JP2022546040A - Pcsk9を阻害するための組成物および方法 - Google Patents
Pcsk9を阻害するための組成物および方法 Download PDFInfo
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- JP2022546040A JP2022546040A JP2022513127A JP2022513127A JP2022546040A JP 2022546040 A JP2022546040 A JP 2022546040A JP 2022513127 A JP2022513127 A JP 2022513127A JP 2022513127 A JP2022513127 A JP 2022513127A JP 2022546040 A JP2022546040 A JP 2022546040A
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- dsrna
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- pcsk9
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/1137—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 enzymes
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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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
- C12N2310/351—Conjugate
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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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/50—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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024180454A JP2025013845A (ja) | 2019-08-27 | 2024-10-16 | Pcsk9を阻害するための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP19306036.5 | 2019-08-27 | ||
| EP19306036 | 2019-08-27 | ||
| PCT/EP2020/073961 WO2021037972A1 (en) | 2019-08-27 | 2020-08-27 | Compositions and methods for inhibiting pcsk9 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024180454A Division JP2025013845A (ja) | 2019-08-27 | 2024-10-16 | Pcsk9を阻害するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022546040A true JP2022546040A (ja) | 2022-11-02 |
| JP2022546040A5 JP2022546040A5 (https=) | 2023-09-04 |
| JPWO2021037972A5 JPWO2021037972A5 (https=) | 2023-09-04 |
Family
ID=67956661
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022513127A Pending JP2022546040A (ja) | 2019-08-27 | 2020-08-27 | Pcsk9を阻害するための組成物および方法 |
| JP2024180454A Pending JP2025013845A (ja) | 2019-08-27 | 2024-10-16 | Pcsk9を阻害するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024180454A Pending JP2025013845A (ja) | 2019-08-27 | 2024-10-16 | Pcsk9を阻害するための組成物および方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20220290156A1 (https=) |
| EP (1) | EP4022061A1 (https=) |
| JP (2) | JP2022546040A (https=) |
| CN (1) | CN115176011B (https=) |
| WO (1) | WO2021037972A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022547888A (ja) * | 2019-09-05 | 2022-11-16 | サノフイ | ヌクレオチド類似体を含有するオリゴヌクレオチド |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| EP3884051A2 (en) | 2018-11-23 | 2021-09-29 | Sanofi | Novel rna compositions and methods for inhibiting angptl8 |
| WO2024002006A1 (zh) * | 2022-06-27 | 2024-01-04 | 大睿生物医药科技(上海)有限公司 | 具有增强的稳定性的核苷酸替代物 |
| JP2025532985A (ja) | 2022-09-30 | 2025-10-03 | 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 | 修飾二本鎖rna剤 |
| WO2024228030A2 (en) * | 2023-05-04 | 2024-11-07 | Argonaute RNA Limited | Dual silencing |
| CN121866332A (zh) * | 2023-06-21 | 2026-04-14 | 美国圣因生物股份有限公司 | 靶向前蛋白转化酶枯草杆菌蛋白酶kexin 9(pcsk9)的双链rna及其使用方法 |
| WO2025077949A1 (zh) * | 2023-11-06 | 2025-04-17 | 杭州天龙药业有限公司 | 提升双链寡核苷酸靶基因抑制效果的修饰模板及其组合与应用 |
Citations (5)
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| JP2010503382A (ja) * | 2006-07-17 | 2010-02-04 | サーナ・セラピューティクス・インコーポレイテッド | 低分子干渉核酸(siNA)を用いたプロタンパク質転換酵素サブチリシンケクシン9(PCSK9)遺伝子発現のRNA干渉媒介性阻害 |
| JP2012530143A (ja) * | 2009-06-15 | 2012-11-29 | 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 | Pcsk9遺伝子を標的とする、脂質で製剤化されたdsrna |
| JP2016506240A (ja) * | 2012-12-05 | 2016-03-03 | 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. | PCSK9iRNA組成物及びその使用方法 |
| WO2019170731A1 (en) * | 2018-03-07 | 2019-09-12 | Sanofi | Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same |
