CR20210371A - Constructos de arni para inhibir la expresión de pnpla3 - Google Patents

Constructos de arni para inhibir la expresión de pnpla3

Info

Publication number
CR20210371A
CR20210371A CR20210371A CR20210371A CR20210371A CR 20210371 A CR20210371 A CR 20210371A CR 20210371 A CR20210371 A CR 20210371A CR 20210371 A CR20210371 A CR 20210371A CR 20210371 A CR20210371 A CR 20210371A
Authority
CR
Costa Rica
Prior art keywords
expression
constructs
pnpla3
arni
inhibit
Prior art date
Application number
CR20210371A
Other languages
English (en)
Inventor
Michael Ollmann
Ingrid Rulifson
Justin K Murray
Oliver Homann
Original Assignee
Amgen 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 Amgen Inc filed Critical Amgen Inc
Publication of CR20210371A publication Critical patent/CR20210371A/es

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    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • C12N9/20—Triglyceride splitting, e.g. by means of lipase
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01051—1-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01003—Triacylglycerol lipase (3.1.1.3)
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a constructos de ARNi para reducir la expresión del gen de PNPLA3. Se describen además métodos de uso de tales constructos de ARNi para tratar o prevenir la enfermedad hepática, enfermedad del 5 hígado graso no alcohólico (NAFLD).
CR20210371A 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresión de pnpla3 CR20210371A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862777714P 2018-12-10 2018-12-10
PCT/US2019/065481 WO2020123508A2 (en) 2018-12-10 2019-12-10 Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

Publications (1)

Publication Number Publication Date
CR20210371A true CR20210371A (es) 2021-08-27

Family

ID=69024749

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20210371A CR20210371A (es) 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresión de pnpla3

Country Status (22)

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US (2) US20220017906A1 (es)
EP (1) EP3894555A2 (es)
JP (2) JP2022511550A (es)
KR (1) KR20210102932A (es)
CN (2) CN113166761B (es)
AR (1) AR117297A1 (es)
AU (1) AU2019396419B2 (es)
BR (1) BR112021011061A2 (es)
CA (1) CA3121510A1 (es)
CL (1) CL2021001489A1 (es)
CO (1) CO2021008997A2 (es)
CR (1) CR20210371A (es)
EA (1) EA202191629A1 (es)
IL (1) IL283473A (es)
JO (1) JOP20210142A1 (es)
MX (2) MX2021006784A (es)
PE (1) PE20211225A1 (es)
PH (1) PH12021551326A1 (es)
SG (1) SG11202105711SA (es)
TW (1) TW202039843A (es)
UY (1) UY38503A (es)
WO (1) WO2020123508A2 (es)

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JP2024522996A (ja) * 2021-06-02 2024-06-25 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 パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
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