Liposome Formulated siRNA View image detail

From Direct to Systemic RNAi delivery

Many of the current RNAi therapeutics development programs employ direct delivery strategies whereby the siRNA is applied directly at the site of disease. This increases the likelihood that the siRNA is taken up before it can either be degraded or secreted from the body, in addition to limiting unnecessary systemic drug exposure.

The eye and the lung are two organs which are amenable to such approaches since appropriate direct delivery technologies have been well established. As a result, siRNAs delivered by inhalation with a nebulizer or intraocular injection are under study for treating conditions such as respiratory syncytial viral (RSV) infection and age-related macular degeneration (AMD), respectively.

While the number of possible indications that can potentially be addressed with current direct delivery capabilities is significant, the successful application of systemic routes of administration such as intravenous or subcutaneous injection would open up RNAi for a vast number of additional diseases representing significant unmet medical needs.