JP6738004B2 - タウ発現を低減するための化合物及び方法 - Google Patents

タウ発現を低減するための化合物及び方法 Download PDF

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JP6738004B2
JP6738004B2 JP2019517059A JP2019517059A JP6738004B2 JP 6738004 B2 JP6738004 B2 JP 6738004B2 JP 2019517059 A JP2019517059 A JP 2019517059A JP 2019517059 A JP2019517059 A JP 2019517059A JP 6738004 B2 JP6738004 B2 JP 6738004B2
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modified
certain embodiments
oligonucleotide
population
modified oligonucleotide
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JP2019529489A5 (enExample
JP2019529489A (ja
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コルダシェヴィッチ，ホリー
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Biogen MA Inc
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Biogen MA Inc
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/46—Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
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    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/31—Chemical structure of the backbone
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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
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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
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    • C12N2310/32—Chemical structure of the sugar
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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
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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 ===---===
    • 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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Microbiology (AREA)
  • Pain & Pain Management (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Botany (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2019517059A 2016-09-29 2017-09-29 タウ発現を低減するための化合物及び方法 Active JP6738004B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662401723P 2016-09-29 2016-09-29
US62/401,723 2016-09-29
US201762450469P 2017-01-25 2017-01-25
US62/450,469 2017-01-25
PCT/US2017/054540 WO2018064593A1 (en) 2016-09-29 2017-09-29 Compounds and methods for reducing tau expression

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JP2019529489A JP2019529489A (ja) 2019-10-17
JP2019529489A5 JP2019529489A5 (enExample) 2020-05-14
JP6738004B2 true JP6738004B2 (ja) 2020-08-12

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JP2020110145A Withdrawn JP2020193199A (ja) 2016-09-29 2020-06-26 タウ発現を低減するための化合物及び方法
JP2022139094A Active JP7477569B2 (ja) 2016-09-29 2022-09-01 タウ発現を低減するための化合物及び方法
JP2024067450A Pending JP2024091814A (ja) 2016-09-29 2024-04-18 タウ発現を低減するための化合物及び方法

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JP2022139094A Active JP7477569B2 (ja) 2016-09-29 2022-09-01 タウ発現を低減するための化合物及び方法
JP2024067450A Pending JP2024091814A (ja) 2016-09-29 2024-04-18 タウ発現を低減するための化合物及び方法

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US (3) US10407680B2 (enExample)
EP (2) EP3519572B1 (enExample)
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KR (2) KR20210097228A (enExample)
CN (2) CN109790543B (enExample)
AU (3) AU2017336093B2 (enExample)
BR (2) BR112019003162B1 (enExample)
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CL (2) CL2019000703A1 (enExample)
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JO (2) JOP20190065B1 (enExample)
LT (1) LT3519572T (enExample)
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PE (1) PE20190655A1 (enExample)
PL (1) PL3519572T3 (enExample)
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RS (1) RS64941B1 (enExample)
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SI (1) SI3519572T1 (enExample)
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Cited By (1)

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JP2020193199A (ja) * 2016-09-29 2020-12-03 バイオジェン・エムエイ・インコーポレイテッドＢｉｏｇｅｎ ＭＡ Ｉｎｃ． タウ発現を低減するための化合物及び方法

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WO2013148283A1 (en) 2012-03-30 2013-10-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP2839006B1 (en) * 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
CN111254145A (zh) 2013-03-14 2020-06-09 Ionis制药公司 用于调节tau表达的组合物和方法
TWI657819B (zh) 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
US12241065B2 (en) 2018-01-12 2025-03-04 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2020007892A1 (en) 2018-07-03 2020-01-09 F. Hoffmann-La Roche Ag Oligonucleotides for modulating tau expression
WO2022125984A1 (en) * 2020-12-11 2022-06-16 Eisai R&D Management Co., Ltd. Tau-targeting oligonucleotide gapmers
JP2024513403A (ja) 2021-04-01 2024-03-25 バイオジェン・エムエイ・インコーポレイテッド 中枢神経系への核酸送達
EP4355759A4 (en) 2021-06-18 2026-05-13 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF IFNAR1
PE20240648A1 (es) * 2021-07-23 2024-04-04 Biogen Ma Inc Metodos para reducir la expresion de tau
US20240401055A1 (en) * 2021-07-23 2024-12-05 University Of Kentucky Research Foundation Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus
MX2024003519A (es) 2021-09-24 2024-04-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
JP2024539596A (ja) * 2021-10-07 2024-10-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド タウ発現を低減するための化合物及び方法
KR20260057473A (ko) * 2023-08-23 2026-04-28 아이오니스 파마수티컬즈, 인코포레이티드 Atn1 발현을 감소시키기 위한 화합물 및 방법
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