AU2013336581A1 - Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types - Google Patents

Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types Download PDF

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Publication number
AU2013336581A1
AU2013336581A1 AU2013336581A AU2013336581A AU2013336581A1 AU 2013336581 A1 AU2013336581 A1 AU 2013336581A1 AU 2013336581 A AU2013336581 A AU 2013336581A AU 2013336581 A AU2013336581 A AU 2013336581A AU 2013336581 A1 AU2013336581 A1 AU 2013336581A1
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Australia
Prior art keywords
conjugate
group
nucleic acid
moiety
conjugate according
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
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AU2013336581A
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English (en)
Inventor
Gabriel G. Alvarado
Francesc Artigas Perez
Analia BORTOLOZZI
Maria del Carmen CARMONA OROZCO
Andres Pablo Montefeltro
Miquel Vila Bover
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Nlife Therapeutics SL
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Nlife Therapeutics SL
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Publication date
Application filed by Nlife Therapeutics SL filed Critical Nlife Therapeutics SL
Publication of AU2013336581A1 publication Critical patent/AU2013336581A1/en
Abandoned legal-status Critical Current

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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, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • 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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (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)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2013336581A 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 Abandoned AU2013336581A1 (en)

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
AU2013336581A1 true AU2013336581A1 (en) 2015-06-11

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ID=47148690

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2013336581A Abandoned AU2013336581A1 (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

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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CA2890112A1 (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
CN106715695B (zh) 2014-02-05 2020-07-31 耶达研究及发展有限公司 用于治疗和诊断的微rna和包含所述微rna的组合物
WO2016186968A1 (en) 2015-05-15 2016-11-24 The Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for treating motor disorders
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10733023B1 (en) * 2015-08-06 2020-08-04 D2Iq, Inc. Oversubscription scheduling
JP6569920B2 (ja) * 2016-01-07 2019-09-04 国立大学法人大阪大学 α−シヌクレイン発現抑制剤
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
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
WO2019009299A1 (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するＥＮＡアンチセンスオリゴヌクレオチド
KR101890925B1 (ko) 2017-08-08 2018-08-24 주식회사 이레산업 캠핑용 그릴 세트
WO2019071028A1 (en) * 2017-10-04 2019-04-11 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
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EP3952919A2 (en) 2019-04-11 2022-02-16 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
JP7671511B2 (ja) * 2019-11-11 2025-05-02 オックスフォード ユニヴァーシティ イノヴェーション リミテッド パーキンソン病の予測及び同定のためのバイオマーカー
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
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof

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Also Published As

Publication number Publication date
US20140120158A1 (en) 2014-05-01
HK1214179A1 (zh) 2016-07-22
EP2911695A1 (en) 2015-09-02
JP6386461B2 (ja) 2018-09-05
MX2015005352A (es) 2015-10-29
CA2890112A1 (en) 2014-05-01
JP2015536927A (ja) 2015-12-24
EP2911695C0 (en) 2023-11-29
WO2014064257A1 (en) 2014-05-01
ES2967530T3 (es) 2024-04-30
US9084825B2 (en) 2015-07-21
EP2911695B1 (en) 2023-11-29
US20160083726A1 (en) 2016-03-24
CN104884094A (zh) 2015-09-02
US10125363B2 (en) 2018-11-13

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