US biotech bluebird bio (Nasdaq: BLUE) has presented new data from its Phase I/II HGB-206 study of its LentiGlobin gene therapy for sickle cell disease (SCD) at ASH.
Results showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in patients from Group C of the trial, with history of VOCs and ACS who had at least six months follow-up, no reports of ACS or serious VOCs at up to 21 months post-treatment.
At six months post-treatment, these patients produced consistent median levels of gene therapy-derived anti-sickling hemoglobin (HbA), ranging from 44% to 59% reducing the median level of abnormal sickle haemoglobin.
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