Gene therapy approved for β‑thalassaemia


  • Dawn O'Shea
  • Univadis Medical News
El acceso al contenido completo es sólo para profesionales sanitarios registrados. El acceso al contenido completo es sólo para profesionales sanitarios registrados.

The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for the gene therapy Zynteglo for the treatment of transfusion-dependent β‑thalassaemia (TDT).

Zynteglo will be available as a 1.2-20×106 cells/mL dispersion for infusion. The active substance of Zynteglo is an autologous CD34+ cell-enriched population that contains haematopoietic stem cells (HSC) transduced with lentiviral vector encoding the βA‑T87Q-globin gene. Zynteglo adds functional copies of a modified β-globin gene into haematopoietic stem cells through transduction of autologous CD34+ cells with BB305 lentivirus vector, thereby addressing the underlying genetic cause of the disease.

Zynteglo has been shown to enable βA‑T87Q-globin expression, which is designed to correct the β/α-globin imbalance in erythroid cells of patients with TDT and has the potential to increase total haemoglobin to normal levels and eliminate dependence on chronic red blood cell transfusions. The most common side effects are thrombocytopenia, abdominal pain, non-cardiac chest pain, pain in the extremities, dyspnoea and hot flush.

The full indication is: “Treatment of patients 12 years and older with transfusion-dependent β‑thalassaemia who do not have a β00 genotype for whom HSC transplantation is appropriate but a human leukocyte antigen-matched-related HSC donor is not available.”