MX377150B - Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas. - Google Patents
Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.Info
- Publication number
- MX377150B MX377150B MX2019009191A MX2019009191A MX377150B MX 377150 B MX377150 B MX 377150B MX 2019009191 A MX2019009191 A MX 2019009191A MX 2019009191 A MX2019009191 A MX 2019009191A MX 377150 B MX377150 B MX 377150B
- Authority
- MX
- Mexico
- Prior art keywords
- fusion proteins
- peptide
- lysosomal
- lysosomal enzyme
- therapeutic purpose
- Prior art date
Links
- 102000037865 fusion proteins Human genes 0.000 title abstract 4
- 108020001507 fusion proteins Proteins 0.000 title abstract 4
- 230000002132 lysosomal effect Effects 0.000 title abstract 4
- 230000001225 therapeutic effect Effects 0.000 title abstract 4
- 108090000790 Enzymes Proteins 0.000 title abstract 2
- 102000004190 Enzymes Human genes 0.000 title abstract 2
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 abstract 2
- 101800001707 Spacer peptide Proteins 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 2
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 abstract 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 208000005340 mucopolysaccharidosis III Diseases 0.000 abstract 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 abstract 1
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 abstract 1
- 208000011580 syndromic disease Diseases 0.000 abstract 1
Classifications
-
- 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
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Toxicology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invención se refiere en general a proteínas de fusión terapéutica útiles para tratar enfermedades de almacenamiento lisosomal y métodos para tratar tales enfermedades. Proteínas de fusión terapéutica ejemplares comprenden una enzima lisosomal, una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador. También se proporcionan composiciones y métodos para tratar Mucopolisacaridosis Tipo IIIB (Síndrome de Sanfilippo B), que comprenden una proteína de fusión terapéutica objetivo que comprende alfa-N-acetilglucosaminidasa (Naglu), una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US201361788968P | 2013-03-15 | 2013-03-15 | |
| PCT/US2013/072287 WO2014085621A1 (en) | 2012-11-27 | 2013-11-27 | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2019009191A MX2019009191A (es) | 2019-10-09 |
| MX377150B true MX377150B (es) | 2025-03-07 |
Family
ID=49753532
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2015006644A MX367024B (es) | 2012-11-27 | 2013-11-27 | Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas. |
| MX2019009191A MX377150B (es) | 2012-11-27 | 2013-11-27 | Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2015006644A MX367024B (es) | 2012-11-27 | 2013-11-27 | Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas. |
Country Status (26)
| Country | Link |
|---|---|
| US (8) | US9376480B2 (es) |
| EP (2) | EP2925776B1 (es) |
| JP (2) | JP6831176B2 (es) |
| KR (4) | KR102262882B1 (es) |
| CN (1) | CN104822701B (es) |
| AR (1) | AR093626A1 (es) |
| AU (1) | AU2013352184B2 (es) |
| BR (1) | BR112015012152B1 (es) |
| CA (1) | CA2892146A1 (es) |
| CL (1) | CL2015001371A1 (es) |
| CY (1) | CY1122555T1 (es) |
| DK (2) | DK2925776T3 (es) |
| ES (2) | ES2729997T3 (es) |
| HR (2) | HRP20181351T1 (es) |
| HU (2) | HUE039334T2 (es) |
| IL (3) | IL238824B (es) |
| LT (1) | LT3115372T (es) |
| MX (2) | MX367024B (es) |
| PL (2) | PL3115372T3 (es) |
| PT (2) | PT3115372T (es) |
| RS (1) | RS58916B1 (es) |
| RU (1) | RU2680581C2 (es) |
| SI (1) | SI3115372T1 (es) |
| TW (2) | TWI626250B (es) |
| WO (1) | WO2014085621A1 (es) |
| ZA (1) | ZA201503509B (es) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HRP20170698T1 (hr) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| HUE039334T2 (hu) | 2012-11-27 | 2018-12-28 | Biomarin Pharm Inc | Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai |
| HK1222093A1 (zh) * | 2013-05-15 | 2017-06-23 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| JP6623213B2 (ja) * | 2014-08-11 | 2019-12-18 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | リソソーム酵素に融合したマンノース−６−リン酸含有ペプチド |
| EP3197503A4 (en) * | 2014-09-25 | 2018-05-16 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| US10556015B2 (en) | 2014-10-24 | 2020-02-11 | Criteo S.A. | Lysosomal targeting of enzymes, and uses thereof |
| TWI752907B (zh) | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| CN107849131A (zh) | 2015-07-06 | 2018-03-27 | 瑞泽恩制药公司 | 多特异性抗原结合分子及其用途 |
| HK1253529A1 (zh) | 2015-11-06 | 2019-06-21 | Biomarin Pharmaceutical Inc. | 用於检测中和溶酶体酶的摄取的抗体或其它因子的基於细胞的测定 |
| RU2751235C2 (ru) * | 2016-02-24 | 2021-07-12 | Байомарин Фармасьютикал Инк. | Терапевтические слитые белки направленного действия на основе лизосомных ферментов, соответствующие составы и их применения |
| SG11201808176TA (en) | 2016-04-15 | 2018-10-30 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| CL2016003282A1 (es) | 2016-12-21 | 2017-08-18 | Univ Chile | Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington |
| EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| SG11202002730XA (en) | 2017-10-02 | 2020-04-29 | Denali Therapeutics Inc | Fusion proteins comprising enzyme replacement therapy enzymes |
| IL322464A (en) | 2018-02-07 | 2025-09-01 | Regeneron Pharma | Methods and compositions for administering therapeutic protein |
