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Evaluation of the alfa-Glucosidase acid production potential by the human cell line HKB-11

Grant number: 17/06338-4
Support type:Scholarships in Brazil - Scientific Initiation
Effective date (Start): July 01, 2017
Effective date (End): October 31, 2019
Field of knowledge:Engineering - Chemical Engineering
Principal Investigator:Kamilla Swiech Antonietto
Grantee:Matheus Henrique dos Santos
Home Institution: Faculdade de Ciências Farmacêuticas de Ribeirão Preto (FCFRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil
Associated research grant:12/04629-8 - Establishment of a production platform for recombinant therapeutic proteins in human cells, AP.JP
Associated scholarship(s):19/00309-8 - Glycosylation profile of the recombinant acid alpha-glucosidase produced in the human cell line HKB-11, BE.EP.IC


The current treatment of Pompe disease, a lysosomal storage disorder, is performed by enzyme replacement therapy using human recombinant alpha-glycosidase, produced in Chinese hamster ovary (CHO) cells. Despite the satisfactory results, cases of serious immunological reactions have been reported due to the differences between the recombinant protein and the native one. There is a growing enthusiasm for the use of human cell lines as a platform for production of recombinant glycoproteins because of their ability to produce glycoproteins as close as possible to those naturally found in the human body, minimizing possible immunogenic reactions. Therefore, the aim of the present work is to evaluate the potential of recombinant alfa-glucosidase production by the human cell line HKB-11. For this, HKB-11 cells will be genetically modified and cultured in serum-free suspension culture, condition compatible with the industrial scale. After the gene modification, it will be characterized the growth kinetics and the cellular metabolism as well as the production kinetics of the enzyme. This work is expected to evaluate the possibility of establishing an alternative platform for the production of this enzyme, capable of generating a product with lower immunogenicity. (AU)