Message :
Required fields
Analyses demonstrate lentiviral vector BB305 unlikely to be the cause of AML in clinical study of LentiGlobin for SCD
bluebird bio has initiated process with regulators to resume clinical studies
Company to hold conference call and webcast today, March 10, 2021, 8:00 AM EST
bluebird bio, Inc. (Nasdaq: BLUE) announced today that based on the analyses completed to date, 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) (bb1111) was related to the BB305 lentiviral vector (LVV).
“In addition to our earlier findings of several well-known genetic mutations and gross chromosomal abnormalities commonly observed in AML in this patient, our latest analyses identified the integration site for the vector within a gene called VAMP4. VAMP4 has no known association with the development of AML nor with processes such as cellular proliferation or genome stability. Moreover, we see no significant gene misregulation attributable to the insertion event,” said Philip Gregory, chief scientific officer, bluebird bio. “In totality, the data from our assessments provide important evidence demonstrating that it is very unlikely our BB305 lentiviral vector played a role in this case and we have shared with the FDA that we believe these results support lifting the clinical holds on our β-thalassemia and sickle cell disease programs.”