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PE20211225A1 - Constructos de arni para inhibir la expresion de pnpla3 - Google Patents

Constructos de arni para inhibir la expresion de pnpla3

Info

Publication number
PE20211225A1
PE20211225A1 PE2021000850A PE2021000850A PE20211225A1 PE 20211225 A1 PE20211225 A1 PE 20211225A1 PE 2021000850 A PE2021000850 A PE 2021000850A PE 2021000850 A PE2021000850 A PE 2021000850A PE 20211225 A1 PE20211225 A1 PE 20211225A1
Authority
PE
Peru
Prior art keywords
expression
constructs
pnpla3
arni
inhibit
Prior art date
Application number
PE2021000850A
Other languages
English (en)
Inventor
Ingrid Rulifson
Justin K Murray
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 PE20211225A1 publication Critical patent/PE20211225A1/es

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    • C—CHEMISTRY; METALLURGY
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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)
    • C—CHEMISTRY; METALLURGY
    • 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.
    • 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
    • C12N2310/31—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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    • C12N2310/30—Chemical structure
    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • 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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invencion se refiere a constructos de ARNi para reducir la expresion del gen de PNPLA3. Se describen ademas metodos de uso de tales constructos de ARNi para tratar o prevenir la enfermedad hepatica, enfermedad del higado graso no alcoholico (NAFLD)
PE2021000850A 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresion de pnpla3 PE20211225A1 (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
PE20211225A1 true PE20211225A1 (es) 2021-07-06

Family

ID=69024749

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021000850A PE20211225A1 (es) 2018-12-10 2019-12-10 Constructos de arni para inhibir la expresion de pnpla3

Country Status (22)

Country Link
US (1) US20220017906A1 (es)
EP (1) EP3894555A2 (es)
JP (2) 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)
PH (1) PH12021551326A1 (es)
SG (1) SG11202105711SA (es)
TW (1) TW202039843A (es)
UY (1) UY38503A (es)
WO (1) WO2020123508A2 (es)

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AR113490A1 (es) * 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
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US12084662B2 (en) 2021-04-14 2024-09-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating PNPLA3 expression
WO2022256395A1 (en) * 2021-06-02 2022-12-08 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
KR20240112993A (ko) * 2021-09-01 2024-07-19 알리고스 테라퓨틱스 인코포레이티드 Pnpla3-표적화 짧은 간섭 rna(sirna) 분자 및 이의 용도
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024120412A1 (en) * 2022-12-07 2024-06-13 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3)
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IL283473A (en) 2021-07-29
US20220017906A1 (en) 2022-01-20
BR112021011061A2 (pt) 2021-08-31
CL2021001489A1 (es) 2022-01-07
EA202191629A1 (ru) 2021-09-03
CN113166761A (zh) 2021-07-23
MX2021006784A (es) 2021-07-15
SG11202105711SA (en) 2021-06-29
AR117297A1 (es) 2021-07-28
JP2024105284A (ja) 2024-08-06
TW202039843A (zh) 2020-11-01
EP3894555A2 (en) 2021-10-20
CA3121510A1 (en) 2020-06-18
JP2022511550A (ja) 2022-01-31
UY38503A (es) 2020-06-30
JOP20210142A1 (ar) 2023-01-30
PH12021551326A1 (en) 2022-04-25
CR20210371A (es) 2021-08-27
AU2019396419A1 (en) 2021-06-17
WO2020123508A2 (en) 2020-06-18
CO2021008997A2 (es) 2021-07-30
WO2020123508A3 (en) 2020-08-13
KR20210102932A (ko) 2021-08-20

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