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US 2018/0296700 A1Published application
Novel drug delivery system based on jcv-vlp
AssigneeLife Science Inkubator Betriebs GmbH & Co. KG
InventorsVictoria Demina, Heiko Manninga, Armin Goetzke, Alexander Glassmann
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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.
Claims10
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.