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
recombinant
wild
protein
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JP2014500722A (ja
JP2014500722A5 (cg-RX-API-DMAC10.html
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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
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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)
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  • Organic Chemistry (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
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  • 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

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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

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JP2014500722A JP2014500722A (ja) 2014-01-16
JP2014500722A5 JP2014500722A5 (cg-RX-API-DMAC10.html) 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 (cg-RX-API-DMAC10.html)
EP (2) EP2638152B1 (cg-RX-API-DMAC10.html)
JP (2) JP6073796B2 (cg-RX-API-DMAC10.html)
KR (1) KR101901467B1 (cg-RX-API-DMAC10.html)
CN (2) CN105296447A (cg-RX-API-DMAC10.html)
BR (1) BR112013011152A2 (cg-RX-API-DMAC10.html)
CA (1) CA2817011C (cg-RX-API-DMAC10.html)
CY (1) CY1118843T1 (cg-RX-API-DMAC10.html)
DK (1) DK2638152T3 (cg-RX-API-DMAC10.html)
ES (2) ES2743825T3 (cg-RX-API-DMAC10.html)
HR (1) HRP20161560T1 (cg-RX-API-DMAC10.html)
HU (1) HUE030932T2 (cg-RX-API-DMAC10.html)
LT (1) LT2638152T (cg-RX-API-DMAC10.html)
PL (1) PL2638152T3 (cg-RX-API-DMAC10.html)
PT (1) PT2638152T (cg-RX-API-DMAC10.html)
RS (1) RS55405B1 (cg-RX-API-DMAC10.html)
SI (1) SI2638152T1 (cg-RX-API-DMAC10.html)
SM (1) SMT201600431B (cg-RX-API-DMAC10.html)
WO (1) WO2012064709A2 (cg-RX-API-DMAC10.html)

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WO2020157248A1 (en) * 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides
CN113795575A (zh) 2019-02-04 2021-12-14 自由行疗法有限公司 多核苷酸
US20220325265A1 (en) 2019-09-09 2022-10-13 Hoffmann-La Roche Inc. Glucocerebrosidase mutants
WO2021199039A1 (en) * 2020-03-29 2021-10-07 Yeda Research And Development Co. Ltd. Variants of beta-glucocerebrosidase for use in treating gaucher disease
KR20230042513A (ko) * 2020-07-29 2023-03-28 프리라인 테라퓨틱스 리미티드 폴리펩티드
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
JPWO2023145813A1 (cg-RX-API-DMAC10.html) * 2022-01-31 2023-08-03
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
WO2023198661A1 (en) 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
CN121406674A (zh) * 2022-10-08 2026-01-27 凌意(杭州)生物科技有限公司 用于治疗GCase缺乏相关疾病的多核苷酸
AU2024394235A1 (en) * 2023-12-08 2026-06-11 Denali Therapeutics Inc. Modified glucocerebrosidase polypeptides and methods of use thereof
TW202617982A (zh) * 2024-06-24 2026-05-01 美商戴納立製藥公司 包含經修飾之葡萄糖腦苷脂酶多肽之融合蛋白及其方法

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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
JP2893481B2 (ja) 1988-12-23 1999-05-24 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
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
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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
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HRP20161560T1 (hr) 2016-12-30
CY1118843T1 (el) 2018-01-10
KR20140009238A (ko) 2014-01-22
US9566316B2 (en) 2017-02-14
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
US20150216950A1 (en) 2015-08-06
US20170157220A1 (en) 2017-06-08
EP2638152B1 (en) 2016-08-31
RS55405B1 (sr) 2017-04-28
US20160184408A1 (en) 2016-06-30
EP3144386A1 (en) 2017-03-22
US9254313B2 (en) 2016-02-09
JP2017099390A (ja) 2017-06-08
US9821038B2 (en) 2017-11-21
JP2014500722A (ja) 2014-01-16
CA2817011C (en) 2019-04-02
BR112013011152A2 (pt) 2016-11-29
EP3144386B1 (en) 2019-05-29
CA2817011A1 (en) 2012-05-18
KR101901467B1 (ko) 2018-11-02
ES2604490T3 (es) 2017-03-07
PT2638152T (pt) 2016-10-25
CN103314105B (zh) 2015-11-25
WO2012064709A2 (en) 2012-05-18
JP6457559B2 (ja) 2019-01-23
US20130344054A1 (en) 2013-12-26
HUE030932T2 (en) 2017-06-28
EP2638152A2 (en) 2013-09-18
SI2638152T1 (sl) 2016-12-30
PL2638152T3 (pl) 2017-02-28
CN105296447A (zh) 2016-02-03
ES2743825T3 (es) 2020-02-20
DK2638152T3 (en) 2016-12-12
LT2638152T (lt) 2016-12-12
SMT201600431B (it) 2017-01-10
EP2638152A4 (en) 2014-04-09
CN103314105A (zh) 2013-09-18

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