JP2020048571A5 - - Google Patents
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- JP2020048571A5 JP2020048571A5 JP2019214352A JP2019214352A JP2020048571A5 JP 2020048571 A5 JP2020048571 A5 JP 2020048571A5 JP 2019214352 A JP2019214352 A JP 2019214352A JP 2019214352 A JP2019214352 A JP 2019214352A JP 2020048571 A5 JP2020048571 A5 JP 2020048571A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- cells
- raav
- raav particles
- particles
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 239000002245 particle Substances 0.000 claims 35
- 210000004027 cell Anatomy 0.000 claims 13
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 8
- 208000014674 injury Diseases 0.000 claims 8
- 239000000203 mixture Substances 0.000 claims 8
- 210000002845 virion Anatomy 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 7
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- 150000007523 nucleic acids Chemical class 0.000 claims 7
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- 208000014018 liver neoplasm Diseases 0.000 claims 6
- 230000026683 transduction Effects 0.000 claims 6
- 238000010361 transduction Methods 0.000 claims 6
- 108090000565 Capsid Proteins Proteins 0.000 claims 5
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- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 3
- 206010028980 Neoplasm Diseases 0.000 claims 3
- 239000002246 antineoplastic agent Substances 0.000 claims 3
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- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
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- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
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- 102000040125 5-hydroxytryptamine receptor family Human genes 0.000 claims 1
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- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
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- 239000005556 hormone Substances 0.000 claims 1
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- 229940079322 interferon Drugs 0.000 claims 1
- 102000002467 interleukin receptors Human genes 0.000 claims 1
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- 239000002502 liposome Chemical class 0.000 claims 1
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- 229940053128 nerve growth factor Drugs 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
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- 239000000137 peptide hydrolase inhibitor Substances 0.000 claims 1
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- 239000013646 rAAV2 vector Substances 0.000 claims 1
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022011418A JP7344595B2 (ja) | 2013-05-21 | 2022-01-28 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/899,481 US9920097B2 (en) | 2007-04-09 | 2013-05-21 | Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use |
| US13/899,481 | 2013-05-21 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016515056A Division JP6628286B2 (ja) | 2013-05-21 | 2014-05-21 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011418A Division JP7344595B2 (ja) | 2013-05-21 | 2022-01-28 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020048571A JP2020048571A (ja) | 2020-04-02 |
| JP2020048571A5 true JP2020048571A5 (enExample) | 2020-06-25 |
| JP7021786B2 JP7021786B2 (ja) | 2022-02-17 |
Family
ID=51023068
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016515056A Active JP6628286B2 (ja) | 2013-05-21 | 2014-05-21 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
| JP2019214352A Active JP7021786B2 (ja) | 2013-05-21 | 2019-11-27 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
| JP2022011418A Active JP7344595B2 (ja) | 2013-05-21 | 2022-01-28 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016515056A Active JP6628286B2 (ja) | 2013-05-21 | 2014-05-21 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022011418A Active JP7344595B2 (ja) | 2013-05-21 | 2022-01-28 | キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| EP (2) | EP2999791B1 (enExample) |
| JP (3) | JP6628286B2 (enExample) |
| KR (4) | KR102649543B1 (enExample) |
| CN (3) | CN105408486B (enExample) |
| AU (3) | AU2014274457B2 (enExample) |
| CA (1) | CA2917018A1 (enExample) |
| DK (1) | DK2999791T3 (enExample) |
| SG (2) | SG11201510757PA (enExample) |
| WO (1) | WO2014193716A2 (enExample) |
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| US5410017A (en) * | 1993-05-21 | 1995-04-25 | Rohm And Haas Company | Continuous thermal polycondensation process for preparing polypeptide polymers |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US20160369298A1 (en) | 2013-09-26 | 2016-12-22 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
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| US20180030096A1 (en) | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
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| WO2018035213A1 (en) * | 2016-08-16 | 2018-02-22 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
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| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| MD3645551T2 (ro) * | 2017-06-27 | 2024-08-31 | Regeneron Pharma | Vectori virali recombinanţi modificați cu tropism și utilizările acestora pentru introducerea țintită a materialului genetic în celulele umane |
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| CN112218880A (zh) * | 2018-03-29 | 2021-01-12 | 阿斯克肋匹奥生物制药公司 | 逃避中和的肝趋向性重组aav6载体 |
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| JP7142815B2 (ja) * | 2018-06-21 | 2022-09-28 | 株式会社遺伝子治療研究所 | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| CN112703198B (zh) | 2018-07-11 | 2025-05-30 | 布里格姆妇女医院 | 用于跨血脑屏障递送试剂的方法和组合物 |
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| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
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| KR102862676B1 (ko) | 2020-11-11 | 2025-09-23 | 유럽피안 몰레큘러 바이올로지 래보러토리 | 유전자 치료를 위한 변형된 바이러스 입자 |
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| US6156303A (en) * | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1486567A1 (en) * | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2359865B1 (en) * | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| EP3492596A1 (en) * | 2007-04-09 | 2019-06-05 | University of Florida Research Foundation, Inc. | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US20120093772A1 (en) * | 2008-05-20 | 2012-04-19 | Alan Horsager | Vectors for delivery of light sensitive proteins and methods of use |
| US9409953B2 (en) * | 2011-02-10 | 2016-08-09 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| CN103060331B (zh) * | 2012-12-05 | 2014-10-08 | 南京医科大学第一附属医院 | 介导膜稳定cd40l基因的衣壳蛋白突变的双链重组腺相关病毒制备方法与应用 |
| US20180030096A1 (en) * | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
-
2014
- 2014-05-21 CN CN201480041247.6A patent/CN105408486B/zh active Active
- 2014-05-21 KR KR1020227033180A patent/KR102649543B1/ko active Active
- 2014-05-21 CN CN202010551899.1A patent/CN111763690A/zh active Pending
- 2014-05-21 EP EP14733803.2A patent/EP2999791B1/en active Active
- 2014-05-21 WO PCT/US2014/039015 patent/WO2014193716A2/en not_active Ceased
- 2014-05-21 KR KR1020217008950A patent/KR102298579B1/ko active Active
- 2014-05-21 SG SG11201510757PA patent/SG11201510757PA/en unknown
- 2014-05-21 JP JP2016515056A patent/JP6628286B2/ja active Active
- 2014-05-21 CA CA2917018A patent/CA2917018A1/en active Pending
- 2014-05-21 KR KR1020217027678A patent/KR20210111878A/ko not_active Ceased
- 2014-05-21 CN CN202010039122.7A patent/CN111411125A/zh active Pending
- 2014-05-21 KR KR1020157036164A patent/KR102234672B1/ko active Active
- 2014-05-21 EP EP18202680.7A patent/EP3492597A3/en active Pending
- 2014-05-21 SG SG10201709552XA patent/SG10201709552XA/en unknown
- 2014-05-21 AU AU2014274457A patent/AU2014274457B2/en active Active
- 2014-05-21 DK DK14733803.2T patent/DK2999791T3/en active
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2019
- 2019-11-27 JP JP2019214352A patent/JP7021786B2/ja active Active
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2020
- 2020-01-03 AU AU2020200041A patent/AU2020200041B2/en active Active
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2022
- 2022-01-28 JP JP2022011418A patent/JP7344595B2/ja active Active
- 2022-06-17 AU AU2022204246A patent/AU2022204246A1/en not_active Abandoned
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