This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
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).
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.
Sequential
VG801
DrugAdministered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Abnormal examination results will be recorded.
Best Corrected Visual Acuity (BCVA)
Abnormal examination results will be recorded.
Optical coherence tomography (OCT)
Abnormal examination results will be recorded.
Fundus autofluorescence
Abnormal examination results will be recorded.
Microperimetry
Abnormal examination results will be recorded.
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