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-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 blood cells, potentiall…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated May 26, 2026 09:29 UTC
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Gene-Editing breakthrough: first patients treated for blood disorder
Disease control CompletedThis early-phase study tested a new gene-editing technique (base editing) on a patient's own blood stem cells to treat severe β-thalassemia, a genetic blood disorder requiring lifelong transfusions. Two children received the edited cells after chemotherapy, and researchers monito…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated May 26, 2026 09:27 UTC
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CRISPR gene editing frees thalassemia patients from lifelong transfusions
Disease control CompletedThis study tested a one-time treatment using CRISPR gene editing to fix a patient's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the modified cells after a short course of chemotherapy. …
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 18, 2026 12:00 UTC