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).
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
Advance world-class knowledge to build
on our expertise in heat shock protein response and lysosomal science
Work tirelessly to conduct the most thorough, diligent research and bring results to the lives of patients as quickly and safely as possible
Build powerful relationships with everyone connected to the diseases we are treating,
to learn from the wider community
At Orphazyme we are dedicated to the development of paradigm-changing medicines to improve the lives of patients affected by diseases of protein misfolding. We are harnessing the power of heat shock protein therapy, and strive to bring together our expert knowledge and world-class meticulous research, to delay or halt disease progression and improve patient lives. We are expediting our patients’ access to treatment by designing smart clinical trials.