CN103314105B - 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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CN103314105B
CN103314105B CN201180064243.6A CN201180064243A CN103314105B CN 103314105 B CN103314105 B CN 103314105B CN 201180064243 A CN201180064243 A CN 201180064243A CN 103314105 B CN103314105 B CN 103314105B
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g1ccerase
glucocerebrosidase
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CN103314105A (zh
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H·杜
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
Callidus Biopharma Inc
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    • 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
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • 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)
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    • 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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CN201180064243.6A 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 Expired - Fee Related CN103314105B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN201510671991.0A CN105296447A (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

Applications Claiming Priority (5)

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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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CN201510671991.0A Division CN105296447A (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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CN103314105A CN103314105A (zh) 2013-09-18
CN103314105B true CN103314105B (zh) 2015-11-25

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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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CA3127162A1 (en) * 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides
CA3128875A1 (en) 2019-02-04 2020-08-13 Freeline Therapeutics Limited Polynucleotides
EP4028048A1 (en) 2019-09-09 2022-07-20 F. Hoffmann-La Roche AG Glucocerebrosidase mutants
JP2023519934A (ja) * 2020-03-29 2023-05-15 イエダ・リサーチ・アンド・デベロツプメント・カンパニー・リミテツド ゴーシェ病の処置において使用するためのベータ－グルコセレブロシダーゼのバリアント
BR112023001583A2 (pt) * 2020-07-29 2023-02-14 Freeline Therapeutics Ltd Polipeptídeos de ss-glucocerebrosidase (gcase) modificado, polinucleotídeo, partícula viral e composição
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
JPWO2023145813A1 (enExample) * 2022-01-31 2023-08-03
JPWO2023145814A1 (enExample) * 2022-01-31 2023-08-03
JP2025514645A (ja) 2022-04-12 2025-05-09 エフ． ホフマン－ラ ロシュ アーゲー 中枢神経系に対して標的化された融合タンパク質
WO2024074142A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of disease associated with gcase deficiency
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

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CN1268186A (zh) * 1997-04-30 2000-09-27 明尼苏达大学评议会 重组诱发剂寡核碱基校正肝细胞遗传损害的体内应用
CN1342206A (zh) * 1998-08-28 2002-03-27 杜克大学 在IVa2,100K和/或preterminal protein序列上有缺失的腺病毒
US20020127219A1 (en) * 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
CN101878303A (zh) * 2007-11-02 2010-11-03 斯克利普斯研究院 使用包含非天然氨基酸的蛋白质进行定向进化

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ES2093642T3 (es) 1988-12-23 1997-01-01 Genzyme Corp Celulas cho que producen gluco-cerebrosidasa re-combinante enzimaticamente activa.
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CN1342206A (zh) * 1998-08-28 2002-03-27 杜克大学 在IVa2,100K和/或preterminal protein序列上有缺失的腺病毒
US20020127219A1 (en) * 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
CN101878303A (zh) * 2007-11-02 2010-11-03 斯克利普斯研究院 使用包含非天然氨基酸的蛋白质进行定向进化

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US20160184408A1 (en) 2016-06-30
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
US20130344054A1 (en) 2013-12-26
PL2638152T3 (pl) 2017-02-28
ES2743825T3 (es) 2020-02-20
JP2014500722A (ja) 2014-01-16
ES2604490T3 (es) 2017-03-07
EP2638152B1 (en) 2016-08-31
EP2638152A2 (en) 2013-09-18
JP6073796B2 (ja) 2017-02-01
US20170157220A1 (en) 2017-06-08
CA2817011A1 (en) 2012-05-18
US9821038B2 (en) 2017-11-21
US9254313B2 (en) 2016-02-09
DK2638152T3 (en) 2016-12-12
SI2638152T1 (sl) 2016-12-30
SMT201600431B (it) 2017-01-10
CY1118843T1 (el) 2018-01-10
HRP20161560T1 (hr) 2016-12-30
LT2638152T (lt) 2016-12-12
HUE030932T2 (en) 2017-06-28
CN103314105A (zh) 2013-09-18
EP2638152A4 (en) 2014-04-09
JP2017099390A (ja) 2017-06-08
EP3144386B1 (en) 2019-05-29
BR112013011152A2 (pt) 2016-11-29
KR101901467B1 (ko) 2018-11-02
EP3144386A1 (en) 2017-03-22
PT2638152T (pt) 2016-10-25
CN105296447A (zh) 2016-02-03
RS55405B1 (sr) 2017-04-28
JP6457559B2 (ja) 2019-01-23
KR20140009238A (ko) 2014-01-22
WO2012064709A2 (en) 2012-05-18
CA2817011C (en) 2019-04-02
US9566316B2 (en) 2017-02-14
US20150216950A1 (en) 2015-08-06

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