Dosing & Uses
AdultPediatric
Sickle Cell Disease
Pending FDA approval as one-time gene therapy for sickle cell disease in patients aged ≥12 years
Sickle Cell Disease
Pending FDA approval as one-time gene therapy for sickle cell disease in patients aged ≥12 years
Next:
Pharmacology
Mechanism of Action
Gene therapy designed to add functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic stem cells (HSCs)
Once patients have the βA-T87Q-globin gene, their RBCs can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of sickle hemoglobin, with the goal of reducing sickled RBCs, hemolysis, and other complications
Previous
Next:
Images
No images available for this drug.
Previous
Next:
Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.
