KR20240063964A - 올리고뉴클레오티드 조성물 및 이의 방법 - Google Patents

올리고뉴클레오티드 조성물 및 이의 방법 Download PDF

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Publication number
KR20240063964A
KR20240063964A KR1020247012540A KR20247012540A KR20240063964A KR 20240063964 A KR20240063964 A KR 20240063964A KR 1020247012540 A KR1020247012540 A KR 1020247012540A KR 20247012540 A KR20247012540 A KR 20247012540A KR 20240063964 A KR20240063964 A KR 20240063964A
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South Korea
Prior art keywords
oligonucleotide
nucleoside
linkage
sfc
modification
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Pending
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KR1020247012540A
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English (en)
Korean (ko)
Inventor
프라샨트 모니안
치크두 샤크티 쉬바닐라
건량 루
챈드라 바기즈
팔로마 호반 기안그란데
파차무투 칸다사미
나오키 이와모토
마모루 시미즈
후이 위
하이린 양
사라 다이앤 라모어
펑자오 장
파드마쿠마르 나라야난
Original Assignee
웨이브 라이프 사이언시스 리미티드
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Priority claimed from PCT/US2021/058495 external-priority patent/WO2022099159A1/en
Application filed by 웨이브 라이프 사이언시스 리미티드 filed Critical 웨이브 라이프 사이언시스 리미티드
Publication of KR20240063964A publication Critical patent/KR20240063964A/ko
Pending legal-status Critical Current

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    • 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
    • C12N15/1137—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 against enzymes
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21011—Elastase (3.4.21.11)

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pulmonology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247012540A 2021-09-26 2022-09-26 올리고뉴클레오티드 조성물 및 이의 방법 Pending KR20240063964A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163248520P 2021-09-26 2021-09-26
US63/248,520 2021-09-26
PCT/US2021/058495 WO2022099159A1 (en) 2020-11-08 2021-11-08 Oligonucleotide compositions and methods thereof
USPCT/US2021/058495 2021-11-08
US202263331756P 2022-04-15 2022-04-15
US63/331,756 2022-04-15
US202263397320P 2022-08-11 2022-08-11
US63/397,320 2022-08-11
PCT/US2022/044765 WO2023049475A1 (en) 2021-09-26 2022-09-26 Oligonucleotide compositions and methods thereof

Publications (1)

Publication Number Publication Date
KR20240063964A true KR20240063964A (ko) 2024-05-10

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KR1020247012540A Pending KR20240063964A (ko) 2021-09-26 2022-09-26 올리고뉴클레오티드 조성물 및 이의 방법

Country Status (11)

Country Link
US (1) US20250333740A1 (https=)
EP (1) EP4396354A4 (https=)
JP (1) JP2024535388A (https=)
KR (1) KR20240063964A (https=)
AU (1) AU2022352779A1 (https=)
CA (1) CA3232070A1 (https=)
CL (1) CL2024000856A1 (https=)
CO (1) CO2024005352A2 (https=)
IL (1) IL311625A (https=)
MX (1) MX2024003612A (https=)
WO (1) WO2023049475A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
CN113748116A (zh) 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
WO2026077951A1 (en) 2024-10-07 2026-04-16 Vico Therapeutics B.V. Improved aon for rna editing

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100048680A1 (en) * 2007-01-11 2010-02-25 University Of Florida Research Foundation Inc. Alpha-1-Antitrypsin Variants and Uses Thereof
JP2019516680A (ja) * 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物およびその方法
WO2020001793A1 (en) * 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
CN114585737A (zh) * 2019-10-06 2022-06-03 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
EP4240849A4 (en) * 2020-11-08 2026-04-01 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES

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WO2023049475A1 (en) 2023-03-30
CL2024000856A1 (es) 2024-09-27
CA3232070A1 (en) 2023-03-30
JP2024535388A (ja) 2024-09-30
EP4396354A1 (en) 2024-07-10
MX2024003612A (es) 2024-05-16
IL311625A (en) 2024-05-01
EP4396354A4 (en) 2025-12-03
AU2022352779A1 (en) 2024-03-28
US20250333740A1 (en) 2025-10-30
CO2024005352A2 (es) 2024-05-30

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