Clinical Research Directory

Inclusion Criteria: * Male or females between 6 and 50 years of age at the time of signing the informed consent form. * Willing to adhere to protocol as evidenced by written informed consent or parental permission and subject assent. * Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and molecular diagnosis of LCA due to RPE65 mutations. * Ability to perform tests of visual and retinal function. * Visual acuity of ≤ 20/80 or visual field less than 20 degrees in the eye to be injected. * Acceptable hematology, clinical chemistry, and urine laboratory parameters. 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 requiring upcoming surgery, corneal or significant lenticular opacities). * Presence of epiretinal membrane by OCT. * Complicating systemic diseases or clinically significant abnormal baseline laboratory values. * Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. * Prior ocular surgery within six months. * Prior gene therapy or oligonucleotide therapy treatments. * Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.