BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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Gene-Editing breakthrough offers hope for blood disorder patients
Disease control CompletedThis early-stage study tested a new gene-edited stem cell transplant in 2 children with severe β-thalassemia, a serious blood disorder requiring lifelong transfusions. The treatment aims to help the body produce healthy red blood cells and reduce or stop the need for transfusions…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Gene editing offers hope for blood disorder patients
Disease control CompletedThis early-phase study tested a new gene-edited stem cell treatment (CS-101) in 5 people with beta-thalassemia, a severe blood disorder requiring regular blood transfusions. The goal was to see if the treatment is safe and can help patients produce healthy red blood cells, potent…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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CRISPR gene editing offers hope for thalassemia patients to ditch transfusions
Disease control CompletedThis study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC