JPWO2022125489A5 - - Google Patents
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- JPWO2022125489A5 JPWO2022125489A5 JP2023534292A JP2023534292A JPWO2022125489A5 JP WO2022125489 A5 JPWO2022125489 A5 JP WO2022125489A5 JP 2023534292 A JP2023534292 A JP 2023534292A JP 2023534292 A JP2023534292 A JP 2023534292A JP WO2022125489 A5 JPWO2022125489 A5 JP WO2022125489A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition
- seq
- unit dose
- effective amount
- 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
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063122249P | 2020-12-07 | 2020-12-07 | |
| US63/122,249 | 2020-12-07 | ||
| PCT/US2021/062112 WO2022125489A1 (en) | 2020-12-07 | 2021-12-07 | Treatment of danon disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023552443A JP2023552443A (ja) | 2023-12-15 |
| JP2023552443A5 JP2023552443A5 (https=) | 2024-11-19 |
| JPWO2022125489A5 true JPWO2022125489A5 (https=) | 2024-11-19 |
Family
ID=81973960
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023534292A Pending JP2023552443A (ja) | 2020-12-07 | 2021-12-07 | ダノン病の治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240033325A1 (https=) |
| EP (1) | EP4255458A4 (https=) |
| JP (1) | JP2023552443A (https=) |
| KR (1) | KR20230129431A (https=) |
| CN (1) | CN116887868A (https=) |
| AU (1) | AU2021396150A1 (https=) |
| CA (1) | CA3201247A1 (https=) |
| CL (1) | CL2023001650A1 (https=) |
| IL (1) | IL303428A (https=) |
| MX (1) | MX2023006694A (https=) |
| WO (1) | WO2022125489A1 (https=) |
Families Citing this family (6)
| 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 |
| 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 |
| WO2025129071A1 (en) * | 2023-12-14 | 2025-06-19 | Sonothera, Inc. | Methods and systems for improved nucleic acid delivery via ultrasound |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20170049887A1 (en) * | 2014-04-25 | 2017-02-23 | University Of Florida Research Foundation, Inc. | Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus |
| DK3405215T3 (da) * | 2016-01-19 | 2022-09-19 | Univ California | Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser |
| CA3049915A1 (en) * | 2017-01-31 | 2018-08-09 | Stephen YOO | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| WO2020014523A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| AU2020221842A1 (en) * | 2019-02-12 | 2021-08-12 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of Danon Disease |
-
2021
- 2021-12-07 CA CA3201247A patent/CA3201247A1/en active Pending
- 2021-12-07 WO PCT/US2021/062112 patent/WO2022125489A1/en not_active Ceased
- 2021-12-07 JP JP2023534292A patent/JP2023552443A/ja active Pending
- 2021-12-07 AU AU2021396150A patent/AU2021396150A1/en active Pending
- 2021-12-07 CN CN202180092975.XA patent/CN116887868A/zh active Pending
- 2021-12-07 IL IL303428A patent/IL303428A/en unknown
- 2021-12-07 EP EP21904214.0A patent/EP4255458A4/en active Pending
- 2021-12-07 KR KR1020237023029A patent/KR20230129431A/ko active Pending
- 2021-12-07 US US18/265,421 patent/US20240033325A1/en active Pending
- 2021-12-07 MX MX2023006694A patent/MX2023006694A/es unknown
-
2023
- 2023-06-07 CL CL2023001650A patent/CL2023001650A1/es unknown
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