Hematological Disorders

Bluebird bio may finally get a gene therapy approved in the US Bluebird bio may get gene therapy approved in the US. The Cambridge, MA biotech declared that the FDA had accepted and given priority review for Zynteglo, its gene therapy for the rare blood disorder beta-thalassemia. The announcement sets up an expedited FDA decision by May 20, 2022. A priority review does not guarantee approval, and more than a few recent biotechs have been annulled after receiving the designation, including Incyte, Sesen, and Provention. But it shows a substantial step forward for a therapy that has seen repeated setbacks and an application that was slapped down with a refuse-to-f...