Patent ID: 8637045
Filing Date: 2014-01-28
Classification: A61K,A61P

Abstract:
1. A method of supplementing an enzymatic deficiency in a cell comprising the steps of: (a) providing an effective amount of a therapeutic delivery system comprising an extracted yeast cell wall defining an internal space and comprising about 6 to about 90 weight percent beta-glucan, a payload trapping molecule and a payload molecule, wherein the payload molecule is selected from the group consisting of a nucleic acid, a peptide, a protein and a mixture thereof in an amount effective to supplement the function of a deficient lysosomal enzyme, wherein the deficient lysosomal enzyme results in an affected lysosomal function, and wherein the affected lysosomal function is a defective degradation of sphingolipid components related to a lysosomal storage disorder selected from the group consisting of Gaucher disease type 1, Gaucher disease type 2 and Gaucher disease type 3; wherein the payload molecule and the payload trapping molecule are soluble in the same solvent system, wherein the payload trapping molecule is contained within the internal space defined by the extracted yeast cell wall, and wherein the payload trapping molecule is present in an amount sufficient to facilitate retention of the payload molecule within the extracted yeast cell wall; and (b) contacting a cell having the enzymatic deficiency with the therapeutic delivery system.