CN118510894A - 用于抑制前激肽释放酶(pkk)蛋白表达的组合物和方法 - Google Patents
用于抑制前激肽释放酶(pkk)蛋白表达的组合物和方法 Download PDFInfo
- Publication number
- CN118510894A CN118510894A CN202380015864.8A CN202380015864A CN118510894A CN 118510894 A CN118510894 A CN 118510894A CN 202380015864 A CN202380015864 A CN 202380015864A CN 118510894 A CN118510894 A CN 118510894A
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- Prior art keywords
- pkk
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- nucleotides
- dsrna
- dsrna agent
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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
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- 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)
- C12Y304/21035—Tissue kallikrein (3.4.21.35)
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- 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
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- 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)
- C12Y304/21008—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
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
-
- 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
- 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
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- 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)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- 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)
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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN118510894A true CN118510894A (zh) | 2024-08-16 |
Family
ID=87470605
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202380015864.8A Pending CN118510894A (zh) | 2022-01-28 | 2023-01-28 | 用于抑制前激肽释放酶(pkk)蛋白表达的组合物和方法 |
Country Status (11)
| Country | Link |
|---|---|
| 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)
| 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)
| 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 |
-
2023
- 2023-01-19 TW TW112102528A patent/TW202346586A/zh unknown
- 2023-01-28 EP EP23746361.7A patent/EP4469570A4/en active Pending
- 2023-01-28 MX MX2024009257A patent/MX2024009257A/es unknown
- 2023-01-28 IL IL314396A patent/IL314396A/en unknown
- 2023-01-28 CA CA3244243A patent/CA3244243A1/en active Pending
- 2023-01-28 KR KR1020247025383A patent/KR20240139057A/ko active Pending
- 2023-01-28 AU AU2023210979A patent/AU2023210979A1/en active Pending
- 2023-01-28 CN CN202380015864.8A patent/CN118510894A/zh active Pending
- 2023-01-28 WO PCT/CN2023/073501 patent/WO2023143483A1/en not_active Ceased
- 2023-01-28 JP JP2024543863A patent/JP2025508324A/ja active Pending
- 2023-01-28 US US18/833,727 patent/US20250145999A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL314396A (en) | 2024-09-01 |
| MX2024009257A (es) | 2024-08-06 |
| KR20240139057A (ko) | 2024-09-20 |
| AU2023210979A1 (en) | 2024-08-01 |
| EP4469570A1 (en) | 2024-12-04 |
| TW202346586A (zh) | 2023-12-01 |
| JP2025508324A (ja) | 2025-03-26 |
| EP4469570A4 (en) | 2026-08-12 |
| US20250145999A1 (en) | 2025-05-08 |
| CA3244243A1 (en) | 2023-08-03 |
| WO2023143483A1 (en) | 2023-08-03 |
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