JP2024504611A5 - - Google Patents

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Publication number
JP2024504611A5
JP2024504611A5 JP2023542653A JP2023542653A JP2024504611A5 JP 2024504611 A5 JP2024504611 A5 JP 2024504611A5 JP 2023542653 A JP2023542653 A JP 2023542653A JP 2023542653 A JP2023542653 A JP 2023542653A JP 2024504611 A5 JP2024504611 A5 JP 2024504611A5
Authority
JP
Japan
Prior art keywords
sequence
seq
gla
nucleic acid
signal peptide
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.)
Withdrawn
Application number
JP2023542653A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024504611A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/070184 external-priority patent/WO2022155665A1/en
Publication of JP2024504611A publication Critical patent/JP2024504611A/ja
Publication of JP2024504611A5 publication Critical patent/JP2024504611A5/ja
Withdrawn legal-status Critical Current

Links

JP2023542653A 2021-01-14 2022-01-13 ファブリー病を治療するための組成物および方法 Withdrawn JP2024504611A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163137235P 2021-01-14 2021-01-14
US63/137,235 2021-01-14
US202163264356P 2021-11-19 2021-11-19
US63/264,356 2021-11-19
PCT/US2022/070184 WO2022155665A1 (en) 2021-01-14 2022-01-13 Compositions and methods for treating fabry disease

Publications (2)

Publication Number Publication Date
JP2024504611A JP2024504611A (ja) 2024-02-01
JP2024504611A5 true JP2024504611A5 (https=) 2025-01-08

Family

ID=82448637

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023542653A Withdrawn JP2024504611A (ja) 2021-01-14 2022-01-13 ファブリー病を治療するための組成物および方法

Country Status (12)

Country Link
US (1) US20240301380A1 (https=)
EP (1) EP4277988A4 (https=)
JP (1) JP2024504611A (https=)
KR (1) KR20230131246A (https=)
AU (1) AU2022207185A1 (https=)
CA (1) CA3208153A1 (https=)
CL (1) CL2023002015A1 (https=)
CO (1) CO2023010599A2 (https=)
IL (1) IL304273A (https=)
MX (1) MX2023008362A (https=)
PE (1) PE20240915A1 (https=)
WO (1) WO2022155665A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4426846A4 (en) * 2021-11-03 2025-11-05 Sangamo Therapeutics Inc METHODS OF USING VIRAL VECTOR CONSTRUCTIONS FOR THE TREATMENT OF FABRY DISEASE
KR20250161626A (ko) * 2023-03-20 2025-11-17 스파크 테라퓨틱스, 인코포레이티드 Ppt1 유전자 치료
US20240360431A1 (en) * 2023-04-30 2024-10-31 Sichuan Real&Best Biotech Co., Ltd. Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
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
WO2025264565A1 (en) * 2024-06-17 2025-12-26 Spark Therapeutics, Inc. Promoters

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
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
MY207355A (en) * 2018-05-16 2025-02-21 Spark Therapeutics Inc Codon-optimized acid lpha-glucosidase expression cassettes and methods of using same
KR20220022107A (ko) * 2018-12-05 2022-02-24 아베오나 테라퓨틱스 인코퍼레이티드 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
US12163425B2 (en) * 2020-03-13 2024-12-10 Baker Hughes Oilfield Operations Llc Automated geosteering based on a distance to oil-water contact

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