Most of the more than 7000 rare diseases described thus far are still orphan

DORPHAN S.A. is a preclinical stage drug development company focusing on targeted therapies for the treatment of rare and orphan diseases

Our strategy is focused on the development of treatments for lysosomal storage diseases, through therapeutic approaches by small molecule-based pharmacological therapies.


Dorphan S.A. was founded in 2011 by the members of the Swiss Sanfilippo Foundation in order to discover and pursue the development of drug candidates for orphan and rare genetic diseases. The company R&D activities are built on worldwide innovative research to identify, evaluate and develop preclinical molecules and deliver promising drug candidates to patients.