JP2024529930A - 低減されたオフターゲット効果を有する修飾低分子干渉ｒｎａ分子 - Google Patents

低減されたオフターゲット効果を有する修飾低分子干渉ｒｎａ分子 Download PDF

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JP2024529930A
JP2024529930A JP2024503797A JP2024503797A JP2024529930A JP 2024529930 A JP2024529930 A JP 2024529930A JP 2024503797 A JP2024503797 A JP 2024503797A JP 2024503797 A JP2024503797 A JP 2024503797A JP 2024529930 A JP2024529930 A JP 2024529930A
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nucleotides
disease
interfering rna
modified sirna
antisense strand
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JP2024529930A5 (enExample
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チャン，イー－チュン
ヤン，チー－ファン
チェン，ホイ－ユー
ヤン，チャ－チュン
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マイクロバイオ （シャンハイ） カンパニー リミテッド
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
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  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2024503797A 2021-07-22 2022-07-21 低減されたオフターゲット効果を有する修飾低分子干渉ｒｎａ分子 Pending JP2024529930A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CNPCT/CN2021/107862 2021-07-22
CN2021107862 2021-07-22
PCT/CN2022/107028 WO2023001234A1 (en) 2021-07-22 2022-07-21 Modified small interfering rna molecules with reduced off-target effects

Publications (2)

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JP2024529930A true JP2024529930A (ja) 2024-08-14
JP2024529930A5 JP2024529930A5 (enExample) 2025-07-30

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JP2024503797A Pending JP2024529930A (ja) 2021-07-22 2022-07-21 低減されたオフターゲット効果を有する修飾低分子干渉ｒｎａ分子

Country Status (7)

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US (1) US20240425853A1 (enExample)
EP (1) EP4373932A4 (enExample)
JP (1) JP2024529930A (enExample)
KR (1) KR20240042457A (enExample)
CN (1) CN118202046A (enExample)
CA (1) CA3227144A1 (enExample)
WO (1) WO2023001234A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007525169A (ja) * 2003-04-02 2007-09-06 ダーマコン， インコーポレイテッド Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
JP2009522304A (ja) * 2005-12-29 2009-06-11 アルコン リサーチ， リミテッド 眼の障害の治療のためのＨＩＦ１ＡのＲＮＡｉ媒介抑制

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080188430A1 (en) * 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
WO2005035759A2 (en) * 2003-08-20 2005-04-21 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20070218551A1 (en) * 2003-10-02 2007-09-20 Chuan-Yuan Li Novel Sirna-Based Approach to Target the Hif-Alpha Factor for Gene Therapy
EP3121281B1 (en) * 2007-08-27 2021-03-31 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
EP3929293A3 (en) * 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
CN106117289B (zh) * 2016-06-24 2019-02-12 郑州大学 2’-o-moe-3’-h-硫代磷酸酯核苷单体及其合成方法
WO2018098328A1 (en) * 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Modified rna agents with reduced off-target effect
EP3946369A4 (en) * 2019-03-26 2023-10-18 University Of Massachusetts MODIFIED OLIGONUCLEOTIDES WITH INCREASED STABILITY
CN110540990B (zh) * 2019-07-16 2024-08-20 武汉泽智生物医药有限公司 沉默小眼畸形转录因子mRNA表达的siRNA分子

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007525169A (ja) * 2003-04-02 2007-09-06 ダーマコン， インコーポレイテッド Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
JP2009522304A (ja) * 2005-12-29 2009-06-11 アルコン リサーチ， リミテッド 眼の障害の治療のためのＨＩＦ１ＡのＲＮＡｉ媒介抑制

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ACS OMEGA, vol. 2, JPN6026025918, 2017, pages 2055 - 2064, ISSN: 0005892097 *
CHEMBIOCHEM, vol. 21, JPN6026025919, 2020, pages 1304 - 1308, ISSN: 0005892098 *
NAT. COMMUN., vol. Vol. 6:6317, JPN6026025921, 2015, pages 1 - 9, ISSN: 0005892100 *
NUCLEIC ACIDS RES., vol. 32, JPN6026025920, 2004, pages 5991 - 6000, ISSN: 0005892099 *

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WO2023001234A1 (en) 2023-01-26
CA3227144A1 (en) 2023-01-26
WO2023001234A9 (en) 2023-03-02
EP4373932A4 (en) 2026-05-20
US20240425853A1 (en) 2024-12-26
KR20240042457A (ko) 2024-04-02
EP4373932A1 (en) 2024-05-29
CN118202046A (zh) 2024-06-14

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