JP2023514204A5 - - Google Patents

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Publication number
JP2023514204A5
JP2023514204A5 JP2022548652A JP2022548652A JP2023514204A5 JP 2023514204 A5 JP2023514204 A5 JP 2023514204A5 JP 2022548652 A JP2022548652 A JP 2022548652A JP 2022548652 A JP2022548652 A JP 2022548652A JP 2023514204 A5 JP2023514204 A5 JP 2023514204A5
Authority
JP
Japan
Prior art keywords
sequence
raav
promoter
sequences
item
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
JP2022548652A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023514204A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/017656 external-priority patent/WO2021163322A1/en
Publication of JP2023514204A publication Critical patent/JP2023514204A/ja
Publication of JP2023514204A5 publication Critical patent/JP2023514204A5/ja
Pending legal-status Critical Current

Links

JP2022548652A 2020-02-14 2021-02-11 Cdkl5欠損障害を処置するための遺伝子治療 Pending JP2023514204A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062976483P 2020-02-14 2020-02-14
US62/976,483 2020-02-14
US202063090492P 2020-10-12 2020-10-12
US63/090,492 2020-10-12
PCT/US2021/017656 WO2021163322A1 (en) 2020-02-14 2021-02-11 Gene therapy for treating cdkl5 deficiency disorder

Publications (2)

Publication Number Publication Date
JP2023514204A JP2023514204A (ja) 2023-04-05
JP2023514204A5 true JP2023514204A5 (https=) 2024-02-20

Family

ID=74873804

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022548652A Pending JP2023514204A (ja) 2020-02-14 2021-02-11 Cdkl5欠損障害を処置するための遺伝子治療

Country Status (12)

Country Link
US (1) US20230054144A1 (https=)
EP (1) EP4103724A1 (https=)
JP (1) JP2023514204A (https=)
KR (1) KR20220140537A (https=)
CN (1) CN115151648A (https=)
AU (1) AU2021218703A1 (https=)
BR (1) BR112022015921A2 (https=)
CA (1) CA3164714A1 (https=)
IL (1) IL294781A (https=)
MX (1) MX2022009982A (https=)
TW (1) TWI904135B (https=)
WO (1) WO2021163322A1 (https=)

Families Citing this family (12)

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WO2021202532A1 (en) * 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
KR20230003569A (ko) * 2020-04-27 2023-01-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물
WO2022225978A1 (en) * 2021-04-21 2022-10-27 The Regents Of The University Of California Use of a split dcas fusion protein system for epigenetic editing
EP4387641A4 (en) * 2021-08-18 2025-07-09 Ultragenyx Pharmaceutical Inc RECOMBINANT AAV FORMULATIONS
EP4349365A1 (en) * 2022-10-07 2024-04-10 Hansa Biopharma AB Co-treatment for gene therapy
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CN116377050A (zh) * 2022-12-16 2023-07-04 湖南家辉生物技术有限公司 一种发育性癫痫性脑病2型致病基因cdkl5突变位点的应用及其检测试剂和应用
EP4713028A1 (en) 2023-05-18 2026-03-25 Ultragenyx Pharmaceutical Inc. Formulations comprising recombinant aav and methods of administering the same
WO2025122548A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv

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