CN110381987A - 使用稳定化α-半乳糖苷酶治疗法布里病的治疗方法 - Google Patents

使用稳定化α-半乳糖苷酶治疗法布里病的治疗方法 Download PDF

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Publication number
CN110381987A
CN110381987A CN201880016068.5A CN201880016068A CN110381987A CN 110381987 A CN110381987 A CN 110381987A CN 201880016068 A CN201880016068 A CN 201880016068A CN 110381987 A CN110381987 A CN 110381987A
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China
Prior art keywords
galactosidase
human alpha
recombinant human
alpha
weeks
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Pending
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CN201880016068.5A
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English (en)
Chinese (zh)
Inventor
埃纳特·阿尔蒙
罗尔·切尔特科夫
萨里·阿隆
尤瑟夫·雪提尔
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Protalix Ltd
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Protalix Ltd
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Priority to CN202410140178.XA priority Critical patent/CN117959455A/zh
Publication of CN110381987A publication Critical patent/CN110381987A/zh
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-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)
  • Public Health (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
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  • 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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Vascular Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
CN201880016068.5A 2017-01-05 2018-01-05 使用稳定化α-半乳糖苷酶治疗法布里病的治疗方法 Pending CN110381987A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410140178.XA CN117959455A (zh) 2017-01-05 2018-01-05 植物重组人类α-半乳糖苷酶制备治疗法布里病的药品的用途

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

Related Child Applications (1)

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CN202410140178.XA Division CN117959455A (zh) 2017-01-05 2018-01-05 植物重组人类α-半乳糖苷酶制备治疗法布里病的药品的用途

Publications (1)

Publication Number Publication Date
CN110381987A true CN110381987A (zh) 2019-10-25

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CN201880016068.5A Pending CN110381987A (zh) 2017-01-05 2018-01-05 使用稳定化α-半乳糖苷酶治疗法布里病的治疗方法
CN202410140178.XA Pending CN117959455A (zh) 2017-01-05 2018-01-05 植物重组人类α-半乳糖苷酶制备治疗法布里病的药品的用途

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US (2) US12194079B2 (enExample)
EP (1) EP3565583A4 (enExample)
JP (2) JP2020504131A (enExample)
KR (2) KR20190103320A (enExample)
CN (2) CN110381987A (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
CN114423427A (zh) * 2019-06-11 2022-04-29 阿米库斯治疗学公司 治疗具有肾损害的患者的法布里病的方法

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
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
CN116419760A (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肾病中的用途

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130295065A1 (en) * 2011-01-20 2013-11-07 Protalix Ltd. Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
US20150320844A1 (en) * 2005-04-06 2015-11-12 Genzyme Corporation Targeting of glycoprotein therapeutics
US20160053247A1 (en) * 2009-11-17 2016-02-25 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
US20160184409A1 (en) * 2002-04-25 2016-06-30 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
US20160324839A1 (en) * 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8742079B2 (en) 2007-08-20 2014-06-03 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
US20120230974A1 (en) 2009-11-17 2012-09-13 Protalix Ltd Alkaline alpha galactosidase for the treatment of fabry disease
HUE030959T2 (en) 2010-03-02 2017-06-28 Protalix Ltd Stabilized alpha-galactosidase and its uses

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160184409A1 (en) * 2002-04-25 2016-06-30 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
US20150320844A1 (en) * 2005-04-06 2015-11-12 Genzyme Corporation Targeting of glycoprotein therapeutics
US20160324839A1 (en) * 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones
US20160053247A1 (en) * 2009-11-17 2016-02-25 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
US20130295065A1 (en) * 2011-01-20 2013-11-07 Protalix Ltd. Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114423427A (zh) * 2019-06-11 2022-04-29 阿米库斯治疗学公司 治疗具有肾损害的患者的法布里病的方法

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Publication number Publication date
NZ755725A (en) 2023-06-30
US20250134968A1 (en) 2025-05-01
CA3048151A1 (en) 2018-07-12
US12194079B2 (en) 2025-01-14
WO2018127920A1 (en) 2018-07-12
RU2019124280A (ru) 2021-02-06
CL2019001867A1 (es) 2019-10-04
KR20190103320A (ko) 2019-09-04
AU2018205891B2 (en) 2024-11-07
CN117959455A (zh) 2024-05-03
JP2020504131A (ja) 2020-02-06
JP2023022244A (ja) 2023-02-14
MX2019008076A (es) 2019-08-29
EP3565583A1 (en) 2019-11-13
EP3565583A4 (en) 2020-12-02
RU2019124280A3 (enExample) 2021-09-14
KR20240042110A (ko) 2024-04-01
IL267863A (en) 2019-09-26
US20200155654A1 (en) 2020-05-21
BR112019013920A2 (pt) 2020-02-04
AU2018205891A1 (en) 2019-08-15
ZA201904850B (en) 2022-04-28

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