CA3197311A1 - Oligonucleotide compositions and methods thereof - Google Patents

Oligonucleotide compositions and methods thereof

Info

Publication number
CA3197311A1
CA3197311A1 CA3197311A CA3197311A CA3197311A1 CA 3197311 A1 CA3197311 A1 CA 3197311A1 CA 3197311 A CA3197311 A CA 3197311A CA 3197311 A CA3197311 A CA 3197311A CA 3197311 A1 CA3197311 A1 CA 3197311A1
Authority
CA
Canada
Prior art keywords
methods
oligonucleotide compositions
compositions
present disclosure
disclosure provides
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
CA3197311A
Other languages
French (fr)
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
Individual
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 Individual filed Critical Individual
Publication of CA3197311A1 publication Critical patent/CA3197311A1/en
Pending 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
    • C12N15/113—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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds 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
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/321—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

Among other things, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders or diseases that can benefit from adenosine modification.
CA3197311A 2020-11-08 2021-11-08 Oligonucleotide compositions and methods thereof Pending CA3197311A1 (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 (1)

Publication Number Publication Date
CA3197311A1 true CA3197311A1 (en) 2022-05-12

Family

ID=81456778

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3197311A Pending CA3197311A1 (en) 2020-11-08 2021-11-08 Oligonucleotide compositions and methods thereof

Country Status (8)

Country Link
EP (1) EP4240849A1 (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 (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
WO2018098264A1 (en) 2016-11-23 2018-05-31 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
JP7557941B2 (en) 2017-06-02 2024-09-30 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
EP3642182A4 (en) 2017-06-21 2020-12-09 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
KR20200035301A (en) 2017-08-08 2020-04-02 웨이브 라이프 사이언시스 리미티드 Oligonucleotide composition and method
KR20200052369A (en) 2017-09-18 2020-05-14 웨이브 라이프 사이언시스 리미티드 Oligonucleotide production technology
CN115989041A (en) * 2020-08-24 2023-04-18 波涛生命科学有限公司 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
JP2024536088A (en) * 2021-09-26 2024-10-04 ウェイブ ライフ サイエンシズ リミテッド Compositions and methods for editing mecp2 transcripts
CA3234835A1 (en) * 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024017817A1 (en) * 2022-07-18 2024-01-25 F. Hoffmann-La Roche Ag Editing oligonucleotide
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
WO2024121373A1 (en) 2022-12-09 2024-06-13 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
WO2024175588A1 (en) 2023-02-21 2024-08-29 Vib Vzw Oligonucleotides for modulating synaptogyrin-3 expression
WO2024175586A2 (en) 2023-02-21 2024-08-29 Vib Vzw Inhibitors of synaptogyrin-3 expression
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2024200472A1 (en) 2023-03-27 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
CN116444593B (en) * 2023-04-28 2023-12-26 南通大学 Synthesis method of 7-deazaguanosine phosphoramidite monomer

Family Cites Families (4)

* 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
KR102501980B1 (en) * 2016-09-01 2023-02-20 프로큐알 테라퓨틱스 Ⅱ 비.브이. Chemically Modified Single-Stranded RNA-Editing Oligonucleotides
KR102678809B1 (en) * 2017-05-18 2024-06-26 더 브로드 인스티튜트, 인코퍼레이티드 Systems, methods and compositions for targeted nucleic acid editing
EP3642182A4 (en) * 2017-06-21 2020-12-09 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis

Also Published As

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

Similar Documents

Publication Publication Date Title
CA3197311A1 (en) Oligonucleotide compositions and methods thereof
MX2022004101A (en) Oligonucleotide compositions and methods of use thereof.
WO2020227691A3 (en) Oligonucleotide compositions and methods of use thereof
WO2021071788A3 (en) Oligonucleotide compositions and methods of use thereof
WO2020219981A3 (en) Oligonucleotide compositions and methods of use thereof
WO2007042554A3 (en) Methods and compositions for treating immune disorders
MX2022011550A (en) Compositions and methods for inhibiting angptl3 expression.
EP2450040A3 (en) Compositions and kits for treating influenza
EP2239328A3 (en) Methods and compositions for treating neurological disease
WO2005004794A3 (en) Method of treating neurodegenerative disease
EP2426203A3 (en) Agents useful in treating facioscapulohumeral muscular dystrophy
EP1676512A3 (en) Skin treatment articles and methods
WO2001098317A3 (en) Alpha-glycosylceramides for treating bacterial and fungal infections
WO2016168762A3 (en) Anti-pacap antibodies and uses thereof
GB2463833A (en) Methods and compositions for the treatment of neurological disorders
WO2022031847A3 (en) Compositions and methods for inhibiting plp1 expression
EP2862867A3 (en) Methods and compositions for the treatment of Marfan syndrome and associated disorders
WO2021081292A8 (en) METHODS FOR TREATING NEUROLOGICAL DISORDERS WITH α1A-AR PARTIAL AGONISTS
WO2005099702A3 (en) R(-)-11-hydroxyaporphine derivatives and uses thereof
WO2019209948A8 (en) Compounds and uses thereof
WO2019175260A3 (en) Modified oligonucleotides for use in treatment of tauopathies
WO2020132378A3 (en) Compositions and methods of treatment for neurological disorders comprising depression
MX2024003612A (en) Oligonucleotide compositions and methods thereof.
WO2023091644A3 (en) Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto
WO2021032777A8 (en) Oligonucleotide conjugate compositions and methods of use