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Funded Studies

Daniel S. Ory, MD

Senior VP of Translational Medicine at Casma Therapeutics

Location: Cambridge, MA United States

Dr. Daniel Ory graduated from Harvard College and Harvard Medical School and received postdoctoral training at the Whitehead Institute at MIT. He joined the faculty at Washington University in 1995. Over the past two decades, his laboratory has made fundamental contributions to our understanding of cholesterol biology and Niemann-Pick disease type C (NPC), a rare neurodegenerative disease. These efforts resulted in discovery of new diagnostic NPC biomarkers. Clinical assays based on these biomarkers have become the worldwide standard for diagnosis of NPC and have led to development of a newborn screen. Working closely with the National Institutes of Health, Dr. Ory led development of the Phase 1/2a trial of intrathecal cyclodextrin and was instrumental in the recruitment of a pharmaceutical partner to conduct the international, multisite Phase 3 trial. In 2018, Dr. Ory joined Casma Therapeutics to accelerate translation of autophagy insights into new treatments for patients with rare genetic and neurodegenerative diseases.

Associated Grants

  • Development of a Novel TRPML1 Activator for Parkinson’s Disease


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