Gene therapy for transfusion-dependent β-thalassemia

The KiTZ is the world’s first center to treat a form of the transfusion-dependent β-thalassemia (TDT) with gene therapy. Treatment with the drug Zynteglo is approved by the European Medicine Agency (EMA) for

  • Patients with TDT from 12 years of age, in whom the mutations of the ß-globin gene allow a certain residual activity of the ß-globin chain synthesis (no β0 / β0 genotype) and who are suitable for a hematopoietic stem cell (HSC) transplantation, but for whom no Human Leukocyte Antigen (HLA) compatible, related HSC donor is available.

The transfusion-dependent ß-thalassemia is an enormous burden for affected patients and families. In addition to health restrictions due to the disease itself, patients need blood transfusions every three to four weeks as a lifelong therapy. In addition, treatment is required to excrete excess iron, which otherwise critically threatens the function of the heart, liver and hormone-producing tissues. This disease therefore reduces life expectancy and leads to a considerable burden on the quality of life of the patient and the affected family.

With the new therapy, the affected patients have for the first time the chance to live a life independent of transfusions. The treatment takes place in the Clinic for Pediatric Oncology, Hematology and Immunology of the Heidelberg University Hospital. The contact person is Professor Dr. Andreas Kulozik, Medical Director of the Heidelberg University Clinic and Director of the KiTZ clinical program.

Consultation „Transfusion-dependent Thalassemia“
Prof. Dr. Andreas Kulozik
Tel.: +49 6221 – 56 4500
E-Mail: andreas.kulozik(at)med.uni-heidelberg.de