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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Japanese (ja)
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JP2017510271A (ja
JP2017510271A5 (enExample
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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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Publication of JP2017510271A publication Critical patent/JP2017510271A/ja
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Priority to JP2021056879A priority Critical patent/JP7194222B2/ja
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    • 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
    • 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
    • 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
    • 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
    • C12N2310/321—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
    • 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
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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
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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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom

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

Publication Number Publication Date
JP2017510271A JP2017510271A (ja) 2017-04-13
JP2017510271A5 JP2017510271A5 (enExample) 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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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

Country Status (25)

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US (4) US10308934B2 (enExample)
EP (2) EP3119888B1 (enExample)
JP (3) JP6902869B2 (enExample)
KR (1) KR102352589B1 (enExample)
CN (2) CN116970607A (enExample)
AU (2) AU2015231130C1 (enExample)
BR (1) BR112016020472B1 (enExample)
CA (1) CA2942340A1 (enExample)
CL (1) CL2016002346A1 (enExample)
CY (1) CY1124526T1 (enExample)
DK (1) DK3119888T3 (enExample)
ES (1) ES2890677T3 (enExample)
HR (1) HRP20211397T1 (enExample)
HU (1) HUE055877T2 (enExample)
IL (1) IL247600B (enExample)
LT (1) LT3119888T (enExample)
MX (1) MX389437B (enExample)
PL (1) PL3119888T3 (enExample)
PT (1) PT3119888T (enExample)
RS (1) RS62526B1 (enExample)
RU (2) RU2019130898A (enExample)
SG (2) SG11201607761UA (enExample)
SI (1) SI3119888T1 (enExample)
SM (1) SMT202100562T1 (enExample)
WO (1) WO2015143246A1 (enExample)

Families Citing this family (21)

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SMT202100562T1 (it) 2014-03-19 2021-11-12 Ionis Pharmaceuticals Inc Composizioni per modulare l'espressione di ataxina 2
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
EP4285912A3 (en) 2015-09-25 2024-07-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
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
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
WO2017117496A1 (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
KR20200075865A (ko) * 2017-10-23 2020-06-26 프리베일 테라퓨틱스, 인크. 신경변성 질환에 대한 유전자 요법
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
AU2019274461B2 (en) 2018-05-22 2025-07-31 Ionis Pharmaceuticals, Inc. Modulators of APOL1 expression
CN112912500A (zh) * 2018-06-05 2021-06-04 豪夫迈·罗氏有限公司 用于调节atxn2表达的寡核苷酸
SG11202100077PA (en) * 2018-07-25 2021-02-25 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn2 expression
EP3890752A1 (en) 2018-12-06 2021-10-13 Biogen MA Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
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
EP3947677A1 (en) * 2019-04-04 2022-02-09 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
EP4188390A4 (en) * 2020-07-29 2025-07-16 Alnylam Pharmaceuticals Inc ATXN2 RNAI COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING ATXN2-ASSOCIATED NEURODEGENERATIVE DISEASES
CA3260484A1 (en) * 2022-06-24 2023-12-28 Eli Lilly And Company RNA INTERFERENCE AGENTS TARGETTING ATXN2
WO2025067482A1 (zh) * 2023-09-28 2025-04-03 大睿生物 调控atxn2表达的dsrna分子
KR20250178496A (ko) 2024-06-19 2025-12-29 이양원 Pcr 장치

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HUE055877T2 (hu) 2021-12-28
CN116970607A (zh) 2023-10-31
AU2021221934A1 (en) 2021-09-23
SG11201607761UA (en) 2016-10-28
CA2942340A1 (en) 2015-09-24
CL2016002346A1 (es) 2017-06-09
AU2015231130B2 (en) 2021-06-10

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