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EP2297341A4 - Procédés et compositions pour le traitement de la maladie de huntington - Google Patents

Procédés et compositions pour le traitement de la maladie de huntington

Info

Publication number
EP2297341A4
EP2297341A4 EP09741640A EP09741640A EP2297341A4 EP 2297341 A4 EP2297341 A4 EP 2297341A4 EP 09741640 A EP09741640 A EP 09741640A EP 09741640 A EP09741640 A EP 09741640A EP 2297341 A4 EP2297341 A4 EP 2297341A4
Authority
EP
European Patent Office
Prior art keywords
huntington
disease
compositions
treatment
methods
Prior art date
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.)
Withdrawn
Application number
EP09741640A
Other languages
German (de)
English (en)
Other versions
EP2297341A1 (fr
Inventor
Michael Hayden
Jeffrey Carroll
Simon Warby
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of British Columbia
Original Assignee
University of British Columbia
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
Application filed by University of British Columbia filed Critical University of British Columbia
Publication of EP2297341A1 publication Critical patent/EP2297341A1/fr
Publication of EP2297341A4 publication Critical patent/EP2297341A4/fr
Withdrawn legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP09741640A 2008-05-09 2009-05-08 Procédés et compositions pour le traitement de la maladie de huntington Withdrawn EP2297341A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US71465208P 2008-05-09 2008-05-09
PCT/CA2009/000645 WO2009135322A1 (fr) 2008-05-09 2009-05-08 Procédés et compositions pour le traitement de la maladie de huntington

Publications (2)

Publication Number Publication Date
EP2297341A1 EP2297341A1 (fr) 2011-03-23
EP2297341A4 true EP2297341A4 (fr) 2013-01-09

Family

ID=41264389

Family Applications (1)

Application Number Title Priority Date Filing Date
EP09741640A Withdrawn EP2297341A4 (fr) 2008-05-09 2009-05-08 Procédés et compositions pour le traitement de la maladie de huntington

Country Status (4)

Country Link
EP (1) EP2297341A4 (fr)
AU (1) AU2009244013B2 (fr)
CA (1) CA2726866A1 (fr)
WO (1) WO2009135322A1 (fr)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
WO2011005761A1 (fr) 2009-07-06 2011-01-13 Ontorii, Inc Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation
WO2011097644A2 (fr) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Réduction sélective de variants alléliques
WO2011097643A1 (fr) * 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Réduction sélective de variants alléliques
EP2620428B1 (fr) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Groupe auxiliaire asymétrique
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
DK2742136T3 (da) 2011-08-11 2017-11-20 Ionis Pharmaceuticals Inc Gapmerforbindelser omfattende 5'-modificerede deoxyribonukleosider i gap og anvendelser deraf
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
KR101835401B1 (ko) 2012-07-13 2018-03-08 신 니뽄 바이오메디칼 라보라토리즈, 엘티디. 키랄 핵산 어쥬번트
EP2872485B1 (fr) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Groupe auxiliaire asymétrique
DK2906256T3 (en) 2012-10-12 2018-11-19 Ionis Pharmaceuticals Inc SELECTIVE ANTISENSE COMPOUNDS AND APPLICATIONS THEREOF
CA2899924A1 (fr) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Composes antisens selectifs et leurs utilisations
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
EP3095459A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
US10221416B2 (en) 2014-04-24 2019-03-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EP4069846A1 (fr) * 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions et méthodes pour le ciblage de htt
WO2024173783A2 (fr) * 2023-02-17 2024-08-22 Ionis Pharmaceuticals, Inc. Composés sélectifs d'allèles et méthodes de modulation de l'expression de la huntingtine

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020187931A1 (en) * 2000-04-13 2002-12-12 Michael Hayden Modulating cell survival by modulating huntingtin function
US20100299768A1 (en) * 2007-06-18 2010-11-25 Commissariat A L'energie Atomique Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and its Application for the Treatment of Huntington's Disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008005562A2 (fr) * 2006-07-07 2008-01-10 University Of Massachusetts Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington
EP2316967A1 (fr) * 2005-06-28 2011-05-04 Medtronic, Inc. Méthodes et séquences pour supprimer préférentiellement l'expression du gène huntingtin muté.
US9273356B2 (en) * 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020187931A1 (en) * 2000-04-13 2002-12-12 Michael Hayden Modulating cell survival by modulating huntingtin function
US20100299768A1 (en) * 2007-06-18 2010-11-25 Commissariat A L'energie Atomique Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and its Application for the Treatment of Huntington's Disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DENOVAN-WRIGHT E M ET AL: "RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 13, no. 6, 1 March 2006 (2006-03-01), pages 525 - 531, XP002452333, ISSN: 0969-7128 *
See also references of WO2009135322A1 *

Also Published As

Publication number Publication date
CA2726866A1 (fr) 2009-11-12
WO2009135322A1 (fr) 2009-11-12
AU2009244013B2 (en) 2015-06-25
AU2009244013A1 (en) 2009-11-12
EP2297341A1 (fr) 2011-03-23

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