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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Canada
Prior art keywords
raav
aav
progranulin
human
grn
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Pending
Application number
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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Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CA3129672A1 publication Critical patent/CA3129672A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P25/28—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2750/14171—Demonstrated 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 (2) US12416017B2 (https=)
EP (1) EP3927381A1 (https=)
JP (2) JP7637058B2 (https=)
KR (1) KR20210131370A (https=)
CN (1) CN113710281A (https=)
AU (1) AU2020225472A1 (https=)
BR (1) BR112021015817A2 (https=)
CA (1) CA3129672A1 (https=)
CL (1) CL2021002172A1 (https=)
CO (1) CO2021011040A2 (https=)
IL (1) IL285654A (https=)
MX (1) MX2021010134A (https=)
PE (1) PE20211819A1 (https=)
SG (1) SG11202108504YA (https=)
TW (1) TW202045730A (https=)
WO (1) WO2020172490A1 (https=)

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GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
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
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
EP4473123A4 (en) * 2022-02-01 2026-03-11 Shape Therapeutics Inc STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION
WO2024245120A1 (en) * 2023-05-26 2024-12-05 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating proteinopathies in central nervous system
WO2024263567A2 (en) * 2023-06-21 2024-12-26 Eli Lilly And Company Compounds for the delivery of granulin across the blood brain barrier
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Also Published As

Publication number Publication date
EP3927381A1 (en) 2021-12-29
BR112021015817A2 (pt) 2021-10-13
AU2020225472A1 (en) 2021-08-26
PE20211819A1 (es) 2021-09-14
US12416017B2 (en) 2025-09-16
JP2022523766A (ja) 2022-04-26
MX2021010134A (es) 2021-09-23
CL2021002172A1 (es) 2022-02-11
CN113710281A (zh) 2021-11-26
US20220136008A1 (en) 2022-05-05
IL285654A (en) 2021-09-30
CO2021011040A2 (es) 2021-09-09
JP2025081439A (ja) 2025-05-27
TW202045730A (zh) 2020-12-16
KR20210131370A (ko) 2021-11-02
WO2020172490A1 (en) 2020-08-27
JP7637058B2 (ja) 2025-02-27
SG11202108504YA (en) 2021-09-29
US20260049335A1 (en) 2026-02-19

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