Bluebird bio: Lentiviral vector BB305 unlikely to be the cause of AML in clinical study of LentiGlobin for Sickle cell disease

Bluebird bio announced that based on the analyses, it is very unlikely the Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) reported in its Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for sickle cell disease (SCD) was related to the BB305 lentiviral vector (LVV). Chief scientific officer reported that the integration site for the vector identified within the gene VAMP4, fact have confirmed by multiple independent analyses. VAMP4, has no known association with the development of AML nor with processes such as cellular proliferation or genome stability and also the insertion into the VAMP4 gene has had no impact on gene expression or gene regulation nor caused any disruption of nearby genes. Given this, the company has initiated engagement with regulators to begin the process of resuming clinical studies for sickle cell disease and β-thalassemia. More info: Bluebird bio press release.