JP2025501768A5 - - Google Patents
Info
- Publication number
- JP2025501768A5 JP2025501768A5 JP2024538396A JP2024538396A JP2025501768A5 JP 2025501768 A5 JP2025501768 A5 JP 2025501768A5 JP 2024538396 A JP2024538396 A JP 2024538396A JP 2024538396 A JP2024538396 A JP 2024538396A JP 2025501768 A5 JP2025501768 A5 JP 2025501768A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- promoter
- mature
- aav
- raav
- 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 |
|---|---|---|---|
| US202163293680P | 2021-12-24 | 2021-12-24 | |
| US63/293,680 | 2021-12-24 | ||
| PCT/US2022/082383 WO2023122803A1 (en) | 2021-12-24 | 2022-12-24 | Gene therapy for lamin a - associated deficiencies |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025501768A JP2025501768A (ja) | 2025-01-23 |
| JP2025501768A5 true JP2025501768A5 (cg-RX-API-DMAC10.html) | 2026-01-07 |
Family
ID=86903820
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024538396A Pending JP2025501768A (ja) | 2021-12-24 | 2022-12-24 | ラミンａ関連欠損症に対する遺伝子療法 |
Country Status (11)
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| WO2025075551A1 (en) * | 2023-10-06 | 2025-04-10 | National University Of Singapore | Method of treating lmna dilated cardiomyopathy |
| KR20260114566A (ko) | 2023-11-14 | 2026-07-22 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7291699B2 (en) * | 2003-03-18 | 2007-11-06 | The Regents Of The University Of Colorado | Product and methods for diagnosis and therapy for cardiac and skeletal muscle disorders |
| WO2013071047A1 (en) * | 2011-11-11 | 2013-05-16 | Children's Medical Center Corporation | Compositions and methods for in vitro transcription of rna |
| US12404522B2 (en) * | 2018-01-19 | 2025-09-02 | Agency For Science, Technology And Research | Disrupting the LINC complex for treating laminopathy |
| WO2020176896A1 (en) * | 2019-02-28 | 2020-09-03 | Encoded Therapeutics, Inc. | Compositions and methods for treating laminopathies |
| EP4198053A1 (en) * | 2021-12-15 | 2023-06-21 | Universitätsklinikum Hamburg-Eppendorf | Lmna gene expression for treatment of laminopathies |
-
2022
- 2022-12-24 US US18/723,236 patent/US20250049949A1/en active Pending
- 2022-12-24 AU AU2022419657A patent/AU2022419657A1/en active Pending
- 2022-12-24 CN CN202280085172.6A patent/CN118434877A/zh active Pending
- 2022-12-24 EP EP22912777.4A patent/EP4453226A4/en active Pending
- 2022-12-24 WO PCT/US2022/082383 patent/WO2023122803A1/en not_active Ceased
- 2022-12-24 MX MX2024007903A patent/MX2024007903A/es unknown
- 2022-12-24 IL IL313734A patent/IL313734A/en unknown
- 2022-12-24 CA CA3241202A patent/CA3241202A1/en active Pending
- 2022-12-24 KR KR1020247022099A patent/KR20240118821A/ko active Pending
- 2022-12-24 JP JP2024538396A patent/JP2025501768A/ja active Pending
-
2024
- 2024-06-24 CO CONC2024/0008188A patent/CO2024008188A2/es unknown