Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07161544

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a gene therapy called AAVB-039 for Stargardt disease, an inherited condition that causes vision loss. About 75 adults with a specific gene mutation will receive a single injection under the retina. The main goal is to check safety, but researchers will also measure whether it slows retinal damage and preserves vision.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

AAVB-039 (a gene therapy given as a single injection under the retina)

What this could lead to

If it works, this could slow or stop vision loss in people with Stargardt disease, potentially preserving sight for years.

What could go wrong

This is an early (Phase 1/2) trial with only 75 participants, so results may not apply to everyone. Gene therapy under the retina carries risks like inflammation or retinal damage.

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.

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Conditions

The condition(s) this trial relates to.

Stargardt disease

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

  • Associated Retina Consultants

    RECRUITING

    Phoenix, Arizona, 85020, United States

    Contact

    Contact Email: •••••@•••••

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

    Contact

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

  • Moorfields Eye Hospital NHS Foundation Trust

    RECRUITING

    London, England, EC1V 2PD, United Kingdom

  • Retina Consultants of Texas

    RECRUITING

    Bellaire, Texas, 77401, United States

    Contact

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

    Contact

    Contact Email: •••••@•••••

  • Retina Vitreous Associates Medical Group

    RECRUITING

    Los Angeles, California, 90211, United States

    Contact

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

  • The Retina Clinic

    RECRUITING

    London, England, W1G 9AX, United Kingdom

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

    Contact Email: •••••@•••••

    Contact