New eye drug shows promise for rare Blindness-Causing mutation

NCT ID NCT05902962

First seen Apr 17, 2026 · Last updated Jun 16, 2026 · Updated 8 times

Summary

This early-stage study tested a new drug called VP-001, given as an injection into the eye, in 17 adults with a rare inherited eye disease caused by a PRPF31 gene mutation. The main goal was to check if the drug is safe and tolerable. The study is complete, and results will help guide future research into treatments for this form of retinal dystrophy.

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Contacts and locations

Locations

  • Bascom Palmer Eye Institute University of Miami

    Miami, Florida, 33136, United States

  • Baylor College of Medicine- Alkek Eye Center

    Houston, Texas, 77030, United States

  • Oregon Health and Science University - Casey Eye Institute

    Portland, Oregon, 97239, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75321, United States

  • University of Florida Health

    Jacksonville, Florida, 32209, United States

  • University of Michigan Kellogg Eye Center

    Ann Arbor, Michigan, 48105, United States

Conditions

Explore the condition pages connected to this study.