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 (enExample) | 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 (enExample) |
| EP (1) | EP3350331A4 (enExample) |
| JP (3) | JP7338970B2 (enExample) |
| KR (1) | KR102799163B1 (enExample) |
| CN (1) | CN108136049A (enExample) |
| AU (3) | AU2016323575B2 (enExample) |
| CA (1) | CA2998636A1 (enExample) |
| CO (1) | CO2018003960A2 (enExample) |
| EA (1) | EA201890543A1 (enExample) |
| HK (1) | HK1257519A1 (enExample) |
| IL (2) | IL314884A (enExample) |
| MX (2) | MX2018003234A (enExample) |
| SA (1) | SA518391113B1 (enExample) |
| WO (1) | WO2017049031A1 (enExample) |
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 |
| JP7153562B2 (ja) | 2016-04-15 | 2022-10-14 | 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マイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| WO2018191666A1 (en) * | 2017-04-14 | 2018-10-18 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells |
| 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 |
| CN111433367A (zh) | 2017-10-20 | 2020-07-17 | 全国儿童医院研究所 | Nt-3基因疗法的方法和材料 |
| EP4591888A3 (en) | 2018-06-18 | 2026-01-21 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| CA3104471A1 (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 |
| CN114828858A (zh) * | 2019-10-25 | 2022-07-29 | 奥登茨治疗公司 | 用于治疗糖原贮积症的组合物和方法 |
| WO2021087007A1 (en) * | 2019-10-28 | 2021-05-06 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| EP4192437A4 (en) * | 2020-08-07 | 2024-09-25 | 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 |
| WO1995013392A1 (en) | 1993-11-09 | 1995-05-18 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
| 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 |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | 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 |
| CA2230758A1 (en) | 1995-09-08 | 1997-03-13 | 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 |
| CN1233291A (zh) | 1996-09-06 | 1999-10-27 | 宾西法尼亚大学托管会 | 重组腺伴随病毒定向基因治疗的方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of 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 |
| AU784376B2 (en) * | 1999-12-13 | 2006-03-23 | Lexicon Pharmaceuticals, Inc. | Novel human transferase proteins and polynucleotides encoding the same |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | ザ・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 | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| 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 |
| 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 |
| 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 |
| WO2013123503A1 (en) | 2012-02-17 | 2013-08-22 | 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 KR KR1020187010628A patent/KR102799163B1/ko active Active
- 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 JP JP2018513504A patent/JP7338970B2/ja active Active
- 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 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 AU AU2016323575A patent/AU2016323575B2/en active Active
- 2016-09-16 HK HK18116463.4A patent/HK1257519A1/zh unknown
-
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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