JP2020514286A5 - - Google Patents
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- Publication number
- JP2020514286A5 JP2020514286A5 JP2019535891A JP2019535891A JP2020514286A5 JP 2020514286 A5 JP2020514286 A5 JP 2020514286A5 JP 2019535891 A JP2019535891 A JP 2019535891A JP 2019535891 A JP2019535891 A JP 2019535891A JP 2020514286 A5 JP2020514286 A5 JP 2020514286A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- promoter
- suspension
- enhancer
- aav
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662440659P | 2016-12-30 | 2016-12-30 | |
| US62/440,659 | 2016-12-30 | ||
| US201762473656P | 2017-03-20 | 2017-03-20 | |
| US62/473,656 | 2017-03-20 | ||
| PCT/US2017/068919 WO2018126116A1 (en) | 2016-12-30 | 2017-12-29 | Gene therapy for treating wilson's disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020514286A JP2020514286A (ja) | 2020-05-21 |
| JP2020514286A5 true JP2020514286A5 (enExample) | 2021-01-28 |
| JP7128190B2 JP7128190B2 (ja) | 2022-08-30 |
Family
ID=62710046
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019535891A Active JP7128190B2 (ja) | 2016-12-30 | 2017-12-29 | ウィルソン病を処置するための遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11473106B2 (enExample) |
| EP (2) | EP4361277A3 (enExample) |
| JP (1) | JP7128190B2 (enExample) |
| KR (2) | KR102863181B1 (enExample) |
| CN (1) | CN110290814A (enExample) |
| BR (1) | BR112019013245A2 (enExample) |
| CA (1) | CA3048044A1 (enExample) |
| DK (1) | DK3562514T3 (enExample) |
| ES (1) | ES3072955T3 (enExample) |
| MX (1) | MX2019007873A (enExample) |
| WO (1) | WO2018126116A1 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102863181B1 (ko) * | 2016-12-30 | 2025-09-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 윌슨병을 치료하기 위한 유전자 치료 |
| CN117821509A (zh) | 2017-04-03 | 2024-04-05 | 编码治疗公司 | 组织选择性转基因表达 |
| CN118345115A (zh) | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| AU2019215152A1 (en) | 2018-02-01 | 2020-08-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof |
| WO2019152841A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for pah gene transfer and methods of use thereof |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| AU2019354296A1 (en) * | 2018-10-01 | 2021-04-29 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| CA3118936A1 (en) * | 2018-11-16 | 2020-05-22 | Encoded Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| KR20210112339A (ko) * | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| SG11202113048SA (en) | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
| CN111088285B (zh) * | 2019-08-15 | 2021-12-28 | 北京锦篮基因科技有限公司 | 携带atp7b基因表达框及变异体的aav载体及应用 |
| KR102490457B1 (ko) * | 2019-11-26 | 2023-01-19 | 한국생명공학연구원 | 단백질 발현용 mRNA 구조체 및 이의 용도 |
| 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 |
| US12686874B2 (en) | 2020-03-31 | 2026-07-21 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| 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 |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| WO2022208342A1 (en) * | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| 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 |
| TW202323528A (zh) * | 2021-10-18 | 2023-06-16 | 美商邏輯生物療法公司 | 用於治療威爾森氏病之基因療法 |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
| CN115216533A (zh) * | 2022-06-30 | 2022-10-21 | 湖南家辉生物技术有限公司 | 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用 |
| EP4677077A4 (en) * | 2023-03-06 | 2026-07-29 | Lingyi Biotech Co Ltd | TRUNCATED ATP7B GENE AND ITS USAGE |
| JP2026508707A (ja) * | 2023-03-22 | 2026-03-11 | スカイライン、セラピューティクス、リミテッド | ウィルソン病の遺伝子療法のための組換えａａｖ |
| US20240360431A1 (en) * | 2023-04-30 | 2024-10-31 | Sichuan Real&Best Biotech Co., Ltd. | Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease |
| 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 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003038049A2 (en) | 2001-10-29 | 2003-05-08 | Renovis, Inc. | Method for isolating cell-type specific mrnas |
| 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 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of 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 |
| EP2665813A2 (en) | 2011-01-21 | 2013-11-27 | Aarhus Universitet | Crystal structure of a type ib p-type atpase |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| 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 |
| CN107208105A (zh) | 2014-12-17 | 2017-09-26 | 基础应用医学研究基金会 | 用于治疗威尔森病的核酸构建体和基因治疗载体 |
| 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 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| KR102863181B1 (ko) * | 2016-12-30 | 2025-09-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 윌슨병을 치료하기 위한 유전자 치료 |
| CA3118936A1 (en) | 2018-11-16 | 2020-05-22 | Encoded Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
-
2017
- 2017-12-29 KR KR1020237039367A patent/KR102863181B1/ko active Active
- 2017-12-29 EP EP24161028.6A patent/EP4361277A3/en active Pending
- 2017-12-29 ES ES17888104T patent/ES3072955T3/es active Active
- 2017-12-29 US US16/474,958 patent/US11473106B2/en active Active
- 2017-12-29 MX MX2019007873A patent/MX2019007873A/es unknown
- 2017-12-29 KR KR1020197021756A patent/KR102604096B1/ko active Active
- 2017-12-29 EP EP17888104.1A patent/EP3562514B1/en active Active
- 2017-12-29 WO PCT/US2017/068919 patent/WO2018126116A1/en not_active Ceased
- 2017-12-29 CN CN201780085376.9A patent/CN110290814A/zh active Pending
- 2017-12-29 BR BR112019013245-7A patent/BR112019013245A2/pt unknown
- 2017-12-29 DK DK17888104.1T patent/DK3562514T3/da active
- 2017-12-29 CA CA3048044A patent/CA3048044A1/en active Pending
- 2017-12-29 JP JP2019535891A patent/JP7128190B2/ja active Active
-
2022
- 2022-07-13 US US17/812,322 patent/US12460228B2/en active Active
-
2025
- 2025-10-07 US US19/351,377 patent/US20260152761A1/en active Pending
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