BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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Scientists test One-Shot gene fix for blood disorder
⭐️ CURE ⭐️ CompletedThis early-stage study tested a new one-time treatment for beta-thalassemia, a serious inherited blood disorder. Researchers collected a small number of patients' own blood stem cells, used gene editing to 'switch on' a protective fetal hemoglobin gene, and then returned the cell…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:32 UTC
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One-Shot CRISPR treatment aims to cure blood disorder, end lifelong transfusions
⭐️ CURE ⭐️ CompletedThis study tested a one-time treatment for people with severe beta-thalassemia, a genetic blood disorder that requires regular blood transfusions to survive. Doctors used CRISPR gene-editing technology to modify a patient's own blood stem cells, then gave them back as a transplan…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:09 UTC