AU2021280943A1 - Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia - Google Patents

Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia Download PDF

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Publication number
AU2021280943A1
AU2021280943A1 AU2021280943A AU2021280943A AU2021280943A1 AU 2021280943 A1 AU2021280943 A1 AU 2021280943A1 AU 2021280943 A AU2021280943 A AU 2021280943A AU 2021280943 A AU2021280943 A AU 2021280943A AU 2021280943 A1 AU2021280943 A1 AU 2021280943A1
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promoter
seq
functional fragment
viral vector
sequence identity
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AU2021280943A
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Inventor
Martina NUBIE
Janine REICHENBACH
Ulrich Siler
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Zurich Universitaet Institut fuer Medizinische Virologie
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Zurich Universitaet Institut fuer Medizinische Virologie
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    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A—HUMAN NECESSITIES
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    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/193—Colony stimulating factors [CSF]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • C07K14/52—Cytokines; Lymphokines; Interferons
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
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    • C07K14/52—Cytokines; Lymphokines; Interferons
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
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    • C07K14/55—IL-2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/56—IFN-alpha
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/57—IFN-gamma
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
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  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Immunology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
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AU2021280943A 2020-05-27 2021-04-07 Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia Pending AU2021280943A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20176939 2020-05-27
EP20176939.5 2020-05-27
PCT/EP2021/059070 WO2021239308A1 (en) 2020-05-27 2021-04-07 Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia

Publications (1)

Publication Number Publication Date
AU2021280943A1 true AU2021280943A1 (en) 2022-07-28

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AU2021280943A Pending AU2021280943A1 (en) 2020-05-27 2021-04-07 Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia

Country Status (11)

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US (1) US20230227847A1 (https=)
EP (1) EP4158035A1 (https=)
JP (2) JP7820823B2 (https=)
KR (1) KR20230018378A (https=)
CN (1) CN115552019A (https=)
AU (1) AU2021280943A1 (https=)
BR (1) BR112022016715A2 (https=)
CA (1) CA3165462A1 (https=)
IL (1) IL298093A (https=)
MX (1) MX2022014766A (https=)
WO (1) WO2021239308A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12071633B2 (en) 2020-10-13 2024-08-27 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
WO2023109911A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Microglia having car and use thereof
WO2024036106A1 (en) * 2022-08-08 2024-02-15 The Board Of Trustees Of The Leland Stanford Junior University Embryo microglia complementation for in vivo microglia manipulation and production of a non-human animal model for validation of gene function and therapeutic screening

Family Cites Families (12)

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Publication number Priority date Publication date Assignee Title
US6379674B1 (en) 1997-08-12 2002-04-30 Georgetown University Use of herpes vectors for tumor therapy
US20110223635A1 (en) 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
WO2016026854A2 (en) 2014-08-18 2016-02-25 Apceth Gmbh & Co. Kg Genetically modified mesenchymal stem cells expressing an immune response-stimulating cytokine to attract and/or activate immune cells
US20190328906A1 (en) 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
US20180186855A1 (en) * 2016-03-23 2018-07-05 Alector Llc Chimeric receptors and methods of use thereof
WO2018169901A1 (en) * 2017-03-13 2018-09-20 Massachusetts Institute Of Technology Synthetic promoters
BR112020001060A2 (pt) 2017-07-27 2020-07-14 Novartis Ag variantes de trem2 resistentes a shedase
CN109868286B (zh) * 2017-12-05 2022-09-16 深圳先进技术研究院 一种慢病毒载体、慢病毒及其制备方法和应用
JP2021517168A (ja) 2018-03-09 2021-07-15 アブロバイオ，インコーポレーテッド パーキンソン病を処置するための組成物及び方法
US20190300870A1 (en) 2018-04-03 2019-10-03 The Broad Institute, Inc. Target recognition motifs and uses thereof
WO2020160458A1 (en) 2019-02-01 2020-08-06 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
MX2022003934A (es) * 2019-10-02 2022-04-25 Univ Zuerich Tratamiento de la enfermedad granulomatosa cronica.

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KR20230018378A (ko) 2023-02-07
JP7820823B2 (ja) 2026-02-26
JP2023526773A (ja) 2023-06-23
EP4158035A1 (en) 2023-04-05
CN115552019A (zh) 2022-12-30
US20230227847A1 (en) 2023-07-20
BR112022016715A2 (pt) 2022-11-16
JP2026062601A (ja) 2026-04-09
CA3165462A1 (en) 2021-12-02
IL298093A (en) 2023-01-01
MX2022014766A (es) 2023-02-22
WO2021239308A1 (en) 2021-12-02

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