JP2023534293A5 - - Google Patents
Info
- Publication number
- JP2023534293A5 JP2023534293A5 JP2023503074A JP2023503074A JP2023534293A5 JP 2023534293 A5 JP2023534293 A5 JP 2023534293A5 JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023534293 A5 JP2023534293 A5 JP 2023534293A5
- Authority
- JP
- Japan
- Prior art keywords
- human
- aav
- fmrp
- vector
- promoter
- 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 |
|---|---|---|---|
| US202063053461P | 2020-07-17 | 2020-07-17 | |
| US63/053,461 | 2020-07-17 | ||
| PCT/US2021/041975 WO2022016055A1 (en) | 2020-07-17 | 2021-07-16 | Methods and compositions for treatment of fragile x syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023534293A JP2023534293A (ja) | 2023-08-08 |
| JP2023534293A5 true JP2023534293A5 (enExample) | 2024-07-24 |
Family
ID=79554278
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023503074A Pending JP2023534293A (ja) | 2020-07-17 | 2021-07-16 | 脆弱ｘ症候群の治療のための方法及び組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230295654A1 (enExample) |
| EP (1) | EP4181877A4 (enExample) |
| JP (1) | JP2023534293A (enExample) |
| KR (1) | KR20230084465A (enExample) |
| CN (1) | CN117136077A (enExample) |
| AU (1) | AU2021308666A1 (enExample) |
| CA (1) | CA3189657A1 (enExample) |
| IL (1) | IL299889A (enExample) |
| WO (1) | WO2022016055A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023182298A1 (ja) * | 2022-03-22 | 2023-09-28 | 国立大学法人京都大学 | 脆弱ｘ症候群の治療または予防薬 |
| GB202206336D0 (en) * | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2024261220A1 (en) | 2023-06-23 | 2024-12-26 | Uniqure Biopharma B.V. | Novel fragile x constructs |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012056440A1 (en) * | 2010-10-28 | 2012-05-03 | Nanodoc Ltd. | COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA |
| EP3285788B1 (en) * | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| GB2574243A (en) * | 2018-05-31 | 2019-12-04 | The Govening Council Of The Univ Of Toronto | Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders |
| US12421524B2 (en) * | 2018-09-24 | 2025-09-23 | Merck Sharp & Dohme Llc | Expression vectors for eukaryotic expression systems |
-
2021
- 2021-07-16 CA CA3189657A patent/CA3189657A1/en active Pending
- 2021-07-16 AU AU2021308666A patent/AU2021308666A1/en active Pending
- 2021-07-16 WO PCT/US2021/041975 patent/WO2022016055A1/en not_active Ceased
- 2021-07-16 EP EP21843256.5A patent/EP4181877A4/en active Pending
- 2021-07-16 KR KR1020237005196A patent/KR20230084465A/ko active Pending
- 2021-07-16 IL IL299889A patent/IL299889A/en unknown
- 2021-07-16 JP JP2023503074A patent/JP2023534293A/ja active Pending
- 2021-07-16 US US18/005,757 patent/US20230295654A1/en active Pending
- 2021-07-16 CN CN202180059850.7A patent/CN117136077A/zh active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220331409A1 (en) | Factor ix gene therapy | |
| JP2023534293A5 (enExample) | ||
| KR102380265B1 (ko) | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 | |
| KR102298579B1 (ko) | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 | |
| JP2021106619A5 (enExample) | ||
| JP2020522269A5 (enExample) | ||
| JP2020510428A5 (enExample) | ||
| JP2022500066A5 (enExample) | ||
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| JP2020514286A5 (enExample) | ||
| JP2018537984A5 (enExample) | ||
| JP2022531809A5 (enExample) | ||
| JP2022505381A5 (enExample) | ||
| JP2025010148A (ja) | タンパク質発現の増加および／またはハプロ不全障害の治療のための方法および組成物 | |
| JP2023534037A5 (enExample) | ||
| JP2021534814A5 (enExample) | ||
| FI3953377T3 (fi) | Geeniterapioita lysosomaalisiin sairauksiin | |
| JP2020503265A5 (enExample) | ||
| JP2023524414A5 (enExample) | ||
| JP2022513318A5 (enExample) | ||
| JP2023520764A5 (enExample) | ||
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| JP2023509443A5 (enExample) | ||
| JP2025090820A5 (enExample) | ||
| JP2020510433A5 (enExample) |