JP6313789B2 - リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 - Google Patents
リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 Download PDFInfo
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- JP6313789B2 JP6313789B2 JP2015558162A JP2015558162A JP6313789B2 JP 6313789 B2 JP6313789 B2 JP 6313789B2 JP 2015558162 A JP2015558162 A JP 2015558162A JP 2015558162 A JP2015558162 A JP 2015558162A JP 6313789 B2 JP6313789 B2 JP 6313789B2
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- certain embodiments
- apociii
- modified
- lpld
- antisense
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- 0 *CC1(C2I)OC(*)C2OC1 Chemical compound *CC1(C2I)OC(*)C2OC1 0.000 description 2
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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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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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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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
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- A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
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- A61P3/00—Drugs for disorders of the metabolism
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
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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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- 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
- C12N2310/315—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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- 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
- C12N2310/322—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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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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- 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
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Steroid Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361764969P | 2013-02-14 | 2013-02-14 | |
| US61/764,969 | 2013-02-14 | ||
| US201361880779P | 2013-09-20 | 2013-09-20 | |
| US61/880,779 | 2013-09-20 | ||
| PCT/US2014/016546 WO2014127268A2 (en) | 2013-02-14 | 2014-02-14 | Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018054633A Division JP6681422B2 (ja) | 2013-02-14 | 2018-03-22 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016513110A JP2016513110A (ja) | 2016-05-12 |
| JP2016513110A5 JP2016513110A5 (enExample) | 2017-03-16 |
| JP6313789B2 true JP6313789B2 (ja) | 2018-04-18 |
Family
ID=51354689
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015558162A Active JP6313789B2 (ja) | 2013-02-14 | 2014-02-14 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
| JP2018054633A Active JP6681422B2 (ja) | 2013-02-14 | 2018-03-22 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
| JP2020050456A Withdrawn JP2020111594A (ja) | 2013-02-14 | 2020-03-23 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018054633A Active JP6681422B2 (ja) | 2013-02-14 | 2018-03-22 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
| JP2020050456A Withdrawn JP2020111594A (ja) | 2013-02-14 | 2020-03-23 | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
Country Status (16)
| Country | Link |
|---|---|
| US (4) | US9593333B2 (enExample) |
| EP (2) | EP3400947B1 (enExample) |
| JP (3) | JP6313789B2 (enExample) |
| KR (2) | KR102169899B1 (enExample) |
| CN (2) | CN108743943A (enExample) |
| AU (3) | AU2014216137B2 (enExample) |
| BR (1) | BR112015019515A8 (enExample) |
| CA (2) | CA3170716A1 (enExample) |
| DK (1) | DK2956176T3 (enExample) |
| ES (2) | ES2680599T3 (enExample) |
| IL (2) | IL240338B (enExample) |
| MX (2) | MX368155B (enExample) |
| NZ (1) | NZ631119A (enExample) |
| RU (2) | RU2018121529A (enExample) |
| WO (1) | WO2014127268A2 (enExample) |
| ZA (1) | ZA201505628B (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2018135341A (ja) * | 2013-02-14 | 2018-08-30 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20190062511A (ko) * | 2011-04-27 | 2019-06-05 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지방단백질 ciii (apociii) 발현의 조정 |
| AU2014284152B2 (en) * | 2013-06-21 | 2020-01-23 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation of target nucleic acids |
| HK1248522A1 (zh) * | 2015-02-27 | 2018-10-19 | Ionis Pharmaceuticals, Inc. | 调节脂肪代谢障碍群体中的载脂蛋白c-iii(apociii)表达 |
| CA3030099A1 (en) | 2016-07-08 | 2018-01-11 | Staten Biotechnology B.V. | Anti-apoc3 antibodies and methods of use thereof |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| JP2020517242A (ja) | 2017-04-21 | 2020-06-18 | スターテン・バイオテクノロジー・ベー・フェー | 抗ApoC3抗体およびその使用方法 |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| BR112020008514A2 (pt) | 2017-10-31 | 2020-10-20 | Staten Biotechnology B.V. | anticorpos anti-apoc3 e métodos de uso dos mesmos |
| US10538583B2 (en) | 2017-10-31 | 2020-01-21 | Staten Biotechnology B.V. | Anti-APOC3 antibodies and compositions thereof |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US20240033280A1 (en) * | 2020-10-02 | 2024-02-01 | Ionis Pharmaceuticals, Inc. | Methods for reducing apociii expression |
| CN116814621B (zh) * | 2022-08-05 | 2025-09-09 | 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 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2018135341A (ja) * | 2013-02-14 | 2018-08-30 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | リポタンパク質リパーゼ欠損(lpld)集団におけるアポリポタンパク質c−iii(apociii)発現の調節 |
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