CA2817011C - Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity - Google Patents
Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity Download PDFInfo
- Publication number
- CA2817011C CA2817011C CA2817011A CA2817011A CA2817011C CA 2817011 C CA2817011 C CA 2817011C CA 2817011 A CA2817011 A CA 2817011A CA 2817011 A CA2817011 A CA 2817011A CA 2817011 C CA2817011 C CA 2817011C
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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
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)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (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)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Hematology (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 (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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2817011A1 CA2817011A1 (en) | 2012-05-18 |
| CA2817011C true CA2817011C (en) | 2019-04-02 |
Family
ID=46051505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2817011A Active CA2817011C (en) | 2010-11-08 | 2011-11-08 | Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity |
Country Status (19)
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 |
| CN113795575A (zh) | 2019-02-04 | 2021-12-14 | 自由行疗法有限公司 | 多核苷酸 |
| US20220325265A1 (en) | 2019-09-09 | 2022-10-13 | Hoffmann-La Roche Inc. | Glucocerebrosidase mutants |
| WO2021199039A1 (en) * | 2020-03-29 | 2021-10-07 | Yeda Research And Development Co. Ltd. | Variants of beta-glucocerebrosidase for use in treating gaucher disease |
| KR20230042513A (ko) * | 2020-07-29 | 2023-03-28 | 프리라인 테라퓨틱스 리미티드 | 폴리펩티드 |
| 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 (cg-RX-API-DMAC10.html) * | 2022-01-31 | 2023-08-03 | ||
| 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 |
| WO2023198661A1 (en) | 2022-04-12 | 2023-10-19 | F. Hoffmann-La Roche Ag | Fusion proteins targeted to the central nervous system |
| CN121406674A (zh) * | 2022-10-08 | 2026-01-27 | 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 |
| AU2024394235A1 (en) * | 2023-12-08 | 2026-06-11 | Denali Therapeutics Inc. | Modified glucocerebrosidase polypeptides and methods of use thereof |
| TW202617982A (zh) * | 2024-06-24 | 2026-05-01 | 美商戴納立製藥公司 | 包含經修飾之葡萄糖腦苷脂酶多肽之融合蛋白及其方法 |
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 |
| JP2893481B2 (ja) | 1988-12-23 | 1999-05-24 | 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 |
| 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 |
| IL150314A0 (en) | 1999-12-30 | 2002-12-01 | 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 |
| AU2008245578A1 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| KR101789000B1 (ko) * | 2007-05-22 | 2017-10-20 | 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 |
| EP2220218A4 (en) | 2007-11-02 | 2010-12-08 | Scripps Research Inst | GENETICALLY ENCODED BORONATE AMINO ACID |
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2011
- 2011-11-08 US US13/884,126 patent/US8962564B2/en active Active
- 2011-11-08 PT PT118394287T patent/PT2638152T/pt unknown
- 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 WO PCT/US2011/059731 patent/WO2012064709A2/en not_active Ceased
- 2011-11-08 ES ES16178095T patent/ES2743825T3/es active Active
- 2011-11-08 PL PL11839428T patent/PL2638152T3/pl unknown
- 2011-11-08 LT LTEP11839428.7T patent/LT2638152T/lt unknown
- 2011-11-08 CN CN201510671991.0A patent/CN105296447A/zh active Pending
- 2011-11-08 KR KR1020137014671A patent/KR101901467B1/ko active Active
- 2011-11-08 SI SI201131025A patent/SI2638152T1/sl unknown
- 2011-11-08 CN CN201180064243.6A patent/CN103314105B/zh not_active Expired - Fee Related
- 2011-11-08 EP EP16178095.2A patent/EP3144386B1/en active Active
- 2011-11-08 DK DK11839428.7T patent/DK2638152T3/en active
- 2011-11-08 JP JP2013538823A patent/JP6073796B2/ja active Active
- 2011-11-08 RS RS20161010A patent/RS55405B1/sr unknown
- 2011-11-08 CA CA2817011A patent/CA2817011C/en active Active
- 2011-11-08 HU HUE11839428A patent/HUE030932T2/en unknown
- 2011-11-08 ES ES11839428.7T patent/ES2604490T3/es active Active
- 2011-11-08 HR HRP20161560TT patent/HRP20161560T1/hr 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
- 2016-11-24 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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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20161102 |
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| MPN | Maintenance fee for patent paid |
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| MPN | Maintenance fee for patent paid |
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| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250916 |
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| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250916 |