TRANSFUSION-DEPENDENT THALASSEMIA
Clinical trials for TRANSFUSION-DEPENDENT THALASSEMIA explained in plain language.
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 02, 2026 07:00 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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One-Shot gene fix for blood disorder enters human testing
Disease control ENROLLING_BY_INVITATIONThis early-stage trial tests a one-time gene therapy called BRL-101 for people with transfusion-dependent beta-thalassemia, a severe blood disorder. The treatment uses the patient's own stem cells, edited with CRISPR to boost healthy hemoglobin production. The goal is to see if a…
Matched conditions: TRANSFUSION-DEPENDENT THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Bioray Laboratories • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC