Transfusion-dependent Beta-thalassemia
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CLINICAL_TRIALS research · 2026-02-03Clinical Trial Update: Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies [Phase 1]
Phase 1 clinical trial evaluating hematopoietic stem cell BCL11A enhancer gene editing for severe sickle cell disease and beta-thalassemia, …
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients
This clinical trial evaluates the use of the Hemanext One Hypoxic Red Blood Cell Storage System for transfusion in transfusion-dependent bet…
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CLINICAL_TRIALS research · 2025-04-02Clinical Trial Update: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
A long-term follow-up study is ongoing to monitor participants with sickle cell disease or transfusion-dependent beta-thalassemia who receiv…
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CLINICAL_TRIALS research · 2025-03-10Clinical Trial Update: Β-Thalassemia Treatment with KL003 Cell Injection [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating KL003 cell injection for transfusion-dependent beta-thalassemia is currently recruiting.
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CLINICAL_TRIALS research · 2024-10-29Clinical Trial Update: Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
A clinical trial update discusses decision-making and quality-of-life considerations for hematologic diseases undergoing gene therapy, with …
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