JP2023530112A - 遅発型ポンペ病を処置するための組成物及び方法 - Google Patents

遅発型ポンペ病を処置するための組成物及び方法 Download PDF

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Publication number
JP2023530112A
JP2023530112A JP2022576792A JP2022576792A JP2023530112A JP 2023530112 A JP2023530112 A JP 2023530112A JP 2022576792 A JP2022576792 A JP 2022576792A JP 2022576792 A JP2022576792 A JP 2022576792A JP 2023530112 A JP2023530112 A JP 2023530112A
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individual
treatment
weeks
increase
pharmaceutical composition
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JP2023530112A5 (enExample
Inventor
パトリック・フィン
アラア・ハメド
クリスティナ・アン・ハーク
キャサリン・ウィルソン
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Genzyme Corp
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Genzyme Corp
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Priority to JP2026060810A priority Critical patent/JP2026117025A/ja
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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • 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
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022576792A 2020-06-14 2021-06-11 遅発型ポンペ病を処置するための組成物及び方法 Pending JP2023530112A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026060810A JP2026117025A (ja) 2020-06-14 2026-04-02 遅発型ポンペ病を処置するための組成物及び方法

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063038857P 2020-06-14 2020-06-14
US63/038,857 2020-06-14
US202063076037P 2020-09-09 2020-09-09
US63/076,037 2020-09-09
US202163147629P 2021-02-09 2021-02-09
US63/147,629 2021-02-09
US202163157490P 2021-03-05 2021-03-05
US63/157,490 2021-03-05
PCT/US2021/037111 WO2021257409A1 (en) 2020-06-14 2021-06-11 Compositions and methods for treating late-onset pompe disease

Related Child Applications (1)

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JP2026060810A Division JP2026117025A (ja) 2020-06-14 2026-04-02 遅発型ポンペ病を処置するための組成物及び方法

Publications (2)

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JP2023530112A true JP2023530112A (ja) 2023-07-13
JP2023530112A5 JP2023530112A5 (enExample) 2024-06-18

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JP2022576792A Pending JP2023530112A (ja) 2020-06-14 2021-06-11 遅発型ポンペ病を処置するための組成物及び方法
JP2026060810A Pending JP2026117025A (ja) 2020-06-14 2026-04-02 遅発型ポンペ病を処置するための組成物及び方法

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US (2) US12533396B2 (enExample)
EP (1) EP4164678A1 (enExample)
JP (2) JP2023530112A (enExample)
KR (1) KR20230025874A (enExample)
CN (1) CN115916245A (enExample)
AU (1) AU2021293021A1 (enExample)
BR (1) BR112022025314A2 (enExample)
CA (1) CA3182409A1 (enExample)
IL (1) IL299015A (enExample)
MX (1) MX2022016019A (enExample)
TW (1) TW202214302A (enExample)
WO (1) WO2021257409A1 (enExample)
ZA (1) ZA202300544B (enExample)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012512313A (ja) * 2008-12-16 2012-05-31 ジェンザイム・コーポレーション オリゴ糖−タンパク複合体

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
JP5137814B2 (ja) 2005-04-06 2013-02-06 ジェンザイム・コーポレーション 治療ターゲティングのための酸不安定リンカーを介するｐｅｇおよびポリシアルリソソーム酵素のコンジュゲート
CN102978185B (zh) 2007-01-18 2015-02-11 建新公司 包含氨基氧基基团的寡糖及其轭合物
US8883979B2 (en) 2012-08-31 2014-11-11 Bayer Healthcare Llc Anti-prolactin receptor antibody formulations
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
EP4100120A1 (en) 2020-02-08 2022-12-14 Genzyme Corporation Compositions and methods for treating pompe disease

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012512313A (ja) * 2008-12-16 2012-05-31 ジェンザイム・コーポレーション オリゴ糖−タンパク複合体

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF INBORN ERRORS OF METABOLISM & SCREENING, vol. 5, JPN6025019540, 2017, pages 355 - 356, ISSN: 0005597288 *
JOURNAL OF INHERITED METABOLIC DISEASE, 2018年, vol. Vol. 41, Suppl. 1, JPN6025019542, pages 187, ISSN: 0005902765 *
JOURNAL OF NEUROLOGY, vol. 259, no. 5, JPN6025019545, 2011, pages 952 - 958, ISSN: 0005902767 *
NEUROLOGY, vol. Vol. 94, No. 15_supplement, JPN6025019544, 14 April 2020 (2020-04-14), ISSN: 0005902766 *
PLOS ONE, vol. 13, no. 1, JPN6025019541, 2018, pages 1 - 15, ISSN: 0005597289 *

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Publication number Publication date
WO2021257409A1 (en) 2021-12-23
US12533396B2 (en) 2026-01-27
US20230256062A1 (en) 2023-08-17
EP4164678A1 (en) 2023-04-19
JP2026117025A (ja) 2026-07-14
IL299015A (en) 2023-02-01
KR20230025874A (ko) 2023-02-23
BR112022025314A2 (pt) 2023-01-03
AU2021293021A1 (en) 2023-02-23
MX2022016019A (es) 2023-04-10
TW202214302A (zh) 2022-04-16
CA3182409A1 (en) 2021-12-23
CN115916245A (zh) 2023-04-04
US20260216302A1 (en) 2026-07-30
ZA202300544B (en) 2026-02-25

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