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A new recombinant factor VIII: from genetics to clinical use
Advances in recombinant technology and knowledge about coagulation factor VIII (FVIII) are building a platform for new therapeutic options in patients with hemophilia A. The development of turoctocog alfa, a novel, high-purity, third-generation, B-domain truncated recombinant FVIII, has been produce...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Dove Medical Press
2014
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4271724/ https://www.ncbi.nlm.nih.gov/pubmed/25548513 http://dx.doi.org/10.2147/DDDT.S73241 |
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author | Santagostino, Elena |
author_facet | Santagostino, Elena |
author_sort | Santagostino, Elena |
collection | PubMed |
description | Advances in recombinant technology and knowledge about coagulation factor VIII (FVIII) are building a platform for new therapeutic options in patients with hemophilia A. The development of turoctocog alfa, a novel, high-purity, third-generation, B-domain truncated recombinant FVIII, has been produced and formulated without the use of animal-derived or human serum-derived components, in the wake of understanding of the new biochemical characteristics of FVIII, namely its protein structure, and glycosylation and sulfating patterns. Culture conditions and a five-step purification process have been developed to optimize the safety of turoctocog alfa. The results of two pilot clinical trials using turoctocog alfa confirmed high safety levels, with no patient developing inhibitors during the period of observation. The purpose of this review is to describe briefly the molecular and biological properties of turoctocog alfa, together with details of its clinical development, with emphasis on the needs of patients with hemophilia A. |
format | Online Article Text |
id | pubmed-4271724 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | Dove Medical Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-42717242014-12-29 A new recombinant factor VIII: from genetics to clinical use Santagostino, Elena Drug Des Devel Ther Review Advances in recombinant technology and knowledge about coagulation factor VIII (FVIII) are building a platform for new therapeutic options in patients with hemophilia A. The development of turoctocog alfa, a novel, high-purity, third-generation, B-domain truncated recombinant FVIII, has been produced and formulated without the use of animal-derived or human serum-derived components, in the wake of understanding of the new biochemical characteristics of FVIII, namely its protein structure, and glycosylation and sulfating patterns. Culture conditions and a five-step purification process have been developed to optimize the safety of turoctocog alfa. The results of two pilot clinical trials using turoctocog alfa confirmed high safety levels, with no patient developing inhibitors during the period of observation. The purpose of this review is to describe briefly the molecular and biological properties of turoctocog alfa, together with details of its clinical development, with emphasis on the needs of patients with hemophilia A. Dove Medical Press 2014-12-12 /pmc/articles/PMC4271724/ /pubmed/25548513 http://dx.doi.org/10.2147/DDDT.S73241 Text en © 2014 Santagostino. This work is published by Dove Medical Press Limited, and licensed under Creative Commons Attribution – Non Commercial (unported, v3.0) License The full terms of the License are available at http://creativecommons.org/licenses/by-nc/3.0/. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. |
spellingShingle | Review Santagostino, Elena A new recombinant factor VIII: from genetics to clinical use |
title | A new recombinant factor VIII: from genetics to clinical use |
title_full | A new recombinant factor VIII: from genetics to clinical use |
title_fullStr | A new recombinant factor VIII: from genetics to clinical use |
title_full_unstemmed | A new recombinant factor VIII: from genetics to clinical use |
title_short | A new recombinant factor VIII: from genetics to clinical use |
title_sort | new recombinant factor viii: from genetics to clinical use |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4271724/ https://www.ncbi.nlm.nih.gov/pubmed/25548513 http://dx.doi.org/10.2147/DDDT.S73241 |
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