New eye injection shows promise for rare Blindness-Causing gene mutation

NCT ID NCT05902962

First seen Apr 17, 2026 · Last updated May 12, 2026 · Updated 3 times

Summary

This early-stage study tested a new drug, VP-001, given as an injection into the eye to see if it is safe for people with a rare inherited eye disease caused by a PRPF31 gene mutation. Seventeen adults took part, and the main goal was to check for any side effects. The study did not aim to cure the disease, but to gather safety information for future research.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for RETINAL DYSTROPHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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.