Transfusion-dependent thalassemia
Clinical trials for Transfusion-dependent thalassemia explained in plain language.
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Gene therapy aims to free patients from lifelong blood transfusions
Cure Recruiting nowThis study follows people who have already received an experimental gene therapy called BRL-101 for transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. Researchers will monitor participants over the long term to check for safety issues, su…
Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA
Sponsor: Bioray Laboratories • Aim: Cure
Last updated Jul 23, 2026 00:00 UTC
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CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients
Disease control Recruiting nowThis 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 s…
Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC