WO2005042777A3 - App/ena antisense - Google Patents

App/ena antisense Download PDF

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Publication number
WO2005042777A3
WO2005042777A3 PCT/GB2004/004483 GB2004004483W WO2005042777A3 WO 2005042777 A3 WO2005042777 A3 WO 2005042777A3 GB 2004004483 W GB2004004483 W GB 2004004483W WO 2005042777 A3 WO2005042777 A3 WO 2005042777A3
Authority
WO
WIPO (PCT)
Prior art keywords
app
strategies
early
deposition
ena
Prior art date
Application number
PCT/GB2004/004483
Other languages
French (fr)
Other versions
WO2005042777A2 (en
Inventor
Peter Estibeiro
David Page
Jenny Godfray
Eleanor Barnard
Makoto Koizumi
Original Assignee
Expresson Biosystems Ltd
Sankyo Co
Peter Estibeiro
David Page
Jenny Godfray
Eleanor Barnard
Makoto Koizumi
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 Expresson Biosystems Ltd, Sankyo Co, Peter Estibeiro, David Page, Jenny Godfray, Eleanor Barnard, Makoto Koizumi filed Critical Expresson Biosystems Ltd
Publication of WO2005042777A2 publication Critical patent/WO2005042777A2/en
Publication of WO2005042777A3 publication Critical patent/WO2005042777A3/en

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    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/12—Applications; Uses in screening processes in functional genomics, i.e. for the determination of gene function

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  • 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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention relates to strategies to address the deposition of APP which is associated with early etiology of disease states, such as Alzheimer's disease, by reducing the early deposition of amyloid ß peptide (Aß) in the brain. The strategies can also be used as research tools. The invention uses silencing reagents in the form of specific antisense molecules or siRNA to modulate APP.
PCT/GB2004/004483 2003-10-24 2004-10-22 App/ena antisense WO2005042777A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0324854.9A GB0324854D0 (en) 2003-10-24 2003-10-24 App/ena antisense
GB0324854.9 2003-10-24

Publications (2)

Publication Number Publication Date
WO2005042777A2 WO2005042777A2 (en) 2005-05-12
WO2005042777A3 true WO2005042777A3 (en) 2005-08-25

Family

ID=29595770

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2004/004483 WO2005042777A2 (en) 2003-10-24 2004-10-22 App/ena antisense

Country Status (2)

Country Link
GB (1) GB0324854D0 (en)
WO (1) WO2005042777A2 (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5683261B2 (en) * 2008-03-11 2015-03-11 学校法人 埼玉医科大学 Double-stranded nucleic acid molecule suitable for cancer prevention or treatment, cancer cell growth inhibitor, and pharmaceutical
EP2638163B1 (en) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-associated non-coding rnas
DK2739311T3 (en) 2011-08-04 2018-04-23 Amgen Inc Method of treating bone slit defects
JP6129844B2 (en) 2011-09-14 2017-05-17 ラナ セラピューティクス インコーポレイテッド Multimeric oligonucleotide compounds
MX354270B (en) 2011-12-28 2018-02-21 Amgen Inc Method of treating alvelor bone loss through the use of anti-sclerostin antibodies.
US10059941B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CA2873809A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
EP3161159B1 (en) 2014-06-25 2020-08-05 The General Hospital Corporation Targeting human satellite ii (hsatii)
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
TW202039844A (en) * 2018-12-19 2020-11-01 美商阿尼拉製藥公司 Amyloid precursor protein (app) rnai agent compositions and methods of use thereof
EP3918073A4 (en) * 2019-01-29 2023-11-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000047599A1 (en) * 1999-02-12 2000-08-17 Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
US6177246B1 (en) * 1991-12-24 2001-01-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulating β-amyloid
WO2001042266A1 (en) * 1999-12-09 2001-06-14 Saint Louis University Antisense modulation of amyloid beta protein expression
WO2002072886A2 (en) * 2001-03-08 2002-09-19 Expresson Biosystems Limited Complex element micro-array and methods of use
US20030232435A1 (en) * 2002-06-14 2003-12-18 Isis Pharmaceuticals Inc. Antisense modulation of amyloid beta protein precursor expression

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6177246B1 (en) * 1991-12-24 2001-01-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulating β-amyloid
WO2000047599A1 (en) * 1999-02-12 2000-08-17 Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
EP1152009A1 (en) * 1999-02-12 2001-11-07 Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
WO2001042266A1 (en) * 1999-12-09 2001-06-14 Saint Louis University Antisense modulation of amyloid beta protein expression
WO2002072886A2 (en) * 2001-03-08 2002-09-19 Expresson Biosystems Limited Complex element micro-array and methods of use
US20030232435A1 (en) * 2002-06-14 2003-12-18 Isis Pharmaceuticals Inc. Antisense modulation of amyloid beta protein precursor expression

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
COULSON E J ET AL: "DOWN-REGULATION OF THE AMYLOID PROTEIN PRECURSOR OF ALZHEIMER'S DISEASE BY ANTISENSE OLIGONUCLEOTIDES REDUCES NEURONAL ADHESION TO SPECIFIC SUBSTRATA", BRAIN RESEARCH, vol. 770, 1997, pages 72 - 80, XP002937688, ISSN: 0006-8993 *
ESTIBEIRO P ET AL: "Antisense as a neuroscience tool and therapeutic agent", TRENDS IN NEUROSCIENCE, vol. 24, 1 November 2001 (2001-11-01), pages 56 - 62, XP004512870, ISSN: 0166-2236 *
GODFRAY J ET AL: "The potential of antisense as a CNS therapeutic", EXPERT OPINION ON THERAPEUTIC TARGETS, vol. 7, no. 3, June 2003 (2003-06-01), pages 363 - 376, XP008044774, ISSN: 1472-8222 *
LUO J-J ET AL: "CHARACTERIZATION OF THE NEUROTROPHIC INTERACTION BETWEEN NERVE GROWTH FACTOR AND SECRETED ALPHA-AMYLOID PRECURSOR PROTEIN", JOURNAL OF NEUROSCIENCE RESEARCH, vol. 63, 2001, pages 410 - 420, XP001053718, ISSN: 0360-4012 *

Also Published As

Publication number Publication date
GB0324854D0 (en) 2003-11-26
WO2005042777A2 (en) 2005-05-12

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