CY1122282T1 - Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α - Google Patents
Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης αInfo
- Publication number
- CY1122282T1 CY1122282T1 CY20191101190T CY191101190T CY1122282T1 CY 1122282 T1 CY1122282 T1 CY 1122282T1 CY 20191101190 T CY20191101190 T CY 20191101190T CY 191101190 T CY191101190 T CY 191101190T CY 1122282 T1 CY1122282 T1 CY 1122282T1
- Authority
- CY
- Cyprus
- Prior art keywords
- compositions
- methods
- arylosulfatase
- cns delivery
- cns
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 102100022146 Arylsulfatase A Human genes 0.000 abstract 2
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 abstract 2
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 238000007913 intrathecal administration Methods 0.000 abstract 1
- 230000002132 lysosomal effect Effects 0.000 abstract 1
- 229950008882 polysorbate Drugs 0.000 abstract 1
- 229920000136 polysorbate Polymers 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
- 239000004094 surface-active agent Substances 0.000 abstract 1
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
-
- 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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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)
-
- 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
- A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Inorganic Chemistry (AREA)
- Molecular Biology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| PCT/US2011/041926 WO2011163650A2 (en) | 2010-06-25 | 2011-06-25 | Methods and compositions for cns delivery of arylsulfatase a |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1122282T1 true CY1122282T1 (el) | 2020-11-25 |
Family
ID=46888844
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20191101190T CY1122282T1 (el) | 2010-06-25 | 2019-11-13 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
| CY20211100942T CY1124910T1 (el) | 2010-06-25 | 2021-11-02 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20211100942T CY1124910T1 (el) | 2010-06-25 | 2021-11-02 | Μεθοδοι και συνθεσεις για παραδοση στο κνς της αρυλοσουλφατασης α |
Country Status (26)
| Country | Link |
|---|---|
| US (1) | US9770410B2 (enExample) |
| EP (3) | EP3626257B1 (enExample) |
| JP (1) | JP6045493B2 (enExample) |
| KR (5) | KR102094094B1 (enExample) |
| CN (1) | CN103282046B (enExample) |
| AU (5) | AU2011270670B2 (enExample) |
| BR (1) | BR112012033216B1 (enExample) |
| CA (1) | CA2803003C (enExample) |
| CY (2) | CY1122282T1 (enExample) |
| DK (1) | DK2585104T3 (enExample) |
| ES (1) | ES2754776T3 (enExample) |
| HR (1) | HRP20191923T1 (enExample) |
| IL (3) | IL291556B2 (enExample) |
| LT (1) | LT2585104T (enExample) |
| ME (1) | ME03647B (enExample) |
| MX (2) | MX382443B (enExample) |
| NZ (2) | NZ605873A (enExample) |
| PL (2) | PL2585104T3 (enExample) |
| RS (1) | RS59468B1 (enExample) |
| RU (1) | RU2671503C2 (enExample) |
| SI (1) | SI2585104T1 (enExample) |
| SM (1) | SMT201900589T1 (enExample) |
| TW (1) | TWI667037B (enExample) |
| UA (1) | UA115650C2 (enExample) |
| WO (1) | WO2011163650A2 (enExample) |
| ZA (1) | ZA201300675B (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT3626258T (pt) | 2010-06-25 | 2021-10-19 | Shire Human Genetic Therapies | Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase |
| HRP20191924T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku iduronat-2-sulfataze u cns |
| DK2588130T3 (en) | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| CN119979504A (zh) | 2013-01-09 | 2025-05-13 | 武田药品工业株式会社 | 含用于纯化芳基硫酸酯酶a的方法 |
| RU2692251C2 (ru) * | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| US10208734B2 (en) * | 2015-04-23 | 2019-02-19 | Continuum Dynamics, Inc. | Lift-driven wind turbine with force canceling blade configuration |
| WO2017009255A1 (en) * | 2015-07-10 | 2017-01-19 | Synthon Biopharmaceuticals B.V. | Compositions comprising antibody-duocarmycin drug conjugates |
| CN108883162A (zh) * | 2016-02-17 | 2018-11-23 | 夏尔人类遗传性治疗公司 | 用于芳基硫酸酯酶a的cns递送的方法和组合物 |
| NZ788906A (en) * | 2016-06-01 | 2025-07-25 | Servier Ip Uk Ltd | Formulations of polyalkylene oxide-asparaginase and methods of making and using the same |
| KR102471458B1 (ko) | 2016-12-28 | 2022-11-25 | 제이씨알 파마 가부시키가이샤 | 동결건조 제제 |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| KR20200107984A (ko) | 2017-12-19 | 2020-09-16 | 샤이어 휴먼 지네틱 테라피즈 인크. | 정제된 아릴설파타아제 a 및 이의 조성물 |
