CN111971051A - 用增加tm的核苷酸修饰的双链核酸抑制剂分子 - Google Patents

用增加tm的核苷酸修饰的双链核酸抑制剂分子 Download PDF

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Publication number
CN111971051A
CN111971051A CN201980025615.0A CN201980025615A CN111971051A CN 111971051 A CN111971051 A CN 111971051A CN 201980025615 A CN201980025615 A CN 201980025615A CN 111971051 A CN111971051 A CN 111971051A
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nucleic acid
nucleotides
double
acid inhibitor
stranded nucleic
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CN201980025615.0A
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Chinese (zh)
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W·王
N·内泽夫
B·D·布朗
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • C12N15/09—Recombinant DNA-technology
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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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    • 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)
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201980025615.0A 2018-04-13 2019-04-11 用增加tm的核苷酸修饰的双链核酸抑制剂分子 Pending CN111971051A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862657428P 2018-04-13 2018-04-13
US62/657428 2018-04-13
US201862778755P 2018-12-12 2018-12-12
US62/778755 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 (1)

Publication Number Publication Date
CN111971051A true CN111971051A (zh) 2020-11-20

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CN201980025615.0A Pending CN111971051A (zh) 2018-04-13 2019-04-11 用增加tm的核苷酸修饰的双链核酸抑制剂分子

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Country Link
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113454221A (zh) * 2018-12-12 2021-09-28 迪克纳制药公司 含有三元环的双链核酸抑制剂分子

Families Citing this family (4)

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Publication number Priority date Publication date Assignee Title
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
US20250188477A1 (en) * 2022-03-16 2025-06-12 Daiichi Sankyo Company, Limited CHEMICALLY MODIFIED OLIGONUCLEOTIDE HAVING RNAi ACTIVITY

Citations (4)

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WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
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
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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US8273866B2 (en) * 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
WO2009117589A1 (en) 2008-03-21 2009-09-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucleosides and methods for their use
KR101728655B1 (ko) * 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
CN103555722B (zh) * 2009-04-03 2016-08-31 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
WO2012027033A1 (en) 2010-07-19 2012-03-01 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
DK2970968T3 (en) * 2013-03-15 2018-03-05 Miragen Therapeutics Inc CONNECTED BICYCLIC Nucleosides
US9862742B2 (en) 2013-03-15 2018-01-09 Universität Bern Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9458457B2 (en) * 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
US10150965B2 (en) * 2013-12-06 2018-12-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
US10036017B2 (en) * 2015-02-17 2018-07-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA
HUE059718T2 (hu) * 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
IL271680B (en) * 2017-06-29 2022-09-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression

Patent Citations (4)

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Publication number Priority date Publication date Assignee Title
WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
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
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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113454221A (zh) * 2018-12-12 2021-09-28 迪克纳制药公司 含有三元环的双链核酸抑制剂分子

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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
KR20200144100A (ko) 2020-12-28
US20190316125A1 (en) 2019-10-17
JP2021520355A (ja) 2021-08-19
EP3773609A1 (en) 2021-02-17
MX2020010802A (es) 2020-10-28
US11873488B2 (en) 2024-01-16

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