Can a gene therapy reverse aging in the eye? new trial begins
NCT ID NCT07290244
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial is testing a single dose of ER-100, a gene therapy designed to reverse age-related changes in retinal cells, in 18 adults with open-angle glaucoma or NAION. Participants receive the therapy via an eye injection and take doxycycline for 8 weeks to activate it. The main goal is to check safety and side effects, not yet to measure vision improvement. Researchers will follow participants for up to 5 years.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ER-100 (a gene therapy that delivers instructions to produce OCT4, SOX2, and KLF4 proteins, activated by doxycycline)
- What this could lead to
- If safe, this could pave the way for a treatment that reverses age-related damage in optic nerve cells, potentially slowing vision loss in glaucoma and NAION.
- What could go wrong
- This is a very early Phase 1 trial with only 18 participants, so safety and effectiveness are not yet known. The therapy involves a virus-based gene therapy and requires 8 weeks of doxycycline, which may cause side effects.
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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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2026
- Expected to finish
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Mar 2032
An estimate. End dates often move.
- 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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40 to 85 years
- 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: * Have clear eye structures and be able to have your pupils safely dilated so the doctor can examine the back of your eye. * Able to understand the study and sign a consent form. * Be between 40 and 85 years old. * Willing and able to follow the study schedule, including all visits and tests, and speak a language for which the study materials are available. * If a participant can become pregnant, must agree to use a condom and one highly effective form of birth control during sex for at least 4 months after receiving the study drug (ER-100). For participants with open-angle glaucoma (OAG): * Diagnosis of open-angle glaucoma in the study eye. * Eye pressure must be less than 30 mmHg, measured with a standard test. * Visual field test must show moderate to advanced vision loss (MD score between -6 and -20 dB). * Not expected to need glaucoma surgery in the study eye within 2 months after receiving ER-100. * Have reasonably good vision in the study eye (at least 20/80 on a standard eye chart). For participants with NAION (non-arteritic anterior ischemic optic neuropathy): * Had a sudden, painless loss of vision in one eye within 14 days before receiving ER-100, confirmed by a specialist. Having had NAION in the other eye is okay. * The affected eye must show swelling of the optic nerve. * Visual field test must show vision loss consistent with optic nerve damage (MD worse than -3.0 dB). * If only one eye is affected, there must be a difference in pupil response between the two eyes. * Have vision in the affected eye between 20/40 and 2/500 on a standard eye chart. Exclusion Criteria: * History of optic neuritis (inflammation of the optic nerve) or repeated episodes of eye inflammation (uveitis) not caused by injury or surgery. * Allergic reactions to tetracycline antibiotics or steroid medications. * Moderate to severe cataracts, macular problems, or corneal issues that could interfere with eye testing. * Unable to keep your eyes focused on a target during testing. * Had cataract surgery or other eye surgery (including laser procedures) within 3 months before receiving the study drug. * Had cancer (except for basal cell skin cancer) within the past 5 years. * Have Type 1 diabetes, or poorly controlled Type 2 diabetes (A1c greater than 7 despite treatment). * Have memory or thinking problems that prevent you from understanding the study or completing the required tests. * Pregnant or breastfeeding. * Have a weakened immune system, including a history of organ transplant, or test positive for HIV, hepatitis B or C, or tuberculosis. * Have any other condition that, in the opinion of the study doctor, could increase your risk from the study drug or procedures, affect the study results, or make it hard for you to complete the study. * Have macular disease, advanced diabetic eye disease, or other eye conditions that limit vision in the study eye. * Eye pressure at screening is 30 mmHg or higher. * Taking certain medications (warfarin, dilantin, carbamazepine, or barbiturates) within 14 days before starting the study or during the first 8 weeks. * Have any other eye or vision problem that, in the opinion of the study doctor, could affect safety or interfere with vision testing. * Have previously received any gene therapy using adeno-associated virus (AAV). Additional Exclusion Criteria for Participants with Open-Angle Glaucoma (OAG): \- Diagnosed with glaucoma before age 40. Additional Exclusion Criteria for Participants with NAION: * Show signs of giant cell arteritis (a type of blood vessel inflammation), based on abnormal blood tests. * Had NAION start in both eyes at the same time.
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Genom att skicka in godkänner du våra Användarvillkor
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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Charleston Neuroscience Institute
RECRUITINGCharleston, South Carolina, 29414, United States
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Columbia University Irving Medical Center/Edward S. Harkness Eye Institute
RECRUITINGNew York, New York, 10032, United States
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Global Research Management, Inc.
RECRUITINGGlendale, California, 91204, United States
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Mass Eye and Ear
RECRUITINGBoston, Massachusetts, 02114, United States
Contact Email: •••••@•••••
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