BR112017021053A2 - composições de irna de angiopoietina do tipo 3 (angptl3) e métodos de uso do mesmo - Google Patents
composições de irna de angiopoietina do tipo 3 (angptl3) e métodos de uso do mesmoInfo
- Publication number
- BR112017021053A2 BR112017021053A2 BR112017021053A BR112017021053A BR112017021053A2 BR 112017021053 A2 BR112017021053 A2 BR 112017021053A2 BR 112017021053 A BR112017021053 A BR 112017021053A BR 112017021053 A BR112017021053 A BR 112017021053A BR 112017021053 A2 BR112017021053 A2 BR 112017021053A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- angpt13
- compositions
- irna compositions
- dsrna
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 102000009840 Angiopoietins Human genes 0.000 title 1
- 108010009906 Angiopoietins Proteins 0.000 title 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 208000031226 Hyperlipidaemia Diseases 0.000 abstract 1
- 208000017170 Lipid metabolism disease Diseases 0.000 abstract 1
- 208000006575 hypertriglyceridemia Diseases 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 229920002477 rna polymer Polymers 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
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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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- 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
- 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
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/34—Spatial arrangement of the modifications
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- C12N2310/30—Chemical structure
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- 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
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
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- Medicinal Chemistry (AREA)
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- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
Abstract
a presente invenção refere-se às composições de ácido ribonucleico de filamento duplo (dsrna) visando o gene angptl3, bem como os métodos de inibição da expressão de angptl3 e métodos de tratamento de indivíduos com um distúrbio do metabolismo lipídico, tais como hiperlipidemia ou hipertrigliceridemia, usando tais composições de dsrna.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562146604P | 2015-04-13 | 2015-04-13 | |
US201562261361P | 2015-12-01 | 2015-12-01 | |
PCT/US2016/027271 WO2016168286A1 (en) | 2015-04-13 | 2016-04-13 | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112017021053A2 true BR112017021053A2 (pt) | 2018-08-14 |
Family
ID=55806850
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112017021053A BR112017021053A2 (pt) | 2015-04-13 | 2016-04-13 | composições de irna de angiopoietina do tipo 3 (angptl3) e métodos de uso do mesmo |
Country Status (13)
Country | Link |
---|---|
US (3) | US10570393B2 (pt) |
EP (1) | EP3283631A1 (pt) |
JP (3) | JP6975641B2 (pt) |
KR (1) | KR20170135932A (pt) |
CN (2) | CN107743522B (pt) |
AU (2) | AU2016247922B2 (pt) |
BR (1) | BR112017021053A2 (pt) |
CA (1) | CA2982450A1 (pt) |
EA (1) | EA201792263A1 (pt) |
IL (2) | IL295159A (pt) |
MX (2) | MX2017013102A (pt) |
TW (2) | TWI723986B (pt) |
WO (1) | WO2016168286A1 (pt) |
Families Citing this family (24)
Publication number | Priority date | Publication date | Assignee | Title |
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RU2711799C2 (ru) | 2011-06-21 | 2020-01-22 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ иРНК ДЛЯ СХОДНОГО С АНГИОПОЭТИНОМ 3(ANGPTL3) БЕЛКА И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
TWI723986B (zh) | 2015-04-13 | 2021-04-11 | 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 |
CN117701562A (zh) * | 2017-09-14 | 2024-03-15 | 箭头药业股份有限公司 | 用于抑制血管生成素-样3(ANGPTL3)的表达的RNAi剂和组合物以及使用方法 |
US11660347B2 (en) | 2017-12-01 | 2023-05-30 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing same, preparation method, and use thereof |
WO2019105418A1 (zh) * | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 |
CN110945130B (zh) * | 2017-12-01 | 2024-04-09 | 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 |
US11414665B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
CN116375774A (zh) | 2017-12-29 | 2023-07-04 | 苏州瑞博生物技术股份有限公司 | 缀合物及其制备方法和用途 |
TW202016304A (zh) * | 2018-05-14 | 2020-05-01 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
CN111655849B (zh) | 2018-08-21 | 2024-05-10 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的药物组合物和缀合物及其用途 |
US11896674B2 (en) | 2018-09-30 | 2024-02-13 | Suzhou Ribo Life Science Co., Ltd. | SiRNA conjugate, preparation method therefor and use thereof |
EP3877524A4 (en) * | 2018-11-09 | 2023-10-11 | Alnylam Pharmaceuticals Inc. | MODIFIED DOUBLE-STRANDED OLIGONUCLEOTIDES |
WO2020099482A2 (en) * | 2018-11-13 | 2020-05-22 | Lipigon Pharmaceuticals Ab | Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism |
JP2023521347A (ja) * | 2020-04-07 | 2023-05-24 | ユニキュアー アイピー ビー.ブイ. | アンジオポエチン様3(angptl3)をサイレンシングするための遺伝子構築物及びその使用 |
CN116887842A (zh) * | 2020-10-16 | 2023-10-13 | 赛诺菲 | 用于抑制angptl3的新型rna组合物和方法 |
CA3212128A1 (en) * | 2021-03-04 | 2022-09-09 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
WO2023044458A1 (en) * | 2021-09-17 | 2023-03-23 | Alnylam Pharmaceuticals, Inc. | Irna compositions and methods for silencing angptl4 |
CN116888263A (zh) * | 2021-09-23 | 2023-10-13 | 上海舶望制药有限公司 | 用于抑制血管生成素样3(angptl3)蛋白表达的组合物和方法 |
WO2023092102A1 (en) * | 2021-11-19 | 2023-05-25 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof |
WO2023122656A1 (en) | 2021-12-22 | 2023-06-29 | Regeneron Pharmaceuticals, Inc. | Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors |
WO2023134705A1 (zh) * | 2022-01-11 | 2023-07-20 | 上海金中锘美生物医药科技有限公司 | 抑制angptl3表达的rna干扰剂及其用途 |
CN116987696A (zh) * | 2022-08-04 | 2023-11-03 | 北京福元医药股份有限公司 | 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途 |
WO2024032608A1 (zh) * | 2022-08-08 | 2024-02-15 | 大睿生物医药科技(上海)有限公司 | 调控ANGPTL3基因活性的siRNA分子 |
US20240158795A1 (en) | 2022-11-10 | 2024-05-16 | Regeneron Pharmaceuticals, Inc. | Treatment Of Kidney Diseases With A Combination of Angiopoietin Like 3 (ANGPTL3) Inhibitors And Solute Carrier Family 5 Member 2 (SLC5A2) Inhibitors |
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CA2982450A1 (en) | 2016-10-20 |
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CN107743522A (zh) | 2018-02-27 |
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US10570393B2 (en) | 2020-02-25 |
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AU2016247922A1 (en) | 2017-11-02 |
EA201792263A1 (ru) | 2018-08-31 |
US20200224201A1 (en) | 2020-07-16 |
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AU2022204634A1 (en) | 2022-07-21 |
JP2018512155A (ja) | 2018-05-17 |
JP2022002518A (ja) | 2022-01-11 |
TW202124714A (zh) | 2021-07-01 |
EP3283631A1 (en) | 2018-02-21 |
US11198872B2 (en) | 2021-12-14 |
WO2016168286A1 (en) | 2016-10-20 |
JP6975641B2 (ja) | 2021-12-01 |
CN115927335A (zh) | 2023-04-07 |
MX2017013102A (es) | 2018-02-23 |
US20220119815A1 (en) | 2022-04-21 |
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