Fidest – Agenzia giornalistica/press agency

Quotidiano di informazione – Anno 32 n° 25

61st ASH Annual Meeting and Exposition

Posted by fidest press agency su giovedì, 12 dicembre 2019

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. 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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