CN105296447A - 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白 - Google Patents

具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白 Download PDF

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CN105296447A
CN105296447A CN201510671991.0A CN201510671991A CN105296447A CN 105296447 A CN105296447 A CN 105296447A CN 201510671991 A CN201510671991 A CN 201510671991A CN 105296447 A CN105296447 A CN 105296447A
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glucocerebrosidase
albumen
g1ccerase
recombinant beta
wild
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Chinese (zh)
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H·杜
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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    • 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
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    • 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
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    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201510671991.0A 2010-11-08 2011-11-08 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白 Pending CN105296447A (zh)

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
CN201180064243.6A CN103314105B (zh) 2010-11-08 2011-11-08 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白

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CN201180064243.6A Division CN103314105B (zh) 2010-11-08 2011-11-08 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白

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CN105296447A true CN105296447A (zh) 2016-02-03

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CN201180064243.6A Expired - Fee Related CN103314105B (zh) 2010-11-08 2011-11-08 具有增强的稳定性和增强的保留催化活性的变体重组β-葡萄糖脑苷脂酶蛋白

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US (4) US8962564B2 (https=)
EP (2) EP3144386B1 (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) ES2743825T3 (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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (11)

* 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
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
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
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
JPWO2023145814A1 (https=) * 2022-01-31 2023-08-03
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
EP4508206A1 (en) 2022-04-12 2025-02-19 F. Hoffmann-La Roche AG Fusion proteins targeted to the central nervous system
CN118715316B (zh) * 2022-10-08 2025-09-19 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
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

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DE68926569T2 (de) 1988-12-23 1996-09-26 Genzyme Corp Cho-zellen befähigt enzymatisch aktive rekombinante-glukocerebrosidase zu produzieren
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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Publication number Priority date Publication date Assignee Title
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

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ES2604490T3 (es) 2017-03-07
BR112013011152A2 (pt) 2016-11-29
US9566316B2 (en) 2017-02-14
RS55405B1 (sr) 2017-04-28
DK2638152T3 (en) 2016-12-12
ES2743825T3 (es) 2020-02-20
EP2638152A2 (en) 2013-09-18
JP6073796B2 (ja) 2017-02-01
CN103314105A (zh) 2013-09-18
US20150216950A1 (en) 2015-08-06
US20160184408A1 (en) 2016-06-30
CA2817011A1 (en) 2012-05-18
CY1118843T1 (el) 2018-01-10
US20130344054A1 (en) 2013-12-26
JP2017099390A (ja) 2017-06-08
SMT201600431B (it) 2017-01-10
CA2817011C (en) 2019-04-02
KR101901467B1 (ko) 2018-11-02
PT2638152T (pt) 2016-10-25
SI2638152T1 (sl) 2016-12-30
JP2014500722A (ja) 2014-01-16
US9821038B2 (en) 2017-11-21
EP3144386A1 (en) 2017-03-22
HRP20161560T1 (hr) 2016-12-30
EP3144386B1 (en) 2019-05-29
HUE030932T2 (en) 2017-06-28
US20170157220A1 (en) 2017-06-08
LT2638152T (lt) 2016-12-12
US9254313B2 (en) 2016-02-09
JP6457559B2 (ja) 2019-01-23
PL2638152T3 (pl) 2017-02-28
CN103314105B (zh) 2015-11-25
EP2638152B1 (en) 2016-08-31
WO2012064709A2 (en) 2012-05-18
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
EP2638152A4 (en) 2014-04-09
KR20140009238A (ko) 2014-01-22

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