JP7211933B2 - 転写プロセシングの調節のための化合物及び方法 - Google Patents

転写プロセシングの調節のための化合物及び方法 Download PDF

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JP7211933B2
JP7211933B2 JP2019501570A JP2019501570A JP7211933B2 JP 7211933 B2 JP7211933 B2 JP 7211933B2 JP 2019501570 A JP2019501570 A JP 2019501570A JP 2019501570 A JP2019501570 A JP 2019501570A JP 7211933 B2 JP7211933 B2 JP 7211933B2
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modified
oligomeric compound
modified oligonucleotide
nucleosides
certain embodiments
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JP2019527549A5 (enExample
JP2019527549A (ja
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プラカシュ，タジャ・ピー
リゴ，フランク
セス，プニット・ピー
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Ionis Pharmaceuticals Inc
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Ionis 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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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JP2019501570A 2016-07-15 2017-07-17 転写プロセシングの調節のための化合物及び方法 Active JP7211933B2 (ja)

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US201662363193P 2016-07-15 2016-07-15
US62/363,193 2016-07-15
PCT/US2017/042465 WO2018014043A1 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of transcript processing

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JP2022188237A (ja) * 2016-07-15 2022-12-20 アイオーニス ファーマシューティカルズ， インコーポレーテッド 転写プロセシングの調節のための化合物及び方法

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BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
AR121446A1 (es) * 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
CN119213005A (zh) 2022-03-16 2024-12-27 艾姆皮瑞克公司 用于提高siRNA生物利用度的GALNAC组合物
WO2023192830A2 (en) 2022-03-28 2023-10-05 Empirico Inc. Modified oligonucleotides

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US20160068845A1 (en) 2010-07-19 2016-03-10 University Of Rochester Modulation of dystrophia myotonica-protein kinase (dmpk) expression

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US20130203836A1 (en) 2010-04-01 2013-08-08 Isis Pharmaceuticals, Inc. 2' and 5' modified monomers and oligonucleotides
US20160068845A1 (en) 2010-07-19 2016-03-10 University Of Rochester Modulation of dystrophia myotonica-protein kinase (dmpk) expression

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022188237A (ja) * 2016-07-15 2022-12-20 アイオーニス ファーマシューティカルズ， インコーポレーテッド 転写プロセシングの調節のための化合物及び方法

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CA3027177A1 (en) 2018-01-18
AU2017297624B2 (en) 2023-11-02
JP2019527549A (ja) 2019-10-03
US20190321387A1 (en) 2019-10-24
US20210315918A1 (en) 2021-10-14
JP2022188237A (ja) 2022-12-20
AU2017297624A1 (en) 2018-11-29
EP3485016A4 (en) 2020-03-18

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