AU2019314513B2 - Improved clinical parameters by expression of factor viii - Google Patents

Improved clinical parameters by expression of factor viii

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Publication number
AU2019314513B2
AU2019314513B2 AU2019314513A AU2019314513A AU2019314513B2 AU 2019314513 B2 AU2019314513 B2 AU 2019314513B2 AU 2019314513 A AU2019314513 A AU 2019314513A AU 2019314513 A AU2019314513 A AU 2019314513A AU 2019314513 B2 AU2019314513 B2 AU 2019314513B2
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AU
Australia
Prior art keywords
seq
sequence
fviii
vector
aav6
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Active
Application number
AU2019314513A
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English (en)
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AU2019314513A1 (en
Inventor
Edward R. CONNER
Brigit E. RILEY
Didier Rouy
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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Publication of AU2019314513A1 publication Critical patent/AU2019314513A1/en
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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14071—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Hematology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2019314513A 2018-08-03 2019-08-02 Improved clinical parameters by expression of factor viii Active AU2019314513B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862714553P 2018-08-03 2018-08-03
US62/714,553 2018-08-03
US201962826887P 2019-03-29 2019-03-29
US62/826,887 2019-03-29
US201962869445P 2019-07-01 2019-07-01
US62/869,445 2019-07-01
PCT/US2019/044946 WO2020028830A1 (en) 2018-08-03 2019-08-02 Improved clinical parameters by expression of factor viii

Publications (2)

Publication Number Publication Date
AU2019314513A1 AU2019314513A1 (en) 2021-01-28
AU2019314513B2 true AU2019314513B2 (en) 2026-04-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019314513A Active AU2019314513B2 (en) 2018-08-03 2019-08-02 Improved clinical parameters by expression of factor viii

Country Status (11)

Country Link
US (1) US12497632B2 (OSRAM)
EP (1) EP3829650A4 (OSRAM)
JP (1) JP7684212B2 (OSRAM)
KR (1) KR20210039428A (OSRAM)
CN (1) CN112533645B (OSRAM)
AU (1) AU2019314513B2 (OSRAM)
BR (1) BR112021001315A2 (OSRAM)
CA (1) CA3105382A1 (OSRAM)
IL (1) IL280402B2 (OSRAM)
MX (1) MX2021001375A (OSRAM)
WO (1) WO2020028830A1 (OSRAM)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023006471A (es) * 2020-12-18 2023-08-11 Sangamo Therapeutics Inc Composiciones farmaceuticas mejoradas que contienen vector viral adenoasociado.

Citations (2)

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WO2017053677A1 (en) * 2015-09-24 2017-03-30 Biomarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
US20170119906A1 (en) * 2015-10-28 2017-05-04 Sangamo Biosciences, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof

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US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7238346B2 (en) 2002-07-08 2007-07-03 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw High capacity recombinant adenoviral vector for treatment of hemophilia A
ATE484591T1 (de) 2003-10-21 2010-10-15 Merck Serono Sa Minimale dna sequenz, die als chromatin-isolator wirkt, und deren verwendung für die protein- expression
WO2006043354A1 (ja) 2004-10-20 2006-04-27 National Institute Of Radiological Sciences 組込型の低線量放射線誘導性ベクター
JP2008543283A (ja) 2005-06-10 2008-12-04 ジェネンテック・インコーポレーテッド インスレーターエレメントを含むベクターを使用する抗体発現の改良
JP2011502478A (ja) 2007-11-01 2011-01-27 ユニバーシティー オブ ロチェスター 安定性が増大した組換え型第ｖｉｉｉ因子
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
WO2009134681A2 (en) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US9150847B2 (en) 2011-09-21 2015-10-06 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
JP2015501844A (ja) * 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
EP2804623B1 (en) 2012-01-12 2019-08-07 Bioverativ Therapeutics Inc. Chimeric factor viii polypeptides and uses thereof
CA2875618C (en) 2012-07-11 2021-04-27 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
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Also Published As

Publication number Publication date
WO2020028830A1 (en) 2020-02-06
CN112533645B (zh) 2026-01-23
JP2021533098A (ja) 2021-12-02
IL280402B1 (en) 2026-01-01
BR112021001315A2 (pt) 2021-04-27
CN112533645A (zh) 2021-03-19
IL280402A (en) 2021-03-01
US12497632B2 (en) 2025-12-16
EP3829650A1 (en) 2021-06-09
US20210171982A1 (en) 2021-06-10
KR20210039428A (ko) 2021-04-09
MX2021001375A (es) 2021-04-19
AU2019314513A1 (en) 2021-01-28
CA3105382A1 (en) 2020-02-06
IL280402B2 (en) 2026-05-01
EP3829650A4 (en) 2022-05-04
JP7684212B2 (ja) 2025-05-27

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