Bonn introduces groundbreaking gene therapy against hemophilia – a glimmer of hope!

Bonn introduces groundbreaking gene therapy against hemophilia – a glimmer of hope!

From 2025, innovative gene therapy for the treatment of hemophilia will be offered in the Hemophilia Center at the University Hospital of Bonn (UKB). The UKB has received approval for the use of this therapy in patients with hemophilia, which provides drug-free treatment and can effectively prevent bleeding.

Hemophilia is an inherited disease characterized by the absence of clotting factors VIII (hemophilia A) or IX (hemophilia B). The usual therapy consists of the regular administration of injectable coagulation factors. With gene therapy, however, a healthy gene is introduced into the liver cells that produces the missing clotting factor. Two gene therapies have been approved so far: Roctavian for hemophilia A and Hemgenix for hemophilia B.

First successes of gene therapy

Two patients have already successfully received gene therapy in clinical trials. A patient with hemophilia A was treated 5.5 years ago, while a patient with hemophilia B received therapy 3.5 years ago. Both patients have not experienced any bleeding since their treatment and do not require additional clotting factors.

The North Rhine Medical Service has given the UKB Hemophilia Center approval to routinely carry out gene therapies in the future. The center is one of the largest in Germany and looks after over 1,500 patients annually, with around 4,200 outpatient examinations and 250 inpatient stays carried out per year.

Additional information on this topic can be found in an article from the Journal of Hematology, which deals with the principles and advances of gene therapy for hemophilia A and B.

For further details on the background of gene therapies and their impact on patients, see the report by Cabinet Online.

– Submitted by West-East media