JP2024546578A5 - - Google Patents

Info

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
Prior art keywords
sequence
hsgsh
raav
mirna target
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
JP2024528581A
Other languages
English (en)
Japanese (ja)
Other versions
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 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 (2)

Publication Number Publication Date
JP2024546578A JP2024546578A (ja) 2024-12-26
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
RU2019140862A (ru) * 2017-05-12 2021-06-15 Дзе Чилдрен'З Хоспитал Оф Филадельфия Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia)
AU2018375164B2 (en) * 2017-11-30 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
CN113631182B (zh) * 2018-10-10 2025-03-21 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
KR20220022107A (ko) * 2018-12-05 2022-02-24 아베오나 테라퓨틱스 인코퍼레이티드 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
JP7585233B2 (ja) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物

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