JP7853399B2 - ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途 - Google Patents

ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

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Publication number
JP7853399B2
JP7853399B2 JP2024500173A JP2024500173A JP7853399B2 JP 7853399 B2 JP7853399 B2 JP 7853399B2 JP 2024500173 A JP2024500173 A JP 2024500173A JP 2024500173 A JP2024500173 A JP 2024500173A JP 7853399 B2 JP7853399 B2 JP 7853399B2
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marc1
rnai
olx
antisense strand
sense strand
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JP2024525078A (ja
JP2024525078A5 (enExample
Inventor
ウ ホン，ソン
ヒョン パク，ジュン
ヨン チェ，チョン
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オリックス ファーマシューティカルズ，インコーポレーテッド
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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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y107/00—Oxidoreductases acting on other nitrogenous compounds as donors (1.7)
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    • 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
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    • C12N2310/11—Antisense
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    • 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
    • C12N2310/10—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
    • 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
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024500173A 2021-07-08 2022-07-08 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途 Active JP7853399B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026067672A JP2026120732A (ja) 2021-07-08 2026-04-16 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
KR10-2021-0089864 2021-07-08
KR20210089864 2021-07-08
PCT/KR2022/009984 WO2023282704A1 (ko) 2021-07-08 2022-07-08 Marc1 유전자를 표적으로 하는 rnai 제제 및 이의 용도

Related Child Applications (1)

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JP2026067672A Division JP2026120732A (ja) 2021-07-08 2026-04-16 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

Publications (3)

Publication Number Publication Date
JP2024525078A JP2024525078A (ja) 2024-07-09
JP2024525078A5 JP2024525078A5 (enExample) 2026-01-20
JP7853399B2 true JP7853399B2 (ja) 2026-04-28

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JP2024500173A Active JP7853399B2 (ja) 2021-07-08 2022-07-08 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途
JP2026067672A Pending JP2026120732A (ja) 2021-07-08 2026-04-16 ＭＡＲＣ１遺伝子を標的にするＲＮＡｉ製剤及びその用途

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Country Status (8)

Country Link
US (1) US20250179498A1 (enExample)
EP (1) EP4368717A4 (enExample)
JP (2) JP7853399B2 (enExample)
KR (1) KR20230010593A (enExample)
CN (1) CN117677697A (enExample)
AU (1) AU2022308807B2 (enExample)
CA (1) CA3225054A1 (enExample)
WO (1) WO2023282704A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024186171A1 (ko) * 2023-03-08 2024-09-12 올릭스 주식회사 비만의 예방 또는 치료용 RNAi 제제 및 이를 이용한 병용 요법

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520312A (ja) 2013-05-22 2016-07-14 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法
US20200241005A1 (en) 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver
US20210038698A1 (en) 2017-11-28 2021-02-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Method of Treating Insulin Resistance
US20210262022A1 (en) 2019-03-01 2021-08-26 The General Hospital Corporation Liver protective marc variants and uses thereof
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023537943A (ja) 2020-08-13 2023-09-06 アムジェン インコーポレイテッド ＭＡＲＣ１発現を阻害するためのＲＮＡｉコンストラクト及び方法
JP2024508069A (ja) 2021-05-28 2024-02-22 ノヴォ ノルディスク アー／エス ミトコンドリアアミドキシム還元成分１（ｍａｒｃ１）発現を阻害するための組成物および方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR090905A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
CN110799192A (zh) * 2017-03-27 2020-02-14 加利福尼亚大学董事会 治疗癌症的组合物和方法
SG11202105900UA (en) * 2018-12-10 2021-07-29 Amgen Inc CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520312A (ja) 2013-05-22 2016-07-14 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＳＥＲＰＩＮＡ１ ｉＲＮＡ組成物およびその使用方法
US20210038698A1 (en) 2017-11-28 2021-02-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Method of Treating Insulin Resistance
US20200241005A1 (en) 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver
US20210262022A1 (en) 2019-03-01 2021-08-26 The General Hospital Corporation Liver protective marc variants and uses thereof
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
JP2023537943A (ja) 2020-08-13 2023-09-06 アムジェン インコーポレイテッド ＭＡＲＣ１発現を阻害するためのＲＮＡｉコンストラクト及び方法
JP2024508069A (ja) 2021-05-28 2024-02-22 ノヴォ ノルディスク アー／エス ミトコンドリアアミドキシム還元成分１（ｍａｒｃ１）発現を阻害するための組成物および方法

Also Published As

Publication number Publication date
AU2022308807A1 (en) 2024-02-01
KR20230010593A (ko) 2023-01-19
CN117677697A (zh) 2024-03-08
WO2023282704A1 (ko) 2023-01-12
JP2024525078A (ja) 2024-07-09
AU2022308807B2 (en) 2026-02-12
CA3225054A1 (en) 2023-02-12
EP4368717A1 (en) 2024-05-15
US20250179498A1 (en) 2025-06-05
JP2026120732A (ja) 2026-07-22
EP4368717A4 (en) 2026-07-01

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