Gene therapy breakthrough: could a single infusion free thalassemia patients from lifelong transfusions?
NCT ID NCT05577312
First seen Jan 18, 2026 · Last updated May 10, 2026 · Updated 19 times
Summary
This study tests a one-time gene therapy called BRL-101 for people with transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. The therapy uses the patient's own blood stem cells, modified with CRISPR-Cas9, to help the body produce healthy red blood cells. The goal is to see if a single infusion can safely reduce or eliminate the need for transfusions. The trial involves 39 participants aged 3 to 35.
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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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Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
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Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510510, China
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021, China
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Xiangya Hospital of Central South University
Changsha, Hunan, 510510, China
Conditions
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