JP2020519629A5 - - Google Patents

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Publication number
JP2020519629A5
JP2020519629A5 JP2019562293A JP2019562293A JP2020519629A5 JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5 JP 2019562293 A JP2019562293 A JP 2019562293A JP 2019562293 A JP2019562293 A JP 2019562293A JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5
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JP
Japan
Prior art keywords
raav
sgsh
particle according
variant
raav particle
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JP2019562293A
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English (en)
Japanese (ja)
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JP2020519629A (ja
JP7196099B2 (ja
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Priority claimed from PCT/US2018/032454 external-priority patent/WO2018209317A1/en
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Publication of JP2020519629A5 publication Critical patent/JP2020519629A5/ja
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JP2019562293A 2017-05-12 2018-05-11 ムコ多糖症ｉｉｉａ型（ｍｐｓ ｉｉｉａ）を処置するための、スルファミダーゼ（ｓｇｓｈ）バリアント、ベクター、組成物並びに方法及び使用 Active JP7196099B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762505423P 2017-05-12 2017-05-12
US62/505,423 2017-05-12
PCT/US2018/032454 WO2018209317A1 (en) 2017-05-12 2018-05-11 Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia)

Publications (3)

Publication Number Publication Date
JP2020519629A JP2020519629A (ja) 2020-07-02
JP2020519629A5 true JP2020519629A5 (https=) 2021-07-26
JP7196099B2 JP7196099B2 (ja) 2022-12-26

Family

ID=64104969

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019562293A Active JP7196099B2 (ja) 2017-05-12 2018-05-11 ムコ多糖症ｉｉｉａ型（ｍｐｓ ｉｉｉａ）を処置するための、スルファミダーゼ（ｓｇｓｈ）バリアント、ベクター、組成物並びに方法及び使用

Country Status (10)

Country Link
US (1) US12221635B2 (https=)
EP (1) EP3622060A4 (https=)
JP (1) JP7196099B2 (https=)
CN (1) CN110809626A (https=)
AU (1) AU2018265869C1 (https=)
BR (1) BR112019023869A2 (https=)
CA (1) CA3061925A1 (https=)
MX (1) MX2019013528A (https=)
RU (1) RU2019140862A (https=)
WO (1) WO2018209317A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108728495A (zh) * 2018-05-31 2018-11-02 深圳市免疫基因治疗研究院 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用
ES3035673T3 (en) * 2018-12-20 2025-09-08 Esteve Pharmaceuticals Sa Recombinant vectors for the long term treatment of mucchopolysacharidosis
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
KR20220116459A (ko) * 2019-11-22 2022-08-23 더 칠드런스 호스피탈 오브 필라델피아 아데노(adeno)-관련 바이러스 벡터 변이체
CA3174928A1 (en) * 2020-03-11 2021-09-16 Remotor Therapeutics, Inc. Methods and materials for disseminating a protein throughout the central nervous system
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神（北京）生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
KR20240133693A (ko) * 2021-11-12 2024-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 점액다당류증 iiia의 치료를 위한 유전자 요법
WO2023086939A1 (en) * 2021-11-12 2023-05-19 Amicus Therapeutics, Inc. Compositions and methods for treating mucopolysaccharidosis iiia
US20250257338A1 (en) * 2022-04-19 2025-08-14 Jcr Pharmaceuticals Co., Ltd. Glycoprotein having reduced number of mannose-6-phosphates
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5863782A (en) * 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
KR20130010121A (ko) * 2010-03-23 2013-01-25 인트렉손 코포레이션 치료적 단백질을 조건부로 발현하는 벡터，상기 벡터를 포함하는 숙주 세포 및 이의 용도
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
EP3166626B1 (en) 2014-07-11 2021-04-28 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes

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