CN113423419A - 用于治疗法布里病的方法和组合物 - Google Patents

用于治疗法布里病的方法和组合物 Download PDF

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CN113423419A
CN113423419A CN202080010090.6A CN202080010090A CN113423419A CN 113423419 A CN113423419 A CN 113423419A CN 202080010090 A CN202080010090 A CN 202080010090A CN 113423419 A CN113423419 A CN 113423419A
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sequence
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cell
protein
gal
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M·W·赫斯顿
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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CN202080010090.6A 2019-01-04 2020-01-03 用于治疗法布里病的方法和组合物 Pending CN113423419A (zh)

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

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CN113423419A true CN113423419A (zh) 2021-09-21

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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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CN110022904A (zh) * 2016-10-20 2019-07-16 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
CN117551636A (zh) * 2023-04-30 2024-02-13 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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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

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110022904A (zh) * 2016-10-20 2019-07-16 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
CN110022904B (zh) * 2016-10-20 2024-04-19 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
CN117551636A (zh) * 2023-04-30 2024-02-13 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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US20200239911A1 (en) 2020-07-30
JP2025142225A (ja) 2025-09-30
MX2021008131A (es) 2021-10-13
AU2020204718B2 (en) 2026-02-12
IL284414A (en) 2021-08-31
KR20210112337A (ko) 2021-09-14
JP7716338B2 (ja) 2025-07-31
BR112021012249A2 (pt) 2021-09-08
SG11202107325TA (en) 2021-08-30
JP2022516317A (ja) 2022-02-25
EP3906043A4 (en) 2022-10-26
WO2020142752A1 (en) 2020-07-09
CA3124564A1 (en) 2020-07-09
AU2020204718A1 (en) 2021-07-08
EP3906043A1 (en) 2021-11-10

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