Small molecules are drugs of low molecular weight that can often be taken by mouth. For prion disease the aim is to lower the amount of normal prion protein, or to stop it from being converted. This section covers Molecular Gates, the Trento PPI-FIT program and related entries. Related work on gene-based approaches is in gene and epigenetic editing.
A Molecular Gate is a pill-form drug from Gate Bioscience that blocks a protein from leaving the cell so that the cell breaks it down instead. A 2025 CJD Foundation grant funds a test of whether a lead compound extends survival in prion-infected mice, in work with the Broad Institute. The company’s prion program is described as preclinical and not yet ready for clinical trials. A group in Trento, linked to CIBIO and Emiliano Biasini, reported in 2021 compounds that lower prion protein by stabilising a folding intermediate. That work used cells only, and animal testing is stated as a future step.