CA3008975A1 - Human alpha-n-acetylgalactosaminidase polypeptide - Google Patents

Human alpha-n-acetylgalactosaminidase polypeptide Download PDF

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Publication number
CA3008975A1
CA3008975A1 CA3008975A CA3008975A CA3008975A1 CA 3008975 A1 CA3008975 A1 CA 3008975A1 CA 3008975 A CA3008975 A CA 3008975A CA 3008975 A CA3008975 A CA 3008975A CA 3008975 A1 CA3008975 A1 CA 3008975A1
Authority
CA
Canada
Prior art keywords
amino acid
polypeptide
human
nagal
fragment
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3008975A
Other languages
English (en)
French (fr)
Inventor
Wouter Vervecken
Steven Geysens
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Oxyrane UK Ltd
Original Assignee
Oxyrane UK Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Oxyrane UK Ltd filed Critical Oxyrane UK Ltd
Publication of CA3008975A1 publication Critical patent/CA3008975A1/en
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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)
    • C12Y302/01049—Alpha-N-acetylgalactosaminidase (3.2.1.49)
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/80—Vectors or expression systems specially adapted for eukaryotic hosts for fungi
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/80—Vectors or expression systems specially adapted for eukaryotic hosts for fungi
    • C12N15/81—Vectors or expression systems specially adapted for eukaryotic hosts for fungi for yeasts
    • C12N15/815—Vectors or expression systems specially adapted for eukaryotic hosts for fungi for yeasts for yeasts other than Saccharomyces
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
CA3008975A 2015-12-24 2016-12-22 Human alpha-n-acetylgalactosaminidase polypeptide Pending CA3008975A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15202729 2015-12-24
EP15202729.8 2015-12-24
PCT/EP2016/082304 WO2017109034A1 (en) 2015-12-24 2016-12-22 Human alpha-n-acetylgalactosaminidase polypeptide

Publications (1)

Publication Number Publication Date
CA3008975A1 true CA3008975A1 (en) 2017-06-29

Family

ID=55129455

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3008975A Pending CA3008975A1 (en) 2015-12-24 2016-12-22 Human alpha-n-acetylgalactosaminidase polypeptide

Country Status (6)

Country Link
US (1) US11040114B2 (cg-RX-API-DMAC10.html)
EP (1) EP3393501B1 (cg-RX-API-DMAC10.html)
JP (1) JP6956417B2 (cg-RX-API-DMAC10.html)
KR (1) KR20180088919A (cg-RX-API-DMAC10.html)
CA (1) CA3008975A1 (cg-RX-API-DMAC10.html)
WO (1) WO2017109034A1 (cg-RX-API-DMAC10.html)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20180088919A (ko) * 2015-12-24 2018-08-07 옥시레인 유케이 리미티드 인간 알파-n-아세틸갈락토사미니다아제 폴리펩티드
WO2019051297A1 (en) * 2017-09-08 2019-03-14 The Nemours Foundation AGENT, DEVICE AND BLOOD CIRCULATION SYSTEM FOR TREATING LYSOSOMAL DISEASES, AND METHODS OF TREATING LYSOSOMAL DISEASES
EP3773702A2 (en) 2018-04-05 2021-02-17 CureVac AG Novel yellow fever nucleic acid molecules for vaccination
WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4879231A (en) 1984-10-30 1989-11-07 Phillips Petroleum Company Transformation of yeasts of the genus pichia
US4929555A (en) 1987-10-19 1990-05-29 Phillips Petroleum Company Pichia transformation
US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6806084B1 (en) 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0922109A1 (en) 1996-05-21 1999-06-16 Novo Nordisk A/S Novel yeast promoters suitable for expression cloning in yeast and heterologous expression of proteins in yeast
US20060014264A1 (en) 2004-07-13 2006-01-19 Stowers Institute For Medical Research Cre/lox system with lox sites having an extended spacer region
WO2006129080A1 (en) 2005-05-31 2006-12-07 The Institute Of Cancer Research: Royal Cancer Hospital Materials and methods for transducing cells with a viral vector
JP4368925B2 (ja) * 2005-11-18 2009-11-18 財団法人 東京都医学研究機構 基質特異性を変換した新規高機能酵素
JP5967861B2 (ja) 2007-04-03 2016-08-10 オキシレイン ユーケー リミテッド 分子のグリコシル化
US8569032B2 (en) * 2007-05-18 2013-10-29 Tokyo Metropolitan Institute Of Medical Science Proteins having acquired A-galactosidase activity
KR102000383B1 (ko) * 2009-09-29 2019-07-15 유니버시테이트 젠트 만노스-1-포스포-6-만노스 결합의 포스포-6-만노스로의 가수분해
BR112013007616B1 (pt) 2010-09-29 2023-01-24 Oxyrane Uk Limited Métodos para retirar o cap ("uncapping") de porções de manose-1- fosfo-6-manose e desmanosilar n-glicanos fosforilados em uma glicoproteína, para direcionar uma glicoproteína para o interior de uma célula de mamífero, e para converter uma glicoproteína de uma primeira forma que não se liga substancialmente a um receptor de manose-6-fosfato em uma segunda forma que se liga substancialmente a um receptor de manose-6-fosfato em uma célula de mamífero
CA2867237C (en) 2012-03-15 2024-01-02 Wouter Vervecken Methods and materials for treatment of pompe's disease
CA2970638A1 (en) * 2014-12-22 2016-06-30 Codexis, Inc. Human alpha-galactosidase variants
KR20180088919A (ko) * 2015-12-24 2018-08-07 옥시레인 유케이 리미티드 인간 알파-n-아세틸갈락토사미니다아제 폴리펩티드

Also Published As

Publication number Publication date
EP3393501A1 (en) 2018-10-31
JP6956417B2 (ja) 2021-11-02
EP3393501B1 (en) 2023-06-07
WO2017109034A1 (en) 2017-06-29
US20180360991A1 (en) 2018-12-20
JP2019503171A (ja) 2019-02-07
US11040114B2 (en) 2021-06-22
EP3393501C0 (en) 2023-06-07
KR20180088919A (ko) 2018-08-07

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