ES2743825T3 - Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada - Google Patents

Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada Download PDF

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Publication number
ES2743825T3
ES2743825T3 ES16178095T ES16178095T ES2743825T3 ES 2743825 T3 ES2743825 T3 ES 2743825T3 ES 16178095 T ES16178095 T ES 16178095T ES 16178095 T ES16178095 T ES 16178095T ES 2743825 T3 ES2743825 T3 ES 2743825T3
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Prior art keywords
glccerase
seq
wild
recombinant
protein
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Active
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ES16178095T
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English (en)
Spanish (es)
Inventor
Hung Do
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • 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)
  • Hematology (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)
ES16178095T 2010-11-08 2011-11-08 Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada Active ES2743825T3 (es)

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
ES2743825T3 true ES2743825T3 (es) 2020-02-20

Family

ID=46051505

Family Applications (2)

Application Number Title Priority Date Filing Date
ES16178095T Active ES2743825T3 (es) 2010-11-08 2011-11-08 Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada
ES11839428.7T Active ES2604490T3 (es) 2010-11-08 2011-11-08 Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES11839428.7T Active ES2604490T3 (es) 2010-11-08 2011-11-08 Proteínas beta-glucocerebrosidasa recombinantes, variantes con estabilidad aumentada y actividad catalítica retenida aumentada

Country Status (19)

Country Link
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)

Families Citing this family (12)

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

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
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
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
IL150314A0 (en) 1999-12-30 2002-12-01 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
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
AU2008245578A1 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
KR101789000B1 (ko) * 2007-05-22 2017-10-20 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
EP2220218A4 (en) 2007-11-02 2010-12-08 Scripps Research Inst GENETICALLY ENCODED BORONATE AMINO ACID

Also Published As

Publication number Publication date
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
JP6073796B2 (ja) 2017-02-01
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
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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