WO2003010320A3 - Recombinant vector derived from adeno-associated virus for gene therapy - Google Patents

Recombinant vector derived from adeno-associated virus for gene therapy Download PDF

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Publication number
WO2003010320A3
WO2003010320A3 PCT/BE2002/000128 BE0200128W WO03010320A3 WO 2003010320 A3 WO2003010320 A3 WO 2003010320A3 BE 0200128 W BE0200128 W BE 0200128W WO 03010320 A3 WO03010320 A3 WO 03010320A3
Authority
WO
WIPO (PCT)
Prior art keywords
adeno
associated virus
gene therapy
recombinant vector
vector derived
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/BE2002/000128
Other languages
French (fr)
Other versions
WO2003010320A2 (en
Inventor
Greve Jacques De
Erik Teugels
Bart Neyns
Ziad Zeinoun
Joanna Vermeij
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.)
Vrije Universiteit Brussel VUB
Universite Libre de Bruxelles ULB
Original Assignee
Vrije Universiteit Brussel VUB
Universite Libre de Bruxelles ULB
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 Vrije Universiteit Brussel VUB, Universite Libre de Bruxelles ULB filed Critical Vrije Universiteit Brussel VUB
Priority to EP02759936A priority Critical patent/EP1419260A2/en
Priority to US10/483,946 priority patent/US20050074886A1/en
Priority to AU2002325683A priority patent/AU2002325683A1/en
Publication of WO2003010320A2 publication Critical patent/WO2003010320A2/en
Anticipated expiration legal-status Critical
Publication of WO2003010320A3 publication Critical patent/WO2003010320A3/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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/495—Transforming growth factor [TGF]
    • 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
    • C12N2750/14141—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention is related to a recombinant adeno-associated viral construct comprising at least:- a first terminal repeat of an Adeno Associated Virus- a strong heterologous promoter- an heterologous DNA with at least 90% homology to the gene encoding for a constitutively activated TGF-b1 peptide, said gene being under the control of said promoter- a polyadenylation signal - a second terminal repeat of an Adeno Associated Virus.
PCT/BE2002/000128 2001-07-26 2002-07-26 Recombinant vector derived from adeno-associated virus for gene therapy Ceased WO2003010320A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP02759936A EP1419260A2 (en) 2001-07-26 2002-07-26 Recombinant vector derived from adeno-associated virus for gene therapy
US10/483,946 US20050074886A1 (en) 2001-07-26 2002-07-26 Recombinant vector derived from adeno-associated virus for gene therapy
AU2002325683A AU2002325683A1 (en) 2001-07-26 2002-07-26 Recombinant vector derived from adeno-associated virus for gene therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP01870167A EP1279740A1 (en) 2001-07-26 2001-07-26 Recombinant vector derived from adeno-associated virus for gene therapy
EP01870167.2 2001-07-26

Publications (2)

Publication Number Publication Date
WO2003010320A2 WO2003010320A2 (en) 2003-02-06
WO2003010320A3 true WO2003010320A3 (en) 2004-02-26

Family

ID=8185005

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/BE2002/000128 Ceased WO2003010320A2 (en) 2001-07-26 2002-07-26 Recombinant vector derived from adeno-associated virus for gene therapy

Country Status (4)

Country Link
US (1) US20050074886A1 (en)
EP (2) EP1279740A1 (en)
AU (1) AU2002325683A1 (en)
WO (1) WO2003010320A2 (en)

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Publication number Priority date Publication date Assignee Title
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (en) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Aadc polynucleotides for treating parkinson's disease
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20230145206A (en) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
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 DETECTION OF NEUTRALIZING ANTIBODIES OF VAA
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102427379B1 (en) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. Compositions and methods for treating Huntington's disease
KR20240056729A (en) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518259A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Huntington's disease treatment compositions and methods
JP2020518258A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Amyotrophic lateral sclerosis (ALS) treatment composition and method
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
TW202413649A (en) 2017-10-16 2024-04-01 美商航海家醫療公司 Treatment of amyotrophic lateral sclerosis (als)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR20210019996A (en) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. Composition and method for the treatment of Parkinson's disease
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0373994A1 (en) * 1988-12-16 1990-06-20 Oncogen Limited Partnership Mutants of simian transforming growth factor-beta 1
WO1995034670A2 (en) * 1994-06-06 1995-12-21 Children's Hospital, Inc. Recombinant aav genome encoding immunodeficiency virus protein
WO1998013070A1 (en) * 1996-09-25 1998-04-02 Cell Genesys, Inc. Non-invasive administration of adeno-associated viral vectors

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5588298A (en) * 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0373994A1 (en) * 1988-12-16 1990-06-20 Oncogen Limited Partnership Mutants of simian transforming growth factor-beta 1
WO1995034670A2 (en) * 1994-06-06 1995-12-21 Children's Hospital, Inc. Recombinant aav genome encoding immunodeficiency virus protein
WO1998013070A1 (en) * 1996-09-25 1998-04-02 Cell Genesys, Inc. Non-invasive administration of adeno-associated viral vectors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ZEINOUN Z. ET AL.,: "Construction of a TGF-b1 carrying vector which induces growth inhibition in epithelial cancer cells", PORC. OF THE AMERICAN ASSOCIATION FOR CANCER RESEARCH, vol. 40, - March 1999 (1999-03-01), pages 174 - 175, XP001026169 *
ZEINOUN Z. ET AL.,: "Insufficient TGF-b1 production inactivates the autocrine growth suppressive circuit in human ovarian cancer cell lines", ANTICANCER RESEARCH, vol. 19, no. 1a, January 1999 (1999-01-01), pages 413 - 420, XP001036877 *

Also Published As

Publication number Publication date
EP1279740A1 (en) 2003-01-29
AU2002325683A1 (en) 2003-02-17
EP1419260A2 (en) 2004-05-19
US20050074886A1 (en) 2005-04-07
WO2003010320A2 (en) 2003-02-06

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