JP2019500034A5 - - Google Patents
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- Publication number
- JP2019500034A5 JP2019500034A5 JP2018531240A JP2018531240A JP2019500034A5 JP 2019500034 A5 JP2019500034 A5 JP 2019500034A5 JP 2018531240 A JP2018531240 A JP 2018531240A JP 2018531240 A JP2018531240 A JP 2018531240A JP 2019500034 A5 JP2019500034 A5 JP 2019500034A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- optionally
- raav
- gnptab
- pharmaceutical composition
- 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
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021210071A JP7878799B2 (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267502P | 2015-12-15 | 2015-12-15 | |
| US62/267,502 | 2015-12-15 | ||
| PCT/US2016/066611 WO2017106313A1 (en) | 2015-12-15 | 2016-12-14 | Adeno-associated viral vectors for treating mucolipidosis type ii |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021210071A Division JP7878799B2 (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500034A JP2019500034A (ja) | 2019-01-10 |
| JP2019500034A5 true JP2019500034A5 (enExample) | 2020-01-30 |
| JP7347932B2 JP7347932B2 (ja) | 2023-09-20 |
Family
ID=57737987
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531240A Active JP7347932B2 (ja) | 2015-12-15 | 2016-12-14 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2021210071A Active JP7878799B2 (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A Pending JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021210071A Active JP7878799B2 (ja) | 2015-12-15 | 2021-12-24 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
| JP2024019140A Pending JP2024056832A (ja) | 2015-12-15 | 2024-02-13 | ムコリピドーシスｉｉ型を治療するためのアデノ随伴ウイルスベクター |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200261598A1 (enExample) |
| EP (1) | EP3390644B1 (enExample) |
| JP (3) | JP7347932B2 (enExample) |
| KR (1) | KR102833376B1 (enExample) |
| CN (1) | CN108603199A (enExample) |
| AU (2) | AU2016372035B2 (enExample) |
| CA (1) | CA3008268A1 (enExample) |
| ES (1) | ES2991277T3 (enExample) |
| IL (1) | IL259964B (enExample) |
| MX (1) | MX2018007330A (enExample) |
| RU (1) | RU2742612C2 (enExample) |
| SG (2) | SG10201912935WA (enExample) |
| WO (1) | WO2017106313A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2020118342A (ru) | 2017-11-07 | 2021-12-08 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены aga и экспрессионные кластеры и их применение |
| MX2021005997A (es) * | 2018-11-21 | 2021-08-11 | Vectores virales recombinantes y acidos nucleicos para producirlos. | |
| JP7781057B2 (ja) * | 2019-10-18 | 2025-12-05 | 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 | Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法 |
| FI4103214T3 (fi) * | 2020-02-13 | 2026-07-03 | Tenaya Therapeutics Inc | Geeniterapian vektorit sydänsairauden hoitoon |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) * | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| HK1039565B (zh) * | 1998-04-27 | 2005-10-21 | Peter M.C. Wong | 治疗内毒素血症的方法 |
| US6534300B1 (en) * | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| 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 |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| EP1497412A4 (en) * | 2002-04-30 | 2006-11-22 | Avior Therapeutics Inc | ADENOVIRUS VECTORS USED IN IMMUNOTHERAPY |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| JP4559429B2 (ja) | 2003-05-21 | 2010-10-06 | ジェンザイム・コーポレーション | 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法 |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| PL2158322T3 (pl) * | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| JP5956331B2 (ja) | 2009-06-16 | 2016-07-27 | ジェンザイム・コーポレーション | 組換えａａｖベクターの改良された精製方法 |
| CA2803166C (en) * | 2010-06-25 | 2023-03-07 | Shire Human Genetic Therapies, Inc. | Cns delivery of therapeutic agents |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CN103502458B (zh) | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| JP2016517278A (ja) * | 2013-03-15 | 2016-06-16 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法 |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
-
2016
- 2016-12-14 SG SG10201912935WA patent/SG10201912935WA/en unknown
- 2016-12-14 ES ES16822578T patent/ES2991277T3/es active Active
- 2016-12-14 RU RU2018124448A patent/RU2742612C2/ru active
- 2016-12-14 CA CA3008268A patent/CA3008268A1/en active Pending
- 2016-12-14 JP JP2018531240A patent/JP7347932B2/ja active Active
- 2016-12-14 WO PCT/US2016/066611 patent/WO2017106313A1/en not_active Ceased
- 2016-12-14 AU AU2016372035A patent/AU2016372035B2/en active Active
- 2016-12-14 MX MX2018007330A patent/MX2018007330A/es unknown
- 2016-12-14 US US16/061,579 patent/US20200261598A1/en active Pending
- 2016-12-14 IL IL259964A patent/IL259964B/en unknown
- 2016-12-14 KR KR1020187019970A patent/KR102833376B1/ko active Active
- 2016-12-14 CN CN201680081705.8A patent/CN108603199A/zh active Pending
- 2016-12-14 EP EP16822578.7A patent/EP3390644B1/en active Active
- 2016-12-14 SG SG11201804994QA patent/SG11201804994QA/en unknown
-
2021
- 2021-12-24 JP JP2021210071A patent/JP7878799B2/ja active Active
-
2023
- 2023-06-28 AU AU2023204116A patent/AU2023204116B2/en active Active
-
2024
- 2024-02-13 JP JP2024019140A patent/JP2024056832A/ja active Pending
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