CA3042423A1 - Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction - Google Patents
Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction Download PDFInfo
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- CA3042423A1 CA3042423A1 CA3042423A CA3042423A CA3042423A1 CA 3042423 A1 CA3042423 A1 CA 3042423A1 CA 3042423 A CA3042423 A CA 3042423A CA 3042423 A CA3042423 A CA 3042423A CA 3042423 A1 CA3042423 A1 CA 3042423A1
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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
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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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- 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
- C07H21/02—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 ribosyl as saccharide radical
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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
- 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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- 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
-
- 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
- C12N2310/10—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
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
-
- 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
-
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Vascular Medicine (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662409816P | 2016-10-18 | 2016-10-18 | |
| US62/409,816 | 2016-10-18 | ||
| US201662422028P | 2016-11-14 | 2016-11-14 | |
| US62/422,028 | 2016-11-14 | ||
| US201762472525P | 2017-03-16 | 2017-03-16 | |
| US62/472,525 | 2017-03-16 | ||
| US201762550426P | 2017-08-25 | 2017-08-25 | |
| US62/550,426 | 2017-08-25 | ||
| PCT/US2017/057218 WO2018075658A1 (en) | 2016-10-18 | 2017-10-18 | Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3042423A1 true CA3042423A1 (en) | 2018-04-26 |
Family
ID=60388127
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3042423A Pending CA3042423A1 (en) | 2016-10-18 | 2017-10-18 | Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US20180104360A1 (https=) |
| EP (2) | EP4397311A3 (https=) |
| JP (4) | JP7181881B2 (https=) |
| KR (3) | KR20250171391A (https=) |
| CN (4) | CN110088283A (https=) |
| AU (2) | AU2017346879B2 (https=) |
| CA (1) | CA3042423A1 (https=) |
| CL (1) | CL2019001047A1 (https=) |
| DK (1) | DK3529360T3 (https=) |
| ES (1) | ES2982911T3 (https=) |
| FI (1) | FI3529360T3 (https=) |
| HR (1) | HRP20240930T1 (https=) |
| HU (1) | HUE067712T2 (https=) |
| IL (1) | IL266066A (https=) |
| LT (1) | LT3529360T (https=) |
| MX (2) | MX2019004573A (https=) |
| PL (1) | PL3529360T3 (https=) |
| PT (1) | PT3529360T (https=) |
| RS (1) | RS65725B1 (https=) |
| SI (1) | SI3529360T1 (https=) |
| WO (1) | WO2018075658A1 (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL292159B1 (en) | 2012-12-05 | 2026-04-01 | Alnylam Pharmaceuticals Inc | PCSK9 iRNA compositions and methods of using them |
| CN108348541A (zh) | 2015-08-25 | 2018-07-31 | 阿尔尼拉姆医药品有限公司 | 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物 |
| EP4397311A3 (en) | 2016-10-18 | 2024-10-16 | Novartis AG | Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction |
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| EP3719126B1 (en) | 2017-12-01 | 2025-01-01 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| CA3083970A1 (en) | 2017-12-01 | 2019-06-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| DK3719128T3 (da) | 2017-12-01 | 2025-03-03 | Suzhou Ribo Life Science Co Ltd | Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf |
| EP3719127A4 (en) | 2017-12-01 | 2021-10-20 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE |
| CN110944675B9 (zh) | 2017-12-01 | 2024-08-09 | 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 |
| AU2018394875B2 (en) | 2017-12-29 | 2023-08-03 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| WO2020038377A1 (zh) | 2018-08-21 | 2020-02-27 | 苏州瑞博生物技术有限公司 | 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 |
