JP6833705B2 - Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 - Google Patents

Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 Download PDF

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JP6833705B2
JP6833705B2 JP2017546624A JP2017546624A JP6833705B2 JP 6833705 B2 JP6833705 B2 JP 6833705B2 JP 2017546624 A JP2017546624 A JP 2017546624A JP 2017546624 A JP2017546624 A JP 2017546624A JP 6833705 B2 JP6833705 B2 JP 6833705B2
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certain embodiments
antisense
nucleoside
modified
compound
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JP2018511307A (ja
JP2018511307A5 (enExample
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グオ，スーリン
アガジャン，マリアム
スウェイジ，エリック・イー
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Ionis Pharmaceuticals Inc
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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
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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JP2017546624A 2015-04-03 2016-04-04 Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 Active JP6833705B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562142986P 2015-04-03 2015-04-03
US62/142,986 2015-04-03
PCT/US2016/025883 WO2016161429A1 (en) 2015-04-03 2016-04-04 Compounds and methods for modulating tmprss6 expression

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JP2018511307A JP2018511307A (ja) 2018-04-26
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JP2021015452A Active JP7037681B2 (ja) 2015-04-03 2021-02-03 Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法
JP2022033166A Withdrawn JP2022104917A (ja) 2015-04-03 2022-03-04 Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法

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JP2022033166A Withdrawn JP2022104917A (ja) 2015-04-03 2022-03-04 Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法

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US (4) US10415038B2 (enExample)
EP (1) EP3277817A4 (enExample)
JP (3) JP6833705B2 (enExample)
KR (1) KR102539587B1 (enExample)
CN (1) CN107429250B (enExample)
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TWI840345B (zh) * 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
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KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
JP2023130528A (ja) * 2020-06-24 2023-09-21 田辺三菱製薬株式会社 Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物
CA3195722A1 (en) 2020-09-17 2022-03-24 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
AU2022265493A1 (en) 2021-04-27 2023-09-21 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
EP4329774A4 (en) * 2021-04-28 2025-11-05 Quiver Holdings Inc THERAPEUTIC COMPOSITIONS FOR THE TREATMENT OF MULTI-TARGET PAIN
CN115247173B (zh) * 2021-11-11 2025-11-11 南京启真基因工程有限公司 构建tmprss6基因突变的缺铁性贫血猪核移植供体细胞的基因编辑系统及其应用
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
EP4596693A1 (en) * 2022-09-29 2025-08-06 Bebetter Med Inc. Sirna or salt thereof and medicament for inhibiting expression of tmprss6 gene, and use thereof
WO2024060649A1 (zh) * 2022-09-29 2024-03-28 广州必贝特医药股份有限公司 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用
WO2024200512A1 (en) 2023-03-27 2024-10-03 Silence Therapeutics Gmbh Compounds and compositions for use in stem cell transplantation
TW202527968A (zh) * 2024-01-08 2025-07-16 大陸商上海舶望製藥有限公司 用於抑制跨膜絲氨酸蛋白酶6（tmprss6）表達的組合物和方法
WO2025206092A1 (ja) * 2024-03-27 2025-10-02 田辺三菱製薬株式会社 Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物

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