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bluebird bio’s gene therapy for sickle cell disease LentiGlobin has been granted an ‘innovation passport’ from the UK Medicines and Healthcare products Regulatory Agency (MHRA).
SCD is a serious, progressively debilitating and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis.
The European Medicines Agency will undertake an accelerated review of bluebird bio’s LentiGlobin, a gene therapy for the treatment of transfusion-dependent beta-thalassemia.