CN110809626A - 磺酰胺酶(sgsh)变体、载体、组合物以及用于治疗iiia型粘多糖病(mps iiia)的方法和用途 - Google Patents

磺酰胺酶(sgsh)变体、载体、组合物以及用于治疗iiia型粘多糖病(mps iiia)的方法和用途 Download PDF

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CN110809626A
CN110809626A CN201880043043.4A CN201880043043A CN110809626A CN 110809626 A CN110809626 A CN 110809626A CN 201880043043 A CN201880043043 A CN 201880043043A CN 110809626 A CN110809626 A CN 110809626A
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sgsh
aav
variant
cells
raav
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B·L·戴维森
陈永红
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • C12N9/14—Hydrolases (3)
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Neurosurgery (AREA)
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  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • 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)
CN201880043043.4A 2017-05-12 2018-05-11 磺酰胺酶(sgsh)变体、载体、组合物以及用于治疗iiia型粘多糖病(mps iiia)的方法和用途 Pending CN110809626A (zh)

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 (1)

Publication Number Publication Date
CN110809626A true CN110809626A (zh) 2020-02-18

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CN201880043043.4A Pending CN110809626A (zh) 2017-05-12 2018-05-11 磺酰胺酶(sgsh)变体、载体、组合物以及用于治疗iiia型粘多糖病(mps iiia)的方法和用途

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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)

Cited By (3)

* 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
CN111718947A (zh) * 2020-06-18 2020-09-29 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
CN115335512A (zh) * 2020-03-11 2022-11-11 瑞莫特治疗公司 用于在整个中枢神经系统中传播蛋白质的方法和材料

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.
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
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
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
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

Cited By (3)

* 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
CN115335512A (zh) * 2020-03-11 2022-11-11 瑞莫特治疗公司 用于在整个中枢神经系统中传播蛋白质的方法和材料
CN111718947A (zh) * 2020-06-18 2020-09-29 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

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CA3061925A1 (en) 2018-11-15
WO2018209317A1 (en) 2018-11-15
JP7196099B2 (ja) 2022-12-26
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
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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Application publication date: 20200218