JP2024506882A - 化学修飾された低分子活性化ｒｎａ - Google Patents

化学修飾された低分子活性化ｒｎａ Download PDF

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JP2024506882A
JP2024506882A JP2023547506A JP2023547506A JP2024506882A JP 2024506882 A JP2024506882 A JP 2024506882A JP 2023547506 A JP2023547506 A JP 2023547506A JP 2023547506 A JP2023547506 A JP 2023547506A JP 2024506882 A JP2024506882 A JP 2024506882A
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rag1
seq
small molecule
nucleotides
rna
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JP2024506882A5 (enExample
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ロンチョン リ，
ムーリン カン，
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ラクティゲン セラピューティクス
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/407—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with other heterocyclic ring systems, e.g. ketorolac, physostigmine
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/30—Chemical structure
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023547506A 2021-02-07 2022-01-28 化学修飾された低分子活性化ｒｎａ Pending JP2024506882A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN202110175652 2021-02-07
CN202110175652.9 2021-02-07
CN2021075958 2021-02-08
CNPCT/CN2021/075958 2021-02-08
PCT/CN2022/074635 WO2022166814A1 (zh) 2021-02-07 2022-01-28 一种化学修饰的小激活rna

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JP2024506882A true JP2024506882A (ja) 2024-02-15
JP2024506882A5 JP2024506882A5 (enExample) 2025-02-04

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JP2023547506A Pending JP2024506882A (ja) 2021-02-07 2022-01-28 化学修飾された低分子活性化ｒｎａ

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US (1) US20240301409A1 (enExample)
EP (1) EP4289952A4 (enExample)
JP (1) JP2024506882A (enExample)
KR (1) KR20230142774A (enExample)
CN (1) CN116829716A (enExample)
WO (1) WO2022166814A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025021186A1 (en) * 2023-07-27 2025-01-30 Ractigen Therapeutics Glycerol nucleic acid modified oligonucleotide agents and use thereof
WO2025224036A1 (en) * 2024-04-22 2025-10-30 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018516545A (ja) * 2015-04-22 2018-06-28 ミナ セラピューティクス リミテッド Ｃ／ＥＢＰアルファｓａＲＮＡ組成物および使用方法
WO2019048631A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
WO2019196887A1 (zh) * 2018-04-10 2019-10-17 中美瑞康核酸技术(南通)研究院有限公司 一种新型小激活rna

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1871426B1 (en) * 2005-04-15 2017-06-07 The Regents of The University of California Small activating rna molecules and their use
US9045751B2 (en) * 2010-04-28 2015-06-02 The Regents Of The University Of California Modified small activating RNA molecules and methods of use
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
SI3071696T1 (sl) * 2013-11-22 2019-11-29 Mina Therapeutics Ltd C/EBP alfa kratko delujoči RNA sestavki in postopki uporabe
CN109563511A (zh) 2016-06-30 2019-04-02 阿布特斯生物制药公司 用于递送信使rna的组合物和方法
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
US20200208152A1 (en) * 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized sarna compositions and methods of use
WO2019196883A1 (zh) * 2018-04-10 2019-10-17 中美瑞康核酸技术(南通)研究院有限公司 一种激活p21基因表达的方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018516545A (ja) * 2015-04-22 2018-06-28 ミナ セラピューティクス リミテッド Ｃ／ＥＢＰアルファｓａＲＮＡ組成物および使用方法
WO2019048631A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
WO2019196887A1 (zh) * 2018-04-10 2019-10-17 中美瑞康核酸技术(南通)研究院有限公司 一种新型小激活rna

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 1, no. 3, JPN6025049422, 2012, pages 1 - 12, ISSN: 0005744024 *

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WO2022166814A1 (zh) 2022-08-11
EP4289952A1 (en) 2023-12-13
CN116829716A (zh) 2023-09-29
KR20230142774A (ko) 2023-10-11
US20240301409A1 (en) 2024-09-12
EP4289952A4 (en) 2025-04-16

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