CO2017002776A2 - Composición de alfa glucosidasa humana recombinante y métodos para su fabricación - Google Patents

Composición de alfa glucosidasa humana recombinante y métodos para su fabricación

Info

Publication number
CO2017002776A2
CO2017002776A2 CONC2017/0002776A CO2017002776A CO2017002776A2 CO 2017002776 A2 CO2017002776 A2 CO 2017002776A2 CO 2017002776 A CO2017002776 A CO 2017002776A CO 2017002776 A2 CO2017002776 A2 CO 2017002776A2
Authority
CO
Colombia
Prior art keywords
methods
recombinant human
composition
human alpha
alpha glucosidase
Prior art date
Application number
CONC2017/0002776A
Other languages
English (en)
Inventor
Russell Gotschall
Hung Do
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CO2017002776A2 publication Critical patent/CO2017002776A2/es

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Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • 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
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • 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
    • C12N2510/02—Cells for production

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Composición de alfa glucosidasa humana recombinante (rhGAA) derivada de células CHO que contiene una composición de glicano más optimizada que consiste en una cantidad de rhGAA que contiene N-glicanos que llevan manosa-6-fosfato (M6P) o bis-M6P mayor que las rhGAAs convencionales, junto con una baja cantidad de glicanos de alto contenido en manosa no fosforilados y una baja cantidad de galactosa terminal en oligosacáridos complejos. Se describen composiciones que contienen la rhGAA y métodos de uso.
CONC2017/0002776A 2014-09-30 2017-03-23 Composición de alfa glucosidasa humana recombinante y métodos para su fabricación CO2017002776A2 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462057842P 2014-09-30 2014-09-30
US201462057847P 2014-09-30 2014-09-30
US201562112463P 2015-02-05 2015-02-05
US201562135345P 2015-03-19 2015-03-19
PCT/US2015/053252 WO2016054231A1 (en) 2014-09-30 2015-09-30 Highly potent acid alpha-glucosidase with enhanced carbohydrates

Publications (1)

Publication Number Publication Date
CO2017002776A2 true CO2017002776A2 (es) 2017-08-10

Family

ID=55631441

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2017/0002776A CO2017002776A2 (es) 2014-09-30 2017-03-23 Composición de alfa glucosidasa humana recombinante y métodos para su fabricación

Country Status (36)

Country Link
US (5) US10208299B2 (es)
EP (2) EP4273241A3 (es)
JP (3) JP6851964B2 (es)
KR (3) KR20220146662A (es)
CN (2) CN107075468B (es)
AU (2) AU2015325028B2 (es)
BR (1) BR112017005810A2 (es)
CA (2) CA3228032A1 (es)
CL (1) CL2017000722A1 (es)
CO (1) CO2017002776A2 (es)
CR (1) CR20170107A (es)
DK (1) DK3201320T3 (es)
DO (1) DOP2017000079A (es)
EA (1) EA038986B1 (es)
EC (1) ECSP17019103A (es)
ES (1) ES2967620T3 (es)
FI (2) FI3201320T3 (es)
FR (1) FR24C1012I1 (es)
HR (1) HRP20240041T1 (es)
HU (2) HUE064629T2 (es)
IL (4) IL307587A (es)
LT (1) LT3201320T (es)
MX (2) MX2017003989A (es)
MY (1) MY186336A (es)
NI (1) NI201700039A (es)
PE (1) PE20170906A1 (es)
PH (1) PH12017500455A1 (es)
PL (1) PL3201320T3 (es)
PT (1) PT3201320T (es)
RS (1) RS65066B1 (es)
SG (2) SG11201702114TA (es)
SI (1) SI3201320T1 (es)
SV (1) SV2017005416A (es)
TN (1) TN2017000082A1 (es)
WO (1) WO2016054231A1 (es)
ZA (2) ZA201701945B (es)

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CN107075468B (zh) 2021-12-24
AU2015325028A1 (en) 2017-03-16
CA2961762A1 (en) 2016-04-07
CL2017000722A1 (es) 2017-12-15
IL277529A (en) 2020-11-30
EA201790724A1 (ru) 2017-08-31
EA038986B1 (ru) 2021-11-18
ES2967620T3 (es) 2024-05-03
KR20170063837A (ko) 2017-06-08
US11591583B2 (en) 2023-02-28
IL251152B (en) 2021-06-30
JP2021011489A (ja) 2021-02-04
US20220162578A1 (en) 2022-05-26
US20210261935A1 (en) 2021-08-26
MX2017003989A (es) 2017-07-07
JP2023078118A (ja) 2023-06-06
JP6851964B2 (ja) 2021-03-31
JP7225176B2 (ja) 2023-02-20
EP4273241A2 (en) 2023-11-08
LT3201320T (lt) 2024-01-25
HRP20240041T1 (hr) 2024-03-29
BR112017005810A2 (pt) 2018-02-27
FR24C1012I1 (fr) 2024-05-24
AU2022203498A1 (en) 2022-06-09
CN114540327A (zh) 2022-05-27
KR20210118977A (ko) 2021-10-01
SI3201320T1 (sl) 2024-02-29
PL3201320T3 (pl) 2024-03-18
US20170298335A1 (en) 2017-10-19
RS65066B1 (sr) 2024-02-29
EP3201320B1 (en) 2023-10-11
US20190276812A1 (en) 2019-09-12
CR20170107A (es) 2017-09-01
PT3201320T (pt) 2024-01-12
IL251152A0 (en) 2017-04-30
ZA201901690B (en) 2022-06-29
US10208299B2 (en) 2019-02-19
SV2017005416A (es) 2017-08-08
TN2017000082A1 (en) 2018-07-04
HUE064629T2 (hu) 2024-04-28
AU2015325028B2 (en) 2022-02-24
NI201700039A (es) 2018-01-05
FI3201320T3 (fi) 2024-01-08
US20230203465A1 (en) 2023-06-29
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