autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein

BIOLOGICAL2 trials

Sponsors

Daniel Bauer

Conditions

Beta-ThalassemiaSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Sickle Cell DiseaseTransfusion Dependent Beta-Thalassaemia

Phase 1

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

RecruitingNCT06647979

Daniel BauerBeta-Thalassemia, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Sickle Cell Disease +1

Start: 2025-12-01End: 2030-12-01Target: 10Updated: 2026-02-03

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

Not yet recruitingNCT07708350

Daniel BauerSickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Sickle Cell Disease

Start: 2026-08-01End: 2030-12-01Target: 5Updated: 2026-07-16