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Transfusion Independence after Exagamglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia
- Source :
- Transplantation and Cellular Therapy; February 2024, Vol. 30 Issue: 2, Number 2 Supplement 1 pS236-S236, 1p
- Publication Year :
- 2024
-
Abstract
- Exagamglogene autotemcel (exa-cel) is a cell therapy designed to reactivate fetal hemoglobin (HbF) via non-viral, ex vivo CRISPR/Cas9 gene-editing at the erythroid enhancer region of BCL11Ain autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). We report results from the first 44 patients (pts) dosed with exa-cel in the CLIMB THAL-111 trial.
Details
- Language :
- English
- ISSN :
- 26666375 and 26666367
- Volume :
- 30
- Issue :
- 2, Number 2 Supplement 1
- Database :
- Supplemental Index
- Journal :
- Transplantation and Cellular Therapy
- Publication Type :
- Periodical
- Accession number :
- ejs65485463
- Full Text :
- https://doi.org/10.1016/j.jtct.2023.12.308