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

NCT ID NCT06455826

First seen Apr 16, 2026 · Last updated Jun 16, 2026 · Updated 14 times

Summary

This early-stage study tested the safety of a new drug called VP-001, given as an injection into the eye, for people with a rare genetic eye disease (PRPF31 mutation) that causes vision loss. Six participants aged 12 and older received multiple doses. The main goal was to check for side effects, not to measure vision improvement.

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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.

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Contacts and locations

Locations

  • 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

The condition(s) this trial relates to.

eye disorder inherited retinal dystrophy retinal degeneration retinal disorder retinitis pigmentosa 11

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.