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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

non-arteritic anterior ischemic optic neuropathy open-angle glaucoma optic nerve disorder retinal disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Charleston Neuroscience Institute

    RECRUITING

    Charleston, South Carolina, 29414, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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

    RECRUITING

    New York, New York, 10032, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Global Research Management, Inc.

    RECRUITING

    Glendale, California, 91204, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Mass Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact