ECSP21027262A - Agentes de ARNi para inhibir la expresión de 17beta-HSD tipo 13 (HSD17B13), composiciones de dichos agentes, y métodos de uso - Google Patents
Agentes de ARNi para inhibir la expresión de 17beta-HSD tipo 13 (HSD17B13), composiciones de dichos agentes, y métodos de usoInfo
- Publication number
- ECSP21027262A ECSP21027262A ECSENADI202127262A ECDI202127262A ECSP21027262A EC SP21027262 A ECSP21027262 A EC SP21027262A EC SENADI202127262 A ECSENADI202127262 A EC SENADI202127262A EC DI202127262 A ECDI202127262 A EC DI202127262A EC SP21027262 A ECSP21027262 A EC SP21027262A
- Authority
- EC
- Ecuador
- Prior art keywords
- hsd17b13
- rnai agents
- methods
- agents
- expression
- Prior art date
Links
- 102100037429 17-beta-hydroxysteroid dehydrogenase 13 Human genes 0.000 title abstract 7
- 108091030071 RNAI Proteins 0.000 title abstract 7
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 7
- 101000806241 Homo sapiens 17-beta-hydroxysteroid dehydrogenase 13 Proteins 0.000 title abstract 6
- 230000014509 gene expression Effects 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 6
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 abstract 4
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 2
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 abstract 2
- 101710194118 17-beta-hydroxysteroid dehydrogenase 13 Proteins 0.000 abstract 1
- 108010070743 3(or 17)-beta-hydroxysteroid dehydrogenase Proteins 0.000 abstract 1
- 102100034067 Dehydrogenase/reductase SDR family member 11 Human genes 0.000 abstract 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 abstract 1
- 206010016654 Fibrosis Diseases 0.000 abstract 1
- 101150000579 Hsd17b13 gene Proteins 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 230000007882 cirrhosis Effects 0.000 abstract 1
- 208000037765 diseases and disorders Diseases 0.000 abstract 1
- 210000003494 hepatocyte Anatomy 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 239000003446 ligand Substances 0.000 abstract 1
- 208000019423 liver disease Diseases 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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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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- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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
- C12Y—ENZYMES
- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862733320P | 2018-09-19 | 2018-09-19 | |
| US201862773707P | 2018-11-30 | 2018-11-30 | |
| US201962890220P | 2019-08-22 | 2019-08-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ECSP21027262A true ECSP21027262A (es) | 2021-05-31 |
Family
ID=69887755
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ECSENADI202127262A ECSP21027262A (es) | 2018-09-19 | 2021-04-19 | Agentes de ARNi para inhibir la expresión de 17beta-HSD tipo 13 (HSD17B13), composiciones de dichos agentes, y métodos de uso |
Country Status (21)
| Country | Link |
|---|---|
| US (1) | US12534728B2 (https=) |
| EP (1) | EP3852769A4 (https=) |
| JP (3) | JP7488254B2 (https=) |
| KR (1) | KR102769565B1 (https=) |
| CN (1) | CN113164509B (https=) |
| AU (1) | AU2019342117A1 (https=) |
| BR (1) | BR112021005137A2 (https=) |
| CA (1) | CA3109553A1 (https=) |
| CL (1) | CL2021000669A1 (https=) |
| CO (1) | CO2021003446A2 (https=) |
| CR (1) | CR20210186A (https=) |
| EC (1) | ECSP21027262A (https=) |
| IL (1) | IL281597A (https=) |
| MX (1) | MX2021003077A (https=) |
| MY (1) | MY209138A (https=) |
| PE (1) | PE20211212A1 (https=) |
| PH (1) | PH12021550423A1 (https=) |
