Gene therapy injection aims to save sight in rare eye disease
NCT ID NCT07439887
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.
Disclaimer
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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.
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.
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
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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RayTx Clinical Site
RECRUITINGBakersfield, California, 93309, United States
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RayTx Clinical Site
RECRUITINGBellaire, Texas, 77401, United States
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UPMC Vision Institute
RECRUITINGPittsburgh, Pennsylvania, 15219, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••