Gene therapy trial aims to save sight in boys and men with rare retinal disease
NCT ID NCT05878860
First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 2 times
Summary
This study tests a gene therapy called ATSN-201 in males aged 6 and older with X-linked retinoschisis, a genetic eye condition that leads to vision loss. The therapy delivers a working copy of the RS1 gene to the retina. The trial has three parts: first, finding the right dose; second, expanding to more people; and third, a randomized phase to see if it improves vision compared to no treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ATSN-201 gene therapy
- What this could lead to
- If successful, this could become a treatment that improves vision and slows vision loss in people with X-linked retinoschisis.
- What could go wrong
- This is an early-to-mid stage trial with a small number of participants. Gene therapy carries risks like inflammation or immune reactions, and it may not work for everyone.
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.
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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Associated Retina Consultants
NOT_YET_RECRUITINGPhoenix, Arizona, 85020, United States
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Bascom Palmer Eye Institute
RECRUITINGMiami, Florida, 33136, United States
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Boston Children's Hospital
NOT_YET_RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital of Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Columbia University
NOT_YET_RECRUITINGNew York, New York, 100032, United States
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Duke University
NOT_YET_RECRUITINGDurham, North Carolina, 27710, United States
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Flaum Eye Institute, University of Rochester
NOT_YET_RECRUITINGRochester, New York, 14642, United States
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Massachusetts Eye and Ear
RECRUITINGBoston, Massachusetts, 02114, United States
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Mayo Clinic
NOT_YET_RECRUITINGRochester, Minnesota, 55905, United States
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Moran Eye Center
NOT_YET_RECRUITINGSalt Lake City, Utah, 84132, United States
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Oregon Health Sciences University
RECRUITINGPortland, Oregon, 97239, United States
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Retina Foundation of the Southwest
NOT_YET_RECRUITINGDallas, Texas, 75231, United States
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Shiley Eye Institute
NOT_YET_RECRUITINGLa Jolla, California, 92093, United States
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Stanford University
NOT_YET_RECRUITINGPalo Alto, California, 94303, United States
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UC San Francisco
NOT_YET_RECRUITINGSan Francisco, California, 94158, United States
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University of Michigan
NOT_YET_RECRUITINGAnn Arbor, Michigan, 48105, United States
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University of Wisconsin
NOT_YET_RECRUITINGMadison, Wisconsin, 53705, United States
Contact
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