AU2021316875B2 - Mutated beta-glucocerebrosidase with improved stability - Google Patents

Mutated beta-glucocerebrosidase with improved stability

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Publication number
AU2021316875B2
AU2021316875B2 AU2021316875A AU2021316875A AU2021316875B2 AU 2021316875 B2 AU2021316875 B2 AU 2021316875B2 AU 2021316875 A AU2021316875 A AU 2021316875A AU 2021316875 A AU2021316875 A AU 2021316875A AU 2021316875 B2 AU2021316875 B2 AU 2021316875B2
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AU
Australia
Prior art keywords
gcase
polypeptide
seq
modified
mutation
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.)
Active
Application number
AU2021316875A
Other languages
English (en)
Other versions
AU2021316875A1 (en
Inventor
Fabrizio Comper
Romuald Corbau
Azadeh Kia
Jenny MCINTOSH
Carlos Miranda
Amit Nathwani
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.)
Spur Therapeutics Ltd
Original Assignee
Spur Therapeutics 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
Priority claimed from GBGB2011813.9A external-priority patent/GB202011813D0/en
Priority claimed from GBGB2100648.1A external-priority patent/GB202100648D0/en
Priority claimed from GBGB2105924.1A external-priority patent/GB202105924D0/en
Application filed by Spur Therapeutics Ltd filed Critical Spur Therapeutics Ltd
Publication of AU2021316875A1 publication Critical patent/AU2021316875A1/en
Assigned to SPUR THERAPEUTICS LIMITED reassignment SPUR THERAPEUTICS LIMITED Amend patent request/document other than specification (104) Assignors: FREELINE THERAPEUTICS LIMITED
Application granted granted Critical
Publication of AU2021316875B2 publication Critical patent/AU2021316875B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Removal Of Specific Substances (AREA)
AU2021316875A 2020-07-29 2021-07-29 Mutated beta-glucocerebrosidase with improved stability Active AU2021316875B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
GBGB2011813.9A GB202011813D0 (en) 2020-07-29 2020-07-29 Polypeptide
GB2011813.9 2020-07-29
GBGB2100648.1A GB202100648D0 (en) 2021-01-18 2021-01-18 Polypeptide
GB2100648.1 2021-01-18
GB2105924.1 2021-04-26
GBGB2105924.1A GB202105924D0 (en) 2021-04-26 2021-04-26 Polypeptide
PCT/GB2021/051969 WO2022023761A2 (en) 2020-07-29 2021-07-29 Polypeptide

Publications (2)

Publication Number Publication Date
AU2021316875A1 AU2021316875A1 (en) 2023-02-23
AU2021316875B2 true AU2021316875B2 (en) 2026-04-02

Family

ID=77265101

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021316875A Active AU2021316875B2 (en) 2020-07-29 2021-07-29 Mutated beta-glucocerebrosidase with improved stability

Country Status (16)

Country Link
US (1) US20240261433A1 (https=)
EP (2) EP4118200B1 (https=)
JP (1) JP2023535808A (https=)
KR (1) KR20230042513A (https=)
CN (1) CN116322744A (https=)
AU (1) AU2021316875B2 (https=)
BR (1) BR112023001583A2 (https=)
CA (1) CA3189801A1 (https=)
DK (1) DK4118200T3 (https=)
ES (1) ES3057448T3 (https=)
FI (1) FI4118200T3 (https=)
HR (1) HRP20251591T1 (https=)
IL (1) IL299811A (https=)
MX (1) MX2023001137A (https=)
PT (1) PT4118200T (https=)
WO (1) WO2022023761A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2025166045A1 (en) 2024-01-31 2025-08-07 Alector Llc β-GLUCOCEREBROSIDASE ENZYMES, FUSION PROTEINS AND COMPLEXES COMPRISING THE SAME, AND METHODS OF USE THEREOF
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框
WO2026006173A1 (en) 2024-06-24 2026-01-02 Denali Therapeutics Inc. Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012064709A2 (en) * 2010-11-08 2012-05-18 Callidus Biopharma, Inc. Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
AU2352201A (en) * 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9574184B2 (en) * 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
WO2020012149A1 (en) 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy
US20220125892A1 (en) * 2019-02-01 2022-04-28 OXYRANE UK Ltd. Glucocerebrosidase polypeptides

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012064709A2 (en) * 2010-11-08 2012-05-18 Callidus Biopharma, Inc. Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Also Published As

Publication number Publication date
US20240261433A1 (en) 2024-08-08
EP4643868A3 (en) 2026-04-08
EP4643868A2 (en) 2025-11-05
ES3057448T3 (en) 2026-03-02
KR20230042513A (ko) 2023-03-28
MX2023001137A (es) 2023-02-22
HRP20251591T1 (hr) 2026-01-30
PT4118200T (pt) 2026-01-08
EP4118200B1 (en) 2025-10-08
WO2022023761A2 (en) 2022-02-03
BR112023001583A2 (pt) 2023-02-14
IL299811A (en) 2023-03-01
CA3189801A1 (en) 2022-02-03
FI4118200T3 (fi) 2025-12-12
EP4118200A2 (en) 2023-01-18
AU2021316875A1 (en) 2023-02-23
DK4118200T3 (da) 2025-12-08
JP2023535808A (ja) 2023-08-21
WO2022023761A3 (en) 2022-03-10
CN116322744A (zh) 2023-06-23

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