Gene therapy offers hope for rare eye disease

NCT ID NCT06196827

First seen Mar 16, 2026 · Last updated May 16, 2026 · Updated 14 times

Summary

This early-stage study tests a gene therapy called LX101 for people with a rare inherited eye disease that causes vision loss. The therapy uses a harmless virus to deliver a working copy of the RPE65 gene to the retina. Nine participants aged 6 and older will be monitored for safety and any improvement in vision.

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Contacts and locations

Locations

  • Beijing Tongren Hospital, Capital Medical University

    Beijing, China

  • Shanghai General Hospital

    Shanghai, China

Conditions

Explore the condition pages connected to this study.