PT1064393E - Composicoes e metodos para a producao de virus adeno-associados recombinantes sem auxiliar - Google Patents
Composicoes e metodos para a producao de virus adeno-associados recombinantes sem auxiliarInfo
- Publication number
- PT1064393E PT1064393E PT99912640T PT99912640T PT1064393E PT 1064393 E PT1064393 E PT 1064393E PT 99912640 T PT99912640 T PT 99912640T PT 99912640 T PT99912640 T PT 99912640T PT 1064393 E PT1064393 E PT 1064393E
- Authority
- PT
- Portugal
- Prior art keywords
- aav
- virus
- gene product
- contaminating
- absence
- Prior art date
Links
Classifications
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- 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
-
- 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
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- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
- C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
- C12N2830/006—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB tet repressible
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/60—Vector systems having a special element relevant for transcription from viruses
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US7890898P | 1998-03-20 | 1998-03-20 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT1064393E true PT1064393E (pt) | 2005-04-29 |
Family
ID=22146942
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT99912640T PT1064393E (pt) | 1998-03-20 | 1999-03-18 | Composicoes e metodos para a producao de virus adeno-associados recombinantes sem auxiliar |
Country Status (9)
| Country | Link |
|---|---|
| EP (1) | EP1064393B1 (pt) |
| JP (1) | JP2002506652A (pt) |
| AT (1) | ATE286138T1 (pt) |
| AU (1) | AU3097399A (pt) |
| CA (1) | CA2324225A1 (pt) |
| DE (1) | DE69922934T2 (pt) |
| ES (1) | ES2235470T3 (pt) |
| PT (1) | PT1064393E (pt) |
| WO (1) | WO1999047691A1 (pt) |
Families Citing this family (94)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6924128B2 (en) | 1994-12-06 | 2005-08-02 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| 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 |
| EP1916258B1 (en) | 1999-08-09 | 2014-04-23 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| WO2001059142A1 (en) * | 2000-02-09 | 2001-08-16 | Medimmune, Inc. | Antibody gene therapy with adeno-associated viral vectors |
| CA2410512A1 (en) * | 2000-06-09 | 2001-12-13 | University Of Florida Research Foundation, Inc. | Recombinant aav vectors for gene therapy of obesity |
| DE10056210A1 (de) * | 2000-11-13 | 2002-05-29 | Arimedes Biotechnology Gmbh | Virales Expressionssystem |
| DE10120265A1 (de) * | 2001-04-25 | 2002-11-14 | Deutsches Krebsforsch | AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren |
| DE10137283A1 (de) * | 2001-08-01 | 2003-02-27 | Deutsches Krebsforsch | AAV-Vektor-Verpackungsplasmide zur Helfervirus-freien Herstellung pseudotypisierter AAV-Partikel über Einzeltransfektion |
| NZ578982A (en) | 2001-11-13 | 2011-03-31 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
| EP1944043A1 (en) | 2001-11-21 | 2008-07-16 | The Trustees of the University of Pennsylvania | Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use |
| EP3108899A1 (en) | 2001-11-21 | 2016-12-28 | The Trustees of the University of Pennsylvania | Simian adenovirus adsv1 nucleic acid and amino acid sequences, vectors containing same, and methods of use |
| JP4769417B2 (ja) | 2001-12-17 | 2011-09-07 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用 |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| JP4811765B2 (ja) * | 2004-06-22 | 2011-11-09 | 学校法人東海大学 | 外来遺伝子の誘導発現の制御が可能な発現ベクター |
| KR101614369B1 (ko) | 2007-11-28 | 2016-04-21 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 유인원 아과 ｃ 아데노바이러스 ｓａｄｖ-40, -31, 및 -34 및 그것의 사용 |
| US8685387B2 (en) | 2007-11-28 | 2014-04-01 | The Trustees Of The University Of Pennsylvania | Simian E adenoviruses SAdV-39, -25.2, -26, -30, -37, and -38 |
| US8470310B2 (en) | 2008-03-04 | 2013-06-25 | The Trustees Of The University Of Pennsylvania | Simian adenoviruses SAdV-36, -42.1, -42.2, and -44 and uses thereof |
| WO2010045659A1 (en) | 2008-10-17 | 2010-04-22 | American Gene Technologies International Inc. | Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules |
| ES2552688T3 (es) | 2008-10-31 | 2015-12-01 | The Trustees Of The University Of Pennsylvania | Adenovirus de simio con proteínas de la cápside hexónica de SAdV-46 y usos del mismo |
| CA2759801C (en) | 2009-05-02 | 2019-04-02 | Marco A. Passini | Gene therapy for neurodegenerative disorders |
| US8846031B2 (en) | 2009-05-29 | 2014-09-30 | The Trustees Of The University Of Pennsylvania | Simian adenovirus 41 and uses thereof |
| AU2011332025B2 (en) | 2010-11-23 | 2015-06-25 | The Trustees Of The University Of Pennsylvania | Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof |
| BR112014028684A2 (pt) | 2012-05-18 | 2017-07-25 | Univ Pennsylvania | subfamília e adenovírus de símio a1302, a1320, a1331 e a1337 e usos dos mesmos |
| PL3116900T3 (pl) | 2014-03-09 | 2021-03-08 | The Trustees Of The University Of Pennsylvania | Kompozycje użyteczne w leczeniu niedoboru transkarbamylazy ornitynowej (otc) |
