JP6386461B2 - 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 - Google Patents

特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 Download PDF

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JP6386461B2
JP6386461B2 JP2015538467A JP2015538467A JP6386461B2 JP 6386461 B2 JP6386461 B2 JP 6386461B2 JP 2015538467 A JP2015538467 A JP 2015538467A JP 2015538467 A JP2015538467 A JP 2015538467A JP 6386461 B2 JP6386461 B2 JP 6386461B2
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JP2015536927A (ja
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マリア、デル、カルメン、カルモナ、オロスコ
アンドレス、パブロ、モンテフェルトロ
ガブリエル、ジー.アルバラド
ミケル、ビラ、ボベル
アナリア、ボルトロッシ
フランセスク、アルティガス、ペレス
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Nlife Therapeutics SL
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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)
JP2015538467A 2012-10-26 2013-10-25 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 Active JP6386461B2 (ja)

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 (3)

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JP2015536927A JP2015536927A (ja) 2015-12-24
JP2015536927A5 JP2015536927A5 (enExample) 2016-12-15
JP6386461B2 true JP6386461B2 (ja) 2018-09-05

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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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US12398176B2 (en) 2018-08-27 2025-08-26 Regeneron Pharmaceuticals, Inc. Use of Raman spectroscopy in downstream purification

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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
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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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US11814625B2 (en) 2017-10-23 2023-11-14 Carleton University Aptamers as a therapeutic tool to prevent protein aggregation in neurodegenerative disease
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
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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
EP4058807A1 (en) * 2019-11-11 2022-09-21 Oxford University Innovation Limited Biomarkers for the prediction and identification of parkinson's disease
CN115168823B (zh) * 2022-08-05 2025-07-18 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 Priority date Publication date Assignee Title
US12398176B2 (en) 2018-08-27 2025-08-26 Regeneron Pharmaceuticals, Inc. Use of Raman spectroscopy in downstream purification

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CA2890112A1 (en) 2014-05-01
EP2911695A1 (en) 2015-09-02
US9084825B2 (en) 2015-07-21
CN104884094A (zh) 2015-09-02
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

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