The recombinant Factor VII (rFVII) has been the main choice of therapy for hemophiliac patients who develop inhibitors against factor VIII and IX used as treatment. Currently, the product used is produced in hamster cells (BHK-21) which has disadvantages considering the complexity of post-translational modification of this protein and the insertion of glycosylations from murine origin highly immunogenic to humans. Thus the production of proteins for therapeutic use in human cell lines appears as a promising alternative. In this context, over the past four years, it was developed at the Blood Center of Ribeirão Preto a recombinant factor VII production platform in human cell lines, in which it was possible to select a cell (Sk-Hep-1) capable of producing about 3 IU/ml rFVII which culminated in the filing of a patent (BR1020150123345). Because of these promising results the present study aims the purification of the recombinant FVII produced in human cell line, evaluate and define pharmacokinetic parameters and characterize the protein glycosylation profile in order to prove their efficacy and safety. In addition, the present study aims to develop a new technology to encapsulation of the protein using nanoparticles to increase their stability and half-life, in order to improve the patient life quality.
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