CY1118843T1 - Παραλλαγμενες ανασυνδυασμενες πρωτεϊνες βητα-γλυκοκερεβροσιδασης με αυξημενη σταθεροτητα και αυξημενη συγκρατουμενη καταλυτικη δραστικοτητα - Google Patents

Παραλλαγμενες ανασυνδυασμενες πρωτεϊνες βητα-γλυκοκερεβροσιδασης με αυξημενη σταθεροτητα και αυξημενη συγκρατουμενη καταλυτικη δραστικοτητα

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Publication number
CY1118843T1
CY1118843T1 CY20161101239T CY161101239T CY1118843T1 CY 1118843 T1 CY1118843 T1 CY 1118843T1 CY 20161101239 T CY20161101239 T CY 20161101239T CY 161101239 T CY161101239 T CY 161101239T CY 1118843 T1 CY1118843 T1 CY 1118843T1
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CY
Cyprus
Prior art keywords
proteins
increased
recombinant
glucocerebrosidase
glykerobreossible
Prior art date
Application number
CY20161101239T
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English (en)
Inventor
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 CY1118843T1 publication Critical patent/CY1118843T1/el

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    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • 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)
    • 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)
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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

Abstract

Εδώ περιγράφονται παραλλαγμένες, ανασυνδυασμένες πρωτεΐνες β-γλυκοκερεβροσιδάσης που χαρακτηρίζονται από το ότι έχουν αυξημένη σταθερότητα σε σχέση με την ανασυνδυασμένη β-γλυκοκερεβροσιδάση άγριου-τύπου. Επίσης παρέχονται εδώ παραλλαγμένες, ανασυνδυασμένες πρωτεΐνες β-γλυκοκερεβροσιδάσης που χαρακτηρίζονται από το ότι συγκρατούν περισσότερη καταλυτική δραστικότητα σε σχέση με την ανασυνδυασμένη β-γλυκοκερεβροσιδάση άγριου τύπου. Επιπλέον εδώ περιγράφονται παραλλαγμένες, ανασυνδυασμένες πρωτεΐνες β- γλυκοκερεβροσιδάσης που μπορούν να έχουν παραλλαγές αμινοξέων σε μία ή περισσότερες από τις ακόλουθες θέσεις: 316, 317, 321 και 145. Περιγράφονται επίσης οι μέθοδοι παρασκευής των παραλλαγμένων, ανασυνδυασμένων πρωτεϊνών β-γλυκοκερεβροσιδάσης, καθώς και οι μέθοδοι θεραπείας ασθενών με νόσους λυσοσωματικής αποθήκευσης.
CY20161101239T 2010-11-08 2016-11-29 Παραλλαγμενες ανασυνδυασμενες πρωτεϊνες βητα-γλυκοκερεβροσιδασης με αυξημενη σταθεροτητα και αυξημενη συγκρατουμενη καταλυτικη δραστικοτητα CY1118843T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41133110P 2010-11-08 2010-11-08
US41218010P 2010-11-10 2010-11-10
PCT/US2011/059731 WO2012064709A2 (en) 2010-11-08 2011-11-08 Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Publications (1)

Publication Number Publication Date
CY1118843T1 true CY1118843T1 (el) 2018-01-10

Family

ID=46051505

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20161101239T CY1118843T1 (el) 2010-11-08 2016-11-29 Παραλλαγμενες ανασυνδυασμενες πρωτεϊνες βητα-γλυκοκερεβροσιδασης με αυξημενη σταθεροτητα και αυξημενη συγκρατουμενη καταλυτικη δραστικοτητα

Country Status (19)

