Overcoming Barriers: Clinical Translation of siRNA Nanomedicines

Abstract RNA interference has garnered new hope for many difficult to treat diseases that have been “undruggable” using conventional small molecule drugs or monoclonal antibodies. Small interfering RNA (siRNA) has shown great strides in targeting difficult to treat diseases as siRNA therapeutics are able to efficiently silence specific genes throughout the body. This review highlights major barriers in siRNA delivery and how nanoparticles have been used to circumvent these barriers. The authors outline many of the recent promising preclinical successes in siRNA delivery using nanoparticles. Importantly, the review examines current FDA‐approved siRNA therapeutics and provides a comprehensive analysis of current siRNA candidates in clinical trials. Lastly, the authors discuss future challenges and opportunities in this expansive field of research.