Sonia Vallabh, PhD
Eric Minikel, PhD
The Broad Institute

Talk title: Advancing oligonucleotide therapeutics for prion disease

Sonia Vallabh and Eric Minikel are a wife-husband team who co-run a prion disease research laboratory at the Broad Institute of MIT and Harvard. Their work is driven by a deeply personal mission: in 2010, Sonia watched her 52-year-old mother die of a rapid, mysterious neurodegenerative illness that was later identified as genetic prion disease. When Sonia learned in 2011 that she had inherited the causal mutation—placing her at greater than 90% lifetime risk of developing the same incurable disease—she and Eric made the decision to leave their previous careers and retrain as scientists. With no prior biology training, they earned their PhDs in Biological and Biomedical Sciences from Harvard Medical School in 2019 and now lead a 16-person lab together.

At the Broad Institute, their research spans the full arc of prion disease drug development, from therapeutic discovery and preclinical development to clinical biomarkers and natural history studies. Sonia’s work focuses on lowering prion protein levels in the brain as a treatment strategy, as well as developing the biomarker and natural history tools needed to enable meaningful clinical trials, including in presymptomatic people at risk. Their work helped give rise to Phase 1 clinical trials for ION717 and divalent siRNA for prion disease.