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DK3519572T3 - Forbindelser og fremgangsmåder til reduktion af tau-ekspression - Google Patents

Forbindelser og fremgangsmåder til reduktion af tau-ekspression Download PDF

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Publication number
DK3519572T3
DK3519572T3 DK17857565.0T DK17857565T DK3519572T3 DK 3519572 T3 DK3519572 T3 DK 3519572T3 DK 17857565 T DK17857565 T DK 17857565T DK 3519572 T3 DK3519572 T3 DK 3519572T3
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Prior art keywords
compounds
methods
tau expression
reducing tau
reducing
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DK17857565.0T
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DK3519572T5 (da
Inventor
Holly Kordasiewicz
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Biogen Ma 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
    • 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
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    • 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
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    • 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
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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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    • C12N2310/33—Chemical structure of the base
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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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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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DK17857565.0T 2016-09-29 2017-09-29 Forbindelser og fremgangsmåder til reduktion af tau-ekspression DK3519572T5 (da)

Applications Claiming Priority (3)

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

Publications (2)

Publication Number Publication Date
DK3519572T3 true DK3519572T3 (da) 2023-12-18
DK3519572T5 DK3519572T5 (da) 2024-09-02

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DK17857565.0T DK3519572T5 (da) 2016-09-29 2017-09-29 Forbindelser og fremgangsmåder til reduktion af tau-ekspression

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US (3) US10407680B2 (da)
EP (2) EP4335923A3 (da)
JP (4) JP6738004B2 (da)
KR (2) KR20210097228A (da)
CN (2) CN109790543B (da)
AU (3) AU2017336093B2 (da)
BR (2) BR122020019785B1 (da)
CA (1) CA3038891A1 (da)
CL (2) CL2019000703A1 (da)
CO (1) CO2019001385A2 (da)
CR (2) CR20210028A (da)
DK (1) DK3519572T5 (da)
ES (1) ES2963428T3 (da)
FI (1) FI3519572T3 (da)
HR (1) HRP20231606T1 (da)
HU (1) HUE065170T2 (da)
IL (3) IL299615A (da)
JO (1) JOP20190065A1 (da)
LT (1) LT3519572T (da)
MD (1) MD3519572T2 (da)
MX (2) MX2019003633A (da)
NZ (1) NZ750642A (da)
PE (1) PE20190655A1 (da)
PL (1) PL3519572T3 (da)
PT (1) PT3519572T (da)
RS (1) RS64941B1 (da)
SG (1) SG11201901140QA (da)
SI (1) SI3519572T1 (da)
WO (1) WO2018064593A1 (da)
ZA (2) ZA201900920B (da)

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AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
EP3336189A1 (en) * 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2014153236A1 (en) 2013-03-14 2014-09-25 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
CN111836624A (zh) 2018-01-12 2020-10-27 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
CR20210058A (es) * 2018-07-03 2021-03-22 Hoffmann La Roche Oligonucleótidos para modular la expresión de tau
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
US11753644B2 (en) 2021-06-18 2023-09-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing IFNAR1 expression
WO2023004426A2 (en) * 2021-07-23 2023-01-26 University Of Kentucky Research Foundation Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus
TW202310854A (zh) * 2021-07-23 2023-03-16 美商百健Ｍａ公司 用於減少ｔａｕ表現之方法

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