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:

• Written informed consent.
• Subjects aged ≥ 6 years.
• Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
• Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
• 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:

• Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
• Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
• History of intraocular surgery within the previous 6 months.
• Previous participation in a gene therapy trial.
• Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
• Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).