JP6466832B2 - Serpinc1iRNA組成物およびその使用方法 - Google Patents
Serpinc1iRNA組成物およびその使用方法 Download PDFInfo
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- JP6466832B2 JP6466832B2 JP2015509141A JP2015509141A JP6466832B2 JP 6466832 B2 JP6466832 B2 JP 6466832B2 JP 2015509141 A JP2015509141 A JP 2015509141A JP 2015509141 A JP2015509141 A JP 2015509141A JP 6466832 B2 JP6466832 B2 JP 6466832B2
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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
- 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
- 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
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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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- 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
- C12N2310/335—Modified T or U
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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
- C12N2310/351—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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3523—Allyl
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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
- C12N2310/35—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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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019002713A JP6933670B2 (ja) | 2012-04-26 | 2019-01-10 | Serpinc1 iRNA組成物およびその使用方法 |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261638952P | 2012-04-26 | 2012-04-26 | |
| US61/638,952 | 2012-04-26 | ||
| US201261669249P | 2012-07-09 | 2012-07-09 | |
| US61/669,249 | 2012-07-09 | ||
| US201261734573P | 2012-12-07 | 2012-12-07 | |
| US61/734,573 | 2012-12-07 | ||
| US13/837,129 US9127274B2 (en) | 2012-04-26 | 2013-03-15 | Serpinc1 iRNA compositions and methods of use thereof |
| US13/837,129 | 2013-03-15 | ||
| PCT/US2013/038218 WO2013163430A2 (en) | 2012-04-26 | 2013-04-25 | Serpinc1 irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019002713A Division JP6933670B2 (ja) | 2012-04-26 | 2019-01-10 | Serpinc1 iRNA組成物およびその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015519047A JP2015519047A (ja) | 2015-07-09 |
| JP2015519047A5 JP2015519047A5 (enExample) | 2016-06-16 |
| JP6466832B2 true JP6466832B2 (ja) | 2019-02-06 |
Family
ID=49484030
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015509141A Active JP6466832B2 (ja) | 2012-04-26 | 2013-04-25 | Serpinc1iRNA組成物およびその使用方法 |
| JP2019002713A Active JP6933670B2 (ja) | 2012-04-26 | 2019-01-10 | Serpinc1 iRNA組成物およびその使用方法 |
| JP2021134158A Active JP7478121B2 (ja) | 2012-04-26 | 2021-08-19 | Serpinc1 iRNA組成物およびその使用方法 |
| JP2024068113A Pending JP2024099663A (ja) | 2012-04-26 | 2024-04-19 | Serpinc1 iRNA組成物およびその使用方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019002713A Active JP6933670B2 (ja) | 2012-04-26 | 2019-01-10 | Serpinc1 iRNA組成物およびその使用方法 |
| JP2021134158A Active JP7478121B2 (ja) | 2012-04-26 | 2021-08-19 | Serpinc1 iRNA組成物およびその使用方法 |
| JP2024068113A Pending JP2024099663A (ja) | 2012-04-26 | 2024-04-19 | Serpinc1 iRNA組成物およびその使用方法 |
Country Status (24)
| Country | Link |
|---|---|
| US (6) | US9127274B2 (enExample) |
| EP (2) | EP2841443B1 (enExample) |
| JP (4) | JP6466832B2 (enExample) |
| KR (5) | KR102139918B1 (enExample) |
| CN (4) | CN104520310B (enExample) |
| AR (1) | AR090869A1 (enExample) |
| AU (5) | AU2013251494A1 (enExample) |
| CA (1) | CA2869922A1 (enExample) |
| CL (1) | CL2016000188A1 (enExample) |
| CO (1) | CO7240361A2 (enExample) |
| ES (1) | ES2934152T3 (enExample) |
| FI (1) | FI2841443T3 (enExample) |
| HR (1) | HRP20221498T1 (enExample) |
| HU (1) | HUE060846T2 (enExample) |
| IL (4) | IL234941B (enExample) |
| LT (1) | LT2841443T (enExample) |
| MX (2) | MX365419B (enExample) |
| PL (1) | PL2841443T3 (enExample) |
| PT (1) | PT2841443T (enExample) |
| RS (1) | RS63838B1 (enExample) |
| RU (1) | RU2691580C2 (enExample) |
| SI (1) | SI2841443T1 (enExample) |
| WO (1) | WO2013163430A2 (enExample) |
| ZA (2) | ZA201407293B (enExample) |
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| KR20250133484A (ko) * | 2011-11-18 | 2025-09-05 | 알닐람 파마슈티칼스 인코포레이티드 | 변형된 RNAi 제제 |
| US9127274B2 (en) | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
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| GB201322091D0 (en) | 2013-12-13 | 2014-01-29 | Cambridge Entpr Ltd | Modified serpins for the treatment of bleeding disorders |
| EA201691587A1 (ru) * | 2014-02-11 | 2017-01-30 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| ES2760263T3 (es) * | 2014-03-21 | 2020-05-13 | Genzyme Corp | Tratamiento génico de la retinitis pigmentaria |
| TW201607559A (zh) * | 2014-05-12 | 2016-03-01 | 阿尼拉製藥公司 | 治療serpinc1相關疾患之方法和組成物 |
| JP6811094B2 (ja) * | 2014-05-22 | 2021-01-13 | 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. | アンジオテンシノーゲン(AGT)iRNA組成物およびその使用 |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
| WO2016069694A2 (en) * | 2014-10-30 | 2016-05-06 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof |
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| TWI851574B (zh) | 2018-05-14 | 2024-08-11 | 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 |
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