JP2023520402A5 - - Google Patents

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Publication number
JP2023520402A5
JP2023520402A5 JP2022559589A JP2022559589A JP2023520402A5 JP 2023520402 A5 JP2023520402 A5 JP 2023520402A5 JP 2022559589 A JP2022559589 A JP 2022559589A JP 2022559589 A JP2022559589 A JP 2022559589A JP 2023520402 A5 JP2023520402 A5 JP 2023520402A5
Authority
JP
Japan
Prior art keywords
sequence
raav
item
promoter
enhancer
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
JP2022559589A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023520402A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/024892 external-priority patent/WO2021202532A1/en
Publication of JP2023520402A publication Critical patent/JP2023520402A/ja
Publication of JP2023520402A5 publication Critical patent/JP2023520402A5/ja
Pending legal-status Critical Current

Links

JP2022559589A 2020-03-31 2021-03-30 プロピオン酸血症を処置するための遺伝子治療 Pending JP2023520402A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063002541P 2020-03-31 2020-03-31
US63/002,541 2020-03-31
PCT/US2021/024892 WO2021202532A1 (en) 2020-03-31 2021-03-30 Gene therapy for treating propionic acidemia

Publications (2)

Publication Number Publication Date
JP2023520402A JP2023520402A (ja) 2023-05-17
JP2023520402A5 true JP2023520402A5 (https=) 2024-04-10

Family

ID=75660311

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022559589A Pending JP2023520402A (ja) 2020-03-31 2021-03-30 プロピオン酸血症を処置するための遺伝子治療

Country Status (6)

Country Link
US (1) US20230129893A1 (https=)
EP (1) EP4127189A1 (https=)
JP (1) JP2023520402A (https=)
AR (1) AR121709A1 (https=)
TW (1) TW202204624A (https=)
WO (1) WO2021202532A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
WO2022198079A1 (en) * 2021-03-19 2022-09-22 Mayo Foundation For Medical Education And Research Methods and materials for treating 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

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NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
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JP6947739B2 (ja) 2016-02-16 2021-10-13 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
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KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
WO2018222503A1 (en) 2017-05-31 2018-12-06 The Regents Of The University Of California Adeno-associated virus with variant capsid and methods of use thereof
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BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
JP2023514204A (ja) * 2020-02-14 2023-04-05 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Cdkl5欠損障害を処置するための遺伝子治療

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