JP2022553307A5 - - Google Patents
Info
- Publication number
- JP2022553307A5 JP2022553307A5 JP2022523408A JP2022523408A JP2022553307A5 JP 2022553307 A5 JP2022553307 A5 JP 2022553307A5 JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022553307 A5 JP2022553307 A5 JP 2022553307A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- seq
- functionally linked
- nucleic acid
- polynucleotide
- 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 |
|---|---|---|---|
| US201962924340P | 2019-10-22 | 2019-10-22 | |
| US62/924,340 | 2019-10-22 | ||
| PCT/US2020/056860 WO2021081201A1 (en) | 2019-10-22 | 2020-10-22 | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022553307A JP2022553307A (ja) | 2022-12-22 |
| JP2022553307A5 true JP2022553307A5 (enExample) | 2023-10-30 |
Family
ID=75620215
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523408A Pending JP2022553307A (ja) | 2019-10-22 | 2020-10-22 | プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス(aav)システム |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20210147872A1 (enExample) |
| EP (1) | EP4048799A4 (enExample) |
| JP (1) | JP2022553307A (enExample) |
| KR (1) | KR20230019402A (enExample) |
| CN (1) | CN115715327A (enExample) |
| AU (1) | AU2020371662A1 (enExample) |
| CA (1) | CA3158516A1 (enExample) |
| IL (1) | IL292382A (enExample) |
| MX (1) | MX2022004812A (enExample) |
| WO (1) | WO2021081201A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210052450A (ko) * | 2018-07-31 | 2021-05-10 | 코넬 유니버시티 | 기관 기능을 제어하기 위한 유전자 요법 방법 |
| IL303239A (en) * | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| WO2024035649A1 (en) * | 2022-08-08 | 2024-02-15 | Shape Therapeutics Inc. | Compositions for progranulin expression and methods of use thereof |
| AU2023378899A1 (en) * | 2022-11-13 | 2025-06-26 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009089635A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| HRP20250897T1 (hr) * | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| WO2016125330A1 (ja) * | 2015-02-02 | 2016-08-11 | 原 英彰 | 網膜再生促進薬 |
| US20190328906A1 (en) * | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| US20190388508A1 (en) * | 2017-01-19 | 2019-12-26 | New York University | Progranulin and progranulin derivatives in treating impaired fracture healing |
| 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 |
| WO2020160458A1 (en) * | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| BR112021015817A2 (pt) * | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
-
2020
- 2020-10-22 MX MX2022004812A patent/MX2022004812A/es unknown
- 2020-10-22 IL IL292382A patent/IL292382A/en unknown
- 2020-10-22 CN CN202080089386.1A patent/CN115715327A/zh active Pending
- 2020-10-22 EP EP20880235.5A patent/EP4048799A4/en active Pending
- 2020-10-22 WO PCT/US2020/056860 patent/WO2021081201A1/en not_active Ceased
- 2020-10-22 JP JP2022523408A patent/JP2022553307A/ja active Pending
- 2020-10-22 KR KR1020227017064A patent/KR20230019402A/ko not_active Ceased
- 2020-10-22 US US17/077,680 patent/US20210147872A1/en not_active Abandoned
- 2020-10-22 CA CA3158516A patent/CA3158516A1/en active Pending
- 2020-10-22 AU AU2020371662A patent/AU2020371662A1/en not_active Abandoned
-
2023
- 2023-09-26 US US18/372,868 patent/US20240318199A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022553307A5 (enExample) | ||
| JP2022549380A5 (enExample) | ||
| US12239692B2 (en) | Adeno-associated virus factor VIII vectors | |
| JP2021106619A5 (enExample) | ||
| JP2021087431A5 (enExample) | ||
| JP2021118724A5 (enExample) | ||
| AU2017301600B2 (en) | Novel adeno-associated virus capsid proteins | |
| JP2022060228A5 (enExample) | ||
| EP3436051B1 (en) | Column-based fully scalable raav manufacturing process | |
| CA3216419A1 (en) | Tissue-targeted modified aav capsids and methods of use thereof | |
| JP2018510648A5 (enExample) | ||
| JP7853952B2 (ja) | Glut1発現のためのアデノ随伴ウイルスベクターおよびその使用 | |
| JP2022542828A5 (enExample) | ||
| JP2022504740A5 (enExample) | ||
| WO2001083797A2 (en) | Polynucleotides for use in recombinant adeno-associated virus virion production | |
| JP2022520803A5 (enExample) | ||
| US20240318199A1 (en) | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders | |
| JP2024543918A5 (enExample) | ||
| JP2022551744A5 (enExample) | ||
| JP2023537980A5 (enExample) | ||
| IL292264B2 (en) | aav transfer cassette | |
| JP2023549124A5 (enExample) | ||
| CA3228365A1 (en) | Treatment of muscular dystrophy | |
| JP2022518931A5 (enExample) | ||
| Youjin et al. | The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy |