for people ages 50-89 (full criteria)
at La Jolla, California and other locations
study started
completion around



RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD.

Official Title

A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD


This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 gene therapy relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to aflibercept. Approximately 465 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.


AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, WetAMD, CNV, Age-Related Macular Degeneration, Neovascular Age-Related Macular Degeneration, Macular Degeneration, Wet Macular Degeneration, Choroidal Neovascularization, Retinal Degeneration, Retinal Diseases, Eye Diseases, Ranibizumab, Aflibercept, Angiogenesis Inhibitors, Angiogenesis Modulating Agents, Growth Substances, Physiological Effects of Drugs, Growth Inhibitors, Antineoplastic Agents, Gene Therapy, Anti-vascular endothelial grown factory therapy, Anti-VEGF therapy, RGX-314 Dose 1, RGX-314 Dose 2, Aflibercept (EYLEA®)


You can join if…

Open to people ages 50-89

  1. Age ≥ 50 years and ≤ 89 years
  2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
  3. Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
  4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
  5. Willing and able to provide written, signed informed consent for this study
  6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry

You CAN'T join if...

  1. CNV or macular edema in the study eye secondary to any causes other than AMD
  2. Subfoveal fibrosis or atrophy in the study eye
  3. Any condition in the investigator's opinion that could limit VA improvement in the study eye
  4. Active or history of retinal detachment or current retinal tear in the study eye
  5. Advanced glaucoma or history of secondary glaucoma in the study eye
  6. Myocardial infarction, cerebrovascular accident, or transient ischemic attach within the past 6 months.
  7. History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
  8. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1.
  9. Prior treatment with gene therapy.


  • UC San Diego accepting new patients
    La Jolla California 92093 United States
  • Retina Consultants of San Diego accepting new patients
    Poway California 92064 United States


accepting new patients
Start Date
Completion Date
Phase 3 research study
Study Type
Expecting 465 study participants
Last Updated