JP2020504131A - 安定化α−ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン - Google Patents

安定化α−ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン Download PDF

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JP2020504131A
JP2020504131A JP2019536488A JP2019536488A JP2020504131A JP 2020504131 A JP2020504131 A JP 2020504131A JP 2019536488 A JP2019536488 A JP 2019536488A JP 2019536488 A JP2019536488 A JP 2019536488A JP 2020504131 A JP2020504131 A JP 2020504131A
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galactosidase
human
recombinant human
weeks
administration
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JP2020504131A5 (enExample
Inventor
エイナット アルモン
エイナット アルモン
ラウル チェルトコフ
ラウル チェルトコフ
サリ アロン
サリ アロン
ヨセフ シャールティエル
ヨセフ シャールティエル
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プロタリクス リミテッド
プロタリクス リミテッド
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Publication of JP2020504131A publication Critical patent/JP2020504131A/ja
Publication of JP2020504131A5 publication Critical patent/JP2020504131A5/ja
Priority to JP2022192939A priority Critical patent/JP2023022244A/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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Vascular Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
JP2019536488A 2017-01-05 2018-01-05 安定化α−ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン Pending JP2020504131A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022192939A JP2023022244A (ja) 2017-01-05 2022-12-01 安定化α－ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762442537P 2017-01-05 2017-01-05
US62/442,537 2017-01-05
PCT/IL2018/050018 WO2018127920A1 (en) 2017-01-05 2018-01-05 Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase

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JP2022192939A Division JP2023022244A (ja) 2017-01-05 2022-12-01 安定化α－ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン

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JP2020504131A true JP2020504131A (ja) 2020-02-06
JP2020504131A5 JP2020504131A5 (enExample) 2021-02-12

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JP2019536488A Pending JP2020504131A (ja) 2017-01-05 2018-01-05 安定化α−ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン
JP2022192939A Pending JP2023022244A (ja) 2017-01-05 2022-12-01 安定化α－ガラクトシダーゼを用いた、ファブリー病の治療のための治療レジメン

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US (2) US12194079B2 (enExample)
EP (1) EP3565583A4 (enExample)
JP (2) JP2020504131A (enExample)
KR (2) KR20240042110A (enExample)
CN (2) CN117959455A (enExample)
AU (1) AU2018205891B2 (enExample)
BR (1) BR112019013920A2 (enExample)
CA (1) CA3048151A1 (enExample)
CL (1) CL2019001867A1 (enExample)
IL (1) IL267863A (enExample)
MX (1) MX2019008076A (enExample)
NZ (1) NZ755725A (enExample)
WO (1) WO2018127920A1 (enExample)
ZA (1) ZA201904850B (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023549323A (ja) * 2020-11-13 2023-11-24 ハンミ ファーマシューティカル カンパニー リミテッド 治療学的酵素融合タンパク質のファブリー病に起因する又は伴う腎臓疾患の予防及び治療用途

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12020820B1 (en) * 2017-03-03 2024-06-25 Cerner Innovation, Inc. Predicting sphingolipidoses (fabry's disease) and decision support
CA3141226A1 (en) * 2019-06-11 2020-12-17 Franklin Johnson Methods of treating fabry disease in patients having renal impairment
CN116419759A (zh) * 2020-11-13 2023-07-11 韩美药品株式会社 治疗酶融合蛋白在预防和治疗由法布里病引起或伴有法布里病的神经病中的用途

Citations (1)

* Cited by examiner, † Cited by third party
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JP2013520986A (ja) * 2010-03-02 2013-06-10 プロタリクス リミテッド 安定化α−ガラクトシダーゼおよびその使用

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US5766897A (en) 1990-06-21 1998-06-16 Incyte Pharmaceuticals, Inc. Cysteine-pegylated proteins
CA2052728A1 (en) 1991-10-03 1993-04-04 Guy Ampleman Glycidyl azide polymer
DK1503788T3 (da) * 2002-04-25 2011-10-17 Shire Human Genetic Therapies Behandling af alpha-galactosidase A-deficiens
WO2006108052A2 (en) * 2005-04-06 2006-10-12 Genzyme Corporation Peg and polysialic lysosomal enzyme conjugates via acid labile linkers for therapeutic targeting
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AU2008290217B2 (en) 2007-08-20 2013-09-05 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
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US9194011B2 (en) * 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
BR112013018516B1 (pt) 2011-01-20 2023-11-07 Protalix Ltd Construto de expressão de ácido nucleico, método de produção de uma proteína alfa-galactosidase humana recombinante, proteína alfagalactosidase humana, composição farmacêutica, e, uso de uma proteína alfagalactosidase humana ou de uma composição
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease

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Publication number Priority date Publication date Assignee Title
JP2023549323A (ja) * 2020-11-13 2023-11-24 ハンミ ファーマシューティカル カンパニー リミテッド 治療学的酵素融合タンパク質のファブリー病に起因する又は伴う腎臓疾患の予防及び治療用途

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Publication number Publication date
BR112019013920A2 (pt) 2020-02-04
ZA201904850B (en) 2022-04-28
CN110381987A (zh) 2019-10-25
US20200155654A1 (en) 2020-05-21
RU2019124280A (ru) 2021-02-06
MX2019008076A (es) 2019-08-29
NZ755725A (en) 2023-06-30
EP3565583A4 (en) 2020-12-02
JP2023022244A (ja) 2023-02-14
KR20240042110A (ko) 2024-04-01
CN117959455A (zh) 2024-05-03
AU2018205891B2 (en) 2024-11-07
AU2018205891A1 (en) 2019-08-15
KR20190103320A (ko) 2019-09-04
US12194079B2 (en) 2025-01-14
US20250134968A1 (en) 2025-05-01
WO2018127920A1 (en) 2018-07-12
EP3565583A1 (en) 2019-11-13
CA3048151A1 (en) 2018-07-12
RU2019124280A3 (enExample) 2021-09-14
CL2019001867A1 (es) 2019-10-04
IL267863A (en) 2019-09-26

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