RS55405B1 - Varijantni, rekombinantni proteini beta-glukocerebrozidaze sa povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću - Google Patents

Varijantni, rekombinantni proteini beta-glukocerebrozidaze sa povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću

Info

Publication number
RS55405B1
RS55405B1 RS20161010A RSP20161010A RS55405B1 RS 55405 B1 RS55405 B1 RS 55405B1 RS 20161010 A RS20161010 A RS 20161010A RS P20161010 A RSP20161010 A RS P20161010A RS 55405 B1 RS55405 B1 RS 55405B1
Authority
RS
Serbia
Prior art keywords
glucocerebrosidase
wild
glccerase
variant
recombinant
Prior art date
Application number
RS20161010A
Other languages
English (en)
Serbian (sr)
Inventor
Hung Do
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of RS55405B1 publication Critical patent/RS55405B1/sr

Links

Classifications

    • 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

Landscapes

  • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)
RS20161010A 2010-11-08 2011-11-08 Varijantni, rekombinantni proteini beta-glukocerebrozidaze sa povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću RS55405B1 (sr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US41133110P 2010-11-08 2010-11-08
US41218010P 2010-11-10 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
EP11839428.7A EP2638152B1 (en) 2010-11-08 2011-11-08 Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Publications (1)

Publication Number Publication Date
RS55405B1 true RS55405B1 (sr) 2017-04-28

Family

ID=46051505

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20161010A RS55405B1 (sr) 2010-11-08 2011-11-08 Varijantni, rekombinantni proteini beta-glukocerebrozidaze sa povećanom stabilnošću i povećanom zadržanom katalitičkom aktivnošću

Country Status (19)

Country Link
US (4) US8962564B2 (enExample)
EP (2) EP3144386B1 (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 (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
PL3850089T3 (pl) 2019-02-04 2024-05-06 Freeline Therapeutics Limited Polinukleotydy
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
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
IL299811A (en) * 2020-07-29 2023-03-01 Freeline Therapeutics Ltd polypeptide
US20250129351A1 (en) * 2022-01-31 2025-04-24 Nippon Shokubai Co., Ltd. Recombinant glucocerebrosidase protein having improved enzyme activity or improved stability
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
JPWO2023145812A1 (enExample) * 2022-01-31 2023-08-03
WO2023198661A1 (en) 2022-04-12 2023-10-19 F. Hoffmann-La Roche Ag Fusion proteins targeted to the central nervous system
JP2025534899A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド ＧＣａｓｅ欠乏関連疾患を治療するためのポリヌクレオチド
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

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
ES2093642T3 (es) 1988-12-23 1997-01-01 Genzyme Corp Celulas cho que producen gluco-cerebrosidasa re-combinante enzimaticamente activa.
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
US6066626A (en) 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method for treating lysosomal storage disease
CA2340276A1 (en) 1998-08-28 2000-03-09 Duke University Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences
WO2001049830A2 (en) * 1999-12-30 2001-07-12 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
WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
ES2490143T3 (es) * 2007-05-22 2014-09-03 Amicus Therapeutics, Inc. Nuevo método para preparar isofagomina y sus derivados
EP2220218A4 (en) 2007-11-02 2010-12-08 Scripps Research Inst GENETICALLY ENCODED BORONATE AMINO ACID
US9249408B2 (en) 2007-11-02 2016-02-02 The Scripps Research Institute Directed evolution using proteins comprising unnatural amino acids

Also Published As

Publication number Publication date
ES2743825T3 (es) 2020-02-20
SI2638152T1 (sl) 2016-12-30
HRP20161560T1 (hr) 2016-12-30
EP3144386A1 (en) 2017-03-22
US8962564B2 (en) 2015-02-24
WO2012064709A3 (en) 2012-08-02
JP2014500722A (ja) 2014-01-16
EP3144386B1 (en) 2019-05-29
US20160184408A1 (en) 2016-06-30
KR101901467B1 (ko) 2018-11-02
CN105296447A (zh) 2016-02-03
US9254313B2 (en) 2016-02-09
PL2638152T3 (pl) 2017-02-28
CN103314105A (zh) 2013-09-18
US9566316B2 (en) 2017-02-14
DK2638152T3 (en) 2016-12-12
CY1118843T1 (el) 2018-01-10
LT2638152T (lt) 2016-12-12
US20150216950A1 (en) 2015-08-06
JP6457559B2 (ja) 2019-01-23
US20130344054A1 (en) 2013-12-26
US20170157220A1 (en) 2017-06-08
EP2638152A4 (en) 2014-04-09
BR112013011152A2 (pt) 2016-11-29
CA2817011A1 (en) 2012-05-18
SMT201600431B (it) 2017-01-10
PT2638152T (pt) 2016-10-25
JP2017099390A (ja) 2017-06-08
EP2638152B1 (en) 2016-08-31
CN103314105B (zh) 2015-11-25
CA2817011C (en) 2019-04-02
WO2012064709A2 (en) 2012-05-18
JP6073796B2 (ja) 2017-02-01
ES2604490T3 (es) 2017-03-07
KR20140009238A (ko) 2014-01-22
US9821038B2 (en) 2017-11-21
HUE030932T2 (en) 2017-06-28
EP2638152A2 (en) 2013-09-18

Similar Documents

Publication Publication Date Title
US9821038B2 (en) Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
JP6587666B2 (ja) リソソーム貯蔵疾患の処置のための方法および組成物
JP7042263B2 (ja) ファブリー病の治療のための方法および組成物
EP3845651B1 (en) Novel nuclease domain and uses thereof
RS55669B1 (sr) Proizvodnja aktivne visoko fosforilisane ljudske n-acetilglaktozamin-6-sulfataze i njene primene
KR20180127319A (ko) 신경 질환의 치료를 위한 방법 및 조성물
EP3393501B1 (en) Human alpha-n-acetylgalactosaminidase polypeptide
HK1189624B (en) Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
RU2845088C2 (ru) Расщепленный с n-конца и/или c-конца растворимый полипептид ph20 и его применение
HK1235826A1 (en) Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
HK1238680A1 (en) Methods and compositions for the treatment of monogenic diseases
HK40002981B (en) Methods and compositions for the treatment of lysosomal storage diseases
HK40002981A (en) Methods and compositions for the treatment of lysosomal storage diseases
HK1238680B (en) Methods and compositions for the treatment of monogenic diseases
HK1209788B (en) Methods and compositions for the treatment of lysosomal storage diseases