Talking Biotech with Dr. Kevin Folta Targeting RNA with Therapeutics - Dr. Matthew Disney
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Oct 5, 2025 Dr. Matthew Disney, an expert in small-molecule therapeutics at the UF Scripps Institute, dives into the revolutionary potential of RNA as a therapeutic target. He explores its pathogenic roles in diseases like ALS and myotonic dystrophy, and discusses innovative strategies to design small molecules that target structured RNA. Disney also highlights how RIBOTACs can recruit RNA-degrading enzymes, and shares insights on targeting viral genomes, including strategies for inhibiting SARS-CoV-2, showcasing the promising future of RNA therapeutics in combating various diseases.
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Use RIBOTACs To Trigger RNA Degradation
- Create small molecules that both bind RNA and recruit endogenous ribonucleases to degrade the target RNA.
- Use ribonuclease targeting chimeras (RIBOTACs) to combine binding specificity with catalytic RNA clearance.
Binding Can Reprogram RNA Processing
- Small-molecule RNA binders can also alter RNA processing and splicing to shift proteins from toxic to non-toxic forms.
- Binding can trigger exon exclusion or decay pathways without requiring catalytic cleavage by the drug itself.
Measure Targets And Effects In Cells
- Map actual cellular targets using covalent cross-linking in live cells to detect off-target binding.
- Pair target engagement data with RNA-seq and proteomics to iteratively design more specific compounds.