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What's New in RNAi Therapeutics?

February 11, 2014
At the 16th Annual BIO CEO & Investor Conference​, Monday morning’s therapeutic workshop, “Delivering the (RNAi) Goods,” featured a panel discussion moderated by Thomas Wei, senior research analyst at Jeffries LLC. The panel gave investors a peek into the novel approaches utilized by four leading companies in this area: Dicerna Pharmaceuticals, Arrowhead Research Corporation, Alnylam Pharmaceuticals, and Regulus Therapeutics. RNAi therapeutics, which include gene silencing, exon skipping, and mircroRNA therapy, are a promising area in the biotech industry, and have seen a renewed interest among investors in recent years.

While the panelists’ companies have been involved in RNA therapeutics for about a decade, the viability of the technology has long been questioned by investors. The panelists described how they saw parallels between the development of the RNA therapeutics segment and the early days of monoclonal antibodies, which one panelist could remember was greeted with similar skepticism back in 2001 because of its’ various technical issues. The panelists credited ISIS Therapeutics for paving the way for other RNA therapeutics; it wasn’t easy for ISIS, but their product Kynamro has finally received FDA approval. The panelists said we are now in a “belief phase” for RNA therapeutics.

According to the panelists, r​egulators tend to like RNA therapeutics because of their precision, and their ability to target conditions that were previously considered “undruggable targets,” such as TTR Amyloidosis and hemophilia under certain conditions. While delivery continues to be a barrier to entry for many biotechs, more companies are exploring RNAi therapeutics as investor interest grows.