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.