People suffering from haemophilia A have a genetic deficiency in clotting factor VIII which causes increased bleeding. In extreme cases, unstoppable bleeding may occur that can be life-threatening, in particular if the head or brain region is affected. Joint bleeds occur frequently that can cause permanent damage and even destruction of joints when left untreated.
Although effective treatment of the symptoms is available, there is no cure at present. Patients have to get lifelong infusions with recombinant factor VIII (rFVIII) several times a week to compensate for the missing clotting factor. The costs of treatment for adults vary between €200,000 and 800,000 per year (GBA).
The aim of HemAcure is to develop a novel curative therapy for severe haemophilia A. The innovative idea: The haemophilia patients' own cells are genetically corrected outside the body to produce the missing clotting factor using precursor cells of endothelial cells flowing in the bloodstream. Subsequently, these cells are transplanted back into the patient's body in Sernova’s Cell Pouch™, which is a small implantable medical device for the survival and function of therapeutic cells. The Cell Pouch™ is implanted in the abdominal region under the skin and becomes vascularized. The genetically modified cells are then transplanted into the device and are expected to continuously produce the clotting factor and release it into the bloodstream for a long period of time. This should mitigate the disease's impact noticeably, increase the patients' quality of life and reduce the costs of therapy.
Coordinator Joris Braspenning on the benefits of the project: "I am highly delighted receiving the opportunity from the European Union to coordinate the HemAcure joined forces of industry and academia. The combination of our complimentary expertise enables a quality by design approach in order to develop better and quicker cell-based medicinal products compliant to European GMP-regulations. This will be of great benefit for tomorrow’s advanced therapies and of course for the patients suffering from severe haemophilia A."