JP2023550485A - Ｄｇａｔ２調節のためのオリゴヌクレオチド - Google Patents

Ｄｇａｔ２調節のためのオリゴヌクレオチド Download PDF

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JP2023550485A
JP2023550485A JP2023530843A JP2023530843A JP2023550485A JP 2023550485 A JP2023550485 A JP 2023550485A JP 2023530843 A JP2023530843 A JP 2023530843A JP 2023530843 A JP2023530843 A JP 2023530843A JP 2023550485 A JP2023550485 A JP 2023550485A
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dsrna
antisense strand
nucleotides
strand
sense strand
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JP2023550485A5 (enExample
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ピー チェック，マイケル
オルベイ イェニルメツ，バトゥハン
コボロバ，アナスタシア
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University of Massachusetts Amherst
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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
    • C—CHEMISTRY; METALLURGY
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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
    • A—HUMAN NECESSITIES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023530843A 2020-11-23 2021-11-22 Ｄｇａｔ２調節のためのオリゴヌクレオチド Pending JP2023550485A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063117005P 2020-11-23 2020-11-23
US63/117,005 2020-11-23
PCT/US2021/060356 WO2022109398A1 (en) 2020-11-23 2021-11-22 Oligonucleotides for dgat2 modulation

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JP2023550485A true JP2023550485A (ja) 2023-12-01
JP2023550485A5 JP2023550485A5 (enExample) 2024-11-29

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JP2023530843A Pending JP2023550485A (ja) 2020-11-23 2021-11-22 Ｄｇａｔ２調節のためのオリゴヌクレオチド

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Country Link
US (1) US20220228141A1 (enExample)
EP (1) EP4247391A4 (enExample)
JP (1) JP2023550485A (enExample)
AU (1) AU2021382621A1 (enExample)
WO (1) WO2022109398A1 (enExample)

Families Citing this family (20)

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US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4181932A4 (en) * 2020-07-16 2025-07-16 Univ Massachusetts CONJUGATED OLIGONUCLEOTIDES FOR SPECIFIC TISSUE DELIVERY
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
WO2024228030A2 (en) * 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing
KR20260006569A (ko) * 2023-05-04 2026-01-13 아르고노트 알엔에이 리미티드 이중 발현 억제
WO2024238752A1 (en) * 2023-05-17 2024-11-21 University Of Massachusetts Oligonucleotides for dgat2, fasn, opn, and nox4 modulations
EP4722365A1 (en) * 2023-05-26 2026-04-08 Visirna Therapeutics (Suzhou) Co., Ltd. Double-stranded nucleotide compound for metabolic diseases and use thereof
CN119464281A (zh) * 2023-08-09 2025-02-18 北京福元医药股份有限公司 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途
WO2025040139A1 (zh) * 2023-08-23 2025-02-27 云合智药（苏州）生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
CN117257764A (zh) * 2023-09-26 2023-12-22 中国药科大学 一种脂滴衍生的核酸药物递送系统及其制备方法和应用
CN121825964A (zh) * 2023-12-26 2026-04-10 苏州吉玛基因股份有限公司 用于抑制DGAT2的siRNA及其修饰物与应用

Citations (3)

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JP2007502616A (ja) * 2003-08-18 2007-02-15 アイシス ファーマシューティカルズ インコーポレイテッド ジアシルグリセロールアシルトランスフェラーゼ２発現の調節
JP2018525030A (ja) * 2015-07-10 2018-09-06 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． ジアシルグリセロールアシルトランスフェラーゼ２（ｄｇａｔ２）の調節剤
WO2022031850A2 (en) * 2020-08-04 2022-02-10 Dicerna Pharmaceuticals Inc. Compositions and methods for the treatment of metabolic syndrome

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JP2018525030A (ja) * 2015-07-10 2018-09-06 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． ジアシルグリセロールアシルトランスフェラーゼ２（ｄｇａｔ２）の調節剤
WO2022031850A2 (en) * 2020-08-04 2022-02-10 Dicerna Pharmaceuticals Inc. Compositions and methods for the treatment of metabolic syndrome

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WO2022109398A1 (en) 2022-05-27
AU2021382621A9 (en) 2024-02-08
WO2022109398A9 (en) 2022-09-29
US20220228141A1 (en) 2022-07-21
AU2021382621A1 (en) 2023-06-22
EP4247391A1 (en) 2023-09-27
EP4247391A4 (en) 2025-11-19

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