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WO2018183808A9 - Thérapeutique antivirale - Google Patents

Thérapeutique antivirale Download PDF

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Publication number
WO2018183808A9
WO2018183808A9 PCT/US2018/025350 US2018025350W WO2018183808A9 WO 2018183808 A9 WO2018183808 A9 WO 2018183808A9 US 2018025350 W US2018025350 W US 2018025350W WO 2018183808 A9 WO2018183808 A9 WO 2018183808A9
Authority
WO
WIPO (PCT)
Prior art keywords
rnas
mrna encoding
cas endonuclease
guide
present disclosure
Prior art date
Application number
PCT/US2018/025350
Other languages
English (en)
Other versions
WO2018183808A1 (fr
Inventor
Derek D. Sloan
Sarah LEDOUX
Xin Cindy XIONG
Michael Patrick HOUSLEY
Original Assignee
Agenovir Corporation
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 Agenovir Corporation filed Critical Agenovir Corporation
Priority to JP2019553095A priority Critical patent/JP2020515258A/ja
Priority to EP18721530.6A priority patent/EP3601568A1/fr
Priority to CN201880035095.7A priority patent/CN110678548A/zh
Publication of WO2018183808A1 publication Critical patent/WO2018183808A1/fr
Publication of WO2018183808A9 publication Critical patent/WO2018183808A9/fr
Anticipated expiration legal-status Critical
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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La présente invention concerne des ARN guides destinés à être utilisés dans le clivage d'acides nucléiques viraux cibles. La présente invention concerne en outre des compositions qui comprennent un ARNm codant pour une endonucléase Cas et un ARN guide ayant des séquences et des modifications particulières, les deux ARN étant encapsulés par des nanoparticules, pour le traitement d'une infection par le VPH. Dans certains modes de réalisation, les ARN guides et le ARNm codant pour une endonucléase Cas sont conditionnés dans une nanoparticule lipidique, qui peut être mise en suspension dans une formulation de vecteurs pour une administration topique ou locale à un tissu infecté. Dans certains modes de réalisation, l'ARN guide et/ou le ARNm codant pour l'endonucléase Cas comprennent également des éléments tels que des nucléotides modifiés qui favorisent l'administration des ARN, et la rétention en leur sein, à des cellules infectées.
PCT/US2018/025350 2017-03-31 2018-03-30 Thérapeutique antivirale Ceased WO2018183808A1 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2019553095A JP2020515258A (ja) 2017-03-31 2018-03-30 抗ウイルス治療剤
EP18721530.6A EP3601568A1 (fr) 2017-03-31 2018-03-30 Thérapeutique antivirale
CN201880035095.7A CN110678548A (zh) 2017-03-31 2018-03-30 抗病毒治疗剂

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762479643P 2017-03-31 2017-03-31
US62/479,643 2017-03-31
US201762507963P 2017-05-18 2017-05-18
US62/507,963 2017-05-18

Publications (2)

Publication Number Publication Date
WO2018183808A1 WO2018183808A1 (fr) 2018-10-04
WO2018183808A9 true WO2018183808A9 (fr) 2018-12-13

Family

ID=62092242

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/025350 Ceased WO2018183808A1 (fr) 2017-03-31 2018-03-30 Thérapeutique antivirale

Country Status (4)

Country Link
EP (1) EP3601568A1 (fr)
JP (1) JP2020515258A (fr)
CN (1) CN110678548A (fr)
WO (1) WO2018183808A1 (fr)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3160394C (fr) 2013-07-30 2023-11-07 Genevant Sciences Gmbh Copolymeres sequences et leurs conjugues ou complexes avec des oligonucleotides
WO2016118697A1 (fr) 2015-01-21 2016-07-28 Phaserx, Inc. Procédés, compositions et systèmes permettant l'administration d'agents thérapeutiques et diagnostiques dans des cellules
WO2018126084A1 (fr) 2016-12-30 2018-07-05 Phaserx, Inc. Molécules de peg ramifié, compositions et procédés associés
JP7384801B2 (ja) * 2017-12-15 2023-11-21 ダニスコ・ユーエス・インク Ｃａｓ９変異体及び使用方法
WO2020154594A2 (fr) * 2019-01-25 2020-07-30 Agenovir Corporation Compositions et procédés de traitement du virus du papillome humain
CN111748551B (zh) * 2019-03-27 2021-07-09 纳昂达(南京)生物科技有限公司 封闭序列、捕获试剂盒、文库杂交捕获方法及建库方法
WO2021168266A1 (fr) * 2020-02-21 2021-08-26 Temple University - Of The Commonwealth System Of Higher Education Éradication de polyomavirus de cellules de merkel
CN111440825A (zh) * 2020-04-07 2020-07-24 嘉晨西海(杭州)生物技术有限公司 一种脂质体载mRNA的方法
IL297193A (en) * 2020-04-09 2022-12-01 Verve Therapeutics Inc Basic editing of pcks9 and methods of using it to treat diseases
EP4150079A1 (fr) * 2020-05-15 2023-03-22 Crispr Therapeutics AG Arn messager codant pour cas9 destiné à être utilisé dans des systèmes d'édition du génome
CN111534518B (zh) * 2020-05-18 2021-07-23 纳昂达(南京)生物科技有限公司 通用封闭序列及其应用
AU2021401231A1 (en) * 2020-12-15 2023-06-29 Flagship Pioneering Innovations V, Inc. Compositions and methods for modulating myc expression
US20220364078A1 (en) * 2021-05-14 2022-11-17 Crispr Therapeutics Ag Mrna large scale synthesis and purification
US20240382622A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025072640A1 (fr) * 2023-09-29 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Éradication ciblée de cellules ebv-positives par réactivation d'ebv médiée par crispr/cas

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102014061B1 (ko) 2011-10-03 2019-08-28 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
US20150307542A1 (en) 2012-10-03 2015-10-29 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
SG11201606819QA (en) * 2014-02-18 2016-09-29 Univ Duke Compositions for the inactivation of virus replication and methods of making and using the same
EP4400584A3 (fr) 2014-12-03 2024-10-16 Agilent Technologies, Inc. Arn guide avec modifications chimiques
JP2017538427A (ja) * 2014-12-18 2017-12-28 インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド Ｃｒｉｓｐｒ系組成物及び使用方法
MA41349A (fr) * 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
EP3280803B1 (fr) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Arn guides chimiquement modifiés pour la régulation génétique médiée par crispr/cas
JP2018516984A (ja) * 2015-05-29 2018-06-28 アジェノビア コーポレーション 細胞を標的にしたｈｐｖ処置のための組成物および方法
JP7107683B2 (ja) * 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるｃｒｉｓｐｒ酵素突然変異

Also Published As

Publication number Publication date
JP2020515258A (ja) 2020-05-28
EP3601568A1 (fr) 2020-02-05
WO2018183808A1 (fr) 2018-10-04
CN110678548A (zh) 2020-01-10

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