Our Approach

At Orphazyme our work is focused on the cell-protective properties of the heat shock response, a natural defence mechanism in all our cells. The heat shock response protects cells from an accumulation of misfolded proteins or other waste products, which would otherwise lead to toxicity and disease. The heat shock response is generated through the production of heat shock proteins, which act as the cells’ lifeguards.

Today, Orphazyme’s technology is based around a drug called arimoclomol, which stimulates an increased production of heat shock proteins in cells experiencing stress or toxicity.

When we began our company, our focus was on making a positive difference for patients with rare diseases with a high unmet need. In line with this, today we are working to create new therapeutic approaches for a number of different rare diseases, including Niemann-Pick type C, Gaucher’s disease, sporadic Inclusion Body Myositis (sIBM) and SOD1-associated Amyotrophic Lateral Sclerosis (SOD1-ALS). Our aim is to slow or even prevent disease progression and we believe our approach has the potential to dramatically improve the lives of those suffering from a range of degenerative diseases which currently don’t have satisfactory treatments.