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
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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
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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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    • C12Q1/00Measuring 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
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    • 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
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    • 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
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • 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
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    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
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    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers

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  • Health & Medical Sciences (AREA)
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  • 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)
  • Immunology (AREA)
  • Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Plant Pathology (AREA)
  • Virology (AREA)
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  • Hospice & Palliative Care (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Optical Modulation, Optical Deflection, Nonlinear Optics, Optical Demodulation, Optical Logic Elements (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
CA3093547A1 (en) * 2018-03-21 2019-09-26 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
TW202400791A (zh) * 2022-05-06 2024-01-01 大陸商蘇州瑞博生物技術股份有限公司 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途

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
DK2855500T3 (da) 2012-05-24 2020-09-14 Ionis Pharmaceuticals Inc Fremgangsmåder og sammensætninger til modulering af apolipoprotein (A)-ekspression
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
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
CN111183234A (zh) 2017-10-11 2020-05-19 雷杰纳荣制药公司 在表达pnpla3 i148m变异的患者的肝病治疗中对hsd17b13的抑制
US20210380985A1 (en) 2018-03-21 2021-12-09 Regeneron Pharmaceuticals, Inc. 17ß-Hydroxysteroid Dehydrogenase Type 13 (HSD17B13) iRNA Compositions And Methods Of Use Thereof
JP7488254B2 (ja) 2018-09-19 2024-05-21 アローヘッド ファーマシューティカルズ インコーポレイテッド 17β-HSD13型(HSD17B13)の発現を阻害するためのRNAi剤、その組成物、および使用方法

Also Published As

Publication number Publication date
AU2019403447B2 (en) 2023-07-27
EP3899024A1 (en) 2021-10-27
CO2021009009A2 (es) 2021-07-30
TW202039846A (zh) 2020-11-01
MX2021007503A (es) 2021-08-05
CL2021001643A1 (es) 2022-01-21
AU2023251525A1 (en) 2023-11-16
US20230310483A1 (en) 2023-10-05
JOP20210158A1 (ar) 2023-01-30
JP7394137B2 (ja) 2023-12-07
BR112021011899A2 (pt) 2021-10-26
CA3122432A1 (en) 2020-06-25
KR20210107027A (ko) 2021-08-31
ECSP21053104A (es) 2021-08-31
CR20210395A (es) 2021-11-05
US20220273691A1 (en) 2022-09-01
SG11202106378VA (en) 2021-07-29
AU2019403447A1 (en) 2021-07-29
WO2020132564A1 (en) 2020-06-25
NI202100056A (es) 2021-12-01
IL283967A (en) 2021-07-29
EP3899024A4 (en) 2023-05-31
JP2022514657A (ja) 2022-02-14
DOP2021000130A (es) 2021-09-30
JP2024037737A (ja) 2024-03-19
PE20211755A1 (es) 2021-09-07
CN113544272A (zh) 2021-10-22

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