| SG (1) | SG11202101698WA (https=) |
| TW (2) | TWI870358B (https=) |
| UY (1) | UY38382A (https=) |
| WO (1) | WO2020061177A1 (https=) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201901841TA (en) * | 2016-09-02 | 2019-03-28 | Arrowhead Pharmaceuticals Inc | Targeting ligands |
| AU2019239971B2 (en) * | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| KR102769565B1 (ko) * | 2018-09-19 | 2025-02-20 | 애로우헤드 파마슈티컬스 인코포레이티드 | 17베타-HSD 유형 13- (HSD17B13)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 |
| CR20210395A (es) * | 2018-12-21 | 2021-11-05 | Ionis Pharmaceuticals Inc | Moduladores de la expresión de hsd17b13 |
| KR20240004092A (ko) * | 2020-06-01 | 2024-01-11 | 암젠 인크 | Hsd17b13 발현을 억제하기 위한 rnai 작제물 및 이의 사용 방법 |
| WO2022098748A1 (en) * | 2020-11-06 | 2022-05-12 | Inipharm, Inc. | Uses for hsd17b13 inhibitors |
| MX2023007587A (es) | 2020-12-23 | 2023-09-21 | Regeneron Pharma | Tratamiento de enfermedades hepaticas con inhibidores de efectores b tipo dffa que inducen la muerte celular (cideb). |
| CA3216332A1 (en) * | 2021-04-22 | 2022-10-27 | Tuojie Biotech (Shanghai) Co., Ltd. | Sirna targeting 17b-hydroxysteroid dehydrogenase type 13 and sirna conjugate |
| 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 |
| CN118234862A (zh) * | 2021-09-08 | 2024-06-21 | 阿利戈斯治疗公司 | 经修饰的短干扰核酸分子(siNA)及其用途 |
| KR20240103029A (ko) * | 2021-11-18 | 2024-07-03 | 노파르티스 아게 | 샤르코 마리 투스 질환 치료용 pmp22 표적화 화합물 |
| EP4433596A4 (en) * | 2021-11-19 | 2026-02-11 | Wave Life Sciences Ltd | DOUBLE-STRANDED OLIGONUCLETIC COMPOSITIONS ASSOCIATED WITH HSD17B13 AND RELATED PROCESSES |
| WO2023109935A1 (zh) * | 2021-12-16 | 2023-06-22 | 上海拓界生物医药科技有限公司 | 一种dsRNA、其制备方法及应用 |
| US20230193392A1 (en) | 2021-12-20 | 2023-06-22 | Regeneron Pharmaceuticals, Inc. | Methods Of Identifying And Evaluating Liver Inflammation And Liver Fibrosis In A Subject By Determining A Stratified Score Based On Gene Expression |
| 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 |
| WO2023196941A1 (en) | 2022-04-08 | 2023-10-12 | Arrowhead Pharmaceuticals, Inc. | Treatment of a non-alcoholic fatty liver disease |
| EP4514805A4 (en) * | 2022-04-28 | 2026-01-21 | Enanta Pharm Inc | TYPE 13 17-BETA-HYDROXYSTEROID DEHYDROGENASE INHIBITORS CONTAINING PYRIMIDINONE |
| CN116515835A (zh) * | 2022-04-29 | 2023-08-01 | 北京福元医药股份有限公司 | 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途 |
| 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 |
| WO2024131916A1 (en) * | 2022-12-22 | 2024-06-27 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of 17beta-hydroxysteroid dehydrogenase type 13 (hsd17b13) |
| WO2024255748A1 (zh) * | 2023-06-16 | 2024-12-19 | 施能康医药科技(苏州)有限公司 | 靶向17-β-羟基类固醇脱氢酶13的核酸及其用途 |
| IL326882A (en) * | 2023-08-30 | 2026-04-01 | Arrowhead Pharmaceuticals Inc | RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions and methods of use |
| US20250120952A1 (en) * | 2023-10-04 | 2025-04-17 | Enanta Pharmaceuticals, Inc. | 17-Beta-Hydroxysteroid Dehydrogenase Type 13 Inhibitors And Methods Of Use Thereof |
| CN121969377A (zh) | 2023-10-06 | 2026-05-01 | 葛兰素史密斯克莱知识产权(第3号)有限公司 | 非酒精性脂肪性肝病的治疗 |
| WO2026082165A1 (zh) * | 2024-10-18 | 2026-04-23 | 正大天晴药业集团股份有限公司 | 修饰的双链核糖核酸 |
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| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
| US5624813A (en) | 1994-04-21 | 1997-04-29 | Mahant; Vijay K. | NAD(P)+ /NAD(P)H based chemiluminescent diagnostics |
| WO1997020942A1 (en) * | 1995-12-05 | 1997-06-12 | Piao Yun Shang | Hsd17b1 promoter, enhancer, silencer and use thereof |
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