| CA3092961A1 (en) * | 2018-03-09 | 2019-09-12 | Avrobio, Inc. | Compositions and methods for treating parkinson's disease |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| EP3802619A1 (en) | 2018-06-08 | 2021-04-14 | F. Hoffmann-La Roche AG | Peptidic linker with reduced post-translational modification |
| US11097015B2 (en) | 2018-10-10 | 2021-08-24 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| AU2019381803A1 (en) * | 2018-11-16 | 2021-06-10 | AskBio Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| WO2021072372A1 (en) * | 2019-10-10 | 2021-04-15 | Amicus Therapeutics, Inc. | Variant igf2 constructs |
| WO2021133822A1 (en) | 2019-12-24 | 2021-07-01 | Juvena Therapeutics,Inc. | Regenerative polypeptides and uses thereof |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| CA3223707A1 (en) | 2021-06-21 | 2022-12-29 | Hanadie Yousef | Regenerative polypeptides and uses thereof |
| WO2022271981A2 (en) | 2021-06-23 | 2022-12-29 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| CN117343197A (zh) * | 2021-10-15 | 2024-01-05 | 中山大学 | 重组融合抗体 |
| KR20250160369A (ko) * | 2023-01-27 | 2025-11-12 | 리제너론 파마슈티칼스 인코포레이티드 | 변형된 랩도바이러스 당단백질 및 이의 용도 |
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| US5206161A (en) | 1991-02-01 | 1993-04-27 | Genentech, Inc. | Human plasma carboxypeptidase B |
| AUPN674895A0 (en) | 1995-11-23 | 1995-12-14 | Women's And Children's Hospital | Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same |
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| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
| ES2300439T3 (es) | 2001-04-30 | 2008-06-16 | Zystor Therapeutics , Inc. | Reconocimiento subcelular de proteinas terapeuticas. |
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| ATE525395T1 (de) | 2003-06-12 | 2011-10-15 | Lilly Co Eli | Fusions proteine von glp-1-analoga |
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| US7678371B2 (en) | 2005-03-04 | 2010-03-16 | Biogen Idec Ma Inc. | Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues |
| HRP20170698T1 (hr) | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| US8697654B2 (en) * | 2008-12-18 | 2014-04-15 | E I Du Pont De Nemours And Company | Peptide linkers for effective multivalent peptide binding |
| US9029530B2 (en) | 2009-01-02 | 2015-05-12 | Biomarin Pharmaceutical Inc. | Detection of oligosaccharides |
| EP2475376B1 (en) * | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| MX362176B (es) | 2010-06-25 | 2019-01-07 | Amcor Rigid Plastics Usa Llc | Sistema de depuracion de oxigeno para un recipiente. |
| MX2013000324A (es) * | 2010-06-25 | 2013-02-01 | Shire Human Genetic Therapies | Tratamiento del sindrome de sanfilippo tipo b. |
| WO2012088461A2 (en) * | 2010-12-23 | 2012-06-28 | Biogen Idec Inc. | Linker peptides and polypeptides comprising same |
| JP6175372B2 (ja) * | 2011-03-04 | 2017-08-02 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ポリペプチド組成物用ペプチドリンカーおよびその使用方法 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| WO2012122477A1 (en) * | 2011-03-10 | 2012-09-13 | Novozymes A/S | Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same |
| HUE039334T2 (hu) | 2012-11-27 | 2018-12-28 | Biomarin Pharm Inc | Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai |
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2013
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 BR BR112015012152-7A patent/BR112015012152B1/pt active IP Right Grant
- 2013-11-27 AR ARP130104367A patent/AR093626A1/es active IP Right Grant
- 2013-11-27 RU RU2015125491A patent/RU2680581C2/ru active
- 2013-11-27 EP EP13802834.5A patent/EP2925776B1/en active Active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 PL PL16182420T patent/PL3115372T3/pl unknown
- 2013-11-27 TW TW102143329A patent/TWI626250B/zh active
- 2013-11-27 MX MX2015006644A patent/MX367024B/es active IP Right Grant
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 2013-11-27 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
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2015
- 2015-05-14 IL IL238824A patent/IL238824B/en active IP Right Grant
- 2015-05-19 ZA ZA2015/03509A patent/ZA201503509B/en unknown
- 2015-05-20 CL CL2015001371A patent/CL2015001371A1/es unknown
- 2015-10-14 US US14/883,193 patent/US9834587B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,211 patent/US9771408B2/en active Active
- 2015-10-14 US US14/883,203 patent/US9834588B2/en active Active
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2017
- 2017-08-28 US US15/688,438 patent/US10301369B2/en active Active
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2018
- 2018-11-13 IL IL262976A patent/IL262976B/en active IP Right Grant
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2019
- 2019-04-08 US US16/378,163 patent/US11254725B2/en active Active
- 2019-05-17 HR HRP20190918TT patent/HRP20190918T1/hr unknown
- 2019-06-06 CY CY20191100600T patent/CY1122555T1/el unknown
- 2019-07-24 JP JP2019136013A patent/JP6913719B2/ja active Active
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2020
- 2020-02-23 IL IL272854A patent/IL272854B/en unknown
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2022
- 2022-01-10 US US17/572,018 patent/US20220127326A1/en not_active Abandoned
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