Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Inclusion Criteria: * Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein); * At least 6 years of age; * Good general health without significant physical examination findings or clinically significant abnormal laboratory results; * Able to perform tests of visual and retinal function; * Visual acuity not better than 20/60 and not worse than hand motion in both the treated eye and the fellow eye; * Visible photoreceptor (outer nuclear) layer on a standard optical coherence tomography (OCT) scan; * Acceptable hematology, clinical chemistry and urine laboratory parameters; * For females of childbearing potential, a negative pregnancy test at screening and at baseline, and agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy; * For males of reproductive potential, agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy Exclusion Criteria: * Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g. glaucoma, corneal or lenticular opacities, or history or retinal detachment); * Presence of epiretinal membrane on OCT; * History of immunodeficiency or other medical cond…