JP2020519629A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
raav
sgsh
particle according
variant
raav particle
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.)
Granted
Application number
JP2019562293A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020519629A (ja
JP7196099B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/032454 external-priority patent/WO2018209317A1/en
Publication of JP2020519629A publication Critical patent/JP2020519629A/ja
Publication of JP2020519629A5 publication Critical patent/JP2020519629A5/ja
Application granted granted Critical
Publication of JP7196099B2 publication Critical patent/JP7196099B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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 (enExample) 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 (enExample)
EP (1) EP3622060A4 (enExample)
JP (1) JP7196099B2 (enExample)
CN (1) CN110809626A (enExample)
AU (1) AU2018265869C1 (enExample)
BR (1) BR112019023869A2 (enExample)
CA (1) CA3061925A1 (enExample)
MX (1) MX2019013528A (enExample)
RU (1) RU2019140862A (enExample)
WO (1) WO2018209317A1 (enExample)

Families Citing this family (9)

* 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
EP3897695B1 (en) * 2018-12-20 2025-06-25 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
AR122285A1 (es) * 2019-07-18 2022-08-31 Lysogene Composiciones y métodos para el tratamiento de la enfermedad de sanfilippo y otros trastornos
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
EP4093859A4 (en) * 2020-03-11 2023-06-14 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
WO2023086939A1 (en) * 2021-11-12 2023-05-19 Amicus Therapeutics, Inc. Compositions and methods for treating mucopolysaccharidosis iiia
JP2023159049A (ja) * 2022-04-19 2023-10-31 Ｊｃｒファーマ株式会社 マンノース－６－リン酸の数を減少させた糖蛋白質
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
CN103038343A (zh) * 2010-03-23 2013-04-10 英特瑞克斯顿股份有限公司 条件表达治疗蛋白的载体，包含所述载体的宿主细胞，及其应用
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
CA2832151C (en) 2011-03-31 2021-06-15 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US20170191041A1 (en) 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes

Similar Documents

Publication Publication Date Title
JP2020519629A5 (enExample)
JP2019511570A5 (enExample)
US20220364117A1 (en) Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
JP2020533959A5 (enExample)
JP2019537576A5 (enExample)
US20230416757A1 (en) Sod1 dual expression vectors and uses thereof
IL278213B1 (en) A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
JP2016503405A5 (enExample)
JP2017529395A5 (enExample)
JP2019529385A5 (enExample)
US20260049336A1 (en) Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof
JP2020533276A5 (enExample)
RU2018145010A (ru) Оптимизированные гены и экспрессионные кассеты cln1, и их применение
IL262852B1 (en) Treatment of complement-mediated disorders
US20210228739A1 (en) Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
JP2020509786A5 (enExample)
FI3373980T3 (fi) Menetelmiä lihasdystrofian hoitamiseksi
US12270033B2 (en) Dual leucine zipper kinase inhibitors for gene therapy
JPWO2020041773A5 (enExample)
JPWO2019226832A5 (enExample)
JPWO2022034130A5 (enExample)
KR102874560B1 (ko) 아데노연관바이러스 변이체
JPWO2023086966A5 (enExample)
JPWO2019200286A5 (enExample)
WO2022221447A4 (en) Materials and methods for neurofibromin 2/merlin (nf2) gene therapy