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 (https=
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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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    • C12Y—ENZYMES
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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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)
  • Health & Medical Sciences (AREA)
  • 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)
  • Zoology (AREA)
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  • Biochemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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
CN2022074535 2022-01-28
CNPCT/CN2022/074535 2022-01-28
PCT/CN2023/073501 WO2023143483A1 (en) 2022-01-28 2023-01-28 Compositions and methods for inhibiting expression of prekallikrein (pkk) protein

Publications (2)

Publication Number Publication Date
JP2025508324A true JP2025508324A (ja) 2025-03-26
JP2025508324A5 JP2025508324A5 (https=) 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

Country Status (11)

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US (1) US20250145999A1 (https=)
EP (1) EP4469570A1 (https=)
JP (1) JP2025508324A (https=)
KR (1) KR20240139057A (https=)
CN (1) CN118510894A (https=)
AU (1) AU2023210979A1 (https=)
CA (1) CA3244243A1 (https=)
IL (1) IL314396A (https=)
MX (1) MX2024009257A (https=)
TW (1) TW202346586A (https=)
WO (1) WO2023143483A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12042509B2 (en) * 2021-10-01 2024-07-23 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102365486B1 (ko) * 2013-08-28 2022-02-18 아이오니스 파마수티컬즈, 인코포레이티드 프리칼리크레인 (pkk) 발현의 조절
HUE052709T2 (hu) * 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
JP6695902B2 (ja) * 2015-05-06 2020-05-20 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法
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
US20220315929A1 (en) * 2019-05-24 2022-10-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof
WO2021126734A1 (en) * 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
BR112022015380A2 (pt) * 2020-02-07 2022-09-27 Intellia Therapeutics Inc Composições e métodos para edição de gene de calicreína (klkb1)

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

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