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
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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
- C12Q2600/156—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)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Analytical Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Pathology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- Oncology (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)
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 (12)
| Country | Link |
|---|---|
| US (4) | US10961583B2 (enExample) |
| EP (2) | EP4234719B1 (enExample) |
| JP (2) | JP7434151B2 (enExample) |
| KR (2) | KR102694809B1 (enExample) |
| CN (1) | CN111183234A (enExample) |
| AU (2) | AU2018348195B2 (enExample) |
| CA (1) | CA3078883A1 (enExample) |
| IL (2) | IL273550B2 (enExample) |
| MX (1) | MX2020003561A (enExample) |
| RU (1) | RU2020115526A (enExample) |
| SG (1) | SG11202003254SA (enExample) |
| WO (1) | WO2019075181A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10208081B2 (en) | 2014-11-26 | 2019-02-19 | Enanta Pharmaceuticals, Inc. | Bile acid derivatives as FXR/TGR5 agonists and methods of use thereof |
| CN118325899A (zh) | 2017-01-23 | 2024-07-12 | 瑞泽恩制药公司 | Hsd17b13变体及其应用 |
| EP4234719B1 (en) | 2017-10-11 | 2025-11-26 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| AU2019403447B2 (en) * | 2018-12-21 | 2023-07-27 | Ionis Pharmaceuticals, Inc. | Modulators of HSD17B13 expression |
| WO2021003295A1 (en) | 2019-07-02 | 2021-01-07 | Regeneron Pharmaceuticals, Inc. | Modulators of hsd17b13 and methods of use thereof |
| WO2021211959A2 (en) * | 2020-04-18 | 2021-10-21 | Inipharm, Inc. | Methods and compositions for treating drug induced steatohepatitis |
| WO2021211981A1 (en) * | 2020-04-18 | 2021-10-21 | Inipharm, Inc. | Substrate selective hsd17b13 inhibitors and uses thereof |
| JP7626423B6 (ja) | 2020-10-16 | 2025-03-03 | 公立大学法人大阪 | 線維症治療薬 |
| US12018259B2 (en) * | 2020-12-23 | 2024-06-25 | 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 |
| 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 |
| IL319486A (en) * | 2022-10-07 | 2025-05-01 | Pfizer | HSD17B13 Inhibitors and/or Retarders |
| WO2024173343A1 (en) | 2023-02-14 | 2024-08-22 | Regeneron Pharmaceuticals, Inc. | Compounds targeting hydroxysteroid 17-beta dehydrogenase (hsd17b) and uses thereof |
| WO2025012038A1 (en) | 2023-07-13 | 2025-01-16 | 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 |
| 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 |
| 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 |
| 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 |
Citations (2)
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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 |
| WO2017048620A1 (en) * | 2015-09-14 | 2017-03-23 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof |
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