Human RNA is an important drug target, but the purposeful targeting of it with small molecules has historically been a challenge.  Inforna, a sequence-based design approach, has delivered compounds that affect the biology of RNA targets from cells to pre-clinical animal models and in multiple disease settings.  In addition to Inforna companion methods to validate the RNA targets of small molecules in cells and animals have also been developed as have frameworks to study how compounds affect phenotype.  Herein, we describe the design of compounds that bind RNA targets with the ability to cleave them substoichiomterically in both cellular and animal models and we apply these approaches across indications.  Design can thus deliver compounds that bind RNA structure and can also affect cleavage of these structures against many targets against many settings.

Small-ish molecule antisense

Macromolecular Chemistry: The Second Century

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ACS Spring 2020 National Meeting & Expo

The official archive and repository for all ACS Meetings & Expositions abstracts and select posters or presentations.