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
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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
WO2020127678A1 (en) * 2018-12-20 2020-06-25 Esteve Pharmaceuticals, S.A. 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
PH12022551247A1 (en) * 2019-11-22 2023-11-13 Childrens Hospital Philadelphia Adeno-associated viral vector variants
JP2023517230A (ja) * 2020-03-11 2023-04-24 リモーター セラピューティクス インコーポレイテッド 中枢神経系全体にわたってタンパク質を散在させるための方法および材料
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
WO2023086939A1 (en) * 2021-11-12 2023-05-19 Amicus Therapeutics, Inc. Compositions and methods for treating mucopolysaccharidosis iiia
EP4430201A4 (en) * 2021-11-12 2026-04-08 Univ Pennsylvania Gene therapy for the treatment of mucopolysaccharidosis IIIA
EP4512892A1 (en) * 2022-04-19 2025-02-26 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
CA2794196A1 (en) * 2010-03-23 2011-09-29 Intrexon Corporation Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
ES2745470T3 (es) 2011-03-31 2020-03-02 Univ Iowa Res Found Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
US20170191041A1 (en) * 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes

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