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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JPWO2019200124A5 (https=
JP2021520355A5 (https=
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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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/52—Physical structure branched
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y102/00—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003—Aldehyde dehydrogenase (NAD+) (1.2.1.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9

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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (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 (3)

Publication Number Publication Date
JP2021520355A true JP2021520355A (ja) 2021-08-19
JPWO2019200124A5 JPWO2019200124A5 (https=) 2022-04-14
JP2021520355A5 JP2021520355A5 (https=) 2022-04-14

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JP2020554092A Withdrawn JP2021520355A (ja) 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

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

Cited By (1)

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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

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
AU2019397247A1 (en) * 2018-12-12 2021-06-24 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop
WO2020226960A1 (en) * 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
US20240287512A1 (en) * 2021-05-11 2024-08-29 Dicerna Pharmaceuticals, Inc. Lipid conjugation for targeting neurons of the central nervous system
WO2023069707A2 (en) * 2021-10-22 2023-04-27 Sirnaomics, Inc. Products and compositions

Citations (3)

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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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WO2009117589A1 (en) 2008-03-21 2009-09-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucleosides and methods for their use
US20110288147A1 (en) 2008-09-22 2011-11-24 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop
AU2009336191B2 (en) * 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
CN104651362A (zh) * 2009-04-03 2015-05-27 戴瑟纳制药公司 利用不对称双链rna特异性抑制kras的方法和组合物
US20140011860A1 (en) 2010-07-19 2014-01-09 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
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CA2902571A1 (en) * 2013-03-15 2014-09-18 MiRagen Therapeutics, Inc. Bridged bicyclic nucleosides
WO2014140348A1 (en) 2013-03-15 2014-09-18 Universität Bern Tricyclic nucleosides and oligomeric compounds prepared therefrom
DK3444350T3 (da) * 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna
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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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MX2020010802A (es) 2020-10-28
EP3773609A4 (en) 2022-05-18
KR20200144100A (ko) 2020-12-28
AU2019252529A1 (en) 2020-10-01
IL277813A (en) 2020-11-30
US20190316125A1 (en) 2019-10-17
WO2019200124A1 (en) 2019-10-17
US11873488B2 (en) 2024-01-16
EP3773609A1 (en) 2021-02-17
CN111971051A (zh) 2020-11-20
CA3094008A1 (en) 2019-10-17

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