EP4240849A4 - OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEM - Google Patents

OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEM

Info

Publication number
EP4240849A4
EP4240849A4 EP21890258.3A EP21890258A EP4240849A4 EP 4240849 A4 EP4240849 A4 EP 4240849A4 EP 21890258 A EP21890258 A EP 21890258A EP 4240849 A4 EP4240849 A4 EP 4240849A4
Authority
EP
European Patent Office
Prior art keywords
oligonucleotide compositions
oligonucleotide
compositions
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
EP21890258.3A
Other languages
German (de)
French (fr)
Other versions
EP4240849A1 (en
Inventor
Prashant Monian
Chikdu Shakti Shivalila
Subramanian Marappan
Chandra Vargeese
Pachamuthu Kandasamy
Genliang Lu
Hui Yu
David Charles Donnell Butler
Luciano Henrique Apponi
Mamoru Shimizu
Stephany Michelle Standley
David John Boulay
Andrew Guzior Hoss
Jigar Desai
Jack David Godfrey
Hailin Yang
Naoki Iwamoto
Jayakanthan Kumarasamy
Anthony Lamattina
Tom Liantang Pu
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.)
Wave Life Sciences Pte Ltd
Original Assignee
Wave Life Sciences Pte Ltd
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 Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of EP4240849A1 publication Critical patent/EP4240849A1/en
Publication of EP4240849A4 publication Critical patent/EP4240849A4/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)

Landscapes

  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP21890258.3A 2020-11-08 2021-11-08 OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEM Pending EP4240849A4 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063111079P 2020-11-08 2020-11-08
US202163175036P 2021-04-14 2021-04-14
US202163188415P 2021-05-13 2021-05-13
US202163196178P 2021-06-02 2021-06-02
US202163248520P 2021-09-26 2021-09-26
PCT/US2021/058495 WO2022099159A1 (en) 2020-11-08 2021-11-08 Oligonucleotide compositions and methods thereof

Publications (2)

Publication Number Publication Date
EP4240849A1 EP4240849A1 (en) 2023-09-13
EP4240849A4 true EP4240849A4 (en) 2026-04-01

Family

ID=81456778

Family Applications (1)

Application Number Title Priority Date Filing Date
EP21890258.3A Pending EP4240849A4 (en) 2020-11-08 2021-11-08 OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEM

Country Status (8)

