CA2468882C - 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
CA2468882C
CA2468882C CA2468882A CA2468882A CA2468882C CA 2468882 C CA2468882 C CA 2468882C CA 2468882 A CA2468882 A CA 2468882A CA 2468882 A CA2468882 A CA 2468882A CA 2468882 C CA2468882 C CA 2468882C
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Prior art keywords
library
nucleic acid
aav
cells
insert
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Expired - Lifetime
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CA2468882A
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English (en)
French (fr)
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CA2468882A1 (en
Inventor
Luca Perabo
Hildegard Buening
Joerg Enssle
Martin Ried
Michael Hallek
Nadja Huttner
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Medigene AG
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Medigene AG
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Communicable Diseases (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)
CA2468882A 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 - Lifetime CA2468882C (en)

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

Publications (2)

Publication Number Publication Date
CA2468882A1 CA2468882A1 (en) 2003-07-03
CA2468882C true CA2468882C (en) 2015-09-29

Family

ID=27407597

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2468882A Expired - Lifetime CA2468882C (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

Country Status (7)

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

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DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
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
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
ES2602610T3 (es) * 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2098536A1 (en) * 2008-03-05 2009-09-09 4-Antibody AG Isolation and identification of antigen- or ligand-specific binding proteins
WO2010093784A2 (en) 2009-02-11 2010-08-19 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
AU2011317846B2 (en) 2010-10-21 2015-07-09 Deutsches Krebsforschungszentrum Retargeting of rat parvovirus H-1PV to cancer cells through genetic engineering of its capsid
CN105755044A (zh) 2011-04-22 2016-07-13 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及其使用方法
WO2013037961A1 (en) 2011-09-15 2013-03-21 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
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
EP3003391B1 (en) 2013-05-31 2021-09-22 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
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
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
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
JP2020534788A (ja) 2017-08-28 2020-12-03 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 アデノ随伴ウイルスカプシド変異体及びその使用方法
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
WO2019084015A1 (en) * 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
CN111770999A (zh) 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (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
JP2022530457A (ja) 2019-04-26 2022-06-29 サンガモ セラピューティクス, インコーポレイテッド 遺伝子操作aav
AU2020368539A1 (en) * 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome

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US6031071A (en) * 1996-01-24 2000-02-29 Biophage, Inc. Methods of generating novel peptides
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EP1181382B1 (en) * 1999-06-01 2005-03-23 The University of Washington Recombinant adenoviral vectors expressing chimeric fiber proteins for cell specific infection and genome integration
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods

Also Published As

Publication number Publication date
AU2008202032B2 (en) 2012-09-20
CA2468882A1 (en) 2003-07-03
US20050106558A1 (en) 2005-05-19
EP1456383A2 (en) 2004-09-15
ES2467156T3 (es) 2014-06-12
WO2003054197A2 (en) 2003-07-03
EP2363487A2 (en) 2011-09-07
EP1456383B1 (en) 2014-03-12
AU2002352261A1 (en) 2003-07-09
AU2002352261B2 (en) 2008-02-07
AU2008202032A1 (en) 2009-11-26
WO2003054197A3 (en) 2004-03-18
EP2363487A3 (en) 2012-03-21
JP2005512569A (ja) 2005-05-12

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