JP7895925B2 - 安定性の改善された変異型ベータ－グルコセレブロシダーゼ - Google Patents

安定性の改善された変異型ベータ－グルコセレブロシダーゼ

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Publication number
JP7895925B2
JP7895925B2 JP2023506111A JP2023506111A JP7895925B2 JP 7895925 B2 JP7895925 B2 JP 7895925B2 JP 2023506111 A JP2023506111 A JP 2023506111A JP 2023506111 A JP2023506111 A JP 2023506111A JP 7895925 B2 JP7895925 B2 JP 7895925B2
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JP
Japan
Prior art keywords
gcase
polypeptide
seq
modified
mutation
Prior art date
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Active
Application number
JP2023506111A
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English (en)
Japanese (ja)
Other versions
JP2023535808A (ja
JP2023535808A5 (enExample
Inventor
カンパー、ファブリツィオ
ナスワニ、アミット
マッキントッシュ、ジェニー
コルバウ、ロムアルド
キア、アザデ
ミランダ、カルロス
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 JP2023535808A publication Critical patent/JP2023535808A/ja
Publication of JP2023535808A5 publication Critical patent/JP2023535808A5/ja
Application granted granted Critical
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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
    • 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
    • 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
    • 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
    • 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

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)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Neurology (AREA)
JP2023506111A 2020-07-29 2021-07-29 安定性の改善された変異型ベータ－グルコセレブロシダーゼ Active JP7895925B2 (ja)

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 (3)

Publication Number Publication Date
JP2023535808A JP2023535808A (ja) 2023-08-21
JP2023535808A5 JP2023535808A5 (enExample) 2024-08-07
JP7895925B2 true JP7895925B2 (ja) 2026-07-28

Family

ID=77265101

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023506111A Active JP7895925B2 (ja) 2020-07-29 2021-07-29 安定性の改善された変異型ベータ－グルコセレブロシダーゼ

Country Status (16)

Country Link
US (1) US20240261433A1 (enExample)
EP (2) EP4643868A3 (enExample)
JP (1) JP7895925B2 (enExample)
KR (1) KR20230042513A (enExample)
CN (1) CN116322744A (enExample)
AU (2) AU2021316875B2 (enExample)
BR (1) BR112023001583A2 (enExample)
CA (1) CA3189801A1 (enExample)
DK (1) DK4118200T3 (enExample)
ES (1) ES3057448T3 (enExample)
FI (1) FI4118200T3 (enExample)
HR (1) HRP20251591T1 (enExample)
IL (1) IL299811A (enExample)
MX (1) MX2023001137A (enExample)
PT (1) PT4118200T (enExample)
WO (1) WO2022023761A2 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024394235A1 (en) 2023-12-08 2026-06-11 Denali Therapeutics Inc. Modified glucocerebrosidase polypeptides and methods of use thereof
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
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001049830A2 (en) 1999-12-30 2001-07-12 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
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 (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CN104383556B (zh) 2007-05-16 2018-06-05 布里格姆妇女医院 突触核蛋白病的治疗
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US9574184B2 (en) * 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
PE20181922A1 (es) * 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
EP3692158A4 (en) 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
AU2019300431A1 (en) 2018-07-13 2021-01-14 Ucl Business Ltd Glucocerebrosidase gene therapy
WO2020157248A1 (en) * 2019-02-01 2020-08-06 Oxyrane Uk Ltd Glucocerebrosidase polypeptides

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001049830A2 (en) 1999-12-30 2001-07-12 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
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
IL299811A (en) 2023-03-01
KR20230042513A (ko) 2023-03-28
EP4118200B1 (en) 2025-10-08
WO2022023761A2 (en) 2022-02-03
AU2026205139A1 (en) 2026-07-16
DK4118200T3 (da) 2025-12-08
AU2021316875A1 (en) 2023-02-23
US20240261433A1 (en) 2024-08-08
EP4118200A2 (en) 2023-01-18
CA3189801A1 (en) 2022-02-03
EP4643868A3 (en) 2026-04-08
WO2022023761A3 (en) 2022-03-10
AU2021316875B2 (en) 2026-04-02
FI4118200T3 (fi) 2025-12-12
ES3057448T3 (en) 2026-03-02
EP4643868A2 (en) 2025-11-05
MX2023001137A (es) 2023-02-22
JP2023535808A (ja) 2023-08-21
CN116322744A (zh) 2023-06-23
PT4118200T (pt) 2026-01-08
HRP20251591T1 (hr) 2026-01-30
BR112023001583A2 (pt) 2023-02-14

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