A biomedical research laboratory based at the Broad Institute, we integrate several approaches in pursuit of one overarching mission: to prevent and treat prion disease in our lifetime.

                       
Mission
Founded by a wife-husband team motivated by a personal genetic diagnosis, our singular goal is to make prion disease a preventable and treatable condition, in our lifetime.
Therapeutics
We work to validate therapeutic hypotheses, discover drug compounds, and understand mechanisms of action through biophysical and biochemical approaches and preclinical models.
Biomarkers
We develop molecular readouts to detect drug activity and disease processes in living humans, as tools to understand disease processes and to enable clinical trials.
Genomics
We use human genomic data to gain in vivo, whole organism insights about the biology of the organism we care most about.
Mechanisms
We build systems, models, and assays to help elucidate how disease works on a molecular level, and inspire the next generation of therapies.

Our clinical trial of divalent siRNA for prion disease is now recruiting. See details: NCT07444580.

We are committed to making drug development regulatory documents publicly available as a service to the rare disease community:

Our story has been told in WIRED, The New York Times, Scientific American, NPR Morning Edition & All Things Considered, The Boston Globe, The Atlantic, and The New Yorker.

We also run a 501(c)(3) non-profit organization, Prion Alliance, through which you can donate to support our work. We co-run a natural history study of biomarkers in people at genetic risk for prion disease at MGH, which is now recruiting, as well as an online registry, PrionRegistry.org, through which you can volunteer to participate in prion disease research. Eric blogs at CureFFI.org. We are affiliated with Broad’s Program in Brain Health, the MGH McCance Center for Brain Health, and the Department of Neurology at MGH.