WO2024112876A3 - Synthèse divisée d'arn longs - Google Patents

Synthèse divisée d'arn longs Download PDF

Info

Publication number
WO2024112876A3
WO2024112876A3 PCT/US2023/080893 US2023080893W WO2024112876A3 WO 2024112876 A3 WO2024112876 A3 WO 2024112876A3 US 2023080893 W US2023080893 W US 2023080893W WO 2024112876 A3 WO2024112876 A3 WO 2024112876A3
Authority
WO
WIPO (PCT)
Prior art keywords
split synthesis
long rnas
rnas
long
split
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.)
Ceased
Application number
PCT/US2023/080893
Other languages
English (en)
Other versions
WO2024112876A2 (fr
Inventor
Ekambareswara Rao KANDIMALLA
Venubabu Kotikam
Meetu SETH
Cong Zhou
Andrew Doyle
David Wiley
Jeffrey HUSSMANN
Jonathan Levy
Seth Alexander
Andrew ANZALONE
Rohan KUMBHARE
Mallikarjuna Putta
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
Priority to EP23821097.5A priority Critical patent/EP4623081A2/fr
Publication of WO2024112876A2 publication Critical patent/WO2024112876A2/fr
Publication of WO2024112876A3 publication Critical patent/WO2024112876A3/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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La présente invention concerne des méthodes de préparation d'ARN longs tels que des ARN guides de « prime editing » (PEgRNA).
PCT/US2023/080893 2022-11-23 2023-11-22 Synthèse divisée d'arn longs Ceased WO2024112876A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP23821097.5A EP4623081A2 (fr) 2022-11-23 2023-11-22 Synthèse divisée d'arn longs

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202263384872P 2022-11-23 2022-11-23
US63/384,872 2022-11-23
US202363586548P 2023-09-29 2023-09-29
US63/586,548 2023-09-29

Publications (2)

Publication Number Publication Date
WO2024112876A2 WO2024112876A2 (fr) 2024-05-30
WO2024112876A3 true WO2024112876A3 (fr) 2024-07-04

Family

ID=89158216

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/080893 Ceased WO2024112876A2 (fr) 2022-11-23 2023-11-22 Synthèse divisée d'arn longs

Country Status (2)

Country Link
EP (1) EP4623081A2 (fr)
WO (1) WO2024112876A2 (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 (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016186745A1 (fr) * 2015-05-15 2016-11-24 Ge Healthcare Dharmacon, Inc. Arn de guidage unique synthétique pour l'édition de gène médiée par cas9
WO2018126176A1 (fr) * 2016-12-30 2018-07-05 Editas Medicine, Inc. Molécules de guidage synthétiques, compositions et procédés associés
WO2020006423A1 (fr) * 2018-06-29 2020-01-02 Editas Medicine, Inc. Molécules de guidage synthétiques, compositions et procédés associés
WO2020191248A1 (fr) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
US20210317495A1 (en) * 2018-10-02 2021-10-14 Toray Industries, Inc. Method of producing hairpin single-stranded rna molecules

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4430189A1 (fr) 2021-11-11 2024-09-18 Prime Medicine, Inc. Compositions et procédés d'édition génomique pour le traitement du syndrome du chromosome x fragile
IL313027A (en) 2021-11-24 2024-07-01 Prime Medicine Inc Modified prime editing guide rnas

