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Notably, LentiGlobin led to a 99.5% mean reduction in the annual rate of such events among 14 patients with a median of eight events in the two years’ prior treatment.Īlso, median levels of HbA T87Q were maintained, contributing to at least 40% of total hemoglobin (up to 60% of defective hemoglobin). No serious VOCs or acute chest syndrome were reported among participants with at least six months of data. They remained off transfusions at three months post-treatment.
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Results showed that all group C patients became free from regular blood transfusions.
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Newly presented data concerned patients in group C (followed for a median of 12.1 months and up to 24.8 months) and particularly the 18 patients followed for at least six months. Previous results from groups A and B up to three years after treatment showed that LentiGlobin resulted in sustained levels of HbA T87Q - present in more than 70% of red blood cells - and reduced frequency of VOCs and acute chest syndrome. An enhanced treatment protocol designed to increase the therapy’s efficiency was used for group C. In groups A and B, patients’ hematopoietic stem cells were extracted from the bone marrow, while in group C they were extracted from the blood. Participants had to have experienced at least four severe VOCs in the two years prior to enrollment.Īs of March 3, a total of 34 patients - seven in group A, two in group B, and 25 in group C - had received the therapy.
#Bluebird app icon trial
The Phase 1/2 HGB-206 trial ( NCT02140554) was designed to evaluate the safety and effectiveness of LentiGlobin in approximately 50 people, ages 12 to 50, with severe SCD. Once the cells differentiate, red blood cells start producing a modified version of hemoglobin, called HbA T87Q.īy promoting the production of this anti-sickling form of hemoglobin, LentiGlobin is expected to lower the proportion of defective hemoglobin in patients’ red blood cells, ultimately preventing their destruction and other complications associated with SCD. The single-dose therapy uses a harmless virus to deliver a modified, but functional, copy of the HBB gene into patients’ red blood cell precursors, or hematopoietic stem cells. LentiGlobin is a gene therapy designed to increase the levels of hemoglobin, the protein in red blood cells that carries oxygen and is defective in people with SCD due to mutations in the HBB gene. More information on contacts and locations is available here. “We are pleased to have reached a general agreement with the FDA on the clinical data required to support a submission for LentiGlobin for SCD and we plan to seek an accelerated approval,” said David Davidson, MD, Bluebird’s chief medical officer.Ī U.S.-based, Phase 3 clinical trial of LentiGlobin ( NCT04293185), called HGB-210, is recruiting approximately 35 children and adults with SCD.
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Food and Drug Administration (FDA), Bluebird expects to submit a biologics license application seeking LentiGlobin’s approval for treating SCD in the second half of 2021. Kanter is from The University of Alabama at Birmingham and presented the data.īased on the data and discussions with the U.S. “The promising results of this study, which show patients have an almost complete elimination of VOCs and ACS, suggest LentiGlobin … has real potential to provide a significant impact for people living with sickle cell disease,” Julie Kanter, MD, said in a press release.
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The findings, “ Outcomes in patients treated with LentiGlobin for sickle cell disease (SCD) gene therapy: updated results from the Phase 1/2 HGB-206 group C study,” were recently presented at the virtual 25th European Hematology Association (EHA) Annual Congress. Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in adolescents and adults with severe sickle cell disease (SCD), according to recent data from a Phase 1/2 clinical trial.