JP2022515744A - Ｋｃｎｔ１関連障害の治療のための組成物及び方法 - Google Patents

Ｋｃｎｔ１関連障害の治療のための組成物及び方法 Download PDF

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JP2022515744A
JP2022515744A JP2021535662A JP2021535662A JP2022515744A JP 2022515744 A JP2022515744 A JP 2022515744A JP 2021535662 A JP2021535662 A JP 2021535662A JP 2021535662 A JP2021535662 A JP 2021535662A JP 2022515744 A JP2022515744 A JP 2022515744A
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oligonucleotide
nucleoside
compound
bond
kcnt1
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JP2022515744A5 (enExample
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スティーブン ペトロー，
マイケル クリストファー マシュー カーリグ，
キラン レディ，
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プラクシス プレシジョン メディシンズ， インコーポレイテッド
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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
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—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
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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 ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • 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)
JP2021535662A 2018-12-20 2019-12-20 Ｋｃｎｔ１関連障害の治療のための組成物及び方法 Withdrawn JP2022515744A (ja)

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

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JP2022515744A true JP2022515744A (ja) 2022-02-22
JP2022515744A5 JP2022515744A5 (enExample) 2022-12-27

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

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JP2023533153A (ja) * 2020-06-25 2023-08-02 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｋｃｎｔ１の発現を低減するための化合物及び方法
JP2024537991A (ja) 2021-10-14 2024-10-18 アーセナル バイオサイエンシズ インコーポレイテッド 共発現されるｓｈＲＮＡと論理ゲートシステムとを有する免疫細胞
EP4441229A1 (en) * 2021-12-01 2024-10-09 Atalanta Therapeutics, Inc. Compositions and methods for treatment of epilepsies
PE20251319A1 (es) 2022-09-13 2025-05-16 Arsenal Biosciences Inc Celulas inmunitarias que coexpresan arnhc de tgfbr
EP4587570A2 (en) 2022-09-16 2025-07-23 Arsenal Biosciences, Inc. Immune cells with combination gene perturbations
IL322949A (en) 2023-03-03 2025-10-01 Arsenal Biosciences Inc Systems targeting PSMA and CA9
US20250297255A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Systems targeting tmprss4 and slc34a2

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