One-Time gene injection aims to restore sight in rare eye disease
NCT ID NCT07054632
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests a gene therapy called LX101 in 30 people with inherited retinal dystrophy caused by RPE65 mutations. Participants receive a single injection of LX101 into the eye, while a control group gets no treatment. The study measures changes in functional vision and light sensitivity over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
LX101 (a gene therapy that delivers a working RPE65 gene to the retina)
What this could lead to
If successful, this could provide a one-time treatment to improve vision and light sensitivity in people with this rare inherited eye disease.
What could go wrong
This is a small, early-phase 3 trial with only 30 participants, so results may not apply to everyone. Gene therapy carries risks like inflammation or retinal damage, and long-term effects are unknown.
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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Shanghai General Hospital
Shanghai, China
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Southwest Hospital of AMU
Chongqing, China
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Tianjin Medical University Ophthalmology Hospital
Tianjin, China
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Zhongshan Ophthalmic Center, Sun Yat sen University
Guangzhou, Guangdong, China
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