Matt Disney: Drugging RNA – Pioneering a New Field in Drug Discovery
Back of the Napkin - Inspiring Stories from Biotech Pioneers
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Targeting RNA with Small Molecules
In this chapter, they discuss how companies are now targeting RNA with small molecules, inspired by a talk by the guest in 2015. They also talk about the concept of ribotax, a heterobifunctional compound that binds to an RNA and a nuclease to cleave the RNA target. They explain how they view ribotax as another layer of controlling RNA function by binding structure, similar to anti-sense oligos.
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