| WO2020135673A1 (zh) | 2018-12-28 | 2020-07-02 | 苏州瑞博生物技术有限公司 | 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 |
| MX2021008661A (es) | 2019-01-18 | 2021-08-19 | Astrazeneca Ab | Inhibidores de la pcsk9 y metodos de uso de los mismos. |
| BR112021013807A2 (pt) | 2019-01-18 | 2021-11-30 | Astrazeneca Ab | Inibidores de pcsk9 e seus métodos de uso |
| KR20220011689A (ko) | 2019-05-22 | 2022-01-28 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도 |
| US12590304B2 (en) | 2019-05-22 | 2026-03-31 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| AU2020280439B2 (en) | 2019-05-22 | 2025-08-14 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| CN113795582B (zh) | 2019-05-24 | 2024-05-24 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
| CN115066498A (zh) | 2020-03-16 | 2022-09-16 | 阿尔戈诺特Rna有限公司 | Pcsk9的拮抗剂 |
| JP2025531341A (ja) * | 2022-09-19 | 2025-09-19 | カイロンノヴァ （シャーメン） バイオファーマ カンパニー，リミテッド | 炭水化物－オリゴヌクレオチド複合体、薬物組成物および治療への応用 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101484588B (zh) * | 2006-05-11 | 2013-11-06 | 阿尔尼拉姆医药品有限公司 | 抑制pcsk9基因表达的组合物和方法 |
| IL292159B1 (en) | 2012-12-05 | 2026-04-01 | Alnylam Pharmaceuticals Inc | PCSK9 iRNA compositions and methods of using them |
| CN108348541A (zh) * | 2015-08-25 | 2018-07-31 | 阿尔尼拉姆医药品有限公司 | 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物 |
| EP4397311A3 (en) | 2016-10-18 | 2024-10-16 | Novartis AG | Methods for preventing cardiovascular events through proprotein convertase subtilisin kexin 9 (pcsk9) protein reduction |
-
2017
- 2017-10-18 EP EP24170268.7A patent/EP4397311A3/en active Pending
- 2017-10-18 CA CA3042423A patent/CA3042423A1/en active Pending
- 2017-10-18 HU HUE17800976A patent/HUE067712T2/hu unknown
- 2017-10-18 MX MX2019004573A patent/MX2019004573A/es unknown
- 2017-10-18 CN CN201780077941.7A patent/CN110088283A/zh not_active Withdrawn
- 2017-10-18 PL PL17800976.7T patent/PL3529360T3/pl unknown
- 2017-10-18 RS RS20240774A patent/RS65725B1/sr unknown
- 2017-10-18 WO PCT/US2017/057218 patent/WO2018075658A1/en not_active Ceased
- 2017-10-18 CN CN202411482008.6A patent/CN119700800A/zh active Pending
- 2017-10-18 SI SI201731532T patent/SI3529360T1/sl unknown
- 2017-10-18 ES ES17800976T patent/ES2982911T3/es active Active
- 2017-10-18 US US15/787,405 patent/US20180104360A1/en not_active Abandoned
- 2017-10-18 KR KR1020257038513A patent/KR20250171391A/ko active Pending
- 2017-10-18 DK DK17800976.7T patent/DK3529360T3/da active
- 2017-10-18 KR KR1020247014347A patent/KR20240067272A/ko not_active Ceased
- 2017-10-18 PT PT178009767T patent/PT3529360T/pt unknown
- 2017-10-18 JP JP2019542356A patent/JP7181881B2/ja active Active
- 2017-10-18 CN CN202411481996.2A patent/CN119345221A/zh active Pending
- 2017-10-18 HR HRP20240930TT patent/HRP20240930T1/hr unknown
- 2017-10-18 KR KR1020197014059A patent/KR20190077411A/ko not_active Ceased
- 2017-10-18 FI FIEP17800976.7T patent/FI3529360T3/fi active
- 2017-10-18 AU AU2017346879A patent/AU2017346879B2/en active Active
- 2017-10-18 LT LTEPPCT/US2017/057218T patent/LT3529360T/lt unknown
- 2017-10-18 CN CN202411479127.6A patent/CN119318663A/zh active Pending
- 2017-10-18 EP EP17800976.7A patent/EP3529360B1/en active Active
-
2019
- 2019-04-16 IL IL266066A patent/IL266066A/en unknown
- 2019-04-17 CL CL2019001047A patent/CL2019001047A1/es unknown
- 2019-04-17 MX MX2024004495A patent/MX2024004495A/es unknown
-
2020
- 2020-06-22 US US16/908,517 patent/US20210093736A1/en active Pending
-
2022
- 2022-04-19 JP JP2022068684A patent/JP7566815B2/ja active Active
-
2023
- 2023-11-09 AU AU2023263519A patent/AU2023263519B2/en active Active
-
2024
- 2024-10-02 JP JP2024173304A patent/JP7850215B2/ja active Active
-
2025
- 2025-12-17 JP JP2025258789A patent/JP2026062719A/ja active Pending
- 2025-12-22 US US19/429,048 patent/US20260108631A1/en active Pending
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