JP2022542829A - 改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス - Google Patents
改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス Download PDFInfo
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- JP2022542829A JP2022542829A JP2022502942A JP2022502942A JP2022542829A JP 2022542829 A JP2022542829 A JP 2022542829A JP 2022502942 A JP2022502942 A JP 2022502942A JP 2022502942 A JP2022502942 A JP 2022502942A JP 2022542829 A JP2022542829 A JP 2022542829A
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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
- C12N15/63—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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
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- 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
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- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0684—Cells of the urinary tract or kidneys
- C12N5/0686—Kidney cells
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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- C12N2820/00—Vectors comprising a special origin of replication system
- C12N2820/60—Vectors comprising a special origin of replication system from viruses
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Urology & Nephrology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16/512,051 US10653731B1 (en) | 2019-07-15 | 2019-07-15 | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
| US16/512,051 | 2019-07-15 | ||
| PCT/US2020/022326 WO2021011034A1 (en) | 2019-07-15 | 2020-03-12 | Recombinantly-modified adeno-associated virus (raav) having improved packaging efficiency |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022542829A true JP2022542829A (ja) | 2022-10-07 |
| JP2022542829A5 JP2022542829A5 (https=) | 2023-03-16 |
| JPWO2021011034A5 JPWO2021011034A5 (https=) | 2023-03-16 |
Family
ID=70736392
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022502942A Pending JP2022542829A (ja) | 2019-07-15 | 2020-03-12 | 改善されたパッケージング効率を有する組換え改変アデノ随伴ウイルス |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US10653731B1 (https=) |
| EP (1) | EP3999648A4 (https=) |
| JP (1) | JP2022542829A (https=) |
| KR (1) | KR20220034214A (https=) |
| CN (1) | CN114375337A (https=) |
| AU (1) | AU2020315513A1 (https=) |
| WO (1) | WO2021011034A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10653731B1 (en) | 2019-07-15 | 2020-05-19 | Vigene Biosciences Inc. | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
| EP4658759A2 (en) * | 2023-02-02 | 2025-12-10 | Agathos Biologics | Baby hamster kidney (bhk) cells transformed with the adenoviral e1 gene for production of recombinant adeno-associated virus |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014503173A (ja) * | 2009-11-05 | 2014-02-13 | プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ | 制御された発現系 |
| JP2018537969A (ja) * | 2015-10-29 | 2018-12-27 | テンプル ユニヴァーシティ − オブ ザ コモンウェルス システム オブ ハイアー エデュケーション | Dnaポリメラーゼシータによる核酸の3’末端の修飾 |
| US20190211357A1 (en) * | 2012-12-14 | 2019-07-11 | Case Western Reserve University | Genomic rna packaging enhancer element |
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| US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6642051B1 (en) | 1997-10-21 | 2003-11-04 | Targeted Genetics Corporation | Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors |
| IT1297074B1 (it) | 1997-11-21 | 1999-08-03 | Angeletti P Ist Richerche Bio | Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| DE19905501B4 (de) | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
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| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US6841357B1 (en) | 2001-03-30 | 2005-01-11 | Stratagene California | Method and kits for titering adeno-associated virus vectors |
| 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 |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| NZ532635A (en) * | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
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| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| US7625570B1 (en) | 2005-03-10 | 2009-12-01 | The Regents Of The University Of California | Methods for purifying adeno-associated virus |
| EP2311967B1 (en) | 2005-10-20 | 2017-09-20 | UniQure IP B.V. | Improved AAV vectors produced in insect cells |
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| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| EP2500434A1 (en) | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| WO2012145509A2 (en) | 2011-04-19 | 2012-10-26 | The Research Foundation Of State University Of New York | Adeno-associated-virus rep sequences, vectors, and viruses |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US9447433B2 (en) | 2013-03-15 | 2016-09-20 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| CN105765066B (zh) | 2013-11-29 | 2018-12-14 | 宝生物工程株式会社 | 用于定量腺伴随病毒的方法 |
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| US11208630B2 (en) | 2015-12-24 | 2021-12-28 | University Of Florida Research Foundation, Incorporated | AAV production using suspension adapted cells |
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| JP7632835B2 (ja) * | 2016-05-20 | 2025-02-19 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 加齢関連疾患及び症状の遺伝子治療法 |
| US10294452B2 (en) | 2016-11-22 | 2019-05-21 | Dao-Yao He | Lysis, extraction and purification of adeno-associated virus and adenovirus from host cells |
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| US10653731B1 (en) | 2019-07-15 | 2020-05-19 | Vigene Biosciences Inc. | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
-
2019
- 2019-07-15 US US16/512,051 patent/US10653731B1/en active Active
-
2020
- 2020-03-12 US US17/627,165 patent/US12156894B2/en active Active
- 2020-03-12 WO PCT/US2020/022326 patent/WO2021011034A1/en not_active Ceased
- 2020-03-12 KR KR1020227004899A patent/KR20220034214A/ko not_active Ceased
- 2020-03-12 CN CN202080064371.XA patent/CN114375337A/zh active Pending
- 2020-03-12 AU AU2020315513A patent/AU2020315513A1/en active Pending
- 2020-03-12 EP EP20839897.4A patent/EP3999648A4/en active Pending
- 2020-03-12 JP JP2022502942A patent/JP2022542829A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014503173A (ja) * | 2009-11-05 | 2014-02-13 | プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ | 制御された発現系 |
| US20190211357A1 (en) * | 2012-12-14 | 2019-07-11 | Case Western Reserve University | Genomic rna packaging enhancer element |
| JP2018537969A (ja) * | 2015-10-29 | 2018-12-27 | テンプル ユニヴァーシティ − オブ ザ コモンウェルス システム オブ ハイアー エデュケーション | Dnaポリメラーゼシータによる核酸の3’末端の修飾 |
Non-Patent Citations (3)
| Title |
|---|
| DARBY L THOMAS ET AL.: "Scalable recombinant adeno-associated virus production using recombinant herpes simplex vir", HUM GENE THER., vol. 20(8), JPN7024001111, 31 August 2009 (2009-08-31), pages 861 - 70, ISSN: 0005286214 * |
| HAIFENG CHEN ET AL.: "Adeno-associated virus vectors for human gene therapy", WORLD JOURNAL OF MEDICAL GENETICS, vol. 5, no. 3, JPN7024001112, 1 January 2015 (2015-01-01), pages 28 - 45, ISSN: 0005286213 * |
| STIFANI SATKUNANATHAN ET AL: "The function of DNA binding protein nucleophosmin in AAV replication", VIROLOGY, vol. 510, JPN7024001110, 31 December 2017 (2017-12-31), pages 46 - 54, ISSN: 0005286215 * |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220034214A (ko) | 2022-03-17 |
| US12156894B2 (en) | 2024-12-03 |
| US10653731B1 (en) | 2020-05-19 |
| US20220257678A1 (en) | 2022-08-18 |
| AU2020315513A1 (en) | 2022-02-17 |
| WO2021011034A1 (en) | 2021-01-21 |
| EP3999648A1 (en) | 2022-05-25 |
| CN114375337A (zh) | 2022-04-19 |
| EP3999648A4 (en) | 2023-08-16 |
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