KR20250171391A - 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법 - Google Patents

전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법

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KR20250171391A
KR20250171391A KR1020257038513A KR20257038513A KR20250171391A KR 20250171391 A KR20250171391 A KR 20250171391A KR 1020257038513 A KR1020257038513 A KR 1020257038513A KR 20257038513 A KR20257038513 A KR 20257038513A KR 20250171391 A KR20250171391 A KR 20250171391A
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rnai agent
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피터 빈가드
데이비드 칼렌드
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노파르티스 아게
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Application filed by 노파르티스 아게 filed Critical 노파르티스 아게
Publication of KR20250171391A publication Critical patent/KR20250171391A/ko
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
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    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21061Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3521Methyl
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • 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)
KR1020257038513A 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법 Pending KR20250171391A (ko)

Applications Claiming Priority (10)

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
KR1020247014347A KR20240067272A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020247014347A Division KR20240067272A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법

Publications (1)

Publication Number Publication Date
KR20250171391A true KR20250171391A (ko) 2025-12-08

Family

ID=60388127

Family Applications (3)

Application Number Title Priority Date Filing Date
KR1020257038513A Pending KR20250171391A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법
KR1020247014347A Ceased KR20240067272A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법
KR1020197014059A Ceased KR20190077411A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법

Family Applications After (2)

Application Number Title Priority Date Filing Date
KR1020247014347A Ceased KR20240067272A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법
KR1020197014059A Ceased KR20190077411A (ko) 2016-10-18 2017-10-18 전구 단백질 전환효소 섭틸리신 켁신 9 (pcsk9) 단백질 감소를 통한 심혈관 사건의 예방 방법

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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Publication number Publication date
JP2025016469A (ja) 2025-02-04
WO2018075658A1 (en) 2018-04-26
JP2026062719A (ja) 2026-04-10
HRP20240930T1 (hr) 2024-10-11
CN110088283A (zh) 2019-08-02
CN119700800A (zh) 2025-03-28
DK3529360T3 (da) 2024-07-15
JP7566815B2 (ja) 2024-10-15
SI3529360T1 (sl) 2024-08-30
AU2017346879B2 (en) 2023-11-30
HUE067712T2 (hu) 2024-11-28
CN119345221A (zh) 2025-01-24
US20260108631A1 (en) 2026-04-23
LT3529360T (lt) 2024-07-25
EP3529360A1 (en) 2019-08-28
AU2023263519A1 (en) 2023-12-07
EP4397311A2 (en) 2024-07-10
JP7181881B2 (ja) 2022-12-01
AU2017346879A1 (en) 2019-05-23
RS65725B1 (sr) 2024-08-30
KR20240067272A (ko) 2024-05-16
AU2023263519B2 (en) 2026-02-12
CN119318663A (zh) 2025-01-17
PL3529360T3 (pl) 2024-08-26
US20210093736A1 (en) 2021-04-01
MX2024004495A (es) 2024-05-08
ES2982911T3 (es) 2024-10-18
CL2019001047A1 (es) 2020-05-08
IL266066A (en) 2019-06-30
BR112019007843A2 (pt) 2019-10-01
KR20190077411A (ko) 2019-07-03
CA3042423A1 (en) 2018-04-26
MX2019004573A (es) 2019-10-07
EP3529360B1 (en) 2024-04-17
JP2022109948A (ja) 2022-07-28
PT3529360T (pt) 2024-07-12
EP4397311A3 (en) 2024-10-16
US20180104360A1 (en) 2018-04-19
JP7850215B2 (ja) 2026-04-22
FI3529360T3 (fi) 2024-07-11
JP2019531357A (ja) 2019-10-31

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