RecruitingPhase 1NCT06647979

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

Studying Sickle cell anemia

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Key facts

Sponsor
Daniel Bauer
Intervention
autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein(biological)
Enrollment
10 enrolled
Eligibility
13-40 years · All sexes
Timeline
2025 – 2030

Study locations (1)

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT06647979 on ClinicalTrials.gov

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