Gene-Editing breakthrough? new therapy aims to stop sickle cell pain crises
NCT ID NCT05456880
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-stage trial is testing a new treatment called BEAM-101 for people with severe sickle cell disease. The therapy uses a patient's own blood stem cells, which are gene-edited in a lab to produce healthy red blood cells. After a short course of chemotherapy, the edited cells are infused back into the patient. The goal is to reduce or eliminate the painful blockages (vaso-occlusive crises) that are a hallmark of the disease. The study is currently enrolling 15 participants aged 12 to 35.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- BEAM-101 (a gene-edited stem cell therapy made from the patient's own blood cells)
- What this could lead to
- If it works, this could provide a long-term reduction in severe pain crises for people with sickle cell disease, potentially reducing the need for frequent hospital visits.
- What could go wrong
- This is an early, small trial (15 people) testing safety and effectiveness. The treatment requires strong chemotherapy before the infusion, which carries serious risks like infection and infertility. It may not work for everyone.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can Co-Designed support help parents embrace sickle cell care?
- Can community hospitals in zambia master severe disease care?
- A Pocket-Sized coach for sickle cell: can an app turn Pill-Taking into a habit?
- Can a phone app keep teens on track with sickle cell treatment?
- A common amino acid may calm sickle cell pain crises — a trial puts it to the test
- A drug that protects the liver may make stem cell transplants safer for children with sickle cell disease