TW202229560A - 治療法布瑞氏症之組成物及方法 - Google Patents

治療法布瑞氏症之組成物及方法 Download PDF

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TW202229560A
TW202229560A TW110137528A TW110137528A TW202229560A TW 202229560 A TW202229560 A TW 202229560A TW 110137528 A TW110137528 A TW 110137528A TW 110137528 A TW110137528 A TW 110137528A TW 202229560 A TW202229560 A TW 202229560A
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hgla
gla
seq
sequence
aavhu68
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TW110137528A
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茱麗葉 豪杜司
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賓州大學委員會
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    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • AHUMAN NECESSITIES
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    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
TW110137528A 2020-10-09 2021-10-08 治療法布瑞氏症之組成物及方法 TW202229560A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063089850P 2020-10-09 2020-10-09
US63/089,850 2020-10-09
US202163146286P 2021-02-05 2021-02-05
US63/146,286 2021-02-05
US202163186092P 2021-05-08 2021-05-08
US63/186,092 2021-05-08

Publications (1)

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TW202229560A true TW202229560A (zh) 2022-08-01

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TW110137528A TW202229560A (zh) 2020-10-09 2021-10-08 治療法布瑞氏症之組成物及方法

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US (1) US20230365955A1 (https=)
EP (1) EP4225906A1 (https=)
JP (1) JP2023545433A (https=)
KR (1) KR20230118075A (https=)
AU (1) AU2021356684A1 (https=)
CA (1) CA3193833A1 (https=)
TW (1) TW202229560A (https=)
WO (1) WO2022076803A1 (https=)

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AR124981A1 (es) * 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
US20240408157A1 (en) * 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
EP4551711A1 (en) * 2022-07-08 2025-05-14 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease
AU2023331251A1 (en) * 2022-08-25 2025-02-27 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof

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EP4225906A1 (en) 2023-08-16
AU2021356684A1 (en) 2023-05-11
US20230365955A1 (en) 2023-11-16
WO2022076803A1 (en) 2022-04-14
WO2022076803A8 (en) 2022-06-09
CA3193833A1 (en) 2022-04-14
KR20230118075A (ko) 2023-08-10
JP2023545433A (ja) 2023-10-30

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