Since the 1990s scientists across the world have begun to harness the power of RNA interference (RNAi) to silence genes in a multitude of diseases. However, many challenges face those aiming to develop RNAi-based therapies including targeted delivery, the design and efficacy of gene silencing agents and the minimization of off-target and immunogenic effects. Today there are over 100 RNAi-based projects under investigation to treat autoimmune and inflammatory diseases, cancer, cardiovascular disease, CNS disorders, metabolic, ocular conditions, and viral infectious diseases. The majority of these projects are in the earliest discovery stages (36%) or in preclinical studies (54%). Of the projects that have reached clinical trials, only a handful have passed initial Phase 1/2 testing, with a total of three being investigated in Phase 2 studies. In 1H09 two late-stage ocular RNAi-based therapies, Opko Health's bevasiranib and Allergan's AGN-745, were terminated in late-stage clinical trials after the drugs failed to meet a key efficacy endpoint. This is a major set back for market and raises questions regarding the potential of unformulated siRNAs as therapeutics. Despite these challenges and others, such as the complexities of regulation and intellectual property, the field retains enormous potential to diagnose and treat diseases with high unmet medical need. Specialists and academics are working hard to find ways around these hurdles in order to deliver a new generation of RNAi-based therapeutics and diagnostics. The market for RNAi-based therapeutics is forecast to grow from 2013 onwards, as the first products enter the marketplace, to generate sales in excess of US$2.9 billion by 2020. The first siRNA based therapeutics will capitalize on the demand to treat viral infections and ocular conditions and in the longer term companies will be able to target niche areas of high unmet clinical need such as cancer, cardiovascular disease, metabolic disorders, inflammatory and neurological conditions.

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