JP2024531728A - 心血管疾患の処置 - Google Patents

心血管疾患の処置 Download PDF

Info

Publication number
JP2024531728A
JP2024531728A JP2024516476A JP2024516476A JP2024531728A JP 2024531728 A JP2024531728 A JP 2024531728A JP 2024516476 A JP2024516476 A JP 2024516476A JP 2024516476 A JP2024516476 A JP 2024516476A JP 2024531728 A JP2024531728 A JP 2024531728A
Authority
JP
Japan
Prior art keywords
nucleic acid
acid molecule
molecule
nucleotide sequence
double
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024516476A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023041508A5 (https=
JP2024531728A5 (https=
Inventor
カーン,ステラ
ミッチェル,ダニエル
カーン,マイケル
Original Assignee
アルゴノート アールエヌエー リミテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from GBGB2113104.0A external-priority patent/GB202113104D0/en
Priority claimed from GBGB2207239.1A external-priority patent/GB202207239D0/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 JP2024531728A publication Critical patent/JP2024531728A/ja
Publication of JPWO2023041508A5 publication Critical patent/JPWO2023041508A5/ja
Publication of JP2024531728A5 publication Critical patent/JP2024531728A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2024516476A 2021-09-14 2022-09-13 心血管疾患の処置 Pending JP2024531728A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2113104.0A GB202113104D0 (en) 2021-09-14 2021-09-14 Antagonist of pcsk9
GB2113104.0 2021-09-14
GBGB2207239.1A GB202207239D0 (en) 2022-05-18 2022-05-18 Treatment of cardiovascular disease 2
GB2207239.1 2022-05-18
PCT/EP2022/075355 WO2023041508A2 (en) 2021-09-14 2022-09-13 Treatment of cardiovascular disease

Publications (3)

Publication Number Publication Date
JP2024531728A true JP2024531728A (ja) 2024-08-29
JPWO2023041508A5 JPWO2023041508A5 (https=) 2025-07-23
JP2024531728A5 JP2024531728A5 (https=) 2025-07-23

Family

ID=83691543

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024516476A Pending JP2024531728A (ja) 2021-09-14 2022-09-13 心血管疾患の処置

Country Status (5)

Country Link
US (1) US20250283076A1 (https=)
EP (1) EP4402263A2 (https=)
JP (1) JP2024531728A (https=)
CA (1) CA3229020A1 (https=)
WO (1) WO2023041508A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
TW202345865A (zh) * 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
EP4680740A1 (en) * 2023-03-14 2026-01-21 Argonaute Rna Limited Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
WO2024228030A2 (en) * 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术(苏州)有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
AU2024321098A1 (en) * 2023-08-09 2026-03-19 Beijing Foyou Pharma Co., Ltd Sirna for inhibiting dgat2 gene expression, sirna conjugate or prodrug and pharmaceutical composition thereof, and use thereof
WO2025040139A1 (zh) * 2023-08-23 2025-02-27 云合智药(苏州)生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
WO2025103391A1 (zh) * 2023-11-15 2025-05-22 北京加科瑞康医药科技有限公司 用于抑制lpa的基因表达的sirna,含其的组合物及其用途

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001064008A2 (en) 2000-03-03 2001-09-07 Smithkline Beecham Biologicals S.A. Vaccine for the treatment of artherosclerosis
GB0121171D0 (en) 2001-08-31 2001-10-24 Glaxosmithkline Biolog Sa Vaccine
GB0305794D0 (en) 2003-03-13 2003-04-16 Glaxosmithkline Biolog Sa Vaccine
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
WO2005045035A2 (en) 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US8067572B2 (en) * 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
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)
ES2804764T3 (es) * 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
WO2012058693A2 (en) * 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
US10125369B2 (en) * 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
WO2014205449A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
BR112019010057A2 (pt) 2016-11-16 2019-09-03 Purdue Research Foundation composição, método para suprimir a ingestão de comida, sequência de shrna, método de inibição da síntese de ácidos graxos
MX2020004897A (es) 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de lpa en una celula.
BR112021009213A2 (pt) * 2018-11-13 2021-08-10 Silence Therapeutics Gmbh ácidos nucleicos para inibição da expressão de lpa em uma célula
US20230027604A1 (en) * 2019-07-02 2023-01-26 Argonaute RNA Limited Apolipoprotein b antagonist
US20230078200A1 (en) * 2019-12-09 2023-03-16 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
US20230183694A1 (en) * 2020-03-16 2023-06-15 Argonaute RNA Limited Antagonist of pcsk9

Also Published As

Publication number Publication date
CA3229020A1 (en) 2023-03-23
WO2023041508A3 (en) 2023-06-08
EP4402263A2 (en) 2024-07-24
US20250283076A1 (en) 2025-09-11
WO2023041508A2 (en) 2023-03-23

Similar Documents

Publication Publication Date Title
JP2024531728A (ja) 心血管疾患の処置
US20190078088A1 (en) RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
Watts et al. Clinical status of duplex RNA
JP2024500035A (ja) 心血管疾患の治療
JP2021185149A (ja) アポリポタンパク質(a)発現の調節
JP2013511964A (ja) miRNA−100を含む医薬組成物ならびに血管増殖および内皮炎症を調節するためのその使用
KR20150006743A (ko) 간암 연관 유전자 특이적 siRNA, 그러한 siRNA를 포함하는 이중나선 올리고 RNA 구조체 및 이를 포함하는 암 예방 또는 치료용 조성물
JP2022543136A (ja) Apoc3関連疾患および障害の処置のための方法
JP2022537987A (ja) アポリポタンパク質bアンタゴニスト
JP5406024B2 (ja) Bcl−XL特異的siNAを用いる癌治療法
CN118103508A (zh) 心血管疾病的治疗
WO2024228030A2 (en) Dual silencing
GB2618915A (en) Treatment of cardiovascular disease
GB2631903A (en) Dual silencing
GB2629617A (en) Dual silencing
TW202526015A (zh) 靶向LPA基因的RNAi劑及其用途
WO2026026890A1 (zh) 用于抑制LPA基因表达的siRNA及其修饰物与应用
EP4680740A1 (en) Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
WO2024222686A1 (zh) 用于抑制LPA表达的RNAi剂及其应用
WO2026017176A1 (zh) 靶向微管相关蛋白Tau基因的寡核苷酸及其用途
WO2025242147A1 (zh) 靶向外周髓鞘蛋白-22基因的寡核苷酸及其用途
WO2026087871A1 (en) Gene silencing of cardiovascular genes
WO2025077916A1 (zh) 靶向转甲状腺素蛋白的寡核苷酸及其用途
CN119563025A (zh) 一种抑制LP(a)基因表达的dsRNA分子及其应用
CN120344664A (zh) 靶向脂蛋白a的寡核苷酸及其用途

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240621

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240725

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250714

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250714