Abstract: The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.
Type:
Application
Filed:
August 19, 2013
Publication date:
February 20, 2014
Applicant:
ORPHAZYME APS
Inventors:
Thomas Kirkegaard Jensen, Marja H. Jaattela
Abstract: The present invention relates to a bioactive agent capable of increasing the intracellular concentration and/or activity of Hsp70 for use in the treatment of a lysosomal storage disease which arise from a defect in an enzyme whose activity is not directly associated with the presence of lysosomal BMP as a co-factor; such as glycogen storage diseases, gangliosidoses, neuronal ceroid lipofuscinoses, cerebrotendinous cholesterosis, Wolman's disease, cholesteryl ester storage disease, disorders of glycosaminoglycan metabolism, mucopolysaccharidoses, disorders of glycoprotein metabolism, mucolipidoses, aspartylglucosaminuria, fucosidosis, mannosidoses, and sialidosis type II.
Type:
Application
Filed:
November 22, 2011
Publication date:
September 5, 2013
Applicant:
ORPHAZYME APS
Inventors:
Thomas Kirkegaard Jensen, Anders Morkeberg Hinsby