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bluebird bio Presents New Data from Ongoing Phase 1/2 HGB-206 Study of LentiGlobin Gene Therapy for Sickle Cell Disease (SCD) at 61st ASH Annual Meeting and Exposition

Nachrichtenquelle: Business Wire (engl.)
07.12.2019, 20:00  |  166   |   |   

bluebird bio, Inc. (Nasdaq: BLUE) announced new data from its ongoing Phase 1/2 HGB-206 study of investigational LentiGlobin gene therapy for sickle cell disease (SCD), including additional patients treated in the study and updated data for those previously reported. These data, as well as results from exploratory assays designed to assess the relationship between drug product characteristics and red blood cell physiology, were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida.

“At ASH, the growing body of data from our clinical studies of LentiGlobin for SCD reflects results from 26 treated patients with up to four years of follow-up,” said David Davidson, M.D., chief medical officer, bluebird bio. “We continue to observe patients treated in Group C producing high levels of gene-therapy derived anti-sickling hemoglobin, HbAT87Q, accounting for at least 40% of total hemoglobin in those with six or more months of follow-up, and exploratory assays show that HbAT87Q is present in most red blood cells of treated patients. The robust production of HbAT87Q was associated with substantial reductions of sickle hemoglobin, HbS, as well as improvement in key markers of hemolysis. Most importantly, patients in Group C have not experienced any episodes of acute chest syndrome or serious vaso-occlusive crises following LentiGlobin for SCD treatment.”

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the β-globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive crises (VOCs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complications—such as acute chest syndrome (ACS), stroke and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD was designed to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, their RBCs can produce anti-sickling hemoglobin that decreases the proportion of sickled hemoglobin, with the goal of reducing sickled RBCs, hemolysis and other complications.

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