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 free patients from lifelong blood transfusions
Disease control CompletedThis study tested a one-time treatment using the gene-editing tool CRISPR to try to help people with severe thalassemia. Doctors took a patient's own blood stem cells, edited a gene in the lab, and then gave them back. The main goal was to see if this single treatment could allow…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC