Open Actively Recruiting

Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD


Brief Summary

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.

Primary Purpose
Study Type
Phase 2/Phase 3


Healthy Volunteers
Minimum Age
50 Years
Maximum Age
89 Years

Inclusion Criteria:

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

Exclusion Criteria:

  • CNV or macular edema in the study eye secondary to any causes other than AMD
  • Subfoveal fibrosis or atrophy in the study eye
  • Any condition in the investigator's opinion that could limit VA improvement in the study eye
  • Active or history of retinal detachment in the study eye
  • Uncontrolled glaucoma in the study eye
  • History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
  • 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.
  • Prior treatment with gene therapy.
  • Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.

Join this Trial

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Study Stats
Protocol No.
Eye/Ocular Disorders
Karina Lozano
  • UCLA Pasadena
  • UCLA Westwood
For Providers
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