JPWO2020172720A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020172720A5 JPWO2020172720A5 JP2021549908A JP2021549908A JPWO2020172720A5 JP WO2020172720 A5 JPWO2020172720 A5 JP WO2020172720A5 JP 2021549908 A JP2021549908 A JP 2021549908A JP 2021549908 A JP2021549908 A JP 2021549908A JP WO2020172720 A5 JPWO2020172720 A5 JP WO2020172720A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- set forth
- sequence set
- effector
- shmir
- 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 |
|---|---|---|---|
| US201962812187P | 2019-02-28 | 2019-02-28 | |
| US62/812,187 | 2019-02-28 | ||
| PCT/AU2020/050182 WO2020172720A1 (en) | 2019-02-28 | 2020-02-28 | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022522166A JP2022522166A (ja) | 2022-04-14 |
| JP2022522166A5 JP2022522166A5 (https=) | 2023-03-03 |
| JPWO2020172720A5 true JPWO2020172720A5 (https=) | 2023-03-03 |
Family
ID=72238736
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549908A Pending JP2022522166A (ja) | 2019-02-28 | 2020-02-28 | 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための組成物及び方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220098614A1 (https=) |
| EP (1) | EP3931317A4 (https=) |
| JP (1) | JP2022522166A (https=) |
| CN (2) | CN121160804A (https=) |
| AU (1) | AU2020229886B2 (https=) |
| BR (1) | BR112021017054A2 (https=) |
| CA (1) | CA3130221A1 (https=) |
| IL (1) | IL285841A (https=) |
| MX (1) | MX2021010412A (https=) |
| SG (1) | SG11202108469XA (https=) |
| WO (1) | WO2020172720A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN112867511A (zh) * | 2018-10-17 | 2021-05-28 | 贝尼泰克生物制药有限公司 | 用于治疗眼咽肌营养不良(opmd)的方法 |
| KR20260030147A (ko) * | 2023-06-28 | 2026-03-05 | 베니텍 아이피 홀딩스 아이엔씨. | 인두 근육에 치료 조성물을 투여하기 위한 장치 및 방법 |
| WO2026011009A1 (en) * | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US7208315B2 (en) | 2000-01-05 | 2007-04-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for efficient AAV vector production |
| EP2017338A1 (en) | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| 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 |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| WO2005072364A2 (en) * | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
| US8008468B2 (en) | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| EP2311967B1 (en) | 2005-10-20 | 2017-09-20 | UniQure IP B.V. | Improved AAV vectors produced in insect cells |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| KR101589259B1 (ko) | 2006-06-21 | 2016-02-01 | 유니큐어 아이피 비.브이. | 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터 |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| EP3024497B1 (en) * | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3039129B1 (en) | 2013-08-30 | 2018-06-27 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
| CN104815338A (zh) * | 2015-05-01 | 2015-08-05 | 许瑞安 | 一种携带kal基因重组腺相关病毒载体的基因治疗药物 |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| EP3555294A4 (en) * | 2016-12-14 | 2020-08-26 | Benitec Biopharma Limited | REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE |
| WO2018139634A1 (ja) * | 2017-01-30 | 2018-08-02 | 学校法人日本医科大学 | アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体 |
| UY37863A (es) | 2017-08-31 | 2019-03-29 | Benitec Biopharma Ltd | Virus adenoasociado (aav) con dominio de fosfolipasa modificado |
-
2020
- 2020-02-28 US US17/434,666 patent/US20220098614A1/en active Pending
- 2020-02-28 CN CN202511142034.9A patent/CN121160804A/zh active Pending
- 2020-02-28 WO PCT/AU2020/050182 patent/WO2020172720A1/en not_active Ceased
- 2020-02-28 JP JP2021549908A patent/JP2022522166A/ja active Pending
- 2020-02-28 BR BR112021017054A patent/BR112021017054A2/pt unknown
- 2020-02-28 SG SG11202108469XA patent/SG11202108469XA/en unknown
- 2020-02-28 EP EP20762817.3A patent/EP3931317A4/en active Pending
- 2020-02-28 CA CA3130221A patent/CA3130221A1/en active Pending
- 2020-02-28 AU AU2020229886A patent/AU2020229886B2/en active Active
- 2020-02-28 MX MX2021010412A patent/MX2021010412A/es unknown
- 2020-02-28 CN CN202080017400.7A patent/CN113631706A/zh active Pending
-
2021
- 2021-08-24 IL IL285841A patent/IL285841A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022500066A5 (https=) | ||
| JP2021522842A5 (https=) | ||
| JP2024095717A5 (https=) | ||
| IL273261B2 (en) | Adeno-associated virus variant capsids and methods of using them | |
| JP2021502060A5 (https=) | ||
| RU2019111648A (ru) | Средство лечения болезни гентингтона на основе aav | |
| JP2019519219A5 (https=) | ||
| JPWO2020069461A5 (https=) | ||
| JP2017536116A5 (https=) | ||
| JPWO2021113270A5 (https=) | ||
| JPWO2019193119A5 (https=) | ||
| JP2022066336A5 (https=) | ||
| JP2019515663A5 (https=) | ||
| JPWO2020223232A5 (https=) | ||
| JPWO2020223236A5 (https=) | ||
| CN119137273A (zh) | 包括工程化核内小RNA（snRNA）的组合物和方法 | |
| JP2021525531A5 (https=) | ||
| JP2020535804A5 (https=) | ||
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| JPWO2022053630A5 (https=) | ||
| JP2024119830A5 (https=) | ||
| JP2020503014A5 (https=) | ||
| JP2020527167A5 (https=) | ||
| JP2021534794A5 (https=) | ||
| JPWO2020172720A5 (https=) |