JP2024537167A5 - - Google Patents
Info
- Publication number
- JP2024537167A5 JP2024537167A5 JP2024520851A JP2024520851A JP2024537167A5 JP 2024537167 A5 JP2024537167 A5 JP 2024537167A5 JP 2024520851 A JP2024520851 A JP 2024520851A JP 2024520851 A JP2024520851 A JP 2024520851A JP 2024537167 A5 JP2024537167 A5 JP 2024537167A5
- Authority
- JP
- Japan
- Prior art keywords
- mpz
- aav
- nucleic acid
- polynucleotide sequence
- combination pharmaceutical
- 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 |
|---|---|---|---|
| US202163253357P | 2021-10-07 | 2021-10-07 | |
| US63/253,357 | 2021-10-07 | ||
| PCT/US2022/077726 WO2023060215A1 (en) | 2021-10-07 | 2022-10-07 | Products and methods for myelin protein zero silencing and treating cmt1b disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024537167A JP2024537167A (ja) | 2024-10-10 |
| JP2024537167A5 true JP2024537167A5 (enExample) | 2025-10-14 |
Family
ID=83995553
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024520851A Pending JP2024537167A (ja) | 2021-10-07 | 2022-10-07 | ミエリンタンパク質ゼロサイレンシング及びｃｍｔ１ｂ疾患を治療するための生成物及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20240401046A1 (enExample) |
| EP (1) | EP4413131A1 (enExample) |
| JP (1) | JP2024537167A (enExample) |
| AU (1) | AU2022360382A1 (enExample) |
| CA (1) | CA3234702A1 (enExample) |
| WO (1) | WO2023060215A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118291543A (zh) * | 2024-02-29 | 2024-07-05 | 神拓生物技术(杭州)有限公司 | 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| KR102066663B1 (ko) * | 2017-02-21 | 2020-01-15 | 사회복지법인 삼성생명공익재단 | 유전성 말초 신경질환 진단용 마커 및 그의 용도 |
| WO2020028533A1 (en) * | 2018-08-01 | 2020-02-06 | Yale University | Compositions and methods for identification of membrane targets for enhancement of t cell activity against cancer |
| WO2020047268A1 (en) * | 2018-08-29 | 2020-03-05 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of mutant gars protein |
| GB201907882D0 (en) | 2019-06-03 | 2019-07-17 | Cyprus Foundation For Muscular Dystrophy Res | Methods |
-
2022
- 2022-10-07 EP EP22794077.2A patent/EP4413131A1/en active Pending
- 2022-10-07 AU AU2022360382A patent/AU2022360382A1/en active Pending
- 2022-10-07 US US18/698,253 patent/US20240401046A1/en active Pending
- 2022-10-07 CA CA3234702A patent/CA3234702A1/en active Pending
- 2022-10-07 JP JP2024520851A patent/JP2024537167A/ja active Pending
- 2022-10-07 WO PCT/US2022/077726 patent/WO2023060215A1/en not_active Ceased
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