JP2025515159A - ポンペ病の治療方法 - Google Patents

ポンペ病の治療方法 Download PDF

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Publication number
JP2025515159A
JP2025515159A JP2024565157A JP2024565157A JP2025515159A JP 2025515159 A JP2025515159 A JP 2025515159A JP 2024565157 A JP2024565157 A JP 2024565157A JP 2024565157 A JP2024565157 A JP 2024565157A JP 2025515159 A JP2025515159 A JP 2025515159A
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atb200
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ert
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Japanese (ja)
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JP2025515159A5 (enExample
Inventor
ジェイ バース，
シーラ シタラマン ダス，
ジェフ カステリ，
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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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Publication of JP2025515159A5 publication Critical patent/JP2025515159A5/ja
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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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38—Cellulose; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Inorganic Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024565157A 2022-05-05 2023-05-05 ポンペ病の治療方法 Pending JP2025515159A (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US202263338601P 2022-05-05 2022-05-05
US63/338,601 2022-05-05
US202263399498P 2022-08-19 2022-08-19
US202263399491P 2022-08-19 2022-08-19
US63/399,498 2022-08-19
US63/399,491 2022-08-19
US202263404475P 2022-09-07 2022-09-07
US63/404,475 2022-09-07
US202263414897P 2022-10-10 2022-10-10
US63/414,897 2022-10-10
US202263428970P 2022-11-30 2022-11-30
US63/428,970 2022-11-30
US202263431920P 2022-12-12 2022-12-12
US63/431,920 2022-12-12
US202263434791P 2022-12-22 2022-12-22
US63/434,791 2022-12-22
US202363447222P 2023-02-21 2023-02-21
US63/447,222 2023-02-21
PCT/US2023/066657 WO2023215865A1 (en) 2022-05-05 2023-05-05 Methods for treating pompe disease

Publications (2)

Publication Number Publication Date
JP2025515159A true JP2025515159A (ja) 2025-05-13
JP2025515159A5 JP2025515159A5 (enExample) 2026-05-15

Family

ID=86692686

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024565157A Pending JP2025515159A (ja) 2022-05-05 2023-05-05 ポンペ病の治療方法

Country Status (13)

Country Link
US (1) US20260041744A1 (enExample)
EP (1) EP4518885A1 (enExample)
JP (1) JP2025515159A (enExample)
KR (1) KR20250007602A (enExample)
CN (1) CN119343147A (enExample)
AU (1) AU2023265155A1 (enExample)
CA (1) CA3252291A1 (enExample)
CL (1) CL2024003377A1 (enExample)
CO (1) CO2024016724A2 (enExample)
IL (1) IL316776A (enExample)
MX (1) MX2024013673A (enExample)
TW (1) TW202400212A (enExample)
WO (1) WO2023215865A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026006625A1 (en) 2024-06-26 2026-01-02 Amicus Therapeutics, Inc. Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
US11382980B2 (en) 2013-10-08 2022-07-12 Taiyo Kagaku Co., Ltd. Oil/fat composition containing polyunsaturated fatty acid
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
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
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
JP2024506346A (ja) * 2021-02-11 2024-02-13 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ルコシダーゼ及びその使用

Also Published As

Publication number Publication date
MX2024013673A (es) 2024-12-06
IL316776A (en) 2025-01-01
CN119343147A (zh) 2025-01-21
CA3252291A1 (en) 2023-11-09
AU2023265155A1 (en) 2024-12-12
KR20250007602A (ko) 2025-01-14
WO2023215865A1 (en) 2023-11-09
CL2024003377A1 (es) 2025-03-28
US20260041744A1 (en) 2026-02-12
CO2024016724A2 (es) 2025-04-28
EP4518885A1 (en) 2025-03-12
TW202400212A (zh) 2024-01-01

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