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Exagamglogene Autotemcel for Severe Sickle Cell Disease

24 Apr 2024

Abstract excerpt

Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 gene editing of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region of BCL11A . Methods We conducted a phase 3, single-group, open-label study of...

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PMID: 38661449
DOI: 10.1056/nejmoa2309676

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