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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Charleston Neuroscience Institute

    RECRUITING

    Charleston, South Carolina, 29414, United States

  • Columbia University Irving Medical Center/Edward S. Harkness Eye Institute

    RECRUITING

    New York, New York, 10032, United States

  • Global Research Management, Inc.

    RECRUITING

    Glendale, California, 91204, United States

  • Mass Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

    Contact Email: •••••@•••••

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