bluebird bio updates on genetic disease portfolio

bluebird bio (Nasdaq: BLUE) today announced business and program updates across its severe genetic disease portfolio, which pushed the US biotech firm’s shares down more than 2% to $27.40 by late-morning trading.

This included a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase I/II study of LentiGlobin for sickle cell disease (SCD) (bb1111), the company’s decision to withdraw Zynteglo (betibeglogene autotemcel, beti-cel) for transfusion-dependent β-thalassemia (TDT) from the German market, and a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs.

Last month, the company reported that it is very unlikely the suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector (LVV). This assessment, along with the re-classification of the originally reported MDS case to transfusion-dependent anemia are important steps in bluebird bio’s path to seeking removal of the clinical hold on studies HGB-206 and HGB-210 of LentiGlobin for SCD.