JP7687685B2 - 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド - Google Patents

組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド Download PDF

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JP7687685B2
JP7687685B2 JP2021555491A JP2021555491A JP7687685B2 JP 7687685 B2 JP7687685 B2 JP 7687685B2 JP 2021555491 A JP2021555491 A JP 2021555491A JP 2021555491 A JP2021555491 A JP 2021555491A JP 7687685 B2 JP7687685 B2 JP 7687685B2
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rna
complementary
dsrna molecule
nucleotides
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コボロバ，アナスタシア
ファーガソン，シャンタル
ロガエフ，エフゲニー
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University of Massachusetts Amherst
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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
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021555491A 2019-03-15 2020-03-13 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド Active JP7687685B2 (ja)

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JP2025081846A JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

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US201962819189P 2019-03-15 2019-03-15
US62/819,189 2019-03-15
US201962864797P 2019-06-21 2019-06-21
US62/864,797 2019-06-21
US201962951441P 2019-12-20 2019-12-20
US62/951,441 2019-12-20
PCT/US2020/022748 WO2020190768A1 (en) 2019-03-15 2020-03-13 Oligonucleotides for tissue specific apoe modulation

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JP2022525208A JP2022525208A (ja) 2022-05-11
JPWO2020190768A5 JPWO2020190768A5 (https=) 2023-03-17
JP2022525208A5 JP2022525208A5 (https=) 2023-03-17
JP7687685B2 true JP7687685B2 (ja) 2025-06-03

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JP2025081846A Pending JP2025156319A (ja) 2019-03-15 2025-05-15 組織特異的なａｐｏｅ調節のためのオリゴヌクレオチド

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US (1) US20200362341A1 (https=)
EP (1) EP3937951A4 (https=)
JP (2) JP7687685B2 (https=)
CN (1) CN113811311A (https=)
AU (1) AU2020239987A1 (https=)
CA (1) CA3133241A1 (https=)
WO (1) WO2020190768A1 (https=)

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JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
JP2022545118A (ja) * 2019-08-23 2022-10-25 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
KR20230018377A (ko) * 2020-04-27 2023-02-07 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 e (apoe) irna 제제 조성물 및 이의 사용 방법
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CN114716518A (zh) * 2021-01-06 2022-07-08 圣诺制药公司 一种能够抑制pcsk9表达的分子构造及药物组合物
WO2022187617A1 (en) * 2021-03-05 2022-09-09 Alnylam Pharmaceuticals, Inc. Dosing of sirna compounds to the cisterna magna
WO2022204430A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
JP2024523466A (ja) * 2021-06-22 2024-06-28 ユニバーシティー オブ マサチューセッツ ＩＦＮ－γシグナル伝達経路の調節のためのオリゴヌクレオチド
WO2023086777A1 (en) * 2021-11-09 2023-05-19 Children's National Medical Center Targeting apoe enhances t-cell killing in cancer immunotherapy
US20240173420A1 (en) * 2022-09-30 2024-05-30 University Of Massachusetts Ocular delivery of oligonucleotides
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WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna
US20250353873A1 (en) * 2024-05-17 2025-11-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apoe expression
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Givosiran的制备方法
WO2026057066A1 (zh) * 2024-09-14 2026-03-19 瑞典瑞博科尔制药公司 寡核苷酸、寡核苷酸缀合物及组合物和用途
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JP2017534255A (ja) 2014-09-12 2017-11-24 ホワイトヘッド インスティテュート フォー バイオメディカル リサーチ アポリポタンパク質ｅを発現する細胞及びその使用
JP2018513986A (ja) 2015-04-10 2018-05-31 キョンブク ナショナル ユニバーシティ ホスピタル コレステロールの蓄積による疾患診断用マーカー組成物及びその利用
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AU2020239987A1 (en) 2021-11-04
CA3133241A1 (en) 2020-09-24
JP2025156319A (ja) 2025-10-14
US20200362341A1 (en) 2020-11-19
JP2022525208A (ja) 2022-05-11
CN113811311A (zh) 2021-12-17
WO2020190768A1 (en) 2020-09-24
EP3937951A1 (en) 2022-01-19
EP3937951A4 (en) 2023-04-05

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