HemAcure: Application of combined gene and cell therapy within an implantable therapeutic device for the treatment of severe haemophilia A
The bleeding disease haemophilia A is caused by a genetic deficiency in clotting factor VIII (FVIII). At stage the therapies for treating haemophilia A mainly involve the application of recombinant factor VIII (rFVIII) which implies that patients need regular infusions with this factor. Thus, a permanent solution in the form of cell therapy is an extremely attractive approach for the treatment of this bleeding disease.
The innovative idea behind HemAcure: The isolation of endothelial cells from peripheral blood of haemophilia A patients and FVIII gene correction of those cells. The transduced cells will then be transplanted into the patient in a medical device designed for therapeutic cells (Cell PouchTM). The cells in this kind of cell bag are connected with the bloodstream of the patient and shall allow a continuous release of FVIII into the patient’s blood. The overall aim of this new approach is to significantly improve the quality of life of haemophilia A patients and reduce the side effects of the disease.
The project is coordinated by the University Hospital of Würzburg (UKW) and started on November 1st, 2015. A total of about 5.6 million Euros has been awarded to the consortium by the European Union within Horizon 2020 to run the project over three years.