| WO2019204021A1 (en) * | 2018-04-18 | 2019-10-24 | Dicerna Pharmaceuticals, Inc. | Pcsk9 targeting oligonucleotides for treating hypercholesterolemia and related conditions |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5665710A (en) | 1990-04-30 | 1997-09-09 | Georgetown University | Method of making liposomal oligodeoxynucleotide compositions |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| WO1994002595A1 (en) | 1992-07-17 | 1994-02-03 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of animal diseases |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6054299A (en) | 1994-04-29 | 2000-04-25 | Conrad; Charles A. | Stem-loop cloning vector and method |
| US5981501A (en) | 1995-06-07 | 1999-11-09 | Inex Pharmaceuticals Corp. | Methods for encapsulating plasmids in lipid bilayers |
| ATE285477T1 (de) | 1995-06-07 | 2005-01-15 | Inex Pharmaceutical Corp | Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer |
| US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| NZ507093A (en) | 1998-04-08 | 2003-08-29 | Commw Scient Ind Res Org | Methods and means for reducing the phenotypic expression of a nucleic acid of interest in a plant |
| AR020078A1 (es) | 1998-05-26 | 2002-04-10 | Syngenta Participations Ag | Metodo para alterar la expresion de un gen objetivo en una celula de planta |
| WO2000003683A2 (en) | 1998-07-20 | 2000-01-27 | Inex Pharmaceuticals Corporation | Liposomal encapsulated nucleic acid-complexes |
| BR9914773A (pt) | 1998-10-09 | 2002-02-05 | Ingene Inc | Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica |
| MXPA01003642A (es) | 1998-10-09 | 2003-07-21 | Ingene Inc | Sintesis enzimatica de adnss. |
| DE19956568A1 (de) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| CN100365122C (zh) * | 2005-12-30 | 2008-01-30 | 中国疾病预防控制中心病毒病预防控制所 | 针对乙型流感病毒多聚酶基因的siRNA序列及其应用 |
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| US20100249214A1 (en) | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
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-
2020
- 2020-08-27 US US17/638,339 patent/US20220290156A1/en active Pending
- 2020-08-27 EP EP20761581.6A patent/EP4022061A1/en active Pending
- 2020-08-27 JP JP2022513127A patent/JP2022546040A/ja active Pending
- 2020-08-27 WO PCT/EP2020/073961 patent/WO2021037972A1/en not_active Ceased
- 2020-08-27 CN CN202080073296.3A patent/CN115176011B/zh active Active
-
2024
- 2024-10-16 JP JP2024180454A patent/JP2025013845A/ja active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010503382A (ja) * | 2006-07-17 | 2010-02-04 | サーナ・セラピューティクス・インコーポレイテッド | 低分子干渉核酸(siNA)を用いたプロタンパク質転換酵素サブチリシンケクシン9(PCSK9)遺伝子発現のRNA干渉媒介性阻害 |
| JP2012530143A (ja) * | 2009-06-15 | 2012-11-29 | 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 | Pcsk9遺伝子を標的とする、脂質で製剤化されたdsrna |
| JP2016506240A (ja) * | 2012-12-05 | 2016-03-03 | 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. | PCSK9iRNA組成物及びその使用方法 |
| WO2019170731A1 (en) * | 2018-03-07 | 2019-09-12 | Sanofi | Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same |
| WO2019204021A1 (en) * | 2018-04-18 | 2019-10-24 | Dicerna Pharmaceuticals, Inc. | Pcsk9 targeting oligonucleotides for treating hypercholesterolemia and related conditions |
Non-Patent Citations (1)
| Title |
|---|
| FRANK-KAMENETSKY, M. ET AL.: "Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in", PROC. NATL. ACAD. SCI. U. S. A., vol. 105, JPN6024027914, 2008, pages 11915 - 11920, ISSN: 0005502476 * |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022547888A (ja) * | 2019-09-05 | 2022-11-16 | サノフイ | ヌクレオチド類似体を含有するオリゴヌクレオチド |
| JP7667775B2 (ja) | 2019-09-05 | 2025-04-23 | サノフイ | ヌクレオチド類似体を含有するオリゴヌクレオチド |
Also Published As
| Publication number | Publication date |
|---|---|
| CN115176011B (zh) | 2025-11-11 |
| EP4022061A1 (en) | 2022-07-06 |
| US20220290156A1 (en) | 2022-09-15 |
| JP2025013845A (ja) | 2025-01-28 |
| CN115176011A (zh) | 2022-10-11 |
| WO2021037972A1 (en) | 2021-03-04 |
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