KR102298080B1 - 재조합 당단백질 및 이의 용도 - Google Patents

재조합 당단백질 및 이의 용도 Download PDF

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KR102298080B1
KR102298080B1 KR1020167013333A KR20167013333A KR102298080B1 KR 102298080 B1 KR102298080 B1 KR 102298080B1 KR 1020167013333 A KR1020167013333 A KR 1020167013333A KR 20167013333 A KR20167013333 A KR 20167013333A KR 102298080 B1 KR102298080 B1 KR 102298080B1
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protein
oligosaccharides
galactosidase
rhaga
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KR20160066550A (ko
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카렌 리
크리스토퍼 황
크리스틴 데마리아
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젠자임 코포레이션
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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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70592—CD52
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/005—Glycopeptides, glycoproteins
    • 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)
    • 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
    • C12N2510/00—Genetically modified cells

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  • General Health & Medical Sciences (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Obesity (AREA)
KR1020167013333A 2013-10-23 2014-10-22 재조합 당단백질 및 이의 용도 Active KR102298080B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020217027690A KR102479886B1 (ko) 2013-10-23 2014-10-22 재조합 당단백질 및 이의 용도

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361894879P 2013-10-23 2013-10-23
US61/894,879 2013-10-23
US201361901942P 2013-11-08 2013-11-08
US61/901,942 2013-11-08
PCT/US2014/061789 WO2015061464A2 (en) 2013-10-23 2014-10-22 Recombinant glycoproteins and uses thereof

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KR1020217027690A Division KR102479886B1 (ko) 2013-10-23 2014-10-22 재조합 당단백질 및 이의 용도

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KR20160066550A KR20160066550A (ko) 2016-06-10
KR102298080B1 true KR102298080B1 (ko) 2021-09-06

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GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7114485B2 (ja) * 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
CN113423419A (zh) 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

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Publication number Publication date
CR20160225A (es) 2016-06-24
TW201900873A (zh) 2019-01-01
TW201610163A (zh) 2016-03-16
US11208644B2 (en) 2021-12-28
AU2020294173A1 (en) 2021-02-11
UA119857C2 (uk) 2019-08-27
JP7296327B2 (ja) 2023-06-22
AU2014340152A1 (en) 2016-06-09
TN2016000140A1 (en) 2017-10-06
SG11201602932WA (en) 2016-05-30
CN114606217A (zh) 2022-06-10
MY197247A (en) 2023-06-08
MX2016005321A (es) 2017-01-05
RS62171B1 (sr) 2021-08-31
UY35794A (es) 2015-05-29
IL272609B (en) 2022-03-01
LT3060660T (lt) 2021-07-26
IL272609A (en) 2020-03-31
CL2016000961A1 (es) 2017-02-03
KR102479886B1 (ko) 2022-12-20
SG10201803366PA (en) 2018-05-30
JP6837334B2 (ja) 2021-03-03
DOP2016000079A (es) 2016-05-30
MX392532B (es) 2025-03-24
DK3060660T3 (da) 2021-09-13
WO2015061464A2 (en) 2015-04-30
PH12016500708B1 (en) 2023-03-01
KR20160066550A (ko) 2016-06-10
EP3060660B1 (en) 2021-06-23
ES2882562T3 (es) 2021-12-02
TWI793159B (zh) 2023-02-21
TWI642782B (zh) 2018-12-01
TW202332774A (zh) 2023-08-16
JP2016538840A (ja) 2016-12-15
EP3060660A2 (en) 2016-08-31
JP2020089379A (ja) 2020-06-11
NZ720384A (en) 2022-04-29
MY178632A (en) 2020-10-19
EA201690826A1 (ru) 2016-08-31
US20220064617A1 (en) 2022-03-03
EA034515B1 (ru) 2020-02-14
EP3778884A1 (en) 2021-02-17
CA2927852A1 (en) 2015-04-30
KR20210111880A (ko) 2021-09-13
PE20160723A1 (es) 2016-08-01
PT3060660T (pt) 2021-08-06
SI3060660T1 (sl) 2021-09-30
PH12016500708A1 (en) 2016-05-30
CN112852784A (zh) 2021-05-28
HUE055236T2 (hu) 2021-11-29
US20160264953A1 (en) 2016-09-15
WO2015061464A3 (en) 2015-09-03
CN105916984A (zh) 2016-08-31
BR112016008086A2 (pt) 2017-10-03
PL3060660T3 (pl) 2021-12-06
IL245255A0 (en) 2016-06-30
IL245255B (en) 2020-02-27
HRP20211197T1 (hr) 2021-11-12

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