CA3110211A1 - Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies - Google Patents

Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies Download PDF

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Publication number
CA3110211A1
CA3110211A1 CA3110211A CA3110211A CA3110211A1 CA 3110211 A1 CA3110211 A1 CA 3110211A1 CA 3110211 A CA3110211 A CA 3110211A CA 3110211 A CA3110211 A CA 3110211A CA 3110211 A1 CA3110211 A1 CA 3110211A1
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Prior art keywords
certain embodiments
modified
oligonucleotide
compound
scn2a
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Pending
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CA3110211A
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English (en)
French (fr)
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Steven Petrou
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Rogcon Inc
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Rogcon Inc
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    • 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
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    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • 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
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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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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    • C12N2310/30—Chemical structure
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3110211A 2018-08-20 2019-08-20 Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies Pending CA3110211A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862765344P 2018-08-20 2018-08-20
US62/765,344 2018-08-20
PCT/US2019/047313 WO2020041348A1 (en) 2018-08-20 2019-08-20 Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies

Publications (1)

Publication Number Publication Date
CA3110211A1 true CA3110211A1 (en) 2020-02-27

Family

ID=69591162

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Application Number Title Priority Date Filing Date
CA3110211A Pending CA3110211A1 (en) 2018-08-20 2019-08-20 Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies

Country Status (9)

Country Link
US (2) US11939582B2 (https=)
EP (1) EP3840733A4 (https=)
JP (1) JP7476199B2 (https=)
CN (1) CN113329739B (https=)
AU (1) AU2019325255A1 (https=)
BR (1) BR112021003224A2 (https=)
CA (1) CA3110211A1 (https=)
EA (1) EA202190581A1 (https=)
WO (1) WO2020041348A1 (https=)

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KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
WO2021174036A1 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
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CN116368227A (zh) * 2020-07-22 2023-06-30 弗洛里神经科学与心理健康研究所 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法
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JP2024091781A (ja) 2024-07-05
CN113329739B (zh) 2024-06-25
EP3840733A1 (en) 2021-06-30
WO2020041348A1 (en) 2020-02-27
US20240294922A1 (en) 2024-09-05
US20210317462A1 (en) 2021-10-14
JP7476199B2 (ja) 2024-04-30
EP3840733A4 (en) 2022-07-20
US11939582B2 (en) 2024-03-26
AU2019325255A1 (en) 2021-04-15
BR112021003224A2 (pt) 2021-07-20

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