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.
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.