KR102254380B1 - SERPINC1 iRNA 조성물 및 그의 이용 방법 - Google Patents
SERPINC1 iRNA 조성물 및 그의 이용 방법 Download PDFInfo
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- KR102254380B1 KR102254380B1 KR1020207021811A KR20207021811A KR102254380B1 KR 102254380 B1 KR102254380 B1 KR 102254380B1 KR 1020207021811 A KR1020207021811 A KR 1020207021811A KR 20207021811 A KR20207021811 A KR 20207021811A KR 102254380 B1 KR102254380 B1 KR 102254380B1
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- irna
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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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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/31—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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- C—CHEMISTRY; METALLURGY
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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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- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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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
- C12N2310/351—Conjugate
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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
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
- C12N2310/3521—Methyl
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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
- C12N2310/3523—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
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
- Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10S530/00—Chemistry: natural resins or derivatives; peptides or proteins; lignins or reaction products thereof
- Y10S530/827—Proteins from mammals or birds
- Y10S530/828—Cancer
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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 |
|---|---|---|---|
| KR1020217014582A KR102375607B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
Applications Claiming Priority (10)
| 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 | 2013-03-15 | ||
| US13/837,129 US9127274B2 (en) | 2012-04-26 | 2013-03-15 | Serpinc1 iRNA compositions and methods of use thereof |
| KR1020147032288A KR102139918B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| PCT/US2013/038218 WO2013163430A2 (en) | 2012-04-26 | 2013-04-25 | Serpinc1 irna compositions and methods of use thereof |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020147032288A Division KR102139918B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217014582A Division KR102375607B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20200093691A KR20200093691A (ko) | 2020-08-05 |
| KR102254380B1 true KR102254380B1 (ko) | 2021-05-25 |
Family
ID=49484030
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020207021811A Active KR102254380B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020227008361A Pending KR20220038521A (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020257029360A Pending KR20250134150A (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020147032288A Active KR102139918B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020217014582A Active KR102375607B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227008361A Pending KR20220038521A (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020257029360A Pending KR20250134150A (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020147032288A Active KR102139918B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
| KR1020217014582A Active KR102375607B1 (ko) | 2012-04-26 | 2013-04-25 | SERPINC1 iRNA 조성물 및 그의 이용 방법 |
Country Status (24)
| Country | Link |
|---|---|
| US (6) | US9127274B2 (https=) |
| EP (2) | EP2841443B1 (https=) |
| JP (5) | JP6466832B2 (https=) |
| KR (5) | KR102254380B1 (https=) |
| CN (4) | CN104520310B (https=) |
| AR (1) | AR090869A1 (https=) |
| AU (5) | AU2013251494A1 (https=) |
| CA (1) | CA2869922A1 (https=) |
| CL (1) | CL2016000188A1 (https=) |
| CO (1) | CO7240361A2 (https=) |
| ES (1) | ES2934152T3 (https=) |
| FI (1) | FI2841443T3 (https=) |
| HR (1) | HRP20221498T1 (https=) |
| HU (1) | HUE060846T2 (https=) |
| IL (4) | IL234941B (https=) |
| LT (1) | LT2841443T (https=) |
| MX (2) | MX365419B (https=) |
| PL (1) | PL2841443T3 (https=) |
| PT (1) | PT2841443T (https=) |
| RS (1) | RS63838B1 (https=) |
| RU (1) | RU2691580C2 (https=) |
| SI (1) | SI2841443T1 (https=) |
| WO (1) | WO2013163430A2 (https=) |
| ZA (2) | ZA201407293B (https=) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| EP2780454A2 (en) * | 2011-11-18 | 2014-09-24 | Alnylam Pharmaceuticals, Inc. | Modified rnai agents |
| US9127274B2 (en) | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| US9703335B2 (en) * | 2013-01-14 | 2017-07-11 | Dell Products L.P. | Information handling system chassis with anisotropic conductance |
| GB201322091D0 (en) | 2013-12-13 | 2014-01-29 | Cambridge Entpr Ltd | Modified serpins for the treatment of bleeding disorders |
| AU2015217301A1 (en) * | 2014-02-11 | 2016-08-25 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (KHK) iRNA compositions and methods of use thereof |
| FI3628334T3 (fi) | 2014-03-21 | 2023-09-15 | Genzyme Corp | Geenihoito verkkokalvon pigmenttirappeuma |
| 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 |
| CA3215908A1 (en) * | 2014-05-22 | 2015-11-26 | Alnylam Pharmaceuticals, Inc. | Angiotensinogen (agt) irna compositions and methods of use thereof |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| TWI864340B (zh) * | 2014-10-10 | 2024-12-01 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| EP3212794B1 (en) * | 2014-10-30 | 2021-04-07 | Genzyme Corporation | Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof |
| US10689647B2 (en) | 2015-05-08 | 2020-06-23 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA |
| FI3386518T3 (fi) * | 2015-12-07 | 2025-09-25 | Genzyme Corp | Menetelmiä ja koostumuksia serpinc1:een liittyvän häiriön hoitamiseksi |
| US10020886B2 (en) * | 2016-10-17 | 2018-07-10 | Panduit Corp. | Methods and systems for fiber optic communication |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| JP7155239B2 (ja) | 2017-04-05 | 2022-10-18 | サイレンス・セラピューティクス・ゲーエムベーハー | 産物及び組成物 |
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| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
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| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| 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 |
| CA3118537A1 (en) | 2018-11-09 | 2020-05-14 | Alnylam Pharmaceuticals, Inc. | Modified double stranded oligonucleotides |
| TW202039844A (zh) * | 2018-12-19 | 2020-11-01 | 美商阿尼拉製藥公司 | 類澱粉前驅蛋白(APP)RNAi劑組成物及其使用方法 |
| KR20210116509A (ko) | 2019-01-16 | 2021-09-27 | 젠자임 코포레이션 | Serpinc1 irna 조성물 및 그의 이용 방법 |
| EP3953473A1 (en) | 2019-04-12 | 2022-02-16 | MiNA Therapeutics Limited | Sirt1-sarna compositions and methods of use |
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