Novel siRNA backbone enhances stability, durability of oligonucleotide therapeutic platform

A biochemical breakthrough using simple carbon atoms, by Ken Yamada, Ph.D., and Anastasia Khvorova, Ph.D., has dramatically improved the stability and efficacy of a potential oligonucleotide therapeutic platform in mice. This discovery, published in Nature Biotechnology, has the potential to improve the durability of oligonucleotide drugs, including small interfering RNAs, and to bring these therapies to cell types outside of the liver for the first time.