Home > Haematology > EHA 2020 > Benign Haematology > SCD LentiGlobin gene therapy: new data on VOC and ACS

SCD LentiGlobin gene therapy: new data on VOC and ACS

Presented By
Dr Julie Kanter, University of Alabama, USA
EHA 2020
HGB-206 Group C study
Sickle cell disease (SCD) patients in the HGB-206 Group C study experienced high-level, sustained expression of gene-therapy-derived haemoglobin, as well as a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS). Dr Julie Kanter (University of Alabama, USA) provided new results on the largest cohort of the study, Group C, in which stem cells were extracted from the peripheral blood of patients by plerixafor mobilisation and apheresis, instead of bone marrow, with an altered protocol for gene therapy transduction with LentiGlobin gene therapy [1]. LentiGlobin therapy transduces autologous CD34+ cells with the BB305 lentiviral vector, encoding a human β-globin gene with the anti-sickling T87Q mutation (βA-T87Q). Patients underwent myeloablation with busulfan before infusio...

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