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
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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.)
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Application number
JP2018531240A
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English (en)
Japanese (ja)
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JP7347932B2 (ja
JP2019500034A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066611 external-priority patent/WO2017106313A1/en
Publication of JP2019500034A publication Critical patent/JP2019500034A/ja
Publication of JP2019500034A5 publication Critical patent/JP2019500034A5/ja
Priority to JP2021210071A priority Critical patent/JP2022046635A/ja
Application granted granted Critical
Publication of JP7347932B2 publication Critical patent/JP7347932B2/ja
Priority to JP2024019140A priority patent/JP2024056832A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018531240A 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 Active JP7347932B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021210071A JP2022046635A (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 JP2022046635A (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 Pending JP2022046635A (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 Pending JP2022046635A (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)

* Cited by examiner, † Cited by third party
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
EP3883954A4 (en) * 2018-11-21 2022-08-10 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
EP4045662A1 (en) * 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
BR112022015979A2 (pt) 2020-02-13 2022-10-11 Tenaya Therapeutics Inc Vetores de terapia genética para tratamento de doenças cardíacas

Family Cites Families (20)

* Cited by examiner, † Cited by third party
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
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999055378A1 (en) * 1998-04-27 1999-11-04 Temple University - Of The Commonwealth System Of Higher Education Method of treating endotoxemia
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
CN103555677B (zh) 2001-11-13 2018-01-30 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
AU2003228792A1 (en) * 2002-04-30 2003-11-17 Avior Therapeutics, Inc. Adenovirus vectors for immunotherapy
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
ES2521682T3 (es) 2003-05-21 2014-11-13 Genzyme Corporation Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & 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
PT3252161T (pt) * 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
HUE040636T2 (hu) 2009-06-16 2019-03-28 Genzyme Corp Javított eljárások rekombináns AAV-vektorok tisztítására
UA115648C2 (uk) * 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Доставка терапевтичних агентів до цнс
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
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015168666A2 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy

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