JP6622214B2 - Ｓｏｄ−１発現を調節するための組成物 - Google Patents

Ｓｏｄ−１発現を調節するための組成物 Download PDF

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JP6622214B2
JP6622214B2 JP2016560543A JP2016560543A JP6622214B2 JP 6622214 B2 JP6622214 B2 JP 6622214B2 JP 2016560543 A JP2016560543 A JP 2016560543A JP 2016560543 A JP2016560543 A JP 2016560543A JP 6622214 B2 JP6622214 B2 JP 6622214B2
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sod
modified
sugar
antisense compound
isis
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JP2017513469A5 (enExample
JP2017513469A (ja
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スウェイジ，エリック・イー
コール，トレーシー
コルダシェヴィッチ，ホリー
フレアー，スーザン・エム
コンドン，トーマス・ピー
ワンツェヴィッチ，エドワード
ロックハート，トリシャ
ヴィカーズ，ティモシー
シング，プリヤム
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Biogen MA Inc
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Biogen MA Inc
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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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61P25/28—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
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    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
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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
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    • C12N2310/31—Chemical structure of the backbone
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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
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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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  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2016560543A 2014-04-01 2015-04-01 Ｓｏｄ−１発現を調節するための組成物 Active JP6622214B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461973803P 2014-04-01 2014-04-01
US61/973,803 2014-04-01
PCT/US2015/023934 WO2015153800A2 (en) 2014-04-01 2015-04-01 Compositions for modulating sod-1 expression

Related Child Applications (1)

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JP2019141933A Division JP2019201664A (ja) 2014-04-01 2019-08-01 Ｓｏｄ−１発現を調節するための組成物

Publications (3)

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JP2017513469A JP2017513469A (ja) 2017-06-01
JP2017513469A5 JP2017513469A5 (enExample) 2018-06-14
JP6622214B2 true JP6622214B2 (ja) 2019-12-18

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JP2016560543A Active JP6622214B2 (ja) 2014-04-01 2015-04-01 Ｓｏｄ−１発現を調節するための組成物
JP2019141933A Withdrawn JP2019201664A (ja) 2014-04-01 2019-08-01 Ｓｏｄ−１発現を調節するための組成物
JP2021014429A Active JP7198298B2 (ja) 2014-04-01 2021-02-01 Ｓｏｄ－１発現を調節するための組成物
JP2022200959A Withdrawn JP2023027299A (ja) 2014-04-01 2022-12-16 Ｓｏｄ－１発現を調節するための組成物
JP2024085318A Pending JP2024105714A (ja) 2014-04-01 2024-05-27 Ｓｏｄ－１発現を調節するための組成物

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JP2021014429A Active JP7198298B2 (ja) 2014-04-01 2021-02-01 Ｓｏｄ－１発現を調節するための組成物
JP2022200959A Withdrawn JP2023027299A (ja) 2014-04-01 2022-12-16 Ｓｏｄ－１発現を調節するための組成物
JP2024085318A Pending JP2024105714A (ja) 2014-04-01 2024-05-27 Ｓｏｄ－１発現を調節するための組成物

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US (3) US10385341B2 (enExample)
EP (3) EP3757214B1 (enExample)
JP (5) JP6622214B2 (enExample)
KR (6) KR20210097225A (enExample)
CN (2) CN111440798A (enExample)
AU (4) AU2015240761B2 (enExample)
BR (1) BR112016021848B1 (enExample)
CA (1) CA2942394A1 (enExample)
CL (3) CL2016002509A1 (enExample)
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FI (1) FIC20240035I1 (enExample)
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PL (2) PL3757214T3 (enExample)
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RS (2) RS60707B1 (enExample)
RU (1) RU2704619C2 (enExample)
SG (3) SG10201910844SA (enExample)
SI (2) SI3126499T1 (enExample)
SM (2) SMT202200479T1 (enExample)
WO (1) WO2015153800A2 (enExample)

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WO2018204786A1 (en) * 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
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EP3743728A1 (en) 2018-01-25 2020-12-02 Biogen MA Inc. Methods of treating spinal muscular atrophy
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
SG11202103794QA (en) * 2018-11-21 2021-05-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing prion expression
CN113365640A (zh) * 2018-12-06 2021-09-07 比奥根Ma公司 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白
UY38506A (es) * 2018-12-14 2020-06-30 Biogen Ma Inc Composiciones y metodos para tratar y prevenir la esclerosis lateral amiotrofica
EP3946375A1 (en) * 2019-03-26 2022-02-09 Rutgers, the State University of New Jersey Compositions and methods for treating neurodegenerative disorders
AU2020264807A1 (en) * 2019-05-01 2021-11-18 Perron Institute for Neurological and Translational Science Limited Treatment for SOD1 associated disease
US11629347B2 (en) * 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
AU2020268415A1 (en) 2019-05-08 2021-12-23 Biogen Ma Inc. Convergent liquid phase syntheses of oligonucleotides
EP4556565A2 (en) * 2019-05-28 2025-05-21 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fus expression
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US20240182903A1 (en) 2021-03-31 2024-06-06 Biogen Ma Inc. Treatment of amyotrophic lateral sclerosis
TW202304473A (zh) 2021-04-01 2023-02-01 美商百健Ｍａ公司 向中樞神經系統之核酸遞送
EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
CN114369130B (zh) * 2021-12-28 2023-10-03 杭州天龙药业有限公司 修饰的硫代寡核苷酸及其应用
JP2025522433A (ja) * 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
KR20240038469A (ko) 2022-09-16 2024-03-25 김정훈 사용자 콘텐츠 기반 홍보 서비스 제공 장치 및 방법
CN120202298A (zh) * 2023-08-29 2025-06-24 石药集团中奇制药技术（石家庄）有限公司 一种抑制sod1基因表达的dsRNA分子及其应用
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PT3757214T (pt) 2022-08-26
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IL247714A0 (en) 2016-11-30
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JP7198298B2 (ja) 2022-12-28
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AU2019268063A1 (en) 2019-12-12
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