Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A

US10189888B2 · kind B2 · utility

Assignee

• Baxalta Incorporated · CH

Inventors

• Falko-Günter Falkner · Orth an der Donau, AT
• Franziska Horling · Gänserndorf, AT
• Johannes Lengler · Wien, AT
• Hanspeter Rottensteiner · Wien, AT
• Friedrich Scheiflinger · Wien, AT

Key dates

• Filing date: Nov 11, 2016
• Grant date: Jan 29, 2019
• Expiry date: Nov 11, 2036

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2840/007
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.