Factor VIII, von Willebrand factor or complexes thereof with prolonged in vivo half-life

US9290561B2 · kind B2 · utility

Assignee

• CSL Behring AG · DE

Inventors

• Thomas Weimer · Gladenbach, DE
• Stefan Schulte · Marburg, DE
• Hubert Metzner · Marburg, DE
• Ulrich Kronthaler · Deisenhofen, DE
• Holger Lind · Marburg, DE
• Wiegand Lang · Cölbe, DE

Key dates

• Filing date: Sep 17, 2013
• Grant date: Mar 22, 2016
• Expiry date: Sep 17, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C07K2319/31
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to modified nucleic acid sequences coding for coagulation factor VIII (FVIII) and for von Willebrand factor (VWF) as well as complexes thereof and their derivatives, recombinant expression vectors containing such nucleic acid sequences, host cells transformed with such recombinant expression vectors, recombinant polypeptides and derivatives coded for by said nucleic acid sequences which recombinant polypeptides and derivatives do have biological activities together with prolonged in vivo half-life and/or improved in vivo recovery compared to the unmodified wild-type protein. The invention also relates to corresponding FVIII sequences that result in improved expression yield. The present invention further relates to processes for the manufacture of such recombinant proteins and their derivatives. The invention also relates to a transfer vector for use in human gene therapy, which comprises such modified nucleic acid sequences.