AR119552A2 - Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos - Google Patents

Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos

Info

Publication number
AR119552A2
AR119552A2 ARP200102165A ARP200102165A AR119552A2 AR 119552 A2 AR119552 A2 AR 119552A2 AR P200102165 A ARP200102165 A AR P200102165A AR P200102165 A ARP200102165 A AR P200102165A AR 119552 A2 AR119552 A2 AR 119552A2
Authority
AR
Argentina
Prior art keywords
acetygalactosamine
sulfatase
production
active human
highly phosphorylated
Prior art date
Application number
ARP200102165A
Other languages
English (en)
Inventor
Michel Claude Vellard
Augustus O Okhamafe
Kidisti Araya
Vish Koppaka
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 AR119552A2 publication Critical patent/AR119552A2/es

Links

Classifications

    • 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)
    • 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)
    • 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

Landscapes

  • 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

La presente proporciona composiciones de la N-aceltigalactosamina-6-sulfatasa (GALNS) humana activa altamente fosforilada y las composiciones y formulaciones farmacéuticas que la incluyen, los métodos para la producción y purificación de GALNS y su uso en el diagnóstico, profilaxis o tratamiento de enfermedades y condiciones, lo que en particular incluye las enfermedades por almacenamiento lisosomal que son ocasionadas por o están asociadas con una deficiencia en la enzima GALNS, por ejemplo, la Mucopolisacaridosis IVa (MPS IVa o síndrome de Morquio A).
ARP200102165A 2010-07-22 2020-07-31 Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos AR119552A2 (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
AR119552A2 true AR119552A2 (es) 2021-12-29

Family

ID=44543776

Family Applications (2)

Application Number Title Priority Date Filing Date
ARP110102649A AR082319A1 (es) 2010-07-22 2011-07-21 Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
ARP200102165A AR119552A2 (es) 2010-07-22 2020-07-31 Producción de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos

Family Applications Before (1)

Application Number Title Priority Date Filing Date
ARP110102649A AR082319A1 (es) 2010-07-22 2011-07-21 Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos

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
KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
US11529397B2 (en) 2018-01-22 2022-12-20 Saint Louis University Method of treating mucopolysaccharidosis type IVA
AU2014243749A1 (en) 2013-03-13 2015-08-06 Shire Human Genetic Therapies, Inc. Method of characterizing lysosomal enzymes
WO2014143622A1 (en) * 2013-03-13 2014-09-18 Seattle Genetics, Inc. ACTIVATED CARBON FILTRATION FOR PURIFICATION OF BENZODIAZEPINE ADCs
WO2014201220A1 (en) * 2013-06-12 2014-12-18 The Board Of Trustees Of The University Of Illinois Methods for detecting prostate cancer
EP3019521A1 (en) 2013-07-12 2016-05-18 EMD Millipore Corporation A method of determining virus removal from a sample containing a target protein using activated carbon
US20170191041A1 (en) * 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes
US10472615B2 (en) 2016-01-21 2019-11-12 Saint Louis University Reduced immunogenic proteins for lysosomal storage disorders
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
RU2763990C2 (ru) * 2020-02-19 2022-01-12 Акционерное общество "ГЕНЕРИУМ" Клетка, продуцирующая с высокой эффективностью активный белок арилсульфатазу в, и способ получения этой клетки
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
TW202403043A (zh) * 2022-03-18 2024-01-16 美商健臻公司 重組人類酸性鞘磷脂酶醫藥組合物及方法

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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
PE20171798A1 (es) 2017-12-28
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
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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