CA3123617A1 - Compositions and methods for the treatment of kcnt1 related disorders - Google Patents

Compositions and methods for the treatment of kcnt1 related disorders Download PDF

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Publication number
CA3123617A1
CA3123617A1 CA3123617A CA3123617A CA3123617A1 CA 3123617 A1 CA3123617 A1 CA 3123617A1 CA 3123617 A CA3123617 A CA 3123617A CA 3123617 A CA3123617 A CA 3123617A CA 3123617 A1 CA3123617 A1 CA 3123617A1
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Canada
Prior art keywords
oligonucleotide
compound
linkage
kcnt1
seq
Prior art date
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Pending
Application number
CA3123617A
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English (en)
French (fr)
Inventor
Steven Petrou
Michael Kristopher Mathieu KAHLIG
Kiran Reddy
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Praxis Precision Medicines Inc
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Praxis Precision Medicines Inc
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Publication of CA3123617A1 publication Critical patent/CA3123617A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3123617A 2018-12-20 2019-12-20 Compositions and methods for the treatment of kcnt1 related disorders Pending CA3123617A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862782877P 2018-12-20 2018-12-20
US62/782,877 2018-12-20
US201962862328P 2019-06-17 2019-06-17
US62/862,328 2019-06-17
US201962884567P 2019-08-08 2019-08-08
US62/884,567 2019-08-08
PCT/US2019/067976 WO2020132521A1 (en) 2018-12-20 2019-12-20 Compositions and methods for the treatment of kcnt1 related disorders

Publications (1)

Publication Number Publication Date
CA3123617A1 true CA3123617A1 (en) 2020-06-25

Family

ID=69187969

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3123617A Pending CA3123617A1 (en) 2018-12-20 2019-12-20 Compositions and methods for the treatment of kcnt1 related disorders

Country Status (7)

Country Link
US (1) US20220056455A1 (OSRAM)
EP (1) EP3898977A1 (OSRAM)
JP (1) JP2022515744A (OSRAM)
CN (1) CN113631709A (OSRAM)
AU (1) AU2019406186A1 (OSRAM)
CA (1) CA3123617A1 (OSRAM)
WO (1) WO2020132521A1 (OSRAM)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115701998A (zh) * 2020-06-25 2023-02-14 Ionis制药公司 用于降低kcnt1表达的化合物和方法
IL311570A (en) 2021-10-14 2024-05-01 Arsenal Biosciences Inc Immune cells with common expression chain and logic gate systems
CN118974255A (zh) * 2021-12-01 2024-11-15 阿塔兰塔治疗公司 用于治疗癫痫的组合物和方法
TW202417626A (zh) 2022-09-13 2024-05-01 美商亞森諾生物科學公司 具有共表現的tgfbr shrna之免疫細胞
CN119968464A (zh) 2022-09-16 2025-05-09 阿森纳生物科学公司 具有组合基因扰动的免疫细胞
JP2026507228A (ja) 2023-03-03 2026-02-27 アーセナル バイオサイエンシズ インコーポレイテッド Psma及びca9を標的とするシステム
IL327100A (en) 2023-09-15 2026-05-01 Praxis Prec Medicines Inc Alsonarsen/PRAX-22 for use in the treatment of SCN2A-related disorders
WO2025199338A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Systems targeting slc34a2 and tmprss4 and methods of use thereof

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