MX2015005352A - Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas. - Google Patents

Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas.

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Publication number
MX2015005352A
MX2015005352A MX2015005352A MX2015005352A MX2015005352A MX 2015005352 A MX2015005352 A MX 2015005352A MX 2015005352 A MX2015005352 A MX 2015005352A MX 2015005352 A MX2015005352 A MX 2015005352A MX 2015005352 A MX2015005352 A MX 2015005352A
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MX
Mexico
Prior art keywords
treatment
transporter
compositions
nucleic acid
methods
Prior art date
Application number
MX2015005352A
Other languages
English (en)
Inventor
Maria Del Carmen Carmona Orozco
Andrés Pablo Montefeltro
Gabriel G Alvarado
Analia Bortolozzi
Francesc Artigas Pérez
Miquel Vila Bover
Original Assignee
Nlife Therapeutics S L
Priority date (The priority date 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 date listed.)
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Publication date
Application filed by Nlife Therapeutics S L filed Critical Nlife Therapeutics S L
Publication of MX2015005352A publication Critical patent/MX2015005352A/es

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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 N.A.

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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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (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)

Abstract

La invención proporciona un conjugado que comprende (i) un agente de selectividad que se une específicamente a uno o más transportadores de neurotransmisores seleccionados del grupo que consiste en un transportador de dopamina (DAT), un transportador de serotonina (SERT) o un transportador de norepinefrina (NET) y (ii) un ácido nucleico capaz de unirse específicamente a una molécula diana que se expresa en la misma célula que el transportador del neurotransmisor en donde dicha molécula diana es a-sinucleína o el ARNm que codifica a a-sinucleína. Los conjugados de la presente invención son útiles para la administración del ácido nucleico a una célula de interés y, por tanto, para el tratamiento de enfermedades que requieren el descenso de la proteína codificada por el ácido nucleico diana así como para la administración de agentes de diagnóstico por imagen a las células para fines diagnósticos.
MX2015005352A 2012-10-26 2013-10-25 Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas. MX2015005352A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261719284P 2012-10-26 2012-10-26
EP12382414 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
MX2015005352A true MX2015005352A (es) 2015-10-29

Family

ID=47148690

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015005352A MX2015005352A (es) 2012-10-26 2013-10-25 Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas.

Country Status (10)

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

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WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP3208338B1 (en) 2011-08-04 2019-04-10 Yeda Research and Development Co., Ltd. Micro-rna mir-15 and compositions comprising same for the treatment of corticotropin-releasing hormone- associated medical conditions
JP6386461B2 (ja) 2012-10-26 2018-09-05 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． 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
CA2936158C (en) 2014-02-05 2023-06-13 Yeda Research And Development Co. Ltd. Use of mir-135 or precursor thereof for the treatment and diagnosis of a bipolar disease
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
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
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Also Published As

Publication number Publication date
AU2013336581A1 (en) 2015-06-11
US20140120158A1 (en) 2014-05-01
US20160083726A1 (en) 2016-03-24
CN104884094A (zh) 2015-09-02
JP6386461B2 (ja) 2018-09-05
HK1214179A1 (zh) 2016-07-22
EP2911695C0 (en) 2023-11-29
US10125363B2 (en) 2018-11-13
EP2911695A1 (en) 2015-09-02
ES2967530T3 (es) 2024-04-30
EP2911695B1 (en) 2023-11-29
JP2015536927A (ja) 2015-12-24
WO2014064257A1 (en) 2014-05-01
US9084825B2 (en) 2015-07-21
CA2890112A1 (en) 2014-05-01

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