Sonia Vallabh, Ph.D.

Sonia Vallabh co-leads the initiative to develop preventive drugs for prion disease at the Broad Institute of MIT and Harvard. She comes to this work with a personal mission. In 2010, Vallabh watched her 52-year-old mother die of a rapid, mysterious, undiagnosed dementia. One year later, Vallabh learned that her mother’s disease had been genetic prion disease, and that she herself was at risk. Vallabh underwent predictive genetic testing and learned that she had inherited the causal mutation, placing her at very high risk of developing the same disease. There was no prevention, treatment, or cure available. Vallabh quit her previous career in law and consulting, and, together with her husband, Eric Minikel, re-trained as a scientist in order to devote her life to finding a way to stop her disease before it starts.

At the Broad, Vallabh is focused on developing preventive drugs for prion disease. Lowering of prion protein (PrP) is a genetically well-validated strategy for delaying the onset of prion disease, and it lends itself to measurement of PrP as a biomarker available before the disease process has begun. Vallabh is working on the discovery and preclinical development of PrP-lowering drugs, including editing, epi-editing, and oligonucleotide strategies; credentialing of PrP in cerebrospinal fluid as a pharmacodynamic biomarker for such drugs; and establishment of a clinical pathway for preventive drug development. In April 2026, a clinical trial called PrP-targeting siRNA Safety & Mechanism Study (PRiSM; NCT07444580), began enrolling participants to evaluate the safety and tolerability of a new siRNA drug candidate developed by Vallabh’s lab in collaboration with University of Massachusetts Chan Medical School. Read Vallabh and Minikel’s blog to see the latest updates on their efforts to move new treatments into clinical testing. Vallabh also co-leads a natural history study at Massachusetts General Hospital devoted to the assessment of fluid biomarkers in individuals at risk for genetic prion disease.

Vallabh holds a B.A. from Swarthmore College, a J.D. from Harvard Law School, and a Ph.D. in biological and biomedical sciences from Harvard University. She trained in Stuart Schreiber’s laboratory at the Broad. Her story has been told in The New Yorker, NPR, and WIRED and in her TED talk entitled, “My quest to cure prion disease – before it’s too late.”

Areas of expertise: prion disease; drug development; biomarkers; rare disease; patient-focused research