PL3740222T3 - Zmodyfikowane białko kapsydu raav do terapii genowej - Google Patents
Zmodyfikowane białko kapsydu raav do terapii genowejInfo
- Publication number
- PL3740222T3 PL3740222T3 PL19701080.4T PL19701080T PL3740222T3 PL 3740222 T3 PL3740222 T3 PL 3740222T3 PL 19701080 T PL19701080 T PL 19701080T PL 3740222 T3 PL3740222 T3 PL 3740222T3
- Authority
- PL
- Poland
- Prior art keywords
- gene therapy
- capsid protein
- raav capsid
- modified raav
- modified
- Prior art date
Links
Classifications
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
-
- 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
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Dermatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18152133 | 2018-01-17 | ||
| PCT/EP2019/051128 WO2019141765A1 (en) | 2018-01-17 | 2019-01-17 | A modified raav capsid protein for gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3740222T3 true PL3740222T3 (pl) | 2023-12-04 |
Family
ID=61022137
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL19701080.4T PL3740222T3 (pl) | 2018-01-17 | 2019-01-17 | Zmodyfikowane białko kapsydu raav do terapii genowej |
Country Status (27)
| Country | Link |
|---|---|
| US (1) | US12480138B2 (pl) |
| EP (1) | EP3740222B1 (pl) |
| JP (1) | JP7389040B2 (pl) |
| KR (1) | KR102919991B1 (pl) |
| CN (1) | CN112004544B (pl) |
| AU (1) | AU2019209595B2 (pl) |
| BR (1) | BR112020014625A2 (pl) |
| CA (1) | CA3087910C (pl) |
| DK (1) | DK3740222T5 (pl) |
| EA (1) | EA202091712A1 (pl) |
| ES (1) | ES2957622T3 (pl) |
| FI (1) | FI3740222T3 (pl) |
| HR (1) | HRP20231126T1 (pl) |
| HU (1) | HUE062774T2 (pl) |
| IL (1) | IL275838B2 (pl) |
| LT (1) | LT3740222T (pl) |
| MX (1) | MX2020006764A (pl) |
| MY (1) | MY202252A (pl) |
| PH (1) | PH12020551096A1 (pl) |
| PL (1) | PL3740222T3 (pl) |
| PT (1) | PT3740222T (pl) |
| RS (1) | RS64499B1 (pl) |
| SG (1) | SG11202006056RA (pl) |
| SI (1) | SI3740222T1 (pl) |
| SM (1) | SMT202300284T1 (pl) |
| WO (1) | WO2019141765A1 (pl) |
| ZA (1) | ZA202004980B (pl) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| JP2020530307A (ja) * | 2017-06-30 | 2020-10-22 | インティマ・バイオサイエンス，インコーポレーテッド | 遺伝子治療のためのアデノ随伴ウイルスベクター |
| RS64499B1 (sr) | 2018-01-17 | 2023-09-29 | Meiragtx Uk Ii Ltd | Modifikovani protein kapsida raav za gensku terapiju |
| JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
| WO2019195423A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| WO2019195444A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| KR20220081971A (ko) * | 2019-07-15 | 2022-06-16 | 메이라지티엑스 유케이 Ii 리미티드 | 관절염 질환 치료용 변형된 aav 캡시드 단백질 |
| AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| KR20220083708A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | Aav 전달 카세트 |
| CN110950934B (zh) * | 2019-12-31 | 2022-11-04 | 复旦大学 | 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用 |
| EP4110792A4 (en) * | 2020-02-25 | 2024-08-21 | Children's Medical Research Institute | POLYPEPTIDES AND CAPSIDIC VECTORS OF ADENO-ASSOCIATED VIRUSES |
| MX2023002016A (es) | 2020-08-19 | 2023-06-26 | Sarepta Therapeutics Inc | Vectores de virus adenoasociados para el tratamiento del sindrome de rett. |
| GB202110014D0 (en) * | 2021-07-12 | 2021-08-25 | Cytiva Bioprocess R & D Ab | A method for separating adeno-associated virus capsids, compositions obtained by said method and uses thereof |
| EP4413147A4 (en) | 2021-10-08 | 2025-07-30 | Dyno Therapeutics Inc | CAPSID VARIANTS AND METHODS OF USE THEREOF |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024213660A1 (en) | 2023-04-13 | 2024-10-17 | Meiragtx Uk Ii Limited | Inflammation-inducible promoters |
| CN119320802A (zh) * | 2023-07-17 | 2025-01-17 | 苏州吉恒基因科技有限公司 | 单链单极DNA重组腺相关病毒的cis包装元件、spAAV载体和应用 |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| AU2002360355B2 (en) | 2001-11-09 | 2005-07-07 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| US8529885B2 (en) * | 2003-09-01 | 2013-09-10 | Academisch Medisch Centrum | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
| DK1664315T3 (da) * | 2003-09-01 | 2012-07-09 | Amc Amsterdam | AAV-vektorer til in vivo genterapi af rheumatoid arthritis |
| AU2010201278B2 (en) * | 2003-09-01 | 2012-11-15 | Academisch Medisch Centrum | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
| AU2006304997B2 (en) | 2005-10-20 | 2012-03-01 | Uniqure Ip B.V. | Improved AAV vectors produced in insect cells |
