In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD.

Age-related macular degeneration (AMD) is a major cause of blindness and visual impairment in older adults. The wet form of AMD, also called neovascular AMD (nAMD) usually causes faster vision loss than the dry form. The most common current treatments of nAMD are products that inhibit vascular endothelial growth factor (VEGF) (including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron) and are delivered by intravitreal injections at 4 to 16 week intervals and continued indefinitely. This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of EXG102-031 gene therapy in subjects with previously treated nAMD. Safety will be assessed over 52 weeks after the administration of EXG102-031.

Inclusion Criteria:

1. Male or female, age ≥ 50 years of age;
2. Diagnosis of nAMD and current active lesion in the study eye at Screening;
3. An ETDRS BCVA letter scores between 73 and 19 letters in the study eye;
4. Response to anti-VEGF treatment during Screening;
5. The study eye must be a pseudophakic lens (post-cataract surgery status); and
6. Voluntarily agree to participate in the clinical trial, understand the trial procedures, and be capable of signing the informed consent form before screening.

Exclusion Criteria:

1. Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye;
2. Presence in the study eye of CNV or macular edema due to causes other than AMD;
3. Presence in the study eye of scarring, fibrosis or atrophy involving the macula;
4. Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters;
5. Active ocular infection in either eye;
6. Presence of advanced glaucoma or uncontrolled glaucoma in the study eye;
7. History of intraocular surgery in the study eye within 90 days of screening; or
8. Prior receipt of any ocular or systemic gene therapy agent.