JP2019528793A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019528793A5 JP2019528793A5 JP2019538714A JP2019538714A JP2019528793A5 JP 2019528793 A5 JP2019528793 A5 JP 2019528793A5 JP 2019538714 A JP2019538714 A JP 2019538714A JP 2019538714 A JP2019538714 A JP 2019538714A JP 2019528793 A5 JP2019528793 A5 JP 2019528793A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- polynucleotide
- adeno
- promoter
- associated virus
- 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
- 239000002157 polynucleotide Substances 0.000 claims 12
- 102000040430 polynucleotide Human genes 0.000 claims 12
- 108091033319 polynucleotide Proteins 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 8
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 230000002103 transcriptional effect Effects 0.000 claims 2
- 108010067390 Viral Proteins Proteins 0.000 claims 1
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16382450.1 | 2016-09-30 | ||
| EP16382450 | 2016-09-30 | ||
| PCT/EP2017/074081 WO2018060097A1 (en) | 2016-09-30 | 2017-09-22 | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019528793A JP2019528793A (ja) | 2019-10-17 |
| JP2019528793A5 true JP2019528793A5 (enExample) | 2020-11-12 |
| JP7059285B2 JP7059285B2 (ja) | 2022-04-25 |
Family
ID=57123944
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019538714A Active JP7059285B2 (ja) | 2016-09-30 | 2017-09-22 | ムコ多糖症の治療のためのアデノ随伴ウイルスベクター |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US11338046B2 (enExample) |
| EP (1) | EP3519569B1 (enExample) |
| JP (1) | JP7059285B2 (enExample) |
| CN (1) | CN109863243B (enExample) |
| AR (1) | AR109752A1 (enExample) |
| AU (1) | AU2017333336B2 (enExample) |
| ES (1) | ES2859661T3 (enExample) |
| PT (1) | PT3519569T (enExample) |
| TW (1) | TW201814044A (enExample) |
| WO (1) | WO2018060097A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9309534B2 (en) | 2010-07-12 | 2016-04-12 | Universidad Autonoma De Barcelona | Gene therapy composition for use in diabetes treatment |
| EP2692868A1 (en) | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| RU2692251C2 (ru) | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| US10973931B2 (en) | 2014-09-16 | 2021-04-13 | Universitat Autònoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
| ES2824457T3 (es) | 2014-12-05 | 2021-05-12 | UNIV AUTòNOMA DE BARCELONA | Vectores virales para el tratamiento de la diabetes |
| ES2900973T3 (es) | 2015-01-07 | 2022-03-21 | UNIV AUTòNOMA DE BARCELONA | Constructo genético de vector individual que comprende genes de insulina y glucoquinasa |
| BR112020021903A2 (pt) | 2018-04-27 | 2021-03-02 | Rocket Pharmaceuticals, Ltd. | terapia genética para degeneração do snc |
| EP3802803A1 (en) * | 2018-05-30 | 2021-04-14 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a |
| CA3106010A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| WO2021007473A1 (en) | 2019-07-10 | 2021-01-14 | Masonic Medical Research Institute | Vgll4 with ucp-1 cis-regulatory element and method of use thereof |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| CN116194154A (zh) | 2020-08-07 | 2023-05-30 | 太空飞船七有限责任公司 | 使用aav载体的plakophilin-2(pkp2)基因疗法 |
| CN114480454B (zh) * | 2020-10-27 | 2024-03-29 | 华东理工大学 | 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7232670B2 (en) * | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| US8227212B2 (en) * | 2003-02-11 | 2012-07-24 | Shire Human Genetic Therapies, Inc. | Cell that expresses a sulfatase and a formylglycine generating enzyme |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CN107982548A (zh) | 2012-02-07 | 2018-05-04 | 全球生物疗法有限公司 | 核酸输送的区室化方法及其组合物和应用 |
| CN105916990B (zh) * | 2013-08-02 | 2020-02-04 | 巴塞罗那自治大学 | 用于转导脂肪组织的腺相关病毒载体 |
| CA2927366A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| MX383227B (es) | 2014-05-14 | 2025-03-13 | UNIV AUTòNOMA DE BARCELONA | Vectores de virus adenoasociados para el tratamiento de trastornos de almacenamiento lisosomico. |
| AU2016219398A1 (en) | 2015-02-10 | 2017-09-28 | Genzyme Corporation | Enhanced delivery of viral particles to the striatum and cortex |
| US9981021B1 (en) * | 2015-04-09 | 2018-05-29 | Kinetiq, Inc. | Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof |
-
2017
- 2017-09-22 PT PT177775418T patent/PT3519569T/pt unknown
- 2017-09-22 US US16/337,953 patent/US11338046B2/en active Active
- 2017-09-22 ES ES17777541T patent/ES2859661T3/es active Active
- 2017-09-22 AU AU2017333336A patent/AU2017333336B2/en active Active
- 2017-09-22 WO PCT/EP2017/074081 patent/WO2018060097A1/en not_active Ceased
- 2017-09-22 EP EP17777541.8A patent/EP3519569B1/en active Active
- 2017-09-22 JP JP2019538714A patent/JP7059285B2/ja active Active
- 2017-09-22 CN CN201780060410.7A patent/CN109863243B/zh active Active
- 2017-09-29 AR ARP170102704A patent/AR109752A1/es unknown
- 2017-09-30 TW TW106133935A patent/TW201814044A/zh unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019528793A5 (enExample) | ||
| JP2018517414A5 (enExample) | ||
| RU2016149206A (ru) | Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления | |
| RU2017142006A (ru) | Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов | |
| Komoto et al. | Reverse genetics system demonstrates that rotavirus nonstructural protein NSP6 is not essential for viral replication in cell culture | |
| JP2020022459A5 (enExample) | ||
| FI3313991T3 (fi) | Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin | |
| JP2014012022A5 (enExample) | ||
| JP2014239686A5 (enExample) | ||
| HRP20180146T1 (hr) | Vektori i sljedovi namijenjeni liječenju bolesti | |
| JP2018520997A5 (enExample) | ||
| BR112019016769A2 (pt) | Métodos e composições para transferência genética através da vasculatura | |
| JP2015523379A5 (enExample) | ||
| JP2019519219A5 (enExample) | ||
| CL2019002280A1 (es) | Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235) | |
| JP2020522269A5 (enExample) | ||
| Gao et al. | Rewiring the RNAs of influenza virus to prevent reassortment | |
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| JP2020500541A5 (enExample) | ||
| JP2016520290A5 (enExample) | ||
| JP2016538885A5 (enExample) | ||
| JP2018534950A5 (enExample) | ||
| JP2013509168A5 (enExample) | ||
| JP2016512683A5 (enExample) | ||
| JP2019522461A5 (enExample) |