Originally published by our sister publication Specialty Pharmacy Continuum
By SPC Staff
The FDA approved the cell-based gene therapy exagamglogene autotemcel (exa-cel; Casgevy, Vertex Pharmaceuticals) for transfusion-dependent beta-thalassemia (TDT) in patients aged 12 years and older.
During the treatment, a patient’s hematopoietic stem cells are modified using the CRISPR-Cas9 genome editing tool and then transplanted back into the patient. The edited stem cells engraft within the bone