AU2014354839B2 - Adeno-associated virus vectors for treatment of glycogen storage disease - Google Patents

Adeno-associated virus vectors for treatment of glycogen storage disease Download PDF

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AU2014354839B2
AU2014354839B2 AU2014354839A AU2014354839A AU2014354839B2 AU 2014354839 B2 AU2014354839 B2 AU 2014354839B2 AU 2014354839 A AU2014354839 A AU 2014354839A AU 2014354839 A AU2014354839 A AU 2014354839A AU 2014354839 B2 AU2014354839 B2 AU 2014354839B2
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g6pc
aav
mice
gpe
raav
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AU2014354839A1 (en
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Barry J. Byrne
Janice J. Chou
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University of Florida Research Foundation Inc
US Department of Health and Human Services
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Government of the United States of America
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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
    • 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
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
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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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    • 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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    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Genetics & Genomics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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AU2014354839A 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease Active AU2014354839B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361908861P 2013-11-26 2013-11-26
US61/908,861 2013-11-26
PCT/US2014/067415 WO2015081101A1 (en) 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease

Publications (2)

Publication Number Publication Date
AU2014354839A1 AU2014354839A1 (en) 2016-06-09
AU2014354839B2 true AU2014354839B2 (en) 2020-06-04

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AU2014354839A Active AU2014354839B2 (en) 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease

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US (3) US9644216B2 (enExample)
EP (2) EP3415620B1 (enExample)
JP (1) JP6649265B2 (enExample)
CN (1) CN105934515B (enExample)
AU (1) AU2014354839B2 (enExample)
CA (1) CA2930872C (enExample)
ES (1) ES2690643T3 (enExample)
IL (1) IL245659B (enExample)
MX (1) MX375360B (enExample)
WO (1) WO2015081101A1 (enExample)

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AU2018212002A1 (en) * 2017-01-30 2019-08-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Recombinant virus vectors for the treatment of glycogen storage disease
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CN105934515B (zh) 2020-08-04
EP3074510B1 (en) 2018-08-01
IL245659A0 (en) 2016-06-30
JP2016538885A (ja) 2016-12-15
EP3074510A1 (en) 2016-10-05
MX2016006774A (es) 2016-10-13
WO2015081101A8 (en) 2016-06-16
AU2014354839A1 (en) 2016-06-09
CA2930872C (en) 2022-05-31
IL245659B (en) 2020-01-30
WO2015081101A1 (en) 2015-06-04
ES2690643T3 (es) 2018-11-21
EP3415620A1 (en) 2018-12-19
US20160376608A1 (en) 2016-12-29
CA2930872A1 (en) 2015-06-04
US11060110B2 (en) 2021-07-13
BR112016011997A2 (pt) 2017-09-26
US10113183B2 (en) 2018-10-30
EP3415620B1 (en) 2021-03-31
US9644216B2 (en) 2017-05-09
CN105934515A (zh) 2016-09-07
US20190017069A1 (en) 2019-01-17
JP6649265B2 (ja) 2020-02-19
MX375360B (es) 2025-03-06
US20170233763A1 (en) 2017-08-17

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