Method of treating hemophilia A

US10792336B2 · kind B2 · utility

Assignees

• St. Jude Children's Research Hospital, Inc. · US
• UCL Business Ltd · GB

Inventors

• Amit Nathwani · London, GB
• Jenny McIntosh · London, GB
• Edward Tuddenham · London, GB
• Andrew Davidoff · Memphis, US

Key dates

• Filing date: Jul 10, 2018
• Grant date: Oct 6, 2020
• Expiry date: Jul 10, 2038

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61K48/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a Factor VIII protein, wherein a B domain portion of the Factor VIII protein is encoded by a nucleotide sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a Factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.