JP2021515784A - 修飾オリゴヌクレオチド及びタウオパチーにおける使用方法 - Google Patents

修飾オリゴヌクレオチド及びタウオパチーにおける使用方法 Download PDF

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JP2021515784A
JP2021515784A JP2020547392A JP2020547392A JP2021515784A JP 2021515784 A JP2021515784 A JP 2021515784A JP 2020547392 A JP2020547392 A JP 2020547392A JP 2020547392 A JP2020547392 A JP 2020547392A JP 2021515784 A JP2021515784 A JP 2021515784A
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oligonucleotide
modified
formula
nucleotides
nucleotide
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JP2021515784A5 (enExample
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エブネス，アンドレアス
オートリヴ ディデワル，コンスタンティン ファン
オートリヴ ディデワル，コンスタンティン ファン
グリャズノフ，セルゲイ
モンテロ，サウール マルティネス
モンテロ，サウール マルティネス
ベイゲルマン，レオニド
クマール ラジワンシ，ヴィヴェク
クマール ラジワンシ，ヴィヴェク
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ヤンセン ファーマシューティカ エヌ．ベー．
ヤンセン ファーマシューティカ エヌ．ベー．
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
    • C12N2310/30—Chemical structure
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    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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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
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/50—Methods for regulating/modulating their activity

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
JP2020547392A 2018-03-13 2019-03-13 修飾オリゴヌクレオチド及びタウオパチーにおける使用方法 Pending JP2021515784A (ja)

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US201862642499P 2018-03-13 2018-03-13
US62/642,499 2018-03-13
PCT/EP2019/056312 WO2019175260A2 (en) 2018-03-13 2019-03-13 Modified oligonucleotides and methods of use in tauopathies

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JP2021515784A true JP2021515784A (ja) 2021-06-24
JP2021515784A5 JP2021515784A5 (enExample) 2022-03-14

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EP (2) EP3922637A1 (enExample)
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KR (1) KR20200131263A (enExample)
CN (1) CN111868068A (enExample)
AU (1) AU2019233612A1 (enExample)
BR (1) BR112020018365A2 (enExample)
CA (1) CA3091979A1 (enExample)
EA (1) EA202092156A1 (enExample)
ES (1) ES3063421T3 (enExample)
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MA (1) MA52149A (enExample)
MX (1) MX2020009532A (enExample)
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CA2866392C (en) 2012-03-30 2023-10-24 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
TWI702046B (zh) 2013-07-19 2020-08-21 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
US20230250125A1 (en) * 2020-04-01 2023-08-10 Janssen Biopharma, Inc. Nucleic acid polymers
US20250320493A1 (en) * 2021-07-23 2025-10-16 Biogen Ma Inc. Methods for reducing tau expression
AU2024313057A1 (en) * 2023-06-22 2025-10-30 Denali Therapeutics Inc. Compositions and methods for modulating tau expression
WO2025212467A1 (en) 2024-03-31 2025-10-09 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of microtubule associated protein tau (mapt), compositions thereof, and methods of use
WO2026025082A1 (en) * 2024-07-25 2026-01-29 Biogen Ma Inc. Methods for treating alzheimer's disease

Citations (7)

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JP2008504022A (ja) * 2004-06-21 2008-02-14 プロティオーム・サイエンシィズ・ピーエルシー スクリーニング方法
WO2013180038A1 (ja) * 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
WO2016108264A1 (ja) * 2014-12-29 2016-07-07 株式会社ボナック 核酸分子を安定に含有する組成物
JP2016528891A (ja) * 2013-07-19 2016-09-23 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タウ発現を調節するための組成物
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JP2018505676A (ja) * 2015-02-04 2018-03-01 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ タウアンチセンスオリゴマー及びその使用

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BRPI0008131B8 (pt) 1999-02-12 2021-05-25 Daiichi Sankyo Co Ltd composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno
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WO2013180038A1 (ja) * 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
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JP2018505676A (ja) * 2015-02-04 2018-03-01 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ タウアンチセンスオリゴマー及びその使用
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EP3765476A2 (en) 2021-01-20
WO2019175260A3 (en) 2019-11-14
IL277238A (en) 2020-10-29
CN111868068A (zh) 2020-10-30
US20240011029A1 (en) 2024-01-11
BR112020018365A2 (pt) 2020-12-29
US20210095284A1 (en) 2021-04-01
EP3765476B1 (en) 2025-11-26
MA52149A (fr) 2021-01-20
AU2019233612A1 (en) 2020-09-10
CA3091979A1 (en) 2019-09-19
KR20200131263A (ko) 2020-11-23
EA202092156A1 (ru) 2021-04-01
SG11202008895WA (en) 2020-10-29
EP3922637A1 (en) 2021-12-15
US11453881B2 (en) 2022-09-27
ES3063421T3 (en) 2026-04-16
WO2019175260A2 (en) 2019-09-19
MX2020009532A (es) 2020-10-05
EP3765476C0 (en) 2025-11-26

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