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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Prior art keywords
oligonucleotide
bond
nucleoside
sfc
nucleobase
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Pending
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JP2024518651A
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Japanese (ja)
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JPWO2023049475A5 (https=
JP2024535388A5 (https=
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 JPWO2023049475A5 publication Critical patent/JPWO2023049475A5/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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    • A61P1/00—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
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    • 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
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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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  • Pulmonology (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
JPWO2023049475A5 JPWO2023049475A5 (https=) 2025-10-07
JP2024535388A5 JP2024535388A5 (https=) 2025-10-07

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JP2024518651A Pending JP2024535388A (ja) 2021-09-26 2022-09-26 オリゴヌクレオチド組成物及びその方法

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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
EP4396354A1 (en) 2024-07-10
KR20240063964A (ko) 2024-05-10
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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