FDA lifts hold on trials of gene therapies for sickle cell disease, beta-thalassemia

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Sumary of FDA lifts hold on trials of gene therapies for sickle cell disease, beta-thalassemia:

  • In March, bluebird bio reported it was very unlikely that the case of AML was related to the BB305 lentiviral vector..
  • In April, bluebird officials announced a revised diagnosis for the reported myelodysplastic syndrome case, indicating the treating investigator determined the accurate diagnosis to be transfusion-dependent anemia….

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