ORPHY

Orphazyme A/S, a biopharmaceutical company, develops therapies for the treatment of neurodegenerative rare diseases. The company offers heat stock proteins that focuses on the treatment of neurodegenerative diseases by harnessing the cell protective properties of the heat shock response, which protects cells from the accumulation of misfolded proteins, aggregated proteins, and dysfunctional lysosomes. Its lead candidate is the Arimoclomol, which is in clinical development for diseases, including Niemann-Pick disease type C, Amyotrophic Lateral Sclerosis, and Inclusion Body Myositis.