Abstract:Background:Sickle cell disease (SCD) is one of the most prevalent inherited disorders worldwide. The ex vivo gene therapy phase 1/2 study HGB‐205 conducted in France, evaluates the treatment of SCD and TDT (Transfusion Dependent Thalassemia) with LentiGlobin Drug Product (DP) containing autologous CD34+ cells transduced with the BB305 lentiviral vector encoding a human β‐globin gene with a single substitution (βA‐T87Q).Aims:We evaluated the clinical impact and the production of gene therapy‐derived hemoglobin … Show more
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