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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JP2014501507A5 (https=
JP2014501507A (ja
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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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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
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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)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (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 (https=) 2016-07-07
JP6126009B2 true JP6126009B2 (ja) 2017-05-10

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ID=46084657

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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 (https=)
EP (2) EP2640853B1 (https=)
JP (2) JP6126009B2 (https=)
AU (2) AU2011329777B2 (https=)
CA (2) CA3077910A1 (https=)
WO (1) WO2012068405A2 (https=)

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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RU2664452C2 (ru) * 2010-04-19 2018-08-17 Нлифе Терапеутикс, С.Л. Конъюгат, медицинское средство и способы лечения и/или профилактики депрессии и болезни, связанной с отложением телец Леви
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
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
RS60230B1 (sr) * 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
HUE067905T2 (hu) 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
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
US9840710B2 (en) 2015-11-18 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
JP6569920B2 (ja) * 2016-01-07 2019-09-04 国立大学法人大阪大学 α−シヌクレイン発現抑制剤
CA3029676A1 (en) * 2016-06-29 2018-01-04 The Regents Of The University Of California Structure-based peptide inhibitors of alpha-synuclein aggregation
EP4035659A1 (en) 2016-11-29 2022-08-03 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
WO2019009299A1 (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するＥＮＡアンチセンスオリゴヌクレオチド
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
AU2019207859A1 (en) * 2018-01-12 2020-07-02 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
EP3830265A1 (en) * 2018-08-03 2021-06-09 Genzyme Corporation Variant rnai against alpha-synuclein
MX2022011536A (es) * 2020-03-18 2023-01-04 Univ Massachusetts Oligonucleotidos para la modulacion de snca.
AU2021353935A1 (en) * 2020-10-01 2023-05-25 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
PE20240696A1 (es) 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
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
WO2024173759A2 (en) * 2023-02-17 2024-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating alpha-synuclein expression
TW202528540A (zh) * 2023-09-20 2025-07-16 日商第一三共股份有限公司 抑制α-突觸核蛋白之表現的siRNA

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