NCT06647979View on ClinicalTrials.gov

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

PHASE1Not yet recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

April 30, 2025

Primary Completion Date

December 31, 2028

Study Completion Date

December 31, 2030

Conditions
Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-ThalassemiaTransfusion Dependent Beta-Thalassaemia
Interventions
BIOLOGICAL

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

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

DEVICE

Sequencing Assay for Variant rs114518452

Device used to carry out the diagnostic testing for exclusion criteria number 12

Trial Locations (1)

02115

Boston Children's Hospital, Boston

All Listed Sponsors
lead

Daniel Bauer

OTHER