JP2024054426A5 - - Google Patents
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- Publication number
- JP2024054426A5 JP2024054426A5 JP2024031460A JP2024031460A JP2024054426A5 JP 2024054426 A5 JP2024054426 A5 JP 2024054426A5 JP 2024031460 A JP2024031460 A JP 2024031460A JP 2024031460 A JP2024031460 A JP 2024031460A JP 2024054426 A5 JP2024054426 A5 JP 2024054426A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- composition
- raav
- acid sequence
- seq
- 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
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962788324P | 2019-01-04 | 2019-01-04 | |
| US62/788,324 | 2019-01-04 | ||
| US201962834830P | 2019-04-16 | 2019-04-16 | |
| US62/834,830 | 2019-04-16 | ||
| PCT/US2020/012131 WO2020142653A1 (en) | 2019-01-04 | 2020-01-03 | Gene therapy constructs for treating wilson disease |
| JP2021538754A JP7531495B2 (ja) | 2019-01-04 | 2020-01-03 | ウイルソン病を処置するための遺伝子治療構築物 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538754A Division JP7531495B2 (ja) | 2019-01-04 | 2020-01-03 | ウイルソン病を処置するための遺伝子治療構築物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024054426A JP2024054426A (ja) | 2024-04-16 |
| JP2024054426A5 true JP2024054426A5 (enExample) | 2025-02-03 |
| JP7746437B2 JP7746437B2 (ja) | 2025-09-30 |
Family
ID=69528954
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538754A Active JP7531495B2 (ja) | 2019-01-04 | 2020-01-03 | ウイルソン病を処置するための遺伝子治療構築物 |
| JP2024031460A Active JP7746437B2 (ja) | 2019-01-04 | 2024-03-01 | ウイルソン病を処置するための遺伝子治療構築物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538754A Active JP7531495B2 (ja) | 2019-01-04 | 2020-01-03 | ウイルソン病を処置するための遺伝子治療構築物 |
Country Status (22)
| Country | Link |
|---|---|
| US (2) | US12338450B2 (enExample) |
| EP (2) | EP3906066B1 (enExample) |
| JP (2) | JP7531495B2 (enExample) |
| KR (1) | KR20210112339A (enExample) |
| CN (1) | CN113518628B (enExample) |
| AU (1) | AU2020204679B2 (enExample) |
| BR (1) | BR112021013140A2 (enExample) |
| CA (1) | CA3124880A1 (enExample) |
| CO (1) | CO2021010088A2 (enExample) |
| DK (1) | DK3906066T5 (enExample) |
| ES (1) | ES2970216T3 (enExample) |
| FI (1) | FI3906066T3 (enExample) |
| HR (1) | HRP20231750T1 (enExample) |
| HU (1) | HUE065782T2 (enExample) |
| IL (1) | IL284554A (enExample) |
| MX (1) | MX2021008135A (enExample) |
| PL (1) | PL3906066T3 (enExample) |
| PT (1) | PT3906066T (enExample) |
| SG (1) | SG11202106855YA (enExample) |
| TW (1) | TWI848038B (enExample) |
| WO (1) | WO2020142653A1 (enExample) |
| ZA (1) | ZA202104515B (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| MX2020010464A (es) | 2018-04-03 | 2021-01-29 | Vectores de virus que evitan anticuerpos. | |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| PL3906066T3 (pl) | 2019-01-04 | 2024-09-02 | Ultragenyx Pharmaceutical Inc. | Konstrukty do terapii genowej do leczenia choroby wilsona |
| JP7698583B2 (ja) | 2019-03-21 | 2025-06-25 | ギンコ バイオワークス インコーポレイテッド | 組換えアデノ随伴ウイルスベクター |
| MX2021013913A (es) * | 2019-05-14 | 2022-03-17 | Univ Duke | Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa. |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| CA3189878A1 (en) | 2020-08-19 | 2022-02-24 | Colin O'BANION | Adeno-associated virus vectors for treatment of rett syndrome |
| CN115216481A (zh) * | 2021-04-21 | 2022-10-21 | 四川至善唯新生物科技有限公司 | 一种表达量提高的截短型atp7b基因及其应用 |
| CN113694218A (zh) * | 2021-08-30 | 2021-11-26 | 昆明理工大学 | 一种atp7b基因p992l突变的基因修复治疗载体 |
