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WO2018183808A9 - Antiviral therapeutic - Google Patents

Antiviral therapeutic 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
French (fr)
Other versions
WO2018183808A1 (en
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/en
Priority to EP18721530.6A priority patent/EP3601568A1/en
Priority to CN201880035095.7A priority patent/CN110678548A/en
Publication of WO2018183808A1 publication Critical patent/WO2018183808A1/en
Publication of WO2018183808A9 publication Critical patent/WO2018183808A9/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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

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  • 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

The present disclosure provides guide RNAs for use in cleaving target viral nucleic acids. The present disclosure further provides compositions that include an mRNA encoding a Cas endonuclease and a guide RNA having particular sequences and modifications, with both RNAs encapsulated by nanoparticles, for the treatment of HPV infection. In some embodiments, the guide RNAs and mRNA encoding a Cas endonuclease are packaged in a lipid nanoparticle, which may be suspended in a carrier formulation for topical or local delivery to infected tissue. In some embodiments, the guide RNA and/or the mRNA encoding the Cas endonuclease also include features such as modified nucleotides that promote the delivery of the RNAs to, and retention within, infected cells.
PCT/US2018/025350 2017-03-31 2018-03-30 Antiviral therapeutic Ceased WO2018183808A1 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2019553095A JP2020515258A (en) 2017-03-31 2018-03-30 Antiviral therapeutic agent
EP18721530.6A EP3601568A1 (en) 2017-03-31 2018-03-30 Antiviral therapeutic
CN201880035095.7A CN110678548A (en) 2017-03-31 2018-03-30 Antiviral therapeutic agent

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 (en) 2018-10-04
WO2018183808A9 true WO2018183808A9 (en) 2018-12-13

Family

ID=62092242

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/025350 Ceased WO2018183808A1 (en) 2017-03-31 2018-03-30 Antiviral therapeutic

Country Status (4)

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

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3160394C (en) 2013-07-30 2023-11-07 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
WO2016118697A1 (en) 2015-01-21 2016-07-28 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
JP7384801B2 (en) * 2017-12-15 2023-11-21 ダニスコ・ユーエス・インク CAS9 variants and methods of use
WO2020154594A2 (en) * 2019-01-25 2020-07-30 Agenovir Corporation Compositions and methods for treatment of human papillomavirus
CN111748551B (en) * 2019-03-27 2021-07-09 纳昂达(南京)生物科技有限公司 Blocking sequence, capture kit, library hybridization capture method and library construction method
WO2021168266A1 (en) * 2020-02-21 2021-08-26 Temple University - Of The Commonwealth System Of Higher Education Eradication of merkel cell polyomavirus
CN111440825A (en) * 2020-04-07 2020-07-24 嘉晨西海(杭州)生物技术有限公司 Method for loading mRNA (messenger ribonucleic acid) by liposome
IL297193A (en) * 2020-04-09 2022-12-01 Verve Therapeutics Inc Base editing of pcsk9 and methods of using same for treatment of disease
EP4150079A1 (en) * 2020-05-15 2023-03-22 Crispr Therapeutics AG Messenger rna encoding cas9 for use in genome-editing systems
CN111534518B (en) * 2020-05-18 2021-07-23 纳昂达(南京)生物科技有限公司 Universal blocking sequence and application thereof
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 (en) * 2023-09-29 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted eradication of ebv-positive cells by crispr/cas-mediated ebv reactivation

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102014061B1 (en) 2011-10-03 2019-08-28 모더나 세라퓨틱스, 인코포레이티드 Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US20150307542A1 (en) 2012-10-03 2015-10-29 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
PT3019619T (en) 2013-07-11 2021-11-11 Modernatx Inc COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING SYNTHETIC CRISPR AND SGARN-RELATED PROTEINS AND METHODS OF USE
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 (en) 2014-12-03 2024-10-16 Agilent Technologies, Inc. Guide rna with chemical modifications
JP2017538427A (en) * 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 composition and method of use
MA41349A (en) * 2015-01-14 2017-11-21 Univ Temple RNA-GUIDED ERADICATION OF HERPES SIMPLEX TYPE I AND OTHER ASSOCIATED HERPES VIRUSES
EP3280803B1 (en) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
JP2018516984A (en) * 2015-05-29 2018-06-28 アジェノビア コーポレーション Compositions and methods for cell targeted HPV treatment
JP7107683B2 (en) * 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 CRISPR enzyme mutations that reduce off-target effects

Also Published As

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

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