KR20230155622A - 재조합 인간 산성 알파-글루코시다제 및 이의 용도 - Google Patents

재조합 인간 산성 알파-글루코시다제 및 이의 용도 Download PDF

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Publication number
KR20230155622A
KR20230155622A KR1020237031038A KR20237031038A KR20230155622A KR 20230155622 A KR20230155622 A KR 20230155622A KR 1020237031038 A KR1020237031038 A KR 1020237031038A KR 20237031038 A KR20237031038 A KR 20237031038A KR 20230155622 A KR20230155622 A KR 20230155622A
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South Korea
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subject
treatment
rhgaa
compared
baseline
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KR1020237031038A
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English (en)
Korean (ko)
Inventor
헝 도
러셀 갓샬
힝 찰
제이 발스
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
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Publication of KR20230155622A publication Critical patent/KR20230155622A/ko
Pending legal-status Critical Current

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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
    • 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
    • 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)
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • 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)
KR1020237031038A 2021-02-11 2022-02-11 재조합 인간 산성 알파-글루코시다제 및 이의 용도 Pending KR20230155622A (ko)

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 (1)

Publication Number Publication Date
KR20230155622A true KR20230155622A (ko) 2023-11-10

Family

ID=82837922

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237031038A Pending KR20230155622A (ko) 2021-02-11 2022-02-11 재조합 인간 산성 알파-글루코시다제 및 이의 용도

Country Status (10)

Country Link
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

Family Cites Families (7)

* 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
JP6851964B2 (ja) * 2014-09-30 2021-03-31 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
IL299470B2 (en) * 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
KR102618519B1 (ko) * 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
CN119113091A (zh) * 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
WO2020163480A1 (en) * 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof

Also Published As

Publication number Publication date
JP2024506346A (ja) 2024-02-13
WO2022174037A9 (en) 2023-06-01
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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PA0105 International application

Patent event date: 20230911

Patent event code: PA01051R01D

Comment text: International Patent Application

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A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250210

Comment text: Request for Examination of Application