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016186745A1 (fr) * 2015-05-15 2016-11-24 Ge Healthcare Dharmacon, Inc. Arn de guidage unique synthétique pour l'édition de gène médiée par cas9
WO2018126176A1 (fr) * 2016-12-30 2018-07-05 Editas Medicine, Inc. Molécules de guidage synthétiques, compositions et procédés associés
WO2020006423A1 (fr) * 2018-06-29 2020-01-02 Editas Medicine, Inc. Molécules de guidage synthétiques, compositions et procédés associés
US20210317495A1 (en) * 2018-10-02 2021-10-14 Toray Industries, Inc. Method of producing hairpin single-stranded rna molecules
WO2020191248A1 (fr) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CHEN ZEXIANG ET AL: "Tetrazine-Ligated CRISPR sgRNAs for Efficient Genome Editing", ACS CHEMICAL BIOLOGY, vol. 17, no. 5, 21 April 2022 (2022-04-21), pages 1045 - 1050, XP093062032, ISSN: 1554-8929, DOI: 10.1021/acschembio.2c00116 *
HIDETO MARUYAMA ET AL: "Chemical ligation of oligonucleotides using an electrophilic phosphorothioester", NUCLEIC ACIDS RESEARCH, vol. 45, no. 12, 18 May 2017 (2017-05-18), GB, pages 7042 - 7048, XP055464361, ISSN: 0305-1048, DOI: 10.1093/nar/gkx459 *
NAKAMOTO KOSUKE ET AL: "Chemically synthesized circular RNAs with phosphoramidate linkages enable rolling circle translation", CHEMICAL COMMUNICATIONS, vol. 56, no. 46, 9 June 2020 (2020-06-09), UK, pages 6217 - 6220, XP055799400, ISSN: 1359-7345, DOI: 10.1039/D0CC02140G *
TAEMAITREE LAPATRADA ET AL: "An artificial triazole backbone linkage provides a split-and-click strategy to bioactive chemically modified CRISPR sgRNA", NATURE COMMUNICATIONS, vol. 10, no. 1, 8 April 2019 (2019-04-08), XP055780838, DOI: 10.1038/s41467-019-09600-4 *

Also Published As

Publication number Publication date
WO2024112876A2 (fr) 2024-05-30
EP4623081A2 (fr) 2025-10-01

Similar Documents

Publication Publication Date Title
ZA202403308B (en) An efficient new process for synthesis of 2-amino-5-chloro-n-,3-dimethylbenzamide
WO2007031548A3 (fr) Procedes de preparation de derives de (ethynyl-benzyl)-benzene substitues par glucopyranosyl et de leurs intermediaires
WO2024112876A3 (fr) Synthèse divisée d'arn longs
WO2023096977A3 (fr) Arn guides d'édition primaire modifiés
NZ796970A (en) Synthesis of quinazoline compounds
CR20230294A (es) Método para preparar pralsetinib
WO2021076835A8 (fr) Procédés de préparation d'acide 5-bromo-2-(3chloro-pyridin-2-yl)-2h-pyrazole-3-carboxylique
EP4410989A3 (fr) Procédé de production de dérivés d'hétérocyclidène acétamide
WO2022031862A3 (fr) Composés hétéroaryle et hétérocyclyle
MX2021013574A (es) Sintesis de imidazo[1,2-a]pirimidinas etiquetadas.
CA3188923A1 (fr) Procedes de production de vecteur lentiviral de qualite clinique
MX2022006142A (es) Proceso para la sintesis de (3-cloro-2-piridil)hidrazina.
EA200602169A1 (ru) Способ получения производных 2-оксо-1-пирролидина внутримолекулярным аллилированием
MX2023002810A (es) Metodo de preparacion de formulaciones de proteinas liofilizadas.
WO2025111452A3 (fr) Modifications chimiques dans pegarn et ngarn
WO2025083699A3 (fr) Polymorphes de resmétirom et procédé associé
ZA202408113B (en) Improved process for the manufacture of osimertinib
WO2020130702A3 (fr) Composition contenant un matériau à changement de phase et procédé de fabrication d'un tel matériau
MX2024009225A (es) Metodos para preparar 3-bromo-1-(3-cloropiridin-2-il)-4,5-dihidro- 1h-pirazol-5-carboxilato de etilo.
MX2021012690A (es) Formas cristalinas y metodos para producir formas cristalinas de un compuesto.
EP4397657A4 (fr) Composé, son procédé de préparation, et molécule unique et oligomère dérivés de celui-ci
WO2024211482A3 (fr) PROCÉDÉS DE SYNTHÈSE DE 14-β-AMINOMORPHANES ET DE SELS DE CEUX-CI
WO2023147057A3 (fr) Procédés de synthèse de cannabidiol, de dérivés de celui-ci et d'autres phytocannabinoïdes
EP4397658A4 (fr) Composé, son procédé de préparation, molécule unique, oligomère et polymère dérivés de celui-ci
MX2025000283A (es) Oligonucleotidos hibridos

Legal Events

Date Code Title Description
WWE Wipo information: entry into national phase

Ref document number: 2023821097

Country of ref document: EP

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2023821097

Country of ref document: EP

Effective date: 20250623

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 23821097

Country of ref document: EP

Kind code of ref document: A2

WWP Wipo information: published in national office

Ref document number: 2023821097

Country of ref document: EP