Gene-Editing breakthrough aims to stop sickle cell pain crises

NCT ID NCT05456880

First seen Jan 11, 2026 · Last updated May 02, 2026 · Updated 18 times

Summary

This study tests a one-time treatment called BEAM-101 for people with severe sickle cell disease. It uses the patient's own blood stem cells, which are gene-edited to produce healthy red blood cells. The goal is to reduce or eliminate painful vaso-occlusive crises. The trial involves 15 participants aged 12 to 35 and monitors safety and effectiveness over time.

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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

  • Boston Children's Hospital

    Boston, Massachusetts, 02215, United States

  • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Henry Ford Cancer Center

    Detroit, Michigan, 48202, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203, United States

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Miami

    Miami, Florida, 33136-1005, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.