BR112020018365A2 - Oligonucleotídeos modificados e métodos para uso em tauopatias - Google Patents

Oligonucleotídeos modificados e métodos para uso em tauopatias Download PDF

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Publication number
BR112020018365A2
BR112020018365A2 BR112020018365-2A BR112020018365A BR112020018365A2 BR 112020018365 A2 BR112020018365 A2 BR 112020018365A2 BR 112020018365 A BR112020018365 A BR 112020018365A BR 112020018365 A2 BR112020018365 A2 BR 112020018365A2
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Brazil
Prior art keywords
oligonucleotide
fact
modified
nucleotides
formula
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BR112020018365-2A
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English (en)
Portuguese (pt)
Inventor
Andreas Ebneth
Constantin Van Outryve D' Ydewalle
Sergei Gryaznov
Saúl Martinez Montero
Leonid Beigelman
Vivek Kumar Rajwanshi
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Janssen Pharmaceutica Nv
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Publication of BR112020018365A2 publication Critical patent/BR112020018365A2/pt

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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
    • 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
    • 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
    • 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
    • C12N2310/30—Chemical structure
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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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    • C12N2310/00—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
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
    • C12N2310/3521—Methyl
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

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  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
BR112020018365-2A 2018-03-13 2019-03-13 Oligonucleotídeos modificados e métodos para uso em tauopatias BR112020018365A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862642499P 2018-03-13 2018-03-13
US62/642,499 2018-03-13
PCT/EP2019/056312 WO2019175260A2 (en) 2018-03-13 2019-03-13 Modified oligonucleotides and methods of use in tauopathies

Publications (1)

Publication Number Publication Date
BR112020018365A2 true BR112020018365A2 (pt) 2020-12-29

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BR112020018365-2A BR112020018365A2 (pt) 2018-03-13 2019-03-13 Oligonucleotídeos modificados e métodos para uso em tauopatias

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US (2) US11453881B2 (https=)
EP (2) EP3922637A1 (https=)
JP (1) JP2021515784A (https=)
KR (1) KR20200131263A (https=)
CN (1) CN111868068A (https=)
AU (1) AU2019233612A1 (https=)
BR (1) BR112020018365A2 (https=)
CA (1) CA3091979A1 (https=)
EA (1) EA202092156A1 (https=)
ES (1) ES3063421T3 (https=)
IL (1) IL277238A (https=)
MA (1) MA52149A (https=)
MX (1) MX2020009532A (https=)
SG (1) SG11202008895WA (https=)
WO (1) WO2019175260A2 (https=)

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EP2831232A4 (en) 2012-03-30 2015-11-04 Univ Washington METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME
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
WO2021198958A1 (en) * 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
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
CN121729493A (zh) * 2023-06-22 2026-03-24 戴纳立制药公司 用于调节τ蛋白表达的组合物和方法
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026025082A1 (en) * 2024-07-25 2026-01-29 Biogen Ma Inc. Methods for treating alzheimer's disease

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US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
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US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
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JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
CN1317291C (zh) 1999-09-10 2007-05-23 杰龙公司 寡核苷酸n3′→p5′硫代氨基磷酸酯,其合成及应用
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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
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Publication number Publication date
SG11202008895WA (en) 2020-10-29
AU2019233612A1 (en) 2020-09-10
KR20200131263A (ko) 2020-11-23
ES3063421T3 (en) 2026-04-16
EP3765476A2 (en) 2021-01-20
WO2019175260A3 (en) 2019-11-14
WO2019175260A2 (en) 2019-09-19
CA3091979A1 (en) 2019-09-19
MA52149A (fr) 2021-01-20
JP2021515784A (ja) 2021-06-24
US11453881B2 (en) 2022-09-27
EP3765476B1 (en) 2025-11-26
US20240011029A1 (en) 2024-01-11
MX2020009532A (es) 2020-10-05
EP3765476C0 (en) 2025-11-26
IL277238A (en) 2020-10-29
EA202092156A1 (ru) 2021-04-01
US20210095284A1 (en) 2021-04-01
EP3922637A1 (en) 2021-12-15
CN111868068A (zh) 2020-10-30

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