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Plataform for recombinant factor VII production in human cells: development of a high eficient and stable national biopharmaceutical

Grant number: 15/19017-6
Support type:Scholarships in Brazil - Post-Doctorate
Effective date (Start): December 01, 2015
Effective date (End): May 31, 2019
Field of knowledge:Health Sciences - Medicine
Principal Investigator:Dimas Tadeu Covas
Grantee:Marcela Cristina Corrêa de Freitas
Home Institution: Hemocentro de Ribeirão Preto. Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da USP (HCMRP). Secretaria da Saúde (São Paulo - Estado). Ribeirão Preto , SP, Brazil
Associated scholarship(s):16/23545-0 - Post-translational modification profile of recombinant factor VII produced in human cell lines, BE.EP.PD

Abstract

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.