Nearly all drugs work by attaching themselves to proteins. But not every protein can be touched with a drug. For over a decade, Scripps Research Professor Matthew Disney, PhD, has focused exclusively on developing approaches to target RNA with small molecules, rather than the usual protein targets. In his Front Row Lecture, Disney described how he developed a computational approach to identifying druggable RNAs, and how he built small molecules to target them via genetic sequence-based design. His platform technologies for healing disease through a focus on altering RNA have opened new potential disease-modifying precision therapies in areas of unmet medical need. These include cancer, neurodegenerative diseases such as ALS, muscular dystrophy, viral diseases and much more.

Presented: October 17, 2019