AU2013203395A1 - Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome - Google Patents

Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome Download PDF

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Publication number
AU2013203395A1
AU2013203395A1 AU2013203395A AU2013203395A AU2013203395A1 AU 2013203395 A1 AU2013203395 A1 AU 2013203395A1 AU 2013203395 A AU2013203395 A AU 2013203395A AU 2013203395 A AU2013203395 A AU 2013203395A AU 2013203395 A1 AU2013203395 A1 AU 2013203395A1
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Prior art keywords
tau
antisense
certain embodiments
nucleic acid
modified
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Abandoned
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AU2013203395A
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Inventor
C. Frank Bennett
Susan M. Freier
Frank Rigo
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Ionis Pharmaceuticals Inc
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Isis Pharmaceuticals Inc
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    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
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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
    • A61P25/28—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
    • A61P35/00—Antineoplastic agents
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • 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 ===---===
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    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
AU2013203395A 2012-03-30 2013-03-14 Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome Abandoned AU2013203395A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261618435P 2012-03-30 2012-03-30
US61/618,435 2012-03-30
US201261660676P 2012-06-15 2012-06-15
US61/660,676 2012-06-15
US201261719149P 2012-10-26 2012-10-26
US61/719,149 2012-10-26
PCT/US2013/031710 WO2013148283A1 (en) 2012-03-30 2013-03-14 Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome

Publications (1)

Publication Number Publication Date
AU2013203395A1 true AU2013203395A1 (en) 2013-10-17

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Family Applications (3)

Application Number Title Priority Date Filing Date
AU2013202595A Active AU2013202595B2 (en) 2012-03-30 2013-03-14 Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome
AU2013203395A Abandoned AU2013203395A1 (en) 2012-03-30 2013-03-14 Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
AU2016202220A Active AU2016202220B2 (en) 2012-03-30 2016-04-11 Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome

Family Applications Before (1)

Application Number Title Priority Date Filing Date
AU2013202595A Active AU2013202595B2 (en) 2012-03-30 2013-03-14 Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2016202220A Active AU2016202220B2 (en) 2012-03-30 2016-04-11 Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome

Country Status (6)

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US (2) US10273474B2 (enExample)
EP (2) EP2831231A1 (enExample)
JP (1) JP6492003B2 (enExample)
AU (3) AU2013202595B2 (enExample)
CA (1) CA2866392C (enExample)
WO (2) WO2013148260A1 (enExample)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
CN109072237A (zh) * 2015-12-21 2018-12-21 诺华股份有限公司 降低tau表达的组合物和方法

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WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
MX2015012621A (es) 2013-03-14 2016-05-31 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tau.
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2016019063A1 (en) * 2014-07-29 2016-02-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
PE20171766A1 (es) * 2015-02-04 2017-12-21 Hoffmann La Roche Oligomeros antisentido de tau y usos de estos
EP3254104B1 (en) * 2015-02-04 2021-08-25 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
US10357543B2 (en) 2015-11-16 2019-07-23 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein
WO2017087282A1 (en) 2015-11-18 2017-05-26 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
CR20210178A (es) * 2018-07-03 2021-05-11 Hoffmann La Roche OLIGONUCLEÓTIDOS PARA MODULAR LA EXPRESIÓN DE TAU (Divisional 2021-0058)
JP7340794B2 (ja) * 2018-07-04 2023-09-08 学校法人 愛知医科大学 タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途
WO2020037234A1 (en) * 2018-08-16 2020-02-20 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Nucleic acids and nucleic acid analogs for treating, preventing, and disrupting pathological polynucleotide-binding protein inclusions
KR20230005194A (ko) * 2020-03-30 2023-01-09 알닐람 파마슈티칼스 인코포레이티드 미세소관 관련된 단백질 TAU (MAPT) iRNA 제제 조성물 및 이의 사용 방법
JP2024543214A (ja) * 2021-12-03 2024-11-19 クラリス コーポレーション Ｕｎｃ１３ａ遺伝子転写物のモジュレーターを使用する神経疾患の処置
EP4655320A1 (en) * 2023-01-27 2025-12-03 The United States Government As Represented By The Department Of Veterans Affairs Compositions for treating tauopathies and methods of use thereof

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