FDA probing hematologic risk with Bluebird’s gene therapy
Dec. 2, 2024
The U.S. FDA said it is investigating the risk of hematologic malignancies associated with Bluebird Bio Inc.’s Skysona (elivaldogene autotemcel), approved in 2022 as a one-time gene therapy for treating early active cerebral adrenoleukodystrophy in boys, ages 4 to 17.