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The biotech company Bluebird Bio intends to resume clinical trials of a sickle cell disease gene therapy that were halted in February after a participant was diagnosed with acute myeloid leukemia five years after treatment. The company released a statement yesterday (March 10) claiming an investigation has found “it is very unlikely” the AML is related to the therapy and the firm is seeking approval from the US Food and Drug Administration (FDA) to resume the trials.
The therapy works by taking a patient’s own hematopoietic stem cells (HCS) and using a lentiviral vector to insert a healthy copy of a β-globin gene. After the patient who had received the gene therapy was diagnosed with AML, Bluebird examined the cancer cells and found the vector DNA incorporated into their genome. Upon additional testing from independent sources, researchers found that the vector landed within the VAMP4 gene ...
