HUE030932T2 - Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins - Google Patents

Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins Download PDF

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Publication number
HUE030932T2
HUE030932T2 HUE11839428A HUE11839428A HUE030932T2 HU E030932 T2 HUE030932 T2 HU E030932T2 HU E11839428 A HUE11839428 A HU E11839428A HU E11839428 A HUE11839428 A HU E11839428A HU E030932 T2 HUE030932 T2 HU E030932T2
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Hungary
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leu
ala
ser
gly
pro
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HUE11839428A
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English (en)
Hungarian (hu)
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Hung Do
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Amicus Therapeutics Inc
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Publication of HUE030932T2 publication Critical patent/HUE030932T2/en

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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
    • 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
    • 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)
    • 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)
    • 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

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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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (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)
HUE11839428A 2010-11-08 2011-11-08 Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins HUE030932T2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US41133110P 2010-11-08 2010-11-08
US41218010P 2010-11-10 2010-11-10

Publications (1)

Publication Number Publication Date
HUE030932T2 true HUE030932T2 (en) 2017-06-28

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
HUE11839428A HUE030932T2 (en) 2010-11-08 2011-11-08 Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins

Country Status (19)

Country Link
US (4) US8962564B2 (enExample)
EP (2) EP2638152B1 (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 (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
FI3850089T3 (fi) 2019-02-04 2024-02-14 Freeline Therapeutics Ltd Polynukleotidejä
JP2022547297A (ja) 2019-09-09 2022-11-11 エフ．ホフマン－ラ ロシュ アーゲー グルコセレブロシダーゼ変異体
US20230132790A1 (en) * 2020-03-29 2023-05-04 Yeda Research And Development Co. Ltd. Variants of beta-glucocerebrosidase for use in treating gaucher disease
CA3189801A1 (en) * 2020-07-29 2022-02-03 Freeline Therapeutics Limited Polypeptide
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 (enExample) * 2022-01-31 2023-08-03
US20250145978A1 (en) * 2022-01-31 2025-05-08 Nippon Shokubai Co., Ltd. Glycosylated protein having glucocerebrosidase activity
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
WO2025122912A1 (en) * 2023-12-08 2025-06-12 Denali Therapeutics Inc. Modified glucocerebrosidase polypeptides and methods of use thereof

Family Cites Families (16)

* Cited by examiner, † Cited by third party
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
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
US6524613B1 (en) 1997-04-30 2003-02-25 Regents Of The University Of Minnesota Hepatocellular chimeraplasty
EP1027069B1 (en) * 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher disease
IL141403A0 (en) * 1998-08-28 2002-03-10 Univ Duke Deleted adenoviruses and methods of making and administering the same
US20020127219A1 (en) * 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
IL150314A0 (en) * 1999-12-30 2002-12-01 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
AU2001253181A1 (en) * 2000-04-06 2001-10-23 Exegenics, Inc. Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase)
WO2002002597A2 (en) 2000-06-30 2002-01-10 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
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
KR20100017879A (ko) * 2007-05-22 2010-02-16 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법
US20090148887A1 (en) 2007-11-02 2009-06-11 The Scripps Research Institute Genetically encoded boronate amino acid
BRPI0819092A2 (pt) 2007-11-02 2014-10-14 Scripps Research Inst Evolução direcionada usando proteínas que compreendem aminoácidos não naturais.

Also Published As

Publication number Publication date
SMT201600431B (it) 2017-01-10
DK2638152T3 (en) 2016-12-12
CN103314105A (zh) 2013-09-18
EP2638152A4 (en) 2014-04-09
JP2014500722A (ja) 2014-01-16
EP3144386A1 (en) 2017-03-22
WO2012064709A2 (en) 2012-05-18
PL2638152T3 (pl) 2017-02-28
RS55405B1 (sr) 2017-04-28
KR101901467B1 (ko) 2018-11-02
CA2817011C (en) 2019-04-02
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
EP3144386B1 (en) 2019-05-29
CN105296447A (zh) 2016-02-03
PT2638152T (pt) 2016-10-25
CN103314105B (zh) 2015-11-25
JP6073796B2 (ja) 2017-02-01
JP2017099390A (ja) 2017-06-08
US20170157220A1 (en) 2017-06-08
LT2638152T (lt) 2016-12-12
KR20140009238A (ko) 2014-01-22
BR112013011152A2 (pt) 2016-11-29
SI2638152T1 (sl) 2016-12-30
ES2604490T3 (es) 2017-03-07
US20130344054A1 (en) 2013-12-26
ES2743825T3 (es) 2020-02-20
JP6457559B2 (ja) 2019-01-23
US9566316B2 (en) 2017-02-14
HRP20161560T1 (hr) 2016-12-30
EP2638152B1 (en) 2016-08-31
US9821038B2 (en) 2017-11-21
EP2638152A2 (en) 2013-09-18
US20160184408A1 (en) 2016-06-30
US9254313B2 (en) 2016-02-09
CY1118843T1 (el) 2018-01-10
CA2817011A1 (en) 2012-05-18
US20150216950A1 (en) 2015-08-06

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