Bluebird Bio Gets Relief, But Gene Therapy Hurdles Remain

bluebird bio said a case of acute myeloid leukemia (AML) in a patient receiving its gene therapy for sickle cell disease (SCD), which was first disclosed in February, probably was not connected to the therapy itself. The 10 March announcement dispersed a significant dark cloud hanging over the program but potential hurdles remain on the road ahead, including adoption of the gene therapy, LentiGlobin.

Cambridge, MA-based bluebird said that analyses to date of data from the Phase I/II HGB-206 study made it “very unlikely” that the AML case reported in the study was