AR093626A1 - Proteinas de fusion de enzima lisosomal y usos de las mismas - Google Patents
Proteinas de fusion de enzima lisosomal y usos de las mismasInfo
- Publication number
- AR093626A1 AR093626A1 ARP130104367A ARP130104367A AR093626A1 AR 093626 A1 AR093626 A1 AR 093626A1 AR P130104367 A ARP130104367 A AR P130104367A AR P130104367 A ARP130104367 A AR P130104367A AR 093626 A1 AR093626 A1 AR 093626A1
- Authority
- AR
- Argentina
- Prior art keywords
- seq
- peptide
- lysosomal
- igf
- therapeutic fusion
- Prior art date
Links
- 102000037865 fusion proteins Human genes 0.000 title abstract 5
- 108020001507 fusion proteins Proteins 0.000 title abstract 5
- 102000004190 Enzymes Human genes 0.000 title abstract 4
- 108090000790 Enzymes Proteins 0.000 title abstract 4
- 230000002132 lysosomal effect Effects 0.000 abstract 5
- 101800001707 Spacer peptide Proteins 0.000 abstract 4
- 230000001225 therapeutic effect Effects 0.000 abstract 4
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 abstract 3
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 2
- 108040004574 (S)-3-O-geranylgeranylglyceryl phosphate synthase activity proteins Proteins 0.000 abstract 1
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 102000057877 human IGF2 Human genes 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
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 208000011580 syndromic disease Diseases 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
- 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)
-
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (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)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Toxicology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Obesity (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261730378P | 2012-11-27 | 2012-11-27 | |
| US201361788968P | 2013-03-15 | 2013-03-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR093626A1 true AR093626A1 (es) | 2015-06-17 |
Family
ID=49753532
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP130104367A AR093626A1 (es) | 2012-11-27 | 2013-11-27 | Proteinas de fusion de enzima lisosomal y usos de las mismas |
Country Status (26)
| Country | Link |
|---|---|
| US (8) | US9376480B2 (enExample) |
| EP (2) | EP3115372B1 (enExample) |
| JP (2) | JP6831176B2 (enExample) |
| KR (4) | KR102521039B1 (enExample) |
| CN (1) | CN104822701B (enExample) |
| AR (1) | AR093626A1 (enExample) |
| AU (1) | AU2013352184B2 (enExample) |
| BR (1) | BR112015012152B1 (enExample) |
| CA (1) | CA2892146A1 (enExample) |
| CL (1) | CL2015001371A1 (enExample) |
| CY (1) | CY1122555T1 (enExample) |
| DK (2) | DK3115372T3 (enExample) |
| ES (2) | ES2679374T3 (enExample) |
| HR (2) | HRP20181351T1 (enExample) |
| HU (2) | HUE043679T2 (enExample) |
| IL (3) | IL238824B (enExample) |
| LT (1) | LT3115372T (enExample) |
| MX (2) | MX377150B (enExample) |
| PL (2) | PL2925776T3 (enExample) |
| PT (2) | PT3115372T (enExample) |
| RS (1) | RS58916B1 (enExample) |
| RU (1) | RU2680581C2 (enExample) |
| SI (1) | SI3115372T1 (enExample) |
| TW (2) | TWI711632B (enExample) |
| WO (1) | WO2014085621A1 (enExample) |
| ZA (1) | ZA201503509B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| KR102521039B1 (ko) | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 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 |
| MX2017001898A (es) * | 2014-08-11 | 2017-04-11 | Shire Human Genetic Therapies | Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. |
| US20180303877A1 (en) * | 2014-09-25 | 2018-10-25 | The General Hospital Corporation | Cell-based targeted delivery of pseudonomas exotoxin |
| EP3220958A1 (en) * | 2014-10-24 | 2017-09-27 | Shire Human Genetic Therapies, Inc. | 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 |
| ES2971045T3 (es) | 2015-07-06 | 2024-06-03 | Regeneron Pharma | Moléculas multiespecíficas de unión a antígeno y usos de las mismas |
| EP3371593B1 (en) | 2015-11-06 | 2020-10-14 | BioMarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| AU2017222620B2 (en) * | 2016-02-24 | 2022-06-16 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof |
| 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 |
| KR102760654B1 (ko) | 2017-06-07 | 2025-02-04 | 리제너론 파마슈티칼스 인코포레이티드 | 효소 내재화를 위한 조성물 및 방법 |
| AU2018335752B2 (en) | 2017-09-22 | 2025-04-10 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
| MX2020002918A (es) | 2017-10-02 | 2020-07-22 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas. |
| MA51796A (fr) | 2018-02-07 | 2020-12-16 | Regeneron Pharma | Procédés et compositions pour l'administration de protéines thérapeutiques |
| JP2021517168A (ja) * | 2018-03-09 | 2021-07-15 | アブロバイオ，インコーポレーテッド | パーキンソン病を処置するための組成物及び方法 |
| US10874750B2 (en) | 2018-04-30 | 2020-12-29 | Amicus Therapeutics, Inc. | Gene therapy constructs and methods of use |
