JP2018517704A - 筋萎縮性側索硬化症（ＡＬＳ）を処置するためのｍｉＲ−１５５阻害剤 - Google Patents

筋萎縮性側索硬化症（ＡＬＳ）を処置するためのｍｉＲ−１５５阻害剤 Download PDF

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JP2018517704A
JP2018517704A JP2017562664A JP2017562664A JP2018517704A JP 2018517704 A JP2018517704 A JP 2018517704A JP 2017562664 A JP2017562664 A JP 2017562664A JP 2017562664 A JP2017562664 A JP 2017562664A JP 2018517704 A JP2018517704 A JP 2018517704A
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oligonucleotide inhibitor
mir
cells
nucleotide
nucleotides
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JP2018517704A5 (enExample
Inventor
エイミー ジャクソン，
エイミー ジャクソン，
クリスティーナ ダルビー，
クリスティーナ ダルビー，
ハワード エル． ウェイナー，
ハワード エル． ウェイナー，
オレグ ブトフスキー，
オレグ ブトフスキー，
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Brigham and Womens Hospital Inc
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Brigham and Womens Hospital Inc
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Publication of JP2018517704A publication Critical patent/JP2018517704A/ja
Publication of JP2018517704A5 publication Critical patent/JP2018517704A5/ja
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    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2017562664A 2015-06-05 2016-06-03 筋萎縮性側索硬化症（ＡＬＳ）を処置するためのｍｉＲ−１５５阻害剤 Pending JP2018517704A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562171743P 2015-06-05 2015-06-05
US62/171,743 2015-06-05
PCT/US2016/035794 WO2016196978A1 (en) 2015-06-05 2016-06-03 Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)

Publications (2)

Publication Number Publication Date
JP2018517704A true JP2018517704A (ja) 2018-07-05
JP2018517704A5 JP2018517704A5 (enExample) 2019-07-04

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JP2017562664A Pending JP2018517704A (ja) 2015-06-05 2016-06-03 筋萎縮性側索硬化症（ＡＬＳ）を処置するためのｍｉＲ−１５５阻害剤

Country Status (6)

Country Link
US (1) US20180161357A1 (enExample)
EP (1) EP3303589A4 (enExample)
JP (1) JP2018517704A (enExample)
CN (1) CN107922947A (enExample)
CA (1) CA2986913A1 (enExample)
WO (1) WO2016196978A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20140074997A (ko) 2011-10-11 2014-06-18 더 브리검 앤드 우먼즈 하스피털, 인크. 신경병성 질환에서 마이크로rna
KR20190118688A (ko) 2015-06-05 2019-10-18 미라젠 세러퓨틱스 인코포레이티드 피부 t 세포 림프종(ctcl) 치료용 mir-155 억제제
US11957713B2 (en) 2016-10-14 2024-04-16 Children's Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
CA3050690A1 (en) 2017-01-17 2018-07-26 Dana-Farber Cancer Institute, Inc. Compositions and methods for diagnosing and treating peroxisomal diseases
WO2018136435A1 (en) 2017-01-17 2018-07-26 Children's Medical Center Corporation Compositions and methods for treating lysosomal storage diseases and disorders
WO2019195304A1 (en) 2018-04-03 2019-10-10 Academia Sinica Mir-17~92 as therapeutic or diagnostic target of motor neuron (mn) degeneration diseases
CN112585280A (zh) 2018-08-23 2021-03-30 罗氏创新中心哥本哈根有限公司 微小rna-134生物标志物
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis
CN114272378B (zh) * 2020-09-27 2023-06-23 四川大学华西医院 一种使cttnbp2nl功能缺失的试剂在制备治疗疾病的药物中的用途
CN112587662A (zh) * 2020-12-22 2021-04-02 上海市徐汇区中心医院 一种miR-155/PEA15信号通路抑制剂在病理性心脏重构药物中的应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009532044A (ja) * 2006-04-03 2009-09-10 サンタリス ファーマ アー／エス ａｎｔｉｍｉＲＮＡアンチセンスオリゴヌクレオチドを含む医薬組成物
JP2010539959A (ja) * 2007-10-04 2010-12-24 サンタリス ファーマ アー／エス マイクロｍｉｒ
JP2014534810A (ja) * 2011-10-11 2014-12-25 ザ ブリガム アンド ウーメンズ ホスピタル，インク 神経変性障害におけるマイクロｒｎａ

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101437942A (zh) * 2006-04-03 2009-05-20 桑塔里斯制药公司 包含抗微小rna反义寡核苷酸的药物组合物
EP2268811A1 (en) * 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
ES2581178T3 (es) * 2009-07-29 2016-09-01 Pharnext Nuevas herramientas de diagnóstico para enfermedad de Alzheimer
WO2013134403A1 (en) * 2012-03-06 2013-09-12 The Washington University Method of treating neurodegenerative diseases with microrna regulators

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009532044A (ja) * 2006-04-03 2009-09-10 サンタリス ファーマ アー／エス ａｎｔｉｍｉＲＮＡアンチセンスオリゴヌクレオチドを含む医薬組成物
JP2010539959A (ja) * 2007-10-04 2010-12-24 サンタリス ファーマ アー／エス マイクロｍｉｒ
JP2014534810A (ja) * 2011-10-11 2014-12-25 ザ ブリガム アンド ウーメンズ ホスピタル，インク 神経変性障害におけるマイクロｒｎａ

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Publication number Publication date
CA2986913A1 (en) 2016-12-08
US20180161357A1 (en) 2018-06-14
CN107922947A (zh) 2018-04-17
WO2016196978A1 (en) 2016-12-08
EP3303589A1 (en) 2018-04-11
EP3303589A4 (en) 2019-01-02

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