DK2638152T3 - Variante, rekombinante beta-glucocerebrosidase-proteiner med forøget stabilitet og forøget bevaret katalytisk aktivitet - Google Patents
Variante, rekombinante beta-glucocerebrosidase-proteiner med forøget stabilitet og forøget bevaret katalytisk aktivitet Download PDFInfo
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- DK2638152T3 DK2638152T3 DK11839428.7T DK11839428T DK2638152T3 DK 2638152 T3 DK2638152 T3 DK 2638152T3 DK 11839428 T DK11839428 T DK 11839428T DK 2638152 T3 DK2638152 T3 DK 2638152T3
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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
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- 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
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- 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
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- 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
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- 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
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- 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
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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
- 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)
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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
- 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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- 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
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- 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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- 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)
Claims (15)
1. Variant, rekombinant humant β-glucocerebrosidase-protein, der har en mutation, der er udvalgt blandt: F316A og L317F; H145L; H145F; H145L, F316A og L317F; K321A; og K321V; i forhold til aminosyresekvensen ifølge SEQ ID NO: 1.
2. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 1, der omfatter mutationen F316A og L317F; eller H145L; og yderligere omfatter en mutation udvalgt blandt K321N, K321A og K321V, hvor mutationerne er i forhold til aminosyresekvensen ifølge SEQ ID NO: 1.
3. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 1 eller krav 2, der har aminosyresekvensen ifølge SEQ ID NO: 2 eller en hvilken som helst af SEQ ID NO: 4 til 16.
4. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge et hvilket som helst af kravene 1 til 3, der er kendetegnet ved at have forøget stabilitet i forhold til vildtype, rekombinant β-glucocerebrosidase.
5. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 4, hvor den forøgede stabilitet er ved fysiologiske betingelser.
6. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 4, hvor den forøgede stabilitet er ved betingelser med neutralt pH og 37 °C.
7. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge et hvilket som helst af kravene 1 til 3, der er kendetegnet ved at have en forlænget tilsyneladende halveringstid, der er mindst 2 gange længere end for vildtype, rekombinant β-glucocerebrosidase.
8. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 7, hvor den forlængede tilsyneladende halveringstid er målt efter inkubation ved betingelser med neutralt pH og 37 °C i et tidsrum på tre timer.
9. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge et hvilket som helst af kravene 1 til 3, der er kendetegnet ved at bevare forøget katalytisk aktivitet i forhold til vildtype, rekombinant β-glucocerebrosidase.
10. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 9, hvor den forøgede katalytiske aktivitet er målt efter inkubation ved betingelser med neutralt pH og 37 °C i et tidsrum på tre timer.
11. Variant, rekombinant humant β-glucocerebrosidase-protein ifølge krav 9, hvor den forøgede katalytiske aktivitet bevares ved fysiologiske betingelser.
12. Sammensætning, der omfatter det variante, rekombinante humane β-glucocerebrosidase-protein ifølge et hvilket som helst af ovennævnte krav og et farmaceutisk acceptabelt bæremateriale.
13. Sammensætning, der omfatter det variante, rekombinante humane β-glucocerebrosidase-protein ifølge et hvilket som helst af kravene 1 til 11 og en farmaceutisk acceptabel buffer.
14. Variant, rekombinant β-glucocerebrosidase-protein ifølge et hvilket som helst af kravene 1 til 11 til anvendelse til behandling af en lysosomal aflejringssygdom.
15. Variant, rekombinant humant β-glucocerebrosidase-protein til anvendelse ifølge krav 14, hvor den lysosomale aflejringssygdom er Gauchers sygdom.
