Gene therapy shot into both eyes aims to save sight in rare blindness
NCT ID NCT07174726
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called laruparetigene zovaparvovec (AGTC-501) in 10 males aged 12-50 with X-linked retinitis pigmentosa, a genetic condition that leads to vision loss. The therapy is injected into both eyes to deliver a working copy of the RPGR gene. The main goal is to check safety, but researchers will also measure if it helps preserve or improve vision.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- laruparetigene zovaparvovec (AGTC-501) gene therapy
- What this could lead to
- If successful, this could slow vision loss or improve sight in people with X-linked retinitis pigmentosa, a genetic cause of blindness.
- What could go wrong
- This is a small, early-phase safety study with only 10 participants. The therapy involves eye surgery and may cause side effects like inflammation or vision changes. It is not yet proven to work.
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
Locations
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Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
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Cincinnati Eye Institute
Cincinnati, Ohio, 45242, United States
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Duke Eye Center
Durham, North Carolina, 27710, United States
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OHSU Casey Eye Institute
Portland, Oregon, 97239, United States
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Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
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University of Florida Jacksonville Ophthalmology
Jacksonville, Florida, 32209, United States