JP2024535388A - オリゴヌクレオチド組成物及びその方法 - Google Patents

オリゴヌクレオチド組成物及びその方法 Download PDF

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JP2024535388A
JP2024535388A JP2024518651A JP2024518651A JP2024535388A JP 2024535388 A JP2024535388 A JP 2024535388A JP 2024518651 A JP2024518651 A JP 2024518651A JP 2024518651 A JP2024518651 A JP 2024518651A JP 2024535388 A JP2024535388 A JP 2024535388A
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Japan
Prior art keywords
oligonucleotide
bond
nucleoside
sfc
nucleobase
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JP2024518651A
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Japanese (ja)
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JP2024535388A5 (enExample
Inventor
モニアン，パラシャント
シャクティ シヴァリラ，チック
ルー，ゲンリャン
ヴァルギーズ，チャンドラ
ホーバン ジャングランデ，パロマ
カンダサミー，パチャムトゥ
直樹 岩本
護 清水
ユ，ホイ
ヤン，ハイリン
ダイアン ラモア，サラ
チャン，フェンジャオ
ナラヤナン，パドマクマール
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Priority claimed from PCT/US2021/058495 external-priority patent/WO2022099159A1/en
Application filed by Wave Life Sciences Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of JP2024535388A publication Critical patent/JP2024535388A/ja
Publication of JP2024535388A5 publication Critical patent/JP2024535388A5/ja
Pending legal-status Critical Current

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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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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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  • 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)
JP2024518651A 2021-09-26 2022-09-26 オリゴヌクレオチド組成物及びその方法 Pending JP2024535388A (ja)

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

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JP2024535388A true JP2024535388A (ja) 2024-09-30
JP2024535388A5 JP2024535388A5 (enExample) 2025-10-07

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US (1) US20250333740A1 (enExample)
EP (1) EP4396354A4 (enExample)
JP (1) JP2024535388A (enExample)
KR (1) KR20240063964A (enExample)
AU (1) AU2022352779A1 (enExample)
CA (1) CA3232070A1 (enExample)
CL (1) CL2024000856A1 (enExample)
CO (1) CO2024005352A2 (enExample)
IL (1) IL311625A (enExample)
MX (1) MX2024003612A (enExample)
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Families Citing this family (11)

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Publication number Priority date Publication date Assignee Title
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
JP2019520339A (ja) 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． オリゴヌクレオチド、その組成物および方法
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
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
JP2022525541A (ja) 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド調製に有用な技術
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
WO2008086524A2 (en) * 2007-01-11 2008-07-17 University Of Florida Research Foundation, Inc. Alpha-1-antitrypsin variants and uses thereof
EP3452596A4 (en) * 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
JP7347830B2 (ja) * 2018-06-29 2023-09-20 エバーハルト カール ウニヴェルジテート テュービンゲン Ｒｎａ編集のための人工核酸
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
JP7766589B2 (ja) * 2019-10-06 2025-11-10 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
KR20230118716A (ko) * 2020-11-08 2023-08-11 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법

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MX2024003612A (es) 2024-05-16
CL2024000856A1 (es) 2024-09-27
CA3232070A1 (en) 2023-03-30
EP4396354A1 (en) 2024-07-10
WO2023049475A1 (en) 2023-03-30
US20250333740A1 (en) 2025-10-30
AU2022352779A1 (en) 2024-03-28
IL311625A (en) 2024-05-01
KR20240063964A (ko) 2024-05-10
CO2024005352A2 (es) 2024-05-30
EP4396354A4 (en) 2025-12-03

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