CA2787827C - A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy - Google Patents
A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy Download PDFInfo
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- CA2787827C CA2787827C CA2787827A CA2787827A CA2787827C CA 2787827 C CA2787827 C CA 2787827C CA 2787827 A CA2787827 A CA 2787827A CA 2787827 A CA2787827 A CA 2787827A CA 2787827 C CA2787827 C CA 2787827C
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- aav vector
- aav
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| PCT/US2011/022371 WO2011094198A1 (en) | 2010-01-28 | 2011-01-25 | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
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| CA2787827C true CA2787827C (en) | 2020-11-10 |
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| PT (1) | PT2529020T (OSRAM) |
| WO (1) | WO2011094198A1 (OSRAM) |
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| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| ES2680915T3 (es) * | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| PT2744895E (pt) * | 2011-09-08 | 2016-02-08 | Uniqure Ip Bv | Remoção de vírus contaminantes de preparações de aav |
| EP2934532B1 (en) | 2012-12-20 | 2019-10-23 | Purdue Research Foundation | Chimeric antigen receptor-expressing t cells as anti-cancer therapeutics |
| FR3002237B1 (fr) * | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
| EP3415620B1 (en) | 2013-11-26 | 2021-03-31 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| MX376190B (es) | 2014-03-21 | 2025-03-07 | Genzyme Corp | PARTÍCULAS DE rAAV QUE CODIFICAN miR-708 Y USOS DEL MISMO. |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| JP6741590B2 (ja) | 2014-04-25 | 2020-08-19 | 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 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるａａｖベクター |
| EP3209311B1 (en) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Recombinant aav variants and uses thereof |
| PT3224376T (pt) * | 2014-11-28 | 2019-10-25 | Uniqure Ip Bv | Impurezas de dna numa composição compreendendo um vírus parvoviral. |
| JP6824169B2 (ja) | 2014-12-23 | 2021-02-03 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
| WO2016114992A2 (en) | 2015-01-13 | 2016-07-21 | Alfa Wassermann, Inc. | Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav) and gradients and flow-through buffers therefore |
| EP3054007A1 (en) * | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| US20180245073A1 (en) * | 2015-02-23 | 2018-08-30 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| ES3009565T3 (en) | 2015-04-16 | 2025-03-27 | Univ Emory | Recombinant promoters and vectors for protein expression in liver and use thereof |
| BR112017022621A2 (pt) | 2015-04-23 | 2018-07-17 | Baker Idi Heart And Diabetes Inst | liberação do gene de smad7 como uma substância terapêutica |
| EP3285780A4 (en) | 2015-04-24 | 2018-12-19 | University of Massachusetts | Modified aav constructions and uses thereof |
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