BR112021015494A2 - Compostos e métodos para reduzir a expressão de kcnt1 - Google Patents

Compostos e métodos para reduzir a expressão de kcnt1 Download PDF

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Publication number
BR112021015494A2
BR112021015494A2 BR112021015494-9A BR112021015494A BR112021015494A2 BR 112021015494 A2 BR112021015494 A2 BR 112021015494A2 BR 112021015494 A BR112021015494 A BR 112021015494A BR 112021015494 A2 BR112021015494 A2 BR 112021015494A2
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BR
Brazil
Prior art keywords
seq
nucleobases
equal length
oligomeric compound
length portion
Prior art date
Application number
BR112021015494-9A
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English (en)
Portuguese (pt)
Inventor
Huynh-Hoa Bui
Susan M. Freier
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Ionis Pharmaceuticals, Inc.
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Application filed by Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of BR112021015494A2 publication Critical patent/BR112021015494A2/pt

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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • 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
    • A61P25/24—Antidepressants
    • 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
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
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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
    • 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
    • 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
    • C12N2310/351—Conjugate
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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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
BR112021015494-9A 2019-03-15 2020-03-13 Compostos e métodos para reduzir a expressão de kcnt1 BR112021015494A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962819344P 2019-03-15 2019-03-15
US62/819,344 2019-03-15
US201962884501P 2019-08-08 2019-08-08
US62/884,501 2019-08-08
PCT/US2020/022680 WO2020190740A1 (en) 2019-03-15 2020-03-13 Compounds and methods for reducing kcnt1 expression

Publications (1)

Publication Number Publication Date
BR112021015494A2 true BR112021015494A2 (pt) 2021-10-05

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BR112021015494-9A BR112021015494A2 (pt) 2019-03-15 2020-03-13 Compostos e métodos para reduzir a expressão de kcnt1

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US (2) US20220177893A1 (https=)
EP (1) EP3938514A4 (https=)
JP (2) JP7564817B2 (https=)
KR (1) KR20210141983A (https=)
CN (3) CN121674396A (https=)
AU (1) AU2020241693B2 (https=)
BR (1) BR112021015494A2 (https=)
CA (1) CA3133247A1 (https=)
CL (1) CL2021002398A1 (https=)
CO (1) CO2021013371A2 (https=)
CR (1) CR20210519A (https=)
IL (1) IL285546A (https=)
JO (1) JOP20210254A1 (https=)
MX (1) MX2021011132A (https=)
PE (1) PE20220168A1 (https=)
PH (1) PH12021552220A1 (https=)
SG (1) SG11202108625WA (https=)
TW (1) TW202102675A (https=)
WO (1) WO2020190740A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10952978B2 (en) 2017-08-28 2021-03-23 Enanta Pharmaceuticals, Inc. Hepatitis B antiviral agents
US11198693B2 (en) 2018-11-21 2021-12-14 Enanta Pharmaceuticals, Inc. Functionalized heterocycles as antiviral agents
WO2021188414A1 (en) 2020-03-16 2021-09-23 Enanta Pharmaceuticals, Inc. Functionalized heterocyclic compounds as antiviral agents
MX2024006709A (es) * 2021-12-01 2024-08-19 Atalanta Therapeutics Inc Composiciones y metodos para el tratamiento de epilepsias.
WO2025036916A1 (en) * 2023-08-16 2025-02-20 Les Laboratoires Servier Oligonucleotides for modulating kcnt1 expression

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288242A1 (en) * 2001-05-18 2005-12-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of RAS gene expression using short interfering nucleic acid (siNA)
US7601501B2 (en) * 2006-08-11 2009-10-13 The Scripps Research Institute Controlling osteogenesis by inhibition of osteogenic suppressors
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
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
US20200129538A1 (en) 2017-06-13 2020-04-30 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1

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Publication number Publication date
CL2021002398A1 (es) 2022-06-03
MX2021011132A (es) 2021-10-14
PH12021552220A1 (en) 2022-05-30
WO2020190740A1 (en) 2020-09-24
EP3938514A4 (en) 2023-05-03
CA3133247A1 (en) 2020-09-24
IL285546A (en) 2021-09-30
CR20210519A (es) 2021-11-24
EP3938514A1 (en) 2022-01-19
CN113661241A (zh) 2021-11-16
KR20210141983A (ko) 2021-11-23
US20250320505A1 (en) 2025-10-16
CN121674396A (zh) 2026-03-17
AU2020241693A1 (en) 2021-09-02
US20220177893A1 (en) 2022-06-09
JP7564817B2 (ja) 2024-10-09
SG11202108625WA (en) 2021-09-29
PE20220168A1 (es) 2022-01-28
JOP20210254A1 (ar) 2023-01-30
TW202102675A (zh) 2021-01-16
CO2021013371A2 (es) 2021-10-20
AU2020241693B2 (en) 2024-01-04
JP2025000805A (ja) 2025-01-07
JP2022526267A (ja) 2022-05-24
CN117106778A (zh) 2023-11-24

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