JP6126009B2 - α−シヌクレイン発現の調節 - Google Patents

α−シヌクレイン発現の調節 Download PDF

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Publication number
JP6126009B2
JP6126009B2 JP2013540028A JP2013540028A JP6126009B2 JP 6126009 B2 JP6126009 B2 JP 6126009B2 JP 2013540028 A JP2013540028 A JP 2013540028A JP 2013540028 A JP2013540028 A JP 2013540028A JP 6126009 B2 JP6126009 B2 JP 6126009B2
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modified
compound
certain embodiments
synuclein
modified oligonucleotide
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JP2014501507A (ja
JP2014501507A5 (enExample
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ベネット，シー・フランク
フレアー，スーザン・エム
マラジョシュラ，ジョティ
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N2310/10—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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/323—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
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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
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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 ===---===
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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

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  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2013540028A 2010-11-17 2011-11-17 α−シヌクレイン発現の調節 Active JP6126009B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41484810P 2010-11-17 2010-11-17
US61/414,848 2010-11-17
PCT/US2011/061245 WO2012068405A2 (en) 2010-11-17 2011-11-17 Modulation of alpha synuclein expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017004099A Division JP2017079786A (ja) 2010-11-17 2017-01-13 α−シヌクレイン発現の調節

Publications (3)

Publication Number Publication Date
JP2014501507A JP2014501507A (ja) 2014-01-23
JP2014501507A5 JP2014501507A5 (enExample) 2016-07-07
JP6126009B2 true JP6126009B2 (ja) 2017-05-10

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JP2013540028A Active JP6126009B2 (ja) 2010-11-17 2011-11-17 α−シヌクレイン発現の調節
JP2017004099A Withdrawn JP2017079786A (ja) 2010-11-17 2017-01-13 α−シヌクレイン発現の調節

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JP2017004099A Withdrawn JP2017079786A (ja) 2010-11-17 2017-01-13 α−シヌクレイン発現の調節

Country Status (6)

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US (4) US9663783B2 (enExample)
EP (2) EP3521451A1 (enExample)
JP (2) JP6126009B2 (enExample)
AU (2) AU2011329777B2 (enExample)
CA (2) CA2817960C (enExample)
WO (1) WO2012068405A2 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11359197B2 (en) 2018-01-12 2022-06-14 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
US12241065B2 (en) 2018-01-12 2025-03-04 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof

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DK2560687T3 (en) * 2010-04-19 2017-09-18 Nlife Therapeutics S L Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
DK2864479T3 (en) 2012-06-25 2018-10-22 Ionis Pharmaceuticals Inc MODULATION OF UBE3A-ATS EXPRESSION
AU2013336581A1 (en) * 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
US10762374B2 (en) * 2014-12-30 2020-09-01 Blinkfire Analytics, Inc. High accuracy image identification system
JP6606557B2 (ja) * 2015-04-16 2019-11-13 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 Ｃ９ｏｒｆ７２発現を調節するための組成物
UA125963C2 (uk) 2015-11-12 2022-07-20 Ф. Хоффманн-Ля Рош Аг Олігонуклеотид для індукції батьківської експресії ube3a
WO2017087282A1 (en) 2015-11-18 2017-05-26 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
US11234995B2 (en) * 2016-01-07 2022-02-01 Osaka University α-synuclein expression inhibitor
US10815271B2 (en) * 2016-06-29 2020-10-27 The Regents Of The University Of California Structure-based peptide inhibitors of alpha-synuclein aggregation
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
WO2019009298A1 (ja) * 2017-07-05 2019-01-10 国立大学法人大阪大学 α－シヌクレイン発現抑制剤
EP3628737A4 (en) * 2017-07-05 2021-01-20 Osaka University ENA-ANTISENSE-OLIGONUCLEOTIDE TO INHIBIT ALPHA-SYNUCLEIN EXPRESSION
TWI809004B (zh) * 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
JP2021511027A (ja) * 2018-01-12 2021-05-06 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ アルファ−シヌクレインアンチセンスオリゴヌクレオチド及びその使用
PH12021550254A1 (en) * 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
CN115777021A (zh) * 2020-03-18 2023-03-10 马萨诸塞大学 用于snca调节的寡核苷酸
WO2022072447A1 (en) * 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
AU2022234522A1 (en) 2021-03-08 2023-10-26 Les Laboratoires Servier Antisense oligonucleotides for inhibiting alpha-synuclein expression
EP4359525A1 (en) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2023215863A2 (en) * 2022-05-06 2023-11-09 Ionis Pharmaceuticals, Inc. Rnai agents for modulating snca
AU2024220937A1 (en) * 2023-02-17 2025-10-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating alpha-synuclein expression
WO2025063239A1 (ja) * 2023-09-20 2025-03-27 第一三共株式会社 α－シヌクレインの発現を抑制するｓｉＲＮＡ

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