CA2991301A1 - Delivery methods and compositions for nuclease-mediated genome engineering - Google Patents

Delivery methods and compositions for nuclease-mediated genome engineering Download PDF

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Publication number
CA2991301A1
CA2991301A1 CA2991301A CA2991301A CA2991301A1 CA 2991301 A1 CA2991301 A1 CA 2991301A1 CA 2991301 A CA2991301 A CA 2991301A CA 2991301 A CA2991301 A CA 2991301A CA 2991301 A1 CA2991301 A1 CA 2991301A1
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Prior art keywords
cell
nuclease
cells
gene
donor
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CA2991301A
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English (en)
French (fr)
Inventor
Gary K. Lee
Brigit E. RILEY
Susan J. ST. MARTIN
Thomas Wechsler
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
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    • A61K40/42—Cancer antigens
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    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203—Receptors for growth factors
    • A61K40/4209—Hepatocyte growth factor receptor [HGFR] or c-met]
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/27—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by targeting or presenting multiple antigens
    • A61K2239/29—Multispecific CARs
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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    • 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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  • General Chemical & Material Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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CA2991301A 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering Pending CA2991301A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562191918P 2015-07-13 2015-07-13
US62/191,918 2015-07-13
US201562247469P 2015-10-28 2015-10-28
US62/247,469 2015-10-28
US201662315438P 2016-03-30 2016-03-30
US62/315,438 2016-03-30
PCT/US2016/042024 WO2017011519A1 (en) 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering

Publications (1)

Publication Number Publication Date
CA2991301A1 true CA2991301A1 (en) 2017-01-19

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CA2991301A Pending CA2991301A1 (en) 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering

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US (2) US10450585B2 (enExample)
EP (1) EP3322297B1 (enExample)
JP (2) JP6954890B2 (enExample)
CN (1) CN108024544B (enExample)
AU (1) AU2016291778B2 (enExample)
CA (1) CA2991301A1 (enExample)
WO (1) WO2017011519A1 (enExample)

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