JP2022068181A5 - - Google Patents
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- Publication number
- JP2022068181A5 JP2022068181A5 JP2022011418A JP2022011418A JP2022068181A5 JP 2022068181 A5 JP2022068181 A5 JP 2022068181A5 JP 2022011418 A JP2022011418 A JP 2022011418A JP 2022011418 A JP2022011418 A JP 2022011418A JP 2022068181 A5 JP2022068181 A5 JP 2022068181A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- aav capsid
- aav
- amino acid
- nucleic acid
- 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.)
- Granted
Links
- 108090000565 Capsid Proteins Proteins 0.000 claims 73
- 102100023321 Ceruloplasmin Human genes 0.000 claims 73
- 241000702421 Dependoparvovirus Species 0.000 claims 61
- 125000003275 alpha amino acid group Chemical group 0.000 claims 20
- 238000006467 substitution reaction Methods 0.000 claims 20
- 102000039446 nucleic acids Human genes 0.000 claims 18
- 108020004707 nucleic acids Proteins 0.000 claims 18
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 16
- 239000002245 particle Substances 0.000 claims 12
- 230000026683 transduction Effects 0.000 claims 12
- 238000010361 transduction Methods 0.000 claims 12
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 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
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 208000014674 injury Diseases 0.000 claims 2
- 241000124008 Mammalia Species 0.000 claims 1
- 208000027418 Wounds and injury Diseases 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 230000008733 trauma Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/899,481 | 2013-05-21 | ||
| US13/899,481 US9920097B2 (en) | 2007-04-09 | 2013-05-21 | Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use |
| JP2019214352A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019214352A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022068181A JP2022068181A (ja) | 2022-05-09 |
| JP2022068181A5 true JP2022068181A5 (enExample) | 2022-08-08 |
| JP7344595B2 JP7344595B2 (ja) | 2023-09-14 |
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 (2)
| 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 |
Country Status (9)
| Country | Link |
|---|---|
| EP (2) | EP2999791B1 (enExample) |
| JP (3) | JP6628286B2 (enExample) |
| KR (4) | KR102298579B1 (enExample) |
| CN (3) | CN111763690A (enExample) |
| AU (3) | AU2014274457B2 (enExample) |
| CA (1) | CA2917018A1 (enExample) |
| DK (1) | DK2999791T3 (enExample) |
| SG (2) | SG11201510757PA (enExample) |
| WO (1) | WO2014193716A2 (enExample) |
Families Citing this family (57)
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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 |
| JP6985795B2 (ja) | 2013-09-26 | 2021-12-22 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
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| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| JP7406783B2 (ja) * | 2015-12-14 | 2023-12-28 | ザ 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 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| BR112019002904A2 (pt) * | 2016-08-16 | 2019-06-25 | The University Of North Carolina At Chapel Hill | métodos e composições para transferência gênica direcionada |
| IL264872B2 (en) * | 2016-08-18 | 2025-02-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| CN110892062A (zh) | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
| DK3645551T5 (da) * | 2017-06-27 | 2024-08-26 | Regeneron Pharma | Tropisme-modificerede rekombinante virusvektorer og anvendelser deraf til targetteret indføring af genetisk materiale i menneskeceller |
| EP3679148A4 (en) * | 2017-09-08 | 2021-06-09 | Generation Bio Co. | LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS |
| JP2021510539A (ja) * | 2018-01-16 | 2021-04-30 | シーエルエス セラピューティクス リミテッドＣＬＳ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ | デオキシリボヌクレアーゼ（ＤＮａｓｅ）活性を有する酵素の肝臓発現による疾患の処置 |
| US12480138B2 (en) * | 2018-01-17 | 2025-11-25 | MeiraGTx Gene Regulation Limited | Modified rAAV capsid protein for gene therapy |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| CN120665951A (zh) * | 2018-02-07 | 2025-09-19 | 吉尼松公司 | 杂合调控元件 |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| 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 |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| JP7331011B2 (ja) | 2018-04-27 | 2023-08-22 | デシベル セラピューティクス インコーポレイテッド | ミオシン１５プロモーター及びその使用 |
