Gene therapy injection aims to slow vision loss in stargardt disease

NCT ID NCT06467344

First seen Jan 11, 2026 · Last updated May 15, 2026 · Updated 18 times

Summary

This early-stage study tests a single injection of ACDN-01 into the eye for people with Stargardt disease or cone-rod dystrophy caused by ABCA4 gene mutations. The main goals are to check safety and find the best dose. About 15 adults with advanced vision loss will take part. The therapy is designed to target the underlying genetic problem, but it is not expected to be a cure.

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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

Study contacts

  • Contact

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

Locations

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

  • Casey Eye Institute OHSU

    RECRUITING

    Portland, Oregon, 97239, United States

  • Cincinnati Eye Institute

    RECRUITING

    Cincinnati, Ohio, 45245, United States

  • Massachusetts Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Retina Consultants of Texas

    RECRUITING

    Houston, Texas, 77401, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75382, United States

  • University of Michigan Kellogg Eye Center

    RECRUITING

    Ann Arbor, Michigan, 48105, United States

  • University of San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

  • Vitreo Retinal Associates

    RECRUITING

    Gainesville, Florida, 32607, United States

  • Wilmer Eye Institute at John Hopkins

    RECRUITING

    Baltimore, Maryland, 21218, United States

Conditions

Explore the condition pages connected to this study.