One-Time gene injection aims to rescue sight in rare childhood blindness

NCT ID NCT07681778

Not yet recruiting Disease control Sponsor: Opus Genetics, Inc Source: ClinicalTrials.gov ↗

First seen Jul 02, 2026 · Last updated Jul 16, 2026 · Updated 4 times

Summary

This study tests a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare condition that leads to severe vision loss from a young age. The treatment is given as a single injection under the retina in the worse-seeing eye. The trial includes adults and adolescents and will check safety and whether vision can be improved or stabilized.

What this could mean

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

Active substance: OPGx-RDH12 (gene therapy)
What this could lead to: If successful, this could slow or partially reverse vision loss in people with a rare genetic form of blindness.
What could go wrong: This is an early, small trial. The therapy may not improve vision, and risks include inflammation or damage from the injection.

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.

Leber congenital amaurosis Leber congenital amaurosis 5

As listed by the trial registrant

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

LCA5 LEBER CONGENITAL AMAUROSIS LEBER CONGENITAL AMAUROSIS (LCA)

Contacts and locations

Locations

Associated Retina Consultants

Phoenix, Arizona, 85020, United States
Perelman School of Medicine, University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
Retina Consultants of Texas & Retina Group Inc.

Houston, Texas, 77056, United States

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