A molecular jewel for hemophilia A treatment

In this issue of Blood, Seth Chhabra et al describe the engineering of a new therapeutic chimera composed of B domain-deleted (BDD) factor VIII (FVIII), FVIII-binding domain of von Willebrand factor (VWF), Fc gamma 1 fragment, and XTEN polypeptides.(1) The molecule, referred to as rFVIIIFc-VWF-XTEN...

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Veröffentlicht in:	Blood 2020-04, Vol.135 (17), p.1417-1419
Hauptverfasser:	Rayes, Julie, Lacroix-Desmazes, Sébastien
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Factor VIII - genetics Hematology Hemophilia A - genetics Hemophilia A - therapy Immunology Life Sciences Life Sciences & Biomedicine Mice Primates Recombinant Fusion Proteins Science & Technology von Willebrand Factor
Online-Zugang:	Volltext
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Beschreibung
Zusammenfassung:	In this issue of Blood, Seth Chhabra et al describe the engineering of a new therapeutic chimera composed of B domain-deleted (BDD) factor VIII (FVIII), FVIII-binding domain of von Willebrand factor (VWF), Fc gamma 1 fragment, and XTEN polypeptides.(1) The molecule, referred to as rFVIIIFc-VWF-XTEN or BIVV001, has a prolonged half-life that is independent from endogenous VWF and is hemostatically competent.
ISSN:	0006-4971 1528-0020
DOI:	10.1182/blood.2020005250