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PE20210633A1 - CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESION DE PNPLA3 Y METODOS DE USO DE LOS MISMOS - Google Patents

CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESION DE PNPLA3 Y METODOS DE USO DE LOS MISMOS

Info

Publication number
PE20210633A1
PE20210633A1 PE2020000745A PE2020000745A PE20210633A1 PE 20210633 A1 PE20210633 A1 PE 20210633A1 PE 2020000745 A PE2020000745 A PE 2020000745A PE 2020000745 A PE2020000745 A PE 2020000745A PE 20210633 A1 PE20210633 A1 PE 20210633A1
Authority
PE
Peru
Prior art keywords
expression
methods
rnai constructs
pnpla3
inhibit
Prior art date
Application number
PE2020000745A
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 PE20210633A1 publication Critical patent/PE20210633A1/es

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    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • 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
    • 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 N.A.
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth 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)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • 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 Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invencion se refiere a constructos de ARNi para reducir la expresion del gen de PNPLA3. Tambien se describe metodos de uso de tales constructos de ARNi para tratar o prevenir enfermedad hepatica, enfermedad por higado graso no alcoholico (NAFLD)
PE2020000745A 2017-12-12 2018-12-12 CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESION DE PNPLA3 Y METODOS DE USO DE LOS MISMOS PE20210633A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762597841P 2017-12-12 2017-12-12
PCT/US2018/065275 WO2019118638A2 (en) 2017-12-12 2018-12-12 Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

Publications (1)

Publication Number Publication Date
PE20210633A1 true PE20210633A1 (es) 2021-03-23

Family

ID=65139138

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2020000745A PE20210633A1 (es) 2017-12-12 2018-12-12 CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESION DE PNPLA3 Y METODOS DE USO DE LOS MISMOS

Country Status (23)

Country Link
US (1) US20210139912A1 (es)
EP (1) EP3724337A2 (es)
JP (2) JP2021506238A (es)
KR (1) KR20200097299A (es)
CN (1) CN111699257A (es)
AR (1) AR113490A1 (es)
AU (1) AU2018386089A1 (es)
BR (1) BR112020011686A2 (es)
CA (1) CA3084133A1 (es)
CL (2) CL2020001543A1 (es)
CO (1) CO2020008485A2 (es)
CR (1) CR20200304A (es)
EA (1) EA202091437A1 (es)
IL (1) IL275029A (es)
JO (1) JOP20200142A1 (es)
MX (1) MX2020006088A (es)
PE (1) PE20210633A1 (es)
PH (1) PH12020550833A1 (es)
SG (1) SG11202005257UA (es)
TW (1) TW201938792A (es)
UY (1) UY38003A (es)
WO (1) WO2019118638A2 (es)
ZA (1) ZA202003982B (es)

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WO2020123508A2 (en) * 2018-12-10 2020-06-18 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression and methods of use thereof
JP2022552249A (ja) * 2019-10-14 2022-12-15 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
AU2020399636A1 (en) * 2019-12-09 2022-06-02 Amgen Inc. RNAi constructs and methods for inhibiting LPA expression
TW202138559A (zh) * 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法
CA3169272A1 (en) 2020-01-28 2021-08-05 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized pnpla3 locus and methods of use
WO2021195467A2 (en) * 2020-03-26 2021-09-30 Arrowhead Pharmaceuticals, Inc. RNAi AGENTS FOR INHIBITING EXPRESSION OF PNPLA3, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE
BR112023001957A2 (pt) 2020-08-05 2023-04-25 Dicerna Pharmaceuticals Inc Composições e métodos para inibir expressão de lpa
US12084662B2 (en) 2021-04-14 2024-09-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating PNPLA3 expression
KR20240112993A (ko) * 2021-09-01 2024-07-19 알리고스 테라퓨틱스 인코포레이티드 Pnpla3-표적화 짧은 간섭 rna(sirna) 분자 및 이의 용도
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)
WO2024125404A1 (zh) * 2022-12-12 2024-06-20 北京福元医药股份有限公司 用于抑制pnpla3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024182446A2 (en) * 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof

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Also Published As

Publication number Publication date
US20210139912A1 (en) 2021-05-13
JP2021506238A (ja) 2021-02-22
AR113490A1 (es) 2020-05-06
KR20200097299A (ko) 2020-08-18
CN111699257A (zh) 2020-09-22
IL275029A (en) 2020-07-30
WO2019118638A3 (en) 2019-08-15
UY38003A (es) 2019-06-28
CL2020001543A1 (es) 2020-08-28
CL2023002358A1 (es) 2024-02-23
SG11202005257UA (en) 2020-07-29
BR112020011686A2 (pt) 2020-11-24
AU2018386089A1 (en) 2020-06-18
CA3084133A1 (en) 2019-06-20
JOP20200142A1 (ar) 2022-10-30
MX2020006088A (es) 2020-08-24
EA202091437A1 (ru) 2020-12-29
TW201938792A (zh) 2019-10-01
JP2024012386A (ja) 2024-01-30
CO2020008485A2 (es) 2020-07-31
PH12020550833A1 (en) 2021-07-05
EP3724337A2 (en) 2020-10-21
ZA202003982B (en) 2021-07-28
WO2019118638A2 (en) 2019-06-20
CR20200304A (es) 2020-09-04

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