JP6073796B2 - 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 - Google Patents

増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 Download PDF

Info

Publication number
JP6073796B2
JP6073796B2 JP2013538823A JP2013538823A JP6073796B2 JP 6073796 B2 JP6073796 B2 JP 6073796B2 JP 2013538823 A JP2013538823 A JP 2013538823A JP 2013538823 A JP2013538823 A JP 2013538823A JP 6073796 B2 JP6073796 B2 JP 6073796B2
Authority
JP
Japan
Prior art keywords
glccerase
glucocerebrosidase
recombinant
wild
protein
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2013538823A
Other languages
English (en)
Japanese (ja)
Other versions
JP2014500722A (ja
JP2014500722A5 (https=
Inventor
ドー，フン
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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 アミカス セラピューティックス インコーポレイテッド, アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2014500722A publication Critical patent/JP2014500722A/ja
Publication of JP2014500722A5 publication Critical patent/JP2014500722A5/ja
Application granted granted Critical
Publication of JP6073796B2 publication Critical patent/JP6073796B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • 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)
    • 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)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (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)
JP2013538823A 2010-11-08 2011-11-08 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質 Active JP6073796B2 (ja)

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
PCT/US2011/059731 WO2012064709A2 (en) 2010-11-08 2011-11-08 Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017000301A Division JP6457559B2 (ja) 2010-11-08 2017-01-05 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質

Publications (3)

Publication Number Publication Date
JP2014500722A JP2014500722A (ja) 2014-01-16
JP2014500722A5 JP2014500722A5 (https=) 2014-12-25
JP6073796B2 true JP6073796B2 (ja) 2017-02-01

Family

ID=46051505

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2013538823A Active JP6073796B2 (ja) 2010-11-08 2011-11-08 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質
JP2017000301A Active JP6457559B2 (ja) 2010-11-08 2017-01-05 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2017000301A Active JP6457559B2 (ja) 2010-11-08 2017-01-05 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質

Country Status (19)

Country Link
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=)

Families Citing this family (12)

* 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
EP4028048A1 (en) * 2019-09-09 2022-07-20 F. Hoffmann-La Roche AG Glucocerebrosidase mutants
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

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
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
WO2000012740A2 (en) 1998-08-28 2000-03-09 Duke University ADENOVIRUSES DELETED IN THE IVa2, 100K AND/OR PRETERMINAL PROTEIN SEQUENCES
US20020127219A1 (en) 1999-12-30 2002-09-12 Okkels Jens Sigurd Lysosomal enzymes and lysosomal enzyme activators
AU2352201A (en) 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
WO2001077307A2 (en) 2000-04-06 2001-10-18 Exegenics, Inc. Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase)
CA2412882A1 (en) * 2000-06-30 2002-01-10 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
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
CA2688850C (en) * 2007-05-22 2015-08-11 Amicus Therapeutics, Inc. New method for preparing isofagomine and its derivatives
JP5513398B2 (ja) 2007-11-02 2014-06-04 ザ スクリプス リサーチ インスティチュート 非天然アミノ酸を含有する蛋白質を使用する指向的進化
WO2009059056A2 (en) 2007-11-02 2009-05-07 The Scripps Research Institute A genetically encoded boronate amino acid

Also Published As

Publication number Publication date
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
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
CN105296447A (zh) 2016-02-03
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

Similar Documents

Publication Publication Date Title
JP6073796B2 (ja) 増加した安定性および増加した保持触媒活性を有する変異型組換えβ−グルコセレブロシダーゼタンパク質
US11898158B2 (en) Methods and compositions for the treatment of lysosomal storage diseases
US20220331447A1 (en) Methods of treating lysosomal storage diseases
AU2017345430B2 (en) Methods and compositions for the treatment of Fabry disease
HK1189624B (en) Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
HK1235826A1 (en) Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
HK40002981A (en) Methods and compositions for the treatment of lysosomal storage diseases
HK40002981B (en) Methods and compositions for the treatment of lysosomal storage diseases
HK1238680B (en) Methods and compositions for the treatment of monogenic diseases
HK1238680A1 (en) Methods and compositions for the treatment of monogenic diseases

Legal Events

Date Code Title Description
A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20140602

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20140602

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20141105

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20141105

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20150924

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20151020

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160120

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20160531

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160829

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20161206

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20170105

R150 Certificate of patent or registration of utility model

Ref document number: 6073796

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250