Tulika Rastogi

By now, news of MIT and Harvard’s Broad Institute’s victory over the CVC group (the University of California-Berkeley, the University of Vienna, and Emmanuelle Charpentier) in a dispute over genome-editing CRISPR technology has spread like wildfire in the biotech industry and academia. To recap, the Patent Trial and Appeal Board (PTAB) of the United States Patent and Trademark Office (USPTO) ruled that the Broad Institute (Broad) was the first to invent single-guide CRISPR-Cas9 gene-editing technology for use in eukaryotes. Furthermore, the judges ruled that “CVC fails to provide sufficient, persuasive evidence of an earlier reduction to practice or conception, as they are legally defined, of each and every element of Count 1 before Broad’s evidence of reduction to practice.” While the decade-long patent battle over the Nobel Prize-winning technology might have come to an end with Broad breathing a sigh of relief and CVC contemplating whether to take it to the next level, this has left some biotech companies scrambling to renegotiate their licensing agreements.

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.