for subsequent clinical evaluation-To evaluate the efficacy of…