Bluebird Bio has halted a phase 3 gene therapy clinical trial against cerebral adrenoleukodystrophy. This trial use lentiviral vectors carrying the healthy adrenoleukodystrophy protein (ADLP) gene.
Patients bone marrow cells were treated ex vivo with the vector before putting the modified cells back into the patients after they underwent chemotherapy. However, one patient developed myelodysplastic syndrome, a pre cursor to leukemia. Bluebird Bio state this is due to the vector design.
The need for assessing the safety profile of vectors has never been more important. Email us to discuss your needs on testing the safety of your vector in our human based in vitro model.