JP2022530678A - 短縮センス鎖を有する二本鎖核酸阻害剤分子 - Google Patents

短縮センス鎖を有する二本鎖核酸阻害剤分子 Download PDF

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JP2022530678A
JP2022530678A JP2021564975A JP2021564975A JP2022530678A JP 2022530678 A JP2022530678 A JP 2022530678A JP 2021564975 A JP2021564975 A JP 2021564975A JP 2021564975 A JP2021564975 A JP 2021564975A JP 2022530678 A JP2022530678 A JP 2022530678A
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nucleotides
double
nucleic acid
strand
nucleotide
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JPWO2020226960A5 (https=
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ボブ デール ブラウン
ウェイミン ワン
ネイム ナゼフ
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Dicerna Pharmaceuticals Inc
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/323—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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • 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
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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

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021564975A 2019-05-03 2020-04-29 短縮センス鎖を有する二本鎖核酸阻害剤分子 Withdrawn JP2022530678A (ja)

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US201962842908P 2019-05-03 2019-05-03
US62/842,908 2019-05-03
PCT/US2020/030398 WO2020226960A1 (en) 2019-05-03 2020-04-29 Double-stranded nucleic acid inhibitor molecules with shortened sense strands

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JP2022530678A true JP2022530678A (ja) 2022-06-30
JPWO2020226960A5 JPWO2020226960A5 (https=) 2023-05-10
JP2022530678A5 JP2022530678A5 (https=) 2023-05-10

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US (1) US12529053B2 (https=)
EP (1) EP3963072A1 (https=)
JP (1) JP2022530678A (https=)
KR (1) KR20220004675A (https=)
CN (1) CN113795581A (https=)
AU (1) AU2020268798A1 (https=)
BR (1) BR112021021686A2 (https=)
CA (1) CA3136676A1 (https=)
CL (1) CL2021002842A1 (https=)
IL (1) IL287354A (https=)
MX (1) MX2021013418A (https=)
SG (1) SG11202111077RA (https=)
WO (1) WO2020226960A1 (https=)

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Publication number Priority date Publication date Assignee Title
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
WO2023081500A2 (en) * 2021-11-08 2023-05-11 Dicerna Pharmaceuticals, Inc. RNAi OLIGONUCLEOTIDE CONJUGATES
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物

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JP2006516191A (ja) * 2002-11-21 2006-06-29 ワイス ループス腎炎の治療用組成物および方法
JP2010532872A (ja) * 2007-07-06 2010-10-14 プロメディオール， インコーポレイテッド 線維化関連障害のための治療および診断方法
JP2012500389A (ja) * 2008-08-12 2012-01-05 ジ・オハイオ・ステイト・ユニバーシティ・リサーチ・ファウンデイション 多発性脊髄腫の診断、予後および治療のためのマイクロｒｎａに基づく組成物および方法
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
WO2018045317A1 (en) * 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
WO2019136157A2 (en) * 2018-01-05 2019-07-11 Dicerna Pharmaceuticals, Inc. Reducing beta-catenin and ido expression to potentiate immunotherapy
WO2019200124A1 (en) * 2018-04-13 2019-10-17 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides

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