BR112013021318B1 - Vetor e usos de um vetor - Google Patents

Vetor e usos de um vetor Download PDF

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Publication number
BR112013021318B1
BR112013021318B1 BR112013021318-3A BR112013021318A BR112013021318B1 BR 112013021318 B1 BR112013021318 B1 BR 112013021318B1 BR 112013021318 A BR112013021318 A BR 112013021318A BR 112013021318 B1 BR112013021318 B1 BR 112013021318B1
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BR
Brazil
Prior art keywords
vector
aav
rep1
genome
sequence
Prior art date
Application number
BR112013021318-3A
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English (en)
Portuguese (pt)
Other versions
BR112013021318A2 (pt
Inventor
Robert Maclaren
Matthew John During
Miguel Seabra
Original Assignee
Isis Innovation Limited
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
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=BR112013021318(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Isis Innovation Limited filed Critical Isis Innovation Limited
Publication of BR112013021318A2 publication Critical patent/BR112013021318A2/pt
Publication of BR112013021318B1 publication Critical patent/BR112013021318B1/pt

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    • C—CHEMISTRY; METALLURGY
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Mycology (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)
BR112013021318-3A 2011-02-22 2012-02-21 Vetor e usos de um vetor BR112013021318B1 (pt)

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
BR112013021318A2 BR112013021318A2 (pt) 2016-10-25
BR112013021318B1 true BR112013021318B1 (pt) 2021-12-07

Family

ID=43881504

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112013021318-3A BR112013021318B1 (pt) 2011-02-22 2012-02-21 Vetor e usos de um vetor

Country Status (18)

Country Link
US (4) US9834788B2 (cg-RX-API-DMAC7.html)
EP (2) EP2678435B2 (cg-RX-API-DMAC7.html)
JP (4) JP2014512171A (cg-RX-API-DMAC7.html)
KR (3) KR102299430B1 (cg-RX-API-DMAC7.html)
CN (2) CN103562396A (cg-RX-API-DMAC7.html)
AU (2) AU2012220404B2 (cg-RX-API-DMAC7.html)
BR (1) BR112013021318B1 (cg-RX-API-DMAC7.html)
CA (1) CA2827975C (cg-RX-API-DMAC7.html)
DK (1) DK2678435T3 (cg-RX-API-DMAC7.html)
ES (1) ES2676550T5 (cg-RX-API-DMAC7.html)
GB (1) GB201103062D0 (cg-RX-API-DMAC7.html)
HK (2) HK1258917A1 (cg-RX-API-DMAC7.html)
IL (1) IL228031B (cg-RX-API-DMAC7.html)
MX (2) MX388146B (cg-RX-API-DMAC7.html)
PL (1) PL2678435T3 (cg-RX-API-DMAC7.html)
PT (1) PT2678435T (cg-RX-API-DMAC7.html)
SG (1) SG192911A1 (cg-RX-API-DMAC7.html)
WO (1) WO2012114090A1 (cg-RX-API-DMAC7.html)

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WO2015082690A1 (en) * 2013-12-06 2015-06-11 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
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US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
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WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
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EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP7766393B2 (ja) 2017-06-14 2025-11-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
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Also Published As

Publication number Publication date
KR20140044793A (ko) 2014-04-15
HK1258917A1 (en) 2019-11-22
CN103562396A (zh) 2014-02-05
CN107881198A (zh) 2018-04-06
JP2017221192A (ja) 2017-12-21
AU2012220404B2 (en) 2017-04-20
US20180135078A1 (en) 2018-05-17
KR102299430B1 (ko) 2021-09-07
MX388146B (es) 2025-03-19
MX356525B (es) 2018-06-01
CA2827975C (en) 2023-06-13
CA2827975A1 (en) 2012-08-30
HK1249548A1 (zh) 2018-11-02
EP2678435A1 (en) 2014-01-01
JP2020054345A (ja) 2020-04-09
ES2676550T5 (es) 2024-06-27
AU2012220404A1 (en) 2013-09-12
US20140107185A1 (en) 2014-04-17
EP2678435B2 (en) 2024-02-07
PL2678435T3 (pl) 2018-11-30
KR20200092422A (ko) 2020-08-03
IL228031A0 (en) 2013-09-30
GB201103062D0 (en) 2011-04-06
US20230332176A1 (en) 2023-10-19
SG192911A1 (en) 2013-09-30
MX2013009604A (es) 2013-09-16
KR102139203B1 (ko) 2020-07-29
AU2017206254A1 (en) 2017-08-10
IL228031B (en) 2018-02-28
US20210032656A1 (en) 2021-02-04
JP2014512171A (ja) 2014-05-22
JP2023036618A (ja) 2023-03-14
KR20210112407A (ko) 2021-09-14
US9834788B2 (en) 2017-12-05
PT2678435T (pt) 2018-07-17
DK2678435T3 (en) 2018-08-27
EP3428283A1 (en) 2019-01-16
ES2676550T3 (es) 2018-07-20
BR112013021318A2 (pt) 2016-10-25
WO2012114090A1 (en) 2012-08-30
EP2678435B1 (en) 2018-05-16

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