AU2002337901B2 - Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use - Google Patents

Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use Download PDF

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Publication number
AU2002337901B2
AU2002337901B2 AU2002337901A AU2002337901A AU2002337901B2 AU 2002337901 B2 AU2002337901 B2 AU 2002337901B2 AU 2002337901 A AU2002337901 A AU 2002337901A AU 2002337901 A AU2002337901 A AU 2002337901A AU 2002337901 B2 AU2002337901 B2 AU 2002337901B2
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AU
Australia
Prior art keywords
factor viii
polypeptide
sequence
seq
factor
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Ceased
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AU2002337901A
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AU2002337901A1 (en
Inventor
John S Lollar
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Expression Therapeutics LLC
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Expression Therapeutics LLC
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Publication of AU2002337901A1 publication Critical patent/AU2002337901A1/en
Assigned to EXPRESSION THERAPEUTICS LLC reassignment EXPRESSION THERAPEUTICS LLC Request for Assignment Assignors: EMORY UNIVERSITY
Application granted granted Critical
Publication of AU2002337901B2 publication Critical patent/AU2002337901B2/en
Anticipated expiration legal-status Critical
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    • 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)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2002337901A 2001-10-05 2002-10-07 Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use Ceased AU2002337901B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US32738801P 2001-10-05 2001-10-05
US60/327,388 2001-10-05
PCT/US2002/033403 WO2003031598A2 (en) 2001-10-05 2002-10-07 Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Publications (2)

Publication Number Publication Date
AU2002337901A1 AU2002337901A1 (en) 2003-07-03
AU2002337901B2 true AU2002337901B2 (en) 2008-01-17

Family

ID=23276347

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2002337901A Ceased AU2002337901B2 (en) 2001-10-05 2002-10-07 Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use

Country Status (9)

Country Link
US (3) US7635763B2 (https=)
EP (1) EP1572889B1 (https=)
JP (2) JP4634036B2 (https=)
AT (1) ATE417618T1 (https=)
AU (1) AU2002337901B2 (https=)
CA (1) CA2461443C (https=)
DE (1) DE60230456D1 (https=)
ES (1) ES2319745T3 (https=)
WO (1) WO2003031598A2 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7560107B2 (en) 1996-06-26 2009-07-14 Emory University Modified factor VIII
WO2003031598A2 (en) * 2001-10-05 2003-04-17 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
WO2005017149A1 (en) 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US7485291B2 (en) 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
SI1750733T1 (sl) * 2004-05-03 2014-03-31 Emory University POSTOPEK DAJANJA PRAĹ IÄŚJEGA fVIII BREZ DOMENE B
KR101468345B1 (ko) 2004-11-12 2014-12-03 바이엘 헬스케어 엘엘씨 Fviii의 부위 지향 변형
CN102112144A (zh) * 2008-05-16 2011-06-29 拜耳医药保健有限公司 靶向性凝固因子及其使用方法
JP5922141B2 (ja) 2010-11-05 2016-05-24 バクスター・インターナショナル・インコーポレイテッドBaxter International Incorp0Rated 増加した比活性を有する抗血友病第viii因子の新規変異体
CN103796670A (zh) * 2011-07-08 2014-05-14 比奥根艾迪克依蒙菲利亚公司 因子viii嵌合和杂合多肽及其使用方法
WO2014008172A2 (en) * 2012-07-03 2014-01-09 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
EP3904376B1 (en) 2013-06-24 2024-11-13 Xiao, Weidong Mutant factor viii compositions and methods
CN115074366A (zh) 2015-04-16 2022-09-20 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
EP3374387A1 (en) 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202092049A1 (ru) 2015-11-13 2020-11-10 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
KR102450833B1 (ko) 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
WO2019040747A1 (en) 2017-08-23 2019-02-28 Wake Forest University Health Sciences IN UTERO TRANSPLANTATION OF CELLS EXPRESSING FACTOR VIII FOR THE TREATMENT OF HEMOPHILIA
US20200360441A1 (en) 2017-08-23 2020-11-19 Wake Forest University Health Sciences Post-natal transplantation of factor viii-expressing cells for treatment of hemophilia
EP3823985A1 (en) 2018-07-16 2021-05-26 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
US11680109B2 (en) 2019-05-29 2023-06-20 Duke University Compositions and methods for detection and measurement of RNA modifications through targeted RNA editing
US20230357360A1 (en) * 2021-01-29 2023-11-09 Expression Therapeutics, Llc Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000071141A1 (en) * 1999-05-20 2000-11-30 Emory University Modified factor viii
US6818439B1 (en) * 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5859204A (en) 1992-04-07 1999-01-12 Emory University Modified factor VIII
US6180371B1 (en) 1996-06-26 2001-01-30 Emory University Modified factor VIII
US5663060A (en) 1992-04-07 1997-09-02 Emory University Hybrid human/animal factor VIII
US5888974A (en) 1992-04-07 1999-03-30 Emory University Hybrid human/animal factor VIII
US5364771A (en) 1992-04-07 1994-11-15 Emory University Hybrid human/porcine factor VIII
US5744446A (en) 1992-04-07 1998-04-28 Emory University Hybrid human/animal factor VIII
CA2162497A1 (en) * 1993-06-10 1994-12-22 Sheila Connelly Adenoviral vectors for treatment of hemophilia
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
WO1999029848A1 (en) * 1997-12-05 1999-06-17 The Immune Response Corporation Novel vectors and genes exhibiting increased expression
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
WO2003031598A2 (en) * 2001-10-05 2003-04-17 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6818439B1 (en) * 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
WO2000071141A1 (en) * 1999-05-20 2000-11-30 Emory University Modified factor viii

Also Published As

Publication number Publication date
CA2461443C (en) 2011-07-12
JP2005518783A (ja) 2005-06-30
DE60230456D1 (de) 2009-01-29
JP2011015698A (ja) 2011-01-27
EP1572889A4 (en) 2006-09-06
US8519111B2 (en) 2013-08-27
US20110077203A1 (en) 2011-03-31
US8188246B2 (en) 2012-05-29
JP4634036B2 (ja) 2011-02-16
WO2003031598A3 (en) 2006-01-05
CA2461443A1 (en) 2003-04-17
ATE417618T1 (de) 2009-01-15
US7635763B2 (en) 2009-12-22
US20050118684A1 (en) 2005-06-02
EP1572889A2 (en) 2005-09-14
HK1080517A1 (en) 2006-04-28
US20120270266A1 (en) 2012-10-25
ES2319745T3 (es) 2009-05-12
EP1572889B1 (en) 2008-12-17
JP5162635B2 (ja) 2013-03-13
WO2003031598A2 (en) 2003-04-17

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PC1 Assignment before grant (sect. 113)

Owner name: EXPRESSION THERAPEUTICS LLC

Free format text: FORMER APPLICANT(S): EMORY UNIVERSITY

FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired