Dosing & Uses
Sickle Cell Disease
Pending FDA approval for sickle cell disease
Beta Thalassemia
Pending FDA approval for transfusion-dependent beta thalassemia
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Pharmacology
Mechanism of Action
Ex vivo CRISPR/Cas9 gene-edited therapy in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in RBCs
HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth
Elevated HbF has potential to reduce or eliminate painful and debilitating vaso-occlusive crisis for patients with sickle cell disease and alleviate transfusion requirements for patients with transfusion-dependent beta thalassemia
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
