Clinical Trial Update: Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies [Phase 1]

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Summary

Phase 1 clinical trial evaluating hematopoietic stem cell BCL11A enhancer gene editing for severe sickle cell disease and beta-thalassemia, currently recruiting…

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Diseases / indications

• Beta-thalassemia
• Sickle Cell Disease
• Transfusion-dependent Beta-thalassemia

Drugs mentioned

• Hematopoietic stem cell BCL11A enhancer gene editing therapy

Snippet

Sponsor: Daniel Bauer | Conditions: Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0) | Phase 1 | Status: RECRUITING