Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes

Abstract

The present invention provides for compositions and methods for treating, ameliorating or preventing a lysosomal storage disease by administering to a patient suffering from a lysosomal storage disease a P97 conjugated with an enzyme which is capable of transportation into the lysosomes of cells on either sides of the blood brain harrier.

1 - 29 . (canceled) 
     
     
         30 . A pharmaceutical composition, comprising a pharmaceutical carrier and a p97 conjugate, where the p97 conjugate comprises a p97 protein or an active fragment or variant thereof covalently linked to a lysosomal storage disease enzyme selected from iduronate-2-sulfatase and α-L-iduronidase. 
     
     
         31 . The pharmaceutical composition of  claim 30 , where the p97 protein is a soluble p97 protein. 
     
     
         32 . The pharmaceutical composition of  claim 30 , where the p97 protein is a human p97 protein. 
     
     
         33 . The pharmaceutical composition of  claim 32 , where the p97 protein is a human, soluble p97 protein. 
     
     
         34 . The pharmaceutical composition of  claim 30 , where the p97 protein is a biologically active fragment of a human p97 protein. 
     
     
         35 . The pharmaceutical composition of  claim 30 , where the p97 protein is covalently linked to said agent of interest with a linker. 
     
     
         36 . The pharmaceutical composition of  claim 30 , where the conjugate is a fusion protein. 
     
     
         37 . The pharmaceutical composition of  claim 30 , comprising a sodium chloride solution or saline. 
     
     
         38 . The pharmaceutical composition of  claim 30 , comprising a phosphate buffer.