Can a single eye injection restore sight in genetic blindness?

NCT ID NCT05203939

Ongoing ⭐️ CURE ⭐️ Sponsor: Ocugen Source: ClinicalTrials.gov ↗

First seen Jul 31, 2026 · Last updated Jul 31, 2026

Summary

This trial tests an experimental gene therapy called OCU400 in people with retinitis pigmentosa or Leber congenital amaurosis, which are inherited conditions that cause progressive vision loss and can lead to blindness. The therapy is given as a single injection into the eye, with the goal of replacing or repairing faulty genes to slow or stop the disease. The study includes adults with specific genetic mutations and will monitor safety and any changes in vision over time.

What this could mean

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

Active substance: OCU400, an experimental gene therapy delivered by a single injection into the eye
What this could lead to: If successful, OCU400 could provide a one-time treatment that slows or stops vision loss in people with certain inherited retinal diseases, potentially preserving sight.
What could go wrong: This is an early-stage trial with a small number of participants, so safety and effectiveness are not yet proven. The injection itself carries risks like infection or retinal damage, and the therapy 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.

congenital stationary night blindness autosomal dominant 1 enhanced S-cone syndrome Leber congenital amaurosis Leber congenital amaurosis 10 Meckel Syndrome, Type 4 retinitis pigmentosa retinitis pigmentosa 37 retinitis pigmentosa 4

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

LEBER CONGENITAL AMAUROSIS RETINITIS PIGMENTOSA

Contacts and locations

Locations

Associated Retina Consultants

Phoenix, Arizona, 85020, United States
Ocugen Site 1 - Retina Foundation of the Southwest

Dallas, Texas, 75231, United States
Ocugen Site 2 - Casey Eye Institute - OHSU

Portland, Oregon, 97239, United States
Ocugen Site 3 - Bascom Palmer Eye Institute

Miami, Florida, 33136, United States
Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute

La Jolla, California, 92093, United States
Ocugen Site 6 - Emory University

Atlanta, Georgia, 30322, United States
Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital

Philadelphia, Pennsylvania, 19107, United States

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