Can a single Gene-Editing injection stop a blinding corneal disease?

NCT ID NCT07750678

First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time

Summary

This trial tests a single injection of GEB-101, a gene-editing treatment, directly into the cornea of people with TGFBI-related corneal dystrophy, a hereditary condition that causes progressive vision loss. The study aims to see if the treatment is safe and whether it can slow or stop the disease by correcting the underlying genetic mutation. Participants are adults aged 18-70 with a confirmed TGFBI gene mutation and symptoms of corneal dystrophy. The trial will monitor for side effects, changes in vision, and how long any benefit lasts.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
GEB-101, a gene-editing injection given directly into the cornea
What this could lead to
If successful, this could lead to a one-time treatment that corrects the genetic cause of TGFBI-related corneal dystrophy, potentially preventing vision loss and the need for repeat surgeries.
What could go wrong
This is an early-phase trial with a small number of participants, so safety and effectiveness are not yet proven. Risks include possible eye inflammation, infection, or the treatment not working as intended.

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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

  • Fudan University Eye & ENT Hospital

    Shanghai, Shanghai Municipality, 200031, China

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

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