Gene Therapy For Thalassemia Needs To Be Safer

Jan 16, 2023 | News

Insertional oncogenesis is an inherent risk of lentiviral vectors because they are able to integrate into thousands of loci within the genome. Bluebird’s Lenti-D integrated into 1 or more oncogenes in nearly all 67 trial subjects with cerebral adrenoleukodystropy; 3 developed malignancy. The synthetic promoter that drives Lenti-D’s ubiquitous expression is culpable. BB305 is constructed with the natural, erythroid specific β-globin promoter, and has not caused malignancy or clonality in 63 with thalassemia or, on detailed analysis, in 50 with sickle cell. Michel Sadelain developed β-globin vector TNS9 in 2000, and in 2021 still believed that a safe and effective genetic cure for thal and sickle cell disease “remains to be achieved”.

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