| WO2016200997A1 (en) * | 2015-06-10 | 2016-12-15 | America Gene Technologies International, Inc. | Non-integrating viral delivery system and methods of use thereof |
| US11980663B2 (en) | 2015-07-08 | 2024-05-14 | American Gene Technologies International Inc. | HIV pre-immunization and immunotherapy |
| US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| WO2017123918A1 (en) | 2016-01-15 | 2017-07-20 | American Gene Technologies International Inc. | Methods and compositons for the activation of gamma-delta t-cells |
| US10137144B2 (en) | 2016-01-15 | 2018-11-27 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
| WO2017139065A1 (en) | 2016-02-08 | 2017-08-17 | American Gene Technologies International Inc. | Hiv vaccination and immunotherapy |
| ES2911448T3 (es) | 2016-03-09 | 2022-05-19 | American Gene Tech Int Inc | Vectores combinados y métodos para el tratamiento del cáncer |
| WO2017213697A1 (en) * | 2016-06-08 | 2017-12-14 | American Gene Technologies International Inc. | Non-integrating viral delivery system and methods related thereto |
| CA3028982A1 (en) | 2016-07-08 | 2018-01-11 | American Gene Technologies International Inc. | Hiv pre-immunization and immunotherapy |
| JP7176756B2 (ja) | 2016-07-21 | 2022-11-22 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | パーキンソン病を処置するためのウイルスベクター |
| KR20190136048A (ko) | 2017-04-03 | 2019-12-09 | 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 | 페닐케톤뇨증을 치료하기 위한 조성물 및 방법 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP4124658A3 (en) | 2017-10-16 | 2023-04-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP3762500A1 (en) | 2018-03-06 | 2021-01-13 | Voyager Therapeutics, Inc. | Insect cell manufactured partial self-complementary aav genomes |
| JP2021521852A (ja) | 2018-04-27 | 2021-08-30 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | Ａａｄｃウイルスベクターの効力を測定する方法 |
| JP2021522813A (ja) * | 2018-05-07 | 2021-09-02 | スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． | プラスミドを用いないａａｖベクター産生細胞株 |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
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| WO2019222441A1 (en) | 2018-05-16 | 2019-11-21 | Voyager Therapeutics, Inc. | Aav serotypes for brain specific payload delivery |
| WO2019222444A2 (en) | 2018-05-16 | 2019-11-21 | Voyager Therapeutics, Inc. | Directed evolution |
| EP3807404A1 (en) | 2018-06-13 | 2021-04-21 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| JP7565218B2 (ja) | 2018-07-02 | 2024-10-10 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
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| WO2021211753A1 (en) | 2020-04-15 | 2021-10-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
| GB202005732D0 (en) | 2020-04-20 | 2020-06-03 | Synpromics Ltd | Regulatory nucleic acid sequences |
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| CN116829725A (zh) * | 2021-02-12 | 2023-09-29 | 富士胶片株式会社 | 用于产生腺相关病毒的试剂盒及其利用 |
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| WO2024059739A1 (en) | 2022-09-15 | 2024-03-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
| WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
| WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
| WO2024145474A2 (en) | 2022-12-29 | 2024-07-04 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5436146A (en) * | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US5173414A (en) * | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US6686200B1 (en) * | 1993-08-31 | 2004-02-03 | Uab Research Foundation | Methods and compositions for the large scale production of recombinant adeno-associated virus |
| US5872005A (en) * | 1994-11-03 | 1999-02-16 | Cell Genesys Inc. | Packaging cell lines for adeno-associated viral vectors |
| AU707862B2 (en) * | 1995-06-07 | 1999-07-22 | University Of North Carolina At Chapel Hill, The | Helper virus-free aav production |
| US6004797A (en) * | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| AU723497C (en) * | 1996-09-06 | 2001-10-11 | Trustees Of The University Of Pennsylvania, The | Method for recombinant adeno-associated virus-directed gene therapy |
-
1999
- 1999-03-18 CA CA002324225A patent/CA2324225A1/en not_active Abandoned
- 1999-03-18 PT PT99912640T patent/PT1064393E/pt unknown
- 1999-03-18 EP EP99912640A patent/EP1064393B1/en not_active Expired - Lifetime
- 1999-03-18 DE DE69922934T patent/DE69922934T2/de not_active Expired - Fee Related
- 1999-03-18 ES ES99912640T patent/ES2235470T3/es not_active Expired - Lifetime
- 1999-03-18 WO PCT/US1999/005870 patent/WO1999047691A1/en active IP Right Grant
- 1999-03-18 AU AU30973/99A patent/AU3097399A/en not_active Abandoned
- 1999-03-18 AT AT99912640T patent/ATE286138T1/de not_active IP Right Cessation
- 1999-03-18 JP JP2000536874A patent/JP2002506652A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| ES2235470T3 (es) | 2005-07-01 |
| EP1064393A1 (en) | 2001-01-03 |
| JP2002506652A (ja) | 2002-03-05 |
| CA2324225A1 (en) | 1999-09-23 |
| DE69922934T2 (de) | 2005-12-08 |
| EP1064393B1 (en) | 2004-12-29 |
| ATE286138T1 (de) | 2005-01-15 |
| WO1999047691A1 (en) | 1999-09-23 |
| AU3097399A (en) | 1999-10-11 |
| DE69922934D1 (de) | 2005-02-03 |
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