EU stamps historic OK on bluebird’s gene therapy for β-thalassemia — now sit back and wait for the price
It’s official: bluebird bio has scored its first drug approval with an EU marketing authorization for its gene therapy for transfusion-dependent β-thalassemia.
Zynteglo is the new name for a treatment once known as LentiGlobin, a process that takes CD34+ cells from a patient’s hematopoietic stem cells, inserts functional copies of the βA-T87Q-globin gene, then reinfuses the modified HSCs. Patients are given chemotherapy to prime their bone marrows before the reinfusion.
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