CA2827975C - Aav-vectors for use in gene therapy of choroideremia - Google Patents

Aav-vectors for use in gene therapy of choroideremia Download PDF

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Publication number
CA2827975C
CA2827975C CA2827975A CA2827975A CA2827975C CA 2827975 C CA2827975 C CA 2827975C CA 2827975 A CA2827975 A CA 2827975A CA 2827975 A CA2827975 A CA 2827975A CA 2827975 C CA2827975 C CA 2827975C
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vector
aav2
seq
composition
sequence encoding
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French (fr)
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CA2827975A1 (en
Inventor
Robert MACLAREN
Miguel SEABRA
Matthew John During
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Oxford University Innovation Ltd
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Oxford University Innovation Ltd
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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
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/12Viral antigens
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1085Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CA2827975A 2011-02-22 2012-02-21 Aav-vectors for use in gene therapy of choroideremia Active CA2827975C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method
GB1103062.4 2011-02-22
PCT/GB2012/050376 WO2012114090A1 (en) 2011-02-22 2012-02-21 Aav -vectors for use in gene therapy of choroideremia

Publications (2)

Publication Number Publication Date
CA2827975A1 CA2827975A1 (en) 2012-08-30
CA2827975C true CA2827975C (en) 2023-06-13

Family

ID=43881504

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2827975A Active CA2827975C (en) 2011-02-22 2012-02-21 Aav-vectors for use in gene therapy of choroideremia

Country Status (18)

Country Link
US (4) US9834788B2 (enExample)
EP (2) EP2678435B2 (enExample)
JP (4) JP2014512171A (enExample)
KR (3) KR20210112407A (enExample)
CN (2) CN107881198A (enExample)
AU (2) AU2012220404B2 (enExample)
BR (1) BR112013021318B1 (enExample)
CA (1) CA2827975C (enExample)
DK (1) DK2678435T3 (enExample)
ES (1) ES2676550T5 (enExample)
GB (1) GB201103062D0 (enExample)
HK (2) HK1258917A1 (enExample)
IL (1) IL228031B (enExample)
MX (2) MX356525B (enExample)
PL (1) PL2678435T3 (enExample)
PT (1) PT2678435T (enExample)
SG (1) SG192911A1 (enExample)
WO (1) WO2012114090A1 (enExample)

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RU2723101C2 (ru) * 2013-12-06 2020-06-08 Инсерм (Инститьют Насьонал Де Ла Санте Ет Де Ла Решерш Медикаль) Вектор, способ лечения хороидермии, способ селективной экспрессии полинуклеотида
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MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
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Also Published As

Publication number Publication date
CN107881198A (zh) 2018-04-06
MX2013009604A (es) 2013-09-16
JP2020054345A (ja) 2020-04-09
DK2678435T3 (en) 2018-08-27
KR20210112407A (ko) 2021-09-14
AU2017206254A1 (en) 2017-08-10
AU2012220404B2 (en) 2017-04-20
ES2676550T5 (es) 2024-06-27
CN103562396A (zh) 2014-02-05
PL2678435T3 (pl) 2018-11-30
US20140107185A1 (en) 2014-04-17
US9834788B2 (en) 2017-12-05
BR112013021318B1 (pt) 2021-12-07
MX388146B (es) 2025-03-19
KR102299430B1 (ko) 2021-09-07
EP2678435B2 (en) 2024-02-07
WO2012114090A1 (en) 2012-08-30
PT2678435T (pt) 2018-07-17
JP2014512171A (ja) 2014-05-22
EP3428283A1 (en) 2019-01-16
EP2678435B1 (en) 2018-05-16
KR102139203B1 (ko) 2020-07-29
KR20200092422A (ko) 2020-08-03
AU2012220404A1 (en) 2013-09-12
SG192911A1 (en) 2013-09-30
GB201103062D0 (en) 2011-04-06
IL228031A0 (en) 2013-09-30
MX356525B (es) 2018-06-01
US20210032656A1 (en) 2021-02-04
CA2827975A1 (en) 2012-08-30
JP2023036618A (ja) 2023-03-14
BR112013021318A2 (pt) 2016-10-25
EP2678435A1 (en) 2014-01-01
US20180135078A1 (en) 2018-05-17
IL228031B (en) 2018-02-28
ES2676550T3 (es) 2018-07-20
HK1249548A1 (zh) 2018-11-02
US20230332176A1 (en) 2023-10-19
HK1258917A1 (en) 2019-11-22
KR20140044793A (ko) 2014-04-15
JP2017221192A (ja) 2017-12-21

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