Gene therapy LentiGlobin Treatment of Sickle Cell Disease: Phase I / Phase II Clinical Positive Results
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Last Update: 2020-06-25
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Source: Internet
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Author: User
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The latest data from Bluebird Bio's ongoing phase I/II study of gene therapy LentiGlobin for the treatment of sickle cell disease (SCD) shows that severevascularaocstatory crisis (VOC) and acute thoracic syndrome (ACS) have been addressedDavid Davidson, chief medical officer of Bluebird Bio, commented: "The complete elimination of VOC shows that LentiGlobin can provide really meaningful clinical benefitsIn addition, the anti-hemolytic HbAT87Q is almost entirely cell-distributed, suggesting that LentiGlobin may significantly alter the pathophysiology of the disease."As of March 3, 34 patients in the HGB-206 trial were treated with LentiGlobin, which showed a 99.8% reduction in each year's VOC and ACSThe company plans to accelerate the approval of LentiGlobin in the SCDsickle cell anemia is caused by a recessive monochromosomal monogene defect in the hemoglobin beta chain, which can lead to the production of sickle cell haemoglobin (HbS)Other forms of sickle cell disease (e.gHbSC or HbSB thalassemia) may occur if hbS is inherited from one parent and another abnormal hemoglobin (or beta thalassemia) is inherited from the other parent
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