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Gene-Edited cells could reduce bleeding in severe hemophilia b

NCT ID NCT06611436

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 29 times

Summary

This study tests a single infusion of BE-101, made from a person's own blood cells that are gene-edited to produce clotting factor IX. It aims to safely raise factor levels and reduce bleeding episodes in 24 adults with moderately severe to severe hemophilia B. Participants must currently use preventive factor treatment and have adequate organ function.

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Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • University of California, Davis

    RECRUITING

    Davis, California, 95616, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • Washington Center for Bleeding Disorders

    RECRUITING

    Seattle, Washington, 98101, United States

Conditions

Explore the condition pages connected to this study.