JP2024546578A5 - - Google Patents

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Publication number
JP2024546578A5
JP2024546578A5 JP2024528581A JP2024528581A JP2024546578A5 JP 2024546578 A5 JP2024546578 A5 JP 2024546578A5 JP 2024528581 A JP2024528581 A JP 2024528581A JP 2024528581 A JP2024528581 A JP 2024528581A JP 2024546578 A5 JP2024546578 A5 JP 2024546578A5
Authority
JP
Japan
Application number
JP2024528581A
Other languages
Japanese (ja)
Other versions
JPWO2023086928A5 (https=
JP2024546578A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/079701 external-priority patent/WO2023086928A2/en
Publication of JP2024546578A publication Critical patent/JP2024546578A/ja
Publication of JPWO2023086928A5 publication Critical patent/JPWO2023086928A5/ja
Publication of JP2024546578A5 publication Critical patent/JP2024546578A5/ja
Pending legal-status Critical Current

Links

JP2024528581A 2021-11-12 2022-11-11 ムコ多糖症iiia型の治療のための遺伝子療法 Pending JP2024546578A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163278775P 2021-11-12 2021-11-12
US63/278,775 2021-11-12
US202163288293P 2021-12-10 2021-12-10
US63/288,293 2021-12-10
PCT/US2022/079701 WO2023086928A2 (en) 2021-11-12 2022-11-11 Gene therapy for treatment of mucopolysaccharidosis iiia

Publications (3)

Publication Number Publication Date
JP2024546578A JP2024546578A (ja) 2024-12-26
JPWO2023086928A5 JPWO2023086928A5 (https=) 2025-11-18
JP2024546578A5 true JP2024546578A5 (https=) 2025-11-18

Family

ID=86336670

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024528581A Pending JP2024546578A (ja) 2021-11-12 2022-11-11 ムコ多糖症iiia型の治療のための遺伝子療法

Country Status (10)

Country Link
US (1) US20250041445A1 (https=)
EP (1) EP4430201A4 (https=)
JP (1) JP2024546578A (https=)
KR (1) KR20240133693A (https=)
AU (1) AU2022388800A1 (https=)
CA (1) CA3237987A1 (https=)
CO (1) CO2024007283A2 (https=)
IL (1) IL312676A (https=)
MX (1) MX2024005735A (https=)
WO (1) WO2023086928A2 (https=)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019013528A (es) * 2017-05-12 2020-07-14 The Children´S Hospital Of Philadelphia Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia).
KR102823833B1 (ko) * 2017-11-30 2025-06-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 뮤코다당류증 iiia형에 대한 유전자 요법
AR116624A1 (es) * 2018-10-10 2021-05-26 Amicus Therapeutics Inc Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso
US12454701B2 (en) * 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN114127275A (zh) * 2019-04-30 2022-03-01 宾夕法尼亚州大学信托人 可用于治疗庞贝病的组合物

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