The Hemophilia Center of Hannover Medical School (MHH) is pioneering the introduction of gene therapy for hemophilia A and B, becoming one of the first centers in Germany to offer this innovative treatment.

Hemophilia, a genetic blood-clotting disorder that affects approximately 6,000 individuals in Germany, is characterized by deficiencies in clotting factors VIII or IX.

Patients suffering from severe hemophilia face increased risks of bleeding, including joint and cerebral hemorrhages, which can lead to long-term joint damage.

Currently, around 150 patients at MHH are under long-term care, relying on frequent self-injections of clotting factor proteins, a process that can be quite challenging.

However, not all patients are eligible for gene therapy due to potential pre-existing antibodies against the viral vector used or underlying liver conditions.

The gene therapy involves a single intravenous injection that utilizes a viral shuttle to deliver the gene responsible for the missing clotting factor into liver cells, enabling the production of the necessary protein.

Clinical studies have indicated that many patients can achieve near-normal coagulation levels and may be able to discontinue previous therapies, though individual outcomes can vary.

This gene therapy is regulated under Advanced Therapy Medical Products (ATMP) guidelines, which ensure the quality and safety of the treatment.

The Hemophilia Center at MHH, along with its partner centers, has received certification from the Joint Federal Committee (G-BA) to provide gene therapy for hemophilia.

Looking ahead, the center plans to extend its gene therapy services to additional hemophilia centers in Lower Saxony through a collaborative approach.