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

Info

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
Authority
AU
Australia
Prior art keywords
factor viii
polypeptide
sequence
seq
factor
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
AU2002337901A
Other languages
English (en)
Other versions
AU2002337901A1 (en
Inventor
John S Lollar
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Expression Therapeutics LLC
Original Assignee
Expression Therapeutics LLC
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 Expression Therapeutics LLC filed Critical Expression Therapeutics LLC
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
Ceased legal-status Critical Current

Links

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)
    • 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

Landscapes

  • 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
ES2319745T3 (es) * 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.
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
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
DK1750733T3 (da) * 2004-05-03 2014-01-20 Univ Emory FREMGANGSMÅDE TIL INDGIVELSE AF SVINE-B-DOMÆNELØS fVIII
PT2363414T (pt) 2004-11-12 2022-08-04 Bayer Healthcare Llc Modificação de fviii direcionada a sítio
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
DK2635297T3 (da) 2010-11-05 2019-05-06 Baxalta GmbH En ny variant af antihæmofil faktor viii, der har en øget specifik aktivitet
PT3513804T (pt) 2011-07-08 2022-06-02 Bioverativ Therapeutics Inc Polipéptidos fator viii híbridos e quiméricos, e métodos de utilização dos mesmos
US8986991B2 (en) * 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
LT3013855T (lt) * 2013-06-24 2021-01-25 Xiao, Weidong Mutavusio viii faktoriaus kompozicijos ir metodai
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CA3005565A1 (en) 2015-11-13 2017-05-18 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
MX2018012537A (es) 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
US20200368286A1 (en) 2017-08-23 2020-11-26 Wake Forest University Health Sciences In utero transplantation of factor viii-expressing cells for treatment of hemophilia
CA3073678A1 (en) 2017-08-23 2019-02-28 Children's Healthcare Of Atlanta, Inc. Post-natal transplantation of factor viii-expressing cells for treatment of hemophilia
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited 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
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
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
US6180371B1 (en) * 1996-06-26 2001-01-30 Emory University Modified factor VIII
EP0710288B1 (en) * 1993-06-10 2006-04-05 Genetic Therapy, Inc. Adenoviral vectors for treatment of hemophilia
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
EP1036177A1 (en) * 1997-12-05 2000-09-20 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
ES2319745T3 (es) * 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.

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

Similar Documents

Publication Publication Date Title
US8519111B2 (en) Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use
AU2002337901A1 (en) Nucleic acid and amino acid sequences encoding high-level expressor factor VIII polypeptides and methods of use
US7205278B2 (en) Stabilized proteins with engineered disulfide bonds
RU2285724C2 (ru) Рекомбинантная молекула днк, кодирующая модифицированный свиной фактор viii (pol 1212), экспрессирующие векторы, модифицированный свиной фактор viii, терапевтическая композиция, способы получения белка модифицированного свиного фактора viii (варианты) и линии клеток (варианты)
JP2005518783A5 (https=)
ES2292255T3 (es) Polipeptido del factor viii con actividad de factor viii:c.
EP1424344A1 (en) Modified cDNA factor VIII and its derivates
AU2001292861A1 (en) Modified factor VIII
WO1995024427A1 (en) Hybrid human/animal factor viii
AU2002310438A1 (en) Stabilized proteins with engineered disulfide bonds
AU2002364509A1 (en) Factor viii c2 domain variants
AU606925B2 (en) A method for producing factor viii in high yield
US20230357360A1 (en) Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
HK1080517B (en) Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
EP1454916A1 (en) Modified cDNA factor VIII and its derivatives

Legal Events

Date Code Title Description
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