BR112020003126A2 - agentes de rnai e composições para inibição da expressão de angiopoietina tipo 3 (angptl3), e métodos de uso - Google Patents
agentes de rnai e composições para inibição da expressão de angiopoietina tipo 3 (angptl3), e métodos de uso Download PDFInfo
- Publication number
- BR112020003126A2 BR112020003126A2 BR112020003126-7A BR112020003126A BR112020003126A2 BR 112020003126 A2 BR112020003126 A2 BR 112020003126A2 BR 112020003126 A BR112020003126 A BR 112020003126A BR 112020003126 A2 BR112020003126 A2 BR 112020003126A2
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- rnai agent
- seq
- nucleotide
- angptl3
- antisense
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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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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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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- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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- 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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- A61P3/00—Drugs for disorders of the metabolism
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- A61P3/00—Drugs for disorders of the metabolism
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- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- A61P9/00—Drugs for disorders of the cardiovascular system
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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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- 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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
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- C12N2310/32—Chemical structure of the sugar
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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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- 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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- 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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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Diabetes (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Endocrinology (AREA)
- Cardiology (AREA)
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- Urology & Nephrology (AREA)
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- Child & Adolescent Psychology (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762558819P | 2017-09-14 | 2017-09-14 | |
| US62/558,819 | 2017-09-14 | ||
| US201762583919P | 2017-11-09 | 2017-11-09 | |
| US62/583,919 | 2017-11-09 | ||
| US201862651284P | 2018-04-02 | 2018-04-02 | |
| US62/651,284 | 2018-04-02 | ||
| US201862694976P | 2018-07-07 | 2018-07-07 | |
| US62/694,976 | 2018-07-07 | ||
| PCT/US2018/050848 WO2019055633A1 (en) | 2017-09-14 | 2018-09-13 | RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112020003126A2 true BR112020003126A2 (pt) | 2020-10-13 |
Family
ID=65630620
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112020003126-7A BR112020003126A2 (pt) | 2017-09-14 | 2018-09-13 | agentes de rnai e composições para inibição da expressão de angiopoietina tipo 3 (angptl3), e métodos de uso |
Country Status (24)
| Country | Link |
|---|---|
| US (2) | US10995335B2 (enExample) |
| EP (1) | EP3681513A4 (enExample) |
| JP (3) | JP7281452B2 (enExample) |
| KR (2) | KR102737464B1 (enExample) |
| CN (2) | CN111343994B (enExample) |
| AU (2) | AU2018331383B2 (enExample) |
| BR (1) | BR112020003126A2 (enExample) |
| CA (1) | CA3074320A1 (enExample) |
| CL (1) | CL2020000666A1 (enExample) |
| CO (1) | CO2020001740A2 (enExample) |
| EC (1) | ECSP20018305A (enExample) |
| IL (2) | IL273173B1 (enExample) |
| JO (1) | JOP20200050A1 (enExample) |
| MX (1) | MX2020001912A (enExample) |
| MY (1) | MY200087A (enExample) |
| NZ (1) | NZ763275A (enExample) |
| PE (1) | PE20201287A1 (enExample) |
| PH (1) | PH12020500309A1 (enExample) |
| SG (1) | SG11201912188RA (enExample) |
| TN (1) | TN2020000038A1 (enExample) |
| TW (2) | TWI823866B (enExample) |
| UY (1) | UY37881A (enExample) |
