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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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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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Mismatched donor stem cell transplant could widen treatment access for sickle cell disease
- Old drugs, new hope: could a thalidomide combo free thalassemia patients from transfusions?
- Talking therapy could help thalassemia patients take their meds
- New stem cell transplant approach shows promise for blood disorders
- Simple blood test could transform iron monitoring in thalassemia