A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia. NCT02453477
A phase 3, open-label, randomized study to compare the efficacy and safety of luspatercept (ACE-536) versus epoetin alpha for the treatment of anemia due to IPSS-R very low, low or intermediate risk due to myelodysplastic syndrome (MDS) ESA in native subjects who require red blood cell transfusions. NCT03682536
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