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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Expired - Fee Related
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Japanese (ja)
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JP2017510271A (ja
JP2017510271A5 (enExample
Inventor
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
Publication of JP2017510271A5 publication Critical patent/JP2017510271A5/ja
Priority to JP2021056879A priority Critical patent/JP7194222B2/ja
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Priority to JP2022196985A priority patent/JP2023027236A/ja
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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
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    • 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
    • C12N2310/321—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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    • C12N2310/00—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
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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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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
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  • 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)
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  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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 Expired - Fee Related 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)

Application Number Title Priority Date Filing Date
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 Expired - Fee Related 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) RU2702838C2 (enExample)
SG (2) SG10201808063PA (enExample)
SI (1) SI3119888T1 (enExample)
SM (1) SMT202100562T1 (enExample)
WO (1) WO2015143246A1 (enExample)

Families Citing this family (21)

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WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
CN116970607A (zh) 2014-03-19 2023-10-31 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
US10533175B2 (en) 2015-09-25 2020-01-14 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
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
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
SG11202010215TA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
IL316397A (en) 2018-05-22 2024-12-01 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
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
TWI906204B (zh) * 2018-12-14 2025-12-01 美商百健Ｍａ公司 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法
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
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
EP3947677A1 (en) * 2019-04-04 2022-02-09 F. Hoffmann-La Roche AG 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
JP2025524468A (ja) * 2022-06-24 2025-07-30 イーライ リリー アンド カンパニー Ａｔｘｎ２ ｒｎａ干渉剤
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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IL247600B (en) 2022-06-01
AU2021221934A1 (en) 2021-09-23
WO2015143246A1 (en) 2015-09-24
HUE055877T2 (hu) 2021-12-28
AU2015231130A1 (en) 2016-09-22
NZ723832A (en) 2024-07-26
KR102352589B1 (ko) 2022-01-18
CY1124526T1 (el) 2022-07-22
EP3978610A3 (en) 2022-08-24
ES2890677T3 (es) 2022-01-21
LT3119888T (lt) 2021-10-11

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