Gene-Editing breakthrough? new therapy aims to stop sickle cell pain crises

NCT ID NCT05456880

Ongoing Disease control Sponsor: Beam Therapeutics Inc. Source: ClinicalTrials.gov ↗

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.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE

Contacts and locations

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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