KR20230118716A - 올리고뉴클레오티드 조성물 및 이의 방법 - Google Patents

올리고뉴클레오티드 조성물 및 이의 방법 Download PDF

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KR20230118716A
KR20230118716A KR1020237019215A KR20237019215A KR20230118716A KR 20230118716 A KR20230118716 A KR 20230118716A KR 1020237019215 A KR1020237019215 A KR 1020237019215A KR 20237019215 A KR20237019215 A KR 20237019215A KR 20230118716 A KR20230118716 A KR 20230118716A
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South Korea
Prior art keywords
oligonucleotide
oligonucleotides
sugar
nucleic acid
composition
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KR1020237019215A
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English (en)
Korean (ko)
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프라샨트 모니안
칙두 샥티 쉬바릴라
수브라마니안 마라판
찬드라 바르게스
파차무투 칸다사미
젠리앙 루
휘 유
데이비드 찰스 도넬 버터
루치아노 헨리께 아포니
마모루 시미즈
스테퍼니 미셸 스탠들리
데이빗 존 불레이
앤드류 귀자이어 호쓰
지가르 데자이
잭 데이빗 갓프리
하이린 양
나오키 이와모토
자야칸탄 쿠마라사미
앤서니 라마티나
탐 롄탕 푸
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웨이브 라이프 사이언시스 리미티드
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Publication of KR20230118716A publication Critical patent/KR20230118716A/ko

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    • 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
    • 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
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    • 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)
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
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    • 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)

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  • 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)
KR1020237019215A 2020-11-08 2021-11-08 올리고뉴클레오티드 조성물 및 이의 방법 KR20230118716A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063111079P 2020-11-08 2020-11-08
US63/111,079 2020-11-08
US202163175036P 2021-04-14 2021-04-14
US63/175,036 2021-04-14
US202163188415P 2021-05-13 2021-05-13
US63/188,415 2021-05-13
US202163196178P 2021-06-02 2021-06-02
US63/196,178 2021-06-02
US202163248520P 2021-09-26 2021-09-26
US63/248,520 2021-09-26
PCT/US2021/058495 WO2022099159A1 (fr) 2020-11-08 2021-11-08 Compositions d'oligonucléotides et procédés associés

Publications (1)

Publication Number Publication Date
KR20230118716A true KR20230118716A (ko) 2023-08-11

Family

ID=81456778

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237019215A KR20230118716A (ko) 2020-11-08 2021-11-08 올리고뉴클레오티드 조성물 및 이의 방법

Country Status (8)

Country Link
EP (1) EP4240849A1 (fr)
JP (1) JP2023548584A (fr)
KR (1) KR20230118716A (fr)
AU (1) AU2021373062A1 (fr)
CA (1) CA3197311A1 (fr)
IL (1) IL302558A (fr)
MX (1) MX2023005326A (fr)
WO (1) WO2022099159A1 (fr)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
EP3544987A4 (fr) 2016-11-23 2020-11-18 Wave Life Sciences Ltd. Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
TW201904587A (zh) 2017-06-02 2019-02-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法
WO2018237194A1 (fr) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Composés, compositions et procédés de synthèse
AU2018316159A1 (en) 2017-08-08 2020-02-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3684755A4 (fr) 2017-09-18 2022-03-02 Wave Life Sciences Ltd. Technologies de préparation d'oligonucléotides
EP4363574A1 (fr) * 2021-06-29 2024-05-08 Korro Bio, Inc. Procédés et compositions pour édition médiée par adar
AU2022349529A1 (en) * 2021-09-26 2024-05-02 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
WO2023069603A1 (fr) * 2021-10-22 2023-04-27 Korro Bio, Inc. Procédés et compositions pour perturber l'interaction de la protéine nrf2-keap1 par l'édition d'arn à médiation adar
WO2023152371A1 (fr) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie
WO2024013361A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
WO2024013360A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar
WO2024017817A1 (fr) * 2022-07-18 2024-01-25 F. Hoffmann-La Roche Ag Oligonucléotide d'édition
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (fr) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de l'hémochromatose hfe héréditaire
WO2024114908A1 (fr) 2022-11-30 2024-06-06 Eberhard Karls Universität Tübingen Oligonucléotides antisens chimiquement modifiés (asos) et compositions les comprenant pour l'édition d'arn
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
WO2024121373A1 (fr) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire
CN116444593B (zh) * 2023-04-28 2023-12-26 南通大学 一种7-脱氮鸟苷亚磷酰胺单体的合成方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014074597A1 (fr) * 2012-11-06 2014-05-15 President And Fellows Of Harvard College Compositions et procédés se rapportant à des nanostructures d'acides nucléiques complexes
NZ751483A (en) * 2016-09-01 2022-07-01 Proqr Therapeutics Ii Bv Chemically modified single-stranded rna-editing oligonucleotides
CA3063739A1 (fr) * 2017-05-18 2018-11-22 The Broad Institute, Inc. Systemes, procedes et compositions d'edition ciblee d'acides nucleiques
WO2018237194A1 (fr) * 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Composés, compositions et procédés de synthèse

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Publication number Publication date
EP4240849A1 (fr) 2023-09-13
AU2021373062A1 (en) 2023-06-08
CA3197311A1 (fr) 2022-05-12
IL302558A (en) 2023-07-01
MX2023005326A (es) 2023-07-31
JP2023548584A (ja) 2023-11-17
WO2022099159A1 (fr) 2022-05-12

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