SINGLE CHAIN FACTOR VIII MOLECULE

The present invention relates to a recombinant Factor VIII protein comprising, in a single chain, a heavy chain portion comprising an A1 and an A2 domain and a light chain portion comprising an A3, C1 and C2 domain of Factor VIII, wherein the B-domain is partially deleted in two deletions, the first...

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Bibliographic Details
Main Authors Ungerer, Christopher, Daufenbach, Jens, Kistner, Steffen, Herbener, Peter
Format Patent
LanguageEnglish
Published 18.08.2022
Subjects
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:The present invention relates to a recombinant Factor VIII protein comprising, in a single chain, a heavy chain portion comprising an A1 and an A2 domain and a light chain portion comprising an A3, C1 and C2 domain of Factor VIII, wherein the B-domain is partially deleted in two deletions, the first leading to the presence of a defined processing sequence cleavable by thrombin, and the second leading to absence of the furin cleavage recognition site at position R1664-R1667. An internal fragment of the B-domain is maintained Nucleic acids encoding said protein, host cells and methods of preparing the protein are also provided, as well as a pharmaceutical composition comprising the protein, nucleic acid or host cell, which may be used for treatment of hemophilia A.
Bibliography:Application Number: US202017438646