JP2019500034A5 - - Google Patents

Download PDF

Info

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
Application number
JP2018531240A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500034A (ja
JP7347932B2 (ja
Filing date
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

Links

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 (https=) 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 (https=)
EP (1) EP3390644B1 (https=)
JP (3) JP7347932B2 (https=)
KR (1) KR102833376B1 (https=)
CN (1) CN108603199A (https=)
AU (2) AU2016372035B2 (https=)
CA (1) CA3008268A1 (https=)
ES (1) ES2991277T3 (https=)
IL (1) IL259964B (https=)
MX (1) MX2018007330A (https=)
RU (1) RU2742612C2 (https=)
SG (2) SG11201804994QA (https=)
WO (1) WO2017106313A1 (https=)

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
WO2020106916A1 (en) * 2018-11-21 2020-05-28 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
CA3158131A1 (en) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
EP4103214B1 (en) * 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease

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
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
JP2005523942A (ja) * 2002-04-30 2005-08-11 アヴィオル セラピューティクス インコーポレイテッド 免疫療法のためのアデノウイルスベクター
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
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
ES2635726T3 (es) * 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
LT2588130T (lt) * 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
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
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
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
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai

Similar Documents

Publication Publication Date Title
KR102200642B1 (ko) 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
ES2230569T3 (es) Funciones accesorias para uso en la produccion de viriones aav recombinantes.
JP6174318B2 (ja) アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物
EP1828390B1 (en) Chimeric vectors
JP2022126765A5 (https=)
JP6363958B2 (ja) Ａａｖ変異体
JP2024009857A5 (https=)
JP2021523718A5 (https=)
JP2021003120A5 (https=)
JP2020048571A5 (https=)
CN116096431A (zh) 肽修饰的aav衣壳
JP2017509632A5 (https=)
JP2018522529A5 (https=)
JP2020522269A5 (https=)
AU2022201920A1 (en) Adeno-associated virus mediated delivery of C1EI as a therapy for angioedema
JP2018506261A5 (https=)
ES2210391T3 (es) Sistema auxiliar de alta eficacia para la produccion de vectores aav.
JP2022046635A5 (https=)
JP2017529395A5 (https=)
JP2019500034A5 (https=)
CN111876432B (zh) 一组肝靶向新型腺相关病毒的获得及其应用
JP2025513851A5 (https=)
RU2018124448A (ru) Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
CN112011571A (zh) 一种用于治疗脊髓性肌萎缩的基因治疗药物
JP2025508743A5 (https=)