Towards a divalent siRNA therapy for prion disease: an academic, patient-scientist led effort

Monday, September 8, 2025 at 12:30 pm US EDT

Prion disease is a rapidly fatal neurodegenerative disease with no current standard or care. Since 2019 we have been collaborating with Anastasia Khvorova's lab at UMass to identify and develop a divalent siRNA molecule capable of lowering prion protein across the human brain, as a treatment and preventive therapy. Leveraging a combination of NIH and philanthropic funds, we have identified a lead candidate, completed GLP toxicology and GMP manufacturing, and filed an IND in Feb 2025 that received partial clearance in March. This talk will cover regulatory feedback received, current challenges, clinical plan, and next steps for this program, in the hopes our experience may in some way prove useful to other low N academic drug development efforts.

Sonia Vallabh co-runs a prion research laboratory at the Broad Institute of MIT and Harvard along with her husband, Eric Minikel. She earned her PhD in Biological and Biomedical Sciences from Harvard Medical School in 2019. Sonia and Eric left their previous careers to devote their lives to biomedical research after learning in 2011 that Sonia had inherited from her mother a mutation that causes genetic prion disease, a rapidly fatal and currently untreatable neurodegenerative disease that typically strikes in midlife. At the Broad Institute, Sonia works on prion disease drug discovery, with a focus on lowering the amount of prion protein in the brain. She also works on biomarkers, natural history and other tools to enable meaningful clinical trials, including in presymptomatic people at risk.

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