While RNAi drug developers continue to pursue major indications such as cancer and hepatitis C, a number of these companies are looking to niche diseases as good opportunities to apply the gene-silencing technology.

Notably, this trend aligns with big pharma’s increasing interest in orphan and ultra-rare diseases, keeping RNAi therapeutics an attractive option for pharmaceutical firms despite the high-profile suspension of in-house RNA drug programs by Roche and Pfizer in late 2010 and early 2011, respectively.

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