Bonn introduces pioneering gene therapy against hemophilia - a glimmer of hope!

Bonn introduces pioneering gene therapy against hemophilia - a glimmer of hope!

From 2025, innovative 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 therapy in patients with hemophilia who enable medication -free treatment and can effectively prevent bleeding.

hemophilia is an inherited disease characterized by the lack of coagulation factors VIII (hemophilia a) or IX (hemophilia b). The common therapy consists in the regular administration of injectable coagulation factors. With gene therapy, on the other hand, a healthy gene is introduced into the liver cells that produce the lack of coagulation factor. So far, two genetic therapies have been approved: Roctavian for hemophilia A and Hemgenix for hemophilia b.

First success of gene therapy

already in clinical studies, two patients successfully received genetic therapy. A patient with hemophilia A was treated 5.5 years ago, while a patient received therapy with hemophilia B 3.5 years ago. Both patients have not suffered bleeding since their treatment and do not require any additional coagulation factors.

The Medical Service North Rhine has given the UKB hemophilia center to approved generous therapies in the future. The center is one of the largest in Germany and supervises over 1,500 patients annually, with around 4,200 outpatient examinations and 250 inpatient stays performing per year.

Supplementary information on this topic can be found in an article from the Journal of Hematology deals with the basics and the progress of gene therapy for hemophilia A and B.

For further details on the background of the gene therapies and their effects on patients, see the report by Kabinett online .

-transmitted by West-East media