Xagamglogene autotemcel cell (exa-cel)

Xagamglogene autotemcel cell (exa-cel) is the ka non-viral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered, regularlyinterspersed, short palindrome repeats (CRISPR)-CAS9 gene editing of autologous CD34 positive in hematopoietic stem and progenitor cells at the erythroid specific enhancer region of BCL11A.

Treatment with exa-cel eliminated vaso-occlusive crisis in 97% of patients with sickle cell disease for a period of 12 months or more.

Treatment with exa-cell, preceded by myeloablation resulted in transfusion independence in 91% of patients with transfusion dependent beta-thalassemia.

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