CN116867510A - 用线粒体甘油-3-磷酸酰基转移酶(gpam)抑制剂治疗肝病 - Google Patents

用线粒体甘油-3-磷酸酰基转移酶(gpam)抑制剂治疗肝病 Download PDF

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Publication number
CN116867510A
CN116867510A CN202280016246.0A CN202280016246A CN116867510A CN 116867510 A CN116867510 A CN 116867510A CN 202280016246 A CN202280016246 A CN 202280016246A CN 116867510 A CN116867510 A CN 116867510A
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China
Prior art keywords
seq
complement
gpam
nucleic acid
nucleotide sequence
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CN202280016246.0A
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English (en)
Chinese (zh)
Inventor
L·A·洛塔
N·维尔韦杰
C·奥杜什莱恩
J·马奇尼
A·巴拉斯
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of CN116867510A publication Critical patent/CN116867510A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12Q2600/00—Oligonucleotides characterized by their use
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    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Analytical Chemistry (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
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  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pathology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280016246.0A 2021-02-27 2022-02-25 用线粒体甘油-3-磷酸酰基转移酶(gpam)抑制剂治疗肝病 Pending CN116867510A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163154693P 2021-02-27 2021-02-27
US63/154,693 2021-02-27
PCT/US2022/017899 WO2022182985A1 (en) 2021-02-27 2022-02-25 Treatment of liver disease with mitochondrial glycerol-3-phosphate acyltransferase (gpam) inhibitors

Publications (1)

Publication Number Publication Date
CN116867510A true CN116867510A (zh) 2023-10-10

Family

ID=80786652

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280016246.0A Pending CN116867510A (zh) 2021-02-27 2022-02-25 用线粒体甘油-3-磷酸酰基转移酶(gpam)抑制剂治疗肝病

Country Status (10)

Country Link
US (2) US12215322B2 (https=)
EP (1) EP4297772A1 (https=)
JP (1) JP2024508824A (https=)
KR (1) KR20230151996A (https=)
CN (1) CN116867510A (https=)
AU (1) AU2022227748A1 (https=)
CA (1) CA3210481A1 (https=)
IL (1) IL304543A (https=)
MX (1) MX2023009986A (https=)
WO (1) WO2022182985A1 (https=)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010000656A1 (en) 2008-07-03 2010-01-07 Santaris Pharma A/S Rna antagonist compounds for the inhibition of expression of mitochondrial glycerol-3-phosphate acyltransferase 1 (mtgpat1)
US9149445B2 (en) 2009-07-27 2015-10-06 The Trustees Of Princeton University Inhibition of glycerol-3-phosphate acyltransferase (GPAT) and associated enzymes for treatment of viral infections
US11702394B2 (en) 2018-02-23 2023-07-18 The Board Of Trustees Of The Leland Stanford Junior University Inhibitors of phospholipid synthesis and methods of use
WO2021252649A2 (en) * 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression

Also Published As

Publication number Publication date
EP4297772A1 (en) 2024-01-03
US20220307032A1 (en) 2022-09-29
US12215322B2 (en) 2025-02-04
US20250197866A1 (en) 2025-06-19
MX2023009986A (es) 2023-09-06
AU2022227748A1 (en) 2023-08-10
CA3210481A1 (en) 2022-09-01
IL304543A (en) 2023-09-01
AU2022227748A9 (en) 2024-05-09
KR20230151996A (ko) 2023-11-02
JP2024508824A (ja) 2024-02-28
WO2022182985A1 (en) 2022-09-01

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