Gene therapy injection aims to halt rare blindness

NCT ID NCT07185256

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a gene therapy called OPGx-BEST1 for two rare inherited eye diseases that cause vision loss. About 10 adults will receive a single injection into one eye to see if it is safe and to find the best dose. The study will follow participants for 5 years to monitor side effects and any vision changes.

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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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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cincinnati Eye Institute

    RECRUITING

    Cincinnati, Ohio, 45242, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Vitreo Retinal Associates

    NOT_YET_RECRUITING

    Gainesville, Florida, 34481, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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