JP7289610B2 - 修飾二本鎖ｒｎａ剤 - Google Patents

修飾二本鎖ｒｎａ剤 Download PDF

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JP7289610B2
JP7289610B2 JP2017509743A JP2017509743A JP7289610B2 JP 7289610 B2 JP7289610 B2 JP 7289610B2 JP 2017509743 A JP2017509743 A JP 2017509743A JP 2017509743 A JP2017509743 A JP 2017509743A JP 7289610 B2 JP7289610 B2 JP 7289610B2
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ome
antisense strand
positions
dsrna agent
dsrna
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JP2017525705A5 (enExample
JP2017525705A (ja
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マルティン・マイアー
ドン・フォスター
スチュアート・ミルスタイン
サティア・クチマンチ
バサント・ジャダブ
カラントッタティル・ラジーブ
ムティア・マノハラン
ルビーナ・パルマル
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of JP2017525705A publication Critical patent/JP2017525705A/ja
Publication of JP2017525705A5 publication Critical patent/JP2017525705A5/ja
Priority to JP2020212528A priority Critical patent/JP7672220B2/ja
Priority to JP2020212527A priority patent/JP7370311B2/ja
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Publication of JP7289610B2 publication Critical patent/JP7289610B2/ja
Priority to JP2023178574A priority patent/JP7654744B2/ja
Priority to JP2025044214A priority patent/JP2025111426A/ja
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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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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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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
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2320/52—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)
  • Saccharide Compounds (AREA)
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JP2017509743A 2014-08-20 2015-08-14 修飾二本鎖ｒｎａ剤 Active JP7289610B2 (ja)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2020212528A JP7672220B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2020212527A JP7370311B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2023178574A JP7654744B2 (ja) 2014-08-20 2023-10-17 修飾二本鎖ｒｎａ剤
JP2025044214A JP2025111426A (ja) 2014-08-20 2025-03-19 修飾二本鎖ｒｎａ剤

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462039507P 2014-08-20 2014-08-20
US62/039,507 2014-08-20
US201462083744P 2014-11-24 2014-11-24
US62/083,744 2014-11-24
US201462093919P 2014-12-18 2014-12-18
US62/093,919 2014-12-18
PCT/US2015/045407 WO2016028649A1 (en) 2014-08-20 2015-08-14 Modified double-stranded rna agents

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JP2020212528A Division JP7672220B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2020212527A Division JP7370311B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤

Publications (3)

Publication Number Publication Date
JP2017525705A JP2017525705A (ja) 2017-09-07
JP2017525705A5 JP2017525705A5 (enExample) 2018-09-20
JP7289610B2 true JP7289610B2 (ja) 2023-06-12

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JP2017509743A Active JP7289610B2 (ja) 2014-08-20 2015-08-14 修飾二本鎖ｒｎａ剤
JP2020212527A Active JP7370311B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2020212528A Active JP7672220B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2023178574A Active JP7654744B2 (ja) 2014-08-20 2023-10-17 修飾二本鎖ｒｎａ剤
JP2025044214A Pending JP2025111426A (ja) 2014-08-20 2025-03-19 修飾二本鎖ｒｎａ剤

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JP2020212527A Active JP7370311B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2020212528A Active JP7672220B2 (ja) 2014-08-20 2020-12-22 修飾二本鎖ｒｎａ剤
JP2023178574A Active JP7654744B2 (ja) 2014-08-20 2023-10-17 修飾二本鎖ｒｎａ剤
JP2025044214A Pending JP2025111426A (ja) 2014-08-20 2025-03-19 修飾二本鎖ｒｎａ剤

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US (12) US10233448B2 (enExample)
EP (4) EP4585686A3 (enExample)
JP (5) JP7289610B2 (enExample)
KR (4) KR102630289B1 (enExample)
CN (3) CN114181942B (enExample)
CA (1) CA2958758A1 (enExample)
EA (1) EA201790420A1 (enExample)
IL (3) IL316808A (enExample)
MX (3) MX2017002144A (enExample)
NZ (1) NZ730296A (enExample)
SG (3) SG11201701166UA (enExample)
WO (1) WO2016028649A1 (enExample)

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TWI865997B (zh) * 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
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