JP7661228B2 - オリゴヌクレオチド組成物及びその方法 - Google Patents

オリゴヌクレオチド組成物及びその方法 Download PDF

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JP7661228B2
JP7661228B2 JP2021541152A JP2021541152A JP7661228B2 JP 7661228 B2 JP7661228 B2 JP 7661228B2 JP 2021541152 A JP2021541152 A JP 2021541152A JP 2021541152 A JP2021541152 A JP 2021541152A JP 7661228 B2 JP7661228 B2 JP 7661228B2
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oligonucleotide
htt
oligonucleotides
composition
wing
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JP2022519019A (ja
JPWO2020160336A5 (https=
JP2022519019A5 (https=
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ブラウン，ジェフリー，マシュー
シュー－メイ バーコヴィッチ，シャウナ
直樹 岩本
ヴァルギーズ，チャンドラ
エム． アクリル，キディスト
デイビッド フランク－カメネツキー，マリア
パーリー ブラウン，ダンカン
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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
    • 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
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    • C12N2320/34—Allele or polymorphism specific uses

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021541152A 2019-02-01 2020-01-30 オリゴヌクレオチド組成物及びその方法 Active JP7661228B2 (ja)

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JP2025060960A JP2025107175A (ja) 2019-02-01 2025-04-02 オリゴヌクレオチド組成物及びその方法

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US201962800409P 2019-02-01 2019-02-01
US62/800,409 2019-02-01
US201962911335P 2019-10-06 2019-10-06
US62/911,335 2019-10-06
PCT/US2020/015971 WO2020160336A1 (en) 2019-02-01 2020-01-30 Oligonucleotide compositions and methods thereof

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JP2022519019A JP2022519019A (ja) 2022-03-18
JPWO2020160336A5 JPWO2020160336A5 (https=) 2023-02-08
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JP7661228B2 true JP7661228B2 (ja) 2025-04-14

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EP (1) EP3917497A4 (https=)
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KR (1) KR20210121199A (https=)
CN (1) CN113423385A (https=)
AU (2) AU2020216186C1 (https=)
BR (1) BR112021014940A2 (https=)
CA (1) CA3126845A1 (https=)
IL (1) IL284882A (https=)
MA (1) MA54875A (https=)
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SG (1) SG11202107318YA (https=)
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