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

Info

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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (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
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 (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=)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
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
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
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
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
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
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
JP4801802B2 (ja) 2004-01-12 2011-10-26 ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア 特定の選択的な電気信号および電磁信号によって生成される電場の印加を介して骨細胞における骨形成タンパク質（ｂｍｐ）遺伝子発現を上方制御するシステムおよび方法
US7893985B1 (en) 2004-03-15 2011-02-22 Grandeye Ltd. Wide angle electronic camera with improved peripheral vision
JP5136766B2 (ja) 2004-12-15 2013-02-06 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 キメラベクター
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
ES2742180T3 (es) 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en 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
CN105755044A (zh) 2011-04-22 2016-07-13 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及其使用方法
WO2013037961A1 (en) 2011-09-15 2013-03-21 Medigene Ag Anti-her2 vaccine based upon aav derived multimeric structures
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US11021519B2 (en) 2015-03-02 2021-06-01 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
AU2017302013B2 (en) 2016-07-29 2022-05-26 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
PE20200488A1 (es) * 2017-06-27 2020-03-03 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids 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
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0567578A1 (en) * 1991-01-25 1993-11-03 The 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
WO1997008298A1 (en) * 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
EP0927044A4 (en) * 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET 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
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The 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
US20100105123A2 (en) 2010-04-29
AU777885B2 (en) 2004-11-04
US7285381B1 (en) 2007-10-23
US20090098634A2 (en) 2009-04-16
JP4938189B2 (ja) 2012-05-23
DE50012863D1 (de) 2006-07-06
JP2003505033A (ja) 2003-02-12
EP1198580B1 (de) 2006-05-31
DE19933719A1 (de) 2001-01-25
US20090246854A2 (en) 2009-10-01
AU6435400A (en) 2001-02-05
WO2001005991A1 (de) 2001-01-25
EP1198580A1 (de) 2002-04-24
ATE328101T1 (de) 2006-06-15
ES2263481T3 (es) 2006-12-16
US20080241906A1 (en) 2008-10-02

Similar Documents

Publication Publication Date Title
CA2379564C (en) Structural protein of adeno-associated virus with modified chromatographic properties, its production and use
US7314912B1 (en) AAv scleroprotein, production and use thereof
CA2332115C (en) Aav structural protein, its preparation and use
US11261463B2 (en) Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
CA2379220C (en) Structural protein of adeno-associated virus with modified antigenicity, its production and its use
Lee et al. Adeno-associated virus (AAV) vectors: Rational design strategies for capsid engineering
Rabinowitz et al. Insertional mutagenesis of AAV2 capsid and the production of recombinant virus
Büning et al. Recent developments in adeno‐associated virus vector technology
AU780231B2 (en) Virus vectors and methods of making and administering the same
Wu et al. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism
EP3773746A1 (en) Aav6 variants
Bhrigu Replication of adeno-associated virus in murine fibroblasts with mouse adenovirus provided helper functions

Legal Events

Date Code Title Description
EEER Examination request
MKLA Lapsed

Effective date: 20180718