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UY38003A - Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas - Google Patents

Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas

Info

Publication number
UY38003A
UY38003A UY0001038003A UY38003A UY38003A UY 38003 A UY38003 A UY 38003A UY 0001038003 A UY0001038003 A UY 0001038003A UY 38003 A UY38003 A UY 38003A UY 38003 A UY38003 A UY 38003A
Authority
UY
Uruguay
Prior art keywords
expression
methods
pnpla3
arni
constructions
Prior art date
Application number
UY0001038003A
Other languages
English (en)
Inventor
Rulifson Ingrid
Justin K Murray
Ollmann Michael
HOMANN Oliver
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 UY38003A publication Critical patent/UY38003A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1025Acyltransferases (2.3)
    • C12N9/1029Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y203/00Acyltransferases (2.3)
    • C12Y203/01Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/010511-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/01Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01003Triacylglycerol lipase (3.1.1.3)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification

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  • 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 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).
UY0001038003A 2017-12-12 2018-12-11 Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas UY38003A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762597841P 2017-12-12 2017-12-12

Publications (1)

Publication Number Publication Date
UY38003A true UY38003A (es) 2019-06-28

Family

ID=65139138

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001038003A UY38003A (es) 2017-12-12 2018-12-11 Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas

Country Status (23)

Country Link
US (2) US20210139912A1 (es)
EP (1) EP3724337A2 (es)
JP (2) JP2021506238A (es)
KR (1) KR20200097299A (es)
CN (1) CN111699257A (es)
AR (1) AR113490A1 (es)
AU (2) 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 (2) 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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EP3283631A1 (en) 2015-04-13 2018-02-21 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
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TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
KR20210102932A (ko) * 2018-12-10 2021-08-20 암젠 인크 Pnpla3 발현을 억제하기 위한 rnai 작제물 및 이의 사용 방법
WO2021074772A1 (en) * 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
US20230078200A1 (en) * 2019-12-09 2023-03-16 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
AU2020404905A1 (en) 2019-12-16 2022-06-23 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
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
KR20220133248A (ko) 2020-01-28 2022-10-04 리제너론 파마슈티칼스 인코포레이티드 인간화 pnpla3 좌위를 포함하는 비-인간 동물 및 사용 방법
IL296718A (en) * 2020-03-26 2022-11-01 Arrowhead Pharmaceuticals Inc RNAi agents for inhibiting expression of pnpla3, their pharmaceutical compositions and methods of use
KR20250077604A (ko) 2020-08-05 2025-05-30 다이서나 파마수이티컬, 인크. Lpa 발현을 저해하기 위한 조성물 및 방법
BR112023021109A2 (pt) 2021-04-14 2023-12-12 Dicerna Pharmaceuticals Inc Composições e métodos para modular a expressão de pnpla3
EP4402262A1 (en) * 2021-09-01 2024-07-24 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
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)
CN117904108A (zh) * 2022-12-12 2024-04-19 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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Publication number Publication date
MX2025010269A (es) 2025-10-01
PE20210633A1 (es) 2021-03-23
IL275029A (en) 2020-07-30
CA3084133A1 (en) 2019-06-20
US20250154512A1 (en) 2025-05-15
AU2025203098A1 (en) 2025-05-22
JOP20200142A1 (ar) 2022-10-30
WO2019118638A2 (en) 2019-06-20
CL2023002358A1 (es) 2024-02-23
KR20200097299A (ko) 2020-08-18
JP2021506238A (ja) 2021-02-22
WO2019118638A3 (en) 2019-08-15
BR112020011686A2 (pt) 2020-11-24
EA202091437A1 (ru) 2020-12-29
EP3724337A2 (en) 2020-10-21
CN111699257A (zh) 2020-09-22
CL2020001543A1 (es) 2020-08-28
PH12020550833A1 (en) 2021-07-05
JP2024012386A (ja) 2024-01-30
SG11202005257UA (en) 2020-07-29
TW201938792A (zh) 2019-10-01
CR20200304A (es) 2020-09-04
ZA202003982B (en) 2021-07-28
US20210139912A1 (en) 2021-05-13
CO2020008485A2 (es) 2020-07-31
AR113490A1 (es) 2020-05-06
AU2018386089A1 (en) 2020-06-18
MX2020006088A (es) 2020-08-24

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