AU2018205891B2 - Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase - Google Patents

Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase Download PDF

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Publication number
AU2018205891B2
AU2018205891B2 AU2018205891A AU2018205891A AU2018205891B2 AU 2018205891 B2 AU2018205891 B2 AU 2018205891B2 AU 2018205891 A AU2018205891 A AU 2018205891A AU 2018205891 A AU2018205891 A AU 2018205891A AU 2018205891 B2 AU2018205891 B2 AU 2018205891B2
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Prior art keywords
galactosidase
leu
weeks
alfa
recombinant human
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AU2018205891A
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AU2018205891A1 (en
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Einat Almon
Sari Alon
Raul CHERTKOFF
Yoseph Shaaltiel
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Protalix Ltd
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Protalix Ltd
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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
    • A61K47/50—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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • 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)
AU2018205891A 2017-01-05 2018-01-05 Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase Active AU2018205891B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2018205891A1 AU2018205891A1 (en) 2019-08-15
AU2018205891B2 true AU2018205891B2 (en) 2024-11-07

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AU2018205891A Active AU2018205891B2 (en) 2017-01-05 2018-01-05 Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase

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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160053247A1 (en) * 2009-11-17 2016-02-25 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof

Family Cites Families (10)

* 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160053247A1 (en) * 2009-11-17 2016-02-25 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF INHERITED METABOLIC DISEASE, vol. 39, no. 1, 12 August 2016, pages S35 - S284, Abstract number O-053. *

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
KR20190103320A (ko) 2019-09-04
US12194079B2 (en) 2025-01-14
MX2019008076A (es) 2019-08-29
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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