JP2018527365A5 - - Google Patents
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- JP2018527365A5 JP2018527365A5 JP2018513504A JP2018513504A JP2018527365A5 JP 2018527365 A5 JP2018527365 A5 JP 2018527365A5 JP 2018513504 A JP2018513504 A JP 2018513504A JP 2018513504 A JP2018513504 A JP 2018513504A JP 2018527365 A5 JP2018527365 A5 JP 2018527365A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- raav
- aav
- nucleotides
- administration
- 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.)
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Links
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021180893A JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562220107P | 2015-09-17 | 2015-09-17 | |
| US62/220,107 | 2015-09-17 | ||
| US201562221068P | 2015-09-20 | 2015-09-20 | |
| US62/221,068 | 2015-09-20 | ||
| US201662301260P | 2016-02-29 | 2016-02-29 | |
| US62/301,260 | 2016-02-29 | ||
| PCT/US2016/052051 WO2017049031A1 (en) | 2015-09-17 | 2016-09-16 | Methods and materials for galgt2 gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021180893A Division JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018527365A JP2018527365A (ja) | 2018-09-20 |
| JP2018527365A5 true JP2018527365A5 (https=) | 2019-08-29 |
| JP7338970B2 JP7338970B2 (ja) | 2023-09-05 |
Family
ID=58289891
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018513504A Active JP7338970B2 (ja) | 2015-09-17 | 2016-09-16 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2021180893A Pending JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A Pending JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021180893A Pending JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A Pending JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US10980897B2 (https=) |
| EP (1) | EP3350331A4 (https=) |
| JP (3) | JP7338970B2 (https=) |
| KR (1) | KR102799163B1 (https=) |
| CN (1) | CN108136049A (https=) |
| AU (3) | AU2016323575B2 (https=) |
| CA (1) | CA2998636A1 (https=) |
| CO (1) | CO2018003960A2 (https=) |
| EA (1) | EA201890543A1 (https=) |
| HK (1) | HK1257519A1 (https=) |
| IL (2) | IL258005B2 (https=) |
| MX (2) | MX2018003234A (https=) |
| SA (1) | SA518391113B1 (https=) |
| WO (1) | WO2017049031A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| ES2977730T3 (es) * | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| EP3596112A2 (en) | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| KR20190139951A (ko) * | 2017-04-14 | 2019-12-18 | 리젠엑스바이오 인크. | 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료 |
| MA50836A (fr) | 2017-10-18 | 2020-08-26 | Res Inst Nationwide Childrens Hospital | Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire |
| US11926653B2 (en) | 2017-10-20 | 2024-03-12 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3807309A1 (en) * | 2018-06-18 | 2021-04-21 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| JP2022554141A (ja) * | 2019-10-25 | 2022-12-28 | オーデンツ セラピューティクス，インコーポレーテッド | 糖原病を治療するための組成物及び方法 |
| WO2021087007A1 (en) * | 2019-10-28 | 2021-05-06 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| CA3190596A1 (en) * | 2020-08-07 | 2022-02-10 | Janssen Biotech, Inc. | Formulations for highly purified viral particles |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
Family Cites Families (23)
| 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 |
| US6692947B2 (en) * | 1999-12-13 | 2004-02-17 | Lexicon Genetics Incorporated | Human transferase proteins and polynucleotides encoding the same |
| 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 |
| EP1390490B1 (en) * | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| US20060194265A1 (en) | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| US20100267575A1 (en) * | 2006-10-17 | 2010-10-21 | Childrens Hospital Medical Center | Gene array technique for predicting response in inflammatory bowel diseases |
| US9434928B2 (en) * | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
-
2016
- 2016-09-16 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 US US15/759,474 patent/US10980897B2/en active Active
- 2016-09-16 CA CA2998636A patent/CA2998636A1/en active Pending
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
- 2016-09-16 EA EA201890543A patent/EA201890543A1/ru unknown
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 IL IL314884A patent/IL314884A/en unknown
- 2016-09-16 EP EP16847351.0A patent/EP3350331A4/en active Pending
- 2016-09-16 CN CN201680054261.9A patent/CN108136049A/zh active Pending
- 2016-09-16 HK HK18116463.4A patent/HK1257519A1/zh unknown
- 2016-09-16 AU AU2016323575A patent/AU2016323575B2/en active Active
- 2016-09-16 JP JP2018513504A patent/JP7338970B2/ja active Active
- 2016-09-16 KR KR1020187010628A patent/KR102799163B1/ko active Active
-
2018
- 2018-03-13 SA SA518391113A patent/SA518391113B1/ar unknown
- 2018-03-14 MX MX2023013509A patent/MX2023013509A/es unknown
- 2018-04-13 CO CONC2018/0003960A patent/CO2018003960A2/es unknown
-
2021
- 2021-03-16 US US17/203,232 patent/US12121595B2/en active Active
- 2021-11-05 JP JP2021180893A patent/JP2022010079A/ja active Pending
-
2023
- 2023-01-31 AU AU2023200499A patent/AU2023200499B2/en active Active
-
2024
- 2024-05-30 JP JP2024087848A patent/JP2024109934A/ja active Pending
- 2024-10-03 US US18/905,388 patent/US20250025578A1/en active Pending
-
2025
- 2025-08-06 AU AU2025213610A patent/AU2025213610A1/en active Pending
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