| WO2007120542A2 (en) * | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| ES2785223T3 (es) | 2006-06-21 | 2020-10-06 | Uniqure Ip Bv | Células de insecto para la producción de vectores de AAV |
| EP2061891B1 (en) | 2006-08-24 | 2012-04-11 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| EP2012122A1 (en) | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| SI2173888T1 (sl) | 2007-07-26 | 2017-01-31 | Uniqure Ip B.V. | Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami |
| ES2644880T3 (es) | 2008-02-19 | 2017-11-30 | Uniqure Ip B.V. | Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto |
| EP2297185A1 (en) | 2008-06-17 | 2011-03-23 | Amsterdam Molecular Therapeutics (AMT) B.V. | Parvoviral capsid with incorporated gly-ala repeat region |
| EP2545165B1 (en) | 2010-03-11 | 2020-07-29 | uniQure IP B.V. | Mutated rep encoding sequences for use in aav production |
| AU2012304993B2 (en) | 2011-09-08 | 2017-09-14 | Uniqure Ip B.V. | Removal of contaminating viruses from AAV preparations |
| WO2013174760A1 (en) * | 2012-05-25 | 2013-11-28 | Deutsches Krebsforschungszentrum | Optimized aav-vectors for high transduction rates in dendritic cells |
| KR102298579B1 (ko) * | 2013-05-21 | 2021-09-07 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| CN104107438A (zh) * | 2014-05-29 | 2014-10-22 | 中国人民解放军军事医学科学院基础医学研究所 | Spry2在制备预防和治疗类风湿性关节炎药物中的应用 |
| WO2016055437A1 (en) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Aav-based gene therapy |
| US10876134B2 (en) * | 2014-12-16 | 2020-12-29 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| DK3402530T3 (da) | 2016-01-13 | 2020-11-23 | Genzyme Corp | Rekombinante aav-vektorer, der udtrykker knoglebeskyttende gener, herunder has2 og lubricin, som er anvendelige til behandling af osteoarthritis og relaterede ledtilstande hos pattedyr |
| US11207382B2 (en) | 2016-08-19 | 2021-12-28 | University Of Florida Research Foundation, Incorporated | Compositions for treating conditions using recombinant self-complementary adeno-associated virus |
| KR20250121159A (ko) | 2016-12-07 | 2025-08-11 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 인터류킨-1 수용체 길항제(IL-1RA) cDNA |
| WO2018189244A1 (en) | 2017-04-11 | 2018-10-18 | Ruprecht-Karls-Universität Heidelberg | Adeno-associated virus library |
| KR102612563B1 (ko) * | 2017-08-31 | 2023-12-11 | 베니텍 아이피 홀딩스 아이엔씨. | 변형된 포스포리파제 도메인을 갖는 아데노-연관 바이러스 (aav) |
| RS64499B1 (sr) | 2018-01-17 | 2023-09-29 | Meiragtx Uk Ii Ltd | Modifikovani protein kapsida raav za gensku terapiju |
| KR20220081971A (ko) * | 2019-07-15 | 2022-06-16 | 메이라지티엑스 유케이 Ii 리미티드 | 관절염 질환 치료용 변형된 aav 캡시드 단백질 |
-
2019
- 2019-01-17 RS RS20230730A patent/RS64499B1/sr unknown
- 2019-01-17 EA EA202091712A patent/EA202091712A1/ru unknown
- 2019-01-17 MX MX2020006764A patent/MX2020006764A/es unknown
- 2019-01-17 WO PCT/EP2019/051128 patent/WO2019141765A1/en not_active Ceased
- 2019-01-17 US US16/962,748 patent/US12480138B2/en active Active
- 2019-01-17 CA CA3087910A patent/CA3087910C/en active Active
- 2019-01-17 PT PT197010804T patent/PT3740222T/pt unknown
- 2019-01-17 SM SM20230284T patent/SMT202300284T1/it unknown
- 2019-01-17 PL PL19701080.4T patent/PL3740222T3/pl unknown
- 2019-01-17 KR KR1020207023174A patent/KR102919991B1/ko active Active
- 2019-01-17 EP EP19701080.4A patent/EP3740222B1/en active Active
- 2019-01-17 ES ES19701080T patent/ES2957622T3/es active Active
- 2019-01-17 DK DK19701080.4T patent/DK3740222T5/da active
- 2019-01-17 FI FIEP19701080.4T patent/FI3740222T3/fi active
- 2019-01-17 LT LTEPPCT/EP2019/051128T patent/LT3740222T/lt unknown
- 2019-01-17 IL IL275838A patent/IL275838B2/en unknown
- 2019-01-17 AU AU2019209595A patent/AU2019209595B2/en active Active
- 2019-01-17 BR BR112020014625-0A patent/BR112020014625A2/pt unknown
- 2019-01-17 HU HUE19701080A patent/HUE062774T2/hu unknown
- 2019-01-17 JP JP2020539801A patent/JP7389040B2/ja active Active
- 2019-01-17 CN CN201980020100.1A patent/CN112004544B/zh active Active
- 2019-01-17 SG SG11202006056RA patent/SG11202006056RA/en unknown
- 2019-01-17 SI SI201930613T patent/SI3740222T1/sl unknown
- 2019-01-17 MY MYPI2020003653A patent/MY202252A/en unknown
- 2019-01-17 HR HRP20231126TT patent/HRP20231126T1/hr unknown
-
2020
- 2020-07-17 PH PH12020551096A patent/PH12020551096A1/en unknown
- 2020-08-12 ZA ZA2020/04980A patent/ZA202004980B/en unknown
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