The FDA must decide whether to approve the therapy by Dec. 8.
A researcher performs a CRISPR process at the Max Delbrück Center for Molecular Medicine. Photo: Gregor Fischer/dpaEditing genes directly in a patient's body could be life-changing for people with debilitating hereditary disorders like sickle cell disease, which the exa-cel therapy under discussion targets.around "off-target" edits that zero in on the wrong genetic sequence and could increase the risk of developing cancer.
A patient's stem cells are edited to produce high levels of fetal hemoglobin, which can offset the effects of defective hemoglobin produced in patients with sickle cell.didn't take issue with the efficacy of exa-cel but questioned if the companies did sufficient analysis to adequately assess the off-target risk.
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