Gene therapy targets second eye in rare blindness disorder
NCT ID NCT06646289
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and vision changes in the other eye. The goal is to see if treating both eyes is safe and may help preserve or improve sight.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
botaretigene sparoparvovec (gene therapy)
What this could lead to
If successful, this could improve vision in the second eye for people with X-linked retinitis pigmentosa, a genetic cause of blindness.
What could go wrong
This is a small, early-phase study (24 participants) focused on safety, so it may not lead to a proven treatment. Gene therapy carries risks like inflammation or vision changes.
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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Locations
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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
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University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States