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

Inclusion Criteria:

• Male or females between 6 and 50 years of age at the time of signing the informedconsent form.

• Willing to adhere to protocol as evidenced by written informed consent or parentalpermission and subject assent.

• Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) and moleculardiagnosis 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 beinjected.

• Acceptable hematology, clinical chemistry, and urine laboratory parameters.

Exclusion Criteria:

• Pre-existing eye conditions that would preclude the planned surgery or interferewith interpretation of study endpoints or complications of surgery (e.g., glaucomarequiring upcoming surgery, corneal or significant lenticular opacities).

• Presence of epiretinal membrane by OCT.

• Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.

• Complicating systemic diseases would include those in which the disease itself, orthe 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 cannotcomply with the protocol requirements or that may place the participant at anunacceptable risk for participation.