Experimental gene therapy aims to restore sight in rare eye disease
NCT ID NCT06196827
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early study tests a gene therapy called LX101 in 9 people with a rare inherited eye disease caused by RPE65 gene mutations. The therapy delivers a working copy of the gene into the retina via a single injection. The main goals are to check safety and see if it can improve vision or light sensitivity.
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 using a harmless virus to deliver a working RPE65 gene into the retina)
- What this could lead to
- If successful, this could point toward a treatment that slows or partially reverses vision loss in people with this rare genetic eye disease.
- What could go wrong
- This is a very early Phase 1 trial with only 9 people, so safety and effectiveness are not yet proven. The therapy involves eye surgery and may not improve vision for everyone.
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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Beijing Tongren Hospital, Capital Medical University
Beijing, China
-
Shanghai General Hospital
Shanghai, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.