FDA Gives Signal to Halted Bluebird Bio's Gene Therapy Trials In Sickle Cell Disease, Thalassemia

The FDA has lifted the clinical hold on Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of Bluebird Bio Inc's BLUE LentiGlobin for sickle cell disease and two Phase 3 studies of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.
The company is working closely with study investigators and clinical trial sites to resume all study activities as soon as possible.
In March, the company said that based on the analyses completed to date, it is very unlikely the suspected unexpected serious adverse reaction of acute myeloid leukemia in its Phase 1/2 study of LentiGlobin was related to the BB305 lentiviral vector.
Later, the company revised the diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin.
The further assessment concluded this is not a case of MDS and revised the diagnosis to transfusion-dependent anemia.
Price Action: BLUE shares are up 1.5% at $31.38 during the market session on the last check Monday.