MECHANISM OF ACTION
ZYNTEGLO addresses the underlying genetic cause of TDT 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 lifelong1
ZYNTEGLO mechanism of action

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

ZYNTEGLO 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 levels1