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About the Seminar
"A Structure-based Approach to Drugging RNA with Small Molecules"
RNA structures play a central role in the complex regulatory network that governs RNA function, particularly splicing and translation. These structures may serve as compelling therapeutic targets for the interdiction of RNA function. We are pursuing small molecules that penetrate cell membranes and bind to specific mRNAs. This requires that we identify ligandable, functionally critical RNA sub-structures and druglike small molecules that bind to those structures selectively, thereby modulating their function. This presentation will focus on some of the unique challenges of drugging RNA and offer some solutions to those challenges. Specifically, we have identified small molecules that bind repeat expansion domains in the DMPK gene of patients that suffer from myotonic dystrophy type 1. In addition to introducing the platform, I will describe binding mode, protein displacement, impact on spliceopathies, and in vivo efficacy.
About the Speaker
Dr. Jennifer Petter is the Founder and Chief Scientific Officer of Arrakis Therapeutics. Previously she was Vice President of Chemistry at Celgene, Vice President of Drug Discovery at Avila Therapeutics, Vice President of Research at Mersana Therapeutics, Director of Small Molecule Drug Discovery at Biogen, Section Head in Oncology Chemistry at Sandoz/Novartis, and Assistant Professor of Chemistry at the University of Pittsburgh. Dr. Petter graduated from Dartmouth College with an AB in chemistry, earned her PhD in organic chemistry at Duke University with Ned Porter, and was a post-doctoral fellow in Ron Breslow’s group at Columbia University. She has ushered more than a dozen compounds into the clinic for the treatment of cancer, cardiovascular disease, autoimmune disorders, and sepsis.