The treatment involves collecting stem cells from the patient’s blood and then using CRISPR gene-editing technology to modify these cells in a lab.
Casgevy operates by editing the genes in a patient’s bone marrow stem cells to enable the production of functional hemoglobin. The treatment involves collecting stem cells from the patient’s blood and then using CRISPR gene-editing technology to modify these cells in a lab. The edited cells are reinfused into the patient, allowing the body to produce functioning hemoglobin. This process targets the faulty gene responsible for the disorders and aims to correct it at the source​​​​.