Gene therapy for rare blindness and deafness condition passes safety check

NCT ID NCT02065011

First seen Jan 21, 2026 · Last updated May 13, 2026 · Updated 20 times

Summary

This study looks at the long-term safety of a gene therapy called SAR421869 in 9 people with Usher syndrome type 1B, a rare genetic disorder that causes vision and hearing loss. Participants received the treatment in an earlier study and are now being followed to see if it remains safe and slows retinal degeneration. The goal is to understand if the therapy can help control the disease over time.

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Contacts and locations

Locations

  • Investigational Site Number : 250001

    Paris, 75012, France

  • Oregon Health and Science University Site Number : 840001

    Portland, Oregon, 97239-3098, United States

Conditions

Explore the condition pages connected to this study.