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

Diagram of BB305 LVV carrying the Beta AT87Q-globin gene

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 approaching patient's HSC to deliver Beta AT87Q-globin gene

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 

Transduced CD34+ HSCs engraft in the patient's bone marrow to produce RBCs with functional Hb that contains βA-T87Q‑globin (HbAT87Q)1

βA‑T87Q-globin expression is designed to correct the β/α‑globin imbalance in erythroid cells and has the potential to increase total Hb to normal levels.1