Scientists test First-Ever gene therapy to halt blindness

NCT ID NCT03374657

Summary

This is the first-ever human study of an experimental gene therapy called CPK850 for a rare, inherited form of vision loss called RLBP1 retinitis pigmentosa. The main goals are to find a safe dose and see if a single injection into the eye can help improve patients' vision, especially in low light. About 12 adult patients will be followed for five years after receiving the treatment.

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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.