Gene therapy trial aims to halt rare eye disease

NCT ID NCT05694598

First seen Jan 07, 2026 · Last updated May 14, 2026 · Updated 20 times

Summary

This early-stage study tests a new gene therapy called VGR-R01 in 12 adults with Bietti Crystalline Dystrophy, a rare inherited eye disease that causes vision loss. The main goal is to check if the treatment is safe and tolerable. Researchers will monitor for side effects and changes in vision over time.

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Contacts and locations

Locations

  • Shanghai Vitalgen Biopharma Co.,Ltd.

    Shanghai, Shanghai Municipality, China

Conditions

Explore the condition pages connected to this study.