Gene therapy injection aims to halt blinding eye disease
NCT ID NCT06467344
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early study tests a new gene therapy called ACDN-01 for people with Stargardt disease, a genetic condition that leads to progressive vision loss. The therapy is given as a one-time injection under the retina. The main goals are to check safety and find the right dose, with a first look at whether it might help preserve sight.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ACDN-01 (a gene therapy given as a one-time injection under the retina)
What this could lead to
If it works, this could slow or stop vision loss in people with Stargardt disease and related retinal conditions.
What could go wrong
This is a very early, first-in-human trial with only 15 participants. It primarily tests safety, not effectiveness. Gene therapies can cause inflammation or other side effects.
Disclaimer
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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.
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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Bascom Palmer Eye Institute
RECRUITINGMiami, Florida, 33136, United States
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Casey Eye Institute OHSU
RECRUITINGPortland, Oregon, 97239, United States
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Cincinnati Eye Institute
RECRUITINGCincinnati, Ohio, 45245, United States
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Massachusetts Eye and Ear
RECRUITINGBoston, Massachusetts, 02114, United States
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Retina Consultants of Texas
RECRUITINGHouston, Texas, 77401, United States
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Retina Foundation of the Southwest
RECRUITINGDallas, Texas, 75382, United States
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University of Michigan Kellogg Eye Center
RECRUITINGAnn Arbor, Michigan, 48105, United States
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University of San Francisco
RECRUITINGSan Francisco, California, 94158, United States
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Vitreo Retinal Associates
RECRUITINGGainesville, Florida, 32607, United States
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Wilmer Eye Institute at John Hopkins
RECRUITINGBaltimore, Maryland, 21218, United States