JP2023552443A5 - - Google Patents

Info

Publication number
JP2023552443A5
JP2023552443A5 JP2023534292A JP2023534292A JP2023552443A5 JP 2023552443 A5 JP2023552443 A5 JP 2023552443A5 JP 2023534292 A JP2023534292 A JP 2023534292A JP 2023534292 A JP2023534292 A JP 2023534292A JP 2023552443 A5 JP2023552443 A5 JP 2023552443A5
Authority
JP
Japan
Application number
JP2023534292A
Other languages
Japanese (ja)
Other versions
JP2023552443A (ja
JPWO2022125489A5 (https=
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/062112 external-priority patent/WO2022125489A1/en
Publication of JP2023552443A publication Critical patent/JP2023552443A/ja
Publication of JP2023552443A5 publication Critical patent/JP2023552443A5/ja
Publication of JPWO2022125489A5 publication Critical patent/JPWO2022125489A5/ja
Pending legal-status Critical Current

Links

JP2023534292A 2020-12-07 2021-12-07 ダノン病の治療 Pending JP2023552443A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063122249P 2020-12-07 2020-12-07
US63/122,249 2020-12-07
PCT/US2021/062112 WO2022125489A1 (en) 2020-12-07 2021-12-07 Treatment of danon disease

Publications (3)

Publication Number Publication Date
JP2023552443A JP2023552443A (ja) 2023-12-15
JP2023552443A5 true JP2023552443A5 (https=) 2024-11-19
JPWO2022125489A5 JPWO2022125489A5 (https=) 2024-11-19

Family

ID=81973960

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023534292A Pending JP2023552443A (ja) 2020-12-07 2021-12-07 ダノン病の治療

Country Status (11)

Country Link
US (1) US20240033325A1 (https=)
EP (1) EP4255458A4 (https=)
JP (1) JP2023552443A (https=)
KR (1) KR20230129431A (https=)
CN (1) CN116887868A (https=)
AU (1) AU2021396150A1 (https=)
CA (1) CA3201247A1 (https=)
CL (1) CL2023001650A1 (https=)
IL (1) IL303428A (https=)
MX (1) MX2023006694A (https=)
WO (1) WO2022125489A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Aavベクターを使用したプラコフィリン-2(pkp2)遺伝子治療
WO2025129071A1 (en) * 2023-12-14 2025-06-19 Sonothera, Inc. Methods and systems for improved nucleic acid delivery via ultrasound

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170049887A1 (en) * 2014-04-25 2017-02-23 University Of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
DK3405215T3 (da) * 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
CA3049915A1 (en) * 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
AU2020221842A1 (en) * 2019-02-12 2021-08-12 Spacecraft Seven, Llc Gene therapy vectors for treatment of Danon Disease

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