HK1210214A1 - Modified adeno-associated virus vector compositions - Google Patents

Modified adeno-associated virus vector compositions

Info

Publication number
HK1210214A1
HK1210214A1 HK15110883.2A HK15110883A HK1210214A1 HK 1210214 A1 HK1210214 A1 HK 1210214A1 HK 15110883 A HK15110883 A HK 15110883A HK 1210214 A1 HK1210214 A1 HK 1210214A1
Authority
HK
Hong Kong
Prior art keywords
associated virus
virus vector
vector compositions
modified adeno
adeno
Prior art date
Application number
HK15110883.2A
Other languages
English (en)
Chinese (zh)
Inventor
Beverly L Davidson
Maria Scheel
Ryan Boudreau
Original Assignee
Univ Iowa Res Found
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 Univ Iowa Res Found filed Critical Univ Iowa Res Found
Publication of HK1210214A1 publication Critical patent/HK1210214A1/xx

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/38Vector systems having a special element relevant for transcription being a stuffer

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HK15110883.2A 2012-07-06 2015-11-04 Modified adeno-associated virus vector compositions HK1210214A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261668839P 2012-07-06 2012-07-06
PCT/US2013/031644 WO2014007858A1 (fr) 2012-07-06 2013-03-14 Compositions de vecteur viral adéno-associé modifiées

Publications (1)

Publication Number Publication Date
HK1210214A1 true HK1210214A1 (en) 2016-04-15

Family

ID=49882407

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15110883.2A HK1210214A1 (en) 2012-07-06 2015-11-04 Modified adeno-associated virus vector compositions

Country Status (12)

Country Link
US (2) US9540659B2 (fr)
EP (1) EP2870237B1 (fr)
JP (1) JP6366581B2 (fr)
CN (1) CN104619832B (fr)
AU (1) AU2013287261B2 (fr)
BR (1) BR112015000161A2 (fr)
CA (1) CA2878401C (fr)
ES (1) ES2703177T3 (fr)
HK (1) HK1210214A1 (fr)
IN (1) IN2015KN00074A (fr)
RU (1) RU2679843C2 (fr)
WO (1) WO2014007858A1 (fr)

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AU2013287261B2 (en) * 2012-07-06 2019-03-07 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
CA2949437C (fr) * 2014-05-20 2023-08-15 University Of Iowa Research Foundation Composes therapeutiques pour la maladie de huntington
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
JP2017535266A (ja) 2014-11-14 2017-11-30 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. 筋萎縮性側索硬化症(als)を治療する組成物および方法
EP3230441A4 (fr) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
WO2017023724A1 (fr) * 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions et méthodes pour le traitement d'une déficience en aadc
WO2017161273A1 (fr) * 2016-03-18 2017-09-21 The Children's Hospital Of Philadelphia Agent thérapeutique pour le traitement de maladies, notamment les maladies touchant le système nerveux central
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
WO2019079242A1 (fr) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
EP4124658A3 (fr) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
CA3094311A1 (fr) 2018-04-03 2019-10-10 Stridebio, Inc. Vecteurs de virus evitant les anticorps
AU2019299861A1 (en) * 2018-07-02 2021-01-14 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
SG11202112078TA (en) 2019-05-14 2021-11-29 Univ Duke Compositions and methods for the treatment of atpase-mediated diseases
CA3157700A1 (fr) 2019-10-17 2021-04-22 Stridebio, Inc. Vecteurs de virus adeno-associes pour le traitement de la maladie de niemann-pick de type c
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
US20230265416A1 (en) * 2020-05-11 2023-08-24 Gordian Biotechnology, Inc. Viral delivery vehicle selection
US20240093189A1 (en) * 2021-02-05 2024-03-21 Maze Therapeutics, Inc. Vectors comprising stuffer polynucleotide sequences
EP4334454A2 (fr) * 2021-05-04 2024-03-13 RegenxBio Inc. Nouveaux vecteurs aav et procédés et utilisations associés
GB2623164A (en) * 2022-08-09 2024-04-10 Univ Sheffield Viral vector

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
JPH1033175A (ja) * 1996-07-24 1998-02-10 Hisamitsu Pharmaceut Co Inc 組換えアデノ随伴ウイルスベクターの製造方法
US6171855B1 (en) 1998-05-28 2001-01-09 The Regents Of The University Of Michigan Vectors
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
ATE529525T1 (de) * 1999-10-29 2011-11-15 Takara Bio Inc Gentransferverfahren
US7132277B1 (en) * 2000-01-31 2006-11-07 Merck & Co., Inc. Helper dependent vector system for gene therapy
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
NZ547185A (en) * 2003-10-20 2009-03-31 Nsgene As In vivo gene therapy of parkinson's disease
WO2009134681A2 (fr) * 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Vecteurs viraux aav7 pour une administration ciblée de cellules rpe
CA3050894C (fr) 2010-04-23 2022-10-18 University Of Massachusetts Constructions d'expression a cistrons multiples
WO2012109667A1 (fr) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Composés thérapeutiques
AU2013287261B2 (en) * 2012-07-06 2019-03-07 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions

Also Published As

Publication number Publication date
RU2015103901A (ru) 2016-08-27
EP2870237A4 (fr) 2015-12-23
CN104619832B (zh) 2021-03-16
AU2013287261B2 (en) 2019-03-07
ES2703177T3 (es) 2019-03-07
US9540659B2 (en) 2017-01-10
US20150184197A1 (en) 2015-07-02
RU2679843C2 (ru) 2019-02-13
JP2015522280A (ja) 2015-08-06
CN104619832A (zh) 2015-05-13
CA2878401C (fr) 2022-07-19
BR112015000161A2 (pt) 2017-06-27
IN2015KN00074A (fr) 2015-07-31
WO2014007858A1 (fr) 2014-01-09
EP2870237B1 (fr) 2018-09-26
EP2870237A1 (fr) 2015-05-13
AU2013287261A1 (en) 2015-01-22
JP6366581B2 (ja) 2018-08-08
US20170306355A1 (en) 2017-10-26
CA2878401A1 (fr) 2014-01-09

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