PT2638152T - Proteínas beta-glucocerebrosidase recombinantes, variantes com estabilidade aumentada e atividade catalítica residual aumentada - Google Patents
Proteínas beta-glucocerebrosidase recombinantes, variantes com estabilidade aumentada e atividade catalítica residual aumentadaInfo
- Publication number
- PT2638152T PT2638152T PT118394287T PT11839428T PT2638152T PT 2638152 T PT2638152 T PT 2638152T PT 118394287 T PT118394287 T PT 118394287T PT 11839428 T PT11839428 T PT 11839428T PT 2638152 T PT2638152 T PT 2638152T
- Authority
- PT
- Portugal
- Prior art keywords
- increased
- variant
- catalytic activity
- recombinant beta
- glucocerebrosidase proteins
- Prior art date
Links
- 102000004547 Glucosylceramidase Human genes 0.000 title 1
- 108010017544 Glucosylceramidase Proteins 0.000 title 1
- 230000003197 catalytic effect Effects 0.000 title 1
- 230000000717 retained effect Effects 0.000 title 1
Classifications
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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
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)
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 |
|---|---|
| PT2638152T true PT2638152T (pt) | 2016-10-25 |
Family
ID=46051505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT118394287T PT2638152T (pt) | 2010-11-08 | 2011-11-08 | Proteínas beta-glucocerebrosidase recombinantes, variantes com estabilidade aumentada e atividade catalítica residual aumentada |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US8962564B2 (https=) |
| EP (2) | EP2638152B1 (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) | ES2604490T3 (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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220125892A1 (en) * | 2019-02-01 | 2022-04-28 | OXYRANE UK Ltd. | Glucocerebrosidase polypeptides |
| BR112021015312A2 (pt) | 2019-02-04 | 2021-11-09 | Freeline Therapeutics Ltd | Polinucleotídeos, partícula viral e composição |
| US20220325265A1 (en) | 2019-09-09 | 2022-10-13 | Hoffmann-La Roche Inc. | Glucocerebrosidase mutants |
| KR20230003492A (ko) * | 2020-03-29 | 2023-01-06 | 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 | 고셔병 치료용 베타-글루코세레브로시다아제 변이체 |
| EP4118200B1 (en) * | 2020-07-29 | 2025-10-08 | Spur Therapeutics Limited | Mutated beta-glucocerebrosidase with improved stability |
| JPWO2023145812A1 (https=) * | 2022-01-31 | 2023-08-03 | ||
| US20250129351A1 (en) * | 2022-01-31 | 2025-04-24 | Nippon Shokubai Co., Ltd. | Recombinant glucocerebrosidase protein having improved enzyme activity or improved stability |
| US20250145979A1 (en) * | 2022-01-31 | 2025-05-08 | Nippon Shokubai Co., Ltd. | Protein having glucocerebrosidase activity and method for producing same |
| 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 |
| WO2025122912A1 (en) * | 2023-12-08 | 2025-06-12 | Denali Therapeutics Inc. | Modified glucocerebrosidase polypeptides and methods of use thereof |
| WO2026006173A1 (en) * | 2024-06-24 | 2026-01-02 | Denali Therapeutics Inc. | Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof |
Family Cites Families (16)
| 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 |
| WO1990007573A1 (en) | 1988-12-23 | 1990-07-12 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
| 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 |
| 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 |
| CA2340276A1 (en) | 1998-08-28 | 2000-03-09 | Duke University | Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences |
| AU2352201A (en) | 1999-12-30 | 2001-07-16 | 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 |
| 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 |
| EP2150254A4 (en) * | 2007-04-26 | 2010-11-10 | Amicus Therapeutics Inc | DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES |
| KR101650726B1 (ko) * | 2007-05-22 | 2016-08-24 | 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 |
| WO2009059056A2 (en) | 2007-11-02 | 2009-05-07 | The Scripps Research Institute | A genetically encoded boronate amino acid |
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2011
- 2011-11-08 SI SI201131025A patent/SI2638152T1/sl unknown
- 2011-11-08 KR KR1020137014671A patent/KR101901467B1/ko active Active
- 2011-11-08 US US13/884,126 patent/US8962564B2/en active Active
- 2011-11-08 DK DK11839428.7T patent/DK2638152T3/en active
- 2011-11-08 HR HRP20161560TT patent/HRP20161560T1/hr unknown
- 2011-11-08 EP EP11839428.7A patent/EP2638152B1/en active Active
- 2011-11-08 PL PL11839428T patent/PL2638152T3/pl unknown
- 2011-11-08 ES ES11839428.7T patent/ES2604490T3/es active Active
- 2011-11-08 HU HUE11839428A patent/HUE030932T2/en unknown
- 2011-11-08 ES ES16178095T patent/ES2743825T3/es active Active
- 2011-11-08 EP EP16178095.2A patent/EP3144386B1/en active Active
- 2011-11-08 RS RS20161010A patent/RS55405B1/sr unknown
- 2011-11-08 CA CA2817011A patent/CA2817011C/en active Active
- 2011-11-08 PT PT118394287T patent/PT2638152T/pt unknown
- 2011-11-08 WO PCT/US2011/059731 patent/WO2012064709A2/en not_active Ceased
- 2011-11-08 JP JP2013538823A patent/JP6073796B2/ja active Active
- 2011-11-08 LT LTEP11839428.7T patent/LT2638152T/lt unknown
- 2011-11-08 CN CN201510671991.0A patent/CN105296447A/zh active Pending
- 2011-11-08 BR BR112013011152A patent/BR112013011152A2/pt not_active Application Discontinuation
- 2011-11-08 CN CN201180064243.6A patent/CN103314105B/zh not_active Expired - Fee Related
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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
- 2016-11-24 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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