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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 CN201880035095.7A priority Critical patent/CN110678548A/zh
Priority to JP2019553095A priority patent/JP2020515258A/ja
Priority to EP18721530.6A priority patent/EP3601568A1/fr
Publication of WO2018183808A1 publication Critical patent/WO2018183808A1/fr
Publication of WO2018183808A9 publication Critical patent/WO2018183808A9/fr

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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (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 WO2018183808A1 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CN201880035095.7A CN110678548A (zh) 2017-03-31 2018-03-30 抗病毒治疗剂
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

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 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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
CN114642735A (zh) 2015-01-21 2022-06-21 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
EP3562510A4 (fr) 2016-12-30 2021-01-06 Genevant Sciences GmbH Molécules de peg ramifié, compositions et procédés associés
US11781125B2 (en) * 2017-12-15 2023-10-10 Danisco Us Inc. Cas9 variants and methods of use
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
CA3179862A1 (fr) 2020-04-09 2021-10-14 Verve Therapeutics, Inc. Edition de base de angptl3 et procedes d'utilisation de celle-ci pour le traitement d'une maladie
WO2021229502A1 (fr) * 2020-05-15 2021-11-18 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
CA3205133A1 (fr) * 2020-12-15 2022-06-23 Flagship Pioneering Innovations V, Inc. Compositions et methodes de modulation de l'expression de myc
WO2022240960A1 (fr) * 2021-05-14 2022-11-17 Crispr Therapeutics Ag Synthèse et purification à grande échelle d'arnm

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE057725T2 (hu) 2011-10-03 2022-06-28 Modernatx Inc Módosított nukleozidok, nukleotidok és nukleinsavak és ezek felhasználása
WO2014093924A1 (fr) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Molécules d'acide nucléique modifiées et leurs utilisations
SMT202100691T1 (it) 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
EP3107999A4 (fr) * 2014-02-18 2017-10-04 Duke University Compositions destinées à l'inactivation de la réplication d'un virus et leurs procédés de fabrication et d'utilisation
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
AU2015364282B2 (en) * 2014-12-18 2021-04-15 Integrated Dna Technologies, Inc. CRISPR-based compositions and methods of use
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
KR20240038141A (ko) * 2015-04-06 2024-03-22 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
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
RU2752834C2 (ru) * 2015-06-18 2021-08-09 Те Брод Инститьют, Инк. Мутации фермента crispr, уменьшающие нецелевые эффекты

Also Published As

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

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