Orphazyme A/S

Established in 2009 and headquartered in Copenhagen, Denmark, Orphazyme A/S is a biopharmaceutical company focused on developing treatments for rare neurodegenerative disorders. The company's innovative strategy centers on deploying heat shock proteins to activate the cell's natural heat shock response. This crucial cellular mechanism offers protection by preventing the accumulation of misfolded or aggregated proteins and mitigating the impact of dysfunctional lysosomes. Orphazyme's leading drug candidate, Arimoclomol, is currently progressing through clinical development for conditions such as Niemann-Pick disease type C, Amyotrophic Lateral Sclerosis (ALS), and Inclusion Body Myositis (IBM).