Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy

• RGX-314 (Genetic): AAV8 vector containing a transgene for anti-VEGF Fab

Primary outcomes

• Incidence of ocular adverse events and any serious adverse events in the study eye [5 years inclusive of parent study]
• Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye [54 weeks]

Secondary outcomes

• Change from baseline in best corrected visual acuity (BCVA) in the study eye [5 years inclusive of parent study]: Mean change and proportion of participants losing greater than or equal to 5, 10, and 15 letters
• Change from baseline in Central Retinal Thickness (CRT) in the study eye [5 years inclusive of parent study]: Mean change from baseline in CRT as measured by spectral domain optical coherence tomography (SD-OCT)
• Number of supplemental anti-VEGF injections in the study eye [5 years inclusive of parent study]: Mean number of supplemental anti-VEGF injections based on chart review
• Number of retinal specialist visits for the study eye [5 years inclusive of parent study]: Mean number of retinal specialist visits attended for nAMD based on chart review
• Fellow eye substudy: Change from baseline in best corrected visual acuity [54 weeks]: Mean change from baseline in BCVA in the fellow eye
• Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye [54 weeks]: Mean change from baseline in CRT in the fellow eye as measured by SD-OCT
• Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye [54 weeks]: Mean number of supplemental anti-VEGF injections in the fellow eye
• Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye [54 weeks]: Fellow eye aqueous and serum RGX-314 TP concentrations at assessed time points
• Fellow eye substudy: Immunogenicity measurements in the fellow eye [54 weeks]: Fellow eye immunogenicity measurements at assessed timepoints

Sex: All, ages: 50 Years to any

Main Observational Study:

Inclusion Criteria:

1. Able and willing to provide written consent
2. Previously enrolled in a clinical study of RGX-314 and received a single subretinal administration of RGX-314

Exclusions Criteria:

1\. None

Fellow Eye Substudy:

Inclusion Criteria

1. Age ≤ 93 years
2. Currently or previously enrolled in the main observational study
3. Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration
4. Active nAMD in the fellow eye
5. BCVA between ≤ 80 and ≥ 20 letters in the fellow eye

Exclusion Criteria:

1. CNV or macular edema in the fellow eye secondary to causes other than nAMD
2. Subfoveal fibrosis or atrophy in the fellow eye
3. Advanced glaucoma or history of secondary glaucoma in the fellow eye or any invasive procedure to treat glaucoma
4. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
5. History of intraocular surgery in the fellow eye within 12 weeks of screening
6. History of intravitreal therapy, such as intravitreal steroid injection or investigational product, other than an intravitreal therapy for nAMD, in the fellow eye within 6 months of screening