Country Link
US (4) US8962564B2 (el)
EP (2) EP3144386B1 (el)
JP (2) JP6073796B2 (el)
KR (1) KR101901467B1 (el)
CN (2) CN103314105B (el)
BR (1) BR112013011152A2 (el)
CA (1) CA2817011C (el)
CY (1) CY1118843T1 (el)
DK (1) DK2638152T3 (el)
ES (2) ES2604490T3 (el)
HR (1) HRP20161560T1 (el)
HU (1) HUE030932T2 (el)
LT (1) LT2638152T (el)
PL (1) PL2638152T3 (el)
PT (1) PT2638152T (el)
RS (1) RS55405B1 (el)
SI (1) SI2638152T1 (el)
SM (1) SMT201600431B (el)
WO (1) WO2012064709A2 (el)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3127162A1 (en) 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides
CN114364794A (zh) * 2019-09-09 2022-04-15 豪夫迈·罗氏有限公司 葡糖脑苷脂酶突变体
KR20230042513A (ko) * 2020-07-29 2023-03-28 프리라인 테라퓨틱스 리미티드 폴리펩티드
WO2023145812A1 (ja) * 2022-01-31 2023-08-03 株式会社日本触媒 グルコセレブロシダーゼ活性を有する糖鎖付加タンパク質
WO2023145813A1 (ja) * 2022-01-31 2023-08-03 株式会社日本触媒 グルコセレブロシダーゼ活性を有するタンパク質およびその製造方法
WO2023145814A1 (ja) * 2022-01-31 2023-08-03 株式会社日本触媒 向上した酵素活性または向上した安定性を有する組換えグルコセレブロシダーゼタンパク質
WO2023198661A1 (en) 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
WO2024074142A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of disease associated with gcase deficiency

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JPH03503721A (ja) * 1988-12-23 1991-08-22 ジェンザイム　コーポレイション 酵素的に活性な組換えグルコセレブロシダーゼ
ES2093642T3 (es) 1988-12-23 1997-01-01 Genzyme Corp Celulas cho que producen gluco-cerebrosidasa re-combinante enzimaticamente activa.
US6524613B1 (en) 1997-04-30 2003-02-25 Regents Of The University Of Minnesota Hepatocellular chimeraplasty
EP0979311A1 (en) 1997-04-30 2000-02-16 Of The University Of Minnesota Regents $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
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AU764686B2 (en) 1998-08-28 2003-08-28 Duke University Adenoviruses deleted in the IVa2, 100K, polymerase and/or preterminal protein sequences
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Also Published As

Publication number Publication date
CN103314105A (zh) 2013-09-18
JP2017099390A (ja) 2017-06-08
CA2817011A1 (en) 2012-05-18
RS55405B1 (sr) 2017-04-28
EP3144386B1 (en) 2019-05-29
SMT201600431B (it) 2017-01-10
EP2638152A2 (en) 2013-09-18
ES2604490T3 (es) 2017-03-07
JP6457559B2 (ja) 2019-01-23
PT2638152T (pt) 2016-10-25
KR20140009238A (ko) 2014-01-22
JP6073796B2 (ja) 2017-02-01
CN103314105B (zh) 2015-11-25
KR101901467B1 (ko) 2018-11-02
US20130344054A1 (en) 2013-12-26
SI2638152T1 (sl) 2016-12-30
EP2638152B1 (en) 2016-08-31
HUE030932T2 (en) 2017-06-28
WO2012064709A2 (en) 2012-05-18
US9254313B2 (en) 2016-02-09
CA2817011C (en) 2019-04-02
LT2638152T (lt) 2016-12-12
PL2638152T3 (pl) 2017-02-28
US20150216950A1 (en) 2015-08-06
US20170157220A1 (en) 2017-06-08
US9566316B2 (en) 2017-02-14
EP2638152A4 (en) 2014-04-09
CN105296447A (zh) 2016-02-03
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
HRP20161560T1 (hr) 2016-12-30
ES2743825T3 (es) 2020-02-20
JP2014500722A (ja) 2014-01-16
EP3144386A1 (en) 2017-03-22
DK2638152T3 (en) 2016-12-12
US9821038B2 (en) 2017-11-21
US20160184408A1 (en) 2016-06-30
BR112013011152A2 (pt) 2016-11-29

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