CN117120619A - 用于治疗与葡萄糖神经酰胺酶β缺陷相关的神经病症的组合物和方法 - Google Patents

用于治疗与葡萄糖神经酰胺酶β缺陷相关的神经病症的组合物和方法 Download PDF

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Publication number
CN117120619A
CN117120619A CN202180066205.8A CN202180066205A CN117120619A CN 117120619 A CN117120619 A CN 117120619A CN 202180066205 A CN202180066205 A CN 202180066205A CN 117120619 A CN117120619 A CN 117120619A
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seq
amino acid
sequence
acid sequence
nucleotide sequence
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G·穆里达兰
J·布朗
E·诺尔
舒颜群
K·贝尔斯
侯金兆
A·阿德鲁伊
B·霍夫曼
S·贾格塔普
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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    • 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)
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/235—Adenoviridae
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN202180066205.8A 2020-07-27 2021-07-26 用于治疗与葡萄糖神经酰胺酶β缺陷相关的神经病症的组合物和方法 Pending CN117120619A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063057265P 2020-07-27 2020-07-27
US63/057,265 2020-07-27
PCT/US2021/043216 WO2022026409A1 (en) 2020-07-27 2021-07-26 Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

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CN117120619A true CN117120619A (zh) 2023-11-24

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US (1) US20230227802A1 (enExample)
EP (1) EP4189095A1 (enExample)
JP (2) JP2023536091A (enExample)
KR (1) KR20230093241A (enExample)
CN (1) CN117120619A (enExample)
AU (1) AU2021315876A1 (enExample)
BR (1) BR112023001456A2 (enExample)
CA (1) CA3190309A1 (enExample)
IL (1) IL299928A (enExample)
MX (1) MX2023000815A (enExample)
TW (1) TW202221125A (enExample)
WO (1) WO2022026409A1 (enExample)

Cited By (1)

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CN118064436A (zh) * 2024-04-18 2024-05-24 上海凌医生物科技有限公司 一种增强基因表达的嵌合启动子

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US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023215851A2 (en) * 2022-05-06 2023-11-09 Apic Bio, Inc. Plasmid optimized for packaging of aav vectors
AU2023427408A1 (en) * 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226761A2 (en) * 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024226790A1 (en) * 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
CN117264912A (zh) * 2023-09-25 2023-12-22 中国科学院深圳先进技术研究院 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用
CN121518479B (zh) * 2026-01-16 2026-04-24 凌意(杭州)生物科技有限公司 基因编辑复合型突变小鼠模型的构建方法

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