JP2021520355A - Tm上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子 - Google Patents

Tm上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子 Download PDF

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JP2021520355A
JP2021520355A JP2020554092A JP2020554092A JP2021520355A JP 2021520355 A JP2021520355 A JP 2021520355A JP 2020554092 A JP2020554092 A JP 2020554092A JP 2020554092 A JP2020554092 A JP 2020554092A JP 2021520355 A JP2021520355 A JP 2021520355A
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nucleic acid
nucleotides
double
acid inhibitor
stranded nucleic
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JP2021520355A5 (enExample
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ウェイミン ワン
ウェイミン ワン
ネイム ナゼフ
ネイム ナゼフ
ボブ デール ブラウン
ボブ デール ブラウン
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Dicerna Pharmaceuticals Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
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    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/52Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
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    • C12Y102/00Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003Aldehyde dehydrogenase (NAD+) (1.2.1.3)
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    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21061Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020554092A 2018-04-13 2019-04-11 Tm上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子 Withdrawn JP2021520355A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862657428P 2018-04-13 2018-04-13
US62/657,428 2018-04-13
US201862778755P 2018-12-12 2018-12-12
US62/778,755 2018-12-12
PCT/US2019/027021 WO2019200124A1 (en) 2018-04-13 2019-04-11 Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides

Publications (2)

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JP2021520355A true JP2021520355A (ja) 2021-08-19
JP2021520355A5 JP2021520355A5 (enExample) 2022-04-14

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US (1) US11873488B2 (enExample)
EP (1) EP3773609A4 (enExample)
JP (1) JP2021520355A (enExample)
KR (1) KR20200144100A (enExample)
CN (1) CN111971051A (enExample)
AU (1) AU2019252529A1 (enExample)
CA (1) CA3094008A1 (enExample)
IL (1) IL277813A (enExample)
MX (1) MX2020010802A (enExample)
WO (1) WO2019200124A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023176863A1 (ja) * 2022-03-16 2023-09-21 第一三共株式会社 RNAi活性を有する化学修飾オリゴヌクレオチド

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WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop
AU2020268798A1 (en) * 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
TW202308662A (zh) * 2021-05-11 2023-03-01 美商黛瑟納製藥公司 用於靶定中樞神經系統的神經元之脂質結合物
WO2023069707A2 (en) * 2021-10-22 2023-04-27 Sirnaomics, Inc. Products and compositions

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JP2012504389A (ja) * 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ, インコーポレイテッド 修飾を有するdsRNAによる遺伝子発現の特異的な阻害のための組成物および方法
WO2016057932A1 (en) * 2014-10-10 2016-04-14 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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KR101728655B1 (ko) * 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
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WO2016057932A1 (en) * 2014-10-10 2016-04-14 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023176863A1 (ja) * 2022-03-16 2023-09-21 第一三共株式会社 RNAi活性を有する化学修飾オリゴヌクレオチド

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EP3773609A4 (en) 2022-05-18
IL277813A (en) 2020-11-30
AU2019252529A1 (en) 2020-10-01
CA3094008A1 (en) 2019-10-17
WO2019200124A1 (en) 2019-10-17
CN111971051A (zh) 2020-11-20
KR20200144100A (ko) 2020-12-28
US20190316125A1 (en) 2019-10-17
EP3773609A1 (en) 2021-02-17
MX2020010802A (es) 2020-10-28
US11873488B2 (en) 2024-01-16

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