Orphazyme's proprietary approach utilizes the lysosome-protective and stabilizing effect of Heat Shock Proteins (HSPs) as a platform for the development of novel therapies for lysosome-related diseases.
The lead program, Orph-001, is a recombinant version of human HSP70 being developed for the treatment of lysosomal storage diseases. HSP70 has been shown to be effective in correcting conditions of lysosomal deficiency and/or stress. The initial studies were published in Nature, January 2010, by Orphazyme's scientific founders (read more). Orph-001 is currently being tested in vivo in animal models for lysosomal storage diseases in collaboration with world class academic laboratories.
The lead program, Orph-001, is a recombinant version of human HSP70 being developed for the treatment of lysosomal storage diseases. HSP70 has been shown to be effective in correcting conditions of lysosomal deficiency and/or stress. The initial studies were published in Nature, January 2010, by Orphazyme's scientific founders (read more). Orph-001 is currently being tested in vivo in animal models for lysosomal storage diseases in collaboration with world class academic laboratories.