Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07439887

Recruiting now Disease control Sponsor: Ray Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a single injection of a gene therapy called RTx-021 in the eye of people with Stargardt disease, a genetic condition that causes vision loss. Up to 18 participants will receive different doses to check safety and tolerability. Researchers will monitor side effects and measure vision changes over 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: RTx-021 (optogenetic gene therapy)
What this could lead to: If it works, this could point toward a treatment that helps preserve or improve vision in people with Stargardt disease.
What could go wrong: This is a very early, small trial (18 people) focused on safety, not effectiveness. The gene therapy is new and may not improve vision or could 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.

Stargardt disease

As listed by the trial registrant

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

STARGARDT DISEASE

Contacts and locations

Locations

RayTx Clinical Site
RECRUITING

Bakersfield, California, 93309, United States
RayTx Clinical Site
RECRUITING

Bellaire, Texas, 77401, United States
UPMC Vision Institute
RECRUITING

Pittsburgh, Pennsylvania, 15219, United States

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