Composition for delivery of genetic material
AssigneeOxford University Innovation Limited
InventorsYiqi Seow, Lydia Alvarez, Matthew Wood
Life of this patent
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Abstract
The present invention relates to exosomes, loaded with genetic material and methods of producing them and to the use of such exosomes for delivering genetic material in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing.
Claims20
A pharmaceutical composition comprising a pharmaceutically acceptable carrier or excipient and an exosome isolated from a cell, wherein the exosome comprises exogenous genetic material loaded into an exosome already isolated from the cell, and the exogenous genetic material comprises a chemically modified oligonucleotide, wherein the oligonucleotide is a morpholino (PMO) or an oligonucleotide comprising at least one chemical modification selected from peptide nucleic acid (PNA), 2′O-methyl, 2′ methoxy-ethyl, phosphoramidate, methylphosphonate, and phosphorothioate.
A pharmaceutical composition comprising a pharmaceutically acceptable carrier or excipient and an exosome isolated from a cell, wherein the exosome comprises exogenousgenetic material loaded into an exosome already isolated from the cell, and the exogenousgenetic material comprises a chemically modified unnatural oligonucleotide, comprising at least one chemical modification selected from peptide nucleic acid (PNA), 2′O-methyl, 2′ methoxy-ethyl, phosphoramidate, methylphosphonate, and phosphorothioate, wherein the exogenousgenetic material is an antisense oligonucleotide, an RNA interference effector moiety, or a morpholino.
A pharmaceutical composition comprising a pharmaceutically acceptable carrier or excipient and an exosome isolated from a cell, wherein the exosome comprises exogenous genetic material loaded into an exosome already isolated from the cell, and the exogenous genetic material comprises a chemically modified oligonucleotide, wherein the exosome has been modified to comprise a targeting moiety expressed on the surface of the exosome which binds to a moiety present on a target cell, wherein the targeting moiety is expressed as a fusion protein with an exosomal transmembrane protein.
Dependencies are inferred from the claim text.