CR20200108A - Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) - Google Patents
Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)Info
- Publication number
- CR20200108A CR20200108A CR20200108A CR20200108A CR20200108A CR 20200108 A CR20200108 A CR 20200108A CR 20200108 A CR20200108 A CR 20200108A CR 20200108 A CR20200108 A CR 20200108A CR 20200108 A CR20200108 A CR 20200108A
- Authority
- CR
- Costa Rica
- Prior art keywords
- apoc3
- rnai agents
- iii
- expresión
- composiciones
- Prior art date
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- 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
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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
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- 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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- 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
- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/04—Anorexiants; Antiobesity agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H3/00—Compounds containing only hydrogen atoms and saccharide radicals having only carbon, hydrogen, and oxygen atoms
- C07H3/02—Monosaccharides
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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
- C12N2310/11—Antisense
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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 N.A.
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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
- 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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- 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
- C12N2310/3233—Morpholino-type ring
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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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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- 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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- 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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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
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- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
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- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Plant Pathology (AREA)
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- Hematology (AREA)
- Diabetes (AREA)
- Child & Adolescent Psychology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Urology & Nephrology (AREA)
- Obesity (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Epidemiology (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
La presente descripción se refiere a agentes de iARN, por ejemplo, agentes de iARN de cadena doble, capaces de inhibir la expresión del gen de la Apolipoproteína C-III (también denominada APOC3, apoC-III, APOC-III, y APO C-III), y composiciones que incluyen agentes de iARN de APOC3. Los agentes de iARN de APOC3 descritos en la presente pueden estar conjugados con ligandos diana, que incluyen ligandos que incluyen N-acetil-galactosamina, para facilitar la administración a células, que incluyen a hepatocitos. También se describen las composiciones farmacéuticas que incluyen uno o más agentes de iARN de APOC3, opcionalmente con uno o más productos terapéuticos adicionales. La administración de los agentes de iARN de APOC3 in vivo proporciona la inhibición de la expresión del gen APOC3, y puede dar como resultado niveles de triglicéridos y/o niveles de colesterol reducidos en el sujeto. Los agentes de iARN de APOC3 se pueden usar en métodos de tratamiento de enfermedades y trastornos relacionados con APOC3 que incluyen hipertrigliceridemia, enfermedad cardiovascular, y otros trastornos y enfermedades metabólicos relacionados.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762556818P | 2017-09-11 | 2017-09-11 | |
| US201862643927P | 2018-03-16 | 2018-03-16 | |
| US201862720434P | 2018-08-21 | 2018-08-21 | |
| PCT/US2018/050248 WO2019051402A1 (en) | 2017-09-11 | 2018-09-10 | RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20200108A true CR20200108A (es) | 2020-06-28 |
Family
ID=65630632
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20200108A CR20200108A (es) | 2017-09-11 | 2018-09-10 | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
Country Status (23)
| Country | Link |
|---|---|
| US (4) | US10597657B2 (es) |
| EP (1) | EP3681516A4 (es) |
| JP (2) | JP7438103B2 (es) |
| CN (2) | CN111107853B (es) |
| AU (1) | AU2018329190A1 (es) |
| BR (1) | BR112020002413A2 (es) |
| CA (1) | CA3074303A1 (es) |
| CL (1) | CL2020000593A1 (es) |
| CO (1) | CO2020002586A2 (es) |
| CR (1) | CR20200108A (es) |
| EC (1) | ECSP20017905A (es) |
| IL (1) | IL273172A (es) |
| JO (1) | JOP20200054A1 (es) |
| MX (1) | MX2020002648A (es) |
| MY (1) | MY202025A (es) |
| PE (1) | PE20201283A1 (es) |
| PH (1) | PH12020500332A1 (es) |
| SG (1) | SG11201912178VA (es) |
| TN (1) | TN2020000039A1 (es) |
| TW (2) | TW202415389A (es) |
| UY (1) | UY37871A (es) |
| WO (1) | WO2019051402A1 (es) |
| ZA (1) | ZA202100806B (es) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113846101A (zh) | 2014-11-17 | 2021-12-28 | 阿尔尼拉姆医药品有限公司 | 载脂蛋白C3（APOC3）iRNA组合物及其使用方法 |
| EP3506913A4 (en) * | 2016-09-02 | 2020-06-10 | Arrowhead Pharmaceuticals, Inc. | TARGETING LIGANDS |
| EP4219713A3 (en) | 2017-04-05 | 2023-08-16 | Silence Therapeutics GmbH | Products and compositions |
| CR20200108A (es) * | 2017-09-11 | 2020-06-28 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| KR20220044551A (ko) * | 2019-08-05 | 2022-04-08 | 애로우헤드 파마슈티컬스 인코포레이티드 | Apoc3-관련 질환 및 장애의 치료 방법 |
| MX2022010052A (es) * | 2020-02-18 | 2022-09-05 | Alnylam Pharmaceuticals Inc | Composiciones de acido ribonucleico de interferencia (arni) contra la apolipoproteina c3 (apoc3) y metodos para su uso. |
| CN114480382A (zh) * | 2020-11-12 | 2022-05-13 | 北京大学 | 用于抑制SURF4基因表达的RNAi试剂和组合物 |
| WO2022162155A1 (en) * | 2021-01-30 | 2022-08-04 | E-Therapeutics Plc | Nucleic acids containing abasic nucleotides |
| WO2022272108A2 (en) | 2021-06-24 | 2022-12-29 | Sirnaomics, Inc. | Products and compositions |
| MX2024000650A (es) * | 2021-07-16 | 2024-01-31 | Suzhou Ribo Life Science Co Ltd | Acido nucleico, composicion y conjugado que contiene el mismo, y metodo de preparacion y uso. |
| CA3232053A1 (en) * | 2021-10-08 | 2023-04-13 | E-Therapeutics Plc | Nucleic acids containing abasic nucleosides |
| MX2024006740A (es) | 2021-12-01 | 2024-06-19 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de apoc3. |
| TW202340469A (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 |
| TW202345865A (zh) * | 2022-01-24 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 抑制LPA(Apo(a))蛋白表達的組合物和方法 |
| CN116917477B (zh) | 2022-01-30 | 2024-04-09 | 大睿生物医药科技(上海)有限公司 | 含有n-乙酰半乳糖胺的靶向配体 |
| WO2024002093A1 (zh) * | 2022-06-27 | 2024-01-04 | 大睿生物医药科技(上海)有限公司 | 抑制载脂蛋白C3表达的siRNA |
| US20240218363A1 (en) * | 2022-07-11 | 2024-07-04 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
| WO2024023254A1 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
| WO2024023256A1 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
| WO2024023262A2 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
| CN116814621A (zh) * | 2022-08-05 | 2023-09-29 | 厦门甘宝利生物医药有限公司 | 一种抑制apoc3基因表达的rna抑制剂及其应用 |
| WO2024032680A1 (zh) * | 2022-08-11 | 2024-02-15 | 益杰立科(上海)生物科技有限公司 | 一种表观编辑靶点的方法及用途 |
| WO2024165571A2 (en) * | 2023-02-06 | 2024-08-15 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2024165568A2 (en) * | 2023-02-06 | 2024-08-15 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2024188173A1 (zh) * | 2023-03-10 | 2024-09-19 | 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其用途 |
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