Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

Inclusion Criteria:

To be eligible for study entry, subjects must satisfy all the following criteria:

1. Written informed consent.

2. Subjects aged ≥ 6 years.

3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessivehereditary macular dystrophy (Stargardt disease).

4. Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compoundheterozygotes).

5. Poor vision in the study eye.

Exclusion Criteria:

Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:

1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy orimplantation of a medical device in the vitreous cavity or subretinal space.

2. Systemic diseases that would preclude the planned surgery or interfere with theinterpretation of study results.

3. History of intraocular surgery within the previous 6 months.

4. Previous participation in a gene therapy trial.

5. Participation in a clinical trial (investigational drug or medical device) withinthe previous 6 months.

6. Any other eye disease that may affect the outcome of the study (e.g., ocularopacities, advanced cataracts, amblyopia, etc.).