CN115701998A - 用于降低kcnt1表达的化合物和方法 - Google Patents

用于降低kcnt1表达的化合物和方法 Download PDF

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Publication number
CN115701998A
CN115701998A CN202180045166.3A CN202180045166A CN115701998A CN 115701998 A CN115701998 A CN 115701998A CN 202180045166 A CN202180045166 A CN 202180045166A CN 115701998 A CN115701998 A CN 115701998A
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Prior art keywords
modified
oligomeric compound
modified oligonucleotide
certain embodiments
tds
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CN202180045166.3A
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English (en)
Chinese (zh)
Inventor
P·杰法尔-内贾德
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of CN115701998A publication Critical patent/CN115701998A/zh
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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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  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
CN202180045166.3A 2020-06-25 2021-06-25 用于降低kcnt1表达的化合物和方法 Pending CN115701998A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063044345P 2020-06-25 2020-06-25
US63/044,345 2020-06-25
US202063079438P 2020-09-16 2020-09-16
US63/079,438 2020-09-16
PCT/US2021/039049 WO2021263082A2 (en) 2020-06-25 2021-06-25 Compounds and methods for reducing kcnt1 expression

Publications (1)

Publication Number Publication Date
CN115701998A true CN115701998A (zh) 2023-02-14

Family

ID=79281885

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180045166.3A Pending CN115701998A (zh) 2020-06-25 2021-06-25 用于降低kcnt1表达的化合物和方法

Country Status (6)

Country Link
US (1) US20240229042A1 (https=)
EP (1) EP4171576A4 (https=)
JP (1) JP2023533153A (https=)
CN (1) CN115701998A (https=)
UY (1) UY39297A (https=)
WO (1) WO2021263082A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025117662A1 (en) * 2023-11-29 2025-06-05 Actio Biosciences, Inc. Heterocyclic compounds useful as kcnt1 inhibitors

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040023328A1 (en) * 2000-06-14 2004-02-05 Applera Corporation Isolated human transporter proteins, nucleic acid molecules encoding human transporter proteins, and uses thereof
WO2005083127A2 (en) * 2004-02-27 2005-09-09 Applera Corporation Genetic polymorphisms associated with stroke, methods of detection and uses thereof
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

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019406186A1 (en) * 2018-12-20 2021-07-15 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of KCNT1 related disorders

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040023328A1 (en) * 2000-06-14 2004-02-05 Applera Corporation Isolated human transporter proteins, nucleic acid molecules encoding human transporter proteins, and uses thereof
WO2005083127A2 (en) * 2004-02-27 2005-09-09 Applera Corporation Genetic polymorphisms associated with stroke, methods of detection and uses thereof
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

Also Published As

Publication number Publication date
WO2021263082A3 (en) 2022-02-10
EP4171576A2 (en) 2023-05-03
JP2023533153A (ja) 2023-08-02
UY39297A (es) 2021-12-31
US20240229042A1 (en) 2024-07-11
WO2021263082A2 (en) 2021-12-30
EP4171576A4 (en) 2025-03-19

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