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WO2000017377A3 - Methods for large-scale production of recombinant aav vectors - Google Patents

Methods for large-scale production of recombinant aav vectors Download PDF

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Publication number
WO2000017377A3
WO2000017377A3 PCT/US1999/022052 US9922052W WO0017377A3 WO 2000017377 A3 WO2000017377 A3 WO 2000017377A3 US 9922052 W US9922052 W US 9922052W WO 0017377 A3 WO0017377 A3 WO 0017377A3
Authority
WO
WIPO (PCT)
Prior art keywords
raav
methods
cells
genomes
scale production
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
PCT/US1999/022052
Other languages
French (fr)
Other versions
WO2000017377A2 (en
Inventor
Barry J Byrne
James E Conway
Gary S Hayward
Nicholas Muzyczka
Sergei Zolotukhin
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.)
University of Florida
Johns Hopkins University
Original Assignee
University of Florida
Johns Hopkins University
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 University of Florida, Johns Hopkins University filed Critical University of Florida
Priority to NZ511171A priority Critical patent/NZ511171A/en
Priority to EP99954648A priority patent/EP1115880A2/en
Priority to AU10949/00A priority patent/AU774706B2/en
Priority to CA002347129A priority patent/CA2347129A1/en
Publication of WO2000017377A2 publication Critical patent/WO2000017377A2/en
Publication of WO2000017377A3 publication Critical patent/WO2000017377A3/en
Anticipated expiration legal-status Critical
Priority to AU2004218673A priority patent/AU2004218673A1/en
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643—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
    • C12N2750/14143—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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/108—Plasmid DNA episomal vectors

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Disclosed are HSV-1 amplicons that supply all necessary helper functions required for rAAV packaging and methods for their use. These HSV-1 amplicons have been shown to be capable of rescuing and replicating all forms of rAAV genomes including rAAV genomes introduced into cells by infection of rAAV virions, rAAV genomes transfected into cells on plasmids or proviral rAAV genomes integrated into cellular chromosomal DNA. Also provided are methods for preparing high-titer rAAV vector compositions suitable for gene therapy and the delivery of exogenous polynucleotides to selected host cells.
PCT/US1999/022052 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors Ceased WO2000017377A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
NZ511171A NZ511171A (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant AAV vectors
EP99954648A EP1115880A2 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors
AU10949/00A AU774706B2 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant AAV vectors
CA002347129A CA2347129A1 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors
AU2004218673A AU2004218673A1 (en) 1998-09-22 2004-10-07 Methods for large-scale production of recombinant AAV vectors

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US10150798P 1998-09-22 1998-09-22
US60/101,507 1998-09-22

Publications (2)

Publication Number Publication Date
WO2000017377A2 WO2000017377A2 (en) 2000-03-30
WO2000017377A3 true WO2000017377A3 (en) 2000-09-08

Family

ID=22285008

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/022052 Ceased WO2000017377A2 (en) 1998-09-22 1999-09-22 Methods for large-scale production of recombinant aav vectors

Country Status (5)

Country Link
EP (1) EP1115880A2 (en)
AU (2) AU774706B2 (en)
CA (1) CA2347129A1 (en)
NZ (2) NZ511171A (en)
WO (1) WO2000017377A2 (en)

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WO2001025253A2 (en) 1999-10-01 2001-04-12 The University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
EP2336339A3 (en) * 2000-09-25 2011-09-14 The Regents of the University of Michigan Production of viral vectors
AU2003272868A1 (en) * 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
ES2634118T3 (en) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Modified virus vectors and methods to manufacture and use them
ES2683695T3 (en) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Inverse restrictive terminal repeats for viral vectors
EP4234571A3 (en) 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
JP6387350B2 (en) 2012-09-28 2018-09-05 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Adeno-associated virus vector targeting oligodendrocytes
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CA2904396A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
RU2727015C2 (en) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Aav vectors aimed at the central nervous system
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
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MX2018009426A (en) 2016-02-22 2018-12-19 Univ North Carolina Chapel Hill Aav-idua vector for treatment of mps i-associated blindness.
JP2019517274A (en) 2016-06-13 2019-06-24 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ Optimized CLN1 genes and expression cassettes and their use
CA3028786A1 (en) 2016-07-26 2018-02-01 The University Of North Carolina At Chapel Hill Vector-mediated immune tolerance in the eye
EP4303225B1 (en) 2017-03-15 2025-11-26 The University of North Carolina at Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
WO2019094061A1 (en) 2017-11-07 2019-05-16 The University Of North Carolina At Chapel Hill Optimized aga genes and expression cassettes and their use
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
CN112041442A (en) 2018-02-28 2020-12-04 北卡罗来纳大学查佩尔希尔分校 Methods and compositions for viral vectors to evade antibodies
CN112543766A (en) 2018-04-03 2021-03-23 斯特里迪比奥公司 Antibody-evasive viral vectors
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
GB201806333D0 (en) * 2018-04-18 2018-05-30 Glaxosmithkline Ip Dev Ltd Parvovirus vector production
EP4659766A2 (en) 2018-06-12 2025-12-10 The University of North Carolina at Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
BR112020025268A2 (en) 2018-06-28 2021-03-09 The University Of North Carolina At Chapel Hill OPTIMIZED EXPRESSION CASSETS AND GENES CLN5 AND THEIR USE
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimization of CLN7 genes and expression cassettes and their use
WO2020097002A1 (en) 2018-11-05 2020-05-14 The University Of North Carolina At Chapel Hill Optimized fig4 genes and expression cassettes and their use
EP3884057A4 (en) 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill OPTIMIZED GALC GENES AND EXPRESSION CASSETTES AND THEIR USE
GB201901571D0 (en) 2019-02-05 2019-03-27 Oxford Genetics Ltd Inducible AAV sysyem
WO2020174368A1 (en) 2019-02-25 2020-09-03 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
CN113727992A (en) 2019-03-21 2021-11-30 斯特里迪比奥公司 Recombinant adeno-associated virus vector
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JP7616668B2 (en) 2019-05-22 2025-01-17 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル UBE3A GENE AND EXPRESSION CASSETTES AND USES THEREOF
TW202128736A (en) 2019-10-17 2021-08-01 美商史崔德生物公司 Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3169548A1 (en) 2020-03-23 2021-09-30 Haiyan Fu Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
US20230257771A1 (en) 2020-04-20 2023-08-17 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
JP2023524577A (en) 2020-05-05 2023-06-12 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Modified adeno-associated virus 5 capsid and uses thereof
US20230285595A1 (en) 2020-07-23 2023-09-14 The University Of North Carolina At Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
TW202227632A (en) 2020-08-19 2022-07-16 美商史崔德生物公司 Adeno-associated virus vectors for treatment of rett syndrome
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Title
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DATABASE WPI Section Ch Week 199933, Derwent World Patents Index; Class B04, AN 1999-386309, XP002136602 *
SHU YUELONG ET AL: "A novel recombinant adeno-associated virus vector packaging system with HSV-1, amplicon providing helper functions.", SCIENCE IN CHINA SERIES C LIFE SCIENCES, vol. 42, no. 5, October 1999 (1999-10-01), pages 465 - 470, XP002136601, ISSN: 1006-9305 *
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Also Published As

Publication number Publication date
AU2004218673A1 (en) 2004-11-04
NZ511171A (en) 2004-02-27
WO2000017377A2 (en) 2000-03-30
AU1094900A (en) 2000-04-10
CA2347129A1 (en) 2000-03-30
AU774706B2 (en) 2004-07-08
NZ528942A (en) 2005-03-24
EP1115880A2 (en) 2001-07-18

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