CN104884094A - 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 - Google Patents

通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 Download PDF

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Publication number
CN104884094A
CN104884094A CN201380068146.3A CN201380068146A CN104884094A CN 104884094 A CN104884094 A CN 104884094A CN 201380068146 A CN201380068146 A CN 201380068146A CN 104884094 A CN104884094 A CN 104884094A
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conjugate
nucleic acid
synuclein
seq
moiety
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Chinese (zh)
Inventor
M·D·C·卡尔莫纳奥罗斯科
A·P·蒙泰费尔特罗
G·G·阿尔瓦拉多
M·维拉博韦尔
A·博尔托洛齐
F·阿蒂加斯佩雷斯
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Nlife Therapeutics SL
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Nlife Therapeutics SL
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Publication of CN104884094A publication Critical patent/CN104884094A/zh
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    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
CN201380068146.3A 2012-10-26 2013-10-25 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 Pending CN104884094A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261719284P 2012-10-26 2012-10-26
EP12382414 2012-10-26
US61/719,284 2012-10-26
EP12382414.6 2012-10-26
PCT/EP2013/072410 WO2014064257A1 (en) 2012-10-26 2013-10-25 Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

Publications (1)

Publication Number Publication Date
CN104884094A true CN104884094A (zh) 2015-09-02

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CN201380068146.3A Pending CN104884094A (zh) 2012-10-26 2013-10-25 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法

Country Status (10)

Country Link
US (2) US9084825B2 (enExample)
EP (1) EP2911695B1 (enExample)
JP (1) JP6386461B2 (enExample)
CN (1) CN104884094A (enExample)
AU (1) AU2013336581A1 (enExample)
CA (1) CA2890112A1 (enExample)
ES (1) ES2967530T3 (enExample)
HK (1) HK1214179A1 (enExample)
MX (1) MX2015005352A (enExample)
WO (1) WO2014064257A1 (enExample)

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CN113286600A (zh) * 2018-11-30 2021-08-20 协和麒麟株式会社 核酸复合物
CN115151822A (zh) * 2019-11-11 2022-10-04 牛津大学创新有限公司 用于预测和鉴定帕金森病的生物标志物
CN115168823A (zh) * 2022-08-05 2022-10-11 聊城大学 基于组平均的多任务功能性近红外光谱信号的身份识别

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WO2014064257A1 (en) 2012-10-26 2014-05-01 Nlife Therapeutics, S.L. Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
US10125365B2 (en) 2014-02-05 2018-11-13 Yeda Research And Development Co. Ltd. Micro-RNAs and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
US11426366B2 (en) 2015-05-15 2022-08-30 Arizona Board Of Regents On Behalf Of The Universsity Of Arizona Compositions and methods for treating motor disorders
CN117965536A (zh) 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
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US20200172903A1 (en) * 2017-07-05 2020-06-04 Osaka University ENA ANTISENSE OLIGONUCLEOTIDE FOR INHIBITION OF alpha-SYNUCLEIN EXPRESSION
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
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EP3737759A1 (en) 2018-01-12 2020-11-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
MY204234A (en) 2018-08-27 2024-08-16 Regeneron Pharma Use of raman spectroscopy in downstream purification
US20220096665A1 (en) * 2018-11-21 2022-03-31 University Of Georgia Research Foundation, Inc. Bimodal nanoparticle conjugates for non-invasive central nervous system tissue imaging
US12478684B2 (en) 2019-04-11 2025-11-25 Northeastern University Oligonucleotide-polymer miktoarm conjugates and methods of use
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof
AU2025205167A1 (en) * 2024-07-09 2026-01-29 China Medical University Targeting vehicles, compositions and uses thereof

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113286600A (zh) * 2018-11-30 2021-08-20 协和麒麟株式会社 核酸复合物
CN115151822A (zh) * 2019-11-11 2022-10-04 牛津大学创新有限公司 用于预测和鉴定帕金森病的生物标志物
CN115168823A (zh) * 2022-08-05 2022-10-11 聊城大学 基于组平均的多任务功能性近红外光谱信号的身份识别

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Publication number Publication date
CA2890112A1 (en) 2014-05-01
EP2911695A1 (en) 2015-09-02
US9084825B2 (en) 2015-07-21
AU2013336581A1 (en) 2015-06-11
EP2911695C0 (en) 2023-11-29
JP2015536927A (ja) 2015-12-24
US20140120158A1 (en) 2014-05-01
WO2014064257A1 (en) 2014-05-01
HK1214179A1 (zh) 2016-07-22
EP2911695B1 (en) 2023-11-29
ES2967530T3 (es) 2024-04-30
MX2015005352A (es) 2015-10-29
US10125363B2 (en) 2018-11-13
US20160083726A1 (en) 2016-03-24
JP6386461B2 (ja) 2018-09-05

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