IN2015KN00074A - - Google Patents

Info

Publication number
IN2015KN00074A
IN2015KN00074A IN74KON2015A IN2015KN00074A IN 2015KN00074 A IN2015KN00074 A IN 2015KN00074A IN 74KON2015 A IN74KON2015 A IN 74KON2015A IN 2015KN00074 A IN2015KN00074 A IN 2015KN00074A
Authority
IN
India
Prior art keywords
adeno
nucleotides
disclosed
length
nucleic acid
Prior art date
Application number
Other languages
English (en)
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 IN2015KN00074A publication Critical patent/IN2015KN00074A/en

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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)
IN74KON2015 2012-07-06 2013-03-14 IN2015KN00074A (fr)

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
IN2015KN00074A true IN2015KN00074A (fr) 2015-07-31

Family

ID=49882407

Family Applications (1)

Application Number Title Priority Date Filing Date
IN74KON2015 IN2015KN00074A (fr) 2012-07-06 2013-03-14

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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BR112015000161A2 (pt) * 2012-07-06 2017-06-27 Univ Iowa Res Found composições de vetor de vírus adeno-associado modificado
JP6663859B2 (ja) * 2014-05-20 2020-03-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション ハンチントン病の治療化合物
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3193811A1 (fr) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions et methodes de traitement de la sclerose laterale amyotrophique (sla)
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
US20190000940A1 (en) * 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
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
EP3448987A4 (fr) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
EP3448874A4 (fr) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
CA3024448C (fr) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. Polynucleotides modulateurs
CA3024449A1 (fr) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions et methodes de traitement de la maladie de huntington
WO2018044933A1 (fr) 2016-08-30 2018-03-08 The Regents Of The University Of California Procédés de ciblage et d'administration biomédicaux, et dispositifs et systèmes pour la mise en œuvre de ceux-ci
EP3618839A4 (fr) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la sclérose latérale amyotrophique (sla)
JP2020518259A (ja) 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. ハンチントン病治療組成物および方法
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
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (fr) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Traitement de la sclérose latérale amyotrophique (sla)
WO2019118516A1 (fr) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Méthodes et compositions liées à cpf1 pour l'édition génique
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN111902164A (zh) 2018-02-01 2020-11-06 同源药物公司 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
CA3094465A1 (fr) 2018-04-03 2019-10-10 Stridebio, Inc. Vecteurs de virus permettant de cibler des tissus ophtalmiques
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US20210254103A1 (en) * 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
CN114072514A (zh) 2019-05-14 2022-02-18 杜克大学 用于治疗atp酶介导的疾病的组合物和方法
EP3999647A1 (fr) * 2019-07-18 2022-05-25 Lysogene Compositions et méthodes de traitement de la maladie de sanfilippo et d'autres troubles
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
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
WO2021231305A2 (fr) * 2020-05-11 2021-11-18 Gordian Biotechnology, Inc. Sélection de véhicules d'administration virale
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
EP4200408A1 (fr) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Vecteurs de type virus adéno-associé pour le traitement du syndrome de rett
KR20230142576A (ko) * 2021-02-05 2023-10-11 메이즈 테라퓨틱스, 인코퍼레이티드 스터퍼 폴리뉴클레오티드 서열을 포함하는 벡터
WO2022235614A2 (fr) * 2021-05-04 2022-11-10 Regenxbio Inc. Nouveaux vecteurs aav et procédés et utilisations associés
WO2024033329A2 (fr) * 2022-08-09 2024-02-15 University Of Sheffield Vecteur viral

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KR20020057975A (ko) * 1999-10-29 2002-07-12 오미야 히사시 유전자 도입 방법
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WO2005039643A2 (fr) * 2003-10-20 2005-05-06 Nsgene A/S Therapie genique in vivo pour la maladie de parkinson
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
WO2011133874A1 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Constructions d'expression à cistrons multiples
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
BR112015000161A2 (pt) * 2012-07-06 2017-06-27 Univ Iowa Res Found composições de vetor de vírus adeno-associado modificado

Also Published As

Publication number Publication date
CN104619832B (zh) 2021-03-16
EP2870237B1 (fr) 2018-09-26
CA2878401A1 (fr) 2014-01-09
US20170306355A1 (en) 2017-10-26
WO2014007858A1 (fr) 2014-01-09
US20150184197A1 (en) 2015-07-02
HK1210214A1 (en) 2016-04-15
EP2870237A1 (fr) 2015-05-13
RU2015103901A (ru) 2016-08-27
RU2679843C2 (ru) 2019-02-13
EP2870237A4 (fr) 2015-12-23
US9540659B2 (en) 2017-01-10
AU2013287261A1 (en) 2015-01-22
CA2878401C (fr) 2022-07-19
ES2703177T3 (es) 2019-03-07
AU2013287261B2 (en) 2019-03-07
JP2015522280A (ja) 2015-08-06
CN104619832A (zh) 2015-05-13
BR112015000161A2 (pt) 2017-06-27
JP6366581B2 (ja) 2018-08-08

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