Our Story

At Orphazyme we bring together world-class expertise and the latest research to develop treatments for protein misfolding diseases, including lysosomal storage diseases with neurological pathologies, and neurodegenerative disorders. These debilitating conditions are often rare and many have no existing treatments.

Orphazyme was founded in 2009 in Copenhagen, Denmark.  Our work is focused on the cell-protective properties of the heat shock response. This natural defence mechanism can be stimulated to defend cells from a toxic accumulation of misfolded proteins or other waste products.

Degenerative diseases progressively deprive patients of opportunities, and in many cases, result in premature death. Orphazyme is developing technology with the potential to dramatically improve the lives of those suffering from a range of degenerative diseases which currently don’t have satisfactory treatments. Our lead therapy in development stimulates a heat shock protein response in cells experiencing stress. Heat shock proteins can stabilise and chaperone misfolded proteins inside the cells and stabilise lysosomes inside cells, alleviating the cellular stress.

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 diseases, including Niemann-Pick type C, Gaucher’s disease, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS).

 

CORE VALUES

At Orphazyme we:

Care for people with rare diseases, and with diseases of a high unmet need

  Have a vision to delay or stop disease progression and make a dramatic improvement in the lives of patients

graphic-about-icons-03.png

Advance world-class knowledge to build
on our expertise in heat shock protein response and lysosomal science

 
Work hard to conduct the most thorough, diligent research and bring results to the lives of patients as quickly and safely as possible  

  Work tirelessly to conduct the most thorough, diligent research and bring results to the lives of patients as quickly and safely as possible

graphic-about-icons-05.png

Build powerful relationships with everyone connected to the diseases we are treating,
to learn from the wider community