HK1258917A1 - 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
HK1258917A1
HK1258917A1 HK19101394.9A HK19101394A HK1258917A1 HK 1258917 A1 HK1258917 A1 HK 1258917A1 HK 19101394 A HK19101394 A HK 19101394A HK 1258917 A1 HK1258917 A1 HK 1258917A1
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Hong Kong
Prior art keywords
composition
aav
vector
sequence encoding
sequence
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HK19101394.9A
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English (en)
Chinese (zh)
Inventor
Maclaren Robert
Seabra Miguel
John During Matthew
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Oxford University Innovation Limited
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=43881504&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1258917(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Oxford University Innovation Limited filed Critical Oxford University Innovation Limited
Publication of HK1258917A1 publication Critical patent/HK1258917A1/en

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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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HK19101394.9A 2011-02-22 2014-05-23 Aav-vectors for use in gene therapy of choroideremia HK1258917A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB201103062 2011-02-22
GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
HK14104853.2A Addition HK1191672B (en) 2011-02-22 2012-02-21 Aav-vectors for use in gene therapy of choroideremia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
HK14104853.2A Division HK1191672B (en) 2011-02-22 2012-02-21 Aav-vectors for use in gene therapy of choroideremia

Publications (1)

Publication Number Publication Date
HK1258917A1 true HK1258917A1 (en) 2019-11-22

Family

ID=43881504

Family Applications (2)

Application Number Title Priority Date Filing Date
HK19101394.9A HK1258917A1 (en) 2011-02-22 2014-05-23 Aav-vectors for use in gene therapy of choroideremia
HK18109118.8A HK1249548A1 (zh) 2011-02-22 2018-07-13 用於无脉络膜的基因疗法的aav载体

Family Applications After (1)

Application Number Title Priority Date Filing Date
HK18109118.8A HK1249548A1 (zh) 2011-02-22 2018-07-13 用於无脉络膜的基因疗法的aav载体

Country Status (18)

Country Link
US (4) US9834788B2 (https=)
EP (2) EP3428283A1 (https=)
JP (4) JP2014512171A (https=)
KR (3) KR102139203B1 (https=)
CN (2) CN103562396A (https=)
AU (2) AU2012220404B2 (https=)
BR (1) BR112013021318B1 (https=)
CA (1) CA2827975C (https=)
DK (1) DK2678435T3 (https=)
ES (1) ES2676550T5 (https=)
GB (1) GB201103062D0 (https=)
HK (2) HK1258917A1 (https=)
IL (1) IL228031B (https=)
MX (2) MX356525B (https=)
PL (1) PL2678435T3 (https=)
PT (1) PT2678435T (https=)
SG (1) SG192911A1 (https=)
WO (1) WO2012114090A1 (https=)

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KR20140044793A (ko) 2014-04-15
BR112013021318B1 (pt) 2021-12-07
JP2014512171A (ja) 2014-05-22
PT2678435T (pt) 2018-07-17
GB201103062D0 (en) 2011-04-06
EP3428283A1 (en) 2019-01-16
MX356525B (es) 2018-06-01
AU2012220404A1 (en) 2013-09-12
KR102139203B1 (ko) 2020-07-29
US20180135078A1 (en) 2018-05-17
ES2676550T5 (es) 2024-06-27
JP2020054345A (ja) 2020-04-09
CN103562396A (zh) 2014-02-05
JP2023036618A (ja) 2023-03-14
AU2012220404B2 (en) 2017-04-20
MX2013009604A (es) 2013-09-16
US20210032656A1 (en) 2021-02-04
CN107881198A (zh) 2018-04-06
US9834788B2 (en) 2017-12-05
KR102299430B1 (ko) 2021-09-07
HK1249548A1 (zh) 2018-11-02
WO2012114090A1 (en) 2012-08-30
US20230332176A1 (en) 2023-10-19
IL228031B (en) 2018-02-28
PL2678435T3 (pl) 2018-11-30
US20140107185A1 (en) 2014-04-17
SG192911A1 (en) 2013-09-30
JP2017221192A (ja) 2017-12-21
DK2678435T3 (en) 2018-08-27
ES2676550T3 (es) 2018-07-20
KR20210112407A (ko) 2021-09-14
KR20200092422A (ko) 2020-08-03
MX388146B (es) 2025-03-19
BR112013021318A2 (pt) 2016-10-25
CA2827975A1 (en) 2012-08-30
CA2827975C (en) 2023-06-13
EP2678435A1 (en) 2014-01-01
IL228031A0 (en) 2013-09-30
EP2678435B2 (en) 2024-02-07
AU2017206254A1 (en) 2017-08-10
EP2678435B1 (en) 2018-05-16

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