NI202100056A - Moduladores de la expresión de hsd17b13 - Google Patents

Moduladores de la expresión de hsd17b13

Info

Publication number
NI202100056A
NI202100056A NI202100056A NI202100056A NI202100056A NI 202100056 A NI202100056 A NI 202100056A NI 202100056 A NI202100056 A NI 202100056A NI 202100056 A NI202100056 A NI 202100056A NI 202100056 A NI202100056 A NI 202100056A
Authority
NI
Nicaragua
Prior art keywords
hsd17b13
expression
modulators
compounds
methods
Prior art date
Application number
NI202100056A
Other languages
English (en)
Inventor
M Freier Susan
F Murray Susan
Original Assignee
Ionis Pharmaceuticals 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of NI202100056A publication Critical patent/NI202100056A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • 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
    • 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C12Q1/6886Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers

Abstract

Se proporcionan procedimientos, compuestos y composiciones útiles para inhibir la expresión de HSD17B13. Tales compuestos, composiciones y procedimientos son útiles para tratar, prevenir o mejorar una enfermedad asociada con HSD17B13.
NI202100056A 2018-12-21 2021-06-21 Moduladores de la expresión de hsd17b13 NI202100056A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862783680P 2018-12-21 2018-12-21
US201962825581P 2019-03-28 2019-03-28
US201962827524P 2019-04-01 2019-04-01

Publications (1)

Publication Number Publication Date
NI202100056A true NI202100056A (es) 2021-12-01

Family

ID=71102009

Family Applications (1)

Application Number Title Priority Date Filing Date
NI202100056A NI202100056A (es) 2018-12-21 2021-06-21 Moduladores de la expresión de hsd17b13

Country Status (21)

Country Link
US (2) US20220273691A1 (es)
EP (1) EP3899024A4 (es)
JP (2) JP7394137B2 (es)
KR (1) KR20210107027A (es)
CN (1) CN113544272A (es)
AU (2) AU2019403447B2 (es)
BR (1) BR112021011899A2 (es)
CA (1) CA3122432A1 (es)
CL (1) CL2021001643A1 (es)
CO (1) CO2021009009A2 (es)
CR (1) CR20210395A (es)
DO (2) DOP2021000130A (es)
EC (1) ECSP21053104A (es)
IL (1) IL283967A (es)
JO (1) JOP20210158A1 (es)
MX (1) MX2021007503A (es)
NI (1) NI202100056A (es)
PE (1) PE20211755A1 (es)
SG (1) SG11202106378VA (es)
TW (1) TW202039846A (es)
WO (1) WO2020132564A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210000906A1 (en) * 2018-03-21 2021-01-07 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
CN112635049A (zh) * 2020-12-23 2021-04-09 无锡市第二人民医院 一种研究HSD17B13 rs72613567基因变异和肾功能损伤之间关系的方法
WO2023091644A2 (en) * 2021-11-19 2023-05-25 Wave Life Sciences Ltd. Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto
TW202340470A (zh) * 2022-01-20 2023-10-16 大陸商上海拓界生物醫藥科技有限公司 一種dsrna、其應用及製備方法
WO2023213284A1 (zh) * 2022-05-06 2023-11-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011003071A1 (en) 2009-07-02 2011-01-06 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Methods of modulating hepatitis c virus infection
HUE051680T2 (hu) 2012-05-24 2021-03-29 Ionis Pharmaceuticals Inc Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények
CN103520724B (zh) * 2013-10-23 2016-05-25 江苏美迪森生物医药有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
KR102619197B1 (ko) * 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
WO2019075181A1 (en) 2017-10-11 2019-04-18 Regeneron Pharmaceuticals, Inc. INHIBITION OF HSD17B13 IN THE TREATMENT OF HEPATIC DISEASE IN PATIENTS EXPRESSING PNPLA3 I148M VARIATION
WO2019183164A1 (en) 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA3109553A1 (en) 2018-09-19 2020-03-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of 17beta-hsd type 13 (hsd17b13), compositions thereof, and methods of use

Also Published As

Publication number Publication date
CN113544272A (zh) 2021-10-22
US20220273691A1 (en) 2022-09-01
AU2019403447B2 (en) 2023-07-27
SG11202106378VA (en) 2021-07-29
CL2021001643A1 (es) 2022-01-21
JP7394137B2 (ja) 2023-12-07
MX2021007503A (es) 2021-08-05
IL283967A (en) 2021-07-29
CA3122432A1 (en) 2020-06-25
DOP2023000251A (es) 2023-12-29
ECSP21053104A (es) 2021-08-31
JP2022514657A (ja) 2022-02-14
PE20211755A1 (es) 2021-09-07
JOP20210158A1 (ar) 2023-01-30
CR20210395A (es) 2021-11-05
AU2019403447A1 (en) 2021-07-29
CO2021009009A2 (es) 2021-07-30
TW202039846A (zh) 2020-11-01
EP3899024A4 (en) 2023-05-31
JP2024037737A (ja) 2024-03-19
EP3899024A1 (en) 2021-10-27
US20230310483A1 (en) 2023-10-05
DOP2021000130A (es) 2021-09-30
AU2023251525A1 (en) 2023-11-16
WO2020132564A1 (en) 2020-06-25
BR112021011899A2 (pt) 2021-10-26
KR20210107027A (ko) 2021-08-31

Similar Documents

Publication Publication Date Title
ECSP21053104A (es) Moduladores de la expresión de hsd17b13
BR112018004620A2 (pt) moduladores da expressão de kras
CO2017013065A2 (es) Moduladores de diaciglicerol aciltransferasa 2 (dgat2)
NI202100018A (es) Moduladores de la expresión de pnpla3
CR20190471A (es) Moduladores de la expresión de pcsk9
EA202092748A1 (ru) Модуляторы экспрессии apol1
CL2021001246A1 (es) Moduladores de expresión irf5.
CO2020003134A2 (es) Moduladores de la expresión de enac
CO2020008769A2 (es) Moduladores de la expresión dnm2
UY38472A (es) Moduladores de la expresión de foxp3
EA202190045A1 (ru) Составы/композиции, содержащие ибрутиниб
EA202191657A1 (ru) Модуляторы экспрессии hsd17b13
AR117079A1 (es) Moduladores de la expresión de foxp3