CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients
NCT ID NCT05477563
First seen Nov 06, 2025 · Last updated Jun 19, 2026 · Updated 39 times
Summary
This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or severe sickle cell disease. The trial involves 26 participants and includes a conditioning step with chemotherapy before the stem cells are infused.
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This is a summary of
the original study
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITINGRome, Italy
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King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITINGAl Mathar Ash Shamali, Saudi Arabia
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Levine Children's Hospital - Hematology
RECRUITINGCharlotte, North Carolina, 28203, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITINGNashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITINGDüsseldorf, Germany
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
CRISPR-edited stem cells (CTX001, also called exagamglogene autotemcel)
What this could lead to
If it works, this could free people with severe sickle cell disease or beta-thalassemia from needing regular blood transfusions or lifelong medication.
What could go wrong
This is a small, early-phase 3 study with only 26 participants. The treatment requires strong chemotherapy before the infusion, which can cause serious side effects. Long-term safety and effectiveness are still unknown.
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.