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MARKET
RNA interference (RNAi) drugs are of significant commercial interest to the pharmaceutical industry because unlike small molecule drugs, RNAi therapeutics are highly specific, potent, and straightforward to design, and can be used toward virtually any molecular target. The problem, however, is that mammalian organisms efficiently protect themselves from invasion by large, charged molecules such as RNAi molecules, through a series of biological barriers.
Consequently, administered RNAi drugs are substantially hindered from circulating in the bloodstream, crossing the vascular endothelium, penetrating extracellular matrix/tissue, crossing the membrane of the target cell, and traversing the cell’s interior to safely reach their molecular target. RNAi delivery companies and researchers have attempted to overcome one or more of these barriers with various formulations and RNAi-modifications over the past decade, but a comprehensive strategy has remained elusive.
It is therefore a generally accepted proposition that the key to unlocking the significant commercial potential of RNAi therapeutics is development of delivery technology that overcomes each of the body’s innate barriers in a format that is clinically relevant (i.e., dosage levels, methods of administration, and shelf stability) and scalable.
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