CN117677697A - 靶向MARC1基因的RNAi制剂及其用途 - Google Patents

靶向MARC1基因的RNAi制剂及其用途 Download PDF

Info

Publication number
CN117677697A
CN117677697A CN202280048413.XA CN202280048413A CN117677697A CN 117677697 A CN117677697 A CN 117677697A CN 202280048413 A CN202280048413 A CN 202280048413A CN 117677697 A CN117677697 A CN 117677697A
Authority
CN
China
Prior art keywords
seq
marc1
group
antisense strand
olx
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202280048413.XA
Other languages
English (en)
Chinese (zh)
Inventor
洪善宇
朴峻贤
崔正容
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Olix Pharmaceuticals Inc
Original Assignee
Olix Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Olix Pharmaceuticals Inc filed Critical Olix Pharmaceuticals Inc
Publication of CN117677697A publication Critical patent/CN117677697A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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)
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/32—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

Landscapes

  • 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)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (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)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280048413.XA 2021-07-08 2022-07-08 靶向MARC1基因的RNAi制剂及其用途 Pending CN117677697A (zh)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CN117677697A true CN117677697A (zh) 2024-03-08

Family

ID=84801791

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280048413.XA Pending CN117677697A (zh) 2021-07-08 2022-07-08 靶向MARC1基因的RNAi制剂及其用途

Country Status (8)

Country Link
US (1) US20250179498A1 (enExample)
EP (1) EP4368717A1 (enExample)
JP (1) 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
KR20250137186A (ko) * 2023-03-08 2025-09-17 올릭스 주식회사 비만의 예방 또는 치료용 RNAi 제제 및 이를 이용한 병용 요법

Family Cites Families (9)

* 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
EA038792B1 (ru) * 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US10835581B2 (en) * 2017-11-28 2020-11-17 University of Pittsburgh—of the Commonwealth System of Higher Education Method of treating insulin resistance
WO2020123410A1 (en) * 2018-12-10 2020-06-18 Amgen Inc. CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF
WO2020154567A1 (en) * 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver
US12215382B2 (en) * 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US20230183707A1 (en) * 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
KR20230046319A (ko) * 2020-08-13 2023-04-05 암젠 인크 MARC1 발현을 억제하기 위한 RNAi 작제물 및 방법
EP4347820A1 (en) * 2021-05-28 2024-04-10 Novo Nordisk A/S Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (marc1) expression

Also Published As

Publication number Publication date
CA3225054A1 (en) 2023-02-12
KR20230010593A (ko) 2023-01-19
EP4368717A1 (en) 2024-05-15
US20250179498A1 (en) 2025-06-05
JP7853399B2 (ja) 2026-04-28
AU2022308807B2 (en) 2026-02-12
WO2023282704A1 (ko) 2023-01-12
JP2024525078A (ja) 2024-07-09
AU2022308807A1 (en) 2024-02-01

Similar Documents

Publication Publication Date Title
JP6440592B2 (ja) アンジオポエチン様３発現の調節
KR101697482B1 (ko) 진단 및 치료 목적을 위한 마이크로ｒｎａ 및 다운스트림 타겟
JP2025011138A (ja) アンジオテンシノーゲンの発現を調節するための化合物及び方法
CN103476931A (zh) Mir-34的合成模拟物
EP3210611A2 (en) Methods of treating vascular inflammatory disorders
US20130195858A1 (en) Treatment of b-cell lymphoma with microrna
CN118773191A (zh) 靶向LPA基因的RNAi剂及其用途
US7968524B2 (en) Methods of enhancing long term memory formation by inhibition of Gpr12
CN117677697A (zh) 靶向MARC1基因的RNAi制剂及其用途
JP2023550906A (ja) 肝障害の治療に使用するための阻害剤
EP4634382A1 (en) Rnai-based therapies targeting claudin-1 for the treatment and prevention of fibrotic diseases
CN116392500B (zh) microRNA及其在诊断和治疗中的用途
WO2022270071A1 (ja) 肝線維化抑制のための剤及び医薬組成物
CN106795556A (zh) VGSC β3 蛋白质用于癌症预防、治疗及诊断检测的靶点
TW202526015A (zh) 靶向ＬＰＡ基因的ＲＮＡｉ劑及其用途
WO2023234410A1 (ja) 心筋細胞死の抑制剤及び心筋障害又は心不全の予防又は治療剤
CN121628899A (zh) 靶向APOC3基因的RNAi剂及其用途
CN119101680A (zh) 多核苷酸、药物组合物及其用途
CN120210192A (zh) Hsd17b13表达抑制物及其用途
JP2023108978A (ja) ＨＢＶ由来ｃｃｃＤＮＡの抑制剤、抑制方法、ＨＢＶ感染症用医療組成物、およびＨＢＶ感染症の治療方法
WO2024166110A1 (en) Targeted reduction of cd59 for use in treating disease
CN121846123A (zh) 多核苷酸序列的用途
CN118256500A (zh) 一种靶向FASN基因的siRNA、载体复合物及其应用
CN101579351A (zh) Acl在筛选防治肥胖相关代谢紊乱疾病的药物中的应用
RS52904B (sr) Mikrornk (mirnk) mir-21 za dijagnozu i terapeutske svrhe

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination