Treatment with ZYNTEGLO is managed through
a highly coordinated process

By using a patient’s own cells, ZYNTEGLO gives patients aged ≥12 years with non-ß00 TDT who do not have a matched related donor the potential to achieve transfusion independence.1 
    Approximately 2 months prior to ZYNTEGLO infusion

    • Cell collection is required for treatment, since ZYNTEGLO uses the patient's own haematopoietic stem cells
    • Includes mobilisation followed by apheresis
    • Mobilisation and apheresis may be repeated to ensure collection of sufficient stem cells for treatment
    • A back-up collection of CD34+ stem cells of at least 1.5×106 CD34+ cells/kg (if collected by apheresis) or >1.0×108 TNC/kg (if collected by bone marrow harvest) is required


    • After cell collection, the patient’s cells are shipped to the licensed manufacturing facility, where functional copies of the βA-T87Q‑globin gene are introduced into the cells via a LVV in order to manufacture ZYNTEGLO
    • ZYNTEGLO is then cryopreserved and, after passing quality release testing, stored until ready to be shipped to the ZYNTEGLO Qualified Treatment Centre

    At least 6 days before ZYNTEGLO infusion
    In clinical trials, a 4-day regimen of busulfan was the only myeloablative conditioning tested with ZYNTEGLO

    • The treating physician should confirm that HSC transplantation is appropriate for the patient before myeloablative conditioning is initiated
    • Full myeloablative conditioning (a chemotherapy given over 4 days) must be administered before infusion of ZYNTEGLO
    • It is recommended that patients maintain Hb ≥11 g/dL for 30 days prior to myeloablative conditioning
    • Iron chelation should be stopped at least 7 days prior to myeloablative conditioning
    • Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended
    • Depending on the myeloablative conditioning agent administered, prophylaxis for seizures should be considered
    • Myeloablative conditioning should not begin until the complete set of infusion bag(s) constituting the dose of ZYNTEGLO has been received and stored at the administration site, and the availability of the back-up collection is confirmed 


    • There must be a minimum of 48 hours of washout before ZYNTEGLO infusion 
    • ZYNTEGLO is administered to the patient via intravenous infusion at a Qualified Treatment Centre
    • Per the clinical judgement of the treating physician, the patient will remain in the hospital until they are ready to be discharged
    • NOTE: Restarting iron chelation after ZYNTEGLO infusion may be necessary and should be based on clinical practice; phlebotomy can be used in lieu of iron chelation, when appropriate

    For up to 15 years following administration of ZYNTEGLO

    • Every patient receiving ZYNTEGLO should be informed about and encouraged to enrol in the product registry for up to 15 years
    • The registry collects data on safety and efficacy of treatment