is a Lead Researcher at St. Jude Children’s Research Hospital. Vibhor is a published author, an associate faculty member for Faculty Opinions, and an ad-hoc reviewer for several scientific journals. Vibhor’s research focuses on investigating novel drug targets involved in childhood leukemia using targeted protein degradation platforms.
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.