Gene-Editing breakthrough: single dose may slash sickle cell pain crises
NCT ID NCT05456880
First seen Jan 11, 2026 · Last updated May 24, 2026 · Updated 22 times
Summary
This early-stage trial tests a one-time treatment called BEAM-101 for people with severe sickle cell disease. The therapy uses the patient's own blood stem cells, which are gene-edited to produce healthy red blood cells and reduce painful blockages. The study aims to see if it is safe and can lower the number of severe pain crises. About 15 participants aged 12 to 35 with frequent crises will be enrolled.
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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.
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
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02215, United States
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Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital
Atlanta, Georgia, 30329, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University Irving Medical Center
New York, New York, 10032, United States
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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Henry Ford Cancer Center
Detroit, Michigan, 48202, United States
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Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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St Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203, United States
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The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
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University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
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University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
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University of Miami
Miami, Florida, 33136-1005, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.