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Gene therapy offers hope for rare blindness

NCT ID NCT03374657

First seen Feb 01, 2026 · Last updated May 24, 2026 · Updated 11 times

Summary

This study tests a new gene therapy, CPK850, for people with a rare inherited eye disease called RLBP1 retinitis pigmentosa, which causes progressive vision loss and trouble seeing in the dark. The treatment is given as a single injection under the retina to deliver a working copy of the faulty gene. The main goals are to check safety and see if it can improve the eye's ability to adjust to darkness. The study involves 12 adults aged 18 to 70 with moderate to severe vision loss.

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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.

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Contacts and locations

Locations

  • Novartis Investigative Site

    Stockholm, SE-112 82, Sweden

Conditions

Explore the condition pages connected to this study.