Gene therapy breakthrough? early trial for blood disorder shows promise
NCT ID NCT06291961
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tested a single dose of a gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body make more healthy hemoglobin. Participants were monitored for side effects and blood improvements over several months.
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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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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
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Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
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Ruijin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, China
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China