KR20190103320A - 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법 - Google Patents

안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법 Download PDF

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KR20190103320A
KR20190103320A KR1020197023039A KR20197023039A KR20190103320A KR 20190103320 A KR20190103320 A KR 20190103320A KR 1020197023039 A KR1020197023039 A KR 1020197023039A KR 20197023039 A KR20197023039 A KR 20197023039A KR 20190103320 A KR20190103320 A KR 20190103320A
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galactosidase
recombinant human
administration
human
weeks
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에이낫 알몬
라울 체르트코프
사리 알론
요세프 샤알티엘
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프로탈릭스 리미티드
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
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    • 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
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    • A61P3/00—Drugs for disorders of the metabolism
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    • 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
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    • 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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  • 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)
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  • Heart & Thoracic Surgery (AREA)
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  • Neurology (AREA)
KR1020197023039A 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법 Ceased KR20190103320A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247008410A KR20240042110A (ko) 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법

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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KR1020247008410A Division KR20240042110A (ko) 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법

Publications (1)

Publication Number Publication Date
KR20190103320A true KR20190103320A (ko) 2019-09-04

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KR1020197023039A Ceased KR20190103320A (ko) 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법
KR1020247008410A Ceased KR20240042110A (ko) 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법

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KR1020247008410A Ceased KR20240042110A (ko) 2017-01-05 2018-01-05 안정화된 알파-갈락토시다제를 이용하는 파브리의 치료를 위한 치료적 요법

Country Status (14)

Country Link
US (2) US12194079B2 (https=)
EP (1) EP3565583A4 (https=)
JP (2) JP2020504131A (https=)
KR (2) KR20190103320A (https=)
CN (2) CN110381987A (https=)
AU (1) AU2018205891B2 (https=)
BR (1) BR112019013920A2 (https=)
CA (1) CA3048151A1 (https=)
CL (1) CL2019001867A1 (https=)
IL (1) IL267863A (https=)
MX (1) MX2019008076A (https=)
NZ (1) NZ755725A (https=)
WO (1) WO2018127920A1 (https=)
ZA (1) ZA201904850B (https=)

Families Citing this family (4)

* 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
AR120055A1 (es) * 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
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
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

Family Cites Families (11)

* 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
EP1503788B1 (en) * 2002-04-25 2011-06-29 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
EP1877099B1 (en) * 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2192924B1 (en) 2007-08-20 2017-10-11 Protalix Ltd. Saccharide-containing protein conjugates and uses thereof
US9194011B2 (en) * 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
WO2011107990A1 (en) 2010-03-02 2011-09-09 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
WO2011061736A1 (en) 2009-11-17 2011-05-26 Protalix Ltd. Alkaline alpha galactosidase for the treatment of fabry disease
CN103443270B (zh) * 2011-01-20 2017-06-06 普罗塔里克斯有限公司 用于在植物和植物细胞中表达α‑半乳糖苷酶的核酸构建体
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease

Also Published As

Publication number Publication date
EP3565583A1 (en) 2019-11-13
IL267863A (en) 2019-09-26
ZA201904850B (en) 2022-04-28
RU2019124280A (ru) 2021-02-06
CN110381987A (zh) 2019-10-25
JP2023022244A (ja) 2023-02-14
RU2019124280A3 (https=) 2021-09-14
JP2020504131A (ja) 2020-02-06
CA3048151A1 (en) 2018-07-12
US20250134968A1 (en) 2025-05-01
NZ755725A (en) 2023-06-30
US12194079B2 (en) 2025-01-14
MX2019008076A (es) 2019-08-29
AU2018205891B2 (en) 2024-11-07
CL2019001867A1 (es) 2019-10-04
EP3565583A4 (en) 2020-12-02
BR112019013920A2 (pt) 2020-02-04
KR20240042110A (ko) 2024-04-01
WO2018127920A1 (en) 2018-07-12
AU2018205891A1 (en) 2019-08-15
CN117959455A (zh) 2024-05-03
US20200155654A1 (en) 2020-05-21

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