One-Time gene injection could end frequent eye shots for wet AMD
NCT ID NCT05903794
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tested a gene therapy called EXG102-031 in 12 people with wet age-related macular degeneration (nAMD), a condition where abnormal blood vessels damage central vision. The therapy was injected into the eye to block these vessels. The main goal was to check safety and tolerability, not yet to prove it works. If safe, larger studies could explore whether it reduces the need for repeated anti-VEGF injections.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- EXG102-031 (a gene therapy injected into the eye to block abnormal blood vessel growth)
- What this could lead to
- If successful, this could lead to a one-time gene therapy that reduces the need for frequent eye injections in people with wet age-related macular degeneration.
- What could go wrong
- This is a very early Phase 1 trial with only 12 people, focused on safety. It is too small to prove effectiveness, and gene therapy carries risks like inflammation or vision changes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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12 people
The number who actually took part.
- Started
-
Jul 2023
- Finished
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Feb 2026
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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50 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Erie Retina Research
Erie, Pennsylvania, 16507, United States
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Sierra Eye Associates
Reno, Nevada, 89502, United States