We are a chemical biology lab dedicated to developing RNA-based therapeutics. Our progress stems from asking the right research questions—those rooted in a deep understanding of the biochemical and pharmacological pathways that define and limit the potential of RNA as a drug.
Our core mission is to unite informaticians, chemists, biochemists, cell biologists, and pharmacologists in collaborative research. This cross-disciplinary approach has enabled transformative advances in RNA therapeutics—from siRNA informatics and insights into RISC (RNA-induced silencing complex) biology to the development of pharmacological principles and two new classes of therapeutic siRNAs.
These novel siRNA classes facilitate delivery to the central nervous system (CNS) and extrahepatic tissues, contributing to three clinical-stage drugs and numerous additional candidates in development.
