SMT201600431B - Proteine β-glucocerebrosidasi ricombinanti, varianti, con aumentata stabilità e aumentata attività catalitica conservata - Google Patents
Proteine β-glucocerebrosidasi ricombinanti, varianti, con aumentata stabilità e aumentata attività catalitica conservataInfo
- Publication number
- SMT201600431B SMT201600431B SM201600431T SM201600431T SMT201600431B SM T201600431 B SMT201600431 B SM T201600431B SM 201600431 T SM201600431 T SM 201600431T SM 201600431 T SM201600431 T SM 201600431T SM T201600431 B SMT201600431 B SM T201600431B
- Authority
- SM
- San Marino
- Prior art keywords
- increased
- recombinant
- variants
- catalytic activity
- 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
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 (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 |
|---|---|
| SMT201600431B true SMT201600431B (it) | 2017-01-10 |
Family
ID=46051505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SM201600431T SMT201600431B (it) | 2010-11-08 | 2016-11-24 | Proteine β-glucocerebrosidasi ricombinanti, varianti, con aumentata stabilità e aumentata attività catalitica conservata |
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)
| 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)
| 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 |
-
2011
- 2011-11-08 HU HUE11839428A patent/HUE030932T2/en unknown
- 2011-11-08 RS RS20161010A patent/RS55405B1/sr unknown
- 2011-11-08 CN CN201180064243.6A patent/CN103314105B/zh not_active Expired - Fee Related
- 2011-11-08 PT PT118394287T patent/PT2638152T/pt unknown
- 2011-11-08 CA CA2817011A patent/CA2817011C/en active Active
- 2011-11-08 EP EP16178095.2A patent/EP3144386B1/en active Active
- 2011-11-08 PL PL11839428T patent/PL2638152T3/pl unknown
- 2011-11-08 CN CN201510671991.0A patent/CN105296447A/zh active Pending
- 2011-11-08 DK DK11839428.7T patent/DK2638152T3/en active
- 2011-11-08 ES ES16178095T patent/ES2743825T3/es active Active
- 2011-11-08 BR BR112013011152A patent/BR112013011152A2/pt not_active Application Discontinuation
- 2011-11-08 JP JP2013538823A patent/JP6073796B2/ja active Active
- 2011-11-08 US US13/884,126 patent/US8962564B2/en active Active
- 2011-11-08 EP EP11839428.7A patent/EP2638152B1/en active Active
- 2011-11-08 HR HRP20161560TT patent/HRP20161560T1/hr unknown
- 2011-11-08 WO PCT/US2011/059731 patent/WO2012064709A2/en not_active Ceased
- 2011-11-08 LT LTEP11839428.7T patent/LT2638152T/lt unknown
- 2011-11-08 ES ES11839428.7T patent/ES2604490T3/es active Active
- 2011-11-08 SI SI201131025A patent/SI2638152T1/sl unknown
- 2011-11-08 KR KR1020137014671A patent/KR101901467B1/ko active Active
-
2015
- 2015-02-02 US US14/611,495 patent/US9254313B2/en active Active
-
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
-
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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