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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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) | IL314884A (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 |
|---|---|---|---|---|
| EA201890543A1 (ru) | 2015-09-17 | 2018-08-31 | Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл | Способы и материалы для генной терапии galgt2 |
| AU2017250791B2 (en) * | 2016-04-15 | 2022-11-24 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| JP2020516291A (ja) * | 2017-04-14 | 2020-06-11 | レジェンクスバイオ インコーポレーテッド | ヒト神経細胞又はグリア細胞により生産された組換えヒトイズロン酸−２−スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉ型の治療 |
| JP7213238B2 (ja) | 2017-10-18 | 2023-01-26 | 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 | 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| AU2018351528B2 (en) | 2017-10-20 | 2025-02-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| CN120393053A (zh) | 2018-06-18 | 2025-08-01 | 全国儿童医院研究所 | 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送 |
| WO2019246125A1 (en) * | 2018-06-18 | 2019-12-26 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| WO2021081338A1 (en) * | 2019-10-25 | 2021-04-29 | Audentes Therapeutics, Inc. | Compositions and methods for treating glycogen storage disorders |
| EP4051324A4 (en) * | 2019-10-28 | 2023-11-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| AU2021320395A1 (en) * | 2020-08-07 | 2023-04-13 | 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 |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| 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 |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | 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 |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved 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 |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| 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 |
| CA2394976A1 (en) * | 1999-12-13 | 2001-06-14 | Lexicon Genetics Incorporated | Novel human transferase proteins and polynucleotides encoding the same |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| EP2017338A1 (en) * | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
| US20060194265A1 (en) | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| WO2008048986A2 (en) * | 2006-10-17 | 2008-04-24 | Children's 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 |
| JP2015503924A (ja) * | 2012-01-05 | 2015-02-05 | ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド | 遺伝子導入のための方法及び組成物 |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| EA201890543A1 (ru) | 2015-09-17 | 2018-08-31 | Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл | Способы и материалы для генной терапии galgt2 |
-
2016
- 2016-09-16 EA EA201890543A patent/EA201890543A1/ru unknown
- 2016-09-16 EP EP16847351.0A patent/EP3350331A4/en active Pending
- 2016-09-16 CA CA2998636A patent/CA2998636A1/en active Pending
- 2016-09-16 IL IL314884A patent/IL314884A/en unknown
- 2016-09-16 US US15/759,474 patent/US10980897B2/en active Active
- 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 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 KR KR1020187010628A patent/KR102799163B1/ko active Active
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
-
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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