HRP20161560T1 - Varijanta, rekombinantnih proteina beta-glukocerebrozidaze s povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću - Google Patents

Varijanta, rekombinantnih proteina beta-glukocerebrozidaze s povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću Download PDF

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Publication number
HRP20161560T1
HRP20161560T1 HRP20161560TT HRP20161560T HRP20161560T1 HR P20161560 T1 HRP20161560 T1 HR P20161560T1 HR P20161560T T HRP20161560T T HR P20161560TT HR P20161560 T HRP20161560 T HR P20161560T HR P20161560 T1 HRP20161560 T1 HR P20161560T1
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Croatia
Prior art keywords
variant
recombinant human
glucocerebrosidase
protein according
glucocerebrosidase protein
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HRP20161560TT
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English (en)
Croatian (hr)
Inventor
Hung Do
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Amicus Therapeutics, Inc.
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Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of HRP20161560T1 publication Critical patent/HRP20161560T1/hr

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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
    • 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
    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • 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
    • 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)
    • 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)
    • 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)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • 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)
  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)
HRP20161560TT 2010-11-08 2011-11-08 Varijanta, rekombinantnih proteina beta-glukocerebrozidaze s povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću HRP20161560T1 (hr)

Applications Claiming Priority (4)

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
EP11839428.7A EP2638152B1 (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
HRP20161560T1 true HRP20161560T1 (hr) 2016-12-30

Family

ID=46051505

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20161560TT HRP20161560T1 (hr) 2010-11-08 2011-11-08 Varijanta, rekombinantnih proteina beta-glukocerebrozidaze s povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću

Country Status (19)

Country Link
US (4) US8962564B2 (enExample)
EP (2) EP3144386B1 (enExample)
JP (2) JP6073796B2 (enExample)
KR (1) KR101901467B1 (enExample)
CN (2) CN103314105B (enExample)
BR (1) BR112013011152A2 (enExample)
CA (1) CA2817011C (enExample)
CY (1) CY1118843T1 (enExample)
DK (1) DK2638152T3 (enExample)
ES (2) ES2743825T3 (enExample)
HR (1) HRP20161560T1 (enExample)
HU (1) HUE030932T2 (enExample)
LT (1) LT2638152T (enExample)
PL (1) PL2638152T3 (enExample)
PT (1) PT2638152T (enExample)
RS (1) RS55405B1 (enExample)
SI (1) SI2638152T1 (enExample)
SM (1) SMT201600431B (enExample)
WO (1) WO2012064709A2 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020157248A1 (en) 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides
PL3850089T3 (pl) 2019-02-04 2024-05-06 Freeline Therapeutics Limited Polinukleotydy
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
CN115348884B (zh) * 2020-03-29 2024-07-30 耶达研究及发展有限公司 用于治疗戈谢病的β-葡糖脑苷脂酶变体
IL299811A (en) * 2020-07-29 2023-03-01 Freeline Therapeutics Ltd polypeptide
US20250129351A1 (en) * 2022-01-31 2025-04-24 Nippon Shokubai Co., Ltd. Recombinant glucocerebrosidase protein having improved enzyme activity or improved stability
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
JPWO2023145812A1 (enExample) * 2022-01-31 2023-08-03
WO2023198661A1 (en) 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
JP2025534899A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド GCase欠乏関連疾患を治療するためのポリヌクレオチド
TW202532096A (zh) * 2023-12-08 2025-08-16 美商戴納立製藥公司 經修飾之葡萄糖腦苷脂酶多肽及其使用方法
WO2026006173A1 (en) * 2024-06-24 2026-01-02 Denali Therapeutics Inc. Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof

Family Cites Families (16)

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Publication number Priority date Publication date Assignee Title
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
NZ500694A (en) 1997-04-30 2001-09-28 Univ Minnesota Use of chimeric mutational vector (CMV), a macromolecule and a ligand for cell surface receptor adhesion molecule (clatherin) in gene therapy; the CMV vector derived from REC2 homologous pairing with Ustilago maydis
US6066626A (en) 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method for treating lysosomal storage disease
CA2340276A1 (en) 1998-08-28 2000-03-09 Duke University Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences
WO2001049830A2 (en) * 1999-12-30 2001-07-12 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
US20020127219A1 (en) 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
IL152110A0 (en) 2000-04-06 2003-07-31 Exegenics Inc Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase)
AU2001267337A1 (en) * 2000-06-30 2002-01-14 Maxygen Aps Peptide extended glycosylated polypeptides
US7855063B2 (en) 2003-04-16 2010-12-21 Yeda Research And Development Co. Ltd. Partially deglycosylated glucocerebrosidase polypeptide and crystals thereof
WO2005089047A2 (en) 2004-03-18 2005-09-29 Glycofi, Inc. Glycosylated glucocerebrosidase expression in fungal hosts
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
ES2490143T3 (es) * 2007-05-22 2014-09-03 Amicus Therapeutics, Inc. Nuevo método para preparar isofagomina y sus derivados
EP2220218A4 (en) 2007-11-02 2010-12-08 Scripps Research Inst GENETICALLY ENCODED BORONATE AMINO ACID
US9249408B2 (en) 2007-11-02 2016-02-02 The Scripps Research Institute Directed evolution using proteins comprising unnatural amino acids

Also Published As

Publication number Publication date
ES2743825T3 (es) 2020-02-20
SI2638152T1 (sl) 2016-12-30
EP3144386A1 (en) 2017-03-22
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
JP2014500722A (ja) 2014-01-16
EP3144386B1 (en) 2019-05-29
US20160184408A1 (en) 2016-06-30
RS55405B1 (sr) 2017-04-28
KR101901467B1 (ko) 2018-11-02
CN105296447A (zh) 2016-02-03
US9254313B2 (en) 2016-02-09
PL2638152T3 (pl) 2017-02-28
CN103314105A (zh) 2013-09-18
US9566316B2 (en) 2017-02-14
DK2638152T3 (en) 2016-12-12
CY1118843T1 (el) 2018-01-10
LT2638152T (lt) 2016-12-12
US20150216950A1 (en) 2015-08-06
JP6457559B2 (ja) 2019-01-23
US20130344054A1 (en) 2013-12-26
US20170157220A1 (en) 2017-06-08
EP2638152A4 (en) 2014-04-09
BR112013011152A2 (pt) 2016-11-29
CA2817011A1 (en) 2012-05-18
SMT201600431B (it) 2017-01-10
PT2638152T (pt) 2016-10-25
JP2017099390A (ja) 2017-06-08
EP2638152B1 (en) 2016-08-31
CN103314105B (zh) 2015-11-25
CA2817011C (en) 2019-04-02
WO2012064709A2 (en) 2012-05-18
JP6073796B2 (ja) 2017-02-01
ES2604490T3 (es) 2017-03-07
KR20140009238A (ko) 2014-01-22
US9821038B2 (en) 2017-11-21
HUE030932T2 (en) 2017-06-28
EP2638152A2 (en) 2013-09-18

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