CO2020008485A2 - Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de los mismos - Google Patents

Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de los mismos

Info

Publication number
CO2020008485A2
CO2020008485A2 CONC2020/0008485A CO2020008485A CO2020008485A2 CO 2020008485 A2 CO2020008485 A2 CO 2020008485A2 CO 2020008485 A CO2020008485 A CO 2020008485A CO 2020008485 A2 CO2020008485 A2 CO 2020008485A2
Authority
CO
Colombia
Prior art keywords
methods
constructs
arni
pnpla3 expression
inhibit
Prior art date
Application number
CONC2020/0008485A
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 CO2020008485A2 publication Critical patent/CO2020008485A2/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)

Abstract

La presente invención se refiere a constructos de ARNi para reducir la expresión del gen de PNPLA3. También se describe métodos de uso de tales constructos de ARNi para tratar o prevenir enfermedad hepática, enfermedad por hígado graso no alcohólico (NAFLD) .
CONC2020/0008485A 2017-12-12 2020-07-10 Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de los mismos CO2020008485A2 (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
CO2020008485A2 true CO2020008485A2 (es) 2020-07-31

Family

ID=65139138

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0008485A CO2020008485A2 (es) 2017-12-12 2020-07-10 Constructos de arni para inhibir la expresión de pnpla3 y métodos 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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US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
JP2022511550A (ja) * 2018-12-10 2022-01-31 アムジエン・インコーポレーテツド ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト
WO2021074772A1 (en) * 2019-10-14 2021-04-22 Astrazeneca Ab Modulators of pnpla3 expression
JP2023504744A (ja) * 2019-12-09 2023-02-06 アムジエン・インコーポレーテツド ＬＰＡ発現を阻害するためのＲＮＡｉコンストラクト及び方法
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
US20210301294A1 (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
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AU2022339846A1 (en) * 2021-09-01 2024-03-14 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof

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Also Published As

Publication number Publication date
WO2019118638A2 (en) 2019-06-20
MX2020006088A (es) 2020-08-24
AU2018386089A1 (en) 2020-06-18
CL2023002358A1 (es) 2024-02-23
JP2021506238A (ja) 2021-02-22
SG11202005257UA (en) 2020-07-29
WO2019118638A3 (en) 2019-08-15
EA202091437A1 (ru) 2020-12-29
JOP20200142A1 (ar) 2022-10-30
JP2024012386A (ja) 2024-01-30
CR20200304A (es) 2020-09-04
CN111699257A (zh) 2020-09-22
IL275029A (en) 2020-07-30
UY38003A (es) 2019-06-28
ZA202003982B (en) 2021-07-28
PE20210633A1 (es) 2021-03-23
CA3084133A1 (en) 2019-06-20
EP3724337A2 (en) 2020-10-21
PH12020550833A1 (en) 2021-07-05
CL2020001543A1 (es) 2020-08-28
BR112020011686A2 (pt) 2020-11-24
AR113490A1 (es) 2020-05-06
KR20200097299A (ko) 2020-08-18
TW201938792A (zh) 2019-10-01
US20210139912A1 (en) 2021-05-13

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