WO2007107789A3 - Treatment of cns conditions - Google Patents

Treatment of cns conditions Download PDF

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Publication number
WO2007107789A3
WO2007107789A3 PCT/GB2007/050128 GB2007050128W WO2007107789A3 WO 2007107789 A3 WO2007107789 A3 WO 2007107789A3 GB 2007050128 W GB2007050128 W GB 2007050128W WO 2007107789 A3 WO2007107789 A3 WO 2007107789A3
Authority
WO
WIPO (PCT)
Prior art keywords
treatment
cns conditions
conditions
cns
modulates
Prior art date
Application number
PCT/GB2007/050128
Other languages
French (fr)
Other versions
WO2007107789A2 (en
Inventor
Anton Ana Isabel Jimenez
Yague Angela Sesto
Gomez Ma Concepcion Jimenez
Gullon Eduardo Gomez-Acebo
Original Assignee
Sylentis Sa
Williams Gareth Owen
Anton Ana Isabel Jimenez
Yague Angela Sesto
Gomez Ma Concepcion Jimenez
Gullon Eduardo Gomez-Acebo
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.)
Filing date
Publication date
Priority to US12/293,230 priority Critical patent/US8871729B2/en
Priority to JP2008558913A priority patent/JP5781722B2/en
Priority to EP07733553.7A priority patent/EP2004823B1/en
Priority to CA2645120A priority patent/CA2645120C/en
Priority to KR1020147013792A priority patent/KR101605144B1/en
Priority to AU2007228570A priority patent/AU2007228570B2/en
Priority to ES07733553.7T priority patent/ES2582649T3/en
Priority to CN200780017978.7A priority patent/CN101448944B/en
Application filed by Sylentis Sa, Williams Gareth Owen, Anton Ana Isabel Jimenez, Yague Angela Sesto, Gomez Ma Concepcion Jimenez, Gullon Eduardo Gomez-Acebo filed Critical Sylentis Sa
Priority to KR1020087025174A priority patent/KR101669944B1/en
Priority to MX2008011731A priority patent/MX2008011731A/en
Publication of WO2007107789A2 publication Critical patent/WO2007107789A2/en
Publication of WO2007107789A3 publication Critical patent/WO2007107789A3/en
Priority to IL194114A priority patent/IL194114A/en
Priority to HK09105618.2A priority patent/HK1126812A1/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • A61K2039/541—Mucosal route
    • A61K2039/543—Mucosal route intranasal
    • 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.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Methods and compositions for the treatment of pathologic conditions of the central nervous system (CNS) by means of intranasal administration of a composition that modulates, by means of RNA interference, the expression and/or activity of genes involved in above-mentioned conditions.
PCT/GB2007/050128 2006-03-17 2007-03-16 Treatment of cns conditions WO2007107789A2 (en)

Priority Applications (12)

Application Number Priority Date Filing Date Title
ES07733553.7T ES2582649T3 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
EP07733553.7A EP2004823B1 (en) 2006-03-17 2007-03-16 Treatment of cns conditions
CA2645120A CA2645120C (en) 2006-03-17 2007-03-16 Treatment of cns conditions
KR1020147013792A KR101605144B1 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
AU2007228570A AU2007228570B2 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
US12/293,230 US8871729B2 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
CN200780017978.7A CN101448944B (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
JP2008558913A JP5781722B2 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
KR1020087025174A KR101669944B1 (en) 2006-03-17 2007-03-16 Treatment of CNS conditions
MX2008011731A MX2008011731A (en) 2006-03-17 2007-03-16 Treatment of cns conditions.
IL194114A IL194114A (en) 2006-03-17 2008-09-15 Use of a rna interference compound in the preparation of medicaments for treating a disorder of the central nervous system, pharmaceutical compositions for intranasal administration comprising one or a plurality of sina targeting a gene expressed in the cns. and methods of reducing expression of a target gene in the cns of a non-human mammal
HK09105618.2A HK1126812A1 (en) 2006-03-17 2009-06-23 Treatment of cns conditions cns

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0605337.5A GB0605337D0 (en) 2006-03-17 2006-03-17 Treatment of CNS conditions
GB0605337.5 2006-03-17

Publications (2)

Publication Number Publication Date
WO2007107789A2 WO2007107789A2 (en) 2007-09-27
WO2007107789A3 true WO2007107789A3 (en) 2008-05-08

Family

ID=36292900

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2007/050128 WO2007107789A2 (en) 2006-03-17 2007-03-16 Treatment of cns conditions

Country Status (14)

