TW202342109A - 於長期停止gaa酶替代療法治療龐貝症的治療性腺相關病毒 - Google Patents

於長期停止gaa酶替代療法治療龐貝症的治療性腺相關病毒 Download PDF

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TW202342109A
TW202342109A TW112107060A TW112107060A TW202342109A TW 202342109 A TW202342109 A TW 202342109A TW 112107060 A TW112107060 A TW 112107060A TW 112107060 A TW112107060 A TW 112107060A TW 202342109 A TW202342109 A TW 202342109A
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gaa
administration
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aav
disclosed
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TW112107060A
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山姆 霍普金斯
愛德華 克林頓 史密斯
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美商艾司科皮歐斯生物製藥有限公司
美國公爵大學
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Publication of TW202342109A publication Critical patent/TW202342109A/zh

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    • 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
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    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
TW112107060A 2022-02-25 2023-02-24 於長期停止gaa酶替代療法治療龐貝症的治療性腺相關病毒 TW202342109A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202263314093P 2022-02-25 2022-02-25
US63/314,093 2022-02-25
US202263319034P 2022-03-11 2022-03-11
US63/319,034 2022-03-11
US202263340972P 2022-05-12 2022-05-12
US63/340,972 2022-05-12

Publications (1)

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TW202342109A true TW202342109A (zh) 2023-11-01

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TW112107060A TW202342109A (zh) 2022-02-25 2023-02-24 於長期停止gaa酶替代療法治療龐貝症的治療性腺相關病毒

Country Status (8)

Country Link
US (1) US20250163457A1 (https=)
EP (1) EP4482516A4 (https=)
JP (1) JP2025508841A (https=)
CN (1) CN119095612A (https=)
AU (1) AU2023224856A1 (https=)
CA (1) CA3252501A1 (https=)
TW (1) TW202342109A (https=)
WO (1) WO2023164060A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025053118A1 (ja) * 2023-09-04 2025-03-13 アステラス製薬株式会社 短縮型ｃｍｖプロモーター配列、及び／又は短縮型ポリアデニル化配列を含む組換えアデノ随伴ウイルス
WO2025235807A1 (en) * 2024-05-10 2025-11-13 Solid Biosciences, Inc. Compositions and methods to improve graft survival

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001269923A1 (en) * 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US8679478B2 (en) * 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
US20250213726A1 (en) * 2022-03-18 2025-07-03 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications

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Publication number Publication date
US20250163457A1 (en) 2025-05-22
AU2023224856A1 (en) 2024-08-15
CA3252501A1 (en) 2023-08-31
EP4482516A4 (en) 2026-03-25
JP2025508841A (ja) 2025-04-10
CN119095612A (zh) 2024-12-06
WO2023164060A3 (en) 2023-10-19
EP4482516A2 (en) 2025-01-01
WO2023164060A2 (en) 2023-08-31

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