AU2016226289B2 - Compositions and methods for intravitreal delivery of polynucleotides to retinal cones - Google Patents

Compositions and methods for intravitreal delivery of polynucleotides to retinal cones Download PDF

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AU2016226289B2
AU2016226289B2 AU2016226289A AU2016226289A AU2016226289B2 AU 2016226289 B2 AU2016226289 B2 AU 2016226289B2 AU 2016226289 A AU2016226289 A AU 2016226289A AU 2016226289 A AU2016226289 A AU 2016226289A AU 2016226289 B2 AU2016226289 B2 AU 2016226289B2
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AU2016226289A1 (en
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Thomas W. Chalberg
Jay Neitz
Maureen Neitz
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University of Washington
Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P27/02—Ophthalmic agents
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    • C07K—PEPTIDES
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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
    • C12N15/861—Adenoviral vectors
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2016226289A 2015-03-02 2016-03-02 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones Active AU2016226289B2 (en)

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AU2021209206A AU2021209206A1 (en) 2015-03-02 2021-07-27 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

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US201562127194P 2015-03-02 2015-03-02
US62/127,194 2015-03-02
US201562134466P 2015-03-17 2015-03-17
US62/134,466 2015-03-17
PCT/US2016/020482 WO2016141078A1 (en) 2015-03-02 2016-03-02 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

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US (2) US11021519B2 (OSRAM)
EP (1) EP3265178A4 (OSRAM)
JP (3) JP7095994B2 (OSRAM)
KR (1) KR20170137730A (OSRAM)
CN (2) CN107405507B (OSRAM)
AU (2) AU2016226289B2 (OSRAM)
BR (1) BR112017018846A2 (OSRAM)
CA (1) CA2978068C (OSRAM)
EA (1) EA201791939A1 (OSRAM)
HK (1) HK1249067A1 (OSRAM)
IL (1) IL254148B (OSRAM)
MX (1) MX2017011041A (OSRAM)
MY (1) MY187898A (OSRAM)
SG (1) SG11201707063TA (OSRAM)
WO (1) WO2016141078A1 (OSRAM)

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