Targeting RNA

Chemist Matthew Disney is leading a revolution in drug discovery

In June 2000, when President Bill Clinton announced that the initial sequencing of the human genome had been completed, it was hailed as a milestone in medicine, promising cures for everything from Alzheimer’s and Parkinson’s to diabetes and cancer.

But Matt Disney had questions.

The teams sequencing the human genome had focused on DNA, counting only about 30,000 protein-coding genes, about the same number as a mustard plant or a worm. What explained human complexity and diversity if humans had similar numbers of genes to plants and invertebrates?

At the time, Disney was working on his Ph.D. at the University of Rochester, focusing primarily on ribonucleic acid, or RNA, which was thought — and has now been shown — to make up 90% of the genome.

Disney thought the answers to his questions about the human genome project, and its real potential, might lie in the RNA. But in 2000, RNA’s structure and functions were still something of a black box, and the idea of making medicines targeted to RNA was rejected as unfeasible, too risky or both.

The gaps in knowledge enticed Disney, who saw translating genetics into treatments as a worthy challenge.

“I thought, well, if we could really turn the human genome and the RNAs that they make into new targets for medicines, that would be transformative.”

Two decades later, Disney and his team at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology in Jupiter, Florida, have discovered more than 200 unique RNA-targeting compounds. His methods and discoveries have changed minds, igniting a global race to treat incurable diseases via their RNA.

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