JP2023537903A5 - - Google Patents
Info
- Publication number
- JP2023537903A5 JP2023537903A5 JP2023507760A JP2023507760A JP2023537903A5 JP 2023537903 A5 JP2023537903 A5 JP 2023537903A5 JP 2023507760 A JP2023507760 A JP 2023507760A JP 2023507760 A JP2023507760 A JP 2023507760A JP 2023537903 A5 JP2023537903 A5 JP 2023537903A5
- Authority
- JP
- Japan
- Prior art keywords
- approximately
- itr
- raav
- adeno
- 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.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063063851P | 2020-08-10 | 2020-08-10 | |
| US63/063,851 | 2020-08-10 | ||
| PCT/US2021/045447 WO2022035903A2 (en) | 2020-08-10 | 2021-08-10 | Gene therapies for lysosomal disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023537903A JP2023537903A (ja) | 2023-09-06 |
| JP2023537903A5 true JP2023537903A5 (https=) | 2024-08-20 |
Family
ID=77864621
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023507760A Pending JP2023537903A (ja) | 2020-08-10 | 2021-08-10 | リソソーム障害に対する遺伝子療法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230310654A1 (https=) |
| EP (1) | EP4192516A2 (https=) |
| JP (1) | JP2023537903A (https=) |
| KR (1) | KR20230051529A (https=) |
| CN (1) | CN116157527A (https=) |
| AU (1) | AU2021324686A1 (https=) |
| CA (1) | CA3190866A1 (https=) |
| IL (1) | IL300407A (https=) |
| MX (1) | MX2023001562A (https=) |
| WO (1) | WO2022035903A2 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20250205365A1 (en) * | 2022-03-18 | 2025-06-26 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector |
| WO2024229161A1 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| US20250152747A1 (en) * | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating gaucher disease and gba-parkinson’s disease |
| WO2025229340A1 (en) | 2024-05-01 | 2025-11-06 | Spur Therapeutics Limited | Methods and compositions for treatment of a synucleinopathy and/or a disease associated with a gcase deficiency |
| KR20260021150A (ko) | 2024-08-05 | 2026-02-13 | 충북대학교 산학협력단 | Gba1 돌연변이 보인자의 dj-1 발현수준에 따른 파킨슨병의 위험도 예측 방법 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008024998A2 (en) | 2006-08-24 | 2008-02-28 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| EP3204503A1 (en) * | 2014-10-06 | 2017-08-16 | Arthrogen BV | Aav-based gene therapy |
| HK1256341A1 (zh) * | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| JP7496667B2 (ja) | 2016-04-21 | 2024-06-07 | ビロベク，インコーポレイテッド | 昆虫細胞中でのａａｖ生成、方法およびその組成物 |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| AU2018346104B2 (en) * | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| JP7664848B2 (ja) * | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
-
2021
- 2021-08-10 JP JP2023507760A patent/JP2023537903A/ja active Pending
- 2021-08-10 WO PCT/US2021/045447 patent/WO2022035903A2/en not_active Ceased
- 2021-08-10 AU AU2021324686A patent/AU2021324686A1/en active Pending
- 2021-08-10 CA CA3190866A patent/CA3190866A1/en active Pending
- 2021-08-10 MX MX2023001562A patent/MX2023001562A/es unknown
- 2021-08-10 IL IL300407A patent/IL300407A/en unknown
- 2021-08-10 CN CN202180062323.1A patent/CN116157527A/zh active Pending
- 2021-08-10 US US18/020,575 patent/US20230310654A1/en active Pending
- 2021-08-10 KR KR1020237008509A patent/KR20230051529A/ko active Pending
- 2021-08-10 EP EP21773671.9A patent/EP4192516A2/en active Pending
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