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)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (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) | EP2638152B1 (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 (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220125892A1 (en) * | 2019-02-01 | 2022-04-28 | OXYRANE UK Ltd. | Glucocerebrosidase polypeptides |
| FI3850089T3 (fi) | 2019-02-04 | 2024-02-14 | Freeline Therapeutics Ltd | Polynukleotidejä |
| 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 |
| US20230132790A1 (en) * | 2020-03-29 | 2023-05-04 | Yeda Research And Development Co. Ltd. | Variants of beta-glucocerebrosidase for use in treating gaucher disease |
| CA3189801A1 (en) * | 2020-07-29 | 2022-02-03 | Freeline Therapeutics Limited | Polypeptide |
| 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 |
| JPWO2023145813A1 (enExample) * | 2022-01-31 | 2023-08-03 | ||
| US20250145978A1 (en) * | 2022-01-31 | 2025-05-08 | Nippon Shokubai Co., Ltd. | Glycosylated protein having glucocerebrosidase activity |
| 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 |
| WO2025122912A1 (en) * | 2023-12-08 | 2025-06-12 | Denali Therapeutics Inc. | Modified glucocerebrosidase polypeptides and methods of use 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 |
| 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 |
| EP1027069B1 (en) * | 1997-10-29 | 2006-07-26 | Genzyme Corporation | Gene therapy for gaucher disease |
| IL141403A0 (en) * | 1998-08-28 | 2002-03-10 | Univ Duke | Deleted adenoviruses and methods of making and administering the same |
| US20020127219A1 (en) * | 1999-12-30 | 2002-09-12 | Okkels Jens Sigurd | Lysosomal enzymes and lysosomal enzyme activators |
| IL150314A0 (en) * | 1999-12-30 | 2002-12-01 | Maxygen Aps | Improved lysosomal enzymes and lysosomal enzyme activators |
| AU2001253181A1 (en) * | 2000-04-06 | 2001-10-23 | Exegenics, Inc. | Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase) |
| WO2002002597A2 (en) | 2000-06-30 | 2002-01-10 | 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 |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| KR20100017879A (ko) * | 2007-05-22 | 2010-02-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 이소파고민과 이의 유도체를 제조하는 새로운 방법 |
| US20090148887A1 (en) | 2007-11-02 | 2009-06-11 | The Scripps Research Institute | Genetically encoded boronate amino acid |
| BRPI0819092A2 (pt) | 2007-11-02 | 2014-10-14 | Scripps Research Inst | Evolução direcionada usando proteínas que compreendem aminoácidos não naturais. |
-
2011
- 2011-11-08 DK DK11839428.7T patent/DK2638152T3/en active
- 2011-11-08 PT PT118394287T patent/PT2638152T/pt unknown
- 2011-11-08 JP JP2013538823A patent/JP6073796B2/ja active Active
- 2011-11-08 ES ES16178095T patent/ES2743825T3/es active Active
- 2011-11-08 RS RS20161010A patent/RS55405B1/sr unknown
- 2011-11-08 ES ES11839428.7T patent/ES2604490T3/es active Active
- 2011-11-08 KR KR1020137014671A patent/KR101901467B1/ko active Active
- 2011-11-08 LT LTEP11839428.7T patent/LT2638152T/lt unknown
- 2011-11-08 US US13/884,126 patent/US8962564B2/en active Active
- 2011-11-08 BR BR112013011152A patent/BR112013011152A2/pt not_active Application Discontinuation
- 2011-11-08 EP EP11839428.7A patent/EP2638152B1/en active Active
- 2011-11-08 CN CN201180064243.6A patent/CN103314105B/zh active Active
- 2011-11-08 SI SI201131025A patent/SI2638152T1/sl unknown
- 2011-11-08 CN CN201510671991.0A patent/CN105296447A/zh active Pending
- 2011-11-08 PL PL11839428T patent/PL2638152T3/pl unknown
- 2011-11-08 HR HRP20161560TT patent/HRP20161560T1/hr unknown
- 2011-11-08 HU HUE11839428A patent/HUE030932T2/en 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 WO PCT/US2011/059731 patent/WO2012064709A2/en not_active Ceased
-
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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