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  • Kaitlyn Choi

Drugging RNA

Another research has identified long non-coding RNAs (lnc RNAs) that are involved in cancer--TROLL-2 and TROLL-3--adding them to a growing list of lnc RNAs that can be targeted for cancer diagnostic and treatment.


Doing some research on drugging RNA, I realized that my knowledge has been limited to antisense oligonucleotides (ASO) and siRNAs. These molecules have sequences complimentary to the target RNAs and some of them have been approved by FDA. What's on the rise in the RNA therapeutics is small molecules. Unlike ASO, small molecules can be orally delivered and target multiple organs. A month ago, FDA approved Roche and PTC Therapeutic's risdiplam, a small molecule that modifies splicing to treat spinal muscular atrophy. A recent paper from Arrakis Tx describes its new tool that could identify where in RNAs a small molecule binds and then provide the information about its selectivity and the structural model of small molecule bound to the target RNA, which helps develop new RNA targeting molecules.


I was drawn to RNA therapeutics just because RNA is my favorite biological molecule (if I can have one). Following this field--either the science or the business/investment side of it--won't bore me for sure.

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