UY38503A - Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos - Google Patents

Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos

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Publication number
UY38503A
UY38503A UY0001038503A UY38503A UY38503A UY 38503 A UY38503 A UY 38503A UY 0001038503 A UY0001038503 A UY 0001038503A UY 38503 A UY38503 A UY 38503A UY 38503 A UY38503 A UY 38503A
Authority
UY
Uruguay
Prior art keywords
methods
constructs
pnpla3
expression
arni
Prior art date
Application number
UY0001038503A
Other languages
English (en)
Inventor
Ingrid Rulifson
K Murray Justin
Michael Ollmann
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 UY38503A publication Critical patent/UY38503A/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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)
    • 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 N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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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    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
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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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    • C12N2310/30—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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (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 hígado graso no alcohólico (NAFLD).
UY0001038503A 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos UY38503A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862777714P 2018-12-10 2018-12-10

Publications (1)

Publication Number Publication Date
UY38503A true UY38503A (es) 2020-06-30

Family

ID=69024749

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001038503A UY38503A (es) 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos

Country Status (21)

Country Link
US (1) US20220017906A1 (es)
EP (1) EP3894555A2 (es)
JP (1) JP2022511550A (es)
KR (1) KR20210102932A (es)
CN (1) CN113166761A (es)
AR (1) AR117297A1 (es)
AU (1) AU2019396419A1 (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 (1) MX2021006784A (es)
PE (1) PE20211225A1 (es)
SG (1) SG11202105711SA (es)
TW (1) TW202039843A (es)
UY (1) UY38503A (es)
WO (1) WO2020123508A2 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202300645A (zh) * 2021-04-14 2023-01-01 美商戴瑟納製藥股份有限公司 用於調節ｐｎｐｌａ３表現之組合物及方法
TW202317762A (zh) * 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）的ｉＲＮＡ組成物及其使用方法
CA3230222A1 (en) * 2021-09-01 2023-03-09 Leonid Beigelman Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds

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Publication number Publication date
WO2020123508A2 (en) 2020-06-18
TW202039843A (zh) 2020-11-01
WO2020123508A3 (en) 2020-08-13
EA202191629A1 (ru) 2021-09-03
CA3121510A1 (en) 2020-06-18
PE20211225A1 (es) 2021-07-06
BR112021011061A2 (pt) 2021-08-31
KR20210102932A (ko) 2021-08-20
US20220017906A1 (en) 2022-01-20
JOP20210142A1 (ar) 2023-01-30
AU2019396419A1 (en) 2021-06-17
EP3894555A2 (en) 2021-10-20
SG11202105711SA (en) 2021-06-29
CR20210371A (es) 2021-08-27
IL283473A (en) 2021-07-29
MX2021006784A (es) 2021-07-15
CL2021001489A1 (es) 2022-01-07
JP2022511550A (ja) 2022-01-31
AR117297A1 (es) 2021-07-28
CN113166761A (zh) 2021-07-23
CO2021008997A2 (es) 2021-07-30

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