Orphazyme A/S (Nasdaq Copenhagen: ORPHA.CO) (Copenhagen, Denmark) is a biopharmaceutical company with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases.
The company develops new therapies that mobilize a group of stress-response proteins, called heat-shock proteins. Heat-shock proteins help restore balance to cells affected by stress arising from misfolded proteins, protein aggregates, and lysosomal dysfunction.
The lead compound, Arimoclomol, is in clinical development in Niemann-Pick Type C, ALS, sIBM and Gaucher.
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