Clinical development of synthetic siRNA therapeutics

In 2001 the Tuschl laboratory reported that synthetic siRNAs could be applied safely to mammalian cells to silence gene expression while avoiding induction of local and systemic innate immune responses to double-stranded RNA. Today there are 19 synthetic siRNAs in clinical studies in 14 different disease indications. Clinical activity has been demonstrated in liver, eye, lung, kidney, skin and solid tumors. Synthetic oligonucleotides, including siRNAs, are poised to become the next therapeutic platform in the pharmaceutical industry.