CA3048151A1 - 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 PDFInfo
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- CA3048151A1 CA3048151A1 CA3048151A CA3048151A CA3048151A1 CA 3048151 A1 CA3048151 A1 CA 3048151A1 CA 3048151 A CA3048151 A CA 3048151A CA 3048151 A CA3048151 A CA 3048151A CA 3048151 A1 CA3048151 A1 CA 3048151A1
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- Prior art keywords
- galactosidase
- alpha
- recombinant human
- weeks
- human
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Classifications
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- 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
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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
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- 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
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- 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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- 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
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- 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
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- 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
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- 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
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- 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)
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- 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)
Landscapes
- Health & Medical Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
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- Medicinal Chemistry (AREA)
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- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
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- Molecular Biology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
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- Cardiology (AREA)
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- Urology & Nephrology (AREA)
- Physics & Mathematics (AREA)
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- Biophysics (AREA)
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Applications Claiming Priority (3)
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| 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 (1)
| Publication Number | Publication Date |
|---|---|
| CA3048151A1 true CA3048151A1 (en) | 2018-07-12 |
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| Application Number | Title | Priority Date | Filing Date |
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| CA3048151A Pending CA3048151A1 (en) | 2017-01-05 | 2018-01-05 | Therapeutic regimen for the treatment of fabry using stabilized alpha-galactosidase |
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| EP (1) | EP3565583A4 (enExample) |
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| KR (2) | KR20190103320A (enExample) |
| CN (2) | CN117959455A (enExample) |
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| 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 |
| EA202290024A1 (ru) * | 2019-06-11 | 2022-03-14 | Амикус Терапьютикс, Инк. | Способы лечения болезни фабри у пациентов с почечной недостаточностью |
| US20230416714A1 (en) * | 2020-11-13 | 2023-12-28 | Hanmi Pharm. Co., Ltd | Use of therapeutic enzyme fusion protein in prevention and treatment of neuropathy caused by or accompanied by fabry disease |
| IL302858A (en) * | 2020-11-13 | 2023-07-01 | Hanmi Pharm Ind Co Ltd | Use of therapeutic enzyme fusion protein in prevention and treatment of renal diseases caused by or accompanied by fabry disease |
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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 |
| EP1503788B1 (en) | 2002-04-25 | 2011-06-29 | Shire Human Genetic Therapies, Inc. | Treatment of alpha-galactosidase a deficiency |
| 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 |
| US9999618B2 (en) | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| CA2696699A1 (en) | 2007-08-20 | 2009-02-26 | Protalix Ltd. | Saccharide-containing protein conjugates and uses thereof |
| WO2011061736A1 (en) | 2009-11-17 | 2011-05-26 | Protalix Ltd. | Alkaline alpha galactosidase for the treatment of fabry disease |
| US9194011B2 (en) | 2009-11-17 | 2015-11-24 | Protalix Ltd. | Stabilized alpha-galactosidase and uses thereof |
| CN102933707B (zh) | 2010-03-02 | 2015-09-30 | 普罗塔里克斯有限公司 | 稳定的α-半乳糖苷酶及其用途 |
| RS60129B1 (sr) | 2011-01-20 | 2020-05-29 | Protalix Ltd | Kompozicije alfa-galaktozidaze |
| 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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2018
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- 2018-01-05 JP JP2019536488A patent/JP2020504131A/ja active Pending
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2022
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Also Published As
| Publication number | Publication date |
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| CL2019001867A1 (es) | 2019-10-04 |
| JP2020504131A (ja) | 2020-02-06 |
| WO2018127920A1 (en) | 2018-07-12 |
| ZA201904850B (en) | 2022-04-28 |
| US12194079B2 (en) | 2025-01-14 |
| CN117959455A (zh) | 2024-05-03 |
| RU2019124280A3 (enExample) | 2021-09-14 |
| IL267863A (en) | 2019-09-26 |
| US20250134968A1 (en) | 2025-05-01 |
| BR112019013920A2 (pt) | 2020-02-04 |
| KR20190103320A (ko) | 2019-09-04 |
| RU2019124280A (ru) | 2021-02-06 |
| AU2018205891B2 (en) | 2024-11-07 |
| NZ755725A (en) | 2023-06-30 |
| MX2019008076A (es) | 2019-08-29 |
| CN110381987A (zh) | 2019-10-25 |
| EP3565583A1 (en) | 2019-11-13 |
| JP2023022244A (ja) | 2023-02-14 |
| AU2018205891A1 (en) | 2019-08-15 |
| KR20240042110A (ko) | 2024-04-01 |
| US20200155654A1 (en) | 2020-05-21 |
| EP3565583A4 (en) | 2020-12-02 |
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