JPWO2020072451A5 - - Google Patents

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Publication number
JPWO2020072451A5
JPWO2020072451A5 JP2021542078A JP2021542078A JPWO2020072451A5 JP WO2020072451 A5 JPWO2020072451 A5 JP WO2020072451A5 JP 2021542078 A JP2021542078 A JP 2021542078A JP 2021542078 A JP2021542078 A JP 2021542078A JP WO2020072451 A5 JPWO2020072451 A5 JP WO2020072451A5
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JP
Japan
Prior art keywords
sequence
raav
pccb
pcca
seq
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.)
Pending
Application number
JP2021542078A
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English (en)
Japanese (ja)
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JP2022513318A (ja
JP2022513318A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/054003 external-priority patent/WO2020072451A1/en
Publication of JP2022513318A publication Critical patent/JP2022513318A/ja
Publication of JP2022513318A5 publication Critical patent/JP2022513318A5/ja
Publication of JPWO2020072451A5 publication Critical patent/JPWO2020072451A5/ja
Pending legal-status Critical Current

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JP2021542078A 2018-10-01 2019-10-01 プロピオン酸血症を処置するための遺伝子治療 Pending JP2022513318A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862739471P 2018-10-01 2018-10-01
US62/739,471 2018-10-01
PCT/US2019/054003 WO2020072451A1 (en) 2018-10-01 2019-10-01 Gene therapy for treating propionic acidemia

Publications (3)

Publication Number Publication Date
JP2022513318A JP2022513318A (ja) 2022-02-07
JP2022513318A5 JP2022513318A5 (https=) 2022-10-12
JPWO2020072451A5 true JPWO2020072451A5 (https=) 2022-10-12

Family

ID=70055045

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021542078A Pending JP2022513318A (ja) 2018-10-01 2019-10-01 プロピオン酸血症を処置するための遺伝子治療

Country Status (11)

Country Link
US (2) US12472268B2 (https=)
EP (1) EP3861131A4 (https=)
JP (1) JP2022513318A (https=)
KR (1) KR20210082460A (https=)
CN (1) CN112955557A (https=)
AR (1) AR116569A1 (https=)
AU (1) AU2019354296A1 (https=)
BR (1) BR112021006052A2 (https=)
CA (1) CA3113975A1 (https=)
TW (1) TW202028472A (https=)
WO (1) WO2020072451A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3990029A1 (en) * 2019-06-27 2022-05-04 The United States of America, as represented by the Secretary, Department of Health and Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
WO2021076797A1 (en) * 2019-10-15 2021-04-22 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase beta
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP4308174A4 (en) * 2021-03-19 2025-07-30 Mayo Found Medical Education & Res METHODS AND MATERIALS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
WO2023130003A2 (en) * 2021-12-29 2023-07-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
CN118166004B (zh) * 2024-05-14 2024-12-03 北京剂泰医药科技有限公司 用于编码人PCCA或PCCB蛋白的mRNA及其用途

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