JPWO2020167996A5 - - Google Patents
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- Publication number
- JPWO2020167996A5 JPWO2020167996A5 JP2021546891A JP2021546891A JPWO2020167996A5 JP WO2020167996 A5 JPWO2020167996 A5 JP WO2020167996A5 JP 2021546891 A JP2021546891 A JP 2021546891A JP 2021546891 A JP2021546891 A JP 2021546891A JP WO2020167996 A5 JPWO2020167996 A5 JP WO2020167996A5
- Authority
- JP
- Japan
- Prior art keywords
- gene therapy
- therapy vector
- raav gene
- raav
- expression
- 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.)
- Pending
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025007633A JP2025063228A (ja) | 2019-02-12 | 2025-01-20 | ダノン病の治療のための遺伝子療法ベクター |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962804521P | 2019-02-12 | 2019-02-12 | |
| US62/804,521 | 2019-02-12 | ||
| US201962934928P | 2019-11-13 | 2019-11-13 | |
| US62/934,928 | 2019-11-13 | ||
| PCT/US2020/017987 WO2020167996A1 (en) | 2019-02-12 | 2020-02-12 | Gene therapy vectors for treatment of danon disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025007633A Division JP2025063228A (ja) | 2019-02-12 | 2025-01-20 | ダノン病の治療のための遺伝子療法ベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022520232A JP2022520232A (ja) | 2022-03-29 |
| JP2022520232A5 JP2022520232A5 (https=) | 2023-02-21 |
| JPWO2020167996A5 true JPWO2020167996A5 (https=) | 2023-02-21 |
Family
ID=72043957
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546891A Pending JP2022520232A (ja) | 2019-02-12 | 2020-02-12 | ダノン病の治療のための遺伝子療法ベクター |
| JP2025007633A Pending JP2025063228A (ja) | 2019-02-12 | 2025-01-20 | ダノン病の治療のための遺伝子療法ベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025007633A Pending JP2025063228A (ja) | 2019-02-12 | 2025-01-20 | ダノン病の治療のための遺伝子療法ベクター |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220143215A1 (https=) |
| EP (1) | EP3924371A4 (https=) |
| JP (2) | JP2022520232A (https=) |
| KR (1) | KR20210125999A (https=) |
| CN (2) | CN113508130A (https=) |
| AU (3) | AU2020221842A1 (https=) |
| BR (1) | BR112021015751A2 (https=) |
| CA (1) | CA3128514A1 (https=) |
| IL (1) | IL285238A (https=) |
| MX (1) | MX2021009696A (https=) |
| SG (1) | SG11202107744SA (https=) |
| WO (1) | WO2020167996A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3021572A1 (en) | 2016-04-20 | 2017-10-26 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas | Compositions and methods for enhanced gene expression of pklr |
| EP3697452A4 (en) | 2017-10-16 | 2021-11-24 | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. | LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS |
| BR112020020841A2 (pt) | 2018-04-11 | 2021-01-19 | Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus | Composições e métodos para transplante de célulastronco |
| CA3096293A1 (en) | 2018-04-27 | 2019-10-31 | Rocket Pharmaceuticals, Ltd. | Gene therapy for cns degeneration |
| WO2020014523A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| JP2023538519A (ja) | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| CA3201247A1 (en) * | 2020-12-07 | 2022-06-16 | Gaurav Shah | Treatment of danon disease |
| CN114874990A (zh) * | 2021-02-05 | 2022-08-09 | 中国科学院苏州纳米技术与纳米仿生研究所 | 一种功能化外泌体及其制备方法和应用 |
| JP7564368B2 (ja) | 2021-09-23 | 2024-10-08 | エルジー エナジー ソリューション リミテッド | 高耐熱性コネクター及びこれを含むバッテリーモジュール、バッテリーパック、自動車 |
| CN114457112A (zh) * | 2022-02-07 | 2022-05-10 | 苏州市立医院 | 一种特异性神经靶向mr分子探针及其制备方法和应用 |
| CN115975051B (zh) * | 2022-11-17 | 2025-09-12 | 东南大学 | 靶向外泌体及制备方法、应用、药物和药物递送系统 |
Family Cites Families (9)
| 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 |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US9434928B2 (en) * | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| AU2013221212B2 (en) * | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| DK3405215T3 (da) * | 2016-01-19 | 2022-09-19 | Univ California | Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser |
| WO2017136536A1 (en) * | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| US11713468B2 (en) * | 2017-08-02 | 2023-08-01 | Dna Twopointo Inc. | DNA vectors and elements for sustained gene expression in eukaryotic cells |
| WO2020014523A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| EP3833745A1 (en) * | 2018-08-10 | 2021-06-16 | REGENXBIO Inc. | Scalable method for recombinant aav production |
-
2020
- 2020-02-12 AU AU2020221842A patent/AU2020221842A1/en not_active Abandoned
- 2020-02-12 CA CA3128514A patent/CA3128514A1/en active Pending
- 2020-02-12 CN CN202080011825.7A patent/CN113508130A/zh active Pending
- 2020-02-12 EP EP20754942.9A patent/EP3924371A4/en active Pending
- 2020-02-12 CN CN202510460177.8A patent/CN120324641A/zh active Pending
- 2020-02-12 WO PCT/US2020/017987 patent/WO2020167996A1/en not_active Ceased
- 2020-02-12 KR KR1020217022811A patent/KR20210125999A/ko active Pending
- 2020-02-12 SG SG11202107744SA patent/SG11202107744SA/en unknown
- 2020-02-12 JP JP2021546891A patent/JP2022520232A/ja active Pending
- 2020-02-12 US US17/430,107 patent/US20220143215A1/en active Pending
- 2020-02-12 BR BR112021015751A patent/BR112021015751A2/pt unknown
- 2020-02-12 MX MX2021009696A patent/MX2021009696A/es unknown
-
2021
- 2021-07-29 IL IL285238A patent/IL285238A/en unknown
-
2023
- 2023-03-01 AU AU2023201237A patent/AU2023201237B2/en active Active
-
2025
- 2025-01-20 JP JP2025007633A patent/JP2025063228A/ja active Pending
- 2025-07-09 AU AU2025205318A patent/AU2025205318A1/en active Pending
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