| WO2020004368A1 (ja) * | 2018-06-25 | 2020-01-02 | Ｊｃｒファーマ株式会社 | 蛋白質含有水性液剤 |
| WO2020227166A1 (en) * | 2019-05-03 | 2020-11-12 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| WO2021061517A1 (en) * | 2019-09-23 | 2021-04-01 | University Of Washington | Assay of arylsulfatase a enzymatic activity in dried blood spots by mass spectrometry and fluorometry |
| WO2024176112A1 (en) | 2023-02-21 | 2024-08-29 | Takeda Pharmaceutical Company Limited | Large scale production of recombinant arylsulfatase a and compositions thereof |
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| US4743265A (en) | 1986-04-23 | 1988-05-10 | Dij Catheter Corp | Articulated catheter placement device |
| US5863782A (en) | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| US6118045A (en) | 1995-08-02 | 2000-09-12 | Pharming B.V. | Lysosomal proteins produced in the milk of transgenic animals |
| ATE225184T1 (de) * | 1997-08-22 | 2002-10-15 | Seikagaku Kogyo Co Ltd | Arzneimittel zur behandlung von durch hernie gestörter intervertebraler bandscheibe |
| WO2000034451A1 (en) | 1998-12-07 | 2000-06-15 | Pharming Intellectual Property B.V. | Treatment of pompe's disease |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US20020052311A1 (en) | 1999-09-03 | 2002-05-02 | Beka Solomon | Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US20020099025A1 (en) | 1999-12-30 | 2002-07-25 | Heywood James A. | Treatment of neurological disorders |
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| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
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| ATE384736T1 (de) | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
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| JP5570677B2 (ja) | 2002-04-25 | 2014-08-13 | 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 | α−ガラクトシダーゼＡ欠損症の治療 |
| EP1514106A4 (en) | 2002-05-29 | 2007-05-09 | Zystor Therapeutics Inc | TARGETED THERAPEUTIC PROTEINS |
| US20040248262A1 (en) | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
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| NZ548871A (en) | 2004-01-30 | 2009-06-26 | Shire Pharmaceuticals Ireland | Use of arylsulfatase A for treating metachromatic leukodystrophy |
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| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
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| PL2631242T3 (pl) * | 2006-04-04 | 2023-03-20 | Takeda Pharmaceutical Company Limited | Sposób zatężania polipeptydu |
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| US20090041741A1 (en) | 2007-03-06 | 2009-02-12 | Saint Louis University | Modified enzyme and treatment method |
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| US7722865B2 (en) * | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
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| MX2013000320A (es) | 2010-06-25 | 2013-06-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa. |
| HRP20191924T1 (hr) | 2010-06-25 | 2020-01-10 | Shire Human Genetic Therapies, Inc. | Postupci i sastavi za isporuku iduronat-2-sulfataze u cns |
| DK2588130T3 (en) | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| NZ605873A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| PT3626258T (pt) | 2010-06-25 | 2021-10-19 | Shire Human Genetic Therapies | Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase |
| US20110318327A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| EP2588132A4 (en) | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
| JP2012062312A (ja) | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
-
2011
- 2011-06-25 NZ NZ605873A patent/NZ605873A/en unknown
- 2011-06-25 LT LT11799037T patent/LT2585104T/lt unknown
- 2011-06-25 SI SI201131776T patent/SI2585104T1/sl unknown
- 2011-06-25 US US13/168,963 patent/US9770410B2/en active Active
- 2011-06-25 UA UAA201214685A patent/UA115650C2/uk unknown
- 2011-06-25 AU AU2011270670A patent/AU2011270670B2/en active Active
- 2011-06-25 PL PL11799037T patent/PL2585104T3/pl unknown
- 2011-06-25 IL IL291556A patent/IL291556B2/en unknown
- 2011-06-25 ES ES11799037T patent/ES2754776T3/es active Active
- 2011-06-25 SM SM20190589T patent/SMT201900589T1/it unknown
- 2011-06-25 EP EP19192508.0A patent/EP3626257B1/en active Active
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