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Nina Oberbeck, PhD, Senior Director of Translational Sciences

Grant Title: Assessing efficacy of a PrP-lowering Molecular Gate as a therapeutic for prion disease

Location: Gate Bioscience

Grant Year: 2025
Gate Bioscience is a biotechnology company developing a new class of drugs called Molecular Gates designed to eliminate harmful proteins, like prion protein, before they can cause disease. Molecular Gates have a precise mechanism of action: they selectively block the export of a harmful protein from cells, causing the protein to be degraded instead of secreted. Molecular Gates are small molecule therapeutics, which means they are taken as a pill instead of injected.

Many studies show that reducing prion protein levels in the brain could be an effective way to treat prion disease. Gate Bioscience is developing a Molecular Gate drug to eliminate prion protein from the brain. By reducing the levels of prion protein, the drug eliminates the substrate required for prion multiplication and spreading, thus stopping the disease in its tracks.

Early lab and animal studies have shown promising results: in mice, a lead compound successfully lowered prion protein levels in the brain by 80% after only a week of once daily dosing. With the support of this CJDF grant and in collaboration with the Vallabh Minikel lab at the Broad Institute, Gate Bioscience will now test whether one of their lead compounds can extend survival in a mouse model of prion disease.

If successful, these data will be pivotal in the race to develop a safe and effective prion protein lowering drug that patients and at-risk individuals can pick up from the pharmacy, place in their medicine cabinet, and take from the comfort of their own home to prevent, slow, or reverse this terrible disease.

About the Researcher:

Nina Oberbeck, PhD, is the Senior Director of Translational Sciences at Gate Bioscience, where she leads the Prion program. Gate Bioscience have been collaborating with the Vallabh Minikel Lab since 2021.

Dr Oberbeck earned her Ph.D. in Molecular Biology from the MRC Laboratory of Molecular Biology, University of Cambridge, where she investigated DNA repair mechanisms under the mentorship of Dr. K.J. Patel, FRS FMedSci. She also holds an MA (Hons) and MSci in Natural Sciences from Magdalene College, University of Cambridge. She completed her postdoctoral training at Genentech Inc. under Dr. Vishva Dixit, focusing on the signaling pathways that regulate epidermal stem cell differentiation. She has authored several first-author publications in leading scientific journals, including Nature and Molecular Cell.

Dr. Oberbeck has presented Gate’s work on prion disease internationally at the Prion 2024 meeting in Nanchang, China, and the 14 th Annual National CJD Conference in Melbourne, Australia, in 2023.