12:00 AM
Nov 07, 2011
 |  BioCentury  |  Emerging Company Profile

Orphazyme: One for all

Denmark's Orphazyme developing Hsp70 for lysosomal storage disorders

Drug development for lysosomal storage disorders has largely centered on enzyme replacement therapies, which yield one drug per disease. Orphazyme ApS believes its recombinant heat shock protein 70 (Hsp70) and small molecules that induce heat shock protein expression will be able to treat a variety of lysosomal storage disorders.

The company was founded based on work by co-founder and CSO Thomas Kirkegaard Jensen while he was a postdoctoral fellow at the Danish Cancer Society's Institute of Cancer Biology.

Jensen and colleagues showed in a January 2010Nature article that Hsp70 stabilized lysosomes in vitro in fibroblasts from Niemann-Pick disease patients and could thus reverse the pathology associated with the disease.

CEO Anders Morkeberg Hinsby believes Orphazyme's recombinant Hsp70, which the company calls Orph-001, could be applied to a panel of diseases.

Orphazyme has unpublished in vitrodata showing Orph-001 returned cellular lysosome levels to close to healthy levels in eight lysosomal diseases: Niemann-Pick types A, B and...

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