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Gene therapy for wet age-related macular degeneration

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

PHASE3 · AbbVie · NCT05407636

This study is testing a one-time gene therapy for people with wet age-related macular degeneration to see if it can improve their vision better than the usual treatment that requires regular injections.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment714 (estimated)
Ages50 Years to 89 Years
SexAll
SponsorAbbVie (industry)
Locations181 sites (Phoenix, Arizona and 180 other locations)
Trial IDNCT05407636 on ClinicalTrials.gov

What this trial studies

This Phase 3 clinical study evaluates the efficacy and safety of ABBV-RGX-314, a one-time gene therapy, in treating neovascular age-related macular degeneration (nAMD). The trial involves approximately 660 participants who will be randomized into three groups to receive either two different doses of RGX-314 or the standard treatment, aflibercept. The primary goal is to assess the change in best-corrected visual acuity (BCVA) in participants receiving the gene therapy compared to those receiving the active comparator. This approach aims to reduce the need for frequent injections currently required for standard treatments.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 50 to 89 with a diagnosis of nAMD who have previously responded to anti-VEGF therapy.

Not a fit: Patients with conditions affecting vision improvement in the study eye or those with other causes of choroidal neovascularization will not benefit from this study.

Why it matters

Potential benefit: If successful, this gene therapy could significantly reduce the treatment burden for patients with wet AMD by potentially eliminating the need for regular injections.

How similar studies have performed: Other studies have shown promise with gene therapy approaches for retinal diseases, indicating potential for success in this novel treatment.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

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 choroidal neovascularization (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

Inclusion Criteria (Bilateral Treatment Substudy)\*:

1. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
2. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
3. Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes
4. Willing and able to provide written, signed informed consent for this study
5. Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study

Exclusion Criteria:

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. Advanced glaucoma or history of secondary glaucoma in the study eye
5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
6. History of intraocular surgery in the study eye within 12 weeks prior to randomization
7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6
8. Prior treatment with gene therapy

Exclusion Criteria (Bilateral Treatment Substudy)\*:

1. CNV or macular edema in either eye secondary to any causes other than AMD
2. Subfoveal fibrosis or atrophy in either eye
3. Any condition in the investigator's opinion that could limit VA improvement in either eye
4. Advanced glaucoma or history of secondary glaucoma in either eye
5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
6. History of intraocular surgery in either eye within 12 weeks prior to randomization
7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6.
8. Prior treatment with gene therapy (\*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.

Note: Other inclusion/exclusion criteria apply

Where this trial is running

Phoenix, Arizona and 180 other locations

+131 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, WetAMD, CNV, Age-Related Macular Degeneration, Neovascular Age-Related Macular Degeneration

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.