TWI642782B - 重組醣蛋白及其用途 - Google Patents

重組醣蛋白及其用途 Download PDF

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Publication number
TWI642782B
TWI642782B TW103136272A TW103136272A TWI642782B TW I642782 B TWI642782 B TW I642782B TW 103136272 A TW103136272 A TW 103136272A TW 103136272 A TW103136272 A TW 103136272A TW I642782 B TWI642782 B TW I642782B
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TW
Taiwan
Prior art keywords
protein
oligosaccharides
linked
percentage
total nitrogen
Prior art date
Application number
TW103136272A
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English (en)
Chinese (zh)
Other versions
TW201610163A (zh
Inventor
凱倫 李
Karen Lee
克里斯多福 黃
Christopher HWANG
克莉絲汀 黛瑪麗亞
Christine Demaria
Original Assignee
健臻公司
Genzyme Corporation
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Publication of TW201610163A publication Critical patent/TW201610163A/zh
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Publication of TWI642782B publication Critical patent/TWI642782B/zh

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate 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
    • 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
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70592CD52
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • 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
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00Preparation of peptides or proteins
    • C12P21/005Glycopeptides, glycoproteins
    • 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)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • C12N2510/00Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Cardiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Vascular Medicine (AREA)
TW103136272A 2013-10-23 2014-10-21 重組醣蛋白及其用途 TWI642782B (zh)

Applications Claiming Priority (4)

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

Publications (2)

Publication Number Publication Date
TW201610163A TW201610163A (zh) 2016-03-16
TWI642782B true TWI642782B (zh) 2018-12-01

Family

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TW103136272A TWI642782B (zh) 2013-10-23 2014-10-21 重組醣蛋白及其用途
TW111139854A TW202332774A (zh) 2013-10-23 2014-10-21 重組醣蛋白及其用途
TW107130267A TWI793159B (zh) 2013-10-23 2014-10-21 重組醣蛋白及其用途

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TW111139854A TW202332774A (zh) 2013-10-23 2014-10-21 重組醣蛋白及其用途
TW107130267A TWI793159B (zh) 2013-10-23 2014-10-21 重組醣蛋白及其用途

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US (2) US11208644B2 (enExample)
EP (2) EP3778884A1 (enExample)
JP (2) JP6837334B2 (enExample)
KR (2) KR102298080B1 (enExample)
CN (3) CN114606217A (enExample)
AU (2) AU2014340152A1 (enExample)
BR (1) BR112016008086A2 (enExample)
CA (1) CA2927852A1 (enExample)
CL (1) CL2016000961A1 (enExample)
CR (1) CR20160225A (enExample)
DK (1) DK3060660T3 (enExample)
DO (1) DOP2016000079A (enExample)
EA (1) EA034515B1 (enExample)
ES (1) ES2882562T3 (enExample)
HR (1) HRP20211197T1 (enExample)
HU (1) HUE055236T2 (enExample)
IL (2) IL245255B (enExample)
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PH (1) PH12016500708B1 (enExample)
PL (1) PL3060660T3 (enExample)
PT (1) PT3060660T (enExample)
RS (1) RS62171B1 (enExample)
SG (2) SG10201803366PA (enExample)
SI (1) SI3060660T1 (enExample)
TN (1) TN2016000140A1 (enExample)
TW (3) TWI642782B (enExample)
UA (1) UA119857C2 (enExample)
UY (1) UY35794A (enExample)
WO (1) WO2015061464A2 (enExample)

Families Citing this family (5)

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GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3896164A1 (en) * 2016-05-18 2021-10-20 ModernaTX, Inc. Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
EP4667565A2 (en) 2016-10-20 2025-12-24 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
TW201829770A (zh) 2017-01-10 2018-08-16 美商阿米庫斯醫療股份有限公司 用於治療fabry氏病之重組α-半乳糖苷酶A
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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US20030152560A1 (en) * 1996-09-13 2003-08-14 Transkaryotic Therapies Inc., A Massachusetts Corporation Nucleic acid encoding a chimeric polypeptide

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US5356804A (en) * 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US5401650A (en) * 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
DK2374876T3 (en) 1996-09-13 2016-05-30 Shire Human Genetic Therapies A process for the purification of alpha-galactosidase A
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AU2011356137A1 (en) * 2011-01-20 2013-08-15 Protalix Ltd. Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells
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Publication number Publication date
US11208644B2 (en) 2021-12-28
KR20210111880A (ko) 2021-09-13
US20220064617A1 (en) 2022-03-03
IL245255A0 (en) 2016-06-30
JP2016538840A (ja) 2016-12-15
IL245255B (en) 2020-02-27
WO2015061464A3 (en) 2015-09-03
KR20160066550A (ko) 2016-06-10
AU2014340152A1 (en) 2016-06-09
ES2882562T3 (es) 2021-12-02
EP3778884A1 (en) 2021-02-17
RS62171B1 (sr) 2021-08-31
EA201690826A1 (ru) 2016-08-31
CA2927852A1 (en) 2015-04-30
LT3060660T (lt) 2021-07-26
JP2020089379A (ja) 2020-06-11
CN112852784A (zh) 2021-05-28
SG11201602932WA (en) 2016-05-30
IL272609A (en) 2020-03-31
EA034515B1 (ru) 2020-02-14
NZ720384A (en) 2022-04-29
DK3060660T3 (da) 2021-09-13
EP3060660A2 (en) 2016-08-31
CN114606217A (zh) 2022-06-10
KR102479886B1 (ko) 2022-12-20
UA119857C2 (uk) 2019-08-27
MX392532B (es) 2025-03-24
UY35794A (es) 2015-05-29
SI3060660T1 (sl) 2021-09-30
CN105916984A (zh) 2016-08-31
MY197247A (en) 2023-06-08
JP7296327B2 (ja) 2023-06-22
PH12016500708A1 (en) 2016-05-30
HRP20211197T1 (hr) 2021-11-12
BR112016008086A2 (pt) 2017-10-03
JP6837334B2 (ja) 2021-03-03
TN2016000140A1 (en) 2017-10-06
HUE055236T2 (hu) 2021-11-29
PH12016500708B1 (en) 2023-03-01
CR20160225A (es) 2016-06-24
EP3060660B1 (en) 2021-06-23
MX2016005321A (es) 2017-01-05
DOP2016000079A (es) 2016-05-30
PL3060660T3 (pl) 2021-12-06
TWI793159B (zh) 2023-02-21
SG10201803366PA (en) 2018-05-30
PT3060660T (pt) 2021-08-06
MY178632A (en) 2020-10-19
CL2016000961A1 (es) 2017-02-03
IL272609B (en) 2022-03-01
TW201610163A (zh) 2016-03-16
TW201900873A (zh) 2019-01-01
US20160264953A1 (en) 2016-09-15
TW202332774A (zh) 2023-08-16
WO2015061464A2 (en) 2015-04-30
PE20160723A1 (es) 2016-08-01
KR102298080B1 (ko) 2021-09-06
AU2020294173A1 (en) 2021-02-11

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