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WO2025111452A3 - Modifications chimiques dans pegarn et ngarn - Google Patents

Modifications chimiques dans pegarn et ngarn Download PDF

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Publication number
WO2025111452A3
WO2025111452A3 PCT/US2024/056877 US2024056877W WO2025111452A3 WO 2025111452 A3 WO2025111452 A3 WO 2025111452A3 US 2024056877 W US2024056877 W US 2024056877W WO 2025111452 A3 WO2025111452 A3 WO 2025111452A3
Authority
WO
WIPO (PCT)
Prior art keywords
pegrna
ngrnas
chemical modifications
same
guide rna
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
PCT/US2024/056877
Other languages
English (en)
Other versions
WO2025111452A2 (fr
Inventor
Ekambareswara Rao KANDIMALLA
Mallikarjuna Putta
Seth Alexander
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Prime Medicine Inc
Original Assignee
Prime Medicine Inc
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 Prime Medicine Inc filed Critical Prime Medicine Inc
Publication of WO2025111452A2 publication Critical patent/WO2025111452A2/fr
Publication of WO2025111452A3 publication Critical patent/WO2025111452A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/335—Modified T or U

Landscapes

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

Abstract

L'invention concerne des séquences modifiées d'ARN guide d'édition primaire (PEgARN) et d'ARN guide de coupure (ngARN) et des compositions les comprenant, ainsi que l'utilisation de celles-ci dans des procédés d'édition de gènes.
PCT/US2024/056877 2023-11-21 2024-11-21 Modifications chimiques dans pegarn et ngarn Pending WO2025111452A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363601415P 2023-11-21 2023-11-21
US63/601,415 2023-11-21

Publications (2)

Publication Number Publication Date
WO2025111452A2 WO2025111452A2 (fr) 2025-05-30
WO2025111452A3 true WO2025111452A3 (fr) 2025-07-03

Family

ID=93924097

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/056877 Pending WO2025111452A2 (fr) 2023-11-21 2024-11-21 Modifications chimiques dans pegarn et ngarn

Country Status (1)

Country Link
WO (1) WO2025111452A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025215514A1 (fr) * 2024-04-08 2025-10-16 Crispr Therapeutics Ag Synthèse d'arn médiée par ponts

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022067130A2 (fr) * 2020-09-24 2022-03-31 The Broad Institute, Inc. Arn guides d'édition primaire, leurs compositions et leurs méthodes d'utilisation
WO2022256714A2 (fr) * 2021-06-03 2022-12-08 Prime Medicine, Inc. Compositions d'édition de génome et méthodes de traitement de la maladie de wilson
WO2023039586A1 (fr) * 2021-09-10 2023-03-16 Agilent Technologies, Inc. Arn guides avec modification chimique pour l'édition primaire
WO2023043856A1 (fr) * 2021-09-14 2023-03-23 Agilent Technologies, Inc. Procédés d'utilisation d'arn guides avec des modifications chimiques
WO2023047338A1 (fr) * 2021-09-24 2023-03-30 Crispr Therapeutics Ag Synthèse de sgrna synthétique prodrug
WO2023070110A2 (fr) * 2021-10-21 2023-04-27 Prime Medicine, Inc. Compositions d'édition génomique et méthodes de traitement de la rétinite pigmentaire

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022067130A2 (fr) * 2020-09-24 2022-03-31 The Broad Institute, Inc. Arn guides d'édition primaire, leurs compositions et leurs méthodes d'utilisation
WO2022256714A2 (fr) * 2021-06-03 2022-12-08 Prime Medicine, Inc. Compositions d'édition de génome et méthodes de traitement de la maladie de wilson
WO2023039586A1 (fr) * 2021-09-10 2023-03-16 Agilent Technologies, Inc. Arn guides avec modification chimique pour l'édition primaire
WO2023043856A1 (fr) * 2021-09-14 2023-03-23 Agilent Technologies, Inc. Procédés d'utilisation d'arn guides avec des modifications chimiques
WO2023047338A1 (fr) * 2021-09-24 2023-03-30 Crispr Therapeutics Ag Synthèse de sgrna synthétique prodrug
WO2023070110A2 (fr) * 2021-10-21 2023-04-27 Prime Medicine, Inc. Compositions d'édition génomique et méthodes de traitement de la rétinite pigmentaire

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
CHEN PETER J. ET AL: "Prime editing for precise and highly versatile genome manipulation", NATURE REVIEWS GENETICS, vol. 24, no. 3, 7 March 2023 (2023-03-07), GB, pages 161 - 177, XP093067691, ISSN: 1471-0056, Retrieved from the Internet <URL:https://www.nature.com/articles/s41576-022-00541-1> DOI: 10.1038/s41576-022-00541-1 *
HAO YIN: "Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing", NATURE BIOTECHNOLOGY, vol. 35, no. 12, 13 December 2017 (2017-12-13), New York, pages 1179 - 1187, XP093161200, ISSN: 1087-0156, Retrieved from the Internet <URL:http://www.nature.com/articles/nbt.4005> DOI: 10.1038/nbt.4005 *
LI HANQIN ET AL: "Highly efficient generation of isogenic pluripotent stem cell models using prime editing", BIORXIV, 15 February 2022 (2022-02-15), pages 1 - 49, XP093103479, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.1101/2022.02.15.480601v1.full.pdf> [retrieved on 20231120], DOI: 10.1101/2022.02.15.480601 *
NELSON JAMES W ET AL: "Engineered pegRNAs improve prime editing efficiency", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 40, no. 3, 4 October 2021 (2021-10-04), pages 402 - 410, XP037720612, ISSN: 1087-0156, [retrieved on 20211004], DOI: 10.1038/S41587-021-01039-7 *
ROZNERS ERIKS: "Chemical Modifications of CRISPR RNAs to Improve Gene-Editing Activity and Specificity", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 144, no. 28, 7 July 2022 (2022-07-07), pages 12584 - 12594, XP093072674, ISSN: 0002-7863, Retrieved from the Internet <URL:https://pubs.acs.org/doi/pdf/10.1021/jacs.2c02633> DOI: 10.1021/jacs.2c02633 *
YIN HAO ET AL: "Supplementary Materials: Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing", NATURE BIOTECHNOLOGY, 13 November 2017 (2017-11-13), XP093250096, Retrieved from the Internet <URL:http://www.nature.com/articles/nbt.4005> DOI: 10.1038/nbt.4005 *

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