Clinical Research Directory

Inclusion Criteria: * Male or females between 8 and 50 years of age at the time of signing theinformed consent form. * Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent. * Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations. * Ability to perform tests of visual and retinal function. * Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected. * Acceptable hematology, clinical chemistry, and urine laboratory parameters. Exclusion Criteria: * OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye. * Presence of epiretinal membrane by OCT. * Complicating systemic diseases or clinically significant abnormal baselinelaboratory values. * Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function. * Prior ocular surgery within six months. * Prior gene therapy or oligonucleotide therapy treatments. * Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.