Advancing an intrathecal divalent siRNA for prion disease as an investigator-initiated program

Eric Minikel co-leads the initiative to develop preventive drugs for prion disease at the Broad Institute of MIT and Harvard. In prion disease, his research spans therapeutic discovery, preclinical development, and clinical biomarker and natural history research. He also studies the genetics of prion and other neurodegenerative diseases, and the application of human genomic datasets to disease epidemiology and drug target validation. His blog, CureFFI.org, covers topics ranging from prion biology to genomics to drug development.