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.