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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Prior art keywords
structural protein
aav
virus
mutation
protein
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Expired - Fee Related
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CA2379564A
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English (en)
French (fr)
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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
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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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or 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
DE19933719.5 1999-07-19
DE19933719A DE19933719A1 (de) 1999-07-19 1999-07-19 Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
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 (https=)
EP (1) EP1198580B1 (https=)
JP (1) JP4938189B2 (https=)
AT (1) ATE328101T1 (https=)
AU (1) AU777885B2 (https=)
CA (1) CA2379564C (https=)
DE (2) DE19933719A1 (https=)
ES (1) ES2263481T3 (https=)
WO (1) WO2001005991A1 (https=)

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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
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
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
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
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
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
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
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
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
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
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
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
PL2403867T3 (pl) 2009-03-04 2019-12-31 Deutsches Krebsforschungszentrum Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z 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
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
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
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
KR102537394B1 (ko) 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
MY187898A (en) 2015-03-02 2021-10-27 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
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
EP3827812B1 (en) 2016-07-29 2025-10-29 The Regents of the University of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
MX2020000244A (es) * 2017-06-27 2020-09-28 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.
WO2019006182A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS 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
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions

Family Cites Families (10)

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WO1992012727A1 (en) * 1991-01-25 1992-08-06 Regents Of The University Of Minnesota Laminin a chain domain vi polypeptides
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
AU722196B2 (en) * 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
CA2251738A1 (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
ES2340230T3 (es) * 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods

Also Published As

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

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Effective date: 20180718