Anastasia Khvorova, Ph.D.

At-Large Director, Scientific & Research Council Chair

For 20 years, I have focused my research efforts on RNA chemical biology and oligonucleotide technology development. I joined the RNA Therapeutics Institute (RTI) at the University of Massachusetts Medical School (UMMS) after a decade of working on different aspects of RNA chemistry, biochemistry, bioinformatics, delivery, and therapeutic development in industry. My diverse expertise in both chemistry and cell biology allowed me to establish a lab that brings together hard-core organic chemists and RNA biologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. I also have established the Nucleic Acid Chemistry Center at the RTI and am now providing access to RNA chemistry expertise to multiple labs within and outside of UMMS.

My lab focuses on chemical engineering of therapeutic RNAs. We have established that full chemical stabilization is essential for conjugate-mediated delivery of oligonucleotides in vivo. We have developed siRNA scaffolds compatible with RISC assembly and supporting productive conjugate-mediated delivery. Recently we engineered lipid conjugates that expand the functional utility of RNAi to multiple tissues, including liver, kidney, spleen, lung, heart, fat, muscle, placenta, and most importantly, central nervous system. With chemistry advances, we have learned how to therapeutically modulate gene expression for periods exceeding six months following a single administration. My industrial tenure gives me additional background and understanding of what it takes to develop RNA products and therapeutics.


ASGCT Policy Summit

September 23-25, 2020