| WO (1) | WO2019055633A1 (enExample) |
| ZA (1) | ZA202103669B (enExample) |
Families Citing this family (78)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6110372B2 (ja) | 2011-06-21 | 2017-04-05 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | アンジオポエチン様3(ANGPTL3)iRNA組成物及びその使用方法 |
| TW202507005A (zh) | 2015-04-13 | 2025-02-16 | 美商阿尼拉製藥公司 | 類血管生成素3(ANGPTL3)iRNA組成物及其用途方法 |
| KR102856522B1 (ko) * | 2016-09-02 | 2025-09-08 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
| CN110573013B (zh) | 2017-03-17 | 2021-10-29 | 明治制果药业株式会社 | 植物病害防除剂 |
| CN110520531A (zh) | 2017-04-05 | 2019-11-29 | 赛伦斯治疗有限责任公司 | 产品和组合物 |
| EP4013870A1 (en) | 2019-08-13 | 2022-06-22 | Alnylam Pharmaceuticals, Inc. | Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof |
| JP2023500661A (ja) | 2019-11-01 | 2023-01-10 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | ハンチンチン(HTT)iRNA剤組成物およびその使用方法 |
| WO2021096763A1 (en) | 2019-11-13 | 2021-05-20 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating an angiotensinogen- (agt-) associated disorder |
| CN115151641A (zh) | 2019-12-13 | 2022-10-04 | 阿尔尼拉姆医药品有限公司 | 人类染色体9开放阅读框72(C9ORF72)iRNA剂组合物及其使用方法 |
| WO2021126734A1 (en) | 2019-12-16 | 2021-06-24 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| JP7735288B2 (ja) | 2020-02-18 | 2025-09-08 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | アポリポタンパク質C3(APOC3)iRNA組成物およびその使用法 |
| WO2021178607A1 (en) | 2020-03-05 | 2021-09-10 | Alnylam Pharmaceuticals, Inc. | Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases |
| BR112022017822A2 (pt) | 2020-03-06 | 2022-11-08 | Alnylam Pharmaceuticals Inc | Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas |
| EP4121534A1 (en) | 2020-03-18 | 2023-01-25 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| BR112022018667A2 (pt) * | 2020-03-18 | 2022-11-29 | Dicerna Pharmaceuticals Inc | Composições e métodos para inibir a expressão de ang-ptl3 |
| TW202204615A (zh) | 2020-03-26 | 2022-02-01 | 美商阿尼拉製藥公司 | 冠狀病毒iRNA組成物及其使用方法 |
| CN115397436B (zh) * | 2020-03-26 | 2025-07-08 | 箭头药业股份有限公司 | 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法 |
| WO2021206922A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof |
| WO2021206917A1 (en) | 2020-04-07 | 2021-10-14 | Alnylam Pharmaceuticals, Inc. | ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| CN115955972A (zh) | 2020-04-27 | 2023-04-11 | 阿尔尼拉姆医药品有限公司 | 载脂蛋白E(APOE)iRNA剂组合物及其使用方法 |
| JP2023523790A (ja) | 2020-04-30 | 2023-06-07 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | 補体因子B(CFB)iRNA組成物およびその使用方法 |
| EP4162050A1 (en) | 2020-06-09 | 2023-04-12 | Alnylam Pharmaceuticals, Inc. | Rnai compositions and methods of use thereof for delivery by inhalation |
| CN116209760A (zh) | 2020-06-18 | 2023-06-02 | 阿尔尼拉姆医药品有限公司 | 黄嘌呤脱氢酶(XDH)iRNA组合物及其使用方法 |
| EP4217489A1 (en) | 2020-09-24 | 2023-08-02 | Alnylam Pharmaceuticals, Inc. | Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof |
| WO2022068923A1 (zh) * | 2020-09-30 | 2022-04-07 | 纳肽得(青岛)生物医药有限公司 | 血管生成素样3(ANGPTL3)的siRNA及其用途 |
| JP2023544413A (ja) | 2020-10-05 | 2023-10-23 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | Gタンパク質共役受容体75(GPR75)iRNA組成物およびその使用方法 |
| WO2022079222A1 (en) * | 2020-10-16 | 2022-04-21 | Sanofi | Novel rna compositions and methods for inhibiting angptl3 |
| EP4232582A1 (en) | 2020-10-23 | 2023-08-30 | Alnylam Pharmaceuticals, Inc. | Mucin 5b (muc5b) irna compositions and methods of use thereof |
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| WO2022150260A1 (en) | 2021-01-05 | 2022-07-14 | Alnylam Pharmaceuticals, Inc. | COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2022182864A1 (en) | 2021-02-25 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | Prion protein (prnp) irna compositions and methods and methods of use thereof |
| WO2022182574A1 (en) | 2021-02-26 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| MX2023010249A (es) | 2021-03-04 | 2023-09-15 | Alnylam Pharmaceuticals Inc | Composiciones de arni de proteina similar a la angiopoyetina 3 (angptl3) y metodos para usarlas. |
| WO2022192519A1 (en) | 2021-03-12 | 2022-09-15 | Alnylam Pharmaceuticals, Inc. | Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof |
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