WO2000026254A3 - An das aav-kapsid bindender, den zelltropismus verändernder antikörper und verfahren zum gerichteten gentransfer - Google Patents

An das aav-kapsid bindender, den zelltropismus verändernder antikörper und verfahren zum gerichteten gentransfer Download PDF

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Publication number
WO2000026254A3
WO2000026254A3 PCT/DE1999/003517 DE9903517W WO0026254A3 WO 2000026254 A3 WO2000026254 A3 WO 2000026254A3 DE 9903517 W DE9903517 W DE 9903517W WO 0026254 A3 WO0026254 A3 WO 0026254A3
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WIPO (PCT)
Prior art keywords
antibodies
binding
cytotropism
gene transfer
targeted gene
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PCT/DE1999/003517
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English (en)
French (fr)
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WO2000026254A2 (de
Inventor
Juergen Kleinschmidt
Christiane Wobus
Andrea Kern
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Deutsches Krebsforsch
Juergen Kleinschmidt
Christiane Wobus
Andrea Kern
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.)
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Publication date
Application filed by Deutsches Krebsforsch, Juergen Kleinschmidt, Christiane Wobus, Andrea Kern filed Critical Deutsches Krebsforsch
Priority to EP99963196A priority Critical patent/EP1124854A2/de
Priority to AU19626/00A priority patent/AU1962600A/en
Publication of WO2000026254A2 publication Critical patent/WO2000026254A2/de
Publication of WO2000026254A3 publication Critical patent/WO2000026254A3/de

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/626—Diabody or triabody
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/859—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from immunoglobulins

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
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  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

Die Erfindung betrifft monoklonale Antikörper oder Fragmente davon, die an das Kapsid von Adeno-assoziierten Viren binden, wodurch die Bindung des Virus an den Virusrezeptor der ursprünglichen Zielzelle verhindert wird. Dieser Antikörper oder das Fragment davon können außerdem mit einem gewünschten Rezeptorliganden fusioniert werden. Nach Bindung eines solchen Antikörpers an das AAV-Kapsid wird ein AAV erhalten, das über einen veränderten Tropismus verfügt, also - in Abhängigkeit von dem fusionierten Liganden - an eine neue Zielzelle binden kann und zur Konstruktion eines AAV-Vektors für gerichteten Gentransfer verwendet werden kann.
PCT/DE1999/003517 1998-10-29 1999-10-29 An das aav-kapsid bindender, den zelltropismus verändernder antikörper und verfahren zum gerichteten gentransfer WO2000026254A2 (de)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP99963196A EP1124854A2 (de) 1998-10-29 1999-10-29 An das aav-kapsid bindender, den zelltropismus verändernder antikörper und verfahren zum gerichteten gentransfer
AU19626/00A AU1962600A (en) 1998-10-29 1999-10-29 Antibodies binding to the aav capsid, antibodies modifying cytotropism, method for targeted gene transfer

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE19849643.5 1998-10-29
DE1998149643 DE19849643A1 (de) 1998-10-29 1998-10-29 An das AAV-Kapsid bindender, den Zelltropismus verändernder Antikörper und Verfahren zum gerichteten Gentransfer

Publications (2)

Publication Number Publication Date
WO2000026254A2 WO2000026254A2 (de) 2000-05-11
WO2000026254A3 true WO2000026254A3 (de) 2000-08-03

Family

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Application Number Title Priority Date Filing Date
PCT/DE1999/003517 WO2000026254A2 (de) 1998-10-29 1999-10-29 An das aav-kapsid bindender, den zelltropismus verändernder antikörper und verfahren zum gerichteten gentransfer

Country Status (4)

Country Link
EP (1) EP1124854A2 (de)
AU (1) AU1962600A (de)
DE (1) DE19849643A1 (de)
WO (1) WO2000026254A2 (de)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
CA2745131C (en) 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
AU2001277531A1 (en) * 2000-07-10 2002-01-21 Novartis Ag Bifunctional molecules and vectors complexed therewith for targeted gene delivery
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
WO2005056807A2 (en) * 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
CN110799524A (zh) * 2017-06-27 2020-02-14 瑞泽恩制药公司 向性修饰的重组病毒载体及其用于将遗传材料靶向引入人细胞内的用途
CN112159467A (zh) * 2020-09-14 2021-01-01 和元生物技术（上海）股份有限公司 能够与aav1-13结合的抗体
CN113583112B (zh) * 2021-07-30 2022-07-19 上海勉亦生物科技有限公司 Aav特异性抗体及其应用
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN117285620B (zh) * 2023-11-27 2024-02-13 恺佧生物科技(上海)有限公司 抗aav9抗体及aav9滴度测定elisa试剂盒

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995011997A1 (en) * 1993-10-28 1995-05-04 Deutsches Krebsforschungszentrum, Stiftung Des Öffentlichen Rechts Adeno-associated virus - its diagnostic use with early abortion
WO1999067393A2 (de) * 1998-06-19 1999-12-29 Medigene Aktiengesellschaft Strukturprotein von aav, seine herstellung und verwendung

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995011997A1 (en) * 1993-10-28 1995-05-04 Deutsches Krebsforschungszentrum, Stiftung Des Öffentlichen Rechts Adeno-associated virus - its diagnostic use with early abortion
WO1999067393A2 (de) * 1998-06-19 1999-12-29 Medigene Aktiengesellschaft Strukturprotein von aav, seine herstellung und verwendung

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BARTLETT J ET AL: "Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody.", NATURE BIOTECHNOLOGY, (1999 FEB) 17, 181-6, XP002135910 *
WISTUBA A ET AL: "Intermediates of Adeno-Associated Virus type 2 assembly: Identification of soluble complexes containing Rep and Cap proteins.", JOURNAL OF VIROLOGY, (1995 SEP) 69 (9) 5311-9, XP002135909 *

Also Published As

Publication number Publication date
DE19849643A1 (de) 2000-05-04
EP1124854A2 (de) 2001-08-22
AU1962600A (en) 2000-05-22
WO2000026254A2 (de) 2000-05-11

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