Exosomes For Delivery Of Biotherapeutics

Abstract

The present invention relates to exosomes, loaded with biotherapeutic protein and/or peptide and methods of producing them and to the use of such exosomes for delivering protein and/or peptide in vivo, in particular the use of such exosomes in methods of therapy.

1 . (canceled) 
     
     
         2 . A composition comprising a therapeutic delivery exosome and a pharmaceutically acceptable carrier, wherein the exosome is loaded with a n exogenous recombinant therapeutic protein, and wherein the exogenous recombinant therapeutic protein is encapsulated inside the exosome and does not elicit an immune response when inside the exosome, and wherein the exosome further comprises a targeting moiety expressed on the surface of the exosome. 
     
     
         3 . The composition of  claim 2 , wherein the targeting moiety is 5-100 amino acids in length. 
     
     
         4 . The composition of  claim 2 , wherein the targeting moiety comprises an antibody, scFv, nanobody, muscle specific protein, rabies glycoprotein or NGF. 
     
     
         5 . The composition of  claim 2 , wherein the targeting moiety comprises a peptide which binds to a moiety present on a cell type to be targeted. 
     
     
         6 . The composition of  claim 5 , wherein the exosome comprises an exosomal transmembrane protein which has been modified to incorporate the targeting moiety. 
     
     
         7 . The composition of  claim 6 , wherein the exosomal transmembrane protein is Lamp-1, Lamp-2, CD 13, CD86, Flotillin, Syntaxin-3, CD2, CD36, CD40, CD40L, CD41a, CD44, CD45, ICAM-1, Integrin alpha4, LiCAM, LFA-1, Mac-1 alpha and beta, Vti-1A and B, CD3 epsilon and zeta, CD9, CD18, CD37, CD53, CD63, CD81, CD82, CXCR4 or tetraspanins. 
     
     
         8 . A method of producing a therapeutic delivery exosome with an exogenous therapeutic protein comprising (a) transforming or transfecting a host cell with a nucleic acid which expresses the therapeutic protein and a nucleic acid that expresses a targeting moiety and (b) producing an exosome from the host cell, wherein the exosome comprises the therapeutic protein and the targeting moiety, and wherein the therapeutic protein is encapsulated inside the exosome and does not elicit an immune response when inside the exosome, and wherein the targeting moiety is expressed on the surface of the exosome.