JP2022046635A5 - - Google Patents

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Publication number
JP2022046635A5
JP2022046635A5 JP2021210071A JP2021210071A JP2022046635A5 JP 2022046635 A5 JP2022046635 A5 JP 2022046635A5 JP 2021210071 A JP2021210071 A JP 2021210071A JP 2021210071 A JP2021210071 A JP 2021210071A JP 2022046635 A5 JP2022046635 A5 JP 2022046635A5
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JP
Japan
Prior art keywords
raav
gnptab
aav
iii
particle
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
Application number
JP2021210071A
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English (en)
Japanese (ja)
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JP2022046635A (ja
JP7878799B2 (ja
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Priority claimed from PCT/US2016/066611 external-priority patent/WO2017106313A1/en
Application filed filed Critical
Publication of JP2022046635A publication Critical patent/JP2022046635A/ja
Publication of JP2022046635A5 publication Critical patent/JP2022046635A5/ja
Priority to JP2024019140A priority Critical patent/JP2024056832A/ja
Application granted granted Critical
Publication of JP7878799B2 publication Critical patent/JP7878799B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021210071A 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 Active JP7878799B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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 (4)

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
JP2018531240A 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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2018531240A Division 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024019140A Division 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

Publications (3)

Publication Number Publication Date
JP2022046635A JP2022046635A (ja) 2022-03-23
JP2022046635A5 true JP2022046635A5 (enExample) 2022-12-09
JP7878799B2 JP7878799B2 (ja) 2026-06-23

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 Before (1)

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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
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)

* 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
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

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