CA2348838A1 - Production amelioree de vecteurs de virus associes aux adenovirus - Google Patents
Production amelioree de vecteurs de virus associes aux adenovirus Download PDFInfo
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- CA2348838A1 CA2348838A1 CA002348838A CA2348838A CA2348838A1 CA 2348838 A1 CA2348838 A1 CA 2348838A1 CA 002348838 A CA002348838 A CA 002348838A CA 2348838 A CA2348838 A CA 2348838A CA 2348838 A1 CA2348838 A1 CA 2348838A1
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- 238000002731 protein assay Methods 0.000 description 1
- 238000010814 radioimmunoprecipitation assay Methods 0.000 description 1
- 238000011552 rat model Methods 0.000 description 1
- 238000009790 rate-determining step (RDS) Methods 0.000 description 1
- 230000006798 recombination Effects 0.000 description 1
- 238000005215 recombination Methods 0.000 description 1
- 230000022532 regulation of transcription, DNA-dependent Effects 0.000 description 1
- 230000004044 response Effects 0.000 description 1
- 108091008146 restriction endonucleases Proteins 0.000 description 1
- 230000000717 retained effect Effects 0.000 description 1
- 235000009566 rice Nutrition 0.000 description 1
- 239000000523 sample Substances 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 238000002415 sodium dodecyl sulfate polyacrylamide gel electrophoresis Methods 0.000 description 1
- 239000007787 solid Substances 0.000 description 1
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- 238000003860 storage Methods 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
- 108010066082 tartrate-sensitive acid phosphatase Proteins 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 238000004448 titration Methods 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
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- 230000009466 transformation Effects 0.000 description 1
- 230000001131 transforming effect Effects 0.000 description 1
- 239000002753 trypsin inhibitor Substances 0.000 description 1
- 238000009281 ultraviolet germicidal irradiation Methods 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
- 238000010200 validation analysis Methods 0.000 description 1
- 230000007442 viral DNA synthesis Effects 0.000 description 1
- 230000006648 viral gene expression Effects 0.000 description 1
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
-
- 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
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)
Abstract
L'invention concerne le domaine des vecteurs viraux produits par génie génétique, et plus particulièrement des vecteurs viraux associés aux adénovirus (AAV), utilisés en thérapie génique. L'invention concerne également un procédé de production de vecteurs viraux associés aux adénovirus recombinants à titrage élevé, essentiellement exempts de virus auxiliaires tel qu'un adénovirus. L'invention concerne, en outre, une cellule d'encapsidation des AAV contenant un acide nucléique codant pour un produit génique qui fournit une fonction d'auxiliaire aux AAV, ce qui permet de produire des AAV recombinants sans produire de virus auxiliaires concomitants.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP98203635 | 1998-10-27 | ||
| EP98203635.2 | 1998-10-27 | ||
| PCT/NL1999/000663 WO2000024916A1 (fr) | 1998-10-27 | 1999-10-27 | Production amelioree de vecteurs de virus associes aux adenovirus |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2348838A1 true CA2348838A1 (fr) | 2000-05-04 |
Family
ID=8234269
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002348838A Abandoned CA2348838A1 (fr) | 1998-10-27 | 1999-10-27 | Production amelioree de vecteurs de virus associes aux adenovirus |
Country Status (6)
| Country | Link |
|---|---|
| EP (1) | EP1124976A1 (fr) |
| JP (1) | JP2002528087A (fr) |
| AU (1) | AU764130B2 (fr) |
| CA (1) | CA2348838A1 (fr) |
| MX (1) | MXPA01004169A (fr) |
| WO (1) | WO2000024916A1 (fr) |
Families Citing this family (77)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1572893B1 (fr) | 2001-11-09 | 2009-01-07 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES | Production d'un virus adeno-associe dans des cellules d'insectes |
| 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 |
| PL1945779T3 (pl) | 2005-10-20 | 2013-08-30 | Uniqure Ip Bv | Ulepszone wektory AAV produkowane w komórkach owadzich |
| GB0602063D0 (en) | 2006-02-02 | 2006-03-15 | Univ Manchester | Cell Culture |
| CA2655957C (fr) | 2006-06-21 | 2016-05-03 | Amsterdam Molecular Therapeutics (Amt) B.V. | Vecteurs presentant un codon d'initiation modifie pour la traduction d'aav-rep78, utilises pour la production de l'aav dans des cellules d'insectes |
| EP2061891B1 (fr) | 2006-08-24 | 2012-04-11 | Virovek, Inc. | Expression dans des cellules d'insecte de gènes ayant des cadres ouverts de lecture chevauchants, procédés et compositions pour cela |
