JP2025508324A - プレカリクレイン（ｐｋｋ）タンパク質の発現を阻害するための組成物及び方法 - Google Patents

プレカリクレイン（ｐｋｋ）タンパク質の発現を阻害するための組成物及び方法 Download PDF

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JP2025508324A
JP2025508324A JP2024543863A JP2024543863A JP2025508324A JP 2025508324 A JP2025508324 A JP 2025508324A JP 2024543863 A JP2024543863 A JP 2024543863A JP 2024543863 A JP2024543863 A JP 2024543863A JP 2025508324 A JP2025508324 A JP 2025508324A
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pkk
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dsrna
dsrna agent
nucleotide
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JP2025508324A5 (enExample
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シュー、ドンシュウ
シャオ、ペンチェン、パトリック
シャー、シーウェイ
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シャンハイ アルゴ バイオファーマシューティカル カンパニー，リミテッド
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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21035—Tissue kallikrein (3.4.21.35)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21008—Kallikrein (3.4.21.8)
    • 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)
    • C12Y304/21034—Plasma kallikrein (3.4.21.34)
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
JP2024543863A 2022-01-28 2023-01-28 プレカリクレイン（ｐｋｋ）タンパク質の発現を阻害するための組成物及び方法 Pending JP2025508324A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CNPCT/CN2022/074535 2022-01-28
CN2022074535 2022-01-28
PCT/CN2023/073501 WO2023143483A1 (en) 2022-01-28 2023-01-28 Compositions and methods for inhibiting expression of prekallikrein (pkk) protein

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JP2025508324A true JP2025508324A (ja) 2025-03-26
JP2025508324A5 JP2025508324A5 (enExample) 2026-02-02

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JP2024543863A Pending JP2025508324A (ja) 2022-01-28 2023-01-28 プレカリクレイン（ｐｋｋ）タンパク質の発現を阻害するための組成物及び方法

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US (1) US20250145999A1 (enExample)
EP (1) EP4469570A4 (enExample)
JP (1) JP2025508324A (enExample)
KR (1) KR20240139057A (enExample)
CN (1) CN118510894A (enExample)
AU (1) AU2023210979A1 (enExample)
CA (1) CA3244243A1 (enExample)
IL (1) IL314396A (enExample)
MX (1) MX2024009257A (enExample)
TW (1) TW202346586A (enExample)
WO (1) WO2023143483A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022357561A1 (en) * 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9670492B2 (en) * 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
HUE052709T2 (hu) * 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
CA2984636A1 (en) * 2015-05-06 2016-11-10 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
EP3649243A1 (en) * 2017-07-07 2020-05-13 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12)
CN109762904A (zh) * 2019-03-05 2019-05-17 中国医学科学院北京协和医院 与胰腺神经内分泌肿瘤相关的分子标记物及其应用
CN113891939B (zh) * 2019-05-24 2024-04-02 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
CN115176007A (zh) * 2019-12-16 2022-10-11 阿尔尼拉姆医药品有限公司 含类PATATIN磷脂酶结构域3(PNPLA3)iRNA组合物及其使用方法
CN115362256A (zh) * 2020-02-07 2022-11-18 因特利亚治疗公司 用于激肽释放酶（klkb1）基因编辑的组合物和方法
AU2022357561A1 (en) * 2021-10-01 2024-04-18 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof

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IL314396A (en) 2024-09-01
MX2024009257A (es) 2024-08-06
CN118510894A (zh) 2024-08-16
KR20240139057A (ko) 2024-09-20
AU2023210979A1 (en) 2024-08-01
EP4469570A1 (en) 2024-12-04
TW202346586A (zh) 2023-12-01
EP4469570A4 (en) 2026-08-12
US20250145999A1 (en) 2025-05-08
CA3244243A1 (en) 2023-08-03
WO2023143483A1 (en) 2023-08-03

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