JP7817791B2 - 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド - Google Patents

遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

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JP7817791B2
JP7817791B2 JP2018566905A JP2018566905A JP7817791B2 JP 7817791 B2 JP7817791 B2 JP 7817791B2 JP 2018566905 A JP2018566905 A JP 2018566905A JP 2018566905 A JP2018566905 A JP 2018566905A JP 7817791 B2 JP7817791 B2 JP 7817791B2
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oligonucleotide
exon
monomer
seq
bna
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JP2019522972A (ja
JP2019522972A5 (https=
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ドイテコム， ジュディス クリスティーナ セオドラ ファン
ヴィッサー， ピーター クリスチャン デ
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バイオマリン テクノロジーズ ベー．フェー．
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Priority to JP2024037011A priority patent/JP2024102041A/ja
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    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2018566905A 2016-07-05 2017-07-05 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド Active JP7817791B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022161723A JP2023024968A (ja) 2016-07-05 2022-10-06 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
JP2024037011A JP2024102041A (ja) 2016-07-05 2024-03-11 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16177973 2016-07-05
EP16177973.1 2016-07-05
PCT/EP2017/066845 WO2018007475A1 (en) 2016-07-05 2017-07-05 Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders

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JP2022161723A Division JP2023024968A (ja) 2016-07-05 2022-10-06 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

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JP2019522972A JP2019522972A (ja) 2019-08-22
JP2019522972A5 JP2019522972A5 (https=) 2024-07-05
JPWO2018007475A5 JPWO2018007475A5 (https=) 2024-07-05
JP7817791B2 true JP7817791B2 (ja) 2026-02-19

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JP2018566905A Active JP7817791B2 (ja) 2016-07-05 2017-07-05 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
JP2022161723A Pending JP2023024968A (ja) 2016-07-05 2022-10-06 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
JP2024037011A Pending JP2024102041A (ja) 2016-07-05 2024-03-11 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

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JP2022161723A Pending JP2023024968A (ja) 2016-07-05 2022-10-06 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
JP2024037011A Pending JP2024102041A (ja) 2016-07-05 2024-03-11 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

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US (2) US20180028554A1 (https=)
EP (2) EP4252845A3 (https=)
JP (3) JP7817791B2 (https=)
KR (3) KR102824513B1 (https=)
CN (2) CN121271865A (https=)
AU (2) AU2017291960B2 (https=)
BR (1) BR112019000061A2 (https=)
CA (2) CA3029772A1 (https=)
IL (3) IL301091A (https=)
MX (2) MX2018016253A (https=)
NZ (2) NZ789904A (https=)
RU (2) RU2022107926A (https=)
WO (1) WO2018007475A1 (https=)

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