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Promising news for gene therapy for sickle cell disease

Dr Olivier Hermine, Necker Hospital, France
EHA 2019
Phase 1/2, HGB-206

An engineered lentivirus delivers functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene or LentiGlobin) into a sickle cell disease (SCD) patient’s own haematopoietic stem cells. Once patients have the βA-T87Q-globin gene, they can make functional red blood cells, with the goal of reducing sickled red blood cells, haemolysis, and ensuing complications [1]. In patients who were at least 6 months post-treatment with lentiviral LentiGlobin for SCD, the median level of abnormal sickle haemoglobin was reduced to ≤50% of total haemoglobin. At up to 15 months post-treatment with LentiGlobin, no serious vaso-occlusive crisis (VOEs) or acute chest syndrome were reported in this cohort.

In the plenary session, Dr Olivier Hermine (Necker Hospital, France) presented the ongoing, phase 1/2 HGB-206 study. Adults and children living with SCD experience unpredictable episodes of pain due...

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