WO2018183808A9 - Antiviral therapeutic - Google Patents
Antiviral therapeutic Download PDFInfo
- 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
Links
- 230000000840 anti-viral effect Effects 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 108020005004 Guide RNA Proteins 0.000 abstract 4
- 102000004533 Endonucleases Human genes 0.000 abstract 3
- 108010042407 Endonucleases Proteins 0.000 abstract 3
- 108020004999 messenger RNA Proteins 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 239000002105 nanoparticle Substances 0.000 abstract 2
- 208000009608 Papillomavirus Infections Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 150000002632 lipids Chemical class 0.000 abstract 1
- 230000014759 maintenance of location Effects 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000000699 topical effect Effects 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
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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
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
Priority Applications (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201880035095.7A CN110678548A (en) | 2017-03-31 | 2018-03-30 | Antiviral therapeutic agent |
JP2019553095A JP2020515258A (en) | 2017-03-31 | 2018-03-30 | Antiviral therapeutic agent |
EP18721530.6A EP3601568A1 (en) | 2017-03-31 | 2018-03-30 | Antiviral therapeutic |
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 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 (13)
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 (en) | 2015-01-21 | 2022-06-21 | 菲泽尔克斯公司 | Methods, compositions and systems for delivering therapeutic and diagnostic agents into cells |
EP3562510A4 (en) | 2016-12-30 | 2021-01-06 | Genevant Sciences GmbH | Branched peg molecules and related compositions and methods |
US11781125B2 (en) * | 2017-12-15 | 2023-10-10 | Danisco Us Inc. | 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 |
CA3179862A1 (en) | 2020-04-09 | 2021-10-14 | Verve Therapeutics, Inc. | Base editing of angptl3 and methods of using same for treatment of disease |
WO2021229502A1 (en) * | 2020-05-15 | 2021-11-18 | 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 |
CA3205133A1 (en) * | 2020-12-15 | 2022-06-23 | Flagship Pioneering Innovations V, Inc. | Compositions and methods for modulating myc expression |
WO2022240960A1 (en) * | 2021-05-14 | 2022-11-17 | Crispr Therapeutics Ag | Mrna large scale synthesis and purification |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
HUE057725T2 (en) | 2011-10-03 | 2022-06-28 | Modernatx Inc | Modified nucleosides, nucleotides and nucleic acids and their uses |
WO2014093924A1 (en) | 2012-12-13 | 2014-06-19 | Moderna Therapeutics, Inc. | Modified nucleic acid molecules and uses thereof |
SMT202100691T1 (en) | 2013-07-11 | 2022-01-10 | Modernatx Inc | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
EP3107999A4 (en) * | 2014-02-18 | 2017-10-04 | Duke University | Compositions for the inactivation of virus replication and methods of making and using the same |
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 (en) * | 2015-01-14 | 2017-11-21 | Univ Temple | RNA-GUIDED ERADICATION OF HERPES SIMPLEX TYPE I AND OTHER ASSOCIATED HERPES VIRUSES |
KR20240038141A (en) * | 2015-04-06 | 2024-03-22 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | 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 |
RU2752834C2 (en) * | 2015-06-18 | 2021-08-09 | Те Брод Инститьют, Инк. | Crispr enzyme mutations reducing non-targeted effects |
-
2018
- 2018-03-30 WO PCT/US2018/025350 patent/WO2018183808A1/en active Application Filing
- 2018-03-30 JP JP2019553095A patent/JP2020515258A/en active Pending
- 2018-03-30 EP EP18721530.6A patent/EP3601568A1/en not_active Withdrawn
- 2018-03-30 CN CN201880035095.7A patent/CN110678548A/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CN110678548A (en) | 2020-01-10 |
EP3601568A1 (en) | 2020-02-05 |
WO2018183808A1 (en) | 2018-10-04 |
JP2020515258A (en) | 2020-05-28 |
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