Orphazyme US, Inc.
Life Sciences Drug DevelopmentNewton, MA
Orphazyme US, Inc. is based in Newton, MA and is a wholly owned subsidiary of Orphazyme A/S headquartered in Copenhagen, Denmark. Orphazyme is biopharmaceutical company focused on the development of treatments for patients living with life-threatening and debilitating rare diseases. Our research is concentrated on heat shock response, a natural cell-protective rescue system against diseases caused by misfolded proteins and lysosomal dysfunction. The company’s lead candidate, arimoclomol, is in development for four severe orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and two lysosomal storage diseases, Niemann-Pick disease Type C (NP-C) and Gaucher disease.