JP2022516317A5 - - Google Patents

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Publication number
JP2022516317A5
JP2022516317A5 JP2021538993A JP2021538993A JP2022516317A5 JP 2022516317 A5 JP2022516317 A5 JP 2022516317A5 JP 2021538993 A JP2021538993 A JP 2021538993A JP 2021538993 A JP2021538993 A JP 2021538993A JP 2022516317 A5 JP2022516317 A5 JP 2022516317A5
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JP
Japan
Prior art keywords
expression construct
gala
item
seq
cells
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JP2021538993A
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English (en)
Japanese (ja)
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JP2022516317A (ja
JP7716338B2 (ja
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Priority claimed from PCT/US2020/012274 external-priority patent/WO2020142752A1/en
Publication of JP2022516317A publication Critical patent/JP2022516317A/ja
Publication of JP2022516317A5 publication Critical patent/JP2022516317A5/ja
Priority to JP2025121650A priority Critical patent/JP2025142225A/ja
Application granted granted Critical
Publication of JP7716338B2 publication Critical patent/JP7716338B2/ja
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JP2021538993A 2019-01-04 2020-01-03 ファブリー病の処置のための方法及び組成物 Active JP7716338B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025121650A JP2025142225A (ja) 2019-01-04 2025-07-18 ファブリー病の処置のための方法及び組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962788439P 2019-01-04 2019-01-04
US62/788,439 2019-01-04
PCT/US2020/012274 WO2020142752A1 (en) 2019-01-04 2020-01-03 Methods and compositions for the treatment of fabry disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025121650A Division JP2025142225A (ja) 2019-01-04 2025-07-18 ファブリー病の処置のための方法及び組成物

Publications (3)

Publication Number Publication Date
JP2022516317A JP2022516317A (ja) 2022-02-25
JP2022516317A5 true JP2022516317A5 (https=) 2023-01-19
JP7716338B2 JP7716338B2 (ja) 2025-07-31

Family

ID=71407140

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021538993A Active JP7716338B2 (ja) 2019-01-04 2020-01-03 ファブリー病の処置のための方法及び組成物
JP2025121650A Pending JP2025142225A (ja) 2019-01-04 2025-07-18 ファブリー病の処置のための方法及び組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025121650A Pending JP2025142225A (ja) 2019-01-04 2025-07-18 ファブリー病の処置のための方法及び組成物

Country Status (12)

Country Link
US (1) US20200239911A1 (https=)
EP (1) EP3906043A4 (https=)
JP (2) JP7716338B2 (https=)
KR (1) KR20210112337A (https=)
CN (1) CN113423419A (https=)
AU (1) AU2020204718B2 (https=)
BR (1) BR112021012249A2 (https=)
CA (1) CA3124564A1 (https=)
IL (1) IL284414A (https=)
MX (1) MX2021008131A (https=)
SG (1) SG11202107325TA (https=)
WO (1) WO2020142752A1 (https=)

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Publication number Priority date Publication date Assignee Title
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
US20220298514A1 (en) * 2019-09-11 2022-09-22 Aav Gene Therapeutics, Inc. Aav-zyme and use for infusion replacement therapy
CA3183951A1 (en) * 2020-07-14 2022-01-20 Birgitte Rono Apc targeting units for immunotherapy
AU2021356684A1 (en) * 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
AR124981A1 (es) * 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
CA3229998A1 (en) * 2021-08-25 2023-03-02 Canbridge Pharmaceuticals, Inc. Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease
EP4426846A4 (en) * 2021-11-03 2025-11-05 Sangamo Therapeutics Inc METHODS OF USING VIRAL VECTOR CONSTRUCTIONS FOR THE TREATMENT OF FABRY DISEASE
US20240408157A1 (en) * 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CN118475373A (zh) * 2021-12-31 2024-08-09 九天生物医药(上海)有限公司 用于法布雷病的基因治疗的重组aav
CN119095612A (zh) * 2022-02-25 2024-12-06 阿斯克肋匹奥生物制药公司 用于在长期停止gaa酶替代疗法的情况下治疗庞贝病的治疗性腺相关病毒
CN117551636B (zh) * 2023-04-30 2024-11-08 四川至善唯新生物科技有限公司 工程化α-GAL A肽及其功能变体和治疗法布里病的相关方法

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001271614B2 (en) * 2000-07-03 2007-05-31 Catalent Pharma Solutions, Llc Host cells containing multiple integrating vectors
KR100545945B1 (ko) * 2000-07-03 2006-01-25 갈라 디자인, 인크. 발현 벡터
GB0024550D0 (https=) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
EP1995309A1 (en) * 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
JPWO2012147370A1 (ja) * 2011-04-28 2014-07-28 国立大学法人山口大学 ターミネータ配列を含む高発現用リバースプライマー及びリニアｄｎａ
EP3285788B1 (en) * 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MX390848B (es) * 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
CN105950664B (zh) * 2016-05-17 2019-03-29 上海优卡迪生物医药科技有限公司 一种靶向cd123的复制缺陷性重组慢病毒car-t转基因载体及其构建方法和应用
WO2017201328A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
KR102712926B1 (ko) * 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CA3089587A1 (en) * 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
DE102020111571A1 (de) * 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren

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