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
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JP2021538993A
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Japanese (ja)
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JP7716338B2 (ja
JP2022516317A (ja
JPWO2020142752A5 (https=
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Priority claimed from PCT/US2020/012274 external-priority patent/WO2020142752A1/en
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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)

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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 (4)

Publication Number Publication Date
JP2022516317A JP2022516317A (ja) 2022-02-25
JP2022516317A5 true JP2022516317A5 (https=) 2023-01-19
JPWO2020142752A5 JPWO2020142752A5 (https=) 2023-01-19
JP7716338B2 JP7716338B2 (ja) 2025-07-31

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ID=71407140

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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

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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)

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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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CA3039673A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
US20220298514A1 (en) * 2019-09-11 2022-09-22 Aav Gene Therapeutics, Inc. Aav-zyme and use for infusion replacement therapy
US20230355733A1 (en) * 2020-07-14 2023-11-09 Evaxion Biotech A/S 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
KR20230150836A (ko) * 2021-02-26 2023-10-31 다케다 야쿠힌 고교 가부시키가이샤 파브리병 치료를 위한 조성물 및 방법
KR20240073025A (ko) * 2021-08-25 2024-05-24 캔브리지 파마슈티컬스, 인크. 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도
EP4426846A4 (en) * 2021-11-03 2025-11-05 Sangamo Therapeutics Inc METHODS OF USING VIRAL VECTOR CONSTRUCTIONS FOR THE TREATMENT OF FABRY DISEASE
WO2023102517A1 (en) * 2021-12-02 2023-06-08 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
WO2023164060A2 (en) * 2022-02-25 2023-08-31 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy
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

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KR100545945B1 (ko) * 2000-07-03 2006-01-25 갈라 디자인, 인크. 발현 벡터
US6852510B2 (en) * 2000-07-03 2005-02-08 Gala Design Inc Host cells containing multiple integrating vectors
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
EP2708600A4 (en) * 2011-04-28 2015-01-07 Univ Yamaguchi REVERSE PRIMER WITH A TERMINATION SEQUENCE FOR OVEREXPRESSION AND LINEAR DNA
CA3019315A1 (en) * 2015-04-23 2016-10-27 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
WO2016210170A1 (en) * 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
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
AU2017266932B2 (en) * 2016-05-18 2023-04-20 Modernatx, Inc. Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease
CA3039673A1 (en) * 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
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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