Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Eligibility 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.
• * 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.