KR20230042513A - 폴리펩티드 - Google Patents

폴리펩티드 Download PDF

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Publication number
KR20230042513A
KR20230042513A KR1020237007296A KR20237007296A KR20230042513A KR 20230042513 A KR20230042513 A KR 20230042513A KR 1020237007296 A KR1020237007296 A KR 1020237007296A KR 20237007296 A KR20237007296 A KR 20237007296A KR 20230042513 A KR20230042513 A KR 20230042513A
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KR
South Korea
Prior art keywords
gcase
polypeptide
seq
modified
mutation
Prior art date
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Pending
Application number
KR1020237007296A
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English (en)
Korean (ko)
Inventor
파브리지오 콤퍼
아미트 나쓰와니
제니 민토쉬
로무알드 코르바우
아자데흐 키아
카를로스 미란다
Original Assignee
프리라인 테라퓨틱스 리미티드
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.)
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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 프리라인 테라퓨틱스 리미티드 filed Critical 프리라인 테라퓨틱스 리미티드
Publication of KR20230042513A publication Critical patent/KR20230042513A/ko
Pending legal-status Critical Current

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    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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

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  • 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)
  • Food Preservation Except Freezing, Refrigeration, And Drying (AREA)
KR1020237007296A 2020-07-29 2021-07-29 폴리펩티드 Pending KR20230042513A (ko)

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 (1)

Publication Number Publication Date
KR20230042513A true KR20230042513A (ko) 2023-03-28

Family

ID=77265101

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237007296A Pending KR20230042513A (ko) 2020-07-29 2021-07-29 폴리펩티드

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
WO2025122912A1 (en) 2023-12-08 2025-06-12 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
WO2026006173A1 (en) 2024-06-24 2026-01-02 Denali Therapeutics Inc. Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU1775901A (en) * 1999-11-17 2001-05-30 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
SI2638152T1 (sl) * 2010-11-08 2016-12-30 Amicus Therapeutics, Inc. Variantni in rekombinantni proteini beta-glukocerebrozidaze s povišano stabilnostjo in povišano ohranjeno katalitično aktivnostjo
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. 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
KR102450833B1 (ko) * 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
WO2020012149A1 (en) 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy
WO2020012164A1 (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

Also Published As

Publication number Publication date
WO2022023761A2 (en) 2022-02-03
EP4643868A3 (en) 2026-04-08
JP2023535808A (ja) 2023-08-21
ES3057448T3 (en) 2026-03-02
FI4118200T3 (fi) 2025-12-12
CN116322744A (zh) 2023-06-23
EP4118200B1 (en) 2025-10-08
IL299811A (en) 2023-03-01
EP4643868A2 (en) 2025-11-05
BR112023001583A2 (pt) 2023-02-14
PT4118200T (pt) 2026-01-08
CA3189801A1 (en) 2022-02-03
MX2023001137A (es) 2023-02-22
DK4118200T3 (da) 2025-12-08
WO2022023761A3 (en) 2022-03-10
EP4118200A2 (en) 2023-01-18
US20240261433A1 (en) 2024-08-08
AU2021316875A1 (en) 2023-02-23
AU2021316875B2 (en) 2026-04-02
HRP20251591T1 (hr) 2026-01-30

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