| EP3151866B1 (fr) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Capsides chimériques |
| KR20170096998A (ko) | 2014-11-05 | 2017-08-25 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드 |
| WO2016077689A1 (fr) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Polynucléotides modulateurs |
| WO2016077687A1 (fr) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de la sclérose latérale amyotrophique (sla) |
| WO2016094783A1 (fr) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions et méthodes pour la production de scaav |
| CA2994160C (fr) | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Sequences virales ancestrales et leurs utilisations |
| EP3368054A4 (fr) | 2015-10-28 | 2019-07-03 | Voyager Therapeutics, Inc. | Expression régulable au moyen d'un virus adéno-associé (vaa) |
| DK3436593T3 (da) | 2016-03-28 | 2023-02-20 | Ultragenyx Pharmaceutical Inc | Fremgangsmåder til varmeinaktivering af adenovirus |
| EP3448987A4 (fr) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | Compositions pour le traitement de maladies |
| EP3448874A4 (fr) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | Compositions pour le traitement de maladies |
| CA3024448C (fr) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | Polynucleotides modulateurs |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | composições e métodos de tratamento da doença de huntington |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| US11795473B2 (en) | 2016-10-14 | 2023-10-24 | Ultragenyx Pharmaceutical Inc. | Use of tonicifying agents to enhance recombinant adeno-associated virus yield |
| EP3601581B1 (fr) | 2017-03-22 | 2025-04-30 | Ultragenyx Pharmaceutical Inc. | Procédés de culture cellulaire faisant intervenir des inhibiteurs de hdac ou des protéines rep |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| WO2018204786A1 (fr) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de la sclérose latérale amyotrophique (sla) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (fr) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Systeme de guide de trajectoire d'appareillage en reseau |
| EP3662060A2 (fr) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions et procedes de délivrance d'aav |
| US20200231986A1 (en) | 2017-09-29 | 2020-07-23 | Massachusetts Eye And Ear Infirmary | Production of adeno-associated viruses in insect cells |
| CA3075656A1 (fr) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Sauvetage de phenotype neurologique central et peripherique de l'ataxie de friedreich par administration intraveineuse |
| EP4454654A3 (fr) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Traitement de la sclérose latérale amyotrophique (sla) |
| WO2019079240A1 (fr) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | Traitement de la sclérose latérale amyotrophique (sla) |
| EA202092229A1 (ru) | 2018-03-21 | 2021-07-05 | Зе Скриппс Рисёрч Инститьют | Мутеины cd4 и способы их применения |
| EP3775266A4 (fr) | 2018-04-05 | 2021-06-30 | Massachusetts Eye and Ear Infirmary | Procédés de fabrication et d'utilisation de bibliothèques combinatoires d'acides nucléiques à code-barres ayant une variation définie |
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| JP2021523914A (ja) | 2018-05-15 | 2021-09-09 | 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． | パーキンソン病を治療するための組成物および方法 |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒（ａａｖ）之組成物和方法 |
| US20210214749A1 (en) | 2018-05-16 | 2021-07-15 | Voyager Therapeutics, Inc. | Directed evolution |
| EP3793686A1 (fr) | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Sérotypes de vaa pour l'administration de charge utile spécifique au cerveau |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (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 |
| US20210355454A1 (en) | 2018-07-24 | 2021-11-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| US20210348242A1 (en) | 2018-10-04 | 2021-11-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| AU2019354793A1 (en) | 2018-10-05 | 2021-05-13 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding AAV production proteins |
| WO2020077165A1 (fr) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions et procédés pour l'administration d'aav |
| EP3867389A1 (fr) | 2018-10-15 | 2021-08-25 | Voyager Therapeutics, Inc. | Vecteurs d'expression pour la production à grande échelle de raav dans le système baculovirus/sf9 |
| US20220064671A1 (en) | 2019-01-18 | 2022-03-03 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| WO2020210507A1 (fr) | 2019-04-12 | 2020-10-15 | Ultragenyx Pharmaceutical Inc. | Lignées cellulaires productrices modifiées et leurs procédés de fabrication et d'utilisation |
| EP3962536A1 (fr) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Système et procédé pour la production de cellules d'insectes infectées par baculovirus (ceib) dans les bioréacteurs |
