Casgevy works by enabling people to produce a fetal form of hemoglobin that doesn't cause sickling. It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production ...

Advances in CRISPR technologies promise to accelerate the burgeoning pipeline of gene-editing therapies and broaden access to these disease-altering therapies.

CASGEVY is a non-viral ... and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin ...

Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY - - Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached ...

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited ...