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
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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 (9)
| Country | Link |
|---|---|
| US (2) | US11473106B2 (enExample) |
| EP (2) | EP3562514B1 (enExample) |
| JP (1) | JP7128190B2 (enExample) |
| KR (2) | KR102863181B1 (enExample) |
| CN (1) | CN110290814A (enExample) |
| BR (1) | BR112019013245A2 (enExample) |
| CA (1) | CA3048044A1 (enExample) |
| MX (1) | MX2019007873A (enExample) |
| WO (1) | WO2018126116A1 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3562514B1 (en) * | 2016-12-30 | 2026-04-29 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
| JP7401432B2 (ja) | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス， インコーポレイテッド | 操作されたｄｎａ結合タンパク質 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| 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 |
| 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 |
| KR20210082460A (ko) | 2018-10-01 | 2021-07-05 | 울트라제닉스 파마수티컬 인코포레이티드 | 프로피온산혈증을 치료하기 위한 유전자 요법 |
| EP3880809A4 (en) * | 2018-11-16 | 2023-01-11 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF WILSON'S DISEASE |
| SG11202106855YA (en) * | 2019-01-04 | 2021-07-29 | Ultragenyx Pharmaceutical Inc | Gene therapy constructs for treating wilson disease |
| WO2020243651A1 (en) | 2019-05-29 | 2020-12-03 | 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 |
| CN114846145A (zh) * | 2019-11-26 | 2022-08-02 | 韩国生命工学研究院 | 用于蛋白质表达的mRNA构建体及其用途 |
| 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 |
| 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 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| 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 |
| WO2023069425A2 (en) * | 2021-10-18 | 2023-04-27 | Logicbio Therapeutics, Inc. | Gene therapy for the treatment of wilson''s disease |
| US20250320509A1 (en) * | 2022-05-23 | 2025-10-16 | The Johns Hopkins University | 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 |
| JP2026509434A (ja) * | 2023-03-06 | 2026-03-19 | リンイー・バイオテック・カンパニー・リミテッド | 切断型ａｔｐ７ｂ及びその使用 |
| EP4684025A1 (en) * | 2023-03-22 | 2026-01-28 | Skyline Therapeutics Limited | Recombinant aav for the gene therapy of wilson disease |
| 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 |
|---|---|---|---|---|
| AU2002350039A1 (en) | 2001-10-29 | 2003-05-12 | 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 |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| WO2005013090A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| KR20130040844A (ko) | 2010-03-29 | 2013-04-24 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 약학적으로 유발된 전이유전자 제거 시스템 |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2012097826A2 (en) | 2011-01-21 | 2012-07-26 | 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 |
| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| JP6865167B2 (ja) | 2014-12-17 | 2021-04-28 | フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター |
| ES2784264T3 (es) | 2014-12-17 | 2020-09-23 | Fundacion Para La Investig Medica Aplicada | Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson y otras afecciones |
| 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 |
| EP3562514B1 (en) | 2016-12-30 | 2026-04-29 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
| EP3880809A4 (en) * | 2018-11-16 | 2023-01-11 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF WILSON'S DISEASE |
| SG11202106855YA (en) | 2019-01-04 | 2021-07-29 | Ultragenyx Pharmaceutical Inc | Gene therapy constructs for treating wilson disease |
-
2017
- 2017-12-29 EP EP17888104.1A patent/EP3562514B1/en active Active
- 2017-12-29 EP EP24161028.6A patent/EP4361277A3/en active Pending
- 2017-12-29 JP JP2019535891A patent/JP7128190B2/ja active Active
- 2017-12-29 MX MX2019007873A patent/MX2019007873A/es unknown
- 2017-12-29 WO PCT/US2017/068919 patent/WO2018126116A1/en not_active Ceased
- 2017-12-29 KR KR1020237039367A patent/KR102863181B1/ko active Active
- 2017-12-29 CA CA3048044A patent/CA3048044A1/en active Pending
- 2017-12-29 CN CN201780085376.9A patent/CN110290814A/zh active Pending
- 2017-12-29 KR KR1020197021756A patent/KR102604096B1/ko active Active
- 2017-12-29 BR BR112019013245-7A patent/BR112019013245A2/pt unknown
- 2017-12-29 US US16/474,958 patent/US11473106B2/en active Active
-
2022
- 2022-07-13 US US17/812,322 patent/US12460228B2/en active Active
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