Applications Claiming Priority (3)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2638152T3 true DK2638152T3 (da) | 2016-12-12 |
Family
ID=46051505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK11839428.7T DK2638152T3 (da) | 2010-11-08 | 2011-11-08 | Variante, rekombinante beta-glucocerebrosidase-proteiner med forøget stabilitet og forøget bevaret katalytisk aktivitet |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US8962564B2 (da) |
| EP (2) | EP3144386B1 (da) |
| JP (2) | JP6073796B2 (da) |
| KR (1) | KR101901467B1 (da) |
| CN (2) | CN105296447A (da) |
| BR (1) | BR112013011152A2 (da) |
| CA (1) | CA2817011C (da) |
| CY (1) | CY1118843T1 (da) |
| DK (1) | DK2638152T3 (da) |
| ES (2) | ES2743825T3 (da) |
| HR (1) | HRP20161560T1 (da) |
| HU (1) | HUE030932T2 (da) |
| LT (1) | LT2638152T (da) |
| PL (1) | PL2638152T3 (da) |
| PT (1) | PT2638152T (da) |
| RS (1) | RS55405B1 (da) |
| SI (1) | SI2638152T1 (da) |
| SM (1) | SMT201600431B (da) |
| WO (1) | WO2012064709A2 (da) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3127162A1 (en) | 2019-02-01 | 2020-08-06 | Oxyrane Uk Ltd | Glucocerebrosidase polypeptides |
| 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 |
| CA3189801A1 (en) * | 2020-07-29 | 2022-02-03 | Freeline Therapeutics Limited | Polypeptide |
| 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 |
| 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 |
| 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 |
| WO2023198661A1 (en) | 2022-04-12 | 2023-10-19 | F. Hoffmann-La Roche Ag | Fusion proteins targeted to the central nervous system |
| WO2024074142A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of disease associated with gcase deficiency |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE138686T1 (de) | 1988-12-23 | 1996-06-15 | Genzyme Corp | Cho-zellen befähigt enzymatisch aktive rekombinante-glukocerebrosidase zu produzieren |
| 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 |
| 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 |
| IL135578A0 (en) | 1997-10-29 | 2001-05-20 | Genzyme Corp | Compositions and methods 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 |
| CA2405120A1 (en) | 2000-04-06 | 2001-10-18 | 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 |
| 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. |
| KR101597499B1 (ko) * | 2007-05-22 | 2016-02-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 이소파고민과 이의 유도체를 제조하는 새로운 방법 |
| WO2009061369A2 (en) | 2007-11-02 | 2009-05-14 | The Scripps Research Institute | Directed evolution using proteins comprising unnatural amino acids |
| US20090148887A1 (en) | 2007-11-02 | 2009-06-11 | The Scripps Research Institute | Genetically encoded boronate amino acid |
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2011
- 2011-11-08 WO PCT/US2011/059731 patent/WO2012064709A2/en active Application Filing
- 2011-11-08 US US13/884,126 patent/US8962564B2/en active Active
- 2011-11-08 PL PL11839428T patent/PL2638152T3/pl unknown
- 2011-11-08 CA CA2817011A patent/CA2817011C/en active Active
- 2011-11-08 CN CN201510671991.0A patent/CN105296447A/zh active Pending
- 2011-11-08 EP EP16178095.2A patent/EP3144386B1/en active Active
- 2011-11-08 CN CN201180064243.6A patent/CN103314105B/zh active Active
- 2011-11-08 ES ES16178095T patent/ES2743825T3/es active Active
- 2011-11-08 DK DK11839428.7T patent/DK2638152T3/da active
- 2011-11-08 PT PT118394287T patent/PT2638152T/pt unknown
- 2011-11-08 JP JP2013538823A patent/JP6073796B2/ja active Active
- 2011-11-08 LT LTEP11839428.7T patent/LT2638152T/lt unknown
- 2011-11-08 KR KR1020137014671A patent/KR101901467B1/ko active IP Right Grant
- 2011-11-08 EP EP11839428.7A patent/EP2638152B1/en active Active
- 2011-11-08 BR BR112013011152A patent/BR112013011152A2/pt not_active Application Discontinuation
- 2011-11-08 ES ES11839428.7T patent/ES2604490T3/es active Active
- 2011-11-08 RS RS20161010A patent/RS55405B1/sr unknown
- 2011-11-08 HU HUE11839428A patent/HUE030932T2/en unknown
- 2011-11-08 SI SI201131025A patent/SI2638152T1/sl unknown
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2015
- 2015-02-02 US US14/611,495 patent/US9254313B2/en active Active
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2016
- 2016-01-04 US US14/987,204 patent/US9566316B2/en active Active
- 2016-11-23 HR HRP20161560TT patent/HRP20161560T1/hr unknown
- 2016-11-24 SM SM201600431T patent/SMT201600431B/it unknown
- 2016-11-29 CY CY20161101239T patent/CY1118843T1/el unknown
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2017
- 2017-01-05 JP JP2017000301A patent/JP6457559B2/ja active Active
- 2017-02-13 US US15/430,682 patent/US9821038B2/en active Active
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