Vlp for the treatment of a lysosomal storage disease
AssigneeNEUWAY Pharma GmbH
InventorsVictoria DEMINA, Marcus STAPF, Sergey Sotnikov, Heiko MANNINGA
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Abstract
The invention relates to virus like particles (VLP) associated with a lysosomal enzyme or an expression vector encoding a lysosomal enzyme which are used in a method for the treatment of a lysosomal storage disease. The invention also relates to a pharmaceutical composition for use in a method for the treatment of a lysosomal storage disease, to an expression vector encoding a lysosomal enzyme and to a method of associating a VLP with an expression vector encoding a lysosomal enzyme.
Claims48
A method of treating Metachromatic Leukodystrophy Disease (MLD), comprising administering a virus-like particle (VLP) to a subject in need thereof,
- wherein said VLP incorporates a lysosomal enzyme, or incorporates an expression vector comprising a nucleotide sequence encoding a lysosomal enzyme, and
- wherein the VLP is derived from JCV.
An expression vector comprising a nucleotide sequence encoding a lysosomal enzyme, and further comprising a promoter selected from the group consisting of CAG and CMV, wherein said expression vector has a size of less than 7 kb.
A method of incorporating a lysosomal enzyme, or incorporating an expression vector comprising a nucleotide sequence encoding a lysosomal enzyme, into a VLP, wherein the method comprises:
- a) providing a composition comprising VP1 proteins,
- b) exposing the VP1 proteins of the composition of a) to conditions inducing the VP1 to assemble into VLP,
- c) exposing the VLP of the composition of b) to conditions disassembling the VLP into pentamers,
- d) exposing the pentamers of the composition of c) to conditions inducing the pentamers to reassemble into VLP,
- e) exposing the VLP of the composition of d) to conditions disassembling the VLP into pentamers, and
- f) exposing the pentamers of the composition of e) to the lysosomal enzyme or the expression vector to conditions inducing the pentamers to assemble into a VLP associated with the lysosomal enzyme or the expression vector.
31-32. (canceled)
Dependencies are inferred from the claim text.