AU2018348195B2 - Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation - Google Patents
Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation Download PDFInfo
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- AU2018348195B2 AU2018348195B2 AU2018348195A AU2018348195A AU2018348195B2 AU 2018348195 B2 AU2018348195 B2 AU 2018348195B2 AU 2018348195 A AU2018348195 A AU 2018348195A AU 2018348195 A AU2018348195 A AU 2018348195A AU 2018348195 B2 AU2018348195 B2 AU 2018348195B2
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q1/00—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
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- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q1/00—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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- 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 nucleic acids [NA]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q2600/00—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)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Analytical Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Pathology (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Oncology (AREA)
- Hospice & Palliative Care (AREA)
- Virology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2025203267A AU2025203267A1 (en) | 2017-10-11 | 2025-05-07 | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762570985P | 2017-10-11 | 2017-10-11 | |
| US62/570,985 | 2017-10-11 | ||
| PCT/US2018/055377 WO2019075181A1 (en) | 2017-10-11 | 2018-10-11 | INHIBITION OF HSD17B13 IN THE TREATMENT OF HEPATIC DISEASE IN PATIENTS EXPRESSING PNPLA3 I148M VARIATION |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025203267A Division AU2025203267A1 (en) | 2017-10-11 | 2025-05-07 | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2018348195A1 AU2018348195A1 (en) | 2020-04-23 |
| AU2018348195B2 true AU2018348195B2 (en) | 2025-05-15 |
Family
ID=64316974
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2018348195A Active AU2018348195B2 (en) | 2017-10-11 | 2018-10-11 | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| AU2025203267A Pending AU2025203267A1 (en) | 2017-10-11 | 2025-05-07 | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2025203267A Pending AU2025203267A1 (en) | 2017-10-11 | 2025-05-07 | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US10961583B2 (https=) |
| EP (3) | EP4685243A3 (https=) |
| JP (3) | JP7434151B2 (https=) |
| KR (2) | KR102694809B1 (https=) |
| CN (1) | CN111183234A (https=) |
| AU (2) | AU2018348195B2 (https=) |
| CA (1) | CA3078883A1 (https=) |
| IL (2) | IL319615A (https=) |
| MX (1) | MX2020003561A (https=) |
| NZ (1) | NZ763161A (https=) |
| RU (1) | RU2020115526A (https=) |
| SG (1) | SG11202003254SA (https=) |
| WO (1) | WO2019075181A1 (https=) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US10208081B2 (en) | 2014-11-26 | 2019-02-19 | Enanta Pharmaceuticals, Inc. | Bile acid derivatives as FXR/TGR5 agonists and methods of use thereof |
| IL314915B1 (en) | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 17β-水酸化ステロイド脱水素酵素13型(HSD17B13)iRNA組成物およびその使用方法 |
| MX2021003077A (es) | 2018-09-19 | 2021-05-27 | Arrowhead Pharmaceuticals Inc | Agentes de arni para inhibir la expresion de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y metodos de uso. |
| EP3899024A4 (en) * | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
| EP3994125B1 (en) | 2019-07-02 | 2025-12-24 | Regeneron Pharmaceuticals, Inc. | Modulators of hsd17b13 and methods of use thereof |
| WO2021211981A1 (en) * | 2020-04-18 | 2021-10-21 | Inipharm, Inc. | Substrate selective hsd17b13 inhibitors and uses thereof |
| WO2021211959A2 (en) * | 2020-04-18 | 2021-10-21 | Inipharm, Inc. | Methods and compositions for treating drug induced steatohepatitis |
| JP7626423B6 (ja) | 2020-10-16 | 2025-03-03 | 公立大学法人大阪 | 線維症治療薬 |
| CA3201624A1 (en) | 2020-12-23 | 2022-06-30 | Regeneron Pharmaceuticals, Inc. | Treatment of liver diseases with cell death inducing dffa like effector b (cideb) inhibitors |
| CN112626199A (zh) * | 2021-01-04 | 2021-04-09 | 杭州艾迪康医学检验中心有限公司 | 检测慢性肝炎转化为肝硬化的风险预测位点的引物和方法 |
| CN118103038A (zh) | 2021-08-20 | 2024-05-28 | 英安塔制药有限公司 | 17β-羟基类固醇脱氢酶13型抑制剂及其使用方法 |
| MX2024007463A (es) | 2021-12-20 | 2024-09-02 | Regeneron Pharma | Metodos para identificar y evaluar la inflamacion hepatica y la fibrosis hepatica en un sujeto mediante la determinacion de una puntuacion estratificada basada en la expresion genetica. |
| CN114262738B (zh) * | 2021-12-31 | 2023-03-31 | 吉林大学第一医院 | Hsd17b13基因多态性在中重度mafld诊断中的应用 |
| 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 |
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| WO2024173343A1 (en) | 2023-02-14 | 2024-08-22 | Regeneron Pharmaceuticals, Inc. | Compounds targeting hydroxysteroid 17-beta dehydrogenase (hsd17b) and uses thereof |
| WO2025012425A1 (en) | 2023-07-13 | 2025-01-16 | Enyo Pharma | Tromethamine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof |
| AU2024295030A1 (en) | 2023-07-13 | 2026-01-15 | Enyo Pharma | L-lysine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof |
| WO2025012422A1 (en) | 2023-07-13 | 2025-01-16 | Enyo Pharma | L-arginine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof |
| WO2025098440A1 (zh) * | 2023-11-08 | 2025-05-15 | 浙江扬厉医药技术有限公司 | 抑制PNPLA3表达的双链siRNA以及其缀合物 |
| WO2026002833A1 (en) | 2024-06-24 | 2026-01-02 | Enyo Pharma | Ethanolamine salt of 4-chloro-5-[4-(2,6-dichlorophenyl)sulfonylpiperazin-1-yl]-1-benzofuran-2-carboxylic acid and various forms thereof |
| CN118813778B (zh) * | 2024-07-01 | 2025-10-21 | 北京泛生子基因科技有限公司 | 一种试剂盒、使用方法及存储介质 |
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| CN103520724A (zh) * | 2013-10-23 | 2014-01-22 | 北京美森生物医药科技有限公司 | Hsd17b13蛋白或其编码基因的抑制剂的新用途 |
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