CA2379564C - Structural protein of adeno-associated virus with modified chromatographic properties, its production and use - Google Patents

Structural protein of adeno-associated virus with modified chromatographic properties, its production and use Download PDF

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Publication number
CA2379564C
CA2379564C CA2379564A CA2379564A CA2379564C CA 2379564 C CA2379564 C CA 2379564C CA 2379564 A CA2379564 A CA 2379564A CA 2379564 A CA2379564 A CA 2379564A CA 2379564 C CA2379564 C CA 2379564C
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Canada
Prior art keywords
structural protein
aav
virus
mutation
protein
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Expired - Fee Related
Application number
CA2379564A
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English (en)
French (fr)
Other versions
CA2379564A1 (en
Inventor
Michael Hallek
Anne Girod
Martin Ried
Christof Stolla
Ulrich Moebius
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Medigene AG
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Medigene AG
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Publication of CA2379564A1 publication Critical patent/CA2379564A1/en
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Publication of CA2379564C publication Critical patent/CA2379564C/en
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

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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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2379564A 1999-07-19 2000-07-18 Structural protein of adeno-associated virus with modified chromatographic properties, its production and use Expired - Fee Related CA2379564C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DE19933719A DE19933719A1 (de) 1999-07-19 1999-07-19 Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
DE19933719.5 1999-07-19
PCT/EP2000/006861 WO2001005991A1 (de) 1999-07-19 2000-07-18 Strukturprotein von adeno-assoziiertem virus mit veränderten chromatographischen eigenschaften, seine herstellung und verwendung

Publications (2)

Publication Number Publication Date
CA2379564A1 CA2379564A1 (en) 2001-01-25
CA2379564C true CA2379564C (en) 2012-03-20

Family

ID=7915238

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2379564A Expired - Fee Related CA2379564C (en) 1999-07-19 2000-07-18 Structural protein of adeno-associated virus with modified chromatographic properties, its production and use

Country Status (9)

Country Link
US (2) US7285381B1 (enExample)
EP (1) EP1198580B1 (enExample)
JP (1) JP4938189B2 (enExample)
AT (1) ATE328101T1 (enExample)
AU (1) AU777885B2 (enExample)
CA (1) CA2379564C (enExample)
DE (2) DE19933719A1 (enExample)
ES (1) ES2263481T3 (enExample)
WO (1) WO2001005991A1 (enExample)

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US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US8313908B2 (en) 2000-02-23 2012-11-20 The Trustees Of The University Of Pennsylvania Regulation of stem cell gene production with specific and selective electric and electromagnetic fields
US7374916B2 (en) 2000-02-23 2008-05-20 The Trustees Of The University Of Pennsylvania Regulation of aggrecan gene expression using specific and selective electrical and electromagnetic signals
US7022506B2 (en) 2000-02-23 2006-04-04 The Trustees Of The University Of Pennsylvania Method and device for treating osteoarthritis, cartilage disease, defects and injuries in the human knee
US7429471B2 (en) 2000-02-23 2008-09-30 The Trustees Of The University Of Pennsylvania Regulation of matrix metalloproteinase gene expression using specific and selective electrical and electromagnetic signals
US7465566B2 (en) 2000-02-23 2008-12-16 The Trustees Of The University Of Pennsylvania Regulation of genes via application of specific and selective electrical and electromagnetic signals
US7465546B2 (en) 2000-02-23 2008-12-16 The Trustees Of The University Of Pennsylvania Regulation of transforming growth factor-beta (TGF-β) gene expression in living cells via the application of specific and selective electric and electromagnetic fields
US7981611B2 (en) 2000-02-23 2011-07-19 The Trustees Of The University Of Pennsylvania Regulation of fibroblastic growth factor-2 (FGF-2) gene expression in living cells with the application of specific and selective electric and electromagnetic fields
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
CN1918285B (zh) 2004-01-12 2013-09-25 宾夕法尼亚大学理事会 通过施加由特异性和选择性的电以及电磁信号产生的场上调骨细胞中骨形态发生蛋白(bmp)基因表达的系统以及方法
US7893985B1 (en) 2004-03-15 2011-02-22 Grandeye Ltd. Wide angle electronic camera with improved peripheral vision
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2158211B1 (en) 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
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
CA2754335C (en) 2009-03-04 2019-12-24 Medigene Ag Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essentially consisting of vp3
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
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
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
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
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
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
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
SI3645551T1 (sl) * 2017-06-27 2024-06-28 Regeneron Pharmaceuticals, Inc. S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice
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.
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
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
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH06507383A (ja) * 1991-01-25 1994-08-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ラミニンのａ鎖のドメイン６のポリペプチド
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5837520A (en) * 1995-03-07 1998-11-17 Canji, Inc. Method of purification of viral vectors
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
WO1997038723A1 (en) * 1996-04-16 1997-10-23 Immusol Incorporated Targeted viral vectors
US20010031463A1 (en) 1997-09-02 2001-10-18 Jurgen Kleinschmidt Method for purifying and concentrating AAV-2 and antigen portions thereof
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
EP1135468B1 (en) * 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods

Also Published As

Publication number Publication date
CA2379564A1 (en) 2001-01-25
US20080241906A1 (en) 2008-10-02
EP1198580B1 (de) 2006-05-31
WO2001005991A1 (de) 2001-01-25
EP1198580A1 (de) 2002-04-24
ES2263481T3 (es) 2006-12-16
JP4938189B2 (ja) 2012-05-23
US20100105123A2 (en) 2010-04-29
JP2003505033A (ja) 2003-02-12
US7285381B1 (en) 2007-10-23
DE19933719A1 (de) 2001-01-25
DE50012863D1 (de) 2006-07-06
US20090098634A2 (en) 2009-04-16
AU777885B2 (en) 2004-11-04
AU6435400A (en) 2001-02-05
ATE328101T1 (de) 2006-06-15
US20090246854A2 (en) 2009-10-01

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Effective date: 20180718