JP6073796B2 - 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 - Google Patents

増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 Download PDF

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JP6073796B2
JP6073796B2 JP2013538823A JP2013538823A JP6073796B2 JP 6073796 B2 JP6073796 B2 JP 6073796B2 JP 2013538823 A JP2013538823 A JP 2013538823A JP 2013538823 A JP2013538823 A JP 2013538823A JP 6073796 B2 JP6073796 B2 JP 6073796B2
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glccerase
glucocerebrosidase
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protein
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JP2014500722A (ja
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ドー，フン
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アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
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    • 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)
    • 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)
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  • Molecular Biology (AREA)
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  • 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)
JP2013538823A 2010-11-08 2011-11-08 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 Active JP6073796B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US41133110P 2010-11-08 2010-11-08
US61/411,331 2010-11-08
US41218010P 2010-11-10 2010-11-10
US61/412,180 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

Related Child Applications (1)

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JP2017000301A Division JP6457559B2 (ja) 2010-11-08 2017-01-05 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質

Publications (3)

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JP2014500722A JP2014500722A (ja) 2014-01-16
JP2014500722A5 JP2014500722A5 (https=) 2014-12-25
JP6073796B2 true JP6073796B2 (ja) 2017-02-01

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JP2017000301A Active JP6457559B2 (ja) 2010-11-08 2017-01-05 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質

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US (4) US8962564B2 (https=)
EP (2) EP2638152B1 (https=)
JP (2) JP6073796B2 (https=)
KR (1) KR101901467B1 (https=)
CN (2) CN105296447A (https=)
BR (1) BR112013011152A2 (https=)
CA (1) CA2817011C (https=)
CY (1) CY1118843T1 (https=)
DK (1) DK2638152T3 (https=)
ES (2) ES2604490T3 (https=)
HR (1) HRP20161560T1 (https=)
HU (1) HUE030932T2 (https=)
LT (1) LT2638152T (https=)
PL (1) PL2638152T3 (https=)
PT (1) PT2638152T (https=)
RS (1) RS55405B1 (https=)
SI (1) SI2638152T1 (https=)
SM (1) SMT201600431B (https=)
WO (1) WO2012064709A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220125892A1 (en) * 2019-02-01 2022-04-28 OXYRANE UK Ltd. Glucocerebrosidase polypeptides
BR112021015312A2 (pt) 2019-02-04 2021-11-09 Freeline Therapeutics Ltd Polinucleotídeos, partícula viral e composição
US20220325265A1 (en) 2019-09-09 2022-10-13 Hoffmann-La Roche Inc. Glucocerebrosidase mutants
KR20230003492A (ko) * 2020-03-29 2023-01-06 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 고셔병 치료용 베타-글루코세레브로시다아제 변이체
EP4118200B1 (en) * 2020-07-29 2025-10-08 Spur Therapeutics Limited Mutated beta-glucocerebrosidase with improved stability
JPWO2023145812A1 (https=) * 2022-01-31 2023-08-03
US20250129351A1 (en) * 2022-01-31 2025-04-24 Nippon Shokubai Co., Ltd. Recombinant glucocerebrosidase protein having improved enzyme activity or improved stability
US20250145979A1 (en) * 2022-01-31 2025-05-08 Nippon Shokubai Co., Ltd. Protein having glucocerebrosidase activity and method for producing same
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
WO2025122912A1 (en) * 2023-12-08 2025-06-12 Denali Therapeutics Inc. Modified glucocerebrosidase polypeptides and methods of use thereof
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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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
WO1990007573A1 (en) 1988-12-23 1990-07-12 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
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
US6524613B1 (en) 1997-04-30 2003-02-25 Regents Of The University Of Minnesota Hepatocellular chimeraplasty
IL135578A0 (en) * 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods 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
AU2352201A (en) 1999-12-30 2001-07-16 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
EP1613264A4 (en) 2003-04-16 2007-08-15 Yeda Res & Dev MEDICAMENT FOR THE TREATMENT OF GAUCHER DISEASE AND METHOD FOR IDENTIFYING IT
US20050265988A1 (en) * 2004-03-18 2005-12-01 Byung-Kwon Choi Glycosylated glucocerebrosidase expression in fungal hosts
EP2150254A4 (en) * 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
KR101650726B1 (ko) * 2007-05-22 2016-08-24 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
US9150849B2 (en) 2007-11-02 2015-10-06 The Scripps Research Institute Directed evolution using proteins comprising unnatural amino acids
WO2009059056A2 (en) 2007-11-02 2009-05-07 The Scripps Research Institute A genetically encoded boronate amino acid

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Publication number Publication date
SI2638152T1 (sl) 2016-12-30
CA2817011A1 (en) 2012-05-18
EP2638152A4 (en) 2014-04-09
JP6457559B2 (ja) 2019-01-23
BR112013011152A2 (pt) 2016-11-29
WO2012064709A2 (en) 2012-05-18
CN105296447A (zh) 2016-02-03
DK2638152T3 (en) 2016-12-12
JP2014500722A (ja) 2014-01-16
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
HUE030932T2 (en) 2017-06-28
PT2638152T (pt) 2016-10-25
CA2817011C (en) 2019-04-02
ES2743825T3 (es) 2020-02-20
KR20140009238A (ko) 2014-01-22
US9821038B2 (en) 2017-11-21
CN103314105B (zh) 2015-11-25
LT2638152T (lt) 2016-12-12
US20130344054A1 (en) 2013-12-26
US9254313B2 (en) 2016-02-09
JP2017099390A (ja) 2017-06-08
RS55405B1 (sr) 2017-04-28
US20160184408A1 (en) 2016-06-30
KR101901467B1 (ko) 2018-11-02
SMT201600431B (it) 2017-01-10
CN103314105A (zh) 2013-09-18
EP3144386A1 (en) 2017-03-22
EP2638152A2 (en) 2013-09-18
CY1118843T1 (el) 2018-01-10
EP3144386B1 (en) 2019-05-29
ES2604490T3 (es) 2017-03-07
US20170157220A1 (en) 2017-06-08
US9566316B2 (en) 2017-02-14
PL2638152T3 (pl) 2017-02-28
EP2638152B1 (en) 2016-08-31
US20150216950A1 (en) 2015-08-06
HRP20161560T1 (hr) 2016-12-30

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