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 (https=
JPWO2019175260A5 (https=
Inventor
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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Publication of JP2021515784A publication Critical patent/JP2021515784A/ja
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

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  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • 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)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (3)

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JP2021515784A true JP2021515784A (ja) 2021-06-24
JP2021515784A5 JP2021515784A5 (https=) 2022-03-14
JPWO2019175260A5 JPWO2019175260A5 (https=) 2022-03-14

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US (2) US11453881B2 (https=)
EP (2) EP3765476B1 (https=)
JP (1) JP2021515784A (https=)
KR (1) KR20200131263A (https=)
CN (1) CN111868068A (https=)
AU (1) AU2019233612A1 (https=)
BR (1) BR112020018365A2 (https=)
CA (1) CA3091979A1 (https=)
EA (1) EA202092156A1 (https=)
ES (1) ES3063421T3 (https=)
IL (1) IL277238A (https=)
MA (1) MA52149A (https=)
MX (1) MX2020009532A (https=)
SG (1) SG11202008895WA (https=)
WO (1) WO2019175260A2 (https=)

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AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2021198958A1 (en) * 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
PE20240648A1 (es) * 2021-07-23 2024-04-04 Biogen Ma Inc Metodos para reducir la expresion de tau
AU2024313057A1 (en) * 2023-06-22 2025-10-30 Denali Therapeutics Inc. Compositions and methods for modulating tau expression
US12551569B2 (en) 2024-03-31 2026-02-17 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

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WO2001018015A1 (en) * 1999-09-10 2001-03-15 Geron Corporation Oligonucleotide n3'→p5' thiophosphoramidates: their synthesis and use
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
WO2017109679A1 (en) * 2015-12-21 2017-06-29 Novartis Ag Compositions and methods for decreasing tau expression
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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US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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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
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
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
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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WO2019175260A3 (en) 2019-11-14
KR20200131263A (ko) 2020-11-23
EA202092156A1 (ru) 2021-04-01
US20210095284A1 (en) 2021-04-01
EP3765476A2 (en) 2021-01-20
CA3091979A1 (en) 2019-09-19
EP3765476B1 (en) 2025-11-26
EP3922637A1 (en) 2021-12-15
MX2020009532A (es) 2020-10-05
MA52149A (fr) 2021-01-20
EP3765476C0 (en) 2025-11-26
ES3063421T3 (en) 2026-04-16
BR112020018365A2 (pt) 2020-12-29
SG11202008895WA (en) 2020-10-29
WO2019175260A2 (en) 2019-09-19
AU2019233612A1 (en) 2020-09-10
IL277238A (en) 2020-10-29
CN111868068A (zh) 2020-10-30
US20240011029A1 (en) 2024-01-11
US11453881B2 (en) 2022-09-27

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