| EP3784697A4 (en) * | 2018-04-27 | 2022-07-06 | Spark Therapeutics, Inc. | Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same |
| 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 |
| EP3820885A4 (en) | 2018-07-11 | 2022-04-20 | The Brigham & Women's Hospital, Inc. | METHODS AND COMPOSITIONS FOR ADMINISTRATION OF DRUGS ACROSS THE BLOOD-BRAIN BARRIER |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| WO2020079256A1 (en) * | 2018-10-19 | 2020-04-23 | Genethon | Modulation, monitoring and prediction of the immune response directed against aav gene therapy vectors |
| AU2020208933B2 (en) | 2019-01-18 | 2025-08-28 | Centre National De La Recherche Scientifique (Cnrs) | AAV-mediated gene therapy restoring the otoferlin gene |
| CA3129422A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| WO2020163743A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| EP3736330A1 (en) * | 2019-05-08 | 2020-11-11 | European Molecular Biology Laboratory | Modified adeno-associated virus (aav) particles for gene therapy |
| EP3999120A4 (en) * | 2019-07-04 | 2024-02-21 | Children's Medical Research Institute | Methods and aav vectors for in vivo transduction |
| US20220282276A1 (en) * | 2019-07-11 | 2022-09-08 | Centre National De La Recherche Scientifique | Chemically-modified adeno-associated virus |
| AU2020347276A1 (en) * | 2019-09-12 | 2022-03-17 | Massachusetts Institute Of Technology | Engineered adeno-associated virus capsids |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| US20230340526A1 (en) * | 2019-11-25 | 2023-10-26 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target hepatocytes and evade the humoral immune response |
| US20230049066A1 (en) * | 2019-11-25 | 2023-02-16 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target human hepatocytes in the liver of humanized mice |
| AU2021206256A1 (en) * | 2020-01-10 | 2022-07-28 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| JP2023524577A (ja) * | 2020-05-05 | 2023-06-12 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 改変アデノ随伴ウイルス５カプシドおよびその使用 |
| 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 |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
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| CN113647359A (zh) * | 2021-08-15 | 2021-11-16 | 芜湖职业技术学院 | 注射衣霉素诱导急性肝损伤小鼠模型的构建方法 |
| WO2023072181A1 (en) * | 2021-10-28 | 2023-05-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Adeno-associated virus capsid |
| WO2023201368A2 (en) * | 2022-04-15 | 2023-10-19 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| CN119380820B (zh) * | 2024-10-10 | 2025-06-17 | 四川大学华西医院 | 一种aav2衣壳蛋白变体设计方法、系统和存储介质 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP2357010B1 (en) * | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| 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 |
| HUE030719T2 (en) * | 2007-04-09 | 2017-05-29 | Univ Florida | RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use |
| MX2010012592A (es) * | 2008-05-20 | 2011-05-05 | Eos Neuroscience Inc | Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas. |
| EP4234571A3 (en) * | 2011-02-10 | 2023-09-27 | 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 AU AU2014274457A patent/AU2014274457B2/en active Active
- 2014-05-21 KR KR1020217008950A patent/KR102298579B1/ko active Active
- 2014-05-21 CN CN202010551899.1A patent/CN111763690A/zh active Pending
- 2014-05-21 JP JP2016515056A patent/JP6628286B2/ja active Active
- 2014-05-21 WO PCT/US2014/039015 patent/WO2014193716A2/en not_active Ceased
- 2014-05-21 EP EP14733803.2A patent/EP2999791B1/en active Active
- 2014-05-21 SG SG11201510757PA patent/SG11201510757PA/en unknown
- 2014-05-21 DK DK14733803.2T patent/DK2999791T3/en active
- 2014-05-21 SG SG10201709552XA patent/SG10201709552XA/en unknown
- 2014-05-21 KR KR1020157036164A patent/KR102234672B1/ko active Active
- 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 CN202010039122.7A patent/CN111411125A/zh active Pending
- 2014-05-21 KR KR1020217027678A patent/KR20210111878A/ko not_active Ceased
- 2014-05-21 CA CA2917018A patent/CA2917018A1/en active Pending
- 2014-05-21 EP EP18202680.7A patent/EP3492597A3/en active Pending
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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