JP2025513785A5 - - Google Patents
Info
- Publication number
- JP2025513785A5 JP2025513785A5 JP2024559143A JP2024559143A JP2025513785A5 JP 2025513785 A5 JP2025513785 A5 JP 2025513785A5 JP 2024559143 A JP2024559143 A JP 2024559143A JP 2024559143 A JP2024559143 A JP 2024559143A JP 2025513785 A5 JP2025513785 A5 JP 2025513785A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- seq
- nucleic acid
- rnai
- identity
- 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 |
|---|---|---|---|
| US202263328241P | 2022-04-06 | 2022-04-06 | |
| US63/328,241 | 2022-04-06 | ||
| US202363483075P | 2023-02-03 | 2023-02-03 | |
| US63/483,075 | 2023-02-03 | ||
| PCT/US2023/065388 WO2023196862A1 (en) | 2022-04-06 | 2023-04-05 | Targeted gene therapy for dm-1 myotonic dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025513785A JP2025513785A (ja) | 2025-04-30 |
| JP2025513785A5 true JP2025513785A5 (https=) | 2026-04-13 |
Family
ID=86272086
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024559143A Pending JP2025513785A (ja) | 2022-04-06 | 2023-04-05 | Ｄｍ－１筋強直性ジストロフィーの標的遺伝子療法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230365968A1 (https=) |
| EP (1) | EP4504936A1 (https=) |
| JP (1) | JP2025513785A (https=) |
| KR (1) | KR20240169111A (https=) |
| CN (1) | CN119234040A (https=) |
| AU (1) | AU2023248463A1 (https=) |
| CA (1) | CA3247417A1 (https=) |
| CO (1) | CO2024013743A2 (https=) |
| IL (1) | IL316078A (https=) |
| MX (1) | MX2024012308A (https=) |
| TW (1) | TW202345914A (https=) |
| WO (1) | WO2023196862A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2025137378A1 (en) * | 2023-12-20 | 2025-06-26 | Aavantibio, Inc. | Aav capsid engineering for cardiac and/or musculoskeletal gene therapy |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP2152874A2 (en) | 2007-04-26 | 2010-02-17 | University of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| DK2164967T3 (en) | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| EP2875133B1 (en) | 2012-07-17 | 2018-01-10 | Université de Genève | Nucleic acids for down-regulation of gene expression |
| WO2018178067A1 (en) * | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| JP2020518258A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| WO2018226638A1 (en) * | 2017-06-05 | 2018-12-13 | The Brigham And Women's Hospital, Inc. | Vero cell lines stably expressing hsv icp0 protein |
| EP3765624A4 (en) | 2018-03-16 | 2022-05-25 | Research Institute at Nationwide Children's Hospital | Increasing tissue specific gene delivery by capsid modification |
| US20210363193A1 (en) | 2018-04-27 | 2021-11-25 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| BR112020023298A2 (pt) * | 2018-05-15 | 2021-03-09 | University Of Washington | composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna |
| KR102931619B1 (ko) * | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
| JP2021534755A (ja) * | 2018-08-22 | 2021-12-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| CA3164335A1 (en) * | 2020-01-10 | 2021-07-15 | Fatih Ozsolak | Viral vector for combination therapy |
-
2023
- 2023-04-05 WO PCT/US2023/065388 patent/WO2023196862A1/en not_active Ceased
- 2023-04-05 KR KR1020247036731A patent/KR20240169111A/ko active Pending
- 2023-04-05 AU AU2023248463A patent/AU2023248463A1/en active Pending
- 2023-04-05 CA CA3247417A patent/CA3247417A1/en active Pending
- 2023-04-05 JP JP2024559143A patent/JP2025513785A/ja active Pending
- 2023-04-05 CN CN202380041468.2A patent/CN119234040A/zh active Pending
- 2023-04-05 US US18/296,283 patent/US20230365968A1/en active Pending
- 2023-04-05 IL IL316078A patent/IL316078A/en unknown
- 2023-04-05 EP EP23720519.0A patent/EP4504936A1/en active Pending
- 2023-04-06 TW TW112112926A patent/TW202345914A/zh unknown
-
2024
- 2024-10-03 MX MX2024012308A patent/MX2024012308A/es unknown
- 2024-10-11 CO CONC2024/0013743A patent/CO2024013743A2/es unknown
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