IL305103A - Recombinant human acid alpha-glucosidase and uses thereof - Google Patents

Recombinant human acid alpha-glucosidase and uses thereof

Info

Publication number
IL305103A
IL305103A IL305103A IL30510323A IL305103A IL 305103 A IL305103 A IL 305103A IL 305103 A IL305103 A IL 305103A IL 30510323 A IL30510323 A IL 30510323A IL 305103 A IL305103 A IL 305103A
Authority
IL
Israel
Prior art keywords
subject
treatment
rhgaa
compared
baseline
Prior art date
Application number
IL305103A
Other languages
English (en)
Hebrew (he)
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of IL305103A publication Critical patent/IL305103A/en

Links

Classifications

    • 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)

Landscapes

  • 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)
IL305103A 2021-02-11 2022-02-11 Recombinant human acid alpha-glucosidase and uses thereof IL305103A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163148596P 2021-02-11 2021-02-11
US202163162683P 2021-03-18 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
IL305103A true IL305103A (en) 2023-10-01

Family

ID=82837922

Family Applications (1)

Application Number Title Priority Date Filing Date
IL305103A IL305103A (en) 2021-02-11 2022-02-11 Recombinant human acid alpha-glucosidase and uses thereof

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
KR20230155622A (ko) 2023-11-10
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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