| IL313427A (en) * | 2021-12-10 | 2024-08-01 | Aavantibio Inc | Methods and preparations for treating BAG-3-related cardiomyopathy with a viral vector |
| IL313304A (en) * | 2021-12-10 | 2024-08-01 | Aavantibio Inc | Compositions comprising KOZAK sequences selected for enhanced expression |
| WO2024183769A1 (en) * | 2023-03-06 | 2024-09-12 | Lingyi Biotech Co., Ltd. | A truncated atp7b and the use thereof |
| WO2024193638A1 (en) * | 2023-03-22 | 2024-09-26 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of wilson disease |
| WO2025002226A1 (zh) * | 2023-06-28 | 2025-01-02 | 迈威(上海)生物科技股份有限公司 | 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用 |
| CN120882865A (zh) * | 2023-07-28 | 2025-10-31 | 凌意(杭州)生物科技有限公司 | 用于治疗威尔逊病的多核苷酸 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| AU2002350039A1 (en) * | 2001-10-29 | 2003-05-12 | Renovis, Inc. | Method for isolating cell-type specific mrnas |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| DE10156121A1 (de) | 2001-11-16 | 2003-05-28 | Wolfgang Stremmel | Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011094198A1 (en) | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| WO2013123503A1 (en) * | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| EP2834259A4 (en) | 2012-04-02 | 2016-08-24 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| WO2015168666A2 (en) * | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| RU2745567C2 (ru) * | 2014-12-17 | 2021-03-29 | Фундасион Пара Ла Инвестигасион Медика Апликада | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний |
| US11118238B2 (en) | 2014-12-17 | 2021-09-14 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| WO2020102723A1 (en) | 2018-11-16 | 2020-05-22 | Encoded Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| PL3906066T3 (pl) | 2019-01-04 | 2024-09-02 | Ultragenyx Pharmaceutical Inc. | Konstrukty do terapii genowej do leczenia choroby wilsona |
| US20250320509A1 (en) * | 2022-05-23 | 2025-10-16 | The Johns Hopkins University | Compositions and methods for treating wilson's disease |
-
2020
- 2020-01-03 PL PL20704633.5T patent/PL3906066T3/pl unknown
- 2020-01-03 WO PCT/US2020/012131 patent/WO2020142653A1/en not_active Ceased
- 2020-01-03 HU HUE20704633A patent/HUE065782T2/hu unknown
- 2020-01-03 DK DK20704633.5T patent/DK3906066T5/da active
- 2020-01-03 US US17/417,619 patent/US12338450B2/en active Active
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 TW TW109100147A patent/TWI848038B/zh active
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko active Pending
- 2020-01-03 JP JP2021538754A patent/JP7531495B2/ja active Active
- 2020-01-03 CA CA3124880A patent/CA3124880A1/en active Pending
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/en active Active
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 EP EP23196931.2A patent/EP4269579A3/en active Pending
- 2020-01-03 CN CN202080007863.5A patent/CN113518628B/zh active Active
- 2020-01-03 AU AU2020204679A patent/AU2020204679B2/en active Active
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
-
2021
- 2021-06-29 ZA ZA2021/04515A patent/ZA202104515B/en unknown
- 2021-07-01 IL IL284554A patent/IL284554A/en unknown
- 2021-07-29 CO CONC2021/0010088A patent/CO2021010088A2/es unknown
-
2024
- 2024-03-01 JP JP2024031460A patent/JP7746437B2/ja active Active
-
2025
- 2025-05-20 US US19/213,623 patent/US20250333763A1/en active Pending
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