bluebird bio says AML case "very unlikely" to be related to gene therapy LentiGlobin
bluebird bio has announced that a recently reported suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) is "very unlikely" to be related to its sickle cell disease (SCD) gene therapy LentiGlobin.
In a statement released today, bluebird bio said that the analyses completed to date includes additional scientific assessments to determine if the BB305 lentiviral vector used in the gene therapy could have caused the AML case.
“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 provides 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 beta-thalassemia and sickle cell disease programmes,” he added.
Read more: http://www.pmlive.com/pharma_news/bluebird_bio_says_aml_case_very_unlikely_to_be_related_to_gene_therapy_lentiglobin_1364991
In a statement released today, bluebird bio said that the analyses completed to date includes additional scientific assessments to determine if the BB305 lentiviral vector used in the gene therapy could have caused the AML case.
“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 provides 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 beta-thalassemia and sickle cell disease programmes,” he added.
Read more: http://www.pmlive.com/pharma_news/bluebird_bio_says_aml_case_very_unlikely_to_be_related_to_gene_therapy_lentiglobin_1364991
