AU2001268373A1 - Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus - Google Patents

Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus

Info

Publication number
AU2001268373A1
AU2001268373A1 AU2001268373A AU6837301A AU2001268373A1 AU 2001268373 A1 AU2001268373 A1 AU 2001268373A1 AU 2001268373 A AU2001268373 A AU 2001268373A AU 6837301 A AU6837301 A AU 6837301A AU 2001268373 A1 AU2001268373 A1 AU 2001268373A1
Authority
AU
Australia
Prior art keywords
associated virus
adeno
methods
previously exposed
virions
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
AU2001268373A
Inventor
Katherine A. High
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.)
Childrens Hospital of Philadelphia CHOP
Original Assignee
Childrens Hospital of Philadelphia CHOP
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 Childrens Hospital of Philadelphia CHOP filed Critical Childrens Hospital of Philadelphia CHOP
Publication of AU2001268373A1 publication Critical patent/AU2001268373A1/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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/23—Parvoviridae, e.g. feline panleukopenia virus
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • 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
    • C12N2750/14134—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
AU2001268373A 2000-06-13 2001-06-13 Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus Abandoned AU2001268373A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US21106600P 2000-06-13 2000-06-13
US60211066 2000-06-13
PCT/US2001/018965 WO2001096587A2 (en) 2000-06-13 2001-06-13 Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus

Publications (1)

Publication Number Publication Date
AU2001268373A1 true AU2001268373A1 (en) 2001-12-24

Family

ID=22785464

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001268373A Abandoned AU2001268373A1 (en) 2000-06-13 2001-06-13 Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus

Country Status (3)

Country Link
US (2) US20020106381A1 (en)
AU (1) AU2001268373A1 (en)
WO (1) WO2001096587A2 (en)

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JP2004532822A (en) * 2001-03-14 2004-10-28 アビジェン， インコーポレイテッド Recombinant adeno-associated virus-mediated gene transfer by retrograde virion infection.
US20040009151A1 (en) * 2002-04-04 2004-01-15 Kay Mark A. Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal
GB0324044D0 (en) * 2003-10-14 2003-11-19 Astrazeneca Ab Protein
JP5461008B2 (en) * 2005-04-13 2014-04-02 アストラゼネカ アクチボラグ vector
ES2398593T3 (en) * 2006-07-25 2013-03-20 Celladon Corporation Prolonged antegrade epicardial coronary infusion of adeno-associated viral vectors comprising SERCA2a for gene therapy
US8206967B2 (en) 2007-07-06 2012-06-26 Medimmune Limited Method for production of recombinant human thrombin
JP2011512408A (en) 2008-02-19 2011-04-21 セラドン・コーポレーション Composition for increasing viral vector uptake into the myocardium
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
KR102599909B1 (en) 2014-11-14 2023-11-09 보이저 테라퓨틱스, 인크. Compositions and methods of treating amyotrophic lateral sclerosis (als)
DK3218386T3 (en) 2014-11-14 2021-06-07 Voyager Therapeutics Inc MODULATORY POLYNUCLEOTID
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
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
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
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
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
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)
US20210395776A1 (en) 2018-09-28 2021-12-23 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN117120619A (en) 2020-07-27 2023-11-24 沃雅戈治疗公司 Compositions and methods for treating neurological disorders associated with a deficiency in glucosylceramidase beta
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders

Family Cites Families (10)

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US6268213B1 (en) * 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
WO1994026915A1 (en) * 1993-05-10 1994-11-24 The Regents Of The University Of Michigan Gene transfer into pancreatic and biliary epithelial cells
US20010009904A1 (en) * 1997-12-30 2001-07-26 Jon A. Wolff Process of delivering a polynucleotide to a cell via the vascular system
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US20030215422A1 (en) * 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
WO1998024479A1 (en) * 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6093392A (en) * 1997-03-14 2000-07-25 Childrens Hospital Of Phildelphia Methods and compositions for use in gene therapy for treatment of hemophilia
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
JP2004532822A (en) * 2001-03-14 2004-10-28 アビジェン， インコーポレイテッド Recombinant adeno-associated virus-mediated gene transfer by retrograde virion infection.

Also Published As

Publication number Publication date
WO2001096587A8 (en) 2002-04-04
WO2001096587A3 (en) 2002-05-16
US20050281784A1 (en) 2005-12-22
WO2001096587A2 (en) 2001-12-20
US20020106381A1 (en) 2002-08-08

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