Learn what Casgevy (exagamglogene autotemcel) is, how this CRISPR-Cas9 gene-editing therapy reactivates fetal hemoglobin by targeting BCL11A, and why it’s a breakthrough for sickle cell disease treatment.
Learn what Casgevy (exagamglogene autotemcel) is, how this CRISPR/Cas9-based gene therapy edits hematopoietic stem cells, reactivates fetal hemoglobin (HbF), and offers long-term relief for sickle cell disease and transfusion-dependent β-thalassemia.