JP6902869B2 - アタキシン２の発現を調節するための組成物 - Google Patents

アタキシン２の発現を調節するための組成物 Download PDF

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Publication number
JP6902869B2
JP6902869B2 JP2016558122A JP2016558122A JP6902869B2 JP 6902869 B2 JP6902869 B2 JP 6902869B2 JP 2016558122 A JP2016558122 A JP 2016558122A JP 2016558122 A JP2016558122 A JP 2016558122A JP 6902869 B2 JP6902869 B2 JP 6902869B2
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modified
nucleoside
ataxin
certain embodiments
nucleic acid
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JP2017510271A (ja
JP2017510271A5 (cg-RX-API-DMAC7.html
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フレアー，スーザン・エム
ハング，ジーン
ベネット，シー・フランク
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • A61P25/14—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
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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
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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
    • C12N2310/323—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
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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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    • C12N2310/30—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

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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Plant Pathology (AREA)
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  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2016558122A 2014-03-19 2015-03-19 アタキシン２の発現を調節するための組成物 Active JP6902869B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021056879A JP7194222B2 (ja) 2014-03-19 2021-03-30 アタキシン２の発現を調節するための組成物
JP2022196985A JP2023027236A (ja) 2014-03-19 2022-12-09 アタキシン２の発現を調節するための組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461955705P 2014-03-19 2014-03-19
US61/955,705 2014-03-19
US201461982131P 2014-04-21 2014-04-21
US61/982,131 2014-04-21
PCT/US2015/021608 WO2015143246A1 (en) 2014-03-19 2015-03-19 Compositions for modulating ataxin 2 expression

Related Child Applications (1)

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JP2021056879A Division JP7194222B2 (ja) 2014-03-19 2021-03-30 アタキシン２の発現を調節するための組成物

Publications (3)

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JP2017510271A JP2017510271A (ja) 2017-04-13
JP2017510271A5 JP2017510271A5 (cg-RX-API-DMAC7.html) 2018-04-26
JP6902869B2 true JP6902869B2 (ja) 2021-07-14

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JP2016558122A Active JP6902869B2 (ja) 2014-03-19 2015-03-19 アタキシン２の発現を調節するための組成物
JP2021056879A Active JP7194222B2 (ja) 2014-03-19 2021-03-30 アタキシン２の発現を調節するための組成物
JP2022196985A Pending JP2023027236A (ja) 2014-03-19 2022-12-09 アタキシン２の発現を調節するための組成物

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JP2022196985A Pending JP2023027236A (ja) 2014-03-19 2022-12-09 アタキシン２の発現を調節するための組成物

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US (4) US10308934B2 (cg-RX-API-DMAC7.html)
EP (2) EP3978610A3 (cg-RX-API-DMAC7.html)
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CN (2) CN116970607A (cg-RX-API-DMAC7.html)
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US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
HRP20211397T1 (hr) 2014-03-19 2021-12-10 Ionis Pharmaceuticals, Inc. Pripravci za modulaciju ekspresije ataksina 2
EP3353303B1 (en) 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
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
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
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US20210261981A1 (en) * 2017-10-23 2021-08-26 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
TW202003541A (zh) 2018-05-09 2020-01-16 美商Ionis製藥公司 用於減少ａｔｘｎ３表現之化合物及方法
MX2020012497A (es) 2018-05-22 2021-02-15 Ionis Pharmaceuticals Inc Moduladores de la expresion de apol1.
WO2019233922A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
CA3106986A1 (en) * 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
KR20210102294A (ko) 2018-12-06 2021-08-19 바이오젠 엠에이 인코포레이티드 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질
UY38506A (es) * 2018-12-14 2020-06-30 Biogen Ma Inc Composiciones y metodos para tratar y prevenir la esclerosis lateral amiotrofica
CN109529018B (zh) * 2019-01-10 2021-11-30 南方医科大学南方医院 Atxn2l用于制备逆转胃癌奥沙利铂耐药的药物的用途
JP7499267B2 (ja) * 2019-04-04 2024-06-13 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を調節するためのオリゴヌクレオチド
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
WO2022026531A1 (en) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
AU2023287741A1 (en) * 2022-06-24 2025-01-09 Eli Lilly And Company Atxn2 rna interference agents
WO2025067482A1 (zh) * 2023-09-28 2025-04-03 大睿生物 调控atxn2表达的dsrna分子

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NZ723832A (en) 2024-07-26
AU2021221934B2 (en) 2024-07-18
US20220195430A1 (en) 2022-06-23
HK1232251A1 (en) 2018-01-05
EP3978610A3 (en) 2022-08-24
AU2015231130B2 (en) 2021-06-10
LT3119888T (lt) 2021-10-11
CN106103717A (zh) 2016-11-09
JP2021104035A (ja) 2021-07-26
SG11201607761UA (en) 2016-10-28
AU2015231130C1 (en) 2021-12-23
EP3119888A4 (en) 2018-02-28
RU2702838C2 (ru) 2019-10-11
US20200087661A1 (en) 2020-03-19
CY1124526T1 (el) 2022-07-22
PT3119888T (pt) 2021-10-25
BR112016020472B1 (pt) 2023-03-07
US20170175114A1 (en) 2017-06-22
IL247600B (en) 2022-06-01
RU2016140897A (ru) 2018-04-19
US11111494B2 (en) 2021-09-07
RU2016140897A3 (cg-RX-API-DMAC7.html) 2018-08-20
IL247600A0 (en) 2016-11-30
EP3119888A1 (en) 2017-01-25
HRP20211397T1 (hr) 2021-12-10
US20240309374A1 (en) 2024-09-19
SG10201808063PA (en) 2018-10-30
EP3978610A2 (en) 2022-04-06
KR20160134710A (ko) 2016-11-23
SI3119888T1 (sl) 2021-11-30
MX2016012123A (es) 2017-01-19
ES2890677T3 (es) 2022-01-21
BR112016020472A2 (pt) 2017-10-10
DK3119888T3 (da) 2021-09-06

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