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
- 241000702421 Dependoparvovirus Species 0.000 claims 10
- 239000003623 enhancer Substances 0.000 claims 8
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims 7
- 208000018839 Wilson disease Diseases 0.000 claims 7
- 239000000725 suspension Substances 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 239000007900 aqueous suspension Substances 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 108091006112 ATPases Proteins 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 102000057290 Adenosine Triphosphatases Human genes 0.000 claims 2
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 claims 2
- 101000727865 Homo sapiens Copper transport protein ATOX1 Proteins 0.000 claims 2
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 102000051131 human ATOX1 Human genes 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 102000013918 Apolipoproteins E Human genes 0.000 claims 1
- 108010025628 Apolipoproteins E Proteins 0.000 claims 1
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 239000003755 preservative agent Substances 0.000 claims 1
- 230000002335 preservative effect Effects 0.000 claims 1
- 239000004094 surface-active agent Substances 0.000 claims 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 1
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) | EP3562514A4 (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 |
|---|---|---|---|---|
| MX2019007873A (es) * | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| AU2018375192B2 (en) | 2017-12-01 | 2023-11-09 | Encoded Therapeutics, Inc. | Engineered DNA binding proteins |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019152843A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function 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 |
| AR116569A1 (es) | 2018-10-01 | 2021-05-19 | Ultragenyx Pharmaceutical Inc | Terapia génica para tratar la acidemia propiónica |
| CN113302290A (zh) * | 2018-11-16 | 2021-08-24 | 编码治疗公司 | 治疗威尔逊氏病的组合物和方法 |
| US12338450B2 (en) * | 2019-01-04 | 2025-06-24 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating Wilson disease |
| TWI872078B (zh) | 2019-05-29 | 2025-02-11 | 美商編碼製藥公司 | 用於選擇性基因調節的組合物和方法 |
| 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 |
| WO2021107647A1 (ko) * | 2019-11-26 | 2021-06-03 | 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 |
| 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 |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | 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 |
| 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 |
| 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 |
| WO2025026198A1 (en) * | 2023-07-28 | 2025-02-06 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of wilson disease |
| 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 |
|---|---|---|---|---|
| US7985553B2 (en) | 2001-10-29 | 2011-07-26 | Nathaniel Heintz | 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 |
| JP4810062B2 (ja) * | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| 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 |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | 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 |
| 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 |
| CN104520428B (zh) | 2012-02-17 | 2018-09-21 | 费城儿童医院 | 将基因转移到细胞、器官和组织的aav载体组合物和方法 |
| AU2013243952A1 (en) | 2012-04-02 | 2014-10-30 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal 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 |
| 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 |
| PT3233129T (pt) * | 2014-12-17 | 2020-04-15 | Fundacion Para La Investig Medica Aplicada | Construções de ácido nucleico e vetores de terapia génica para utilização no tratamento de doença de wilson e outras condições |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | 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. |
| CN113302290A (zh) * | 2018-11-16 | 2021-08-24 | 编码治疗公司 | 治疗威尔逊氏病的组合物和方法 |
| US12338450B2 (en) | 2019-01-04 | 2025-06-24 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating Wilson disease |
-
2017
- 2017-12-29 MX MX2019007873A patent/MX2019007873A/es unknown
- 2017-12-29 CN CN201780085376.9A patent/CN110290814A/zh active Pending
- 2017-12-29 EP EP17888104.1A patent/EP3562514A4/en active Pending
- 2017-12-29 EP EP24161028.6A patent/EP4361277A3/en active Pending
- 2017-12-29 WO PCT/US2017/068919 patent/WO2018126116A1/en not_active Ceased
- 2017-12-29 JP JP2019535891A patent/JP7128190B2/ja active Active
- 2017-12-29 US US16/474,958 patent/US11473106B2/en active Active
- 2017-12-29 KR KR1020237039367A patent/KR102863181B1/ko active Active
- 2017-12-29 CA CA3048044A patent/CA3048044A1/en active Pending
- 2017-12-29 KR KR1020197021756A patent/KR102604096B1/ko active Active
- 2017-12-29 BR BR112019013245-7A patent/BR112019013245A2/pt unknown
-
2022
- 2022-07-13 US US17/812,322 patent/US12460228B2/en active Active