AU2002352261B2 - A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism - Google Patents

A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism Download PDF

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Publication number
AU2002352261B2
AU2002352261B2 AU2002352261A AU2002352261A AU2002352261B2 AU 2002352261 B2 AU2002352261 B2 AU 2002352261B2 AU 2002352261 A AU2002352261 A AU 2002352261A AU 2002352261 A AU2002352261 A AU 2002352261A AU 2002352261 B2 AU2002352261 B2 AU 2002352261B2
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library
virions
nucleic acid
cell
aav
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AU2002352261A
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AU2002352261A1 (en
Inventor
Hildegard Buning
Jorg Enssle
Michael Hallek
Nadja Huttner
Luca Perabo
Martin Ried
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Medigene AG
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Medigene AG
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Communicable Diseases (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2002352261A 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism Expired AU2002352261B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2008202032A AU2008202032B2 (en) 2001-12-21 2008-05-07 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US34413101P 2001-12-21 2001-12-21
US60/344,131 2001-12-21
US36234902P 2002-03-07 2002-03-07
US60/362,349 2002-03-07
US40711602P 2002-08-30 2002-08-30
US60/407,116 2002-08-30
PCT/EP2002/014750 WO2003054197A2 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2008202032A Division AU2008202032B2 (en) 2001-12-21 2008-05-07 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

Publications (2)

Publication Number Publication Date
AU2002352261A1 AU2002352261A1 (en) 2003-07-09
AU2002352261B2 true AU2002352261B2 (en) 2008-02-07

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AU2002352261A Expired AU2002352261B2 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
AU2008202032A Expired AU2008202032B2 (en) 2001-12-21 2008-05-07 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

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Country Status (7)

Country Link
US (1) US20050106558A1 (enExample)
EP (2) EP1456383B1 (enExample)
JP (1) JP2005512569A (enExample)
AU (2) AU2002352261B2 (enExample)
CA (1) CA2468882C (enExample)
ES (1) ES2467156T3 (enExample)
WO (1) WO2003054197A2 (enExample)

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US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
CN101511373B (zh) * 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2158211B1 (en) * 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
EP2098536A1 (en) * 2008-03-05 2009-09-09 4-Antibody AG Isolation and identification of antigen- or ligand-specific binding proteins
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP5765790B2 (ja) * 2010-10-21 2015-08-19 ドイッチェス・クレープスフォルシュングスツェントルムＤｅｕｔｓｃｈｅｓ Ｋｒｅｂｓｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ラットパルボウイルスｈ−１ｐｖのそのカプシドの遺伝子操作による癌細胞への再標的化
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
US9821043B2 (en) 2011-09-15 2017-11-21 Medigene Ag Anti-HER2 vaccine based upon AAV derived multimeric structures
EP2692731A1 (en) * 2012-07-31 2014-02-05 Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery
EP2940131B1 (en) * 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
EP3221456B1 (en) * 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6836999B2 (ja) 2015-03-24 2021-03-03 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
CA3034089A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
KR20250023600A (ko) 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
WO2019084015A1 (en) * 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
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US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3715358A1 (en) 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
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JP2022551986A (ja) * 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
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Also Published As

Publication number Publication date
WO2003054197A3 (en) 2004-03-18
CA2468882A1 (en) 2003-07-03
AU2008202032A1 (en) 2009-11-26
CA2468882C (en) 2015-09-29
AU2002352261A1 (en) 2003-07-09
WO2003054197A2 (en) 2003-07-03
ES2467156T3 (es) 2014-06-12
EP1456383A2 (en) 2004-09-15
EP1456383B1 (en) 2014-03-12
EP2363487A3 (en) 2012-03-21
US20050106558A1 (en) 2005-05-19
EP2363487A2 (en) 2011-09-07
AU2008202032B2 (en) 2012-09-20
JP2005512569A (ja) 2005-05-12

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