PL3198018T3 - Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby - Google Patents

Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby

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Publication number
PL3198018T3
PL3198018T3 PL15775884T PL15775884T PL3198018T3 PL 3198018 T3 PL3198018 T3 PL 3198018T3 PL 15775884 T PL15775884 T PL 15775884T PL 15775884 T PL15775884 T PL 15775884T PL 3198018 T3 PL3198018 T3 PL 3198018T3
Authority
PL
Poland
Prior art keywords
adeno
methods
high efficiency
associated virus
virus vector
Prior art date
Application number
PL15775884T
Other languages
English (en)
Inventor
Saswati Chatterjee
Laura Smith
Kamehameha Wong
Original Assignee
City Of Hope
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
Application filed by City Of Hope filed Critical City Of Hope
Publication of PL3198018T3 publication Critical patent/PL3198018T3/pl

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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A—HUMAN NECESSITIES
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
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    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2800/00—Nucleic acids vectors
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Diabetes (AREA)
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  • Obesity (AREA)
  • Communicable Diseases (AREA)
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PL15775884T 2014-09-24 2015-09-23 Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby PL3198018T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462054899P 2014-09-24 2014-09-24
US201462063587P 2014-10-14 2014-10-14
US201562209862P 2015-08-25 2015-08-25
PCT/US2015/051785 WO2016049230A1 (en) 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
EP15775884.8A EP3198018B1 (en) 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

Publications (1)

Publication Number Publication Date
PL3198018T3 true PL3198018T3 (pl) 2021-07-19

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Application Number Title Priority Date Filing Date
PL15775884T PL3198018T3 (pl) 2014-09-24 2015-09-23 Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby

Country Status (15)

Country Link
US (6) US9890396B2 (pl)
EP (2) EP3800260A1 (pl)
JP (3) JP6683691B2 (pl)
KR (2) KR20230067694A (pl)
CN (2) CN113667696A (pl)
AU (2) AU2015320694B2 (pl)
BR (1) BR112017005892A2 (pl)
CA (1) CA2961555A1 (pl)
DK (1) DK3198018T3 (pl)
ES (1) ES2856090T3 (pl)
MX (2) MX388392B (pl)
PL (1) PL3198018T3 (pl)
PT (1) PT3198018T (pl)
SG (3) SG11201702073XA (pl)
WO (1) WO2016049230A1 (pl)

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AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
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SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
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