JP6594902B2 - ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 - Google Patents
ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 Download PDFInfo
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- JP6594902B2 JP6594902B2 JP2016568484A JP2016568484A JP6594902B2 JP 6594902 B2 JP6594902 B2 JP 6594902B2 JP 2016568484 A JP2016568484 A JP 2016568484A JP 2016568484 A JP2016568484 A JP 2016568484A JP 6594902 B2 JP6594902 B2 JP 6594902B2
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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
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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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- 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
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- 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
- A61P3/04—Anorexiants; Antiobesity agents
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- 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
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- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- 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
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/322—2'-R Modification
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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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
- C12Y207/01003—Ketohexokinase (2.7.1.3)
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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Diabetes (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Emergency Medicine (AREA)
- Urology & Nephrology (AREA)
- Endocrinology (AREA)
- Gastroenterology & Hepatology (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461938567P | 2014-02-11 | 2014-02-11 | |
| US61/938,567 | 2014-02-11 | ||
| PCT/US2015/015367 WO2015123264A1 (en) | 2014-02-11 | 2015-02-11 | Ketohexokinase (khk) irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019173883A Division JP7062623B2 (ja) | 2014-02-11 | 2019-09-25 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017509354A JP2017509354A (ja) | 2017-04-06 |
| JP2017509354A5 JP2017509354A5 (enExample) | 2018-03-22 |
| JP6594902B2 true JP6594902B2 (ja) | 2019-10-23 |
Family
ID=52573741
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016568484A Active JP6594902B2 (ja) | 2014-02-11 | 2015-02-11 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| JP2019173883A Active JP7062623B2 (ja) | 2014-02-11 | 2019-09-25 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| JP2022069642A Active JP7559001B2 (ja) | 2014-02-11 | 2022-04-20 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| JP2024161345A Pending JP2024178284A (ja) | 2014-02-11 | 2024-09-18 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019173883A Active JP7062623B2 (ja) | 2014-02-11 | 2019-09-25 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| JP2022069642A Active JP7559001B2 (ja) | 2014-02-11 | 2022-04-20 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
| JP2024161345A Pending JP2024178284A (ja) | 2014-02-11 | 2024-09-18 | ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US10370666B2 (enExample) |
| EP (2) | EP3960860A3 (enExample) |
| JP (4) | JP6594902B2 (enExample) |
| KR (3) | KR20230152154A (enExample) |
| CN (3) | CN118845818A (enExample) |
| AU (3) | AU2015217301A1 (enExample) |
| CA (1) | CA2938857A1 (enExample) |
| EA (1) | EA201691587A1 (enExample) |
| WO (1) | WO2015123264A1 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4385568A3 (en) | 2010-04-06 | 2025-02-12 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of cd274/pd-l1 gene |
| EA201691587A1 (ru) | 2014-02-11 | 2017-01-30 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| KR20170110083A (ko) | 2014-12-23 | 2017-10-10 | 인털렉츄얼 프라퍼티 어쏘시에이츠, 엘엘씨 | 경피 투여를 위한 방법 및 제형 |
| EA201890619A1 (ru) * | 2015-09-02 | 2018-09-28 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ iRNA ЛИГАНДА 1 БЕЛКА ЗАПРОГРАММИРОВАННОЙ ГИБЕЛИ КЛЕТОК 1 (PD-L1) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| US10982215B2 (en) | 2015-12-09 | 2021-04-20 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting programmed cell death 1 ligand 1 (PD-L1) and methods of use thereof |
| MX376072B (es) | 2015-12-29 | 2025-03-07 | Pfizer | 3-azabiciclo[3.1.0]hexanos sustituidos como inhibidores de cetohexoquinasa |
| WO2017127750A1 (en) | 2016-01-22 | 2017-07-27 | Modernatx, Inc. | Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof |
| WO2017180917A2 (en) | 2016-04-13 | 2017-10-19 | Modernatx, Inc. | Lipid compositions and their uses for intratumoral polynucleotide delivery |
| CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
| US11680254B2 (en) * | 2017-04-21 | 2023-06-20 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the PCSK9 gene |
| JP7285220B2 (ja) | 2017-05-18 | 2023-06-01 | モデルナティエックス インコーポレイテッド | 連結したインターロイキン-12(il12)ポリペプチドをコードするポリヌクレオチドを含む脂質ナノ粒子 |
| US11603532B2 (en) * | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP3681479B1 (en) | 2017-09-15 | 2024-01-31 | Dyve Biosciences, Inc. | Sodium bicarbonate for use in the treatment of gout and related disorders |
| CN111727252A (zh) * | 2017-12-18 | 2020-09-29 | 阿尔尼拉姆医药品有限公司 | 高速泳动族盒-1(HMGB1)iRNA组合物及其使用方法 |
| TWI851574B (zh) * | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
| US20210332367A1 (en) * | 2018-09-18 | 2021-10-28 | Alnylam Pharmaceuticals, Inc. | KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| US20220363711A1 (en) * | 2019-06-25 | 2022-11-17 | Amgen Inc. | Purification methods for carbohydrate-linked oligonucleotides |
| CN114981430A (zh) * | 2019-08-27 | 2022-08-30 | 赛伦斯治疗有限责任公司 | 用于抑制细胞中的c3表达的核酸 |
| KR20220084399A (ko) * | 2019-10-22 | 2022-06-21 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 성분 C3 iRNA 조성물 및 이의 사용 방법 |
| WO2021096763A1 (en) * | 2019-11-13 | 2021-05-20 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating an angiotensinogen- (agt-) associated disorder |
| BR112022017822A2 (pt) * | 2020-03-06 | 2022-11-08 | Alnylam Pharmaceuticals Inc | Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas |
| WO2022182574A1 (en) | 2021-02-26 | 2022-09-01 | Alnylam Pharmaceuticals, Inc. | KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| KR20230170732A (ko) | 2021-04-12 | 2023-12-19 | 베링거 인겔하임 인터내셔날 게엠베하 | 케토헥소키나제 (khk)를 억제하기 위한 조성물 및 방법 |
| TW202430191A (zh) | 2022-10-11 | 2024-08-01 | 德商百靈佳殷格翰國際股份有限公司 | 用於治療伴有晚期纖維化及/或硬化之nash的方法 |
| TW202430192A (zh) | 2022-10-11 | 2024-08-01 | 德商百靈佳殷格翰國際股份有限公司 | 用於治療nash之劑量方案 |
| WO2024175062A1 (zh) * | 2023-02-23 | 2024-08-29 | 大睿生物医药科技(上海)有限公司 | 调控KHK基因活性的dsRNA分子 |
| WO2024260379A1 (zh) * | 2023-06-20 | 2024-12-26 | 施能康医药科技(苏州)有限公司 | 靶向已酮糖激酶的核酸及其用途 |
| WO2025016294A1 (zh) * | 2023-07-14 | 2025-01-23 | 云合智药(苏州)生物科技有限公司 | 用于抑制己酮糖激酶表达的RNAi剂及其应用 |
| WO2025128632A1 (en) * | 2023-12-12 | 2025-06-19 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating ketohexokinase (khk)-associated diseases |
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| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
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