JP2022522166A5 - - Google Patents
Info
- Publication number
- JP2022522166A5 JP2022522166A5 JP2021549908A JP2021549908A JP2022522166A5 JP 2022522166 A5 JP2022522166 A5 JP 2022522166A5 JP 2021549908 A JP2021549908 A JP 2021549908A JP 2021549908 A JP2021549908 A JP 2021549908A JP 2022522166 A5 JP2022522166 A5 JP 2022522166A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- effector
- seq
- sequence described
- 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.)
- Granted
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 true JP2022522166A5 (enExample) | 2023-03-03 |
| JP7873074B2 JP7873074B2 (ja) | 2026-06-11 |
Family
ID=72238736
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549908A Active JP7873074B2 (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 (12)
| Country | Link |
|---|---|
| US (1) | US20220098614A1 (enExample) |
| EP (1) | EP3931317A4 (enExample) |
| JP (1) | JP7873074B2 (enExample) |
| KR (1) | KR102959609B1 (enExample) |
| CN (2) | CN113631706A (enExample) |
| AU (1) | AU2020229886B2 (enExample) |
| BR (1) | BR112021017054A2 (enExample) |
| CA (1) | CA3130221A1 (enExample) |
| IL (1) | IL285841A (enExample) |
| MX (1) | MX2021010412A (enExample) |
| SG (1) | SG11202108469XA (enExample) |
| WO (1) | WO2020172720A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3866859A4 (en) * | 2018-10-17 | 2023-08-09 | Benitec IP Holdings Inc. | METHODS OF TREATMENT FOR OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) |
| EP4493703A4 (en) * | 2022-03-18 | 2026-08-05 | Univ Florida | METHODS AND COMPOSITIONS FOR TREATING TMEM43-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
| 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 |
| JPH11514853A (ja) | 1995-09-08 | 1999-12-21 | ジエンザイム コーポレイション | 遺伝子治療のための改良されたａａｖベクター |
| US7208315B2 (en) | 2000-01-05 | 2007-04-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for efficient AAV vector production |
| EP1390490B1 (en) | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| 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 |
| EP1572893B1 (en) | 2001-11-09 | 2009-01-07 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of 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 |
| US20060166363A1 (en) * | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | 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 |
| PT3272872T (pt) | 2005-10-20 | 2020-06-26 | Uniqure Ip Bv | Vetores aav melhorados produzidos em células de insetos |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| EP2242840B1 (en) | 2008-01-29 | 2019-07-24 | Applied Genetic Technologies Corporation | Recombinant adeno-associated virus production using bhk cells in suspension |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| US11078464B2 (en) | 2013-08-30 | 2021-08-03 | 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 |
| CA3012699C (en) | 2016-04-21 | 2023-11-21 | Virovek, Inc | Aav production in insect cells, methods and compositions therefor |
| KR102495222B1 (ko) * | 2016-12-14 | 2023-02-09 | 베니텍 아이피 홀딩스 아이엔씨. | 안구인두근위축증 (opmd)의 치료제 및 이의 사용 |
| EP3575398A4 (en) | 2017-01-30 | 2020-11-11 | Nippon Medical School Foundation | ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT |
| UY37863A (es) | 2017-08-31 | 2019-03-29 | Benitec Biopharma Ltd | Virus adenoasociado (aav) con dominio de fosfolipasa modificado |
-
2020
- 2020-02-28 CA CA3130221A patent/CA3130221A1/en active Pending
- 2020-02-28 JP JP2021549908A patent/JP7873074B2/ja active Active
- 2020-02-28 EP EP20762817.3A patent/EP3931317A4/en active Pending
- 2020-02-28 US US17/434,666 patent/US20220098614A1/en active Pending
- 2020-02-28 WO PCT/AU2020/050182 patent/WO2020172720A1/en not_active Ceased
- 2020-02-28 BR BR112021017054A patent/BR112021017054A2/pt not_active Application Discontinuation
- 2020-02-28 CN CN202080017400.7A patent/CN113631706A/zh active Pending
- 2020-02-28 CN CN202511142034.9A patent/CN121160804A/zh active Pending
- 2020-02-28 AU AU2020229886A patent/AU2020229886B2/en active Active
- 2020-02-28 MX MX2021010412A patent/MX2021010412A/es unknown
- 2020-02-28 SG SG11202108469XA patent/SG11202108469XA/en unknown
- 2020-02-28 KR KR1020217031046A patent/KR102959609B1/ko active Active
-
2021
- 2021-08-24 IL IL285841A patent/IL285841A/en unknown
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