Novel drug delivery system based on jcv-vlp

Abstract

The invention relates to VLP derived from human polyoma virus loaded with a drug (cargo) as a drug delivery system for transporting said drug into the CNS, in particular of living humans.

1 : A VLP derived from human polyoma virus loaded with a drug for use in a therapeutic or diagnostic procedure as a drug delivery system for transporting said drug into the CNS, in particular of living humans. 
     
     
         2 : The VLP as recited in  claim 1 , whereas the VLP is derived from JCV. 
     
     
         3 : The VLP as recited in  claim 1 , whereas the drug delivery system crosses the blood-brain barrier to enter the CNS together with the drug encapsulated by the VLP. 
     
     
         4 : The VLP as recited in  claim 1 , wherein the drug delivery system crosses the physiologically intact blood-brain barrier. 
     
     
         5 : The VLP as recited in  claim 1 , wherein the VLP is composed of VP1 proteins of JC virus. 
     
     
         6 : The VLP as recited in  claim 1 , wherein the VLP is prepared for intravenous administration. 
     
     
         7 : The VLP as recited in  claim 1 , wherein the VP1 comprises an amino acid sequence which is at least 80% identical to the amino acid sequence according to SEQ ID NO: 1 over its entire length and/or wherein the VP2 comprises an amino acid sequence which is at least 80% identical to the amino acid sequence according to SEQ ID NO: 3 over its entire length. 
     
     
         8 : The VLP as recited in  claim 1 , wherein the drug is selected from the group consisting of a cytotoxic agent, a detectable agent such as a radionuclide, a protein, a peptide and a nucleic acid, in particular selected from the group consisting of nucleic acids encoding a desired protein such as mRNA, cDNA, a plasmid or vector; inhibitory nucleic acids such as siRNA or miRNA; and nucleic acids having catalytic activity such as a ribozyme. 
     
     
         9 : A pharmaceutical composition comprising the VLP as recited in  claim 1 , wherein at least 1%, preferably at least 15%, more preferably at least 50%, in particular preferred at least 95% of the total amount of drug substance (cargo) is fully encapsulated in the hull of the VLP. 
     
     
         10 : The pharmaceutical composition as recited in  claim 9 , wherein the VLP are non-aggregated.