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BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
NCT06647979Phase 1Recruiting
- Start
- Dec 2025
- Primary completion
- Dec 2028
- Modality
- CRISPR-Cas9, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of autologous CD34+ HSPCs electroporated with BCL11A enhancer-targeting Cas9 ribonucleoprotein in sickle cell disease (SCD) and beta thalassemia. Primary endpoint: Primary engraftment at 42 days. Started Dec 2025; status: recruiting.
02
Drugs
autologous CD34+ HSPCs electroporated with BCL11A enhancer-targeting Cas9 ribonucleoprotein
03
Conditions
sickle cell disease (SCD)beta thalassemia
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