TW201918555A - 用於抑制去唾液酸醣蛋白受體1表現之RNAi試劑及組合物 - Google Patents
用於抑制去唾液酸醣蛋白受體1表現之RNAi試劑及組合物 Download PDFInfo
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- TW201918555A TW201918555A TW107136576A TW107136576A TW201918555A TW 201918555 A TW201918555 A TW 201918555A TW 107136576 A TW107136576 A TW 107136576A TW 107136576 A TW107136576 A TW 107136576A TW 201918555 A TW201918555 A TW 201918555A
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- 238000004659 sterilization and disinfection Methods 0.000 description 1
- 125000001424 substituent group Chemical group 0.000 description 1
- IIACRCGMVDHOTQ-UHFFFAOYSA-N sulfamic acid Chemical compound NS(O)(=O)=O IIACRCGMVDHOTQ-UHFFFAOYSA-N 0.000 description 1
- 229940124530 sulfonamide Drugs 0.000 description 1
- BDHFUVZGWQCTTF-UHFFFAOYSA-M sulfonate Chemical compound [O-]S(=O)=O BDHFUVZGWQCTTF-UHFFFAOYSA-M 0.000 description 1
- 239000011593 sulfur Substances 0.000 description 1
- 239000004094 surface-active agent Substances 0.000 description 1
- 239000000375 suspending agent Substances 0.000 description 1
- 238000013268 sustained release Methods 0.000 description 1
- 239000012730 sustained-release form Substances 0.000 description 1
- 239000003765 sweetening agent Substances 0.000 description 1
- 208000011580 syndromic disease Diseases 0.000 description 1
- 238000007910 systemic administration Methods 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 239000002562 thickening agent Substances 0.000 description 1
- 150000003568 thioethers Chemical group 0.000 description 1
- 238000013519 translation Methods 0.000 description 1
- 108091005703 transmembrane proteins Proteins 0.000 description 1
- 102000035160 transmembrane proteins Human genes 0.000 description 1
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 description 1
- 230000003827 upregulation Effects 0.000 description 1
- 238000001291 vacuum drying Methods 0.000 description 1
- 238000009777 vacuum freeze-drying Methods 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
- 229940075420 xanthine Drugs 0.000 description 1
Classifications
-
- 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
- C12N15/1138—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 receptors or cell surface proteins
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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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
- C12N2310/11—Antisense
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
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- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Dermatology (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762573206P | 2017-10-17 | 2017-10-17 | |
| US62/573,206 | 2017-10-17 | ||
| US201762608606P | 2017-12-21 | 2017-12-21 | |
| US62/608,606 | 2017-12-21 | ||
| US201862635277P | 2018-02-26 | 2018-02-26 | |
| US62/635,277 | 2018-02-26 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW201918555A true TW201918555A (zh) | 2019-05-16 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| TW107136576A TW201918555A (zh) | 2017-10-17 | 2018-10-17 | 用於抑制去唾液酸醣蛋白受體1表現之RNAi試劑及組合物 |
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| Country | Link |
|---|---|
| US (1) | US11492624B2 (https=) |
| EP (1) | EP3697909A4 (https=) |
| JP (2) | JP7473472B2 (https=) |
| CN (1) | CN111212909A (https=) |
| AU (1) | AU2018352379A1 (https=) |
| CA (1) | CA3079413A1 (https=) |
| TW (1) | TW201918555A (https=) |
| UY (1) | UY37936A (https=) |
| WO (1) | WO2019079294A1 (https=) |
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| MX2018009853A (es) * | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
| IL302530A (en) * | 2020-11-06 | 2023-07-01 | Arbutus Biopharma Corp | Targeted conjugates involving modified siRNA |
| CN116617390A (zh) * | 2022-02-11 | 2023-08-22 | 武汉大学 | Asgr1抑制剂在促进胆固醇外排和治疗非酒精性脂肪性肝病中的应用 |
| WO2024046297A1 (zh) * | 2022-09-02 | 2024-03-07 | 北京福元医药股份有限公司 | 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途 |
Family Cites Families (16)
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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. |
| DE60040274D1 (de) | 1999-03-10 | 2008-10-30 | Phogen Ltd | Verabreichung von nukleinsäuren und proteinen an zellen |
| US8137695B2 (en) | 2006-08-18 | 2012-03-20 | Arrowhead Madison Inc. | Polyconjugates for in vivo delivery of polynucleotides |
| CN1300312C (zh) * | 2004-08-23 | 2007-02-14 | 中国人民解放军军事医学科学院放射与辐射医学研究所 | 靶向去唾液酸糖蛋白受体的抗乙型肝炎病毒反义寡核苷酸的结构和用途 |
| JP2010505897A (ja) * | 2006-10-11 | 2010-02-25 | マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ | インフルエンザターゲット |
| EP2295543A1 (en) * | 2009-09-11 | 2011-03-16 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Method for the preparation of an influenza virus |
| DK2539451T3 (da) | 2010-02-24 | 2016-04-04 | Arrowhead Res Corp | Sammensætninger til målrettet tilførsel af siRNA |
| US8501930B2 (en) | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
| WO2013032829A1 (en) | 2011-08-26 | 2013-03-07 | Arrowhead Research Corporation | Poly(vinyl ester) polymers for in vivo nucleic acid delivery |
| JP2015515530A (ja) | 2012-04-18 | 2015-05-28 | アローヘッド リサーチ コーポレイション | インビボ核酸送達のためのポリ（アクリラート）ポリマー |
| CA2997444A1 (en) * | 2015-09-29 | 2017-04-06 | Amgen Inc. | Asgr inhibitors for reducing cholesterol levels |
| JOP20160211B1 (ar) * | 2015-10-01 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| KR102515329B1 (ko) | 2016-03-07 | 2023-03-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
| UY37376A (es) * | 2016-08-26 | 2018-03-23 | Amgen Inc | Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso |
| MX2018009853A (es) | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
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2018
- 2018-10-16 US US16/756,440 patent/US11492624B2/en active Active
- 2018-10-16 WO PCT/US2018/056077 patent/WO2019079294A1/en not_active Ceased
- 2018-10-16 AU AU2018352379A patent/AU2018352379A1/en not_active Abandoned
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- 2018-10-17 TW TW107136576A patent/TW201918555A/zh unknown
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2024
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| EP3697909A4 (en) | 2021-10-13 |
| WO2019079294A1 (en) | 2019-04-25 |
| CA3079413A1 (en) | 2019-04-25 |
| AU2018352379A1 (en) | 2020-06-04 |
| JP2024086876A (ja) | 2024-06-28 |
| CN111212909A (zh) | 2020-05-29 |
| US20200283777A1 (en) | 2020-09-10 |
| EP3697909A1 (en) | 2020-08-26 |
| JP7473472B2 (ja) | 2024-04-23 |
| UY37936A (es) | 2019-05-31 |
| JP2020537653A (ja) | 2020-12-24 |
| US11492624B2 (en) | 2022-11-08 |
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