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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    • C12N15/00—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
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • 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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US (1) US20220228141A1 (enExample)
EP (1) EP4247391A4 (enExample)
JP (1) JP2023550485A (enExample)
AU (1) AU2021382621A1 (enExample)
WO (1) WO2022109398A1 (enExample)

Families Citing this family (22)

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EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 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
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova 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
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
AU2024265748A1 (en) * 2023-05-04 2025-10-23 Argonaute RNA Limited Dual silencing
WO2024238752A1 (en) * 2023-05-17 2024-11-21 University Of Massachusetts Oligonucleotides for dgat2, fasn, opn, and nox4 modulations
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术（苏州）有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
TW202515586A (zh) * 2023-08-09 2025-04-16 大陸商北京福元醫藥股份有限公司 抑制DGAT2基因表達的siRNA、siRNA綴合物或其前藥和藥物組合物及用途
CN120035670A (zh) * 2023-08-23 2025-05-23 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
WO2026057029A1 (zh) * 2024-09-14 2026-03-19 信达生物制药(苏州)有限公司 一种dsRNA、其应用及制备方法
WO2026130386A1 (zh) * 2024-12-17 2026-06-25 苏州时安生物技术有限公司 一种调节dgat2表达的双链rna组合物、其缀合物和药物组合物及用途

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

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