AU2001287720A1 - Host cells for packing a recombinant adeno-associated virus (RAAV), method for the production and use thereof - Google Patents

Host cells for packing a recombinant adeno-associated virus (RAAV), method for the production and use thereof

Info

Publication number
AU2001287720A1
AU2001287720A1 AU2001287720A AU8772001A AU2001287720A1 AU 2001287720 A1 AU2001287720 A1 AU 2001287720A1 AU 2001287720 A AU2001287720 A AU 2001287720A AU 8772001 A AU8772001 A AU 8772001A AU 2001287720 A1 AU2001287720 A1 AU 2001287720A1
Authority
AU
Australia
Prior art keywords
raav
packing
production
host cells
associated virus
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.)
Abandoned
Application number
AU2001287720A
Inventor
Joan Bertran
Markus Horer
Ulrich Moebius
Bernd Rehberger
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.)
Medigene AG
Original Assignee
Medigene AG
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 Medigene AG filed Critical Medigene AG
Publication of AU2001287720A1 publication Critical patent/AU2001287720A1/en
Abandoned 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
AU2001287720A 2000-09-08 2001-09-07 Host cells for packing a recombinant adeno-associated virus (RAAV), method for the production and use thereof Abandoned AU2001287720A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DE10044384.2 2000-09-08
DE10044384A DE10044384A1 (en) 2000-09-08 2000-09-08 Host cells for packaging recombinant adeno-associated virus (rAAV), process for their preparation and their use
PCT/EP2001/010370 WO2002020748A2 (en) 2000-09-08 2001-09-07 Host cells for packing a recombinant adeno-associated virus (raav), method for the production and use thereof

Publications (1)

Publication Number Publication Date
AU2001287720A1 true AU2001287720A1 (en) 2002-03-22

Family

ID=7655468

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001287720A Abandoned AU2001287720A1 (en) 2000-09-08 2001-09-07 Host cells for packing a recombinant adeno-associated virus (RAAV), method for the production and use thereof

Country Status (7)

Country Link
US (1) US20040087026A1 (en)
EP (1) EP1315798A2 (en)
JP (1) JP2004508041A (en)
AU (1) AU2001287720A1 (en)
CA (1) CA2421442A1 (en)
DE (2) DE10066104A1 (en)
WO (1) WO2002020748A2 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110423776A (en) * 2019-06-20 2019-11-08 广州辉园苑医药科技有限公司 The carrier and its construction method of expression recombinant blood coagulation factor FIX and application
CN113355292A (en) * 2021-06-04 2021-09-07 天康制药(苏州)有限公司 Porcine circovirus gene modified attenuated strain, construction method and application thereof

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EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN112553229A (en) 2014-11-05 2021-03-26 沃雅戈治疗公司 AADC polynucleotides for treating Parkinson's disease
DK3218386T3 (en) 2014-11-14 2021-06-07 Voyager Therapeutics Inc MODULATORY POLYNUCLEOTID
KR102599909B1 (en) 2014-11-14 2023-11-09 보이저 테라퓨틱스, 인크. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
CN109195636B (en) * 2016-03-03 2022-07-05 马萨诸塞大学 End-blocked linear duplex DNA for non-viral gene transfer
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
AU2017321488B2 (en) 2016-08-30 2022-10-20 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020507331A (en) 2017-02-17 2020-03-12 ロンザ リミテッドＬｏｎｚａ Ｌｉｍｉｔｅｄ Mammalian cells for producing adeno-associated virus
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
CN108103102B (en) * 2017-05-09 2021-04-13 北京锦篮基因科技有限公司 AAV1 virus-mediated skeletal muscle specific PCK1 gene expression vector and application thereof
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
CA3071978A1 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019051289A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Lipid nanoparticle formulations of non-viral, capsid-free dna vectors
MA50100A (en) * 2017-09-08 2020-07-15 Generation Bio Co MODIFIED CLOSED END DNA (CEDNA)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TWI804518B (en) 2017-10-16 2023-06-11 美商航海家醫療公司 Treatment of amyotrophic lateral sclerosis (als)
JPWO2022172986A1 (en) * 2021-02-12 2022-08-18
WO2023086822A2 (en) * 2021-11-09 2023-05-19 Asimov Inc. Stable production systems for aav vector production

Family Cites Families (9)

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Publication number Priority date Publication date Assignee Title
IL116816A (en) * 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
EP1359223A3 (en) * 1995-08-03 2007-09-05 Avigen, Inc. Recombinant AAV preparation substantially free of wt aav virus
JP2001506133A (en) * 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション AAV split-packaging genes and cell lines containing such genes for use in producing recombinant AAV vectors
WO1999014354A1 (en) * 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
CA2304168A1 (en) * 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
EP1025251A1 (en) * 1997-10-21 2000-08-09 Targeted Genetics Corporation Amplifiable adeno-associated virus (aav) packaging cassettes for the production of recombinant aav vectors
US6642051B1 (en) * 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
DE19905501B4 (en) * 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie A method of producing a recombinant adeno-associated virus, suitable compositions therefor, and use for the manufacture of a medicament

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110423776A (en) * 2019-06-20 2019-11-08 广州辉园苑医药科技有限公司 The carrier and its construction method of expression recombinant blood coagulation factor FIX and application
CN110423776B (en) * 2019-06-20 2023-04-11 广州辉园苑医药科技有限公司 Vector for expressing recombinant blood coagulation factor FIX and construction method and application thereof
CN113355292A (en) * 2021-06-04 2021-09-07 天康制药(苏州)有限公司 Porcine circovirus gene modified attenuated strain, construction method and application thereof
CN113355292B (en) * 2021-06-04 2024-02-06 天康制药股份有限公司 Porcine circovirus gene modified attenuated strain, construction method and application thereof

Also Published As

Publication number Publication date
WO2002020748A2 (en) 2002-03-14
WO2002020748A3 (en) 2003-03-20
WO2002020748A8 (en) 2004-03-04
US20040087026A1 (en) 2004-05-06
DE10066104A1 (en) 2003-01-09
DE10044384A1 (en) 2002-04-18
EP1315798A2 (en) 2003-06-04
JP2004508041A (en) 2004-03-18
CA2421442A1 (en) 2003-03-06

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Legal Events

Date Code Title Description
MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application