WO2015054439A3 - Hybrid factor viii polypeptides for use to treat hemophilia a - Google Patents

Hybrid factor viii polypeptides for use to treat hemophilia a Download PDF

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Publication number
WO2015054439A3
WO2015054439A3 PCT/US2014/059787 US2014059787W WO2015054439A3 WO 2015054439 A3 WO2015054439 A3 WO 2015054439A3 US 2014059787 W US2014059787 W US 2014059787W WO 2015054439 A3 WO2015054439 A3 WO 2015054439A3
Authority
WO
WIPO (PCT)
Prior art keywords
hrfviii
bdd
naturally occurring
expression vectors
domain deletion
Prior art date
Application number
PCT/US2014/059787
Other languages
French (fr)
Other versions
WO2015054439A2 (en
Inventor
Tommy E. Howard
Vincent La Terza
Original Assignee
Haplomics, Inc.
The Regents Of The University Of California
Department Of Veterans Affairs
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Haplomics, Inc., The Regents Of The University Of California, Department Of Veterans Affairs filed Critical Haplomics, Inc.
Publication of WO2015054439A2 publication Critical patent/WO2015054439A2/en
Publication of WO2015054439A3 publication Critical patent/WO2015054439A3/en

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)

Abstract

Hybrid recombinant FVIII polypeptides (hrFVIII) comprising non-naturally occurring combinations of amino acid modifications, cDNAs encoding such hrFVIII, expression vectors comprising nucleic acids encoding such hrFVIII, cells comprising expression vectors comprising nucleic acids encoding such hrFVIII, and methods of treating subjects having hemophilia A by administering to the subject such hrFVIII are disclosed herein. The non- naturally occurring amino acid modifications occur at sites of naturally occurring nonsynonymous-Single Nucleotide Polymorphisms (ns-SNP). The hrFVIII can be full length, having a B-domain deletion (BDD), having a B-domain deletion 2 (BDD-2), or having a B-domain deletion 3 (BDD-3). Methods of making the hrFVIIIs, cDNAs, expression vectors, cells are also disclosed.
PCT/US2014/059787 2013-10-08 2014-10-08 Hybrid factor viii polypeptides for use to treat hemophilia a WO2015054439A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361888373P 2013-10-08 2013-10-08
US61/888,373 2013-10-08

Publications (2)

Publication Number Publication Date
WO2015054439A2 WO2015054439A2 (en) 2015-04-16
WO2015054439A3 true WO2015054439A3 (en) 2015-06-04

Family

ID=52813741

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/059787 WO2015054439A2 (en) 2013-10-08 2014-10-08 Hybrid factor viii polypeptides for use to treat hemophilia a

Country Status (1)

Country Link
WO (1) WO2015054439A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006063031A2 (en) 2004-12-06 2006-06-15 Haplomics Allelic variants of human factor viii
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
PE20231949A1 (en) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTS OF FACTOR VIII REDUCED WITH CpG, COMPOSITIONS AND METHODS AND USES FOR THE TREATMENT OF HEMOSTASY DISORDERS

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100256062A1 (en) * 2004-12-06 2010-10-07 Howard Tommy E Allelic Variants of Human Factor VIII
US20120297494A1 (en) * 2009-10-16 2012-11-22 Tommy Eugene Howard Compositions and methods of treatment of black hemophiliac patients

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100256062A1 (en) * 2004-12-06 2010-10-07 Howard Tommy E Allelic Variants of Human Factor VIII
US20120297494A1 (en) * 2009-10-16 2012-11-22 Tommy Eugene Howard Compositions and methods of treatment of black hemophiliac patients

Also Published As

Publication number Publication date
WO2015054439A2 (en) 2015-04-16

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