JP2022538497A - ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法 - Google Patents

ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法 Download PDF

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JP2022538497A
JP2022538497A JP2022500533A JP2022500533A JP2022538497A JP 2022538497 A JP2022538497 A JP 2022538497A JP 2022500533 A JP2022500533 A JP 2022500533A JP 2022500533 A JP2022500533 A JP 2022500533A JP 2022538497 A JP2022538497 A JP 2022538497A
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vector
lysosomal
lsd
lysosomal enzyme
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JP2022538497A5 (https=
JPWO2021003442A5 (https=
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クオン ドー，
リン リウ，
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エム６ピー セラピューティクス （スイッツァランド） ゲーエムベーハー
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    • 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
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    • C12N9/2465—Hydrolases (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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2477—Hemicellulases not provided in a preceding group
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
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JP2022500533A 2019-07-02 2020-07-02 ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法 Pending JP2022538497A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962869808P 2019-07-02 2019-07-02
US201962869781P 2019-07-02 2019-07-02
US62/869,808 2019-07-02
US62/869,781 2019-07-02
PCT/US2020/040770 WO2021003442A1 (en) 2019-07-02 2020-07-02 Vector compositions and methods of using same for treatment of lysosomal storage disorders

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JP2022538497A true JP2022538497A (ja) 2022-09-02
JP2022538497A5 JP2022538497A5 (https=) 2023-07-13
JPWO2021003442A5 JPWO2021003442A5 (https=) 2023-07-13

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US (1) US12492412B2 (https=)
EP (1) EP3994253A1 (https=)
JP (1) JP2022538497A (https=)
KR (1) KR20220069917A (https=)
CN (1) CN114616000A (https=)
AU (1) AU2020298575A1 (https=)
BR (1) BR112021026866A2 (https=)
CA (1) CA3145662A1 (https=)
TW (1) TW202122579A (https=)
WO (1) WO2021003442A1 (https=)

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JP2024505885A (ja) * 2021-01-27 2024-02-08 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル リソソーム酸リパーゼ欠損症（ｌａｌ－ｄ）の治療のための材料及び方法
CN113238053B (zh) * 2021-04-30 2022-05-13 四川大学华西医院 一种用于检测stat3二聚化的质粒
WO2023150051A1 (en) * 2022-02-04 2023-08-10 M6P Therapeutics, Inc. Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders
WO2023150388A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising glucocerebrocidase and methods of use thereof
WO2025238106A1 (en) 2024-05-15 2025-11-20 M6P Therapeutics (Switzerland) GmbH Lysosome-targeting degradation fusion design
CN119950766A (zh) * 2025-02-11 2025-05-09 中国医学科学院阜外医院 一种治疗pnpla2突变致心律失常性心肌病的基因药物及其制备方法和应用

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US20220380800A1 (en) 2022-12-01
CN114616000A (zh) 2022-06-10
EP3994253A1 (en) 2022-05-11
US12492412B2 (en) 2025-12-09
KR20220069917A (ko) 2022-05-27
WO2021003442A1 (en) 2021-01-07
TW202122579A (zh) 2021-06-16
CA3145662A1 (en) 2021-01-07
BR112021026866A2 (pt) 2022-03-03

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