For more than a decade, Scripps Research chemist Matthew Disney, PhD, has been inventing new approaches to treat incurable diseases such as ALS, Parkinson’s, cancers and now, COVID-19.
Ribonucleic acid (RNA) is a molecule essential in the regulation and expression of genes. Disney has long been focused on RNA-targeted drug development—one of the hottest fields in the pharmaceutical industry. Disney invented a system for finding rare structures of RNA stable enough to bind with targeted medicines. He then built a database of complementary compounds and a suite of chemical “side cars” that eliminate disease-causing segments. He’s using this approach against COVID-19, as well as Parkinson’s and other genetic diseases. As a result, the National Institutes of Health recently gave Disney a rare Blueprint Award for his Parkinson’s research.
At Disney’s spinoff company in Jupiter—Expansion Therapeutics—he’s advancing treatments for familial ALS, muscular dystrophy, and other diseases. “There is great potential for drugging RNA in all disease settings,” Disney says.
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