Vibhor Mishra, Ph.D.

Small interfering RNA (siRNA) therapeutics have shown tremendous promise in targeting diseases with poor prognoses, transforming the pharmaceutical landscape. They have allowed a paradigm shift from a conventional inhibitor-based approach to RNA-induced targeted gene silencing. Rational siRNA design and delivery methods have significantly improved their stability, limited immune activation, and increased target affinity resulting in an influx of siRNA-based clinical trials. The U.S. Food and Drug Administration (FDA) has since approved three therapeutic siRNA drugs, ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO® (lumasiran) developed and marketed by Alnylam® Pharmaceuticals. In addition, several other drugs are in the late stages of clinical trials.