MECHANISM OF ACTION
By using a patient’s own cells, ZYNTEGLO gives patients the potential to achieve transfusion independence without the need for a donor1
ZYNTEGLO is a genetically modified autologous CD34+ cell–enriched population that contains HSCs transduced with lentiviral vector (LVV) encoding the βA-T87Q-globin gene1
Following successful engraftment and achievement of transfusion independence, the effects of ZYNTEGLO are expected to be lifelong.1
ZYNTEGLO mechanism of action: addressing the underlying genetic cause of TDT1

BB305 LVV, used to manufacture ZYNTEGLO, is a replication-incompetent, self-inactivating LVV that carries the βA-T87Q‑globin gene (a modified β‑globin gene)1

BB305 LVV adds functional copies of the βA‑T87Q‑globin gene to the patient’s own HSCs ex vivo via transduction of autologous CD34+ cells with BB305 LVV1
