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

NCT06647979 · clinicaltrials.gov ↗

PHASE1

Phase

RECRUITING

Status

10

Enrollment

OTHER

Sponsor class

Conditions

Sickle Cell Disease
Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Beta-Thalassemia
Transfusion Dependent Beta-Thalassaemia

Interventions

BIOLOGICAL: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
DEVICE: Sequencing Assay for Variant rs114518452

Sponsor

Daniel Bauer