BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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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 17, 2026 04:17 UTC
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Gene-Edited stem cells offer hope for blood disorder patients
Disease control CompletedThis study tested a new treatment for β-thalassemia major, a severe blood disorder that requires lifelong blood transfusions. Two children received their own stem cells that were gene-edited in a lab to produce healthier red blood cells. The goal was to see if the treatment is sa…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated May 17, 2026 04:14 UTC
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Gene editing frees thalassemia patients from transfusions?
Disease control CompletedThis early study tested a new gene therapy (CS-101) in 5 people with beta-thalassemia, a blood disorder requiring frequent transfusions. The treatment uses the patient's own stem cells, edited to produce healthy red blood cells. The main goals were safety and whether patients cou…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated May 15, 2026 11:55 UTC