JP7196099B2 - 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 - Google Patents

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 Download PDF

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JP7196099B2
JP7196099B2 JP2019562293A JP2019562293A JP7196099B2 JP 7196099 B2 JP7196099 B2 JP 7196099B2 JP 2019562293 A JP2019562293 A JP 2019562293A JP 2019562293 A JP2019562293 A JP 2019562293A JP 7196099 B2 JP7196099 B2 JP 7196099B2
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composition
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mammal
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JP2020519629A (ja
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ビバリー， エル． デイビッドソン，
ヨンホン チェン，
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Children's Hospital of Philadelphia
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y310/00—Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01—Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001—N-Sulfoglucosamine sulfohydrolase (3.10.1.1)

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  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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)

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JP2020519629A JP2020519629A (ja) 2020-07-02
JP2020519629A5 JP2020519629A5 (enExample) 2021-07-26
JP7196099B2 true JP7196099B2 (ja) 2022-12-26

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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 (12)

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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
WO2021011841A1 (en) * 2019-07-18 2021-01-21 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
CN115023242B (zh) 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
KR20220153021A (ko) * 2020-03-11 2022-11-17 리모터 테라퓨틱스, 인코포레이티드 중추신경계를 통한 단백질을 확산시키기 위한 방법 및 재료
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
AU2022388800A1 (en) * 2021-11-12 2024-06-27 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of mucopolysaccharidosis iiia
US20250034533A1 (en) * 2021-11-12 2025-01-30 Amicus Therapeutics, Inc. Compositions And Methods For Treating Mucopolysaccharidosis IIIA
WO2023204202A1 (ja) * 2022-04-19 2023-10-26 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
CN120379648A (zh) * 2022-12-23 2025-07-25 株式会社绿十字 具有改善的稳定性的包含乙酰肝素n-硫酸酯酶的药物组合物
WO2026162810A1 (en) * 2025-02-03 2026-08-06 Institut National de la Santé et de la Recherche Médicale Methods for the intravenous treatment of sanfilippo syndrome type iiia

Citations (1)

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JP2013531490A (ja) 2010-06-10 2013-08-08 ラボラトリオス・デル・ドクトル・エステベ・ソシエダッド・アノニマ 疾患の治療のためのベクター及び配列

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US5863782A (en) * 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
EP2550359B1 (en) * 2010-03-23 2017-08-09 Intrexon Corporation Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
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
EP3166626B1 (en) * 2014-07-11 2021-04-28 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013531490A (ja) 2010-06-10 2013-08-08 ラボラトリオス・デル・ドクトル・エステベ・ソシエダッド・アノニマ 疾患の治療のためのベクター及び配列

Non-Patent Citations (2)

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Title
527. Overcoming Limitations Inherent in Sulfamidase to Improve MPS IIIA Gene Therapy，Molecular Therapy, May 2017，Vol.25, No.5S1，p.243
Biochemical and Biophysical Research Communications, 2001年，Vol.280，p.1251-1257，doi:10.1006/bbrc.2001.4265

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WO2018209317A1 (en) 2018-11-15
AU2018265869C1 (en) 2025-01-30
CA3061925A1 (en) 2018-11-15
AU2018265869A1 (en) 2019-11-21
US20210324354A1 (en) 2021-10-21
RU2019140862A3 (enExample) 2021-08-17
JP2020519629A (ja) 2020-07-02
EP3622060A1 (en) 2020-03-18
EP3622060A4 (en) 2021-02-17
BR112019023869A2 (pt) 2020-06-09
RU2019140862A (ru) 2021-06-15
MX2019013528A (es) 2020-07-14
CN110809626A (zh) 2020-02-18
AU2018265869B2 (en) 2024-10-24
US12221635B2 (en) 2025-02-11

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