| US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| WO2021030125A1 (fr) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs |
| TW202122582A (zh) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
| WO2021046155A1 (fr) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Édition vectorisée d'acides nucléiques pour corriger des mutations manifestes |
| KR20220076510A (ko) | 2019-10-08 | 2022-06-08 | 트러스티스 오브 보스톤 칼리지 | 다수의 서로 다른 비천연 아미노산을 함유하는 단백질 및 이러한 단백질의 제조 및 사용 방법 |
| WO2022074464A2 (fr) | 2020-03-05 | 2022-04-14 | Neotx Therapeutics Ltd. | Méthodes et compositions pour le traitement du cancer à l'aide de cellules immunitaires |
| EP4121520A1 (fr) | 2020-03-16 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Procédés d'amélioration du rendement de virus adéno-associé recombinant |
| WO2021211753A1 (fr) | 2020-04-15 | 2021-10-21 | Voyager Therapeutics, Inc. | Composés de liaison à la protéine tau |
| WO2021247995A2 (fr) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de la douleur neuropathique |
| EP4189095A1 (fr) | 2020-07-27 | 2023-06-07 | Voyager Therapeutics, Inc. | Compositions et procédés pour le traitement des troubles neurologiques liés au déficit en glucosylcéramidase bêta |
| US20230285596A1 (en) | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| WO2022032153A1 (fr) | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs |
| JP7733110B2 (ja) | 2020-11-02 | 2025-09-02 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルスを濃縮するためのプロセス |
| EP4006141A1 (fr) * | 2020-11-25 | 2022-06-01 | CEVEC Pharmaceuticals GmbH | Procédé de production d'aav |
| CA3216491A1 (fr) | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Virions de vaa a polyploide rationnel traversant la barriere hemato-encephalique et declenchant une reponse humorale reduite |
| WO2023034996A1 (fr) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Compositions capsidiques de vaa et méthodes d'administration |
| WO2023034980A1 (fr) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Compositions capsidiques de vaa et méthodes d'administration |
| WO2023034990A1 (fr) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Compositions capsidiques de vaa et méthodes d'administration |
| US20250122530A1 (en) | 2021-09-03 | 2025-04-17 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4396199A1 (fr) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Compositions capsidiques de vaa et méthodes d'administration |
| WO2023034994A1 (fr) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Compositions capsidiques de vaa et méthodes d'administration |
| US20250223376A1 (en) | 2021-09-20 | 2025-07-10 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| US20250019668A1 (en) | 2021-10-29 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Engineered cell lines for increased production of recombinant adeno-associated virus (raav) |
| WO2023091949A2 (fr) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de troubles neurologiques liés à un déficit en bêta glucosylcéramidase |
| WO2023240236A1 (fr) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale |
| CN120019071A (zh) | 2022-09-15 | 2025-05-16 | 沃雅戈治疗公司 | Tau结合化合物 |
| KR20250129743A (ko) | 2022-12-29 | 2025-08-29 | 보이저 테라퓨틱스, 인크. | Mapt를 조절하기 위한 조성물 및 방법 |
| WO2024163012A1 (fr) | 2023-02-02 | 2024-08-08 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de troubles neurologiques liés à un déficit en bêta glucosylcéramidase |
| WO2024226761A2 (fr) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions et méthodes de traitement de la sclérose latérale amyotrophique |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP4108746B2 (ja) * | 1995-06-07 | 2008-06-25 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ヘルパーウイルスを含まないａａｖ産生 |
| KR100470180B1 (ko) * | 1995-06-15 | 2005-02-04 | 크루셀 홀란드 비.브이. | 유전자 요법에 사용할 수 있는 인간의 재조합아데노바이러스 패키징 시스템 |
| JPH11514853A (ja) * | 1995-09-08 | 1999-12-21 | ジエンザイム コーポレイション | 遺伝子治療のための改良されたａａｖベクター |
| US6004797A (en) * | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| WO1997030167A1 (fr) * | 1996-02-13 | 1997-08-21 | The Trustees Of The University Of Pennsylvania | Procede de traitement des troubles du foie |
-
1999
- 1999-10-27 EP EP99971042A patent/EP1124976A1/fr not_active Withdrawn
- 1999-10-27 CA CA002348838A patent/CA2348838A1/fr not_active Abandoned
- 1999-10-27 JP JP2000578468A patent/JP2002528087A/ja not_active Withdrawn
- 1999-10-27 AU AU11885/00A patent/AU764130B2/en not_active Ceased
- 1999-10-27 WO PCT/NL1999/000663 patent/WO2000024916A1/fr not_active Ceased
- 1999-10-27 MX MXPA01004169A patent/MXPA01004169A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MXPA01004169A (es) | 2002-06-04 |
| AU764130B2 (en) | 2003-08-14 |
| EP1124976A1 (fr) | 2001-08-22 |
| AU1188500A (en) | 2000-05-15 |
| WO2000024916A1 (fr) | 2000-05-04 |
| JP2002528087A (ja) | 2002-09-03 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FZDE | Discontinued |