Zynteglo badge 1st Approved for Gene Therapy 

Transfusion-dependent
β-thalassaemia

People arranged in a double-helical formation on a field releasingblood bags into the air like balloons

Help them let go of
chronic transfusions1

ZYNTEGLO (betibeglogene autotemcel) is the first and only one-time gene therapy for transfusion-dependent β-thalassaemia (TDT) that gives patients the potential to achieve transfusion independence*2

See how ZYNTEGLO works
 
In the pivotal trial 90% of evaluable patients achieved transfusion independence by Month 24†1
There are no reports of lentiviral vector (LVV)-mediated insertional mutagenesis resulting in oncogenesis1
For patients with TDT ≥12 years of age who do not have a β00 genotype and who do not have an HLA-matched related donor1
INDICATION1

ZYNTEGLO is indicated for the treatment of patients 12 years and older with TDT who do not have a β00 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)–matched related HSC donor is not available.

Full common name: A genetically modified autologous CD34+ cell–enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene.1

*In clinical trials, transfusion independence was defined as a weighted average haemoglobin (Hb) of ≥9 g/dL with no red blood cell (RBC) transfusions for a continuous period of ≥12 months at any time during the study after infusion of ZYNTEGLO.1

†As the Phase 3 trial is ongoing, data are for patients who are currently evaluable for assessment of transfusion independence.1