Country Link
EP (1) EP4240849A4 (en)
JP (1) JP2023548584A (en)
KR (1) KR20230118716A (en)
AU (1) AU2021373062A1 (en)
CA (1) CA3197311A1 (en)
IL (1) IL302558A (en)
MX (1) MX2023005326A (en)
WO (1) WO2022099159A1 (en)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2872485T3 (en) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymmetric auxiliary group
BR112016016400A2 (en) 2014-01-16 2017-10-03 Wave Life Sciences Ltd COMPOSITIONS OF CHIRALLY CONTROLLED OLIGONUCLEOTIDES, THEIR USE, THEIR PHARMACEUTICAL COMPOSITION, AND METHODS
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
JP2019520339A (en) 2016-06-03 2019-07-18 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (en) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ Chemically modified RNA editing single stranded oligonucleotides
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
EP3664816A4 (en) 2017-08-08 2021-05-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CA3072110A1 (en) 2017-09-18 2019-03-21 Wave Life Sciences Ltd. Technologies for oligonucleotide preparation
SG11202009877XA (en) 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
JP2022525541A (en) 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド Techniques useful for oligonucleotide preparation
WO2022046667A1 (en) * 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
EP4363574A1 (en) * 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
EP4404942A2 (en) * 2021-09-26 2024-07-31 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
EP4396354A4 (en) * 2021-09-26 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND METHOD FOR THEM
WO2023069603A1 (en) * 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
US20260027237A1 (en) 2022-07-15 2026-01-29 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
JP2025524566A (en) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Oligonucleotides for ADAR-mediated RNA editing and uses thereof
JP2025525564A (en) * 2022-07-18 2025-08-05 エフ. ホフマン-ラ ロシュ アーゲー Oligonucleotide editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
WO2024114908A1 (en) 2022-11-30 2024-06-06 Eberhard Karls Universität Tübingen Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
EP4669750A2 (en) 2023-02-21 2025-12-31 Vib Vzw HIBITORS OF SYNAPTOGYRIN-3 EXPRESSION
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4689110A1 (en) 2023-03-24 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of neurological disorders
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024220934A1 (en) 2023-04-21 2024-10-24 University Of Rochester Nucleic acid molecules for pseudouridylation
CN116444593B (en) * 2023-04-28 2023-12-26 南通大学 Synthesis method of 7-deazaguanosine phosphoramidite monomer
TW202516003A (en) 2023-06-16 2025-04-16 荷蘭商Proqr治療上市公司Ii Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025015338A1 (en) * 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CN121794378A (en) 2023-08-15 2026-04-03 艾尔纳公司 Chemically modified antisense oligonucleotides (ASOs) and compositions for RNA editing
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
AU2024351975A1 (en) * 2023-09-28 2026-04-16 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
AU2024354150A1 (en) * 2023-09-28 2026-03-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025073902A1 (en) 2023-10-06 2025-04-10 Eberhard Karls Universität Tübingen Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing
WO2025083268A1 (en) 2023-10-20 2025-04-24 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025128799A1 (en) * 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
WO2025160090A1 (en) * 2024-01-22 2025-07-31 Wave Life Sciences Ltd. Technol. ogies useful for oligonucleotide preparation
WO2025172372A1 (en) 2024-02-12 2025-08-21 Airna Corporation Conjugate for targeted delivery of an antisense oligonucleotide
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
GB202405143D0 (en) 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2026080897A1 (en) 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000004189A1 (en) * 1998-07-14 2000-01-27 Isis Pharmaceuticals, Inc. Modified oligonucleotides
WO2001023613A1 (en) * 1999-09-30 2001-04-05 Isis Pharmaceuticals, Inc. Human rnase h and oligonucleotide compositions thereof
WO2018057946A2 (en) * 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
WO2020001793A1 (en) * 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
WO2020154344A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014074597A1 (en) * 2012-11-06 2014-05-15 President And Fellows Of Harvard College Compositions and methods relating to complex nucleic acid nanostructures
CN110352244B (en) * 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ Chemically modified RNA editing single stranded oligonucleotides
US11866697B2 (en) * 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US11718638B2 (en) * 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
JP7766589B2 (en) * 2019-10-06 2025-11-10 ウェイブ ライフ サイエンシズ リミテッド Oligonucleotide compositions and methods of use thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000004189A1 (en) * 1998-07-14 2000-01-27 Isis Pharmaceuticals, Inc. Modified oligonucleotides
WO2001023613A1 (en) * 1999-09-30 2001-04-05 Isis Pharmaceuticals, Inc. Human rnase h and oligonucleotide compositions thereof
WO2018057946A2 (en) * 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
WO2020001793A1 (en) * 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
WO2020154344A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
ANZAHAEE MARYAM YAHYAEE ET AL: "Arabinonucleic Acids: 2'-Stereoisomeric Modulators of siRNA Activity", NUCLEIC ACID THERAPEUTICS, vol. 24, no. 5, 11 September 2014 (2014-09-11), US, pages 336 - 343, XP093060210, ISSN: 2159-3337, Retrieved from the Internet <URL:https://www.liebertpub.com/doi/pdf/10.1089/nat.2014.0496> DOI: 10.1089/nat.2014.0496 *
DELEAVEY G. F. ET AL: "Designing chemically modified common general oligonucleotides for targeted gene silencing", CHEM BIOL., vol. 19, no. 8, 23 August 2012 (2012-08-23), pages 937 - 954, XP055905926 *
HABIBIAN MARYAM ET AL: "Effect of 2'-5'/3'-5' phosphodiester linkage heterogeneity on RNA interference", NUCLEIC ACIDS RESEARCH, vol. 48, no. 9, 21 May 2020 (2020-05-21), GB, pages 4643 - 4657, XP093134741, ISSN: 0305-1048, Retrieved from the Internet <URL:https://watermark.silverchair.com/gkaa222.pdf?token=AQECAHi208BE49Ooan9kkhW_Ercy7Dm3ZL_9Cf3qfKAc485ysgAAA2UwggNhBgkqhkiG9w0BBwagggNSMIIDTgIBADCCA0cGCSqGSIb3DQEHATAeBglghkgBZQMEAS4wEQQMPjlmya_Sg5wroXc0AgEQgIIDGAtyt6-G8374AZjr4A3wDFG15Dqe98ZmkUBqGbtljI3egSWUPRJ0P-qPfIP__lSS7nCSd7WbHoJsFLT4MkQUV6x95y_O> DOI: 10.1093/nar/gkaa222 *
PRAKASH ET AL: "RNA interference by 2',5'-linked nucleic acid duplexes in mammalian cells", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, ELSEVIER, AMSTERDAM NL, vol. 16, no. 12, 15 June 2006 (2006-06-15), pages 3238 - 3240, XP005422615, ISSN: 0960-894X, DOI: 10.1016/J.BMCL.2006.03.053 *
PRAKASH T P ET AL: "RNA interference by 2',5'-linked nucleic acid duplexes in mammalian cells", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, ELSEVIER, AMSTERDAM NL, vol. 16, no. 12, 15 June 2006 (2006-06-15), pages 3238 - 3240, XP027965587, ISSN: 0960-894X, [retrieved on 20060615] *
See also references of WO2022099159A1 *
SHEN WEN ET AL: "2'-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF", NUCLEIC ACIDS RESEARCH, vol. 43, no. 9, 19 May 2015 (2015-05-19), GB, pages 4569 - 4578, XP055863433, ISSN: 0305-1048, Retrieved from the Internet <URL:https://academic.oup.com/nar/article-pdf/43/9/4569/5153015/gkv298.pdf> DOI: 10.1093/nar/gkv298 *

Also Published As

Publication number Publication date
WO2022099159A1 (en) 2022-05-12
CA3197311A1 (en) 2022-05-12
AU2021373062A9 (en) 2024-10-24
IL302558A (en) 2023-07-01
JP2023548584A (en) 2023-11-17
AU2021373062A1 (en) 2023-06-08
KR20230118716A (en) 2023-08-11
MX2023005326A (en) 2023-07-31
EP4240849A1 (en) 2023-09-13

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