Revolutionary gene therapy: End of bleeding in hemophilia patients!

From 2025, gene therapy for the treatment of hemophilia is offered in the hemophilia center of the University Hospital Bonn (UKB). The UKB has received approval for the use of this innovative therapy for hemophilia patients. This method enables medication -free treatment that effectively prevents bleeding.

hemophilia is an inherited disease in which coagulation factors VIII (hemophilia a) or IX (hemophilia b) are missing. The common therapy currently has regularly administration of coagulation factors that have to be injected. Gent therapy, on the other hand, introduces a healthy gene into the liver cells that produce the lack of coagulation factor. Two gene therapies have recently been approved: Roctavian for hemophilia A and Hemgenix for hemophilia b.

success of the clinical studies

In the context of clinical studies, two patients have already been successfully treated with genetic therapies. A patient with hemophilia A received therapy 5.5 years ago, while another patient with hemophilia B received this 3.5 years ago. Both patients have not suffered bleeding since their treatments and do not require any additional coagulation factors. The Medical Service of North Rhine has given the UKB hemophilia center to routinely carried out genetic therapies.

The UKB hemophilia center is one of the largest facilities of its kind in Germany and looks after over 1,500 patients every year. Around 4,200 outpatient examinations and 250 inpatient stays are carried out per year, which underlines the importance of this institution.

Additional information on gene therapy for hemophilia A and B can be read in an article from the Medical University of Vienna, which explains the basic principles of this therapy and documents progress in this area, such as ovid reported .