Bluebird resumes clinical trials of gene therapy for sickle cell disease

Published: 9 June 2021

The FDA lifted clinical holds after concluding that a leukemia case is unlikely to be related to the autologous stem cell-based gene therapy.

US gene therapy company Bluebird Bio halted two clinical trials of LentiGlobin in February after two suspected cases of blood cancer in trial participants sparked concerns about the safety of the lentiviral vector used.

Further investigations revealed that one of the cases was not cancer after all and the FDA agreed with the company that the remaining case of acute myeloid leukemia was unlikely to be related to the treatment. The FDA has now given Bluebird the green light to resume Phase II/I and Phase III clinical trials of LentiGlobin for sickle cell anemia, along with two Phase III studies of beta-thalassemia therapy beti-cel (Zynteglo), which uses the same vector.