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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…

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…