Severe hemophilia B is a rare blood clotting disorder. Due to a mistake in the DNA, patients do not produce clotting factor IX. As a result, they have a high risk of bleeding in joints and muscles, and they receive coagulation factors via an IV twice a week.
Big result with gene therapy
The new gene therapy activates the production of clotting factors after just one treatment, the study shows. Meijer therefore calls the results very spectacular. “For the first time, there is a concrete treatment that ensures that patients with this specific form of hemophilia B are cured. Coagulation factor IX was produced in all patients, and we saw a significant reduction in the number of bleeding. It was no longer necessary to administer coagulation factors twice a week. This has given our patients enormous freedom.”
How it works
After a healthy factor IX gene is introduced into the patient, this gene ensures the production of coagulation factor IX. The healthy factor IX gene is packaged in a virus particle and travels through the bloodstream to the liver. There, the gene is taken up by the liver cells, which then start producing coagulation factor IX. This increases the amount of clotting factor in the blood and decreases the number of bleeding.
Follow-up research
According to Meijer, the study has clearly shown that the gene therapy works. Due to follow-up research, it is now necessary to investigate how long the therapy works and what the long-term effects are. According to Meijer, the omens are favourable.
Recently, the European Medicines Agency (EMA) has approved gene therapy for haemophilia B. We are now waiting for gene therapy to be given as regular therapy in the Netherlands. In mid-2022, another new and innovative GMP production and development facility for NecstGen cell and gene therapies was opened at the Leiden University Medical Center (LUMC).