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filingDate 2017-11-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2021-10-25-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_62684a37fedf5437289e222b701ac7e4
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publicationDate 2021-10-25-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber ES-2869339-T3
titleOfInvention Truncated von Willebrand factor polypeptides for the treatment of hemophilia
abstract A polypeptide comprising a truncated von Willebrand factor (VWF) and a half-life extending moiety, for use in treating a blood coagulation disorder, said treatment comprising administering the polypeptide to a subject having a blood coagulation disorder and has endogenous factor VIII (FVIII), wherein the level of endogenous FVIII activity in said subject prior to treatment with said polypeptide is reduced relative to the level of FVIII activity in normal human plasma (NHP) provided that the activity level of endogenous FVIII in said subject is at least 0.5% of the activity level of endogenous FVIII in normal human plasma (NHP), wherein the truncated VWF comprises an amino acid sequence that has at least 90% sequence identity to amino acids 764 to 1242 of SEQ ID NO:4, and wherein the polypeptide is capable of binding endogenous FVIII, wherein von Willebrand factor (VWF) tru ncado provides the ability of the polypeptide to bind endogenous FVIII; wherein the polypeptide is a dimer and the affinity of said dimer to FVIII is greater than the affinity of a monomeric polypeptide to said FVIII, said monomeric polypeptide having the same amino acid sequence as a monomeric subunit of the dimeric polypeptide; wherein the polypeptide has a binding affinity for FVIII characterized by a dissociation constant KD of less than 1 nM; wherein the half-life extending moiety is a heterologous amino acid sequence fused to the truncated VWF or conjugated to the polypeptide; and wherein the level of endogenous FVIII is increased after administration of said polypeptide and wherein - (i) said polypeptide is administered for prophylactic prevention of a bleeding event, wherein a) said treatment or not comprises co-administration of FVIII exogenous, or b) said treatment comprises administering an exogenous FVIII and thereby providing endogenous FVIII in said subject; or - (ii) said polypeptide is co-administered with exogenous FVIII for treatment of a bleeding event or for initiation of a prophylactic treatment regimen, wherein for follow-up treatments said polypeptide is co-administered without co-administration of exogenous FVIII.
priorityDate 2016-11-11-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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