JP2024533371A - 修飾された短い干渉核酸(siNA)分子及びそれらの使用 - Google Patents

修飾された短い干渉核酸(siNA)分子及びそれらの使用 Download PDF

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JP2024533371A
JP2024533371A JP2024515376A JP2024515376A JP2024533371A JP 2024533371 A JP2024533371 A JP 2024533371A JP 2024515376 A JP2024515376 A JP 2024515376A JP 2024515376 A JP2024515376 A JP 2024515376A JP 2024533371 A JP2024533371 A JP 2024533371A
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nucleotide
nucleotides
sina
nucleotide sequence
antisense strand
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JP2024533371A5 (https=
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ベイジェルマン,レオニド
クマル ラジュワンシ,ヴィヴェク
ホスバッハ,マルクス
ケー. パンディ,ラジェンドラ
ホン,ジン
エルテプ,ラクスマン
マルティネス モンテロ,サウル
コスタ,エヌ.ティラニ エス. デ
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アリゴス セラピューティクス,インコーポレーテッド
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
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    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • C12N15/113Non-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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates

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JP2024515376A 2021-09-08 2022-09-07 修飾された短い干渉核酸(siNA)分子及びそれらの使用 Pending JP2024533371A (ja)

Applications Claiming Priority (3)

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US202163241935P 2021-09-08 2021-09-08
US63/241,935 2021-09-08
PCT/US2022/042808 WO2023039005A2 (en) 2021-09-08 2022-09-07 Modified short interfering nucleic acid (sina) molecules and uses thereof

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JP2024533371A true JP2024533371A (ja) 2024-09-12
JP2024533371A5 JP2024533371A5 (https=) 2025-09-16

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US (2) US12454691B2 (https=)
EP (1) EP4399217A2 (https=)
JP (1) JP2024533371A (https=)
KR (1) KR20240099159A (https=)
CN (1) CN118201942A (https=)
AU (1) AU2022344131A1 (https=)
CA (1) CA3231695A1 (https=)
TW (1) TW202320809A (https=)
WO (1) WO2023039005A2 (https=)

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CN118271378A (zh) * 2022-12-29 2024-07-02 中国科学院深圳先进技术研究院 一种糖环修饰的核苷亚磷酰胺单体及其制备方法和应用
CN119836430A (zh) * 2023-05-12 2025-04-15 北京炫景瑞医药科技有限公司 修饰的核苷类似物及其用途与含核苷酸类似物的双链寡核苷酸及其用途
AU2024318590A1 (en) * 2023-07-31 2026-02-26 Basecure Therapeutics Inc. Conjugates of n-acetyl-galactosamine (galnac) and oligonucleotides and uses thereof
CN117534717A (zh) * 2024-01-09 2024-02-09 凯莱英生命科学技术(天津)有限公司 5′-(e)-乙烯基磷酸酯的合成方法

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RU2487716C2 (ru) * 2007-10-03 2013-07-20 Кварк Фармасьютикалс, Инк. Новые структуры малых интерферирующих рнк (sirna)
WO2011094953A1 (en) 2010-02-08 2011-08-11 F. Hoffmann-La Roche Ag Compounds for the treatment and prevention of influenza
WO2011139699A2 (en) * 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
AU2014302711A1 (en) 2013-06-26 2015-12-10 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
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HUE059718T2 (hu) 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
EP3735252A4 (en) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
WO2019217397A2 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
PE20211783A1 (es) 2018-11-08 2021-09-08 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigenos y metodos
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US20240352464A1 (en) * 2023-03-07 2024-10-24 Aligos Therapeutics, Inc. Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof

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US12454691B2 (en) 2025-10-28
CA3231695A1 (en) 2023-03-16
US20230159929A1 (en) 2023-05-25
US20260002157A1 (en) 2026-01-01
KR20240099159A (ko) 2024-06-28
CN118201942A (zh) 2024-06-14
WO2023039005A2 (en) 2023-03-16
TW202320809A (zh) 2023-06-01
AU2022344131A1 (en) 2024-03-28
EP4399217A2 (en) 2024-07-17
WO2023039005A3 (en) 2023-07-20

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