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.
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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
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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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