| US12252544B2 (en) | 2018-05-17 | 2025-03-18 | 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 |
| WO2020077114A2 (en) | 2018-10-10 | 2020-04-16 | 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 |
| AU2020361703A1 (en) * | 2019-10-10 | 2022-04-28 | Amicus Therapeutics, Inc. | Variant IGF2 constructs |
| EP4081241A4 (en) | 2019-12-24 | 2024-05-01 | Juvena Therapeutics, Inc. | REGENERATIVE POLYPEPTIDES AND THEIR USES |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| CN117999278A (zh) | 2021-06-21 | 2024-05-07 | 裘美娜治疗公司 | 再生性多肽及其用途 |
| US20240279360A1 (en) | 2021-06-23 | 2024-08-22 | Lycia Therapeutics, Inc. | Bifunctional compounds containing igf-2 polypeptides |
| CN115975039A (zh) * | 2021-10-15 | 2023-04-18 | 中山大学 | 重组融合抗体和抗体-药物偶联物及其用途 |
| CN120936364A (zh) * | 2023-01-27 | 2025-11-11 | 瑞泽恩制药公司 | 修饰的弹状病毒糖蛋白及其用途 |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| DE10035433C2 (de) | 2000-07-20 | 2002-07-18 | Tuma Wolfgang | Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben |
| US20040005309A1 (en) | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| IL158623A0 (en) * | 2001-04-30 | 2004-05-12 | Symbiontics Inc | Subcellular targeting of therapeutic proteins |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| AU2002362930A2 (en) | 2001-10-16 | 2003-04-28 | Zystor Therapeutics, Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| US7485314B2 (en) | 2002-05-06 | 2009-02-03 | Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center | Induction of antigen specific immunologic tolerance |
| AU2003237314B2 (en) | 2002-05-29 | 2010-03-04 | Biomarin Pharmaceutical Inc. | Targeted therapeutic proteins |
| DK1641823T3 (da) | 2003-06-12 | 2011-12-12 | Lilly Co Eli | GLP-1-analog fusionsproteiner |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050244400A1 (en) * | 2004-02-10 | 2005-11-03 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| WO2006096653A2 (en) | 2005-03-04 | 2006-09-14 | Biogen Idec Ma Inc. | Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues |
| HUE034850T2 (en) * | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
| 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 |
| EP3679942A1 (en) * | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| MX343088B (es) | 2010-06-25 | 2016-10-24 | Amcor Ltd | Sistema de depuración de oxígeno para un recipiente. |
| WO2011163652A2 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| EP2655624B1 (en) * | 2010-12-23 | 2017-11-29 | Biogen MA Inc. | Linker peptides and polypeptides comprising same |
| CN103764162B (zh) | 2011-03-04 | 2017-03-08 | 夏尔人类遗传治疗公司 | 用于多肽组成物的肽连接物及其使用方法 |
| 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 |
| KR102521039B1 (ko) | 2012-11-27 | 2023-04-12 | 바이오마린 파머수티컬 인크. | 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들 |
-
2013
- 2013-11-27 KR KR1020227011416A patent/KR102521039B1/ko active Active
- 2013-11-27 AU AU2013352184A patent/AU2013352184B2/en active Active
- 2013-11-27 ES ES13802834.5T patent/ES2679374T3/es active Active
- 2013-11-27 CN CN201380061935.4A patent/CN104822701B/zh active Active
- 2013-11-27 EP EP16182420.6A patent/EP3115372B1/en active Active
- 2013-11-27 KR KR1020157017224A patent/KR102262882B1/ko active Active
- 2013-11-27 MX MX2019009191A patent/MX377150B/es active IP Right Grant
- 2013-11-27 ES ES16182420T patent/ES2729997T3/es active Active
- 2013-11-27 WO PCT/US2013/072287 patent/WO2014085621A1/en not_active Ceased
- 2013-11-27 DK DK16182420.6T patent/DK3115372T3/da active
- 2013-11-27 HR HRP20181351TT patent/HRP20181351T1/hr unknown
- 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 PL PL13802834T patent/PL2925776T3/pl unknown
- 2013-11-27 CA CA2892146A patent/CA2892146A1/en active Pending
- 2013-11-27 DK DK13802834.5T patent/DK2925776T3/en active
- 2013-11-27 RS RS20190585A patent/RS58916B1/sr unknown
- 2013-11-27 PT PT16182420T patent/PT3115372T/pt unknown
- 2013-11-27 US US14/092,336 patent/US9376480B2/en active Active
- 2013-11-27 KR KR1020217017007A patent/KR102385392B1/ko active Active
- 2013-11-27 HU HUE16182420A patent/HUE043679T2/hu unknown
- 2013-11-27 HU HUE13802834A patent/HUE039334T2/hu unknown
- 2013-11-27 PT PT138028345T patent/PT2925776T/pt unknown
- 2013-11-27 SI SI201331450T patent/SI3115372T1/sl unknown
- 2013-11-27 JP JP2015544209A patent/JP6831176B2/ja active Active
- 2013-11-27 TW TW107114660A patent/TWI711632B/zh active
- 2013-11-27 LT LTEP16182420.6T patent/LT3115372T/lt unknown
- 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 KR KR1020237012007A patent/KR20230054482A/ko not_active Ceased
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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 US14/883,211 patent/US9771408B2/en active Active
- 2015-10-14 US US14/883,169 patent/US9845346B2/en active Active
- 2015-10-14 US US14/883,193 patent/US9834587B2/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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