Country Link
US (1) US8871729B2 (en)
EP (1) EP2004823B1 (en)
JP (2) JP5781722B2 (en)
KR (2) KR101605144B1 (en)
CN (1) CN101448944B (en)
AU (1) AU2007228570B2 (en)
CA (1) CA2645120C (en)
ES (1) ES2582649T3 (en)
GB (1) GB0605337D0 (en)
HK (1) HK1126812A1 (en)
IL (1) IL194114A (en)
MX (1) MX2008011731A (en)
RU (1) RU2426544C2 (en)
WO (1) WO2007107789A2 (en)

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JP2013511990A (en) 2009-11-26 2013-04-11 クォーク ファーマシューティカルズ インコーポレーティッド SiRNA compounds containing terminal substitutions
US20110135613A1 (en) * 2009-12-03 2011-06-09 The J. David Gladstone Institutes Methods for treating apolipoprotein e4-associated disorders
WO2011072091A1 (en) 2009-12-09 2011-06-16 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the cns
JP6081798B2 (en) * 2010-02-26 2017-02-15 メモリアル スローン−ケタリング キャンサー センター Methods and compositions and miRNA inhibitors and targets for detecting and treating cancers associated with miRNA
ES2638309T3 (en) 2010-04-19 2017-10-19 Nlife Therapeutics S.L. Compositions and methods for the selective distribution of oligonucleotide molecules to specific types of neurons
EP2380595A1 (en) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
ES2663009T3 (en) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibition of RNA-mediated gene expression using short interference nucleic acids (ANic)
WO2012078536A2 (en) 2010-12-06 2012-06-14 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
US9949923B2 (en) * 2011-03-15 2018-04-24 Optinose As Nasal delivery
EP2766495B1 (en) * 2011-10-14 2018-04-04 Centre National De La Recherche Scientifique Method of diagnosis, prognostic or treatment of neurodegenerative diseases
WO2013134777A1 (en) * 2012-03-09 2013-09-12 Northeastern University Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
CA2866392C (en) 2012-03-30 2023-10-24 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
US9611473B2 (en) 2012-09-12 2017-04-04 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
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
RU2745324C2 (en) 2013-03-14 2021-03-23 Ионис Фармасьютикалз, Инк. Compositions and methods for modulating expression of tau
JP2016523980A (en) 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク MicroRNA that suppresses tau expression
TWI772856B (en) 2013-07-19 2022-08-01 美商百健Ｍａ公司 Compositions for modulating tau expression
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
WO2015089375A1 (en) * 2013-12-13 2015-06-18 The General Hospital Corporation Soluble high molecular weight (hmw) tau species and applications thereof
WO2016151523A1 (en) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
JOP20200228A1 (en) * 2015-12-21 2017-06-16 Novartis Ag Compositions and methods for decreasing tau expression
JOP20190065A1 (en) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing tau expression
WO2019070894A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EP3692151A4 (en) 2017-10-03 2021-07-14 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
MX2020009532A (en) * 2018-03-13 2020-10-05 Janssen Pharmaceutica Nv Modified oligonucleotides for use in treatment of tauopathies.
CN115552006A (en) * 2020-03-18 2022-12-30 马萨诸塞大学 Oligonucleotides for MAPT modulation
CN113817728A (en) * 2020-06-19 2021-12-21 四川大学华西医院 Recombinant lentivirus capable of effectively interfering Tau protein expression and application thereof
TW202400792A (en) * 2022-05-12 2024-01-01 美商戴瑟納製藥股份有限公司 Compositions and methods for inhibiting mapt expression
WO2024145474A2 (en) * 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

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Also Published As

Publication number Publication date
CN101448944A (en) 2009-06-03
US20090176728A1 (en) 2009-07-09
EP2004823A2 (en) 2008-12-24
IL194114A (en) 2012-06-28
AU2007228570A8 (en) 2008-10-23
RU2426544C2 (en) 2011-08-20
IL194114A0 (en) 2011-08-01
KR101669944B1 (en) 2016-10-28
GB0605337D0 (en) 2006-04-26
JP5781722B2 (en) 2015-09-24
ES2582649T3 (en) 2016-09-14
CA2645120A1 (en) 2007-09-27
HK1126812A1 (en) 2009-09-11
KR20080111063A (en) 2008-12-22
CA2645120C (en) 2015-02-24
AU2007228570B2 (en) 2013-09-19
JP2013234197A (en) 2013-11-21
EP2004823B1 (en) 2016-05-25
JP5923466B2 (en) 2016-05-24
JP2009530257A (en) 2009-08-27
US8871729B2 (en) 2014-10-28
RU2008141162A (en) 2010-04-27
AU2007228570A1 (en) 2007-09-27
WO2007107789A2 (en) 2007-09-27
KR101605144B1 (en) 2016-03-21
MX2008011731A (en) 2009-03-05
KR20140076634A (en) 2014-06-20
CN101448944B (en) 2014-02-12

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