Discovery of direct DUX4 inhibitors using AI-guided covalent chemistry and high-scale proteomics

Investigator: Talus Bioscience

Category: Research - Basic

Talus Bio’s MARMOT platform uses next-generation proteomics to discover drugs for proteins that regulate our genome, including transcription factors such as DUX4. In this project, we will leverage an AI algorithm we developed for predicting transcription factor modulators to create a library of candidate DUX4 inhibitors, which we will then test in human muscle cells expressing DUX4. We will generate multiple lines of experimental evidence to confirm that inhibitors we discover bind directly to the DUX4 protein and prevent it from activating the genes that it regulates, including in muscle cels derived from individuals with FSHD. Crucialy, we believe the data we produce will be highly attractive to potential pharmaceutical companies who can partner with Talus Bio to develop these smal-molecule inhibitors into FSHD therapies.