JP2024506346A - 組換えヒト酸性アルファグルコシダーゼ及びその使用 - Google Patents

組換えヒト酸性アルファグルコシダーゼ及びその使用 Download PDF

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JP2024506346A
JP2024506346A JP2023548587A JP2023548587A JP2024506346A JP 2024506346 A JP2024506346 A JP 2024506346A JP 2023548587 A JP2023548587 A JP 2023548587A JP 2023548587 A JP2023548587 A JP 2023548587A JP 2024506346 A JP2024506346 A JP 2024506346A
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treatment
rhgaa
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JP2024506346A5 (enExample
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ハング ドゥ，
ラッセル ゴッシャル，
ヒング チャー，
ジェイ バース，
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アミカス セラピューティックス インコーポレイテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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)
    • C12Y302/01115—Branched-dextran exo-1,2-alpha-glucosidase (3.2.1.115)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Molecular Biology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2023548587A 2021-02-11 2022-02-11 組換えヒト酸性アルファグルコシダーゼ及びその使用 Pending JP2024506346A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163148596P 2021-02-11 2021-02-11
US63/148,596 2021-02-11
US202163162683P 2021-03-18 2021-03-18
US63/162,683 2021-03-18
PCT/US2022/016124 WO2022174037A1 (en) 2021-02-11 2022-02-11 Recombinant human acid alpha-glucosidase and uses thereof

Publications (2)

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JP2024506346A true JP2024506346A (ja) 2024-02-13
JP2024506346A5 JP2024506346A5 (enExample) 2025-02-19

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JP2023548587A Pending JP2024506346A (ja) 2021-02-11 2022-02-11 組換えヒト酸性アルファグルコシダーゼ及びその使用

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US (1) US20240197839A1 (enExample)
EP (1) EP4291225A4 (enExample)
JP (1) JP2024506346A (enExample)
KR (1) KR20230155622A (enExample)
AU (1) AU2022218792A1 (enExample)
BR (1) BR112023016212A2 (enExample)
CA (1) CA3207917A1 (enExample)
IL (1) IL305103A (enExample)
TW (1) TW202245830A (enExample)
WO (1) WO2022174037A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
KR102618519B1 (ko) 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2023215865A1 (en) * 2022-05-05 2023-11-09 Amicus Therapeutics, Inc. Methods for treating pompe disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537604A (ja) * 2014-09-30 2017-12-21 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
JP2019501178A (ja) * 2015-12-30 2019-01-17 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための強化酸性アルファ−グルコシダーゼ
JP2020519638A (ja) * 2017-05-15 2020-07-02 アミカス セラピューティックス インコーポレイテッド 組換えヒト酸性アルファグリコシダーゼ
WO2020163480A1 (en) * 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8679478B2 (en) * 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
CA2867237C (en) * 2012-03-15 2024-01-02 Wouter Vervecken Methods and materials for treatment of pompe's disease
KR102618519B1 (ko) * 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537604A (ja) * 2014-09-30 2017-12-21 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
JP2019501178A (ja) * 2015-12-30 2019-01-17 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための強化酸性アルファ−グルコシダーゼ
JP2020519638A (ja) * 2017-05-15 2020-07-02 アミカス セラピューティックス インコーポレイテッド 組換えヒト酸性アルファグリコシダーゼ
WO2020163480A1 (en) * 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof

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Publication number Publication date
WO2022174037A9 (en) 2023-06-01
KR20230155622A (ko) 2023-11-10
IL305103A (en) 2023-10-01
US20240197839A1 (en) 2024-06-20
EP4291225A1 (en) 2023-12-20
WO2022174037A1 (en) 2022-08-18
TW202245830A (zh) 2022-12-01
AU2022218792A1 (en) 2023-08-24
BR112023016212A2 (pt) 2023-11-28
CA3207917A1 (en) 2022-08-18
EP4291225A4 (en) 2025-01-08
AU2022218792A9 (en) 2024-10-10

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