Expression cassette for packaging and expression of variant factor VIII for the treatment of hemostasis disorders

US11014975B2 · kind B2 · utility

Assignee

• THE CHILDREN'S HOSPITAL OF PHILADELPHIA · US

Inventors

• Denise Sabatino · Havertown, US
• Katherine A. High · Yeadon, US
• Liron ELKOUBY · Modiin-Maccabim-Reut, IL

Key dates

• Filing date: Aug 13, 2015
• Grant date: May 25, 2021
• Expiry date: Aug 13, 2035

Classification

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

Abstract

Factor VIII variants and methods of use thereof are disclosed. In particular embodiments, Factor VIII variants are expressed more efficiently by cells over wild-type Factor VIII proteins, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced activity over wild-type Factor VIII proteins and are packaged more efficiently into viral vectors.