JP2021506238A - ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト - Google Patents

ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト Download PDF

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JP2021506238A
JP2021506238A JP2020530513A JP2020530513A JP2021506238A JP 2021506238 A JP2021506238 A JP 2021506238A JP 2020530513 A JP2020530513 A JP 2020530513A JP 2020530513 A JP2020530513 A JP 2020530513A JP 2021506238 A JP2021506238 A JP 2021506238A
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pnpla3
rnai construct
rnai
nucleotides
expression
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JP2021506238A5 (https=
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リュリフソン，イングリッド
マレー，ジャスティン・ケイ
オルマン，マイケル
ホフマン，オリバー
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アムジエン・インコーポレーテツド
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Publication of JP2021506238A5 publication Critical patent/JP2021506238A5/ja
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Priority to JP2025152531A priority patent/JP2025186376A/ja
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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
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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]
    • 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
    • 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
    • C12N2310/32—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—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)

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  • Chemical & Material Sciences (AREA)
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  • 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)
JP2020530513A 2017-12-12 2018-12-12 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト Pending JP2021506238A (ja)

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JP2023183884A JP2024012386A (ja) 2017-12-12 2023-10-26 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト
JP2025152531A JP2025186376A (ja) 2017-12-12 2025-09-12 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト

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US201762597841P 2017-12-12 2017-12-12
US62/597,841 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

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JP2021506238A5 JP2021506238A5 (https=) 2022-01-11

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JP2023183884A Pending JP2024012386A (ja) 2017-12-12 2023-10-26 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト
JP2025152531A Pending JP2025186376A (ja) 2017-12-12 2025-09-12 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト

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JP2025152531A Pending JP2025186376A (ja) 2017-12-12 2025-09-12 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト

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US (2) US20210139912A1 (https=)
EP (1) EP3724337A2 (https=)
JP (3) JP2021506238A (https=)
KR (1) KR20200097299A (https=)
CN (1) CN111699257A (https=)
AR (1) AR113490A1 (https=)
AU (2) AU2018386089A1 (https=)
BR (1) BR112020011686A2 (https=)
CA (1) CA3084133A1 (https=)
CL (2) CL2020001543A1 (https=)
CO (1) CO2020008485A2 (https=)
CR (1) CR20200304A (https=)
EA (1) EA202091437A1 (https=)
IL (1) IL275029A (https=)
JO (1) JOP20200142A1 (https=)
MX (2) MX2020006088A (https=)
PE (1) PE20210633A1 (https=)
PH (1) PH12020550833A1 (https=)
SG (1) SG11202005257UA (https=)
TW (1) TW201938792A (https=)
UY (1) UY38003A (https=)
WO (1) WO2019118638A2 (https=)
ZA (1) ZA202003982B (https=)

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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
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US20170349903A1 (en) * 2016-06-03 2017-12-07 Purdue Research Foundation siRNA COMPOSITIONS THAT SPECIFICALLY DOWNREGULATE EXPRESSION OF A VARIANT OF THE PNPLA3 GENE AND METHODS OF USE THEREOF FOR TREATING A CHRONIC LIVER DISEASE OR ALCOHOLIC LIVER DISEASE (ALD)

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US20210139912A1 (en) 2021-05-13
CO2020008485A2 (es) 2020-07-31
JP2025186376A (ja) 2025-12-23
EA202091437A1 (ru) 2020-12-29
CA3084133A1 (en) 2019-06-20
IL275029A (en) 2020-07-30
ZA202003982B (en) 2021-07-28
US20250154512A1 (en) 2025-05-15
SG11202005257UA (en) 2020-07-29
PH12020550833A1 (en) 2021-07-05
MX2025010269A (es) 2025-10-01
CN111699257A (zh) 2020-09-22
WO2019118638A2 (en) 2019-06-20
CL2023002358A1 (es) 2024-02-23
AU2018386089A1 (en) 2020-06-18
WO2019118638A3 (en) 2019-08-15
UY38003A (es) 2019-06-28
AU2025203098A1 (en) 2025-05-22
AU2025203098A9 (en) 2026-03-05
JOP20200142A1 (ar) 2022-10-30
BR112020011686A2 (pt) 2020-11-24
EP3724337A2 (en) 2020-10-21
AR113490A1 (es) 2020-05-06
KR20200097299A (ko) 2020-08-18
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