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Gene therapy offers hope for rare blindness

NCT ID NCT06196827

First seen Mar 16, 2026 · Last updated May 23, 2026 · Updated 16 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 treatment uses a harmless virus to deliver a working copy of the RPE65 gene to the retina. The main goals are to check safety and see if it can improve vision in 9 participants aged 6 and older.

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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.