Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07161544

Recruiting now Disease control Sponsor: AAVantgarde Bio Srl Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

STARGARDT DISEASE

Contacts and locations

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Locations

Associated Retina Consultants
RECRUITING

Phoenix, Arizona, 85020, United States
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Moorfields Eye Hospital NHS Foundation Trust
RECRUITING

London, England, EC1V 2PD, United Kingdom
Retina Consultants of Texas
RECRUITING

Bellaire, Texas, 77401, United States
Retina Foundation of the Southwest
RECRUITING

Dallas, Texas, 75231, United States
Retina Vitreous Associates Medical Group
RECRUITING

Los Angeles, California, 90211, United States
The Retina Clinic
RECRUITING

London, England, W1G 9AY, United Kingdom

Contact Email: •••••@•••••
University of Florida Health Science Center
RECRUITING

Jacksonville, Florida, 32209, United States
University of Utah
RECRUITING

Salt Lake City, Utah, 84112, United States

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