CA3129672A1 - Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration - Google Patents

Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration Download PDF

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Publication number
CA3129672A1
CA3129672A1 CA3129672A CA3129672A CA3129672A1 CA 3129672 A1 CA3129672 A1 CA 3129672A1 CA 3129672 A CA3129672 A CA 3129672A CA 3129672 A CA3129672 A CA 3129672A CA 3129672 A1 CA3129672 A1 CA 3129672A1
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Prior art keywords
raav
aav
progranulin
human
grn
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CA3129672A
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English (en)
French (fr)
Inventor
Christian HINDERER
Nimrod Miller
James M. Wilson
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect

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  • 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)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3129672A 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration Pending CA3129672A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962809329P 2019-02-22 2019-02-22
US62/809,329 2019-02-22
US201962923812P 2019-10-21 2019-10-21
US62/923,812 2019-10-21
US202062969108P 2020-02-02 2020-02-02
US62/969,108 2020-02-02
PCT/US2020/019149 WO2020172490A1 (en) 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

Publications (1)

Publication Number Publication Date
CA3129672A1 true CA3129672A1 (en) 2020-08-27

Family

ID=70058449

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3129672A Pending CA3129672A1 (en) 2019-02-22 2020-02-21 Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration

Country Status (16)

Country Link
US (1) US12416017B2 (enExample)
EP (1) EP3927381A1 (enExample)
JP (2) JP7637058B2 (enExample)
KR (1) KR20210131370A (enExample)
CN (1) CN113710281A (enExample)
AU (1) AU2020225472A1 (enExample)
BR (1) BR112021015817A2 (enExample)
CA (1) CA3129672A1 (enExample)
CL (1) CL2021002172A1 (enExample)
CO (1) CO2021011040A2 (enExample)
IL (1) IL285654A (enExample)
MX (1) MX2021010134A (enExample)
PE (1) PE20211819A1 (enExample)
SG (1) SG11202108504YA (enExample)
TW (1) TW202045730A (enExample)
WO (1) WO2020172490A1 (enExample)

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SG11202108504YA (en) 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
EP4204014A1 (en) * 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
WO2022131322A1 (ja) * 2020-12-17 2022-06-23 国立大学法人京都大学 神経細胞賦活剤
EP4473123A2 (en) * 2022-02-01 2024-12-11 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
WO2024245120A1 (en) * 2023-05-26 2024-12-05 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating proteinopathies in central nervous system
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Publication number Publication date
SG11202108504YA (en) 2021-09-29
PE20211819A1 (es) 2021-09-14
MX2021010134A (es) 2021-09-23
KR20210131370A (ko) 2021-11-02
BR112021015817A2 (pt) 2021-10-13
IL285654A (en) 2021-09-30
JP2025081439A (ja) 2025-05-27
CO2021011040A2 (es) 2021-09-09
AU2020225472A1 (en) 2021-08-26
US12416017B2 (en) 2025-09-16
US20220136008A1 (en) 2022-05-05
JP2022523766A (ja) 2022-04-26
TW202045730A (zh) 2020-12-16
WO2020172490A1 (en) 2020-08-27
JP7637058B2 (ja) 2025-02-27
CL2021002172A1 (es) 2022-02-11
CN113710281A (zh) 2021-11-26
EP3927381A1 (en) 2021-12-29

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