KR20230093241A - 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법 - Google Patents

글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법 Download PDF

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KR20230093241A
KR20230093241A KR1020237006825A KR20237006825A KR20230093241A KR 20230093241 A KR20230093241 A KR 20230093241A KR 1020237006825 A KR1020237006825 A KR 1020237006825A KR 20237006825 A KR20237006825 A KR 20237006825A KR 20230093241 A KR20230093241 A KR 20230093241A
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기리다르 무랄리드하란
제프리 브라운
엘리자베스 놀
옌췬 슈
켈리 베일즈
진자오 호우
아데베일 아델루이
브렛 호프만
스미타 잭탭
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보이저 테라퓨틱스, 인크.
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    • C—CHEMISTRY; METALLURGY
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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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/235—Adenoviridae
    • 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
    • A61P25/14—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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    • 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
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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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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Plant Pathology (AREA)
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  • Immunology (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020237006825A 2020-07-27 2021-07-26 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법 Pending KR20230093241A (ko)

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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KR20230093241A true KR20230093241A (ko) 2023-06-27

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KR1020237006825A Pending KR20230093241A (ko) 2020-07-27 2021-07-26 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법

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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)

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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
CN118064436B (zh) * 2024-04-18 2024-07-26 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
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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