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Sean W. Clark, PhD

Principal Investigator at Amicus Therapeutics

Location: Cranbury, NJ United States

Dr. Clark is the principal investigator of the Research Biology Department at Amicus Therapeutics. Dr. Clark received both his BS and PhD from UCLA. In the decade prior to Amicus, he was a visiting research fellow and research scientist at Princeton University.

Since 2005, Dr. Clark has led Amicus’ investigation of the biochemical link between Gaucher disease and Parkinson’s disease. He has utilized cell and pre-clinical models that recapitulate changes in GCase enzyme activity (Gaucher disease) and the accumulation of, or tolerance to, alpha-synuclein (Parkinson’s disease). Amicus Therapeutics’ platform technology is the Pharmacological Chaperone, which represents a new way to increase cellular enzyme activity. Under an award from the MJFF Therapeutics Development Initiative, Dr. Clark demonstrated that the in vivo enhancement of GCase enzyme activity with a Pharmacological Chaperone was able to reduce the accumulation of alpha-synuclein in a pre-clinical model over expressing alpha-synuclein.

Associated Grants

  • Use of the Orally Administered Pharmacological Chaperone AT3375 to Reduce Alpha-synuclein Levels in the Brain of Thy-1 Alpha-synuclein Small Models


  • Treatment of Parkinson’s Disease with AT2101, a compound that increases the activity of endogenous Glucocerebrosidase


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