PE20171798A1 - Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada - Google Patents

Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada

Info

Publication number
PE20171798A1
PE20171798A1 PE2017001625A PE2017001625A PE20171798A1 PE 20171798 A1 PE20171798 A1 PE 20171798A1 PE 2017001625 A PE2017001625 A PE 2017001625A PE 2017001625 A PE2017001625 A PE 2017001625A PE 20171798 A1 PE20171798 A1 PE 20171798A1
Authority
PE
Peru
Prior art keywords
sulfatase
acetylgalactosamine
galns
enzyme
highly active
Prior art date
Application number
PE2017001625A
Other languages
English (en)
Inventor
Vish Koppaka
Michel Claude Vellard
Augustus O Okhamafe
Kidisti Araya
Original Assignee
Biomarin Pharm 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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of PE20171798A1 publication Critical patent/PE20171798A1/es

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    • 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)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • 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)
    • 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)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K1/00General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14Extraction; Separation; Purification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06004N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Analytical Chemistry (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Se refiere a una formulacion que comprende una enzima N-acetilgalactosamina-6-sulfatasa (GALNS) humana recombinante purificada, buffer fosfato para reducir la desfosforilacion de la enzima y uno o mas agentes estabilizadores seleccionados de sales de aminoacidos, buffers de aminoacido, surfactantes y polioles, donde la formulacion tiene pH 5.0-5.8. Dicha GALNS es util en el diagnostico, profilaxis o tratamiento de enfermedades y condiciones, tales como las enfermedades por almacenamiento lisosomal que son ocasionadas por o estan asociadas con una deficiencia en la enzima GALNS, por ejemplo, la Mucopolisacaridosis IVa (MPS IVa o sindrome de Morquio A)
PE2017001625A 2010-07-22 2011-07-22 Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada PE20171798A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US36671410P 2010-07-22 2010-07-22

Publications (1)

Publication Number Publication Date
PE20171798A1 true PE20171798A1 (es) 2017-12-28

Family

ID=44543776

Family Applications (3)

Application Number Title Priority Date Filing Date
PE2017001625A PE20171798A1 (es) 2010-07-22 2011-07-22 Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada
PE2013000110A PE20131140A1 (es) 2010-07-22 2011-07-22 Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
PE2017001626A PE20171799A1 (es) 2010-07-22 2011-07-22 Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada

Family Applications After (2)

Application Number Title Priority Date Filing Date
PE2013000110A PE20131140A1 (es) 2010-07-22 2011-07-22 Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
PE2017001626A PE20171799A1 (es) 2010-07-22 2011-07-22 Formulacion que comprende n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada

Country Status (28)

Country Link
US (3) US8765437B2 (es)
EP (1) EP2595650B1 (es)
JP (2) JP6030553B2 (es)
KR (2) KR101598897B1 (es)
CN (1) CN103037895B (es)
AR (2) AR082319A1 (es)
AU (1) AU2011280937B2 (es)
BR (1) BR112013001558B1 (es)
CA (2) CA3146151A1 (es)
CL (2) CL2013000219A1 (es)
CY (1) CY1118934T1 (es)
DK (1) DK2595650T3 (es)
ES (1) ES2616263T3 (es)
HR (1) HRP20170245T1 (es)
HU (1) HUE033163T2 (es)
IL (2) IL224125B (es)
LT (1) LT2595650T (es)
MX (1) MX341838B (es)
PE (3) PE20171798A1 (es)
PL (1) PL2595650T3 (es)
PT (1) PT2595650T (es)
RS (1) RS55669B1 (es)
RU (1) RU2607376C2 (es)
SG (1) SG187580A1 (es)
SI (1) SI2595650T1 (es)
SM (1) SMT201700106B (es)
WO (1) WO2012012718A2 (es)
ZA (1) ZA201300286B (es)

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HUE032074T2 (en) * 2008-01-18 2017-08-28 Biomarin Pharm Inc Preparation and use of active, highly phosphorylated, human lisosomal sulfatase enzymes
US8956859B1 (en) 2010-08-13 2015-02-17 Aviex Technologies Llc Compositions and methods for determining successful immunization by one or more vaccines
WO2013119715A1 (en) * 2012-02-07 2013-08-15 Saint Louis University Determination of immunogenic peptides in lysosomal enzymes and induction of oral tolerance
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US11529397B2 (en) 2018-01-22 2022-12-20 Saint Louis University Method of treating mucopolysaccharidosis type IVA
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US20170191041A1 (en) * 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes
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WO2017189432A1 (en) 2016-04-26 2017-11-02 R.P. Scherer Technologies, Llc Antibody conjugates and methods of making and using the same
WO2019045149A1 (en) * 2017-08-31 2019-03-07 Green Cross Corporation PROCESS FOR PURIFYING SULFATASE PROTEIN
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WO2021216460A1 (en) * 2020-04-19 2021-10-28 Figene, Llc Gene modified fibroblasts for therapeutic applications
WO2023150387A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising acid alpha glucosidase and methods of use thereof
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Also Published As

Publication number Publication date
RU2607376C2 (ru) 2017-01-10
HUE033163T2 (en) 2017-11-28
US20120189605A1 (en) 2012-07-26
BR112013001558B1 (pt) 2022-08-16
KR20150038715A (ko) 2015-04-08
RU2013107765A (ru) 2014-08-27
CA2805673C (en) 2022-04-05
WO2012012718A2 (en) 2012-01-26
CL2013000219A1 (es) 2014-07-11
IL260282B (en) 2020-05-31
KR101949906B1 (ko) 2019-04-22
ZA201300286B (en) 2014-03-26
PE20171799A1 (es) 2017-12-28
CA3146151A1 (en) 2012-01-26
RS55669B1 (sr) 2017-06-30
EP2595650A2 (en) 2013-05-29
SMT201700106B (it) 2017-03-08
BR112013001558A2 (pt) 2020-08-04
MX341838B (es) 2016-09-05
US8765437B2 (en) 2014-07-01
SG187580A1 (en) 2013-03-28
AU2011280937B2 (en) 2016-06-09
KR101598897B1 (ko) 2016-03-03
AU2011280937A1 (en) 2013-01-31
DK2595650T3 (en) 2017-02-27
JP2013532986A (ja) 2013-08-22
AR082319A1 (es) 2012-11-28
US20140341878A1 (en) 2014-11-20
ES2616263T3 (es) 2017-06-12
CA2805673A1 (en) 2012-01-26
US8940513B2 (en) 2015-01-27
CN103037895B (zh) 2016-11-09
US9567572B2 (en) 2017-02-14
JP6030553B2 (ja) 2016-11-24
CN103037895A (zh) 2013-04-10
WO2012012718A3 (en) 2012-04-05
PT2595650T (pt) 2017-02-22
EP2595650B1 (en) 2016-11-16
KR20130105605A (ko) 2013-09-25
IL260282A (en) 2018-07-31
IL224125B (en) 2019-02-28
AR119552A2 (es) 2021-12-29
CL2017000498A1 (es) 2017-11-24
CY1118934T1 (el) 2018-01-10
LT2595650T (lt) 2017-03-10
PE20131140A1 (es) 2013-10-17
HRP20170245T1 (hr) 2017-04-21
MX2013000908A (es) 2013-07-05
JP2016178950A (ja) 2016-10-13
US20160186149A1 (en) 2016-06-30
SI2595650T1 (sl) 2017-04-26
PL2595650T3 (pl) 2017-06-30

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