DOP2023000251A - Compuestos oligoméricos que inhiben la expresión de hsd17b13 - Google Patents

Compuestos oligoméricos que inhiben la expresión de hsd17b13

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Publication number
DOP2023000251A
DOP2023000251A DO2023000251A DO2023000251A DOP2023000251A DO P2023000251 A DOP2023000251 A DO P2023000251A DO 2023000251 A DO2023000251 A DO 2023000251A DO 2023000251 A DO2023000251 A DO 2023000251A DO P2023000251 A DOP2023000251 A DO P2023000251A
Authority
DO
Dominican Republic
Prior art keywords
hsd17b13
expression
inhibit
oligomeric compounds
useful
Prior art date
Application number
DO2023000251A
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 DOP2023000251A publication Critical patent/DOP2023000251A/es

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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
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    • 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
    • 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
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C12Q1/6886—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Amplifiers (AREA)
  • Optical Modulation, Optical Deflection, Nonlinear Optics, Optical Demodulation, Optical Logic Elements (AREA)
  • Saccharide Compounds (AREA)

Abstract

Se proporciona un compuestos oligomérico o una sal farmacéuticamente aceptable del mismo, con la estructura: y composiciones farmacéuticas que lo comprenden, útiles para inhibir la expresión de HSD17B13 . Tales compuestos y composiciones son útiles para preparar un medicamento para tratar, prevenir o mejorar una enfermedad asociada con HSD17B13.
DO2023000251A 2018-12-21 2023-11-14 Compuestos oligoméricos que inhiben la expresión de hsd17b13 DOP2023000251A (es)

Applications Claiming Priority (4)

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
PCT/US2019/068051 WO2020132564A1 (en) 2018-12-21 2019-12-20 Modulators of hsd17b13 expression

Publications (1)

Publication Number Publication Date
DOP2023000251A true DOP2023000251A (es) 2023-12-29

Family

ID=71102009

Family Applications (2)

Application Number Title Priority Date Filing Date
DO2021000130A DOP2021000130A (es) 2018-12-21 2021-06-21 Moduladores de la expresion de hsd17b13
DO2023000251A DOP2023000251A (es) 2018-12-21 2023-11-14 Compuestos oligoméricos que inhiben la expresión de hsd17b13

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DO2021000130A DOP2021000130A (es) 2018-12-21 2021-06-21 Moduladores de la expresion 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
WO2023138650A1 (zh) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 一种dsRNA、其应用及制备方法
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
EP2855500B1 (en) 2012-05-24 2020-07-01 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein(a) expression
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
BR112019014841A2 (pt) 2017-01-23 2020-04-28 Regeneron Pharma rna guia, uso do rna guia, rna antissentido, sirna ou shrna, uso do rna antissentido, do sirna ou do shrna, ácido nucleico isolado, vetor, composição, célula, e, métodos para modificar um gene hsd17b13 em uma célula, para diminuir a expressão de um gene hsd17b13 em uma célula, para modificar uma célula e para tratar um indivíduo que não é portador da variante de hsd17b13
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
SG11202007583SA (en) 2018-03-21 2020-09-29 Regeneron Pharma 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
SG11202101698WA (en) 2018-09-19 2021-04-29 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
CO2021009009A2 (es) 2021-07-30
CR20210395A (es) 2021-11-05
CL2021001643A1 (es) 2022-01-21
EP3899024A4 (en) 2023-05-31
MX2021007503A (es) 2021-08-05
SG11202106378VA (en) 2021-07-29
NI202100056A (es) 2021-12-01
KR20210107027A (ko) 2021-08-31
IL283967A (en) 2021-07-29
WO2020132564A1 (en) 2020-06-25
US20230310483A1 (en) 2023-10-05
US20220273691A1 (en) 2022-09-01
EP3899024A1 (en) 2021-10-27
AU2019403447B2 (en) 2023-07-27
DOP2021000130A (es) 2021-09-30
CA3122432A1 (en) 2020-06-25
JP2024037737A (ja) 2024-03-19
JP7394137B2 (ja) 2023-12-07
PE20211755A1 (es) 2021-09-07
JOP20210158A1 (ar) 2023-01-30
JP2022514657A (ja) 2022-02-14
BR112021011899A2 (pt) 2021-10-26
TW202039846A (zh) 2020-11-01
ECSP21053104A (es) 2021-08-31
CN113544272A (zh) 2021-10-22
AU2023251525A1 (en) 2023-11-16
AU2019403447A1 (en) 2021-07-29

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