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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ビバリー， エル． デイビッドソン，
ヨンホン チェン，
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Childrens Hospital of Philadelphia CHOP
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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
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    • 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
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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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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

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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
MX2022006188A (es) * 2019-11-22 2022-08-22 Childrens Hospital Philadelphia Variantes del vector viral adenoasociado.
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
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

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

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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
TWI688395B (zh) * 2010-03-23 2020-03-21 英翠克頌公司 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途
US9849195B2 (en) * 2011-03-31 2017-12-26 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

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)

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

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