JP2023519140A - Ｐｃｓｋ９のアンタゴニスト - Google Patents

Ｐｃｓｋ９のアンタゴニスト Download PDF

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JP2023519140A
JP2023519140A JP2022552336A JP2022552336A JP2023519140A JP 2023519140 A JP2023519140 A JP 2023519140A JP 2022552336 A JP2022552336 A JP 2022552336A JP 2022552336 A JP2022552336 A JP 2022552336A JP 2023519140 A JP2023519140 A JP 2023519140A
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nucleic acid
molecule
acid molecule
seq
strand
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Japanese (ja)
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JPWO2021185765A5 (https=
JP2023519140A5 (https=
Inventor
ミッチェル，ダニエル
カーン，マイケル
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アルゴノート アールエヌエー リミテッド
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Priority claimed from GBGB2003756.0A external-priority patent/GB202003756D0/en
Priority claimed from GBGB2010276.0A external-priority patent/GB202010276D0/en
Priority claimed from GBGB2013998.6A external-priority patent/GB202013998D0/en
Priority claimed from GBGB2020553.0A external-priority patent/GB202020553D0/en
Application filed by アルゴノート アールエヌエー リミテッド filed Critical アルゴノート アールエヌエー リミテッド
Publication of JP2023519140A publication Critical patent/JP2023519140A/ja
Publication of JPWO2021185765A5 publication Critical patent/JPWO2021185765A5/ja
Publication of JP2023519140A5 publication Critical patent/JP2023519140A5/ja
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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
    • 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
    • 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
    • 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
    • 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)
    • C12Y304/21111—Aqualysin 1 (3.4.21.111)
    • 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)
    • C12Y304/21112—Site-1 protease (3.4.21.112), i.e. subtilisin kexin isozyme-1
    • 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
    • C12N2310/11—Antisense
    • 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]
    • 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
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022552336A 2020-03-16 2021-03-15 Ｐｃｓｋ９のアンタゴニスト Ceased JP2023519140A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
GBGB2003756.0A GB202003756D0 (en) 2020-03-16 2020-03-16 Antagonist of PCSK9
GB2003756.0 2020-03-16
GBGB2010276.0A GB202010276D0 (en) 2020-07-03 2020-07-03 PCSK9 antagonist
GB2010276.0 2020-07-03
GB2013998.6 2020-09-07
GBGB2013998.6A GB202013998D0 (en) 2020-09-07 2020-09-07 Antagonist of pcsk9
GB2020553.0 2020-12-23
GBGB2020553.0A GB202020553D0 (en) 2020-12-23 2020-12-23 Antagonist of pcsk9
PCT/EP2021/056540 WO2021185765A1 (en) 2020-03-16 2021-03-15 Antagonist of pcsk9

Publications (3)

Publication Number Publication Date
JP2023519140A true JP2023519140A (ja) 2023-05-10
JPWO2021185765A5 JPWO2021185765A5 (https=) 2024-03-15
JP2023519140A5 JP2023519140A5 (https=) 2024-03-15

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JP2022552336A Ceased JP2023519140A (ja) 2020-03-16 2021-03-15 Ｐｃｓｋ９のアンタゴニスト

Country Status (7)

Country Link
US (1) US20230183694A1 (https=)
EP (1) EP4081642A1 (https=)
JP (1) JP2023519140A (https=)
CN (1) CN115066498A (https=)
CA (1) CA3167849A1 (https=)
GB (1) GB2594788B (https=)
WO (1) WO2021185765A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
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
GB202020534D0 (en) * 2020-12-23 2021-02-03 Argonaute Rna Ltd Conjugate
US20250304966A1 (en) * 2020-12-23 2025-10-02 Argonaute RNA Limited Treatment of cardiovascular disease
WO2023041508A2 (en) * 2021-09-14 2023-03-23 Argonaute RNA Limited Treatment of cardiovascular disease
GB2618915B (en) * 2022-05-18 2024-08-14 Argonaute Rna Ltd Treatment of cardiovascular disease
CN117106781B (zh) * 2023-10-16 2024-03-22 深圳市茵冠生物科技有限公司 修饰的核酸及其产品和应用
CN117210468B (zh) * 2023-11-06 2024-02-20 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN117384907B (zh) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 抑制PCSK9表达的siRNA分子及其应用
WO2025252223A1 (zh) * 2024-06-07 2025-12-11 迈威(上海)生物科技股份有限公司 一种抑制PCSK9基因表达的RNAi制剂及其应用

Citations (3)

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Publication number Priority date Publication date Assignee Title
WO2006125977A2 (en) * 2005-05-25 2006-11-30 The University Of York Hybrid interfering rna
US20110065644A1 (en) * 2008-03-09 2011-03-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
JP2013545736A (ja) * 2010-10-29 2013-12-26 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遺伝子の阻害のための組成物および方法

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JP2004261002A (ja) * 2003-01-08 2004-09-24 Tsutomu Suzuki ｓｉＲＮＡの製造方法
ES2874149T3 (es) * 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
CN200950065Y (zh) 2006-05-24 2007-09-19 王锦峰 一种半自动夹板装置
AU2007275365A1 (en) 2006-07-17 2008-01-24 Sirna Therapeutics Inc. RNA interference mediated inhibition of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
MX2010008394A (es) 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
US9493774B2 (en) * 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
WO2011028938A1 (en) * 2009-09-02 2011-03-10 Alnylam Pharmaceuticals, Inc. Methods for lowering serum cholestrol in a subject using inhibition of pcsk9
EP3358014B1 (en) * 2010-04-19 2020-12-23 TAGCyx Biotechnologies Inc. Method for stabilizing functional nucleic acids
ES2770667T3 (es) * 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
CN118697894A (zh) 2015-08-25 2024-09-27 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
LT3529360T (lt) 2016-10-18 2024-07-25 Novartis Ag Metodai, skirti širdies ir kraujagyslių ligų prevencijai mažinant proproteinų konvertazės subtilizino keksino 9 (pcsk9) baltymo kiekį
CN109957565B (zh) * 2017-12-26 2023-04-07 广州市锐博生物科技有限公司 一种修饰的siRNA分子及其应用
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Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006125977A2 (en) * 2005-05-25 2006-11-30 The University Of York Hybrid interfering rna
US20110065644A1 (en) * 2008-03-09 2011-03-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
JP2013545736A (ja) * 2010-10-29 2013-12-26 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遺伝子の阻害のための組成物および方法

Non-Patent Citations (2)

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Title
SIMON J ALLISON ET AL.: ""RNA Interference by Single- and Double-stranded siRNA With a DNA Extension Containing a 3′ Nuclea", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 3, JPN6024049640, January 2014 (2014-01-01), pages 141, ISSN: 0005480757 *
TARANEH DJAVANBAKHT SAMANI ET AL.: ""Best Minimally Modified Antisense Oligonucleotides According to Cell Nuclease Activity"", ANTISENSE AND NUCLEIC ACID DRUG DEVELOPMENT, vol. 11, no. 3, JPN6024049635, June 2001 (2001-06-01), pages 129 - 136, ISSN: 0005480758 *

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Publication number Publication date
CA3167849A1 (en) 2021-09-23
CN115066498A (zh) 2022-09-16
GB2594788A (en) 2021-11-10
US20230183694A1 (en) 2023-06-15
GB202103594D0 (en) 2021-04-28
EP4081642A1 (en) 2022-11-02
GB2594788B (en) 2023-06-07
WO2021185765A1 (en) 2021-09-23

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