JPWO2023086928A5 - - Google Patents
Info
- Publication number
- JPWO2023086928A5 JPWO2023086928A5 JP2024528581A JP2024528581A JPWO2023086928A5 JP WO2023086928 A5 JPWO2023086928 A5 JP WO2023086928A5 JP 2024528581 A JP2024528581 A JP 2024528581A JP 2024528581 A JP2024528581 A JP 2024528581A JP WO2023086928 A5 JPWO2023086928 A5 JP WO2023086928A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- seq
- hsgsh
- raav
- mirna target
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163278775P | 2021-11-12 | 2021-11-12 | |
| US63/278,775 | 2021-11-12 | ||
| US202163288293P | 2021-12-10 | 2021-12-10 | |
| US63/288,293 | 2021-12-10 | ||
| PCT/US2022/079701 WO2023086928A2 (en) | 2021-11-12 | 2022-11-11 | Gene therapy for treatment of mucopolysaccharidosis iiia |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024546578A JP2024546578A (ja) | 2024-12-26 |
| JPWO2023086928A5 true JPWO2023086928A5 (https=) | 2025-11-18 |
| JP2024546578A5 JP2024546578A5 (https=) | 2025-11-18 |
Family
ID=86336670
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024528581A Pending JP2024546578A (ja) | 2021-11-12 | 2022-11-11 | ムコ多糖症ｉｉｉａ型の治療のための遺伝子療法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20250041445A1 (https=) |
| EP (1) | EP4430201A4 (https=) |
| JP (1) | JP2024546578A (https=) |
| KR (1) | KR20240133693A (https=) |
| AU (1) | AU2022388800A1 (https=) |
| CA (1) | CA3237987A1 (https=) |
| CO (1) | CO2024007283A2 (https=) |
| IL (1) | IL312676A (https=) |
| MX (1) | MX2024005735A (https=) |
| WO (1) | WO2023086928A2 (https=) |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2019013528A (es) * | 2017-05-12 | 2020-07-14 | The Children´S Hospital Of Philadelphia | Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). |
| KR102823833B1 (ko) * | 2017-11-30 | 2025-06-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 뮤코다당류증 iiia형에 대한 유전자 요법 |
| AR116624A1 (es) * | 2018-10-10 | 2021-05-26 | Amicus Therapeutics Inc | Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso |
| US12454701B2 (en) * | 2018-12-05 | 2025-10-28 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN114127275A (zh) * | 2019-04-30 | 2022-03-01 | 宾夕法尼亚州大学信托人 | 可用于治疗庞贝病的组合物 |
-
2022
- 2022-11-11 EP EP22893867.6A patent/EP4430201A4/en active Pending
- 2022-11-11 WO PCT/US2022/079701 patent/WO2023086928A2/en not_active Ceased
- 2022-11-11 JP JP2024528581A patent/JP2024546578A/ja active Pending
- 2022-11-11 AU AU2022388800A patent/AU2022388800A1/en active Pending
- 2022-11-11 US US18/709,415 patent/US20250041445A1/en active Pending
- 2022-11-11 IL IL312676A patent/IL312676A/en unknown
- 2022-11-11 CA CA3237987A patent/CA3237987A1/en active Pending
- 2022-11-11 KR KR1020247019262A patent/KR20240133693A/ko active Pending
-
2024
- 2024-05-09 MX MX2024005735A patent/MX2024005735A/es unknown
- 2024-06-11 CO CONC2024/0007283A patent/CO2024007283A2/es unknown
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